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1.066zM136.15 39.2c.523-.133.813.067.755.6.29-.467.755-.667 1.22-.667.812 0 1.102.667.986 1.534L138.879 42c-.058.333-.058.467.29.4l-.058.4c-.58.133-.87 0-.755-.8l.233-1.333c.116-.667-.175-1.067-.697-1.067s-.987.4-1.103 1.067l-.406 2.066h-.523l.465-2.533c.116-.533.174-.733-.29-.667l.116-.333zM140.272 41.733c-.058.534.348.6.812.6.407 0 .93-.2.987-.6.058-.4-.232-.533-.754-.6-.523-.066-1.161-.266-1.161-.866 0-.8.754-1.2 1.509-1.2.638 0 1.219.333 1.103 1h-.465c.058-.4-.29-.534-.696-.534-.406 0-.929.2-.929.667 0 .267.29.4.755.467.638.066 1.277.266 1.103 1.133-.116.733-.813 1-1.568 1.067-.696 0-1.277-.267-1.16-1.067h.464v-.067z"></path></g> </g> </g> </g> </svg></a></div> </div> </div> <div id="top-utility" class="flex-center"> <div id="Header_TBB0D3FF1011_Col01" class="sf_colsIn" data-sf-element="Top Utility" data-placeholder-label="Top Utility"> <div > <div class="sfContentBlock sf-Long-text" ><a class="button back" 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class="row" data-sf-element="Row"> <div id="Main_C006_Col00" class="sf_colsIn col-md-12" data-sf-element="Column 1" data-placeholder-label="Column 1"> <script> window.mgConfexProgramConfig = window.mgConfexProgramConfig || {}; window.mgConfexProgramConfig.dateLinks = [{"url":"https://www.ispor.org/conferences-education/conferences/past-conferences/ispor-europe-2023/program/posters/poster-detail/euro2023-3784","activeClass":"active","title":"Poster Session 1","date":"Mon, 13 Nov"},{"url":"https://www.ispor.org/conferences-education/conferences/past-conferences/ispor-europe-2023/program/posters/poster-detail/euro2023-3785","activeClass":"","title":"Poster Session 2","date":"Mon, 13 Nov"},{"url":"https://www.ispor.org/conferences-education/conferences/past-conferences/ispor-europe-2023/program/posters/poster-detail/euro2023-3786","activeClass":"","title":"Poster Session 3","date":"Tue, 14 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Moorfields Biomedical Research Centre--Patient Public Involvement Representative, London, UK, 3Moorfields Eye Hospital NHS FT, London, UK, 4Ubisense Ltd., Cambridge, UK, 5NIHR Moorfields Biomedical Research Centre at MEH NHS FT, London, UK","locationCode":"4027","description":"\r\n\t<div>OBJECTIVES: A new pop-up eyecare diagnostic model has been piloted in London to help clear the ophthalmology outpatient appointment backlog. If successful, this can set a new blueprint for efficient high-volume, low-complexity (HVLC) care delivery systems. We identified key service features stakeholders consider for HVLC services for stable eye conditions and developed a discrete choice experiment (DCE) to explore trade-offs between these features. METHODS: We used a brief survey completed mostly by patients to shortlist six key features that were identified from the literature, qualitative interviews, and discussions with the multidisciplinary research team including Patient and Public Involvement (PPI) representatives. We optimized the DCE using D-factorial design and distributed it nationally, targeting eye patients, healthcare professionals (HCP), and the public. Conditional logistic regressions were used to estimate the strength of stated preferences and the trade-offs between attributes—willingness to wait (WTW) and willingness to travel (WTT). RESULTS: We received 39 responses for the initial shortlisting survey and found that level of expertise of person doing test, how well results are explained, appointment delays, and accessibility of venue by public transport were very important. We received 389 responses to the DCE itself—141 patients, 97 HCP, 151 public. Test staff expertise was consistently ranked as most important whilst parking availability was consistently least important. Respondents were willing to wait 7.8 additional months or travel 2.6 hours more to see an optometrist or doctor versus seeing a technician and receiving results via post. Patients were willing to travel almost 4 times as long as the public to see an optometrist or doctor. CONCLUSIONS: The option to discuss test results was important and should be considered in designing new diagnostic service delivery models. Improving other public services such as public transport, regarding both journey length and complexity, is important in service implementation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132837","diseases":[{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Nudging into a Stem Cell Donor Registry: Evidence from Lab and Field","id":"9447ced0-d04d-42dc-8d71-0173bc98fa5f","sessionCode":"HPR30","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Stem cell transplants are used as a last resort treatment for blood cancer. While a quarter of patients can receive a transplant from a relative's stem cell donation, most rely on voluntary donors. However, the likelihood of finding a match between donor and patient is low, necessitating a large pool of registered donors. Despite efforts to increase the donor pool through public advertising and recruitment events, 10% of patients still cannot find a suitable donor. This study examines the impact of altering the registration choice frame to increase the number of potential donors. METHODS: We conducted an online and field experiment. In the online experiment, we adapted an interactive decision game for stem cell donor registration and compared registration rates for two different choice frames and two information treatments. In the second part, participants were asked if they wanted to register with a German donor center. We replicated the different choice frames from the online experiment for participants to request a registration link and also implemented a treatment where participants received buccal swab registration sets at their participation addresses. RESULTS: From March to September 2022, 309 subjects participated in the experiment. Preliminary results show that both default and information treatments affected registration decisions in the lab. Further analyses will examine the influence of personal characteristics and dynamics within the simulated decision game. CONCLUSIONS: Our findings can improve stem-cell donor registration decision design and potentially increase registration rates. Additionally, our unique setup allows us to measure the external validity of decisions in laboratory experiments, contributing to experimental literature on donation behavior.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130987","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Analysis of Sugammadex for Neuromuscular Block Reversal for Laparoscopic Surgery from a Single Hospital System in China","id":"65c3875c-8836-4b1e-8d5c-02493259ce5e","sessionCode":"EE110","topDisplay":"Chen L1, Zhou K2, Zhang M3, Xuan J3 1Health Economic Research Institute, School of Pharmacy, Sun Yat-sen University, guangzhou, 44, China, 2Shanghai Centennial Scientific, Shanghai, 31, China, 3Health Economic Research Institute, School of Pharmacy, Sun Yat-sen University, Guangzhou, China","locationCode":"2047","description":"\r\n\t<div>OBJECTIVES: This study aims to compare the incidence of residual neuromuscular blockade (rNMB) and postoperative complications for patients undergoing laparoscopy in China, as well as assessing the impact on operating room (OR) and post anesthesia care unit (PACU) efficiency, and the potential cost savings with sugammadex (SUG), neostigmine (NEO) or spontaneous recovery from the hospital perspective. METHODS: A decision tree model was developed within the perioperative period and 1000 patients were simulated for each group. The incidence of postoperative complications were calculated, including rNMB- and pneumoperitoneum (PP)-related complications and treatment-related adverse events. The time spent in OR and PACU were also reported. Model parameters were obtained from published literature, public data and expert interviews. Costs were expressed in 2023 CNY (¥). RESULTS: Compared with NEO and spontaneous recovery, brand-name SUG would lead to 292 and 397 fewer postoperative complications respectively. Additionally, the OR time saw a decrease with brand-name SUG use (35.5 hours versus NEO, 82.0 hours versus spontaneous recovery), which could be used to perform 15 and 34 extra laparoscopic surgery respectively. The PACU time was also estimated to decline by 42.6 hours and 98.4 hours respectively. Total time saved in PACU with brand-name SUG could be used to monitor 31 and 71 extra patients. Moreover, SUG was found with more encouraging efficacy in elderly subgroups, and with higher subsequent economic benefits and OR/PACU running efficiency for Chinese hospital (mean net monetary gain were 442 yuan versus generic SUG and 4,887 yuan versus NEO). CONCLUSIONS: Sugammadex could effectively avoid postoperative complications compared with either NEO or spontaneous recovery, meanwhile reducing both the OR and PACU occupancy, despite a substantially higher medication cost might be followed. It indicates that sugammadex is likely to be an acceptable reversal agent choice in laparoscopy from the Chinese hospital perspective.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee110-poster20231030115951131192-pdf.pdf?sfvrsn=16b4a902_0","title":"EE110 Poster_20231030115951131192.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131192","diseases":[{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimating the Incidence and Characteristics of Female Patients in Norway With Early Breast Cancer Who Are at High Risk of Disease Recurrence","id":"d889ed97-4427-4f65-982e-02e09b73fef8","sessionCode":"EPH29","topDisplay":"Walid F1, Singh R2, Emde A2, Haro JM3 1Eli Lilly and Company, Bracknell, LON, UK, 2Eli Lilly and Company, Indianapolis, IN, USA, 3Institut de Recerca Sant Joan de Deu, Sant Boi de Llobregat, B, Spain","locationCode":"3031","description":"\r\n\t<div>OBJECTIVES: This retrospective study described the incidence and characteristics of female patients in Norway with early breast cancer with high-risk of disease recurrence, who met the high-risk criteria in monarchE (cohort 1), a Ph3 trial of abemaciclib for the adjuvant treatment of HR+, HER2-, node-positive, high-risk, early breast cancer. METHODS: Data from female patients with HR+, HER2-, Stage I-IIIC breast cancer, diagnosed between 2010-2019 and who had surgery were obtained from the Norwegian Breast Cancer Register. High-risk of recurrence was defined as per monarchE trial (cohort 1) criteria: having ≥4 positive lymph nodes or 1–3 positive nodes and grade 3 and/or primary tumor size ≥5cm. Patients not meeting this criteria were classified into low/moderate risk group. Survival rate was estimated using Kaplan-Meier curve. RESULTS: 20,632 female patients were included. 2,624 of them (12.7%) met the inclusion criteria for high risk of recurrence, 17,637 (85.5%) were considered low/moderate risk, while the rest were unknown (1.8%). Mean cases per year for the high-risk population was 262 (SD 22.9), and for the low/moderate risk 1,764 (SD 269.5). Majority of patients with stage III cancer were high-risk (78.9% of all stage IIIA/IIIB (n=1083) and 91.8% (n=302) for stage IIIC. In the high-risk population, 34.4% (n=908) had 1-3 positive lymph nodes (LN) and 65.6% (n=1721) ≥4 LN. The majority had tumor Grade 2 (40.1%; n=1,051) or 3 (40.5%, n=1,062) and a tumor size ≥2- <5cm (54.2%, n=1422) or ≥ 5cm (21.2%, n=557). 5-year overall survival rate was significantly lower for high-risk compared to low/moderate risk population (83.5% vs 94.3%; p<0.0001). CONCLUSIONS: About 13% of female patients with HR+, HER2-, Stage I-IIIC breast cancer in Norway diagnosed between 2010 and 2019 met the inclusion criteria for cohort1 high-risk of recurrence used in the monarchE trial and could benefit from more effective adjuvant treatments.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23fakhourieph29poster131374-pdf.pdf?sfvrsn=a434512d_0","title":"ISPOREurope23_Fakhouri_EPH29_POSTER131374.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131374","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Current Usage and Challenges of Goal Attainment Scaling in Clinical Settings","id":"606c4d20-8d4b-489a-b119-03dc18aab789","sessionCode":"PCR46","topDisplay":"Nesto S, Stanley J, Howlett SE, Chapman CA, Rockwood K, Sevinc G Ardea Outcomes, Halifax, NS, Canada","locationCode":"6045","description":"\r\n\t<div>OBJECTIVES: Goal Attainment Scaling (GAS) is a patient-centric outcome measure that quantifies the impact of interventions on personalized goals. GAS interviewers (clinicians/academics) use GAS and incorporate input from patients/caregivers on clinically meaningful goals and their attainment. We explored GAS usage, GAS challenges and effects of the SARS-CoV-2 pandemic on GAS practice. METHODS: A semi-structured guide was used to interview eleven GAS interviewers from Canada, US, UK and Australia. Recorded interviews were transcribed, themes were identified, and data were coded in NVivo 12. RESULTS: Four interviewers had used GAS for 20+ years, 5/11 were introduced to GAS through an article/conference and 7/11 were currently using GAS. Six interviewers used a 5-point scale (-2, -1, 0, +1, 2) with varying baseline levels. An advantage of GAS was that patients/caregivers set personally meaningful goals related to daily life. Results were also readily interpretable and actionable. For example, a decline in GAS scores indicated a need for higher levels of patient care. Seven interviewers commented that GAS was reliable and helped identify issues meaningful and relevant to patients/caregivers. Use of a goal inventory or prompting focused GAS interviews (10/11) to shorten interview time (11/11). Good interviewing skills were found essential (8/11) to setting quality goals. Most interviewers (8/11) were open to new processes for conducting GAS, including innovations to simplify interviewing. Several interviewers thought that those not using GAS were resistant to change or did not realize its value (4/11). Interestingly, the SARS-CoV-2 pandemic had several positive impacts on GAS use, as virtual appointments eliminated travel for patients and/or interviewers. CONCLUSIONS: GAS interviewers stressed the importance of interviewing skills, setting goals that were meaningful to patients/caregivers, and using goal inventories or prompts. Innovative GAS methods would be welcomed if they were time-saving and improved GAS usage.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23nestopcr46poster130209-pdf.pdf?sfvrsn=1fef96a2_0","title":"ISPOREurope23_Nesto_PCR46_POSTER130209.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130209","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Relationship between Health-Related Quality of Life and Productivity Loss and Indirect Costs of Leukemia and Lymphoma Patients and Caregivers: Estimates from a Cross-Sectional Study from Portugal","id":"4d2169d7-25df-4f85-9326-03ddcb11beec","sessionCode":"EE61","topDisplay":"Dimitrovova K1, Reis A1, Barbosa I2, Cunha L3, Ferreira MF4, Oliveira J4, Viana DS5 1MOAI Consulting, Lisboa, Portugal, 2Associação Portuguesa de Leucemias e Linfomas (APLL), Porto, Portugal, 3Associação Portuguesa Contra a Leucemia (APCL), Lisbon, Portugal, 4Associação de Apoio aos Doentes com Leucemia e Linfoma (ADL), Maia, Portugal, 5Janssen-cilag Farmacêutica, Lda, Oeiras, Portugal","locationCode":"2001","description":"\r\n\t<div>OBJECTIVES: To estimate overall work impairment, and indirect costs, of Leukemia and Lymphoma (L&L) patients and caregivers in Portugal, and to explore the relationship of HRQoL with these variables. METHODS: This cross-sectional study was based on the Work Productivity and Activity Impairment Specific Health (WPAI-SH) questionnaire, shared online, with the support of three national Patient Associations. Indirect costs were calculated using official National unit costs and variables from WPAI-SH. Two generalized linear models (GLM), for patients and caregivers, were used to explore the relationship between HRQoL and overall work impairment (using FACT-G for patients and QASCI for caregivers, a Portuguese instrument for the assessment of caregiver burden), adjusted by age, sex, education, ongoing treatment, and time since diagnosis (for patients) and duration of care (for caregivers). For indirect costs, a two-part model (logit and GLM) was used. RESULTS: 112 (n=45 Leukemia; n=67 Lymphoma) and 90 currently employed patients and caregivers answered the questionnaire between October-December 2022. Mean FACT-G score was 73.5(SD=16.2); mean QASCI score was 71.3(SD=17.6), measured from 0 to 160 (highest burden). Patients reported an overall work impairment of 33%(SD=38%) (e.g., 25% for chronic leukemia; 39% for acute leukemia), and caregivers 53%(SD=37%) (e.g., 35% for chronic leukemia; 68% for acute leukemia). Mean annual indirect cost per patient and caregiver was 6,157€ (SD=7,093€) and 9,951€ (SD=7,025€), respectively. When FACT-G score decreases by one-unit, overall work impairment increases by 0.68 p.p. (p<0.01), and indirect costs increase by 169€ (p<0.01). For caregivers, when QASCI score increases by one-unit, overall work impairment increases by 0.75 p.p. (p<0.01), and indirect costs increase by 148€ (p<0.01). CONCLUSIONS: FACT-G and QASCI are significantly related with overall work impairment, and consequently with indirect costs, in L&L patients and caregivers, respectively. Further studies, with a larger number of participants, should be conducted to explore differences within each type of Leukemia and Lymphoma.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dimitrovovaee61poster131724-pdf.pdf?sfvrsn=d77172e4_0","title":"ISPOREurope23_Dimitrovova_EE61_POSTER131724.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131724","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The EU Patient Community's Perspectives on Data Protection and Data Control","id":"87dab3e5-ba17-427d-8e30-041ead9c34e6","sessionCode":"PCR19","topDisplay":"Lalova-Spinks T1, Saesen R1, Silva M2, Geissler J3, Shakhnenko I4, Camaradou JC5, Huys I1 1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, VBR, Belgium, 2EUPATI Belgium vzw, Brussels, Belgium, 3Patvocates GmbH, Riemerling, Germany, 4European Organisation for Research and Treatment of Cancer, Brussels, Belgium, 5Patient partner / Independent researcher, Plymouth, UK","locationCode":"6026","description":"\r\n\t<div>OBJECTIVES: The EU General Data Protection Regulation (GDPR) is the subject of sensitive debates, particularly with regard to the challenges it poses for health research. While researchers’ experiences of navigating the complex legal landscape are increasingly captured in the literature, there has been little empirical investigation of patients’ perspectives on data protection and data control. The aim of this study was to fill this gap in scholarship by providing information about the EU/EEA patient community’s knowledge of the GDPR, experiences of exercising their GDPR rights, and views on data control. In addition, the study aimed to inform ongoing debates on the European Health Data Space proposal with insights into patients’ perspectives on the use of their personal data for health research. METHODS: An online survey (distributed from December 2022 to March 2023). Data were analyzed descriptively and inferentially. RESULTS: Two hundred and twenty people from twenty-eight European countries participated in the study. More than half of the participants had received training in drug development, clinical research, or patient engagement. Respondents had most experience in the disease areas of cancer (39%), infectious diseases (17%), and rare diseases (17%). While the majority of respondents had a high level of awareness about the GDPR (90%) and appreciated the opportunity to exercise control over the use of their personal data, few had previously exercised their individual rights under the GDPR. Survey participants were more likely to share personal data when patient representatives were involved in the decision-making processes of governance bodies. CONCLUSIONS: The findings highlighted the importance of awareness raising and training on the application of the GDPR in the context of health research, and provided important points to consider when building the future European Health Data Space.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-presentation-isporteodora-lalova-spinks128921-pdf.pdf?sfvrsn=cb97276_0","title":"Poster presentation ISPOR_Teodora Lalova-Spinks128921.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128921","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Canadian Pustular Psoriasis Study (CAPPS): Examining Disease Burden, Treatments, and Healthcare Resource Utilization for Generalized Pustular Psoriasis Flares","id":"012e4fca-1482-460a-8012-048837114a7d","sessionCode":"EPH32","topDisplay":"Milan R1, Read S2, Couture-Lapointe C3, Gniadecki R4, Gooderham M5, Khachatryan A6, Kim M1, Kirchhof M7, Martinez M8, Netchiporouk E9, Sarda V10, Zaidi S11, Veillette H3, Chandran N1 1Boehringer Ingelheim Canada Ltd, Burlington, ON, Canada, 2Certara Inc. Evidence & Access, Edinburgh, Midlothian, UK, 3Université Laval, Quebec City, QC, Canada, 4Northern Alberta Clinical Trials & Research Centre, Edmonton, AB, Canada, 5SKiN Centre for Dermatology, Peterborough, ON, Canada, 6Certara Inc. Evidence & Access, London, London, UK, 7University of Ottawa and the Ottawa Hospital, Ottawa, ON, Canada, 8Certara Inc. Evidence & Access, Madrid, Madrid, Spain, 9Research Institute of the McGill University Health Centre, Montreal, QC, Canada, 10Certara Inc. Evidence & Access, Secunderabad, Telangana, India, 11Certara Inc. Evidence & Access, Montreal, QC, Canada","locationCode":"3034","description":"\r\n\t<div>OBJECTIVES: To characterize generalized pustular psoriasis (GPP) and describe treatments, and healthcare resource utilization (HCRU) associated with GPP flares in Canada. METHODS: This was a retrospective, observational cohort study based on data extracted from medical charts of patients diagnosed with GPP between January 2011 and December 2020 at participating dermatology centers in Canada. Patients were followed from diagnosis date until the earliest of June 2021 or death. RESULTS: Fifteen patients from four centers in Quebec, Ontario and Alberta were included (mean age 60.3 years, 77.3% female). Sixty percent of patients had concomitant plaque psoriasis and 20% had depression and/or anxiety. Eight (53.3%) patients had public healthcare insurance. During follow-up, one (6.7%) patient died while 66.7% of patients had at least one flare, resulting in a rate of 0.5 (IQR: 0.3-0.9) flares per person-year. Nine (60%) patients had at least one emergency department visit or hospitalization related to GPP. Among these patients, the median cumulative length of stay per person-year was 5.8 days (3.4-26.0). Two (13.3%) patients had a record of receiving sickness or disability benefits. Overall, 73.3% of patients received topical corticosteroids for the treatment of GPP flare, 46.7% received oral corticosteroids and 13.3% received a biologic. The majority of treatments administered for GPP flares resulted in no or partial response. CONCLUSIONS: GPP is a rare disease and patients in Canada face considerable disease burden with high HCRU associated with flares. The absence of approved medications for GPP flares at the time of the study or a consensus in clinical guidelines for the management of GPP may account for the varied treatments that are given off-label to patients. As reported in our study, treatments currently administered for GPP flares are often ineffective, highlighting the high unmet need for new treatments for patients with GPP.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23chandraneph32poster132844-pdf.pdf?sfvrsn=c690eb64_0","title":"ISPOREurope23_Chandran_EPH32_Poster132844.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132844","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health System Costs and Resources for Stage-Specific Breast Cancer: A Population-Based Approach (Updated)","id":"4ff6dd4a-d068-42e0-9ef8-04a3536a84a7","sessionCode":"EE85","topDisplay":"Mittmann N1, Seung SJ2, Ante Z3, Gatley JM3, Liu N3, Chiarelli AM4, Wolfson M5, Simard J6, Earle C3 1CADTH, Toronto, ON, Canada, 2Sunnybrook Research Institute, Mississauga, ON, Canada, 3ICES, Toronto, ON, Canada, 4Ontario Health, Toronto, ON, Canada, 5University of Ottawa, Ottawa, ON, Canada, 6University of Laval, Laval, QC, Canada","locationCode":"2015","description":"\r\n\t<div>OBJECTIVES: To determine the health system costs and resources utilization of women diagnosed with breast cancer (BC) in Ontario, Canada. METHODS: This was a retrospective, population-based study using provincial-level databases to identify women diagnosed with BC in the Ontario Cancer Registry between April 1, 2015 to March 31, 2019, with a minimum of 2 years follow-up. Collaborative staging (stages I-IV, unknown or missing [UNK/M]) was used. Total mean health care cost per patient (Canadian dollars) was calculated using an individual person-level costing methodology. Treatment-specific person-level costs related to radiation therapy and drug therapy were calculated separately. Healthcare resource utilization (HCRU) per patient per year was also determined. RESULTS: We identified 32,037 women with BC, of which the majority were early stage (stage I=16,549, stage II=9,934, stage III=3,213, stage IV=898, UNK/M=1,443). The total mean BC diagnosis cost of the cohort was $52,752±$49,102, with costs increasing with advanced disease stage (stage 0=$32,519±$33,237, stage I=$38,621±$11,359, stage II=$59,950±$15,592, stage III=$85,041±$23,075, stage IV=$139,925±$97,930, UNK/M= $39,606±$13,269). Drug therapies were received by 80% of the cohort and stage IV diagnoses had the highest mean drug therapy cost at $78,090±$132,200. Radiation therapy was received by 90% of the cohort and stage III diagnoses had the highest mean radiation cost at $17,556±$7,931. Across all stages, the mean number of visits per year was 8.2 for cancer clinics, 4.9 for outpatient clinics, 28 for any physician encounters, and 15.4 for home care. In the first year after diagnosis, the mean number of visits were higher (26.5 for cancer clinics, 11.7 for outpatient clinics, 48.7 for any physician encounters and 25.8 for home care). CONCLUSIONS: This analysis showed that the mean healthcare cost per BC patient was high and increased with advanced disease stage, and that HCRU was highest in the first year after diagnosis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/eurisporposterbccostoct29uploaded128666-pdf.pdf?sfvrsn=3944be2a_0","title":"EurISPORposter_BCCost_Oct29uploaded128666.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128666","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Modelling the Effects of Smoking on Healthy Life Expectancy (HLE) in England","id":"41eb0638-0dbb-41dd-b632-06834173fa81","sessionCode":"HPR35","topDisplay":"Pijper A1, Chan MS2, Xie P2, Cairns A3, Mayhew L4 1Health Analytics, Lane Clark & Peacock LLP, London, LON, UK, 2Health Analytics, Lane Clark & Peacock LLP, London, UK, 3Heriot Watt University, Edinburgh, UK, 4Bayes Business School, London, UK","locationCode":"4008","description":"\r\n\t<div>OBJECTIVES: The UK Government has a target of increasing HLE by 5 years by 2035 and narrowing geographical inequalities in HLE, where smoking patterns are a significant driver. We explored the quantitative link between smoking and HLE by modelling and projecting the potential impacts of smoking interventions on HLE at local and national levels. METHODS: We analysed variations in smoking prevalence across the 149 upper-tier local authorities (UTLAs) in England and compared these to variations in HLE. To attempt to control for confounding demographic factors and extract a direct link between smoking and HLE, we focused our analysis on pairs of UTLAs with similar geographic and demographic features. We applied the Sullivan method to construct life tables with health and mortality components for current smokers, ex-smokers and never-smokers. We used these to convert age-specific changes in smoking prevalence into changes in overall population HLE at a local and national level. This provided a tool for quantifying the impact of smoking intervention programmes and evaluating to what extent these can help deliver the government’s target of increasing HLE by 5 years by 2035. RESULTS: We found a strong correlation between levels of deprivation and smoking prevalence (+0.76), and between smoking prevalence and HLE (-0.73). However, we found that reducing smoking prevalence would likely have a relatively modest impact on HLE in the short term: even in an extreme scenario where everyone in the most deprived UTLA quits smoking overnight, the modelled gain in HLE in that UTLA is less than 3 years. CONCLUSIONS: The link between deprivation, smoking and healthy life expectancy suggests that smoking intervention programmes have a role to play in reducing health inequalities. However, to meet the government’s ambitious HLE targets, smoking interventions would need to be combined with broader action across the multiple drivers of poor health outcomes in the UK.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23pijperhpr35poster132920-pdf.pdf?sfvrsn=5fe934ca_0","title":"ISPOREurope23_Pijper_HPR35_POSTER132920.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132920","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evolution of the Utilization of the Aifa 5% Fund for Orphan Drugs and Rare Diseases","id":"63f2fd68-3789-416c-aa9a-06baab808c26","sessionCode":"HPR29","topDisplay":"Tartarelli F1, Viola V2, Lidonnici D3, Marcellusi A4 1Sapienza università di Roma, ROMA, Italy, 2Pharmavalue, Roma, Roma, Italy, 3More Than Access Srl SB, Legnano, MI, Italy, 4Economic Evaluation and HTA (EEHTA CEIS), Department of Economics and Finance, Faculty of Economics, University of Rome “Tor Vergata”, Rome, Italy","locationCode":"4010","description":"\r\n\t<div>OBJECTIVES: Since 2003 law 326 established the creation of a National Fund at AIFA to be used for the acquisition of orphan drugs for treatment of rare diseases and drugs that represent a therapeutic hope for specific and serious conditions. The analysis aims to understand the evolution of the utilization and allocation of economic resources for the so-called AIFA 5% from 2015 to 2021. METHODS: The information available in the 'transparent administration' section of the AIFA website was examined to understand the allocation of resources for the AIFA 5% Fund. Yearly economic parameters such as initial budget, allocation, total expenditure, and final balance were considered. Through OSMED reports, the 10 drugs with the greatest impact and the number of patients treated were also identified. This analysis provides a partial overview of the use of financial resources according to Law 326/2003. RESULTS: From 2015 to 2019, the Fund maintained a budget surplus, particularly considering the significant allocations: the highest expenditure was € 8.318.000 in 2019, while allocations remained almost stable at around € 17.000.000 per year. From 2019 onwards, higher annual expenditures were observed up to € 40.441.000 in 2021, with a reversal in allocations that decreased to approximately € 10.000.000. In fact, in 2021, AIFA decided to temporarily suspend the activities related to the fund to initiate a simplification and optimization of the system. CONCLUSIONS: Over recent years, the AIFA 5% Fund has seen an exponential increase in its utilization. Comparing these data with OSMED reports, we also found some discrepancies, as the expenditure in the OSMED reports is higher than that in the AIFA balance sheets, highlighting the need for a more transparent and univocal reporting. We look forward to 2023 data to understand if the trend of expenditure compared to the allocation is confirmed.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23tartarellihpr29poster131988-pdf.pdf?sfvrsn=88a5c0b3_0","title":"ISPOREurope23_Tartarelli_HPR29_POSTER131988.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131988","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Burden of HIV/AIDS Assessment and Forecast for 2030 in Turkiye","id":"b135d1f7-ba16-480f-8a5b-073757ebd89f","sessionCode":"HPR3","topDisplay":"Erkut Y1, Kurnaz M1, Okcun S1, Kockaya G2 1ECONiX Research, Istanbul, Turkey, 2ECONiX Research, Samsun, 55, Turkey","locationCode":"3060","description":"\r\n\t<div>OBJECTIVES: HIV affects 38.4 million people worldwide, with 34,000 cases in Türkiye. The impact of HIV continues to grow, without proper treatment, it can lead to life-threatening AIDS-related complications. A descriptive analysis of published official data on HIV/AIDS was conducted to raise social awareness and provide valuable insights. This study aims to forecast the potential number of cases and treatment costs up to 2030 by utilizing available statistics in Türkiye. METHODS: Linear regression was used to forecast the number of HIV-infected patients, deaths, and healthcare costs until 2030, utilizing data from literature and official authorities, published between 1985 and 2022. To estimate deaths, the proportion of past deaths attributed to AIDS was considered, using this forecast the number of patients living with HIV was indirectly estimated. Treatment expenditures associated with HIV/AIDS in Türkiye were estimated by considering published healthcare cost data, the number of living patients, the potential increase in HIV/AIDS cases, future inflation & currency predictions from the Turkish Central Bank. RESULTS: Projections indicate that in 2030, the number of new HIV/AIDS cases is expected to increase from 3,002 in 2021 to 5,709 among a total of 74,227 living patients. The reported total deaths as 598 between 1985 and 2022 are predicted to rise to total as 1,256 by 2030. The estimated total treatment cost for HIV/AIDS in 2021 was TRY1,051,026,183 (USD118,683,580), projected to reach TRY15,432,842,049 (USD296,450,696) by 2030. This includes expenses for outpatient visits (TRY246,710,838 [USD4,739,088]), laboratory tests (TRY2,072,489,815 [USD39,810,623]), inpatient visits (TRY1,352,192,479 [USD25,974,373]), management of complications (TRY3,371,126,960 [USD64,756,247]), and antiretroviral therapy drugs (TRY8,390,321,957 [USD161,170,365]). CONCLUSIONS: By 2030, the prevalence of HIV/AIDS in Türkiye is expected to rise by 137%, with the economic burden increasing by 14.7 times in TRY (2.5 times in USD) compared to 2021. This highlights the urgency for relevant policy measures to address the situation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/gsk-burden-of-hiv130940-pdf.pdf?sfvrsn=c1a6f910_0","title":"GSK-Burden of HIV130940.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130940","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Baseline Characteristics of Patients Applying for Reimbursment of Liraglutide (Saxenda®) for Weight Management Under a Managed Access Protocol in Ireland","id":"fe9da655-4dfd-4654-9527-084c6100bfee","sessionCode":"HPR2","topDisplay":"Gorry C1, Daly M1, Doran S1, Finnigan K2, Clarke S1, Barry M1 1HSE Medicines Management Programme, Dublin, Dublin, Ireland, 2HSE Medicines Management Programme, Dublin 15, Ireland","locationCode":"3054","description":"\r\n\t<div>OBJECTIVES: To review baseline characteristics of applicants seeking reimbursement for liraglutide (Saxenda®) for weight management, under a managed access protocol (MAP) operated by the Health Service Executive-Medicines Management Programme (HSE-MMP) in Ireland. In Phase I, Saxenda® is reimbursed for patients with a body mass index (BMI)>35kg/m2, with prediabetes and high-risk for cardiovascular disease. METHODS: Data was extracted from electronic applications submitted via the Individual Patient Reimbursement Request online application system operated by the HSE Primary Care Reimbursement Service (HSE-PCRS), and analysed in Microsoft Excel™. Applications made between 1 January 2023 and 30 April 2023 under Phase I of the MAP were eligible for inclusion. RESULTS: From 1 January 2023 to 30 April 2023, 3,081 applications were received for liraglutide reimbursement via the online application system; 2,950 (95.7%) were extractable for this analysis. The majority of applications were for females (75.1%), with an average age of 50.6 years and an average BMI of 43.5 kg/m2. A minority of applicants were reported to have obesity secondary to endocrinological or eating disorders (14.4%), and 97.5% were reported to be currently participating in non-pharmacological interventions for weight management. High-risk for cardiovascular disease was reported in 83.4% applications, with 37.9% reported to be in receipt of lipid-lowering therapy and 50.9% in receipt of blood pressure-lowering therapy. A confirmed diagnosis of pre-diabetes was reported in 48.2% of the applications. The average fasting total cholesterol level was 5.83 mmol/L and the average HbA1c level was 39.9mmol/mol. Reimbursement was approved for 1,575 (53.4%) applications that met all the criteria for the MAP. CONCLUSIONS: Data collected will serve to inform future reimbursement decisions for weight management interventions. In phase II of the MAP, outcome data on weight change will be recorded; continued reimbursement for approved patients is conditional on ≥ 5% weight loss over the Phase I treatment period.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gorryhpr2posterv2132814-pdf.pdf?sfvrsn=8368b249_0","title":"ISPOREurope23_Gorry_HPR2_POSTERV2132814.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132814","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparison of the Use of Real-World Evidence (RWE) by Health Technology Assessment (HTA) Agencies in Reviews of Drugs for Rare Diseases (DRDS)","id":"dcbcdca7-e68a-4fae-8721-0b2b2c3659e6","sessionCode":"RWD23","topDisplay":"Kirby J, Tadros N, Gosain S PDCI Market Access a division of McKesson Canada Corporation, Mississauga, ON, Canada","locationCode":"6075","description":"\r\n\t<div>OBJECTIVES: The use of real-world evidence (RWE) by health technology assessment (HTA) agencies varies around the world. This may impact decisions for drugs for rare diseases (DRDs) where it may not be possible to meet traditional evidence standards. The objective of this analysis was to determine how Canadian and international HTA agencies are using RWE, to understand how RWE is influencing recommendations, and to better inform stakeholders on best practices for including RWE in submissions. METHODS: Canadian Agency for Drugs and Technologies in Health (CADTH) DRD recommendations issued between January 2020 and June 2023 were reviewed. Recommendations for the same drugs were then identified from HTA agencies in Quebec, the United Kingdom (England, Scotland), France, the Netherlands, Australia, and New Zealand. The use of RWE in the evaluation, the RWE methods, and the recommendations were extracted and compared to identify the role and impact of including RWE on the recommendations. RESULTS: CADTH reviewed 40 DRDs and 9 of the submissions included RWE. The number of DRD recommendations that included RWE ranged from 0 (Scotland) to 9 (Canada). Submissions considered RWE from simulation and observational studies (including registry analyses and both retrospective and prospective cohort studies). Among the identified submissions to all HTA agencies combined, RWE was mostly used in the assessment of safety (n=15) and efficacy (n=18). Two drug products (trientine hydrochloride (2 formulations)) were given positive recommendations based on RWE alone by 4 HTA agencies. The same RWE studies were submitted across HTA agencies for the same drugs. CONCLUSIONS: The use and impact of RWE varied across agencies and transparency in reporting limited the comparisons that could be drawn. It is encouraging that RWE is being included in DRD submissions and in situations where evidence from randomized controlled studies is not feasible, drugs may be recommended based on RWE.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133528","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Consequences of Using Clevidipine in Acute Hypertension from the Perspective of a US Hospital","id":"1c0e588a-547d-452f-bfb0-0c0e5a2638e6","sessionCode":"EE125","topDisplay":"Gutierrez M1, Wild S2, Compton A3, Paine E3, Jensen I4, Shah A5 1Chiesi USA,Inc, HUNTINGTON BEACH, CA, USA, 2Chiesi USA,Inc, Lindenhurst, IL, USA, 3Chiesi USA,Inc, Cary, NC, USA, 4Precision Health Economics & Outcomes Research, Boston, MA, USA, 5Precision HEOR, Chestnut hill, MA, USA","locationCode":"2056","description":"\r\n\t<div>OBJECTIVES: Clevidipine is an intravenous (IV) dihydropyridine calcium channel blocker indicated for the reduction of blood pressure (BP) when oral therapy is not feasible or not desirable in US. The aim of the cost consequence analysis was to estimate the economics and consequences of varying clevidipine utilization in patients with acute hypertension (aHTN). METHODS: A decision analytic model was developed to simulate the costs and consequences associated with the use of clevidipine, labetalol, and nicardipine in patients with severe aHTN in the emergency room or critical care setting. The outcomes were quantified from a US hospital perspective over a 3-year time horizon. The utilization of IV-antihypertensives was calculated by a combination of purchase history and Diagnosis Related Group (DRG) claims. Low and high clevidipine adopter profiles were formed using a retrospective analysis of IV-antihypertensive purchases in hospitals above the median of aHTN-claims. A change in utilization was modelled from the low adopter profile with a linear increase over 3 years to the high adopter profile. Infusion rates were based on customer survey data on file. Drug costs were based on wholesale acquisition cost from ProspectoRx. Clinical inputs, and dosing information were based on literature. RESULTS: For a hypothetical caseload of 100 patients with aHTN, the use of clevidipine over 24 hours resulted in 2 more patients reaching BP target in 30 minutes and the average time to reach BP target was 7.2 min faster. Additionally, there were 27 fewer cases of concomitant or subsequent IV-antihypertensive use. The average primary drug infusion volume was reduced by 549 mL per patient. The average drug costs decreased by $84/patient. CONCLUSIONS: The increased use of clevidipine in patients with aHTN results in more patients reaching BP target in less time with a decrease in the cost over the 3 years.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/3-chs64433-ccmcleviprexposteracutehypertensionee125isporeu2023130709-pdf.pdf?sfvrsn=ef07ba4_0","title":"3. CHS64433.CCMCleviprexPoster_AcuteHypertension_EE125_ISPOREU2023130709.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130709","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Technology Management - The Future of the Post-Reimbursement Phase?","id":"6bc4dc34-041b-48a8-b3c1-0e0a6205e4f6","sessionCode":"HSD23","topDisplay":"Smith A1, Clarke S2, Barry M2 1HSE Medicines Management Programme, Dublin 8, Ireland, 2HSE Medicines Management Programme, Dublin, Dublin, Ireland","locationCode":"4036","description":"\r\n\t<div>OBJECTIVES: Health technology management (HTM) has been described as ‘measures put in place to enhance the safe, effective, and cost-effective use of medicines thereby controlling utilization and expenditure’1. HTM measures are becoming increasingly common as affordability is an ongoing issue for health systems, particularly with the introduction of high-cost medications for prevalent diseases. This review aims to further describe HTM and the significant impact these measures have on cost-effective prescribing in Ireland. METHODS: This review will describe a number of examples of HTM, including; <ol> <li>Reimbursement application systems (eg. Lidocaine medicated plaster (Versatis®))</li> <li>Best-value biological medicines (BVB) initiatives (eg. adalimumab and etanercept)</li> <li>Managed access protocols (MAPs) for certain high-cost or high-budget impact medicines (eg. calcitonin gene-related peptide monoclonal antibodies (CGRP MABs))</li> </ol> RESULTS: The HTM approach may be applied to new or already reimbursed technologies. The introduction of a reimbursement application system for Versatis® ensured reimbursement was confined to patients with the licensed indication - this resulted in savings of approximately €2.75 million per month to the Health Service Executive (HSE)*. The identification of BVBs for TNF-α inhibitors, adalimumab and etanercept, lead to a significant shift in prescribing from the originator products to the designated best-value biosimilars. In one year, this initiative resulted in combined estimated savings and avoided costs of €22.7 million2. The CGRP MABs are available subject to a managed access protocol; this ensures reimbursement is confined to the patient cohort for whom cost-effectiveness has been demonstrated. Since September 2021, just over 1,000 patients have been dispensed a CGRP MAB under the MAP. CONCLUSIONS: Healthcare payers, such as the HSE, are increasingly recognising the benefits of HTM. This review highlights the significant efficiencies that can be achieved through HTM.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeuropesmithhsd23poster133332-pdf.pdf?sfvrsn=e3e0c6e4_0","title":"ISPOREurope_Smith_HSD23_POSTER133332.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133332","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparing Statistical Adjustments to Correct Misclassification of Respiratory Syncytial Virus Infections (RSV) in Claims Data","id":"d28d4e25-afae-4caf-92ec-0e60be5a4495","sessionCode":"MSR3","topDisplay":"Steinmann M, Schmidt J, Greiner W Department for Health Economics and Health Care Management, Bielefeld University, Bielefeld, NW, Germany","locationCode":"5049","description":"\r\n\t<div>OBJECTIVES: Measurement errors or misclassification, incomplete data and unmeasured confounding, among others, could lead to biased and imprecise estimators. Especially extensive data sets, e.g. claims data, may be more prone to misclassification due to their collection method and purpose. Misclassification is handled as a missing data problem; using multiple imputation with chained equations (MICE) as current standard practice for adjustment. However, less is known about the performance of multiple imputation (MI) in more complex models and its use for epidemiological and health economic studies with misclassified claims data. An adaptable substitute for MI is the Full Bayesian technique (FB), which integrates imputation and analysis within a single estimation process. METHODS: This study aims to compare the performance of MI and FB for a (potentially) misclassified binary outcome; whether or not subjects had a hospitalized, i.e. confirmed, ICD-10 diagnosis of RSV. We use a split-sample approach where hospitalization is the assumed accurate estimator. To obtain information about the relation between the true and the apparent disease status, an infallible classifier (\"gold-standard\") of the true infection status is estimated. Finally, we use the classifier in our MI and FB approach to adjust the rate of misclassification in claims data for RSV. RESULTS: We assess the performance of MI for adjusting for misclassified data and compare its performance with FB which simultaneously imputes missing values and estimates parameters of the epidemiological model. Findings demonstrate that MI may be the most parsimonious choice, but necessitates an accurate specification. In contrast, FB does not require explicit specification of how the outcome enters the imputation models. CONCLUSIONS: Both methods could perform well when handling missing data in epidemiological studies. Addressing misclassification with MI or FB can yield significant benefits in improving the risk estimation for claims data analyses.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23steinmannmsr3poster133459-pdf.pdf?sfvrsn=27b0326a_0","title":"ISPOREurope23_Steinmann_MSR3_POSTER133459.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133459","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Costs of Dose Escalation Among Patients with Inflammatory Bowel Disease Treated with Vedolizumab and Ustekinumab in Portugal","id":"cb6e9f4d-a47e-432c-9999-0ee5df740d01","sessionCode":"RWD6","topDisplay":"Lopes da Cruz JP1, Faria M2, Aleixo AS3, Pereira F4 1Centro Hospitalar Universitario de Lisboa Norte, Lisboa, Lisboa, Portugal, 2Takeda Farmacêuticos Portugal, Oeiras, Portugal, 3IQVIA Portugal, Porto Salvo, Lisboa, Portugal, 4IQVIA Portugal, Porto Salvo, Portugal","locationCode":"6059","description":"\r\n\t<div>OBJECTIVES: Patients with inflammatory bowel diseases (IBD) are commonly treated with biologic therapies such as ustekinumab and vedolizumab and usually undergo dose escalations (DE), which impacts the treatment costs of these treatments. This study aims to evaluate the treatment costs based on the real-life dosages of ustekinumab and vedolizumab administered to patients in Portugal. METHODS: Using retrospective and longitudinal data provided by a set of 16 public hospitals, patients treated with vedolizumab and ustekinumab in gastroenterology departments were identified. Those who started maintenance treatment from January 2020 onwards and completed one year of therapy were selected. The annual maintenance dosage per patient was obtained by summing all doses consumed over a one-year period. Additionally, dose escalation prevalence and magnitude were used to quantify the equivalent patient treatment rate representing the number of patients per 100 that could have been treated with the standard dosing. The average annual treatment cost per patient was compared to the annual costs if the defined standard SmPC dosages were followed, using list prices. Finally, a sensitivity analysis on the prices of both molecules was performed to evaluate the differences in DE. RESULTS: 39% of vedolizumab and 93% of ustekinumab patients undergo DE in their first year of maintenance. The patient equivalence is higher for ustekinumab (161) than vedolizumab (117). The real-life annual treatment cost with vedolizumab (10.215€) and ustekinumab (14.958€) increase 17% and 61%, respectively, when compared to the expected cost if the standard SmPC dose was followed. Considering real-life DE and the list prices, Vedolizumab is 32% less expensive than ustekinumab. CONCLUSIONS: During the first year of maintenance, patients treated in Portugal with ustekinumab undergo DE more often than vedolizumab. This DE causes an unexpected increase in the annual treatment costs, with greater relevance for ustekinumab.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023-10-30-ispor2023-costs-rwd6130512-pdf.pdf?sfvrsn=efe734ba_0","title":"2023.10.30 ISPOR2023.Costs.RWD6130512.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130512","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Prioritization of Social Disability Management Using a Patient Reported Outcome Questionnaire in Patients Suffering from Multiple Sclerosis (MS)","id":"b037ee6b-e186-4098-ac98-0f99a3387e37","sessionCode":"PCR14","topDisplay":"Donze C1, Mekies C2, Mouzawak C3, Paillot G4, Brechenmacher L5, Montagu G6, Nevoret C7, Duburcq A7, Civet A8, Pau D9, Berdeaux G10, Cohen M11 1Hôpital Saint Philibert, Lille, Finland, 2Clinique des Cèdres, Toulouse, France, 3Hôpital du Vésinet, Le Vesinet, France, 4Association Aventure Hustive, Grenoble, France, 5Roche SAS, Boulogne-Billancourt, France, 6Unknowns, Paris, France, 7CEMKA, Bourg-la-Reine, France, 8Roche, Boulogne Billancourt, 92, France, 9Roche, Boulogne-Billancourt, 92, France, 10BERDEAUX CONSULTING SAS, PARIS, France, 11Hopital Pasteur 2, Nice, France","locationCode":"6016","description":"\r\n\t<div>OBJECTIVES: Focal-MS is a PROM dedicated to help patient social disability management during a doctor visit. This questionnaire is psychometrically validated following FDA recommendations. Ten social dimensions were identified. For each dimension, the perceived importance is documented by the patient using a Lickert scale (0 to 10). This abstract reports on how importance might impact on the final decision making. METHODS: A cross-sectional survey was conducted in collaboration with MS patient associations, in France. Severity stratification was performed using SymptoMScreen. Data from patients having completed 80% of the questionnaire were included in the analysis. Scores were estimated based on author recommendations and weighted score used the Lickert importance scale. Weighted and unweighted scores were ranked separately at a patient level. For each level of priority, from 1 (top priority) to 10 (last priority), a concordance table crossing the 10 dimensions, weighted versus unweighted, was produced. RESULTS: 653 patients were analyzed. Socio-demographics were aligned with French MS estimates. A strong ceiling effect was observed on perceived importance with very few scores below 5 showing the impact of MS on social activities. The top-3 importance was parental role, ability to live at home, MS care decision making empowerment. Taking perceived importance into account changed dramatically the evaluation of patient social need leading to very different prioritization of the patient social support. For the patient #1 priority, ie the one to be addressed first, taking into patient perceived importance would change the social dimension support in 23% of the patients. Decision changes while considering perceived importance would occur in in 43%, 51%, 54%, 48%, 41%, 37%, and 33% of the patients, from #2 to #8 priority, respectively. CONCLUSIONS: Accounting for patient perceived importance when evaluating social handicap management is key at allocating the right resources to improve patient MS quality of life.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023-posterdv-ocr94444final128661-pdf.pdf?sfvrsn=90fb684e_0","title":"ISPOR 2023 Poster_DV-OCR94444_final128661.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128661","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Quantifying the Future Impact of Chronic Kidney Disease Screening Programs in Belgium","id":"ef37a340-4679-42e4-9b28-0ff5c7387cc6","sessionCode":"EE116","topDisplay":"Vadia R1, Vandendriessche E2, Meeus G3, Mahieu E4, Jouret F5, Van Pottelbergh G6, Maris M2, Retat L7, Jadoul M8, Vankeirsbilck A2, Garcia Sanchez JJ9 1AstraZeneca BeLux, Brussels, VBR, Belgium, 2AstraZeneca BeLux, Groot-Bijgaarden, Flanders, Belgium, 3AZ Groeninge, Kortrijk, West Flanders, Belgium, 4AZ Glorieux, Ronse, East Flanders, Belgium, 5CHU Liege, Liege, Wallonia, Belgium, 6KU Leuven, Leuven, Flemish Brabant, Belgium, 7HealthLumen, London, Greater London, UK, 8Cliniques universitaires Saint-Luc, Brussels, Brussels, Belgium, 9Health Economic and Payer Evidence, AstraZeneca, Cambridge, Camebridgeshire, UK","locationCode":"2051","description":"\r\n\t<div>OBJECTIVES: Chronic kidney disease (CKD) affects approximately 12% of the population in Belgium, with diagnosis mainly made in the later stages. Earlier diagnosis of CKD offers the opportunity to implement guideline-based interventions sooner, thereby delaying disease-progression and improving patient-outcomes. Inside CKD, using validated, patient-level microsimulation, aims to estimate benefits of investing in screening strategies to diagnose CKD in 10 years’ timeline. METHODS: The microsimulation model constructs a virtual baseline population from currently available Belgium-specific data on demographics, CKD status, comorbidities, all-cause mortality, and associated costs. Two hypothetical screening strategies were compared to “current scenario”: i) 2 estimated glomerular filtration rate (eGFR) tests and ii) 2 eGFR tests + 1 urinary albumin-to-creatine ratio (UACR) test. The “current scenario” reflects the present situation of lower diagnoses in Belgium as reported in literature. The model projects cost-effectiveness of these strategies from 2022 to 2032, across the full CKD population (≥ 45 years) and several subgroups at high risk: type-2 diabetes, hypertension, cardiovascular disease, and age ≥ 65 years. RESULTS: The implementation of both screening programs increased the diagnosis rate by 21.8% from 2022 to 2032. Compared to the “current scenario” for the full CKD population, the 2eGFR only strategy showed incremental cost-effectiveness ratios (ICERs) of 5,925.88 €/QALY which further decreased to 5,143.73 €/QALY when adding a UACR test to the 2eGFR only. Furthermore, when assessed only in high-risk subgroups, the ICERs lower down substantially up to ~3,000€/QALY. CONCLUSIONS: Both screening strategies are cost-effective in 10-year timeline for the Belgian healthcare system, considering a willingness-to-pay equal to Belgian gross domestic product per capita (i.e., approx. 43,300 €). Implementation of continuous screening, preferably UACR test in addition to 2eGFR tests in primary care, particularly for high-risk subgroups, may lead to timely and optimal treatment delaying CKD progression, improving patient outcomes, and subsequently reducing healthcare burden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterscreening130433-pdf.pdf?sfvrsn=b87004cf_0","title":"Poster_screening130433.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130433","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparison of the Clinical and Economic Impact of Two MRNA COVID-19 Vaccines in High Risk Individuals in Japan","id":"ae20434f-ffa7-4c60-af4f-1034a43de3f8","sessionCode":"EE140","topDisplay":"Kohli M1, Hagiwara Y2, Igarashi A3, Joshi K2, Lee A1, Maschio M1, Van de Velde N2, Beck E4 1Quadrant Health Economics Inc, Cambridge, ON, Canada, 2Moderna, Inc., Cambridge, MA, USA, 3Yokohama City University School of Medicine, Yokohama, Japan, 4Moderna, Inc., Munich, BY, Germany","locationCode":"2069","description":"\r\n\t<div>OBJECTIVES: Immunocompromised (IC) individuals and those aged ≥65 years are considered at high risk of severe disease following COVID-19 infections. In Japan, COVID-19 vaccines have been recommended to protect these vulnerable populations. Recent analyses found the Moderna mRNA vaccine more effective in preventing medical encounters following COVID-19 infections than the Pfizer-BioNTech vaccine in both populations (e.g., relative risk for hospitalization of 0.83 in IC individuals). The objectives of the study were to separately estimate the impact of differential vaccine effectiveness on clinical outcomes and treatment costs for individuals aged ≥65 years, and IC individuals aged ≥18 in Japan. METHODS: A decision-analytic model was used to predict clinical and economic outcomes of vaccinating with either the Moderna or Pfizer-BioNTech COVID-19 vaccines over a 1-year time horizon. Base-case and low to high infection incidence scenarios were created based on the observed incidence in Tokyo during the Omicron period. Fall 2023 vaccine coverage was estimated from previous vaccination campaigns. Consequences of infection (outpatient and hospitalizations, myocarditis, death, long-COVID) and COVID-19 treatment costs were calculated. RESULTS: Considering the base-case and low to high incidence scenarios, vaccinating persons aged ≥65 years with the Moderna vaccine could prevent additional COVID-19 outcomes compared with the Pfizer-BioNTech vaccine: 22,170 (16,620-17,710) hospitalizations, 1,740 (1,310-2,180) deaths and 19,480 (14,610-24,350) cases of long-COVID. The reduced morbidity corresponds to ¥13.9B-¥23.2B in treatment costs saved. Compared to the Pfizer-BioNTech vaccine, the Moderna vaccine could prevent an additional 5,800 (4,350-7,250) hospitalizations, 490 (370-610) deaths, and 4,090 (3,070-5,110) cases of long-COVID in the IC population, saving to ¥3.5B-¥5.9B in treatment costs. CONCLUSIONS: Vaccinating those aged ≥65 years and IC individuals aged ≥18 years with the Moderna vaccine in Japan is estimated to prevent more cases of COVID-19 related hospitalizations, deaths, and long-COVID, and associated treatment costs compared with the Pfizer-BioNTech vaccine.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/bimjapanposterisporeu2023133198-pdf.pdf?sfvrsn=540ebf40_0","title":"BIM_Japan_Poster_ISPOR_EU_2023133198.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/bimjapanpostersuppisporeu2023133198-pdf.pdf?sfvrsn=19ce1712_0","title":"BIM_Japan_Poster_Supp_ISPOR_EU_2023133198.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133198","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Disease Diagnosis and Severity Prediction: Artificial Intelligence Applications in Hemophilia A","id":"48ea5832-eef6-4486-a574-107e437108b9","sessionCode":"MSR19","topDisplay":"Khatib M1, Kumar P2, Shabran S3, Subudhi S3, Oliver C4, Net P5 1Syneos Health, Bengaluru, KA, India, 2Syneos Health, London, UK, 3Syneos Health, Gurugram, Haryana, India, 4Syneos Health, New York, NY, USA, 5Syneos Health, Montrouge, France","locationCode":"5061","description":"\r\n\t<div>OBJECTIVES: Despite recent advances, the hemophilia A diagnosis accuracy in claims/clinical settings remains unknown. Artificial intelligence (AI) is an emerging reality that has the potential to bring a paradigm shift in hemophilia A. Machine learning (ML) has encouraging results in hemophilia diagnosis, severity prediction, user-centered app, gene therapy, myocardial infarction risk estimation, identification of factor V and CRISPR/Cas9 nuclease off-treatment target. The objective of this review was to explore the AI use in hemophilia A diagnosis and severity prediction. METHODS: PubMed, EMBASE and secondary searches were conducted on the AI in hemophilia A in clinical trials/real-world settings, from inception till June 2023. Outcomes included diagnosis and severity prediction aiding clinical decision-making using AI. RESULTS: Of the 108 hits, ten studies utilized AI for diagnostic decision-making (n=5), severity prediction (n=4), and both (n=1) in hemophilia A. Different AI models included ML (Case-Based Reasoning, Expert system, Haemaxpert, Hema-class) and Deep learning (DL) (Graph-based neural network). Validation was described by four studies. Reference standards included biological thrombin generation assay and genetic testing. ML model accuracy for severity prediction ranged between 62%-73.86%; for diagnosis ranged between 80-95.57% with 65% positive predictive value. At a probability threshold of 0.6, 94.4% sensitivity, and 90.1% specificity were seen. A cascade of ML models accurately diagnosed hemophilia (99.18%), its type (98.1%), and severity (96.23%). DL model accuracy for severity prediction was 69%. CONCLUSIONS: Though AI models for hemophilia A can deal with highly abstract data features and different data types assisting diagnosis and severity prediction, they lack comprehensive quality datasets and face operational, ethical, interpretability, clinically irrelevant performance metrics, and methodological research concerns. To endorse best practices for AI in hemophilia A, it is crucial to develop critical safeguards, transparent policies, and robust data infrastructure. Further research on adapting AI models for implementation in clinical practice is warranted.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23khatibmsr19poster131759-pdf.pdf?sfvrsn=80292392_0","title":"ISPOREurope23_Khatib_MSR19_POSTER131759.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131759","diseases":[{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Portuguese Clinical Practice and Costs in the Treatment of Acid Sphingomyelinase Deficiency Disease","id":"03c7068b-ca88-404d-ae5a-10cc594dd17c","sessionCode":"HTA26","topDisplay":"Silva Miguel L1, Carvalho P2, Oliveira A3, Guimas A4, Leão Teles E5, Ferreira S6, Bulhosa C2, Pinheiro B1, Borges M7 1IQVIA Portugal, Salvo, Oeiras, 13, Portugal, 2IQVIA Portugal, Lisboa, Portugal, 3Centro Hospitalar Universitário de Lisboa Norte, Lisboa, Portugal, 4Centro Hospitalar Universitário de Santo António, Porto, Portugal, 5Centro Hospitalar Universitário de São João, Porto, Portugal, 6Centro Hospitalar Universitário de Coimbra, Coimbra, Portugal, 7Laboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal","locationCode":"4065","description":"\r\n\t<div>OBJECTIVES: To describe the clinical practice and estimate the costs related to the treatment of acid sphingomyelinase deficiency disease (ASMD), type B or A/B, in the Portuguese National Health Service. METHODS: A panel with four experts (pediatrics and internal medicine), from four reference centers for lysosomal storage diseases, geographically distributed, was conducted, during 2023, in order to characterize the Portuguese routine care. Experts replied individually to a structured electronic questionnaire about their experience on ASMD treatment. Responses were collected and aggregated results were produced based on arithmetic averages, according to Portuguese methodological guidelines. Resource use related to ASMD complications was based on the expert panel, Diagnosis-Related Group (DRGs) database and study SPHINGO-302. The main sources for unit costs were national legislation and official drug cost databases. RESULTS: The panel estimates that 6.1% of ASMD patients are hospitalized and 16.3% resort to emergency, per year. On average, patients go to 10.0 healthcare professionals’ visits and do 10.1 laboratory tests and 6.9 exams per year. Regarding medication, these patients use vitamin D (67.5%), flu vaccination (67.5%), antibiotics (61.3%), bronchodilators (35.8%), statins (28.8%), among others. Combining resource use and unit costs, we estimate a yearly follow-up cost of 923€ per ASMD patient. A total of 78 complications, grouped in five classes, were analyzed, and a mean cost per complication type was calculated: € 828 (respiratory), € 1,786 (spleen), € 1,283 (liver), € 3,668 (cardiovascular) and € 124 (major bleeding). CONCLUSIONS: ASMD is a rare disease, therefore, data to characterize disease treatment is scarce. This study provides some insights on patient management, including disease complications, and estimates costs associated with ASMD in Portugal.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23silvamiguelhta26poster132924-pdf.pdf?sfvrsn=46c7c615_0","title":"ISPOREurope23_SilvaMiguel_HTA26_POSTER132924.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132924","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Comparative Analysis of Missing Value Imputation Techniques: Parametric Imputation with eCDF (PIE) Vs Markov Chain Monte Carlo Imputation (MCMC)","id":"a5ad5519-454c-4285-ae86-11df59e57642","sessionCode":"MSR29","topDisplay":"Paul Choudhury S, Dutta Majumdar A, Sil A, Dutta S PharmaQuant Insights Pvt. Ltd., Kolkata, WB, India","locationCode":"5066","description":"\r\n\t<div>OBJECTIVES: Missing data is a common challenge in healthcare research, and various imputation methods have been developed to address this issue. Markov-chain Monte Carlo (MCMC) imputation is a well-accepted method for handling continuous missing data in primary studies. Our primary objective is to propose an alternative parametric imputation using empirical cumulative distribution function (eCDF) curve (PIE) and compare its performance with the standard MCMC method. METHODS: Data on diastolic blood pressure (DBP) and 2-hour serum insulin levels (SIL) for 768 patients were selected for analysis from a publicly available dataset (Smith 1988). We randomly removed 10% of the data from this primary dataset to create a missing value dataset (MVD). Different parametric distributions (Normal, Cauchy, Log-normal, Gamma, Weibull, Exponential, Pareto etc.) were fitted on MVD. Model fit was compared using Akaike’s Information Criteria (AIC) and eCDF. Each missing value was imputed with the mean of one hundred samples from the best-fitting distribution. Root-mean-square error (RMSE) was estimated to compare the accuracy of the two techniques. Sensitivity analysis for the estimated parameters of the best-fitting distribution was performed using bootstrap sampling to check for parametric uncertainty and results were summarized using RMSE. RESULTS: Log-normal distribution was the best-fitting distribution for both the scenarios. RMSEs were estimated to be lower for PIE compared to MCMC for both DBP [PIE: 7.9; MCMC (five-seeds): 9.8 to 11.9] and SIL [PIE: 108.1; MCMC (five-seeds): 196.6 to 209.5]. Bootstrapped sampling yielded RMSE ranges of 7.5 to 8.2 for DBP and 103.1 to 120.8 for SIL. The estimated RMSE to check the parametric uncertainty in PIE imputation was lower compared to that of MCMC imputation. CONCLUSIONS: The results of this comparative analysis suggest that PIE imputation outperforms MCMC imputation in terms of RMSE, indicating its potential as a viable alternative.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/eu-ispor-2303oct23final-version130469-pdf.pdf?sfvrsn=47056af0_0","title":"EU-ISPOR 23_03OCT23_Final version130469.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130469","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patient Preferences and Expectations Regarding Leukemia and Lymphoma Treatment: Improving Patient Engagement in Portugal through Quantitative and Qualitative Findings","id":"88a15064-563d-4a70-97ae-120e19f32a85","sessionCode":"PCR21","topDisplay":"Dimitrovova K1, Reis A2, Barbosa I3, Cunha L4, Ferreira MF5, Oliveira J5, Viana DS6 1MOAI Consulting, Lisbon, 11, Portugal, 2MOAI Consulting, Lisbon, Portugal, 3Associação Portuguesa de Leucemias e Linfomas (APLL), Porto, Portugal, 4Associação Portuguesa Contra a Leucemia (APCL), Lisbon, Portugal, 5Associação de Apoio aos Doentes com Leucemia e Linfoma (ADL), Maia, Portugal, 6Janssen-cilag Farmacêutica, Lda, Oeiras, Portugal","locationCode":"6001","description":"\r\n\t<div>OBJECTIVES: Quantify the level of Leukemia and Lymphoma (L&L) patient involvement in treatment decisions, patient preferences regarding the most valued criteria when choosing a treatment and ascertain the main drivers of such engagement and preferences. METHODS: An exploratory sequential mixed-methods design was implemented. The initial phase consisted of quantitative data collection through an online questionnaire, followed by focus group meetings. The involvement of L&L patients was performed with the support of three national Patient Associations. Descriptive data analysis was complemented with content analysis, and qualitative insights from the focus groups. RESULTS: 190 patients (n=75 Leukemia; n=115 Lymphoma) answered the questionnaire (October-December 2022) with 32% report wanting to be more involved in treatment decisions. Of these, 66% considered they were not involved due to “disease’s lack of knowledge” and 26% due to “lack of physicians’ openness”. Among those who did not wish to have been more involved, 36% also claimed “disease’s lack of knowledge”. The most valued criteria when choosing a treatment were: 1.Lifespan increase, 2.Duration of response, followed by 3.Duration of treatment, 3.Route of administration and 3.Adverse events (on the same preference level), 4.Frequency of administration and lastly 5.Time spent on each administration. 57%(n=107) of patients stated oral administration preference over intravenous (increasing to 91% (n=21) in CML; 80% (n=12) in CLL and 79% (n=11) in AML). 10 patients participated in 3 focus groups (June-2023). Overall, qualitative insights were consistent with the main quantitative findings. However, most participants felt fully involved in treatment choice and the desire of not being involved (mostly in 1L-treatment) was essentially due to shock after diagnosis and confidence in physicians’ decision. CONCLUSIONS: This study reinforces that L&L patient perceptions on lack of knowledge regarding their disease can influence involvement in treatment decisions, and highlights that improving health literacy may contribute to a greater patient involvement during all treatment stages.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dimitrovovapcr21poster131771-pdf.pdf?sfvrsn=d30b8a5_0","title":"ISPOREurope23_Dimitrovova_PCR21_POSTER131771.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131771","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Use of Inpatient Systemic Chemotherapy and/or Radiotherapy and Related Predictive Factors for Metastatic Urothelial Cancer (mUC): Findings from a Retrospective Observational Study in a Clinical Practice Setting in Italy","id":"7df9d503-7c78-4981-b079-12e692539eec","sessionCode":"CO20","topDisplay":"Kearney M1, Thompson A2, Kirker M2, Gharibian N2, Costa N3, Furegato M4, Pacheco C4, Issa S4, Sciattella P5, Scortichini M5, Mennini FS5 1Merck Healthcare KGaA, Darmstadt, HE, Germany, 2Pfizer, New York, NY, USA, 3Pfizer, Porto Salvo, Portugal, 4Cerner Enviza France SAS, Paris, France, 5Economic Evaluation and HTA (EEHTA-CEIS), DEF Department, Faculty of Economics, University of Rome 'Tor Vergata', Rome, Italy","locationCode":"1016","description":"\r\n\t<div>OBJECTIVES: This study describes the treatment rate and related predictive factors, demographics, and clinical characteristics of patients with mUC who received systemic chemotherapy and/or radiotherapy (or not), in the inpatient setting in Italy. METHODS: This retrospective observational analysis used hospital discharge data (SDO, Scheda di Dimissione Ospedaliera) to describe incident adult (≥18 years) patients with a first hospitalization for mUC (index) in 2017-2018, identified by a combination of International Classification of Diseases (ICD-9-CM), medical procedure, and Diagnosis Related Group (DRG) codes. A multivariable logistic regression model was fitted to identify factors associated with receiving inpatient chemotherapy and/or radiotherapy (or not). RESULTS: A total of 3,674 patients with mUC were identified, of whom 27.6% were treated with inpatient chemotherapy and/or radiotherapy, and 72.4% were with neither. In treated and untreated patients, median age at index was 71 and 78 years, 36.5% and 61.5% were ≥75 years old, and mean (SD) Charlson Comorbidity Index (CCI) score was 0.3 (0.8) and 0.6 (1.1), respectively. Primary tumor location was bladder in 87.2% of patients. Cardiovascular disease and renal function impairment were more prevalent in untreated (22.6% and 13.2%) than treated patients (16.7% and 7.8%), respectively. Older age (odds ratio [OR], 0.94 [95% CI, 0.93-0.95]), female sex (0.82 [0.68-0.99]), and higher CCI score (0.83 [0.73-0.93]) were all associated with a lower likelihood of receiving inpatient chemotherapy and/or radiotherapy. CONCLUSIONS: These real-world findings from a 2017-2018 retrospective longitudinal cohort study indicate that nearly three fourths of patients with mUC did not receive inpatient systemic chemotherapy and/or radiotherapy in Italy, driven by older age, female sex, and high comorbidity burden. Although this study provides a partial capture of inpatient systemic chemotherapy and/or radiotherapy use in Italy, the results are consistent with other European studies with similar designs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kearneyco20posterv3128667-pdf.pdf?sfvrsn=d9f3be4e_0","title":"ISPOREurope23_Kearney_CO20_POSTERV3128667.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128667","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Resource Consumption Analysis in Patients with Hemophilia A Without Inhibitors on Prophylaxis with FVIII Before Switching to Emicizumab","id":"84ef40f0-6c27-4e12-af1c-1470d9868de4","sessionCode":"EE96","topDisplay":"Giacomini E1, Leogrande M2, Motta L3, Tempre R4, Bendinelli S4, Degli Esposti L2 1CliCon S.r.l. Società Benefit Health, Economics & Outcomes Research, Bologna, Italy, 2CliCon S.r.l. Società Benefit Health, Economics & Outcomes Research, Bologna, BO, Italy, 3Roche S.p.A., Monza, MB, Italy, 4Roche S.p.A., MONZA, MB, Italy","locationCode":"2029","description":"\r\n\t<div>OBJECTIVES: The standard therapy for patients with hemophilia A is the prophylactic treatment with replacement factor VIII (FVIII), with a consumption variability depending on the patient's weight and the therapy personalization. In 2018, emicizumab become available in Italy, becoming the first therapy to offer subcutaneous prophylaxis with less frequent administration (QW, Q2W or Q4W). The aim of this analysis was to compare the prophylactic treatment with FVIII (short and long acting) in terms of consumption and direct costs versus the emicizumab theoretical cost in patients with hemophilia A without FVIII inhibitors. METHODS: A retrospective analysis was performed using administrative database from Local Health Units (~9 million inhabitants) including male patients with hemophilia A who started the treatment with emicizumab between January 2018 to September 2022. FVIII consumption was assessed over 12 months before the first prescription of emicizumab. The prophylaxis regimen was identified using a literature-validated algorithm. RESULTS: A total of 72 patients treated with emicizumab were identified; 32 patients (mean age 28.2±21.2 years, N=12 <13 years, estimated mean weight 58.7±21.2 kg) were previously on prophylaxis with FVIII short-acting (N=21, 7 of which <13 years) or long-acting (N=11, 5 of which <13 years). In the year before starting emicizumab, patients had an average annual FVIII consumption of 382,398±339,979 IU with a total average annual cost of €262.678 (VAT included) per patient. In the same period, patients had a mean number of 5.5±7.4 prescriptions for drugs not related to hemophilia and an average number of 4.2±5.1 prescriptions for specialist tests/visits. CONCLUSIONS: This preliminary analysis shows that the mean FVIII prophylaxis consumption (382,398IU) is higher than the previous mean consumption from Italian data. Considering the annual maintenance cost of emicizumab, estimated on an average patient of the same weight, the switch to emicizumab could result in a theoretical cost reduction up to approximately 20% per patient.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23mottaee96poster130077-pdf.pdf?sfvrsn=b8d603e0_0","title":"ISPOREurope23_MOTTA_EE96_POSTER130077.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130077","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical Work of the Carmel Medical Center Pharmacy Unit","id":"8864763e-6781-4485-9a96-1545f58673fe","sessionCode":"HSD26","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"4035","description":"\r\n\t<div>OBJECTIVES: Clinical work of the Carmel Medical Center pharmacy services system. The pharmacy unit at Carmel Medical Center includes l BSc, MSc, PharmD pharmacists. METHODS: Clinical consultations given by the pharmacists in the first half of 2022 were mapped. RESULTS: Clinical consultations given by the pharmacists in the first half of 2022 were mapped. We reviewed the instructions, entered the patients' Electronic health record The selected categories of intervention are medication reconciliation, anticoagulant treatment, antibiotic treatment, therapeutic doubling, adjustment of medication for patients with decreased renal function, counseling on intravenous feeding, medications during breastfeeding, adjustment of medication after bariatric surgery, hematology clinic, polypharmacy, TDM therapeutic drug monitoring. During the first median, 658 consultations were given in the departments. 24% of the consultations given were regarding the drug treatment regimen of antibiotics, 20% with anticoagulants, 16% medication reconciliation, 8% medication dose adjustment for patients with decreased renal function. The pharmacists also performed 2942 pharmacy assessments in the hospital. It was possible to see that only 66 percent of the consultations were accepted by the doctor and any changes in the medication were made. Following this data, a refresher training on medication reconciliation was carried out for the entire staff of the pharmacy unit by a clinical pharmacist. In the second quarter, following the intervention, 89 percent of the consultations were accepted by the doctors and a change of instruction was made. CONCLUSIONS: It can be seen that clinical consultations of the pharmacy system influence the medication treatment in hospitalization. The consultation is important for the improvement and the high quality of medical treatment.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-poster128134-pdf.pdf?sfvrsn=7c1ba567_0","title":"ispor poster128134.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128134","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Digging into the Medical Technology HTA Process in Tunisia","id":"5949bf55-758e-4ae3-a9f2-1573739d9cde","sessionCode":"MT13","topDisplay":"Mordin M1, Warttig S2, Gildea L2, Long J3, D'Souza V3, Kinderas M2, Ling C3, Hartley L3 1RTI Health Solutions, Oak Hill, VA, USA, 2RTI Health Solutions, Manchester, LAN, UK, 3RTI Health Solutions, Manchester, UK","locationCode":"5040","description":"\r\n\t<div>OBJECTIVES: Publicly available information on the process and methods for health technology assessment (HTA) of medical technologies (MT) can be difficult to find. Our objective was to understand the process and methods for MT HTA used by the National Authority for Evaluation and Accreditation in Health (INEAS) in Tunisia. METHODS: A review of publicly available information from the INEAS website was performed and supplemented by an online survey that was sent to and completed by INEAS. The survey requested information on the selection process, types of technologies evaluated, and types of evidence considered as part of the HTA process and timelines. Quantitative and qualitative data were obtained and collated in Excel. RESULTS: Although the INEAS website provides brief information about process and methods used, it is not clear if and how MTs are considered. The online survey revealed that INEAS can evaluate invasive and noninvasive devices, diagnostics, and digital technology such as apps or software. The selection process does not differ depending on technology type. The same external referral process is used. However, INEAS can use either a general HTA process (e.g., the same process used for assessing pharmaceuticals) or a dedicated HTA process specifically designed for assessing MTs. For the dedicated process, INEAS considers clinical and economic data as well as opinions from healthcare professionals and patients. Unpublished data are not considered. It usually takes INEAS 3 to 6 months to complete an HTA for MT. CONCLUSIONS: A major challenge for MT companies is establishing whether a technology requires or is eligible for HTA in different markets. In Tunisia, information about this was not explicitly clear on the INEAS website. MT companies should be prepared to contact HTA agencies directly to obtain necessary information to inform market access strategies and HTA submission plans.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23mordinmt13poster130177-pdf.pdf?sfvrsn=7ff255dc_0","title":"ISPOREurope23_Mordin_MT13_Poster130177.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130177","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Liquid Metformin for Pediatric Patients with Metabolic Syndrome at Risk of Developing DMT2: Cost-Effectiveness Analysis in Mexican Public Health Institutions","id":"f0e592fe-ed71-4cf2-979e-1579eb88b24b","sessionCode":"EE87","topDisplay":"Gay Molina J1, Gonzalez Canudas J2, Castillo V1, Romero Y2, Valls M1 1T.I. Salud, Miguel Hidalgo, Mexico, 2Laboratorio Silanes, Mexico City, DF, Mexico","locationCode":"2023","description":"\r\n\t<div>OBJECTIVES: This study aims to present an economic evaluation of using liquid metformin for pediatric patients with metabolic syndrome at risk of developing DM2 within the context of Mexican public health institutions. METHODS: First, we modeled the effectiveness of the analyzed alternatives using the incidence rates of DM2 in each treatment group; the first group received lifestyle change recommendations, while the second group received metformin in addition to the above. Then, we incorporated these results into a Markov chain analysis over a 4-year time horizon for each treatment group. For sensitivity analysis, we conducted a probabilistic analysis to assess our evaluation in various uncertainty scenarios. RESULTS: We found that liquid metformin use is associated with 0.24 additional years without developing DM2 and incremental savings of $20,457.01, resulting in a dominant intervention compared to the untreated control. The probabilistic sensitivity analysis revealed that 100% of iterations favored liquid metformin over the untreated control, indicating that it not only has superior effectiveness in terms of DM2-free years but also generates significant incremental savings. This finding maintains liquid metformin's dominance over no-treatment alternatives. CONCLUSIONS: Preventive intervention using metformin was found to be a cost-saving and effective strategy for managing type 2 diabetes risk. Liquid metformin increases type 2 diabetes-free life years for pediatric patients, directly contributing to improved life expectancy. From the perspective of public health institutions and decision-makers, this intervention saves more money than preventive conferences or standard lifestyle advice in a population at risk of developing the disease.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129006","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Review of the New NICE Highly Specialised Technologies Criteria: How Do NICE Judge Criteria 3 and 4 to be Met?","id":"838e9f0e-8a17-4b2f-bd24-158d28ae4e38","sessionCode":"HTA62","topDisplay":"Berkley A1, Campbell-Hill S2 1Takeda Pharmaceuticals UK, London, LON, UK, 2Takeda Pharmaceuticals UK, London, London, UK","locationCode":"5001","description":"\r\n\t<div>OBJECTIVES: Since February 2022, eligibility for the National Institute of Health and Care Excellence (NICE) Highly Specialized Technologies (HST) program has been based on four revised criteria. Criteria 1 and 2 are based on the rarity of the treated condition and are quantitatively defined, whereas Criterion 3 (significantly shortens life/severely impairs quality of life [QoL]) and Criterion 4 (no satisfactory treatments/significant benefit over existing treatments) require judgement. We investigated how NICE judge Criteria 3 and 4 to be met. METHODS: A targeted literature review was conducted to assess previous research into this topic, finding no published research that analyzed the outcome of applications using the new HST criteria. NICE ‘guidance in development’ and HST committee meeting minutes published from 01/02/2022 to 03/03/2023 (date of search) were systematically reviewed to identify all published HST checklists; these summarize whether a technology meets each criterion and why. RESULTS: Ten checklists were identified; of these, six and three met Criteria 3 and 4, respectively. A median life expectancy of 66.5 years and a 20-year survival rate of 75% were both sufficient to meet Criterion 3 based on life expectancy and cholestatic pruritus (itching) was sufficient to demonstrate a severe impairment on QoL. Criterion 4 was only met based on lack of treatment options, rather than significant benefit over existing therapies. If alternative treatments exist, evidence of a statistically significant efficacy benefit over standard of care seems to be required (improved convenience/safety is insufficient). Lack of adequate treatments must also apply to a significant proportion of the patient population. CONCLUSIONS: Criterion 3 can be met with a median life expectancy of 66.5 years or 20-year survival rate of 75%. Criterion 4 is the most difficult to meet, with high thresholds for evidence given the rarity of the conditions involved.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23berkleyhta62poster129631-pdf.pdf?sfvrsn=fe5aa90a_0","title":"ISPOREurope23_Berkley_HTA62_POSTER129631.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129631","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Budget Impact Analysis of the Introduction of Dexcom ONE for the Treatment of Patients With T2 Diabetes on an Intensive Insulin Schedule From the Perspective of the Italian Healthcare Payer","id":"8931c4e0-43ca-4f2b-8d1b-1608b30c8634","sessionCode":"EE16","topDisplay":"Genugten M1, Grifi M2 1Dexcom International Ltd, San Diego, CA, USA, 2Dexcom International Ltd, Morges, Switzerland","locationCode":"1061","description":"\r\n\t<div>OBJECTIVES: Studies have shown the value of continuous glucose monitoring (CGM) systems in the treatment of patients with T2 diabetes on insulin. However, no studies are available directly comparing real-time CGM with Flash CGM (rt-CGM and FGM, respectively). The objective of this research is to compare the cost of the new Dexcom ONE rt-CGM and the currently used Free Style Libre 2 (FSL2) FGM in the diabetes type 2 population on an intensive insulin schedule from the perspective of the Italian healthcare payer. METHODS: A budget impact model was developed to estimate the cost difference between Dexcom ONE and FSL2 through an indirect comparison via self-monitoring of blood glucose (SMBG). Clinical data on HbA1c reduction and reductions in acute hospitalisations for severe hypoglycemic events and diabetes ketoacidosis with Dexcom ONE and FGM versus SMBG, were derived from a targeted review of available literature. Both RCT and real-world data were included. Baseline rates for acute hospitalisations were drawn from epidemiological literature on Italy. Healthcare costs including cost of strips, sensors and acute hospitalisations were also derived from Italian sources. One-way sensitivity analysis varying HbA1c reduction, acute hospitalization rates and costs were performed to investigate the robustness of the analysis. RESULTS: From the perspective of the Italian healthcare payer, the cost for the acquisition of Dexcom ONE (EUR 332 per patient per year) was offset by savings in HbA1c-related costs, such as hospitalizations, doctor visits and additional medication use (EUR 529 per patient per year). This resulted in net savings of EUR 197 per patient per year with Dexcom ONE compared to FSL2. The one-way sensitivity analysis showed the robustness of the results in favor of Dexcom ONE. CONCLUSIONS: Dexcom ONE is a potential cost saving strategy for the treatment of patients with T2 diabetes on an intensive insulin schedule in Italy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23griffi-ee16posterv2132805-pdf.pdf?sfvrsn=dd1f7d56_0","title":"ISPOREurope23_Griffi_ EE16_POSTERV2132805.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132805","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Perianal Fistulas on Crohn’s Disease Patients","id":"9f5ddb10-7682-4b74-9fc4-164f1b35de3d","sessionCode":"PCR6","topDisplay":"Sampaio A1, Cruz C1, Reis A2, Dimitrovova K2, Dias A2 1APDI - Associação Portuguesa da Doença Inflamatória do Intestino, Colite Ulcerosa e Doença de Crohn, Matosinhos, Portugal, 2MOAI Consulting, Lisbon, Portugal","locationCode":"6000","description":"\r\n\t<div>OBJECTIVES: This study aimed to characterize perianal fistulas (PAF) in patients with Crohn’s Disease (CD) as well as their impact on patients’ life, through the evaluation of the personal, familiar, marital, social, and professional domains. METHODS: A structured questionnaire was implemented, targeting CD patients with and without PAF, to compare the main differences due to PAF presence. Classification levels scored from 1 (no impact) to 10 (major impact) were used to assess the global domains’ impact on patient’s life. The anonymized data were self-reported, collected through an online platform, and shared by the Portuguese Association of Inflammatory Bowel Disease (APDI). RESULTS: A total of 193 CD patients answered the questionnaire between May and July of 2022. The mean age was of 47 years, 56% were female, and 27% reported moderate to severe symptoms. 90 CD patients reported a presence of PAF related to CD, of which 41% were diagnosed with complex PAF and 9% reported having rectovaginal fistulas. Over time, the most felt symptoms related to PAF were pain (83%), anal area swelling (62%), and itch (59%). CD patients with PAF revealed higher levels of impact (score 8 to 10) in their life when compared with CD patients without PAF. On a personal level, CD patients with PAF feel more uncomfortable and less understood, despite highlighting a more communicative attitude about the disease with friends and family. Although no significant differences were identified in the marital dimension, 42% of CD patients with PAF evidenced sexual restrictions compared to 32% without PAF. Regarding the professional dimension, higher levels of impact were reported by CD patients with PAF (24% vs 10%). CONCLUSIONS: This study reveals how the presence of PAF in CD patients can impact their life, from specific symptoms and complications to daily life restrictions, such as the need of changing jobs or personal relationships.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129520","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Genetic-Guided Pharmacotherapy for Cardiovascular Disease: A Systematic and Critical Review of Economic Evaluations","id":"5d3d6c9a-6024-44d0-b4d7-16d57d1d8916","sessionCode":"EE131","topDisplay":"Lim KK1, Koleva-Kolarova R2, Kamaruzaman HF3, Laurence Y4, Kamil AA1, Chowienczyk P1, Wolfe C1, Fox-Rushby J1 1King's College London, London, UK, 2University of Oxford, Oxford, OXF, UK, 3University of Glasgow, Glasgow, UK, 4King's College London, London, LON, UK","locationCode":"1049","description":"\r\n\t<div>OBJECTIVES: Genetic-guided pharmacotherapy (PGx) is not recommended in clinical guidelines for patients at risk of or diagnosed with cardiovascular diseases (CVD). We examine the extent and quality of evidence from economic evaluations (EEs) of PGx in CVD, and the associations between PGx characteristics and findings. METHODS: From systematic searches across six databases, two independent reviewers screened, extracted data, and rated the methodological quality of EEs. We described the type of CVDs, country, purpose of testing, comparator, type of EE, model design and findings. RESULTS: The 92 papers included examined PGx for coronary artery disease (n=35), atrial fibrillation (n=18), familial hypercholesterolemia (n=18), venous thromboembolism (n=10), and others (n=11), largely from North America (n=42) or Europe (n=34). Most EEs were model-based cost-utility/effectiveness analyses comparing PGx testing to; select the best pharmacotherapy (n=40), select the best dose (n=20), detect CVD early (n=16), or predict the disease prognosis (n=12), against no testing (n=89). Of 362 comparisons reporting quality-adjusted life years, 256 found a positive INMB (199 cost-effective, 60 dominant) and 98 negative INMB (76 not cost-effective, 22 dominated) for PGx. Most accounted for the effect of PGx using relative risk reduction (n=41), lower probabilities of adverse events (n=23), time within treatment biomarker’s therapeutic range (n=15) or test accuracy (n=13). Of 783 variables tested in one-way sensitivity analyses, 154 changed conclusions; these were epidemiological variables (n=74), cost/unit price/resource use (n=29), effectiveness/relative effectiveness (n=35), or utility (n=13). CONCLUSIONS: Economic evaluation have included a range of CVDs, settings, purposes of PGx and designs. We will discuss our findings with respect to data collection and model structures, PGx characteristics associated with value for money, challenges to data and methods of analysis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee131-ispor-europe-2023-kk-lim128713-pdf.pdf?sfvrsn=ee7d066d_0","title":"EE131 ISPOR Europe 2023 KK Lim128713.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128713","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Prevalence, Risk Factors, and Characterization of Individuals with Long COVID Using Electronic Health Records in Over 1.5 Million COVID Cases in England","id":"d2b070f0-00d9-4fe5-bd09-16ef47550d80","sessionCode":"EPH11","topDisplay":"Wang HI1, Doran T1, Crooks M2, Khunti K3, Heightman M4, gonzalez-Izquierdo A5, Arfeen M5, Banerjee A5, van der Feltz - Cornelis C1 1University of York, York, YOR, UK, 2Hull York Medical School, York, North Yorkshire, UK, 3University of Leicester, Leicester, Leicester, UK, 4University College London Hospitals NHS Foundation Trust, London, London, UK, 5University College London, London, London, UK","locationCode":"3018","description":"\r\n\t<div>OBJECTIVES: Long-COVID has a significant and long-lasting impact on individuals' health and well-being. This study uses electronic health records in England to describe the prevalence of long-COVID symptoms among individuals seeking medical attention and explores the risk factors for having long-COVID. METHODS: This is a population-based longitudinal cohort study using linked primary care data derived from the Clinical Practice Research Datalink (CPRD) for 1,554,040 individuals with confirmed SARS-CoV-2 infection. Descriptive statistics were used to explore the prevalence of long-COVID diagnosis and symptoms after 12-weeks from index date and Cox regression models for risk factors for having long-COVID. Sensitivity analysis was conducted to test the impact of right-censoring data. This study is funded by NIHR (COV-LT2-0043) as part of the Symptoms, Trajectory, Inequalities and Management: Understanding Long-COVID to Address and Transform Existing Integrated Care Pathways (STIMULATE-ICP) study. RESULTS: Over average 400 days follow-up, 7.4% of those requiring medical attention following acute COVID-19 had at least one long-COVID symptom. However, only 0.5% had long-COVID diagnostic codes recorded in the electronic medical record. The most frequently recorded long-COVID symptoms were cough (17.7%), back pain (15.2%), stomach ache (11.2%), headache (11.1%), and sore throat (10.0%). Risk factors associated with long-COVID were: female sex (HR: 1.171, 95% CI: 1.156 to 1.186), non-white ethnicity (HR: 1.145, 95% CI: 1.130 to 1.161), obesity (HR: 1.100, 95% CI: 1.085 to 1.116), and pre-existing medical conditions such as anxiety, depression, type II diabetes, and somatic symptom disorders. CONCLUSIONS: Our study is the first to investigate prevalence of, and risk factors for, clinically confirmed long-COVID symptoms in the general population using electronic health records. These findings could help clinicians identify higher risk individuals for timely intervention and allow decision-makers to more efficiently allocate resources for managing long-COVID.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterstimulate129389-pdf.pdf?sfvrsn=f2de1ae_0","title":"Poster_STIMULATE129389.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129389","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Endometrial Cancer First Line Treatment in Argentina: ECHOS-A Real-World Study","id":"223462aa-858e-4677-b49e-17239c7b9836","sessionCode":"RWD14","topDisplay":"Abreu G1, Queiroz J1, Nogueira da Silva TL1, Soares C1, Menezes P1, Felice R2, Carrizo M2, Scibona P3, Simonovich VA3, Riggi MC3, Saadi J J3, Cravero F3, Jotimliansky L2 1GSK, Rio de Janeiro, Brazil, 2GSK, Buenos Aires, Argentina, 3Hospital Italiano de Buenos Aires, Buenos Aires, Argentina","locationCode":"6066","description":"\r\n\t<div>OBJECTIVES: Endometrial Cancer Health Outcomes Study (ECHOS) is a multicountry real-world data study evaluating the treatment practices and health outcomes in patients with endometrial cancer (EC) in Argentina, Brazil, and Colombia. This analysis describes clinical characteristics and treatment patterns in patients from Argentina (ECHOS-A), focusing on first line (1L) antineoplastic therapy use. METHODS: A retrospective cohort study using electronic medical records from patients with EC affiliated to a private health insurance plan (Hospital Italiano de Buenos Aires) receiving antineoplastic therapy between 01/2010 and 12/2019. Index (proxy for diagnosis) was the first date of an EC-related health term or procedure/treatment. Patients were classified as treated when exposed to surgery, radiotherapy, or antineoplastic therapy (hormone therapy [HT], chemotherapy [CT], or immunotherapy [IT]). Proportions of patients receiving systemic therapies in the 1L (dispensed within the initial 30-day cycle) and in each year of follow-up were calculated. International Federation of Gynecology and Obstetrics (FIGO) staging was evaluated among patients receiving treatment. RESULTS: In the observation period, 805 patients received an EC diagnosis and 623 (77.4%) were treated. Among the 544 treated patients with a FIGO staging classification, 101 (18.6%) were considered advanced (III and IV), of whom only 63 (62.4%) received systemic therapy. 198 patients (31.8%) received 1L systemic therapy (CT, 82.8%; HT, 17.7%; IT, 1.0%), 81.3% within the first year. Platinum-based CT was the most frequent 1L scheme, received by 146 patients (73.7%), of which, 106 patients received the carboplatin-paclitaxel combination. CONCLUSIONS: Among patients with EC treated with antineoplastic drugs, the majority received carboplatin and paclitaxel. However, around one-fifth received no treatment and over one-third of advanced cases were not treated with antineoplastic drugs. Efforts to better understand and address the reasons behind this are needed to help improve outcomes in the future. Funding: GSK (217348) </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23abreurwd14poster129983-pdf.pdf?sfvrsn=9207daf_0","title":"ISPOREurope23_Abreu_RWD14_POSTER129983.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129983","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Opioid Tapering and Its Associations with Risk of Opioid Use Disorder and Mortality Among Older Adults","id":"a7a85ba3-ebf2-4616-a9bc-174e5ac9553b","sessionCode":"PT10","topDisplay":"Yang Y1, Prajapati P2, Ramachandran S3, Bhattacharya K3, Maharjan S2, Eriator I4, Bazzazzadehgan S1, Bentley J3 1Department of Pharmacy Administration, University of Mississippi School of Pharmacy, University, MS, USA, 2Department of Pharmacy Administration, University of Mississippi School of Pharmacy, Oxford, MS, USA, 3Department of Pharmacy Administration, University of Mississippi School of Pharmacy, Center for Pharmaceutical Marketing and Management, University of Mississippi School of Pharmacy, University, MS, USA, 4University of Mississippi Medical Center, Jackson, MS, USA","locationCode":"1B","description":"\r\n\t<div>OBJECTIVES: Opioid tapering has increased in recent years, however, evidence regarding its safety profile is lacking. The purpose of this study was to examine the relationships between opioid tapering and subsequent opioid use disorder (OUD) and all-cause mortality among older adults on long-term opioid therapy (LTOT). METHODS: This study used a nested case-control design. A cohort of older (≥65 years) Medicare beneficiaries with chronic non-cancer pain who were on LTOT was identified from 2012-2018 5% national Medicare claims data. Within this LTOT cohort, cases (beneficiaries who experienced either an OUD or mortality) and controls (identified using incidence density sampling) were identified and matched 1:2 on age (±1 year) and time of cohort entry (±30 days). Opioid tapering was operationalized as a monthly dose change percentage with four levels: steady dose (±10% dose change), tapering (10-40% dose reduction), rapid tapering (> 40% dose reduction), and dose escalation (>10% dose increase). Conditional logistic regression was conducted on the matched samples to evaluate the associations between opioid tapering and OUD and mortality. RESULTS: A cohort of 42,091 patients met our inclusion criteria. Among them, 2,670 cases of OUD and 4,614 cases of mortality were identified. After controlling for patient socio-demographics and clinical characteristics, compared with steady dose, the odds of OUD were significantly lower (aOR=0.52; 95%CI=0.42-0.64) for rapid tapering and significantly higher (aOR=1.58; 95%CI=1.31-1.90) for dose escalation. Compared to steady dose, significantly higher odds for all-cause mortality were found among patients undergoing tapering (aOR=1.64, 95%CI=1.44-1.86), rapid tapering (aOR=2.33; 95%CI=2.10-2.59), and dose escalation (aOR=2.21, 95%CI=1.95-2.52). CONCLUSIONS: The results suggest that opioid tapering was associated with increased risk of all-cause mortality but not OUD; opioid dose escalation was associated with both OUD and mortality. Clinicians should evaluate patients on LTOT to weigh the benefits and risks of treatment that incorporates evolving evidence surrounding dose changes.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23yang-pt10poster131186-pdf.pdf?sfvrsn=ba163195_0","title":"ISPOREurope23_Yang-PT10_POSTER131186.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131186","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Expanding the HTA Cost-Effectiveness Analyses for CheckMate 9LA: Nivolumab Plus Ipilimumab Plus Chemotherapy As First-Line Strategy for Non-Small Cell Lung Cancer","id":"3b662092-ce76-46fe-861f-179e203c8a3f","sessionCode":"HTA14","topDisplay":"Orsini I1, Venkatachalam M1, Yuan Y2, Lee A3, Penrod JR4 1PRECISIONheor, London, LON, UK, 2Bristol Myers Squibb, Plainsboro, NJ, USA, 3Bristol Myers Squibb, Uxbridge, LON, UK, 4Bristol Myers Squibb, Princeton, NJ, USA","locationCode":"4058","description":"\r\n\t<div>OBJECTIVES: The evidence required by health technology appraisal (HTA) agencies to assess the cost-effectiveness of new therapies is often limited to quality-adjusted survival and direct costs. The inclusion of additional elements of value meaningful to patients is often excluded by HTAs. A limited number of studies have been conducted to assess the impact of including novel value elements in HTA cost-effectiveness analyses (CEAs). METHODS: To evaluate the impact of including novel value elements in a United States (US) HTA-compliant CEA developed for CheckMate 9LA (24-month minimum follow-up). CheckMate 9LA compared nivolumab plus ipilimumab plus 2 cycles of platinum doublet chemotherapy (N+I+PDC) versus 4 cycles of PDC in first-line metastatic non-small cell lung cancer (mNSCLC). The CEA was adapted to include patients’ and caregivers’ indirect costs, patients’ hope, option value, and insurance value. These novel value elements were quantified after having identified mNSCLC-specific studies. The analysis compared the net monetary benefit (NMB) of N+I+PDC versus PDC in a traditional payer, traditional societal, and broad societal setting. RESULTS: N+I+PDC resulted in higher costs and quality-adjusted life-years (QALYs) compared with PDC performing a traditional payer perspective CEA ($190,281 incremental costs, 1.23 incremental QALYs). The NMB was -$6,232 at a willingness-to-pay threshold of $150,000/QALY, suggesting that costs exceed benefits. A slight increase in incremental costs was observed after expanding the CEA to a traditional societal perspective ($190,281 versus $190,292). A significant improvement in incremental QALY was observed (1.23 versus 2.71) after further expanding the CEA to a broad societal perspective. This perspective, resulting in +$210,834 NMB, indicated that N+I+PDC benefits outweighed its costs. CONCLUSIONS: HTA-compliant CEAs for N+I+PDC in first-line mNSCLC have previously demonstrated acceptable value for money in the US. This study showed that novel value elements provide a more favorable and complete value assessment and should therefore be considered by HTAs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23orsinihta14poster-submitted132525-pdf.pdf?sfvrsn=5951b1b1_0","title":"ISPOREurope23_Orsini_HTA14_Poster Submitted132525.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132525","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Humanistic Burden in Relapsed or Refractory (R/R) Follicular Lymphoma (FL): A Systematic Literature Review (SLR)","id":"ee729ce0-9ceb-4abc-ba65-18aaaafc2772","sessionCode":"PCR18","topDisplay":"Forbes C1, Pustulka I2, Nunez-Gonzalez S3, Kumar J4 1Evidera PPD, London, UK, 2Evidera PPD, Warsaw, Poland, 3Evidera PPD, Waltham, MA, USA, 4Bristol Myers Squibb, Princeton, NJ, USA","locationCode":"6023","description":"\r\n\t<div>OBJECTIVES: To conduct an SLR on humanistic burden, focusing on health-related quality of life (HRQOL), patient-reported outcomes (PRO), and utility/disutility values in patients with R/R FL. METHODS: The SLR followed Cochrane methodologies to identify evidence on HRQOL, PROs, and utility/disutility values. Literature searches were conducted in September 2022 (limited to 2012‒2022) in MEDLINE, Embase, the Cochrane Library (CENTRAL), and PsycInfo. Only English-language publications were included. RESULTS: Nineteen publications met the inclusion criteria as follows: 9 economic models, 7 health technology assessment (HTA) reports, 2 clinical trials, and 1 observational study. Four publications (2 in the R/R population and 2 in the third-line or later [3L+] setting) reported on HRQOL/PROs and 15 publications (12 in the R/R population and 3 in the 3L+ setting) reported on utility/disutility values. In all 4 HRQOL/PRO publications, the primary instrument used was the Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym), with 1 study also using the 36-Item Short Form Survey (SF-36). In the observational study, patients with active relapsed FL demonstrated lower mean total FACT-Lym scores, indicating a poorer HRQOL compared with patients who achieved partial or complete response and those classified as disease free. In clinical trials conducted in the R/R population, the use of bendamustine plus obinutuzumab and tisagenlecleucel resulted in approximately 40%‒49% of patients experiencing a clinically meaningful improvement in HRQOL based on their FACT-Lym and/or SF-36 scores. The majority of economic models and HTAs relied on a single primary source (Wild et al. Value in Health 2006) for utility values, which were subsequently adjusted for the specific characteristics of each model and the population evaluated. CONCLUSIONS: Data on HRQOL and utility/disutility values relevant to 3L+ treatment in R/R FL are scarce. Limited data are available for each subsequent line of therapy beyond the third line, highlighting the need for further research to inform future HTA submissions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23forbespcr18posterv2131280-pdf.pdf?sfvrsn=c0a703b9_0","title":"ISPOREurope23_Forbes_PCR18_POSTERV2131280.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131280","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Use of Copilot, a Generative Artificial Intelligence Tool, as VBA Programming Assistant in Excel-Based Health Economic Models","id":"140effa8-2e87-47cd-800b-18b5f309384d","sessionCode":"MSR26","topDisplay":"Poirrier JE1, Bergemann R2 1Parexel International, Wavre, WBR, Belgium, 2Parexel International, Loerrach, Germany","locationCode":"5070","description":"\r\n\t<div>OBJECTIVES: This study aims 1) to test how health economists can take advantage of Copilot, a GPT-3-based artificial intelligence tool developed by GitHub and OpenAI, in their cost-effectiveness model development in MS Excel/VBA and 2) to draw attention on some non-programming issues related to the use of AI in programming. METHODS: An existing CEM for the treatment of Urinary Tract Infection in hospitalized patients was stripped from its VBA code for some interface interactions, for the creation of the cost-effectiveness (CE) frontier and for the Probabilistic Sensitivity Analysis (PSA). Copilot was then prompted to rewrite the code from scratch, first with general prompts, then with step-by-step requests. The generated code was analyzed by an experienced VBA developer to assess quality, verboseness, and performance. RESULTS: General prompts failed at generating any effective VBA code. Deconstructed algorithms for the CE frontier and the PSA generated effective working code. Performances (runtime) and quality were similar to code generated by an experienced developer. CONCLUSIONS: This study showed it is feasible to use Copilot to generate VBA code in a CEM. However, the quality and verboseness of the generated code is depending on the user’s detailed requests. The CEM-specific user interface built in sheets contributes to difficulties in generating usable VBA code with Copilot. Copilot will probably accelerate model development for junior developers. But more experienced programmers will need to wait for improvements from GPT-4 and an increased VBA codex. Further research and development of more intelligent GAI algorithms is therefore necessary for the introduction on daily routine. More broadly, potential issues of code explainability, ownership and licensing need to be clarified.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23poirriermsr26poster133745-pdf.pdf?sfvrsn=c94872c8_0","title":"ISPOREurope23_Poirrier_MSR26_POSTER133745.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133745","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost of Illness Study of Migraine in Bulgarian Patients","id":"62e51fde-cb68-47d9-9247-18cd8a886b58","sessionCode":"EE47","topDisplay":"Dacheva A1, Slavchev G2, Vutova Y3, Djambazov S4 1Medtronic International Trading Sàrl, Sofia, 23, Bulgaria, 2Medtronic International Trading Sàrl, Sofia, 22, Bulgaria, 3HTA Ltd., Sofia, 22, Bulgaria, 4Medical University Pleven, Sofia, 23, Bulgaria","locationCode":"1038","description":"\r\n\t<div>OBJECTIVES: Migraine is an idiopathic chronic disease characterized by episodes of moderate to severe headache. Migraine is a common cause of disability and loss of work capacity. The analysis aimed to estimate the social costs and health losses associated with Migraine patients in Bulgaria. Currently, ICD code of Migraine is not included in the List of diseases paid by the NHIF in Bulgaria. METHODS: The costs of illness were estimated using a prevalence-based approach. The analysis aims to cover all costs related to the control of Migraine from the perspective of the payer (NHIF) and society. The analysis examines direct medical costs primarily associated with pharmacotherapy and medical care to manage the disease and indirect costs associated with lost productivity. The time horizon of the analysis is one year. The main sources of information were hospitals database; expert opinions of KoLs in Bulgaria; medicine consumption and price data. RESULTS: The results show that the direct costs per patient amount to BGN 848.29 per year, and the indirect costs amount to BGN 1 386.50 per year. The result of the economic evaluation shows that the direct medical costs for the migraine-affected population amount to BGN 165,620,297.38, of which BGN 138,999,298.02 are for pharmacotherapy paid by the patients. The cost of medical activities amounted to BGN 26 620 999,36. Indirect costs are estimated at BGN 270 700 517,89 (BGN 151 283 810,52 as a result of absenteeism and BGN 119 416 707,37 as a result of presenteeism (60% impaired performance). CONCLUSIONS: The current analysis recommends the inclusion of the disease with the relevant code of the ICD-10 in the list of diagnoses for which the NHIF pays for treatment. The inclusion of Migraine in local pharmacotherapeutic guidelines will enable physicians to conduct treatment consistent with international clinical practice.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dachevaee47poster128975-pdf.pdf?sfvrsn=141a823a_0","title":"ISPOREurope23_Dacheva_EE47_POSTER128975.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128975","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Analysis of Managing Brain Metastases in Anaplastic Lymphoma Kinase Positive Non-Small Cell Lung Cancer in Greece","id":"80c06841-2014-4868-bd0b-18ea3a5b414b","sessionCode":"EE75","topDisplay":"Zisimopoulou O1, Liavas A1, Tzanetakos C2, Gourzoulidis G3 1Pfizer Hellas, Athens, Greece, 2Health Through Evidence, Athens, Greece, 3Health Through Evidence, Athens, A1, Greece","locationCode":"1043","description":"\r\n\t<div>OBJECTIVES: Patients with anaplastic lymphoma kinase-positive (ALK+) advanced non-small cell lung cancer (aNSCLC) are at high risk of developing brain metastases (BM), thus entailing significant clinical and economic burden. The aim of this study was to estimate the annual cost of managing aNSCLC ALK+ patients with and without BM and compare the relevant costs of BM patients treated with alectinib or lorlatinib in Greece. METHODS: An excel-based model with one-year time horizon was adapted to evaluate the management cost of ALK+ aNSCLC patients with and without BM. A proportion of patients develops BM based on cumulative annual incidence of BM extracted from the ALEX trial for alectinib and the CROWN trial for lorlatinib. Resource utilization of patients with and without BM included diagnostic/laboratory tests, medical visits, hospitalizations, and medical procedures associated with BM treatment, as sourced from a published study. Direct medical costs (€,2023) were extracted from official publicly available Greek sources. RESULTS: An annual management cost of €936.85 per patient was estimated for ALK+ aNSCLC patients without BM, and €2,904.68 per patient for those with BM. The presence of BM was associated with an annual cost increase of €1,967.82 per patient compared to non-BM, due to increased monitoring and resource utilization. Moreover, treatment with lorlatinib was associated with an annual management cost reduction of €130 per patient compared to alectinib due to lower BM incidence. CONCLUSIONS: Analysis results link lorlatinib’s lower 12-month cumulative incidence of progression of BM with lower healthcare resource utilization compared to alectinib and therefore translates to cost-savings from payer’s perspective. As BM are common in ALK+ aNSCLC, there is a crucial need for treatments that have protective effect against BM development and delay CNS progression. Additionally, these treatments may offer benefits for the healthcare systems since management cost of ALK+ aNSCLC patients is higher when BM are present.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/cost-analysis-of-managing-brain-metastases--ispor-2023261023130192-pdf.pdf?sfvrsn=baefba00_0","title":"Cost analysis of managing brain metastases _ ISPOR 2023_261023130192.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130192","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Oral Semaglutide as the Second-Line and Third-Line Treatment for Type 2 Diabetes Patient","id":"6d574338-0c4c-411f-bdbf-1b5942b78af9","sessionCode":"EE15","topDisplay":"Tan ECH1, Yang MC2 1Department of Health Services Administration, China Medical University, Taichung, TXG, Taiwan, 2National Taiwan University, Taipei, TPE, Taiwan","locationCode":"1068","description":"\r\n\t<div>OBJECTIVES: The clinical goal in the treatment of T2D is to achieve reasonable glycemia control with minimal hypoglycemia and other side effects to avoid micro- and macrovascular complications. The study aimed to assess the long-term cost-effectiveness of oral semaglutide for the treatment of patients with T2D with inadequate glycemic control on metformin. METHODS: The intervention is oral semaglutide and the comparators were empagliflozin, sitagliptin, liraglutide, and dulaglutide in different settings of T2D patients with HbA1c >8.5% and BMI >27kg/m2 and for patients with high CV risk. The healthcare payer perspective in Taiwan was used in study. Cost-effectiveness analyses were performed using the IQVIA CORE Diabetes Model. The baseline characteristics of different cohorts were obtained from real-world data. The cycle length of 1-year and a lifetime horizon was used. The annual discount rate is 3.0%. Treatment effect data were taken from the PIONEER trials. The direct medical cost of diabetes-related complications and adverse event costs were obtained from the National Health Insurance claims data. Utilities and disutilities of complications and adverse events were obtained from published studies. RESULTS: Compared with empagliflozin, sitagliptin, liraglutide, and dulaglutide, the ICER per QALY gained of oral semaglutide were US$36,939.3, US$74,084.5, US$17,491.6, and US$10,775.3, respectively. When using 3 times GDP per capita in 2022 as the willingness-to-pay threshold, the probability of oral semaglutide being cost-effective were 85.8% (vs. empagliflozin), 56.2% (vs. sitagliptin), 80.8% (vs. liraglutide), and 56.2% (vs. dulaglutide). Compared with liraglutide and dulaglutide, the ICER per QALY gained of oral semaglutide were US$22,779.1 and US$39,274.7. The probability of oral semaglutide being cost-effective was 69.3% (vs. liraglutide) and 52.9% (vs. dulaglutide) for patient with high CV risk. CONCLUSIONS: Oral semaglutide has the potential to be a cost-effective option for the second-line treatment of T2D patients with obesity and uncontrolled glycemia and with major CV event.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023elise-90cm120cm131423-pdf.pdf?sfvrsn=99289755_0","title":"ISPOR EU 2023_Elise 90cm120cm131423.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131423","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Insights Into NICE Technology Appraisal Outcomes in Oncology","id":"86457f3c-5ae2-4fdb-80ea-1b775e91b68d","sessionCode":"HTA19","topDisplay":"Champsi S1, Howard D2, Ali A3, Sus J3 1Amgen Ltd, London, UK, 2Amgen Ltd, Uxbridge, LON, UK, 3Amgen Ltd, Uxbridge, England, UK","locationCode":"4048","description":"\r\n\t<div>OBJECTIVES: In January 2021, the Medicines and Healthcare products Regulatory Agency (MHRA) introduced the Innovative Licensing Access Pathway (ILAP), and in January 2022 the National Institute for Health and Care Excellence (NICE) replaced the end of life (EOL) criteria with a severity-based decision modifier. This analysis aims to describe the outcomes of NICE oncology single technology appraisals (STAs), including those entering Cancer Drug Fund (CDF) managed entry agreements (MEAs), following recent changes in regulatory and reimbursement processes. METHODS: All oncology drug STAs from January 2021 to May 2023 were identified, and key information relating to reimbursement processes and decisions were extracted, including ILAP status, NICE recommendations, and EOL criteria. Severity modifier was estimated for appraisals where standard of care quality-adjusted life years (QALYs) were unredacted. RESULTS: One hundred ten published appraisals were reviewed. Sixty-five appraisals were recommended for routine commissioning (of which 18 exited the CDF) and 13 appraisals were recommended via the CDF. Seven appraisals were not recommended, one of which was previously recommended via the CDF, and 25 appraisals were terminated. Amongst appraisals that entered into CDF MEAs, the most frequently cited clinical uncertainties related to maturity of trial data, followed by issues with uncertainties around subsequent treatments. We identified 10 ILAP therapies, of which 4 entered the CDF, 4 were recommended, 1 was not recommended and 1 was terminated. Severity modifier was estimated for 16 appraisals. CONCLUSIONS: The full impact of changes to the UK regulatory and reimbursement landscape remains uncertain. Redactions in appraisals make it difficult to ascertain near-term implications of ILAP and NICE methods update, as ILAP status was not readily available for terminated appraisals and severity modifiers were not estimable for most appraisals. It therefore remains unclear whether drugs that entered CDF with EOL criteria would also be exiting with a similar threshold.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23champsihta19poster132889-pdf.pdf?sfvrsn=3b91082f_0","title":"ISPOREurope23_Champsi_HTA19_POSTER132889.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132889","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Implementing a Value-Based Payment Model for Patients with Osteoarthritis","id":"13d5826e-1c48-4bf7-87e8-1be3622cd78f","sessionCode":"HPR5","topDisplay":"Vutova Y1, Djambazov S2, Dacheva A3, Slavchev G4 1HTA Ltd., Sofia, 22, Bulgaria, 2Medical University Pleven, Sofia, 23, Bulgaria, 3Medtronic International Trading Sàrl, Sofia, 23, Bulgaria, 4Medtronic International Trading Sàrl, Sofia, 22, Bulgaria","locationCode":"3062","description":"\r\n\t<div>OBJECTIVES: This paper presents a proposal to introduce a program aimed at measuring health and patient-related outcomes and implementing bundled payment for healthcare facilities to cover the complete cycle of care for patients with osteoarthritis (OA) of the hip and knee. The objective is to integrate the treatment process, incentivize improved outcomes, and lower complications and associated costs. METHODS: The International Consortium for Health Outcome Measurement (ICHOM) standardized set was selected to measure outcomes and compare clinical care for patients with hip and knee OA. Patients eligible for bundled payment include those with OA requiring surgical treatment and categorized as ASA I and ASA II according to the American Society of Anesthesiologists classification. The program is designed as a one-year pilot project in Bulgaria, with voluntary participation from healthcare facilities. RESULTS: The program aims to achieve high value by focusing on health outcomes achieved per unit of monetary value spent. By implementing bundled payments and measuring outcomes, the program seeks to improve quality of care, promote learning and improvement, reduce costs, and facilitate the adoption of best practices. It also allows patients to choose the most suitable facility while promoting the identification and dissemination of best practices. CONCLUSIONS: By implementing a value-based payment model and measuring outcomes, the proposed program has the potential to improve the quality of care for patients with OA. The pilot project will evaluate the program's effectiveness based on feedback from participating healthcare facilities and an analysis of its impact on clinical outcomes. The expected outcomes include improved patient health, reduced complication rates and associated costs, and increased value for patients, healthcare facilities, regulators, and the overall health system.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23vutovahpr5poster128963-pdf.pdf?sfvrsn=7d8d60c7_0","title":"ISPOREurope23_Vutova_HPR5_POSTER128963.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128963","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Implementing an Enhanced Recovery from Surgery Pathway to Reduce Hospital Length of Stay After Primary Hip and Knee Arthroplasty: A Budget Impact Analysis for Australia","id":"cbf79307-a0e8-4d18-9611-1cc275f089f1","sessionCode":"EE132","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: To determine the impact on healthcare costs of implementing an enhanced short-stay model of care (ESS-MOC) for arthroplasty at a national level. METHODS: A budget impact analysis was conducted for Australian publicly and privately funded hospitals providing hip and knee arthroplasty over the years 2023-2030. Population-based sample projections obtained from clinical registry and administrative datasets of all individuals receiving minor-complexity elective hip or knee arthroplasty for osteoarthritis were applied. The ESS-MOC assigned a conservative 30% of eligible patients to an enhanced recovery from surgery (ERAS) pathway comprising shortened acute ward stay (average 2 days versus 4 days with current care) and outpatient rehabilitation. The remaining 70% received a current practice clinical pathway. The primary outcome was total healthcare cost savings post-implementation of the ESS-MOC, with return on investment (ROI) ratio and hospital bed days utilised also estimated. Costs are presented in Australian dollars (AUD), at 2023 prices. RESULTS: Estimated cost savings for 2023-2030 from implementing the ESS-MOC pathway were AUD641million (95% CI: AUD99million to AUD1250million). This corresponds to a ROI ratio of AUD9.88 (AUD2.3 to AUD18.9). Savings would be 8-fold higher in the private sector (AUD571million vs. AUD70million), primarily attributable to the >80,000 rehabilitation bed days saved annually in this sector. For the period 2023-2030, an estimated 337,000 (261,000 to 412,000) acute bed days could be saved (private sector 262,000 [200,000 to 324,000]; public sector 74,000 [57,000 to 92,000]). Total implementation costs for the ESS-MOC were estimated at AUD38million and AUD34million for the private and public sectors, respectively. Only a small proportion of eligible patients (2% and 10% in private and public, respectively) would need to move into the ERAS pathway to realize cost savings. CONCLUSIONS: Implementation of an ESS-MOC for eligible arthroplasty patients in Australia would generate significant cost and resource savings, particularly in the private hospital sector.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128752","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Mortality and Clinical Complications Among Patients with Transfusion-Dependent Beta-Thalassemia in France","id":"379f7023-ae7b-4817-b451-1d0c77120816","sessionCode":"CO34","topDisplay":"Baldwin J1, Udeze C1, Li N1, Boulmerka L2, Dahal L1, Pesce G3, Quignot N4, Jiang H3, Galacteros F5 1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Formerly of Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 3Certara France, Paris, France, 4Certara France, Paris, 75, France, 5Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, Créteil, France","locationCode":"1033","description":"\r\n\t<div>OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is a rare hereditary disorder wherein patients have reduced or absent β-globin and require regular red blood cell transfusions (RBCTs). Patients with TDT experience significant complications associated with the disease and iron overload that can lead to early mortality and significant clinical burden. This study describes the mortality and clinical complications in patients with TDT in France. METHODS: This longitudinal, retrospective cohort study utilized the système national des données de santé’ (SNDS) database in France. Patients were identified by having an inpatient claim or registration in the long-term condition database (ALD, affection longue durée) with a diagnosis of β-thalassemia between January 1, 2012, and March 1, 2019. Eligible patients with TDT were required to have ≥8 RBCTs/year in any 2 consecutive years. Patients were required to have data for ≥1 year before and after their index date (i.e., the date of the eighth transfusion in the second year of 2 consecutive years). Patients were followed from index until death or study period end (March 1, 2020). Demographics were assessed at index. Mortality (proportion of total population, rate [deaths per 100 person-years], and mean age of death) and clinical complications (proportion of total population) were summarized descriptively during the follow-up period. RESULTS: In total, 331 patients with TDT were included. Their mean age was 26.1 years, and 164 (49.5%) patients were female. Mean length of follow-up was 4.9 years. During follow-up, 15 (4.5%) patients in the cohort died. Mortality rate was 1.16 deaths per 100 person-years. Mean age of death was 52.5 years. The most prevalent complications were endocrine complications (26%), hepatobiliary complications (23%), cardiovascular complications (19%), and renal complications (9%). CONCLUSIONS: Despite available care, patients with TDT experience numerous TDT-related clinical complications and increased mortality, underscoring the need for innovative therapies in this space.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23baldwinco34posterv2129205-pdf.pdf?sfvrsn=ddcb00e8_0","title":"ISPOREurope23_Baldwin_CO34_POSTER_V2129205.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129205","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Incurred Post-Kidney Transplant: A Systematic Review","id":"8cd758f4-168b-41fb-9b5c-1d2772d08ee3","sessionCode":"EE118","topDisplay":"Rabie H, Tickell LA, Lim KK King's College London, London, UK","locationCode":"1048","description":"\r\n\t<div>OBJECTIVES: While kidney transplant is the preferred treatment for end-stage kidney disease, the high cost incurred post-kidney-transplant (PKT) may limit its provision by health systems. This study systematically examined the cost incurred PKT. METHODS: We performed systematic searches on three bibliographic databases (Medline, Embase, EconLit) in June 2022. Two independent researchers screened the titles / abstracts, followed by full texts. We included costing studies published in 2012-2022. We extracted and standardised per-person cost to USD 2021. Costs were summarised according to time periods PKT, event, or resource categories where specified. RESULTS: Of 3286 unique articles screened, 27 studies were included. These were mostly cohort studies (62%) conducted in hospitals (37%), community (33%) or transplant centres (26%) from high-income countries (78%) published between 2018-2022 (78%). Majority studies reported healthcare cost (93%) for specific events (56%) including infection (19%), graft failure / delayed graft function (15%), antibody-mediated rejection (11%), and surgical complications (7%). Where costs reported were event-specific, inpatient cost was the highest with graft failure (USD175k 1-year PKT), followed by lymphoproliferative disorder (USD99k 1-year PKT, USD19k >1-year PKT), surgical complications (USD10k for <1-year, USD14k 1-year, USD39k >1-year PKT), early cancer (USD33k 1-year, USD12k-15k >1-year PKT), rejection (USD 1k-13k 1-year, USD30k >1-year post-diagnosis) and infection (USD1k 1-year PKT). Meanwhile, outpatient cost was the highest for graft failure (USD35k 1-year PKT), followed by early cancer (USD33k 1-year PKT), lymphoproliferative disorder (USD29k 1-year PKT), rejection (USD10k-14k >1-year post-diagnosis) and cytomegalovirus infection (USD1.8k <1-year PKT). Where cost reported were not event-specific, inpatient cost ranged USD18-USD58k, outpatient USD26-USD11k, and travel USD135 1-year PKT. While most studies reported follow-up duration, 48% did not report methods for cost estimation. CONCLUSIONS: Cost incurred PKT varied widely according to time periods, events, and resource categories. Most cost estimates came from single or a small number of studies, hence the findings may not be generalisable.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee118-ispor-europe-2023-poster-rabieh132160-pdf.pdf?sfvrsn=7e10f762_0","title":"EE118 ISPOR Europe 2023 Poster RabieH132160.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132160","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Impact of Response Assessment Timing on Indirect Treatment Comparisons (ITCS) – A Simulation Study","id":"01133cb2-4d7d-4173-8c4d-1d37918e4c38","sessionCode":"MSR15","topDisplay":"Kanters S1, Wennersbusch D1, Harrigan S1, Zannat NE2, Stilla AM1, Yang L1, Zoratti MJ1, Limbrick-Oldfield EH1 1RainCity Analytics, Vancouver, BC, Canada, 2RainCity Analytics, Langley, BC, Canada","locationCode":"5051","description":"\r\n\t<div>OBJECTIVES: Progression-free and event-free survival are common primary endpoints in oncology trials. Initial response assessments conducted at fixed times (e.g., every 12 weeks) often lead to left-censoring. We sought to determine the impact of left-censoring in primary analyses and in ITCs through a simulation analysis. METHODS: We simulated data from two trials with divergent time-to-event evaluation schedules. Our simulations compared 108 parameter permutations: true hazard ratio (HR; 0.25, 0.50, 0.75, or 1.00); sample size (50, 100, or 500); distribution (Weibull, Exponential, or Gompertz); and time intervals of evaluation for each trial with differences of 3, 6 and 12 weeks. Data were generated over 500 simulations for each combination of parameters using the simsurv package in R. Events occurring between timed evaluations were changed to the set evaluation time. We measured the mean squared error and mean bias of the log hazard ratios for both individual studies and ITCs to compare Cox proportional hazards regression to interval censoring regression. RESULTS: The use of Cox regression, in the presence of left censoring, led to some degree of bias in the estimated HR of a given study. This varied from negligible to a difference of 0.059 on the log-hazard scale (i.e., HR of 1.061 as an estimate for 1.00). Similar results were observed using interval censoring, albeit ranging from negligible to 0.040 (HR 1.041 as an estimate for 1.00). Factors associated with larger bias were larger between-trial differences in time of evaluation and smaller sample sizes. Despite bias in the individual estimates, this did not translate to bias in the ITCs as all bias was negligible. CONCLUSIONS: Our simulation study suggests that, despite biased estimates of HR in individual studies, ITCs are unaffected. This supports the use of Cox regression in place of interval censoring regression.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133852","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of S-Adenosylmethionine for the Treatment of Intrahepatic Cholestasis in China","id":"283efe3b-a35d-4e88-be4c-1d4655a2e0cd","sessionCode":"EE25","topDisplay":"Kim H1, Lyu J1, Royle R1, Kim K2, Byrnes J3 1Griffith University, Nathan, QLD, Australia, 2Abbott Products Operations AG, Basel, Basel-Stadt, Switzerland, 3Griffith University, Brisbane, QLD, Australia","locationCode":"1069","description":"\r\n\t<div>OBJECTIVES: Intrahepatic cholestasis (IHC) is a dysfunction of hepatocytes or the lesion/obstruction of bile canaliculi or duct. Symptoms with cholestatic-specificity are jaundice, pruritus, steatorrhea, fatigue, and osteoporosis. Persistent IHC can result in fibrosis, cirrhosis, possible hepatocellular carcinoma, and subsequent liver failure. In China the treatment for IHC caused by a variety of liver diseases is S-Adenosylmethionine (SAMe). The aim of this study is to assess the cost effectiveness of SAMe in China. METHODS: A Markov model with three health states (moderate/severe IHC, mild ICH and dead) was constructed to compare SAMe with ‘no treatment’ (noTREAT). The probability of patients moving between moderate/severe ICH and mild ICH depended on the liver function tests. Published clinical trial data was used to inform the change in liver function test for SAMe and noTREAT. Cycle length was set to 8 weeks with a time horizon of the 2 years. Costs and quality of life utilities were obtained from published sources and through surveys of experts. The incremental cost-effectiveness ratio (ICER) threshold was set to 3xGDP/capita for China which was CNY 270.000 in 2022. One sensitivity analysis and probabilistic sensitivity analysis were performed. RESULTS: Treatment of ICH with SAMe resulted in 1.25 quality adjusted life years (QALY) at a cost of CNY 55,239 vs 1.05 QALY and CNY 44,119 for noTREAT. This resulted in an ICER of CNY 56,451/QALY. One way sensitivity analysis showed that the main driver of the model is the cost of SAMe with the ICER almost doubling to CNY 102,884/QALY with a 25% increase in the cost of SAMe. 80% of the PSA simulations were below the ICER threshold. CONCLUSIONS: This study shows that SAMe is a cost-effective option for the treatment of liver cholestasis in China with the ICER (CNY 56,451/QALY) being lower than the threshold.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133757","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization and Costs in Patients Experiencing Severe Cardiac Events Following a COPD Exacerbation: Results from EXACOS-CV Studies in Spain, Germany, the Netherlands and Canada","id":"20975453-b8f7-4148-abce-1d567fc5eda9","sessionCode":"RWD25","topDisplay":"Parsekar K1, Kossack N2, Hernández I3, Vogelmeier CF4, Kolb N5, Baak B6, Swart-Polinder KMA6, Simons S7, Bengtsson C8, Vojinovic-Dees D9, Sin D10, Hawkins N10, McMullen S11, Pham T11, Corregidor García C12, Martinez N12, Sánchez-Covisa Hernández J12, Abram M13, Halbach M13, van Burk L14, Randhawa A15, Nordon C1 1AstraZeneca, Cambridge, UK, 2WIG2 GmbH, Leipzig, SN, Germany, 3Atrys Health, Madrid, Spain, 4Philipps University of Marburg, Marburg, Germany, 5ZEG – Berlin Center for Epidemiology and Health Research, Berlin, Germany, 6PHARMO Institute for Drug Outcomes Research, Utrecht, Netherlands, 7Maastricht University Medical Centre, Maastricht, Netherlands, 8IQVIA, Solna, Sweden, 9IQVIA, The Netherlands, Netherlands, 10University of British Columbia, Vancouver, BC, Canada, 11Medlior Health Outcomes Research, Calgary, AB, Canada, 12AstraZeneca, Madrid, Spain, 13AstraZeneca, Hamburg, Germany, 14AstraZeneca, Den Haag, Netherlands, 15AstraZeneca, Mississauga, ON, Canada","locationCode":"6079","description":"\r\n\t<div>OBJECTIVES: To describe cardiovascular (CV) hospitalizations and estimate related costs in patients who experienced a severe CV event following an exacerbation of Chronic Obstructive Pulmonary Disease (ECOPD). METHODS: EXACOS-CV program is a set of observational cohort studies. This analysis includes patients with COPD from Canada, Germany, Spain, the Netherlands (NL), identified in secondary databases between 2014-2018. For the present analyses we selected patients who had (hospitalization for acute coronary syndrome, heart failure, stroke or arrhythmias, or death) and an ECOPD within 12 months preceding it. CV-related, respiratory-related and all-cause hospitalizations were described during the 12 months pre-outcome and the 1-3 and 3-12 months post-outcome. The respective across-country cost per capita of hospitalizations were computed in the 12-month prior and 12-month post-outcome. RESULTS: 43,546 patients were included. In the 12 months pre-outcome, between 23% (Germany) and 44% (NL) of patients already had 1+ CV-related hospitalization. Immediately post-outcome (0-1 month), 32% (Spain), 48% (the NL), 50% (Germany), and 69% (Canada) of patients were no longer in the cohort (attrition primarily due to death). Attrition reached up to 72% at 3-12 months post-outcome (Canada). In the 1-3 months post-outcome, CV-related re-hospitalizations were observed in 3% (Spain) to 21% (Germany) of patients. In the 3-12 months post-outcome, these rates ranged between 10% (Spain) and 32% (NL). The average cost of CV hospitalizations doubled over 12 months post-outcome (€12,551, range: €3582 – €27,394) compared to prior-outcome (€5,010, range: €671 – €10,340) due to both outcome-related hospitalization and re-hospitalizations. Similar trends were seen for all-cause and COPD-related hospitalizations. CONCLUSIONS: Severe CV events increase HCRU and costs in exacerbating COPD patients namely due to cardiac re-hospitalizations, highlighting the economic burden incurred by increased cardiopulmonary risk in COPD and the urgency for comprehensive clinical management.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/exacos-cvispor-eu-2023final-upload3-nov129353-pdf.pdf?sfvrsn=a5c345a3_0","title":"EXACOS-CV_ISPOR-EU 2023_Final upload_3 Nov129353.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129353","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patients’ and Healthcare Professionals’ Preferences Regarding Lipid-Lowering Therapies for Patients with Very High Cardiovascular Risk and Established Atherosclerotic Cardiovascular Disease","id":"7ba705e7-ee9d-4a6b-bf14-1d92432fb8db","sessionCode":"PCR2","topDisplay":"Parrondo García FJ1, Fernandez Olmo MR2, Álvarez Hermida AB3, Castellanos M4, Gomez Cerezo J5, Igual Guaita MJ6, Jansen Chaparro SJ7, Obaya Rebollar JC8, Martin Mitjana L9, Cosin-Sales J10 1Novartis Spain, Coslada, M, Spain, 2Hospital Universitario de Jaen, Jaen, Jaen, Spain, 3Centro de Salud Goya, Madrid, Madrid, Spain, 4A Coruña University Hospital & Biomedical Research institute, A Coruña, Spain, 5Infanta Sofia University Hospital, Madrid, Spain, 66Farmacia de Atención Primaria del Departamento de Salud de Gandía, Gandía, Valencia, Spain, 7Hospital Regional Universitario de Málaga, Málaga, Málaga, Spain, 8Centro de Salud Chopera, Madrid, Madrid, Spain, 9Novartis Spain, Barcelona, Barcelona, Spain, 10Hospital Arnau de Vilanova, Valencia, Valencia, Spain","locationCode":"6009","description":"\r\n\t<div>OBJECTIVES: Controlling LDL cholesterol (LDL-C) can reduce atherosclerotic risk. However, not all available therapies are equally effective and safe, or use the same route of administration. Involving patients and considering their preferences in treatment decision-making can help treatment success. The main goal was to assess patients’ and healthcare professionals’ (HCP) preferences regarding the characteristics (attributes) of lipid-lowering therapies. METHODS: A discrete choice experiment (DCE) was conducted to determine participants’ preferences. A literature review and two focus groups (patients=4; HCP=8) were conducted to identify the attributes and levels to be included in the DCE; five attributes (safety, medication collection location, LDL-C reduction, prevention of cardiovascular problems, route of administration) with 2-3 levels each were included, resulting in 36 scenarios. The relative importance (RI) given to each attribute was estimated using a conditional logit model. RESULTS: A total of 42 patients and 89 HCP participated in the study. A third of the patients were women with a mean age of 55.6 years. The most common atherosclerotic cardiovascular disease was myocardial infarction (47.6%) and the most common treatment received was atorvastatin (61.1%). Regarding HCP (primary care: 16.9%; cardiology: 16.9%; neurology: 16.9%: internal medicine: 15.7%; nursing: 16.9%; clinical pharmacy: 16.9%), 42.7% were women, with a mean professional experience of 23.9 years. Most of the HCP (76.4%) worked in hospitals with >300 beds. The estimated RI were: safety: Patients: 24.0%, HCP: 10.2%; medication collection location: Patients: 12.1%, HCP: 9.5%; LDL-C reduction: Patients: 26.8%, HCP: 31.6%; prevention of cardiovascular problems: Patients: 26.8%, HCP: 37.7%; and route of administration: Patients: 10.3%, HCP: 10.9%. CONCLUSIONS: When selecting a lipid-lowering treatment, patients valued similarly efficacy (LDL-C reduction and prevention of cardiovascular problems) and safety, while HCP considered efficacy the most important attribute, followed by route of administration. The results of the study can contribute to improve decision-making.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23parrondopcr2130943-pdf.pdf?sfvrsn=825ad535_0","title":"ISPOREurope23_Parrondo_PCR2130943.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130943","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Utility Analysis of Lorlatinib for First-Line Treatment for ALK Positive Advanced Non-Small Cell Lung Cancer in China","id":"33530f1c-76a9-4cd9-b975-1e7cffe3233c","sessionCode":"EE69","topDisplay":"He X, Fu S School of Pharmaceutical Science and Technology, Tianjin University, Tianjin, China","locationCode":"2006","description":"\r\n\t<div>OBJECTIVES: This study aims to estimate the cost-utility of lorlatinib, a third-generation anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor (TKI), compared with alectinib in patients with previously untreated ALK-positive locally advanced non-small cell lung cancer (ALK+ aNSCLC) from China healthcare system perspective. METHODS: A partition survival model with four health states including progression-free, non-central nervous system [CNS] progressed disease, CNS-progressed disease, and death was developed. A life-time horizon was used in the model. The clinical, utility and cost parameters required for the model were obtained from key clinical trials, published literature and clinical experts. An indirect treatment comparison was performed to inform the efficacy of lorlatinib vs alectinib due to a lack of head-to-head comparison. Three times the per capita gross domestic product (GDP, CNY 85,698 in 2022) is used as the willingness to pay threshold. Sensitivity analysis were performed to test the stability of the model. RESULTS: In the base-case scenario, lorlatinib resulted in a gain of 1.38 QALYs（5.28 vs. 3.90） and an incremental cost of CNY 113,810（CNY 847,992 vs. CNY 734,182） compared with alectinib. The incremental cost per QALY gain was CNY 82,824/ QALY, which is lower than one time GDP per capita. One-way sensitivity analysis presented that the hazard ratio (HR) of overall survival (alectinib vs. crizotinib), HR of progression-free survival (alectinib vs. crizotinib) and the drug price of alectinib were the main factors that had the impact on the model outcomes. Probability sensitivity analysis showed that the outcomes were consistent. CONCLUSIONS: At current price, lorlatinib is more cost-effective than alectinib as the first-line treatment in patients withALK+ aNSCLC.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23fuee69poster132461-pdf.pdf?sfvrsn=2fc547ed_0","title":"ISPOREurope23_Fu_EE69_POSTER132461.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132461","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Discrepancy of Health State Utility Values in Cost-Utility Analyses of Interventions Against Infectious Diseases in Older Adults","id":"93bf3665-e046-4aea-9103-1f3019e316ea","sessionCode":"SA12","topDisplay":"Pham TH1, Mihajlović J2, Loncar I2, Zeevat F3, Dagne AW4, Meszaros K5, van der Schans J3 1University of Groningen, University Medical Center Groningen, Groningen, GR, Netherlands, 2Mihajlović Health Analytics, Novi Sad, Serbia, 3University of Groningen, University Medical Center Groningen, Groningen, Netherlands, 4University of Gondar, Gondar, 1, Ethiopia, 5GSK, Wavre, Belgium","locationCode":"7020","description":"\r\n\t<div>OBJECTIVES: The choice of health state utility values (HSUVs) can significantly influence cost utility analysis (CUA) results, particularly in older adults with infections whose HSUVs might be heterogeneous due to age, comorbidity, or frailty levels. However, despite the importance, the rigorous selection of HSUVs is often overlooked, leading to potential misleading outcomes. This study aims to analyze the methods used to elicit HSUVs in CUAs of infectious diseases in older adults. METHODS: A comprehensive literature review was conducted on PubMed in January 2023 to identify published CUAs of infectious diseases in older adults. These studies underwent critical appraisal independently by two reviewers using the modified checklist developed by Nerich et al. to evaluate the elicitation methods of HSUVs. RESULTS: (Preliminary) The review identified 113 CUAs, and a total of 60 studies have been analyzed. Out of these, 6 studies (10%) cited original health state utility studies, while 45 studies (75%) cited economic evaluations. Most studies did not provide an explanation for their choice or description of the techniques used to elicit HSUVs. Twenty-four studies (40%) used HSUVs that were appropriate in terms of comparability between populations and countries. In 44 studies (73%), outdated references (more than 10 years older than the CUA) were used. Inconsistencies between cited HSUVs and the references were observed in 12 studies. Only 5 studies (8%) discussed limitations regarding the elicitation of HSUVs. CONCLUSIONS: This study highlights the poor practices in the elicitation of HSUVs in CUAs of interventions against infectious diseases in older adults. It is crucial to encourage health economists to adopt a systematic approach in choosing and eliciting HSUVs for accurate health economic evaluations. Additionally, prioritizing data collection of HSUVs is necessary to support future CUAs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130481","diseases":[{"id":"dc752772-538c-4933-b6a5-3b5b6d079673","parentId":"00000000-0000-0000-0000-000000000000","title":"Geriatrics","urlName":"Geriatrics"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Usage of Social Return on Investment Modelling in the Analysis of Healthcare Interventions: A Review of Current Utilization","id":"b6bce93c-0699-4a2c-945c-1f4a1acd3f37","sessionCode":"EE150","topDisplay":"Harrop D1, Hirst A1, Hughes R2, Weston G1 1Adelphi Values PROVE, Bollington, Cheshire, UK, 2Adelphi Values PROVE, Bollington, UK","locationCode":"2079","description":"\r\n\t<div>OBJECTIVES: The quality of public health has far-reaching effects on economic, social, and environmental factors. The growing importance of social and environmental outcomes in decision making, not currently captured within traditional health economic evaluation models, has created a gap in evidence generation for healthcare interventions. This study investigates the applicability of Social Return on Investment (SROI) in modelling of healthcare interventions, considering economic, social, and environmental outcomes beyond the current scope considered in traditional modelling and HTA submissions. Furthermore, this study evaluates the current level of utilization of SROI modelling within healthcare interventions. METHODS: A landscaping review was conducted to investigate published SROI models within healthcare interventions, health improvement programs, and health-base policies. The review evaluated the methods, outcomes, and narratives employed to assess the current utilization of SROI models and their potential effectiveness in generating evidence concerning the benefits of healthcare interventions. RESULTS: The review found 22 publications for SROI models and 10 publications relevant to SROI methodology within the healthcare setting have been published since 2013. The analysis of papers revealed that SROI modelling holds significant promise as a tool, capturing the impact on a wide array of factors influencing the patient’s lifestyle and society and could be seen as the natural evolution of the traditional health economic models. However, most SROI models took a qualitative approach to outcomes, with the general lack of data and established methods surrounding the valuation of non-monetizable parameters limiting the application of quantitative value. CONCLUSIONS: The usage of SROI modelling can provide a more comprehensive assessment of health interventions compared to traditional health economic evaluations, allowing the demonstration of a much wider range of value sources for healthcare interventions. However, standardization of the valuation of non-monetizable parameters would be required to improve comparability between SROI models and accommodate their wider usage.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee150---usage-of-social-return-on-investment-modelling-in-the-analysis-of-healthcare-interventions-a-review-of-current-utilisation-v10130090-pdf.pdf?sfvrsn=d10baf92_0","title":"EE150 - Usage of Social Return on Investment modelling in the analysis of healthcare interventions a review of current utilisation v1_0130090.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130090","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Use of Expert Opinions, Patient, and Carer Perspectives and Real-World Evidence in Orphan Disease Submissions Differs for Health Technology Assessment Agencies Across the World","id":"dd73f0e7-a6d8-4340-8048-1f79121cfe8c","sessionCode":"HTA28","topDisplay":"Ben Taieb A1, Withers K1, Hardy EJ2, Swart N1, Redhead G3, Lang S2, Foy C1, Leadley RM2, Swift SL2 1Mtech Access, Bicester, Oxfordshire, UK, 2Mtech Access, York, North Yorkshire, UK, 3Mtech Access, Manchester, Greater Manchester, UK","locationCode":"4062","description":"\r\n\t<div>OBJECTIVES: To determine the contributions of expert opinions, patient/carer perspectives and real-world evidence (RWE) in global orphan disease health technology assessment (HTA) submissions. METHODS: The NICE database was searched on 18th April 2023 to identify orphan disease HTA submissions (using Orphanet identifiers) for the previous 12 months. Matched submissions for the same drug, manufacturer and disease were subsequently searched for Australia (PBAC), Canada (CADTH), France (HAS), Germany (G-BA/IQWiG) and the EU (EUNETHTA). Data were extracted into Excel® by one reviewer and checked by a second reviewer to reduce error and bias. A thematic analysis was conducted. RESULTS: After identifying 11 relevant submissions to NICE, we sourced matched submissions with decisions from G‑BA/IQWiG (n=9), HAS (n=8), CADTH (n=7) PBAC (n=2) and EUNETHTA (n=1). Preliminary data indicate that, out of a total of 38 submissions, 33 (86.8%) were recommended, 3 (7.9%) were not recommended and 2 (5.3%) had mixed recommendations. 35 of 38 submissions (92.1%) used randomized controlled trial (RCT) data (+/- RWE) as the primary information source. 14 were informed by RCT evidence alone. Clinical experts contributed to 30 of 38 (78.9%) submissions (100% of NICE submissions), predominantly to inform and validate model assumptions. Surveys and testimonies from patients/carers contributed to 25 of 38 (65.8%) submissions (100% of NICE submissions), predominantly to provide information on treatment and quality of life. CONCLUSIONS: An unexpectedly high proportion of orphan disease submissions use RCTs as key data sources. Initial trends suggest that NICE and HAS tend to be more accepting of and place a greater emphasis on RWE and patient testimony compared with other agencies, although this may depend on the position of a new drug in existing treatment pathways. G-BA/IQWiG focused almost exclusively on RCT evidence. Given the emerging importance of RWE, its acceptance should be more widely embedded across HTA agencies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurpoe23langhta28poster131005-pdf.pdf?sfvrsn=8dd9fd9e_0","title":"ISPOREurpoe23_Lang_HTA28_POSTER131005.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131005","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Stroke Treatment Units: Requirements and Recommendations","id":"64a6e4fc-8c38-4c26-9d8f-1fc87cddd92f","sessionCode":"OP1","topDisplay":"Slavchev G1, Dacheva A2, Djambazov S3, Vutova Y4 1Medtronic International Trading Sàrl, Sofia, 22, Bulgaria, 2Medtronic International Trading Sàrl, Sofia, 23, Bulgaria, 3Medical University Pleven, Sofia, 23, Bulgaria, 4HTA Ltd., Sofia, 22, Bulgaria","locationCode":"5075","description":"\r\n\t<div>OBJECTIVES: This study aimed to outline the requirements and recommendations for establishing specialized stroke treatment units. METHODS: The criteria for specialized stroke treatment units were categorized into seven areas and analyzed based on existing recommendations and guidelines. These areas included infrastructure, early diagnosis, diagnostic and therapeutic infrastructure, therapeutic interventions, multidisciplinary mobilization and rehabilitation, expertise of medical staff, and emergency departments. RESULTS: The infrastructure of a specialized stroke treatment unit should consist of two functional segments: Segment A for acute phase treatment and monitoring, and Segment B for post-acute phase treatment. Rapid neurological assessment and access to a neurologist or internist trained in stroke treatment are crucial within 30 minutes of admission. Diagnostic procedures such as CT scans, echocardiography, and Doppler or duplex sonography should be available within specific timeframes. Therapeutic interventions, including thrombolysis and thrombectomy, should be initiated promptly. Multidisciplinary mobilization and rehabilitation should be provided, addressing nursing care, physiotherapy, speech therapy, cognitive rehabilitation, and patient education. The expertise of physicians and nursing staff should be continuously developed through training programs. CONCLUSIONS: The establishment of specialized stroke treatment units requires adherence to specific requirements and recommendations. These units should have appropriate infrastructure, rapid and accurate diagnostic procedures, efficient therapeutic interventions, and multidisciplinary mobilization and rehabilitation. Ongoing training of medical staff is essential to maintain expertise. Access to emergency departments with trained professionals is crucial for continuous stroke care. Implementing these recommendations can improve stroke treatment outcomes and enhance patient recovery.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"bb8fd992-de86-4d1a-8bec-f6e2c928db96","parentId":"00000000-0000-0000-0000-000000000000","title":"Organizational Practices","urlName":"organizational-practices"}],"categoryIds":["bb8fd992-de86-4d1a-8bec-f6e2c928db96"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23slavchevop1poster128949-pdf.pdf?sfvrsn=4bfdc12c_0","title":"ISPOREurope23_Slavchev_OP1_POSTER128949.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128949","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"In Silico Clinical Trials Using Mechanistic Knowledge-Based Model, an Innovative Approach to Accelerate Data Generation and Support Health Technology Assessment","id":"bd1a68c3-39c1-427e-8f96-1fec8de96cf7","sessionCode":"SA1","topDisplay":"Pham E1, Porte S2, Courcelles E2, Peyronnet E2, Wang Y2, Diatchenko A2, Gomez G2, Amarenco P3, Angoulvant D4, Boccara F5, Cariou B6, Mahé G7, Marie-Natacha M8, Bastien A9, Portal L9, Boissel JP2, Bechet E2, Granjeon-Noriot S2, Steg PG10 1Novadiscovery, Lyon, France, 2Novadiscovery, Lyon, Rhone-Alpes, France, 3Department of Neurology and Stroke center, APHP, Bichat Hospital, Université Paris-Cité, Paris, France, 4Cardiology department, Hôpital Trousseau, CHRU de Tours & EA4245 Transplantation Immunologie Inflammation, Université de Tours, Tours, France, 5Sorbonne Université, GRC n°22, C2MV-Complications Cardiovasculaires et Métaboliques chez les patients vivant avec le Virus de l'immunodéficience humaine, Inserm UMR_S 938, Centre de Recherche Saint-Antoine, Institut Hospitalo-Universitaire de Cardio-mé, Paris, Ile de France, France, 6Nantes Université, CHU Nantes, CNRS, Inserm, l'institut du thorax, Nantes, France, 7Vascular Medicine Unit, CHU Rennes, Univ Rennes CIC1414, Rennes, France, 8Novartis, Rueil Malmaison, 92, France, 9Novartis, Rueil-Malmaison, France, 10Université Paris-Cité, AP-HP, Hôpital Bichat, and INSERM U-1148/LVTS, Paris, France","locationCode":"7010","description":"\r\n\t<div>OBJECTIVES: Demonstrating cardiovascular (CV) benefits with lipid-lowering therapy (LLT) requires long-term randomized clinical trials (RCT) with thousands of patients. Innovative approaches such as in silico trials applying a disease computational model to virtual patients receiving alternative treatments provide a valuable option to complement RCTs by rapidly generating supplementary comparative effectiveness data and facilitating drug value demonstration to health technology assessment (HTA) bodies. This study aimed at building a computational model of atherosclerotic cardiovascular disease (ASCVD). Once validated, the model will be used to run in silico clinical trials to compare the benefit of inclisiran, an siRNA targeting PCSK9 mRNA, vs other LLT on CV events in patients with ASCVD. METHODS: A mechanistic computational model of ASCVD was built from an extensive literature review, combining mechanisms of lipoprotein metabolism, with atherosclerotic plaque evolution leading to clinical events. Impact of ASCVD risk factors and standard-of-care LLTs were also integrated. A panel of 6 multidisciplinary clinical experts validated modelling hypotheses and the strategy of calibration/validation by selecting relevant RCTs and registry data. Calibration is the process of determining the values of unknown model parameters such that the model reproduces relevant data. A virtual population was generated to account for inter-patient variability. RESULTS: The model was calibrated to reproduce the pharmacokinetics of atorvastatin, rosuvastatin and ezetimibe, the clinical benefit of combinations of LLTs, including evolocumab and inclisiran and its surrogate markers of efficacy as observed in landmark trials such as <a href=\"https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.117.032235\" target=\"_blank\" rel=\"noopener\">FOURIER</a> and <a href=\"https://www.nejm.org/doi/10.1056/NEJMoa1912387\" target=\"_blank\" rel=\"noopener\">ORION 10</a>. CONCLUSIONS: The model is successfully calibrated. Next step is model validation before using it to predict long term effect of inclisiran on CV events. In silico clinical trials will potentially provide evidence of the clinical benefit of Inclisiran earlier than RCTs. Therefore, the acceptance of this new emerging approach by the HTA bodies could accelerate patient access to innovative drugs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23phamsa1poster131547-pdf.pdf?sfvrsn=163456bb_0","title":"ISPOREurope23_Pham_SA1_POSTER131547.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131547","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Hospital Care Pathway of Patients Treated By Axi-Cel and Tisa-Cel between 2018 and 2021 in France: A National Study Based on the Comprehensive Inpatient Stays Database","id":"15c6fc3c-97cf-4b24-97aa-20331227a449","sessionCode":"HSD10","topDisplay":"Thieblemont C1, Caillot D2, Pierre M3, Branchoux S4, Lemasson H4, Caron A5, Torreton E5, Petel A6, Despas F7 1Hôpital Saint-Louis, Paris, France, 2CHU de Dijon, Dijon, France, 3Bristol Myers Squibb, Rueil-Malmaison, 75, France, 4Bristol Myers Squibb, RUEIL MALMAISON, France, 5CEMKA, Bourg-la-Reine, France, 6Bristol Myers Squibb, Rueil-Malmaison, France, 7Centre Hospitalier Universitaire, Toulouse, France","locationCode":"4021","description":"\r\n\t<div>OBJECTIVES: Since 2018, the lymphoma patient’s pathway has evolved with the innovative CAR T-Cell therapies and the progressive opening of qualified centers. The study objective was to describe the pathway of the patients treated by axi-cel and tisa-cel from 2018 to 2021, and to study its predictors. METHODS: A retrospective cohort study was performed using the French comprehensive hospital database (Programme de Médicalisation des Systèmes d'Information, PMSI). Patients hospitalized for axi-cel and tisa-cel administration discharged between January 1, 2018, and December 31, 2021, were included. Patients treated for acute lymphoblastic leukemia and/or minors were excluded. The patient’s journey was described using the following indicators: the bridging therapy duration and setting, the length from CAR T-cell infusion to discharge, and the discharge modes. The predictors were identified among the patients' and centers’ characteristics, the CAR T-cell infused and the coverage method, using linear and logistic mixed-effect regression models. RESULTS: A total of 901 patients were included, 548 treated by axi-cel and 353 by tisa-cel. The mean age was 59.8 years (±12.6) and 61.3% were male (n=552). On average, the durations were 36.6 days (±15.8) for bridging therapies, and 18.1 days (±11.4) from CAR T-cell infusion to discharge. The proportion of patients attending another center for the bridging therapy was 43.5% (n=199). Most of the patients were discharged at home (74.8%, n=674). Center experience was the main predictor of patients’ pathways. Higher experience was associated with shorter post-infusion’s length of stay, higher probability to receive the bridging therapy in another center and to be discharged at home. Among patient-level characteristics, the absence of transplant’s history, and the treatment by tisa-cel were associated with shorter length of stay. CONCLUSIONS: Center experience was the main driver of the patient’s journey. These results should be updated with the growing experience of the qualified centers.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/27-10-2023--the-hospital-care-pathway-of-patients-treated-by-axi-cel-and-tisa-cel-between-2018-and-2021-in-france---ispor-eu-2023-131995-pdf.pdf?sfvrsn=66ae2fa1_0","title":"27-10-2023- The hospital care pathway of patients treated by axi cel and tisa cel between 2018 and 2021 in France - ISPOR EU 2023 131995.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131995","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"To What Extent European Value-Based Pricing Methodologies Take Into Account Patient Perspectives and Broader Societal Benefits?","id":"cc16404e-0081-4380-9570-20c8839bfa55","sessionCode":"PCR53","topDisplay":"Vassoler H Red Nucleus, Brighton , BNH, UK","locationCode":"6052","description":"\r\n\t<div>OBJECTIVES: Recognizing broader benefits of healthcare interventions depends on how budgets and price setting methodologies are defined. This research explores the extent to which patient and broader societal benefits are recognized by healthcare payers in Europe in the context of value-based pricing frameworks, and the main obstacles to fully shifting to patient-centric decision-making. METHODS: Literature searches were conducted to identify the value attribute categories considered as part of value-frameworks for HTA assessments. A semi-quantitative payer survey was conducted across four European countries (UK, FR, ES and IT) between April- May 2023, to test what value attributes have highest impact on pharmaceutical pricing decisions. RESULTS: Clinical and economic value attributes were found to have highest impact on price setting (i.e., objectively-measured investigator-assessed clinical endpoints and evidence of cost-offsets from a healthcare perspective). In contrast, non-clinical parameters such as patient perspectives and broader societal impact, such as QoL impact on caregivers, were found to have lowest impact. The results of this survey suggest that pricing decisions are largely based on the added clinical benefit and the healthcare cost-offsets provided; reflecting payer’s pursuit of maximizing health impact with a finite budget. Methodological difficulties in objectively measuring patient and societal benefits, as well as budget silos, were the main obstacles preventing payers from adopting a more holistic approach to recognizing broader value attributes for price setting. CONCLUSIONS: This research highlights the need for policy makers to address budget silos that exist between healthcare and other social-welfare services, to create incentives for the development of innovative therapies that improve not only clinical outcomes but also deliver broader patient and societal benefits, that deliver efficiency gains outside of healthcare budgets. There is also a need for the development of more robust methodologies that can objectively measure such endpoints.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23vassolerpcr53poster133820-pdf.pdf?sfvrsn=5e311e6b_0","title":"ISPOREurope23_Vassoler_PCR53_POSTER133820.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133820","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Preferences for Cancer Treatments: A Discrete-Choice Experiment with Adult Patients with Cancer in Europe","id":"cb8c6125-5058-48b6-adc3-20d2063899bc","sessionCode":"PCR20","topDisplay":"Botha W1, Philpott S2, Postmus D3, Pignatti F4, Rodriguez-Leboeuf AM3 1IQVIA, Thornton-Cleveleys, LAN, UK, 2IQVIA, Reading, RDG, UK, 3European Medicines Agency, Netherlands, Netherlands, 4European Medicines Agency, Amsterdam, Amsterdam, Netherlands","locationCode":"6021","description":"\r\n\t<div>OBJECTIVES: To evaluate patient preferences for cancer treatments in Europe. METHODS: An online discrete-choice experiment (DCE) survey among adult patients with cancer was conducted in 3 countries: Spain (N=253), Italy (N=250), and Croatia (N=100). Pairs of hypothetical cancer treatments had four attributes with varying levels: time until disease progression (3, 6, 9, and 12 months), treatment administration (oral pill/tablet or non-oral), treatment location (home or hospital/clinic), and impact of side effects on quality of life (QoL) (mild, moderate, or severe). A random-parameters logit model estimated country-specific preferences and assessed the conditional relative importance of each attribute. Swait and Louviere test was subsequently used to assess whether country-specific data could be pooled together. Subgroup and latent class (LC) analyses explored subpopulation preferences. RESULTS: Mean age across countries was 54 years, 54% had stage I cancer. Approximately 42% were undergoing treatment. Preferences were similar across countries. The impact of side effects on QoL was valued most, then, time until disease progression, treatment administration, and treatment location, respectively. Preferences varied by disease stage, education, and comprehension of the DCE task. LC analysis revealed 3 classes: class 1 valued time until disease progression over the impact of side effects on QoL, class 2 exhibited risk aversion and prioritized the impact of side effects on QoL over time until disease progression, class 3 displayed greater risk aversion and valued the impact of side effects on QoL, over time until disease progression, treatment administration, and treatment location. CONCLUSIONS: While time until progression is a commonly used endpoint in cancer clinical trials, notably in this study, patients prioritized impacts of side effects on their QoL. The impact of side effects on patients' QoL in cancer clinical trials should be evaluated to better understand the overall benefit-risk profile of cancer treatments and make informed decisions regarding patient care.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bothapcr20poster130065-pdf.pdf?sfvrsn=eaa05654_0","title":"ISPOREurope23_Botha_PCR20_POSTER130065.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130065","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Validation of a Scoring Algorithm for the Clinician-Reported Outcome ”Prurigo Activity and Severity (PAS)’’ Tool: Results Based on Clinical Studies of Dupilumab in Adults with Prurigo Nodularis","id":"8a479f2a-fc4a-43b1-946c-2166fff19d0b","sessionCode":"CO32","topDisplay":"Zeidler C1, Stander S1, Rhoten S2, Wratten S3, Zhang D4, Msihid J5, Brookes E6, O’Malley J7, Bansal A8, Wiggins S6, Zahn J8, Thomas RB8, Bahloul D5 1Center for Chronic Pruritus, University Hospital Münster, Munster, Germany, 2IQVIA, San Francisco, CA, USA, 3IQVIA, Manchester, UK, 4IQVIA, Falls Church, VA, USA, 5Sanofi, Chilly-Mazarin, France, 6Sanofi, Reading, UK, 7Sanofi, Cambridge, MA, USA, 8Regeneron, Tarrytown, NY, USA","locationCode":"1031","description":"\r\n\t<div>OBJECTIVES: Prurigo nodularis (PN), a pruritic skin disease, is characterized by multiple localized/generalized pruriginous lesions. The prurigo activity and severity (PAS) tool, a clinician-reported outcome (ClinRO) measure, is a clinically relevant indicator of activity and severity of PN. A 5-item PAS version (adapted from 7-item PAS) has been used in two phase-3 PN dupilumab trials. This study validated the psychometric properties of a PAS score (range: 0 to 11) derived as the sum of the items assessing the estimated number of pruriginous lesions, percentage of pruriginous lesions with excoriations/crusts, and percentage of healed pruriginous lesions and estimated the within-patient improvement threshold of PAS score in PN patients participating in the dupilumab clinical trials. METHODS: Psychometric properties of PAS score were assessed using ClinRO and patient-reported outcome data pooled from the phase-3 trials (N=311; NCT04183335, NCT04202679) of dupilumab in adult patients with PN uncontrolled on topical therapies. Within-patient meaningful improvement thresholds were established using target anchors (patient global impression of severity [PGIS], PGI change [PGIC], investigator’s global assessment for PN activity [IGA PN-A] and stage [IGA PN-S]). RESULTS: PAS score had good internal consistency (α > 0.70) and excellent test-retest reliability (intraclass correlation coefficient ≥ 0.8). Mostly moderate-to-strong correlations (absolute r = 0.32-0.87) with conceptually related-measures, and weaker-to-moderate correlations (absolute r <0.5) with less-related measures, supported the construct validity. PAS score differentiated well between groups known to be different at baseline, Week 12, and Week 24 (p<0.0001). Significant differences in mean PAS score changes over time were observed for groups defined using the target anchors (p<0.0001). Using anchor-based approach, an absolute change of 3 points (range: 2.0–4.0) represents a within-patient meaningful improvement threshold for PAS score. CONCLUSIONS: The PAS score is a fit-for-purpose tool for assessing disease activity and severity in adults with PN uncontrolled on topical therapies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23msihidco32poster132517-pdf.pdf?sfvrsn=f1c3640_0","title":"ISPOREurope23_Msihid_CO32_POSTER132517.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132517","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"People Living With HIV’s Lived Experiences of Long-Acting Injectable Antiretroviral Treatment Administrations in France Show the Central Role of Hospital Nurses in Care: A Qualitative Study (PANTER)","id":"9c375a5e-91b7-469d-9abd-220c7787463b","sessionCode":"PCR7","topDisplay":"Roucoux G1, Rousset-Torrente O1, Petit AS1, Allavena C2, Hocqueloux L3, Palacios C4, Zucman D5, Chassany O1, Duracinsky M6 1Patient-Centered Outcomes (PROQOL), University Paris Cité, Paris, 75, France, 2Institut de Médecine et Epidémiologie Appliquée, Hôpital Bichat, Paris, France, 3Regional Hospital Center of Orléans, Orléans, Loiret, France, 4Tenon Hospital, APHP, Paris, Ile de France, France, 5Foch Hospital, Suresnes, France, 6PROQOL Unit - URC-ECO, APHP - ECEVE, Paris Cité University, INSERM, Paris, Ile de France, France","locationCode":"6013","description":"\r\n\t<div>OBJECTIVES: Long-acting injectable treatment (LAIT) for HIV has been available in France since December 2021. The first three injections are administered as outpatient interventions at hospitals while the subsequent injections can be done by a district nurse (DN). We identified the LAIT administration patterns from patients living with HIV(PWH)s’ narratives. METHODS: Qualitative study using an Inductive General Approach, participant questionnaire and semi-structured interviews. Inclusion criteria was defined to generate a purposive sample with maximum variation. Recruitment occurred through HIV specialists in French hospitals. Data collection proceeded until saturation. Interviews were transcribed and coded in NVivo. Analysis was inductive thematic. Data reported according to COREQ guidelines. RESULTS: Twenty-four PWH participated. Most were men, French-born, residing in the provinces, single, childless and employed with a median age of 53 years old (23 to 77). The median HIV duration was 13 years (2 to 34) and median LAIT 7 months (2 to 24). Three-quarters were continuing LAIT at the time of interview. Three themes were found: (1) Assistance: hospital nurses (HN) continued to order and administer LAIT and organized patient-appointments. Assistance limited organizational problems. (2) Autonomy: patients ordered and collected LAIT from their usual pharmacy, kept it at home (Rilpivirine in refrigerator) or collected it just before injections, found a DN, arranged appointments in the practice or at home, educated pharmacists and DN about LAIT. Autonomy replicated core oral treatment habits. (3) Assistance to reach autonomy: HN taught patients and trained DN about LAIT, gave patients educational information and contact details for DN, and exceptionally accepted patients with DN appointment issues. HN had a central role. CONCLUSIONS: HIV professionals facilitated LAIT administration by fully or partially assisting patients to remain ART-autonomous, which mostly depend on HN. Patients' priority was to follow LAIT and they accepted the organization model implemented in their center.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/panter-ispor-poster-pcr7-oc132699-pdf.pdf?sfvrsn=ee3f46cb_0","title":"PANTER-ISPOR-Poster-PCR7-OC132699.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132699","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Effect of Daratumumab on Pneumonia in Patients with Multiple Myeloma Using Population Based Real-World Data","id":"434cc878-470d-4800-9c1d-22c39ecbd02f","sessionCode":"CO16","topDisplay":"Kim Y1, Kang KW2, Suh HS3 1Department of Regulatory Science, Graduate School, Kyung Hee University, Seoul, Korea, Republic of (South), 2Division of Hematology-Oncology, Department of Internal Medicine, Korea University College of Medicine, Seoul, Korea, Republic of (South), 3Department of Pharmacy, College of Pharmacy, Kyung Hee University, Seoul, Korea, Republic of (South)","locationCode":"1020","description":"\r\n\t<div>OBJECTIVES: Infections are major causes of morbidity and mortality in multiple myeloma (MM). Daratumumab improved patient outcomes but increased the risk of infectious complications, including pneumonia. The aim of this study was to identify the effect of daratumumab on pneumonia using real-world data. METHODS: Using the National Health Insurance database in Korea, patients with a history from the first- to fourth-line therapy of MM from 2007 to 2021 were included. We set the date of fourth-line therapy as the index date. We divided patients into two groups by the history of daratumumab. The start date for the was identified based on the earliest of the following: date of the first claim for new MM drugs or the first claim date for any MM therapy with a gap greater than 180 days after. The occurrence of pneumonia was defined as the first diagnosis of pneumonia during . Two groups were matched using 1:1 propensity score on age, sex, the year of the first-line therapy, pneumonia risk factors, etc. To compare the occurrence of pneumonia between the pre- and post-index period, we conducted the difference-in-differences (DID) analysis using the Cox proportional hazards model. RESULTS: After matching, 578 patients (N=289 in each group) were identified. Comparing the baseline characteristics using standardized differences, only the year of the first-line therapy differed. Comparing daratumumab group and non-daratumumab group, there was a non-significant hazard of pneumonia in pre-index period (hazard ratio (HR), 1.019; 95% confidence interval (CI), 0.818-1.271) and in post-index period (HR, 1.097; 95% CI, 0.869-1.389). After adjusting for the year of the first-line therapy, the hazards before and after index date were still statistically non-significant between the two groups (DID HR, 0.930; 95% CI, 0.673-1.282). CONCLUSIONS: In this study, daratumumab had no significant hazard on pneumonia in MM.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeu2023mmfinal132465-pdf.pdf?sfvrsn=86a11576_0","title":"ISPOR_EU_2023_MM(Final)132465.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132465","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Barriers and Facilitators for the Use of Point of Care Diagnostics in Patients With Respiratory Tract Infections in Outpatient Care Related to the Peri-Launch Phase","id":"4e5f523c-cfe2-41ff-8081-22d7caa4283c","sessionCode":"MT7","topDisplay":"Steigenberger C1, Windisch F2, Vogler S2 1WHO Collaborating Centre for Pharmaceutical Pricing and Reimbursement Policies, Gesundheit Österreich (Austrian National Public Health Institute/GÖG), Vienna, 9, Austria, 2WHO Collaborating Centre for Pharmaceutical Pricing and Reimbursement Policies, Gesundheit Österreich (Austrian National Public Health Institute/GÖG), Vienna, Austria","locationCode":"5034","description":"\r\n\t<div>OBJECTIVES: Inappropriate use of antibiotics contributes to antimicrobial resistance, one of the key global health threats. Measures to reduce the overuse of antibiotics are urgently needed, particularly in the treatment of respiratory infections in community care settings. The introduction of rapid point-of-care diagnostics offers a promising solution. However, the use of these diagnostic tests is limited in most European countries. This study aims to identify the barriers and facilitators related to pharmaceutical policies in the peri-launch phase that could influence the use of rapid diagnostics for community-acquired respiratory infections, including health technology assessment (HTA), funding, pricing, and procurement. METHODS: We conducted case studies with expert interviews with representatives from public authorities in five European countries: Austria, Estonia, France, Poland, and Sweden. RESULTS: Hindering and facilitating factors for the reduction of inappropriate use of antibiotics are strongly influenced by the specific country context and health care system. In addition, findings specific to the different policy areas emerged: HTA processes face challenges due to insufficient evidence and the use of methods developed for medicines without appropriate adaptation to diagnostic tests. The level of funding for diagnostic tests was closely linked to their use. Pricing strategies relied primarily on indirect mechanisms such as procurement procedures, with Poland being the only country to implement a price cap. CONCLUSIONS: Strengthening policies in the peri-launch phase could improve funding mechanisms and facilitate evaluation of the benefits associated with the use of rapid diagnostics. This study highlights the need for tailored approaches that take into account the unique characteristics of each country's health care system to promote effective adoption of these technologies to combat antimicrobial resistance.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23steigenbergermt7poster132995-pdf.pdf?sfvrsn=e767e7f5_0","title":"ISPOREurope23_Steigenberger_MT7_POSTER132995.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132995","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Analysis of Average Treatment Costs for Atrial Fibrillation in a Tertiary Care Hospital of Nepal","id":"c5809cf9-6f63-4c33-b10e-23508ba83470","sessionCode":"EE2","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1039","description":"\r\n\t<div>OBJECTIVES: To analyze the average treatment costs for atrial fibrillation in a tertiary care hospital of Nepal METHODS: The study was performed as a retrospective cohort study from January 2018 to June 2023. All the AF hospitalized patients aged 18 years and older were identified from the database of hospital. Cost related information were obtained from Dhulikhel Hospital. RESULTS: Out of 64 patient 28(43.75%) patient were prescribed with warfarin and 36(56.25%) patients with NOACs (rivaroxaban, 51.56% and dabigatran 4.68%). The total treatment cost for patient prescribed with warfarin was $46.64 ± 36.59 per patient with median of 5 days of hospitalization whereas 49.64 ± 23.26 per patient with median of 4.5 days of hospitalization for NOACs. The largest portion of the expenses was attributed by hospitalization cost (83.904%) followed by laboratory investigations cost (10.624%) and drug cost (5.471%) in the patient prescribed with warfarin. Patient prescribed with NOACs also showed the similar fashion as hospitalization cost (69.32%) followed by laboratory investigations cost (23.85%) and drug cost (6.817%). Independent t test between two group reveled that there was no significant difference in cost of treatment between two groups (p value 0.697) at 95 % of confidence interval. CONCLUSIONS: It has been found that cost of drug and hospitalization cost is nearly similar but the laboratory cost related to warfarin is slightly higher comparative to NOACs.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132500","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"dc752772-538c-4933-b6a5-3b5b6d079673","parentId":"00000000-0000-0000-0000-000000000000","title":"Geriatrics","urlName":"Geriatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Tips for Excel(lent) Modelling or R There Better Alternatives?","id":"a0320a78-27f5-4067-ae73-2529fe62cf7d","sessionCode":"MSR32","topDisplay":"Maervoet J1, Poirrier JE2, Bergemann R3 1Parexel International, Wavre, Belgium, 2Parexel International, Wavre, WBR, Belgium, 3Parexel International, Loerrach, Germany","locationCode":"5068","description":"\r\n\t<div>OBJECTIVES: Whilst the use of R in health decision sciences has recently been increasing, Excel is still being used very commonly for development of health economic (HE) models. In the HE literature and textbooks, there is a paucity of hands‐on, practical guidance for the construction of efficient Excel models. In this study, we wanted to: 1) highlight strengths and limitations of Excel for development of HE models, 2) provide hands‐on practical tips to improve Excel’s efficiency, 3) consider when it may be better to use alternative software. METHODS: A targeted search was conducted to identify literature on the use / comparison of software for development of HE models. The Parexel HEOR modelling team convened a workshop to discuss findings of the literature review and share experience in April 2023. RESULTS: Excel has many built-in statistical and econometric functions that can be further extended using VBA. Excel can, however, become slow when complex HE models or computationally demanding analyses must be run. A list of tips for structuring, formatting, and coding efficient HE models in Excel has been put together and will be presented. Modern programming languages are better suited to conduct complex, computationally demanding, and/or real time analysis. Clear examples include value of information analysis, model calibration in dynamic transmission models, and making real-time updates to statistical analyses of patient data and the resulting HE outcomes. CONCLUSIONS: HE models are often built in Excel, owing to its wide-spread accessibility, user familiarity, and perceived transparency. Methodological and computational advances have allowed HE models to become more sophisticated and to better reflect clinical reality over time. Although modern programming languages are better suited to conduct complex, computationally demanding, and/or real-time analysis, ambiguity over the acceptance of such HE models by health technology assessment bodies remains a barrier to their adoption.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23maervoetmsr32poster131972-pdf.pdf?sfvrsn=24194c65_0","title":"ISPOREurope23_Maervoet_MSR32_POSTER131972.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131972","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Improving Analysis of Continuous Predictors: Advantages of Fractional Polynomial Transformations (FP) and Interpretation of “Non-Linear” Odds Ratios (OR) or Hazard Ratios (HR)","id":"4dbf34e0-4bc1-4801-ad7f-25351568a892","sessionCode":"MSR33","topDisplay":"Valveny N1, O´Mahoney T2, Alfonso V1, Mansilla I1, Shala A1 1TFS HealthScience, Barcelona, Spain, 2TFS HealthScience, Newbridge, Ireland","locationCode":"5073","description":"\r\n\t<div>OBJECTIVES: Assessing continuous predictors (e.g., cholesterol) with a categorical outcome (e.g., mortality) under a “linearity assumption” may lead to wrong decisions, especially if the variable is categorized (data-driven approach). We aimed to review methods for analyzing continuous predictors in regression models from RWE studies, to describe advantages of FP over traditional methods, and to develop a new visualization tool to help understanding “non-linear” ORs or HRs. METHODS: We searched in Pubmed RWE studies from the last 2 years (2020-2022) and summarized main methods, including advantages and limitations. We described FP methodology and developed a “FP-Risk Score Calculator” to translate model parameters into 10 risk zones (intuitive, traffic-light-like ranging from green to dark red) for the predictor values. RESULTS: Most common methods are, by decreasing order: 1) cut-points (49.7%); 2) untransformed variable (47.6%); 3) cubic-spline transformations (2.5%); 4) FP-transformations (0.2%). FP was the most efficient method selecting the best fit based on power and alpha error: 44 single or double-term transformations using “fraction powers” comprising most biologically plausible risk shapes (linear/non-linear, monotonic/unimodal). FP can be tested in univariate or multivariate models using a hierarchical function selection procedure, to select the best (and simplest) fit, which may also be linear (<a href=\"http://biom131.imbi.uni-freiburg.de/biom/mfp/\">http://biom131.imbi.uni-freiburg.de/biom/mfp/</a>). “Non-linear” OR/HRs are x-dependent, i.e., vary along X. We developed a “FP-Risk Score Calculator” to translate model parameters into 10 Risk Zones for the observed range of X values. Each zone is linked to 10 evenly divided outcome probabilities and (for simplicity) to the OR/HR of the mid-point. CONCLUSIONS: Continuous variables should not be assumed by default to have a linear relationship with the outcome nor categorized using pre-defined or data-driven cut-points. Systematic FP-transformations are easy to implement and allow selecting the best (and simplest) fit. The new FP-Risk Score Calculator divides predictor values into 10 risk zones to facilitate clinical interpretation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23valvenymsr33posterv2128301-pdf.pdf?sfvrsn=576b6db5_0","title":"ISPOREurope23_Valveny_MSR33_POSTERV2128301.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128301","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimating the Prevalence of Hepatitis Delta in the UK: A Migration Model Approach Applied in Practice","id":"2d91a26a-9a2c-495e-9382-26fffd708396","sessionCode":"EPH12","topDisplay":"Talbot-Watt N Gilead Life Sciences Ltd, London, LON, UK","locationCode":"3017","description":"\r\n\t<div>OBJECTIVES: Hepatitis delta (HDV) is a rare condition, affecting a subset of HBV+ patients. EMA have recently approved the first treatment for this condition. Prevalence of HDV in the UK is unknown. Objective of this research was to estimate prevalence of HDV in England, Scotland & Wales in preparation for local and national budget discussions with clinicians and payers. METHODS: Using a multivariate model framework outlined previously, population data for England, Scotland and Wales by country of origin were fed into the model to estimate HDV prevalence based on prior HBV and HDV prevalence by country of origin. Data sets were sought at individual regions within England and by health board within Wales & Scotland. RESULTS: Total HDV prevalence within England, Scotland and Wales was estimated at 12-14k, 900-1k and 200-300 patients respectively. Highest regional prevalence for HDV in England was found in London (~45% of total England prevalence), with top 5 countries of birth for HDV prevalence identified as Romania, Nigeria, India, Pakistan & Somalia, accounting for ~35% of overall HDV. The overall co-infection rate for HBV+ HDV was calculated at 7%, 6% and 5% for England, Scotland & Wales respectively. Results were highly dependent on granularity of population data by country of origin. CONCLUSIONS: HDV prevalence is highly dependant on migration populations and background rates of HBV infection. Overall across the UK, there are an estimated 14.5k HDV+ patients, however it is unlikely that the majority of these patients will be diagnosed / aware of their condition. Due to the diverse cultural backgrounds of potential patients, careful consideration will need to be given to how these patient access care and should be supported for their infection.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23talbot-watteph12poster129296-pdf.pdf?sfvrsn=6567c04d_0","title":"ISPOREurope23_Talbot-Watt_EPH12_POSTER129296.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129296","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Success Rate of Cancer Drugs Fund Reappraisals","id":"bcaa55cb-bc07-4ee0-93d8-28506db91d0b","sessionCode":"HTA9","topDisplay":"Liu X, Pannett S, Wallis J, Bertram J MAP Patient Access Limited, Cambridge, CAM, UK","locationCode":"4054","description":"\r\n\t<div>OBJECTIVES: The aim of this study was to investigate the outcomes of products that were recommended by the National Institute for Health and Care Excellence (NICE) for use within the Cancer Drugs Fund (CDF), and to provide pharmaceutical companies with insight on the success rate of NICE reappraisal after CDF exit. METHODS: A retrospective data analysis from the NICE website was conducted. 53 CDF recommendations and 27 CDF reappraisals were identified between 1 October 2016 and 14 April 2023. The status of each CDF recommendation was investigated on the website of NICE guidance, and each reappraisal was linked to the original appraisal to investigate the outcome of the CDF exit. RESULTS: Between 1 October 2016 and 14 April 2023, 53 products were recommended for use within the CDF, of which 25 (47%) are still in the CDF, 27 (52%) have been reviewed, and one (1%) has been withdrawn because the product no longer has a marketing authorization. Of the 27 products that have been reappraised after exiting the CDF, 9 (33%) were recommended for routine commissioning, 15 (56%) were recommended with restrictions for routine commissioning, two (7%) were not recommended, and one (4%) was terminated because the company did not provide an evidence submission. CONCLUSIONS: Following CDF exit, 89% reappraisals received a positive recommendation for routine use in the NHS, indicating that additional data collection and commercial re-negotiations reduced uncertainty enough for NICE to make a positive recommendation. If there are data uncertainties in the appraisal process, engaging early with NICE can provide an opportunity to allow medicines to be made accessible despite these uncertainties. The CDF provides an access route and allows the generation of data to address these data uncertainties, ensuring optimized access for patients and reimbursement for companies, rather than postponing the appraisal process to allow for additional clinical data generation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/success-rate-of-the-cancer-drugs-fund-reappraisals-v1-12oct2023129176-pdf.pdf?sfvrsn=9d3bd11_0","title":"Success rate of The Cancer Drugs Fund reappraisals v1 12Oct2023129176.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129176","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Understanding Equity Considerations in HTA Decisions for CAR-T Treatments and the Path to Inclusive Access","id":"1e6e21e9-b12a-4994-8516-28bcd3d094f5","sessionCode":"HPR22","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3075","description":"\r\n\t<div>OBJECTIVES: Chimeric antigen receptor T-cell (CAR-T) therapies are innovative treatments that have shown remarkable efficacy in rare or hard-to-treat cancers. Studies involving CAR-T treatments often involve limited clinical evidence availability and small sample sizes that pose challenges in assessing the equity implications under current health technology assessment (HTA) processes. This review aimed to evaluate limitations within the clinical evidence base submitted to HTA agencies and document equity issues observed in CAR-T submissions. METHODS: Published appraisals from NICE (UK), ZiN (Netherlands), SMC (Scotland), CADTH (Canada), HAS (France), MSAC (Australia), TLV (Sweden), AIFA (Italy), IQWiG/GBA (Germany) and NIPH (Japan) available on agency websites were screened (06/2023) to identify CAR-T submissions. Appraisals were reviewed and extracted in a standardized manner to analyze patient characteristics from clinical studies against critique documented by HTA agencies regarding equity concerns. Equity considerations for the same CAR-T therapy and indication were compared across agencies. RESULTS: Fifty-one HTA appraisals were identified, comprising six CAR-T therapies over ten indications. Two of the ten agencies (NICE, CADTH) had a section within appraisal documents outlining equity issues. Twenty appraisals (39%) across four agencies (NICE, HAS, CADTH, MSAC) referenced equity considerations within the appraisal. Fourteen appraisals (27%) critiqued the underrepresentation in clinical evidence across demographics (e.g., age, ethnicity, socioeconomic status, geography). Common issues raised across appraisals involved: geography and access to treatment centers (39%), age restrictions (31%), ethnic minority underrepresentation (14%), and out-of-pocket costs (6%). CONCLUSIONS: Equity considerations were documented in a substantial proportion of HTA appraisals involving CAR-T treatments, especially regarding geography and age restrictions. These findings emphasize the need for improved representation of diverse patient populations in CAR-T clinical studies and the importance of addressing equity concerns within HTA processes to expand access to these transformative therapies. Real-world evidence transportability can play a crucial role in informing decision-makers about patients traditionally underrepresented in clinical trials.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133633","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Targeted Literature Review on the Economic and Quality of Life Burden Caused by Acne","id":"b9fa3ef6-1750-492a-bef8-2932a50c8d71","sessionCode":"EE33","topDisplay":"Proshenska D1, Bougeard C1, Fraisier B2, Kwong X3, Alvarez F2 1Syneos Health, Montrouge, France, 2Sanofi, Lyon, France, 3Sanofi, Hong Kong, Hong Kong","locationCode":"1073","description":"\r\n\t<div>OBJECTIVES: Acne affects approximately 3% of the global population. This study aimed at analyzing the economic and humanistic burden of acne, providing a detailed view for a limited subset of countries, while identifying critical gaps in health economics data. METHODS: A targeted literature review in PubMed and EMBASE was conducted on the economic, quality of life (QoL) and psychological (PS) burden of acne in the USA and five major European countries (EU5). Publication years were restricted from 2010 until 2022. RESULTS: 34 studies on acne costs, and 139 on acne QoL and PS were analyzed. In 2019, acne affected 231 million people worldwide (95% CI, 208 – 255 million), resulting in 4.96 million disability adjusted life years (DALYs) (95% CI, 2.98 – 7.85 million). Direct costs primarily arise from prescription medications and healthcare visits, which highly depend on treatment choices, adherence, and duration. The main factors affecting cost variability include female gender, younger age, disease severity, and choices of treatment. Acne significantly impacts QoL and psychological well-being, with increased anxiety and depression scores reflecting its substantial PS burden. Critical gaps exist in economic and clinical severity data, cost-effectiveness of acne treatment, and information on acne-associated indirect costs. The underreporting of PS burden highlights the need for targeted interventions. CONCLUSIONS: Acne imposes a significant economic and QoL burden to patients, healthcare systems, and societies. This burden is primarily driven by direct medical costs, including medication costs, and physician visits, and indirect costs associated with reduced productivity and QoL. Effective acne prevention and treatment strategies are essential in mitigating this burden. It is worth noting that a systematic literature review was beyond the scope of this study.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/acne-ispor-poster-stcfinal133555-pdf.pdf?sfvrsn=838a482b_0","title":"Acne+ISPOR+Poster+STC_final133555.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133555","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Budget Impact of the Jada System for the Treatment of Patients with Abnormal Post-Partum Bleeding or Hemorrhage in the United States","id":"4a96d850-eba5-464c-b215-2974f1025a74","sessionCode":"EE128","topDisplay":"Yong C1, Fox A2, Hirst A2, Hughes R2, Mountain G2, Seal B1, Rood KM3 1Organon, Jersey City, NJ, USA, 2Adelphi Values PROVE, Bollington, UK, 3Ohio State University, Columbus, OH, USA","locationCode":"2062","description":"\r\n\t<div>OBJECTIVES: Postpartum hemorrhage (PPH) is the leading cause of maternal morbidity and mortality globally. With increasing rates of PPH, a novel intrauterine vacuum-induced hemorrhage-control device has been developed for treatment. Our objective was to estimate the budget impact (BI) of adding this device (intervention) as a treatment for PPH. METHODS: A BI model (1-year time horizon) was developed to compare the costs of treating PPH patients from the hospital perspective. Two alternate treatment scenarios were defined: current practice (1) without the intervention and (2) with the intervention. Patients in the model received uterotonic drugs +/- nonsurgical treatments (uterine balloon tamponade (UBT) or the intervention), and can then progress to subsequent surgical procedures, followed by hysterectomy, with potential maternal death. Probabilities of treatment progression and health resource use (HRU) were sourced from clinical trial data and published literature. Cost inputs were estimated from real-world analyses of US Premier hospital database (2016-2022). Model inputs were varied by blood loss categories to assess the impact of earlier vs. later intervention use. RESULTS: In a hypothetical cohort of 10,000 births, 1,470 (14.7%) had PPH requiring treatment with at least 1 second-line uterotonic. The intervention scenario had lower HRU compared to UBT when considering ICU admission, rate of hysterectomy, and major blood transfusions with a reduction of 2%, 2% and 4.7% respectively. Total BI varied depending on the proportion of patients receiving the intervention in each blood loss category. There were cost offsets associated with the intervention due to lower rates of major blood transfusion, ICU admission and hysterectomy compared to UBT; with estimated annual cost savings of -$981,633, -$305,066, -$161,114 respectively. CONCLUSIONS: The device offers a valuable treatment option for patients with PPH, due to potential cost savings from a hospital perspective given the earlier positioning, ahead of invasive treatment options and major HRU.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023posterjada-bimfaa2130671-pdf.pdf?sfvrsn=b09aeba9_0","title":"ISPOR 2023_Poster_Jada BIM_FAA2130671.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130671","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Reductions in Travel Time and CO2 Emissions Arising From Patient Transportation to Hospitals in Ireland for Treatment With Intravenous Iron: An Analysis Comparing Ferric Derisomaltose With Ferric Carboxymaltose","id":"ff0db829-43ae-4351-b8e0-2a44026f25fb","sessionCode":"EE114","topDisplay":"Ahmed W1, Greer T2, Murphy K2, Bird I2, Fahy J2, Pollock R1 1Covalence Research Ltd, Harpenden, HRT, UK, 2Pharmacosmos UK Ltd, Reading, Berkshire, UK","locationCode":"2042","description":"\r\n\t<div>OBJECTIVES: Intravenous (IV) iron is the preferred treatment for patients with iron deficiency anemia (IDA) requiring rapid iron replenishment, or in whom oral iron is contraindicated, not tolerated, or ineffective. Two high-dose, rapid-infusion IV iron formulations are available in Ireland: ferric derisomaltose (FDI) and ferric carboxymaltose (FCM). Dosing differences between formulations can drive differences in the number of infusions required to treat IDA. We aimed to quantify the effect of these differences on patient travel time, distance, and annual carbon dioxide (CO2) emissions in the IDA population in Ireland. METHODS: A list of public Irish hospitals was obtained from the Health Service Executive. The mean distance from 18,627 “small areas” defined by The National Institute of Regional and Spatial Analysis to the nearest hospital was calculated, adjusted using a detour index, and weighted by population. Transport modality and CO2 emissions data were obtained from Government sources. Numbers of FCM and FDI infusions were modeled using a published and validated methodology, combined with annual estimates of patients receiving IV iron treatment, and integrated into the transportation model to calculate mean changes in patient travel time, distance, and CO2 emissions associated with using exclusively FDI versus exclusively FCM. RESULTS: FDI would reduce mean iron infusions per treatment course by 0.42 from 1.77 to 1.35 (23.7%) versus FCM. Based on a multi-modality transport model to Irish hospitals, FDI was projected to reduce the mean total annual distance travelled by 235,500 km (standard deviation [SD]: 53,847) from 992,465 km (SD: 226,926 km) to 756,965 km (SD: 173,079 km) versus FCM, saving 4,688 hours (SD: 1,077 hours) of patient time and 43,400 kg (SD: 10,026 kg) of CO2 emissions. CONCLUSIONS: Compared with FCM, FDI reduced the number of IV infusions required, substantially reducing travel time, distance, and travel-related CO2 emissions for patients with IDA in Ireland.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ahmed-ireland-co2-ispor-poster-2023-10-30133437-pdf.pdf?sfvrsn=d4cb1120_0","title":"Ahmed Ireland CO2 ISPOR Poster 2023 10 30133437.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133437","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Incidence, Prevalence, and Clinical Characteristics of Patients with Chronic Lymphocytic Leukemia (CLL) in Spain: Natural Language Processing (NLP) Analysis of Electronic Health Records (EHRs)","id":"e01745a1-3dfe-4abb-a54a-2a584e49b99d","sessionCode":"MSR10","topDisplay":"Hernandez JA1, Bahar N2, Bas C3, Prieto Patron A4, Ortiz M5, Castillo E6, Gutierrez A7 1University Hospital Infanta Leonor, Madrid, Spain, 2BeiGene International, GmbH, Basel, Switzerland, 3BeiGene ESP, SL, Madrid, Spain, 4BeiGene International, GmbH, Pully, Switzerland, 5Regional University Hospital Malaga, Malaga, Spain, 6Savana Research, Madrid, Spain, 7Lymphoma Unit, Department of Hematology, Son Espases University Hospital/IdISBa, Palma, Spain","locationCode":"5056","description":"\r\n\t<div>OBJECTIVES: Unlike registries or claim-based real-world data, NLP analysis of EHRs analyzes diverse datasets, thereby reducing selection bias to provide accurate patient data. This study used free-text data extracted with NLP from EHRs to determine the incidence, prevalence, and primary clinical characteristics of CLL in Spanish patients to reduce knowledge gaps and enhance disease management. METHODS: This was a multicenter, retrospective study in adult patients with CLL from 1 Jan 2016 to 31 Dec 2021 in 3 Spanish hospitals. EHRs were evaluated using EHRead technology, a data-driven system based on NLP and machine learning, according to clinical terminology (SNOMED CT). Incidence and prevalence were estimated, and descriptive statistics were determined for 205 variables. RESULTS: A total of 697 patients with CLL were included in the study, out of a population of 2,069,341 patients with a total of 88,872,628 EHRs. The overall age-standardized (2013 European population) incidence and prevalence for the study period were 3.38 (95% CI, 2.55-4.22) and 49.81 (95% CI, 47.65-51.98) cases per 100,000 person-years, respectively. The mean age was 72.1 (SD, 12.6) years and 299 patients (42.9%) were female. Among patients with available information, most were smokers or ex-smokers (76.9%) and 38.2% were alcohol drinkers. The most common clinical alterations observed were lymphocytosis (65.9%), lymphadenopathy (35.4%), anemia (26.3%), infections (18.4%), thrombocytopenia (14.2%), and splenomegaly (13.9%). Eastern Cooperative Oncology Group performance status (ECOG PS) was reported in 13.5% of patients, most frequently ECOG PS 0 (43.6%). Cytogenetic alterations were reported in 27.5% of patients. CONCLUSIONS: This study presents comprehensive information on patients with CLL in Spain, obtained through NLP analysis of EHRs. These findings confirm the clinical characteristics described in the literature and reinforce the role of artificial intelligence and NLP as reliable methods for studying disease incidence and prevalence.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23prietomsr10poster130040-pdf.pdf?sfvrsn=47302fb9_0","title":"ISPOREurope23_Prieto_MSR10_POSTER130040.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130040","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Methodology for the Development of a Data Collection Tool","id":"e3050cae-1c59-4242-940e-2ad2b58ce518","sessionCode":"RWD17","topDisplay":"Gonzalez Durio J1, Hex N2 1Becton Dickinson, San Agustín del Guadalix, M, Spain, 2York Health Economics Consortium, York, UK","locationCode":"6069","description":"\r\n\t<div>OBJECTIVES: While carrying out time motion studies, a need was identified to reduce the burden of data collection during the preparation of IV treatments in aseptic units across UK hospitals. The objective was to build a tool to harmonise the process of data collection, allowing standardisation and basic data analysis. METHODS: York Health Economics Consortium were engaged to explore solutions. Support was provided by an on-site qualified pharmacist, who co-developed data collection sheets and verified data quality. Treatment preparation stages were identified and mapped, and workflow was assessed. A paper data collection sheet was designed to record times per task and errors made. An Excel Spreadsheet was developed to store data and perform basic data analysis, such as preparation time per medication per working day; time per task; times per working day and task. This had the advantages of in-time data collection and the facilitation of basic data analysis. However, there was heavy reliance on technicians accurately recording data, with the risk of errors during data collection and transcription phases. To address this the spreadsheet evolved to a ´Google Sheets´, with the same advantages, as well as the ability to input data mimicking the paper sheet, reducing the risk of errors during the transcription phase, and being accessible from different sites to facilitate multicenter studies. RESULTS: The tool has been developed and successfully used with data from different sites and different therapeutical fields (Chemotherapy, IV Antibiotics and Parenteral Nutrition), requiring adaption due to variation in tasks in the three different workflows. Staff reported ease of use of the standarised paper sheets. CONCLUSIONS: The tool developed facilitates the collection and analysis of data from multiple centres. Other areas of the hospital (beside the aseptic unit) will benefit from the use of a similar tool, such as the central dispensary and hospital wards. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gonzalezduriorwd17poster134077-pdf.pdf?sfvrsn=f8f71bc9_0","title":"ISPOREurope23_GonzalezDurio_RWD17_POSTER134077.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/134077","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"G-BA Vs. InEK: Navigating Divergent Requirements Between the Two Key Bodies Behind Inpatient Drug Reimbursement in Germany","id":"4b7299ca-9f67-4aee-9d6e-2b9769f18566","sessionCode":"HTA35","topDisplay":"Macaulay R1, Hartikainen P2, Wang C2, Currell D2 1PRECISIONadvisors, Edinburgh, UK, 2PRECISIONadvisors, London, LON, UK","locationCode":"4038","description":"\r\n\t<div>OBJECTIVES: In Germany, there are two national bodies that determine the pricing and reimbursement of inpatient drugs. The G-BA conducts a benefit assessment that precedes a price negotiation whilst InEK establishes the price and appropriate funding route. This research explores the relationship between the G-BA and InEK in driving successful reimbursement outcomes. METHODS: A quantitative analysis of inpatient drugs in the 2022 NUB list plus a 60-minute qualitative interview with a German payer advisor. RESULTS: 100 NUB outcomes were identified: 77% achieved NUB 1 (positive outcome) and 23% achieved a NUB 2 (negative outcome). Of these, 69% received an additional benefit and 31% received no additional benefit from the G-BA. Notably, there were discrepancies between favourable outcomes by InEK vs. those by the GBA: 28.6% of therapies that received NUB 1 received no additional benefit whilst conversely, 60.9% of therapies that received NUB 2 received additional benefit. Payer insights revealed that the G-BA and InEK operate independently with different criteria. The G-BA focuses on added clinical benefit, which informs national price. In contrast, InEK assesses a drug’s novelty, whether it can be funded from DRGs, and how justified the cost-differential is. The two bodies only convene if the nationally negotiated price is lower, which is then also applied by InEK. After at least 5 years, the DRG may be updated to absorb the cost differential. CONCLUSIONS: The contrasting decision-making processes complicate patient access to inpatient drugs in Germany, with the G-BA and InEK performing their assessments according to different criteria. To secure inpatient reimbursement following the G-BA’s benefit assessment, manufacturers must also ensure they meet InEK’s criteria, assess and justify their drug’s cost-differential, prepare a well-written submission, and ensure all hospitals apply annually with the same application.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23macaulayhta35poster133212-pdf.pdf?sfvrsn=c5bd7da9_0","title":"ISPOREurope23_Macaulay_HTA35_POSTER133212.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133212","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Unleashing the Power of Social Media Listening: A Promising Tool for Patient-Focused Research","id":"2064a2e7-f3d6-4dc1-85c7-2c331123e865","sessionCode":"PCR55","topDisplay":"Gautam R1, Purkayastha P2, Sharma R3, Srivastava T1 1ConnectHEOR, London, UK, 2ConnectHEOR, Delhi, India, 3ConnectHEOR, Edmonton, AB, Canada","locationCode":"6050","description":"\r\n\t<div>OBJECTIVES: Social media (SM) platforms generated posts provide a unique and extensive source for generating patient insights and perspectives. This study aims to analyze the trends in using SM for patient insights and perspective generation in recent years. METHODS: A systematic literature review was conducted in PubMed to identify studies reporting patient insights and perspective generated using SM listening (SML) methodology from January 2012 to March 2023. Only full-text articles published in English were included. Themes that were discussed by patients or their caregivers were analyzed. RESULTS: Of the 1,088 articles retrieved, 37 studies met the inclusion criteria. Eight studies (22%) analyzed SM posts from one country (US, 50%; France, 25%; UK, 12.5%; Malaysia, 12.5%), 14 (38%) from multi-countries, whereas country was unclear in 15 (40%). Approximately half of the studies (43% [n=16]) analyzed posts from one SM platform, while others utilized multiple platforms. Twitter (57% [n=21]) emerged as the most used SM source for posts, followed by Forums (41% [n=15]), Instagram (32% [n=12]), Facebook (27% [n=10]), Blogs (22% [n=8]), Reddit (16% [n=6]), YouTube (14% [n=5]), and Tumblr (11% [n=4]). The studies comprised several therapeutic areas, including cancers (19% [n=7]), neurological diseases (14% [n=5]), skin problems, inflammatory diseases (each 11% [n=4]), eye diseases (8% [n=3]), genetic, and rare diseases (each 5% [n=2]). Six studies (16%) were about treatments/surgery. Patients discussed dominant themes were treatments/disease management (51% [n=19]), symptoms (43% [n=16]), diagnosis (30% [n=11]), seeking disease information, psychological impact (each 27% [n=10]), quality of life, physical impact (each 24% [n=9]), treatment outcomes/side-effects, impact on work/activities of daily living (each 22% [n=8]), emotional impact (19% [n=7]), and social impact (16% [n=6]). CONCLUSIONS: With the increasing demand for patient-centric research in decision-making process, SML represents an unexplored opportunity for generating valuable patient insights by exploring untold experiences and outcomes that can complement data collected through traditional methods.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gautampcr55poster131350-pdf.pdf?sfvrsn=143f033a_0","title":"ISPOREurope23_Gautam_PCR55_POSTER131350.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131350","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Study of the Mental Health of Pharmacists During Wartime in Ukraine","id":"7b748bff-01fc-4c32-8c9f-2c390fafab89","sessionCode":"RWD8","topDisplay":"Maksymovych N, Zaliska O, Semenov O Danylo Halytsky Lviv National Medical University, Lviv, Ukraine","locationCode":"6061","description":"\r\n\t<div>OBJECTIVES: According to the data of national survey in 2022, almost 71% of people felt severe stress due to wartime. The main causes were: war, financial difficulties, deterioration of health. Only 41% of Ukrainians have a satisfactory state of their own mental health. During the wartime, pharmacists are the first to communicate with people who feel anxiety, fear, and irritation. METHODS: Survey of pharmacists using the Google forms, analysis of the list of medicines for stress treatment. RESULTS: We conducted a survey of 125 pharmacists about the state of their mental health during wartime in March-May, 2023. Pharmacists noted the main 5 conditions (52%-75% of the respondents) anxiety, poor sleep, lack of motivation, depression, eating disorders. We found that 78% of pharmacists are not productive while working in a pharmacy, and 28% of pharmacists are unable to continue working due to attention deficit disorder. It was found that 35% of pharmacists experienced mood dysfunction due to the war due to poor sleep, because there is a great fear of not hearing the alarm signal about the danger at night. Only 25% of pharmacists informed employers about their inability to work due to mental health problems. Pharmacists from community pharmacies noted that the following OTC medicines for improving mental health are most often used to treat stress: magnesium preparations, St. John's wort, combined herbal medicines. However, 42% of pharmacists with an average level of stress don’t experience relief of symptoms from the use of OTC sedatives CONCLUSIONS: We established that during the wartime, 82% of pharmacists noted a significant impact of the war on their mental health, social activities. Also, 42% of pharmacists noted that concomitant diseases progress against the background of stress. So there is a need for additional professional training to improve the mental health of pharmacists.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23maksymovychrwd8poster-132213-pdf.pdf?sfvrsn=9dd553c5_0","title":"ISPOREurope23_Maksymovych_RWD8_POSTER 132213.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132213","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Data Standardization in Brazil: An OMOP Common Data Model Approach in a DATASUS Cohort","id":"f1c1978a-7c62-4082-a20d-2c9185d34bec","sessionCode":"PT7","topDisplay":"Oliveira JCB1, Julian G2, Maruyama JM1 1Precision Data, São Paulo, SP, Brazil, 2Pfizer, Sao Paulo, Sao Paulo, Brazil","locationCode":"4B","description":"\r\n\t<div>OBJECTIVES: Common data models (CDM), such as Observational Medical Outcomes Partnership (OMOP), are crucial tools to develop quick multi-site and country real world evidence generation. Therefore, this study aims to describe the methods and quality results of the creation of an OMOP dataset with linked outpatient and inpatient information of Brazilian claims data. METHODS: This study included data from Hospital Information System (SIH) and Outpatient Information System (SIA) from January 2008 until May 2023. The information available in the datasets include all outpatient procedures, consultations, ICD-10 codes of a primary and secondary diagnosis, medicines, and encrypted/anonymized personal data, such as date of birth, residential address, zip code, and sex. All original records from SIA an SIH databases were analyzed to assess the consistency of information for a unique patient key. Patients with inconsistencies in primary key or basic information were excluded from the database. After the cleaning and pre-processing stage, a deterministic linkage algorithm was developed to connect inpatient with outpatient records using the key information of zip code, date of birth, and gender. Subsequently, this dataset underwent transformation into the OMOP CDM. The data quality check was conducted with DataQualityDashboard version 2.1.1 RESULTS: A total of 5.82 million patients were included in the final dataset. In this dataset, there were 3,666 queries tested assessing the quality mapping of the OMOP database. In those queries, the database reached plausibility, conformance and completeness verifications of 99%, 93% and 97%, respectively, reaching an overall pass rate of 98%, indicating a satisfactory index of the mapping quality. CONCLUSIONS: Our study provides the first results of a high-quality cohort with outpatient and inpatient information in Brazil in OMOP format. CMD approaches in Latin America are very scarce and may be a crucial tool to boost real world evidence generation in the region.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023oliveirapt7poster133843-pdf.pdf?sfvrsn=e84ead06_0","title":"ISPOREurope2023_Oliveira_PT7_POSTER133843.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133843","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Value of Looking Beyond Overall Survival: The Role of Oncology-Relevant Endpoints in HTA / Payer Decision-Making","id":"107e3356-880d-4b99-ba49-2d113146a572","sessionCode":"HTA6","topDisplay":"Fameli A1, Fernandez C2, Krueger T3, Podkonjak T4, Cattaneo I5, Rotaru M6, Ryan J7, McLaughlan B8, Marques S9, Calzada V10, Carreras M11, Sapede C12, Gross-Langenhoff M13, Čaić F14 1GSK, London, London, UK, 2Sanofi, GENTILLY, France, 3MSD, Luzern, Switzerland, 4Takeda Pharmaceuticals International AG, Zurich, ZH, Switzerland, 5Novartis Farma S.p.A, Origgio (VA), Italy, 6European Federation of Pharmaceutical Industries and Associations, Brussels, Belgium, 7AstraZeneca, Cambridge, CAM, UK, 8Astellas Pharma Europe, Surrey, Surrey, UK, 9AstraZeneca, Zug, Zug, Switzerland, 10Sanofi, Paris, France, 11F. Hoffmann -La Roche Ltd., Basel, Basel, Switzerland, 12Novartis, Basel, Basel, Switzerland, 13Astellas Pharma Europe, Munich, Munich, Germany, 14Merck Group, Brussels, Brussels, Belgium","locationCode":"4049","description":"\r\n\t<div>OBJECTIVES: HTA bodies / payers have used overall survival (OS) as the main measure of a medicine’s efficacy. However, reliance on OS poses three challenges. First, time to mature, statistically significant OS is longer in clinical trials for early-stage cancers than for metastatic disease. Second, the vulnerability of OS to confounding during this time may not accurately reflect the benefit of a medicine assessed solely on OS data. Finally, other endpoints more effectively measure important outcomes for patients or outcomes that better characterise the disease and treatment setting. As such, there is a need to improve value recognition of oncology-relevant endpoints (OREs) beyond OS in HTA / payer decision-making. METHODS: This study investigates perceptions of OREs by 13 interviews with physicians, patient advocacy groups and former payers in US and Europe; 3 roundtable discussions; and a literature review. RESULTS: OREs beyond OS, including time-to-event, response-related endpoints and patient-reported outcomes, have value as earlier indicators of a medicine’s efficacy and as standalone indicators of clinical and patient benefit. Although many OREs beyond OS are accepted by regulators, HTA bodies / payers may undervalue these endpoints due to perceived uncertainty they translate into long-term benefits for patients and healthcare systems. Several actions are recommended. Early cross-stakeholder engagement should agree the most suitable OREs by disease and treatment setting, evaluate the existing evidence base supporting the surrogate or standalone value and identify research needs to address gaps. Alignment between stakeholders can then inform standardized methodologies to collect them, ensuring data consistency and comparability. CONCLUSIONS: Increased value recognition of OREs beyond OS in HTA / payer decision-making can improve timely access to life-improving medicines, ensure optimal outcomes for patients. Cross-stakeholder collaboration can support increased ORE incorporation in value assessment frameworks and HTA / payer decision-making that results in timely patient access to innovation medicines. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor23famelithe-value-of-looking-beyond-overall-survivalthe-role-of-oncology-relevant-endpoints-in-hta-and-payer-decision-making131581-pdf.pdf?sfvrsn=16a1aa17_0","title":"ISPOR23_Fameli_The value of looking beyond overall survival_The role of oncology-relevant endpoints in HTA and payer decision-making131581.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131581","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Qualitative Analysis of Value-Based Contract Experience, Perception, and Future Adoption across Geographies","id":"2ab821a0-ae5c-4dfb-8a29-2d3b3dacc7e4","sessionCode":"HTA63","topDisplay":"Sathi C1, Dumoulin O2, Mestre-Ferrandiz J3, Pascual-Argente N4, Grandy A5, Bento G6, Chouman-Arcas S7, Towle P8, Bechara A6 1Alira Health, Barcelona, B, Spain, 2Alira Health, Basel, BS, Switzerland, 3Independent Economics Consultant, Universidad Carlos III de Madrid, Madrid, Spain, 4Universitat Pompeu Fabra, Barcelona, B, Spain, 5Alira Health, Paris, France, 6Alira Health, Basel, Switzerland, 7Takeda, Zurich, Zurich, Switzerland, 8Takeda Pharmaceuticals International AG, Zürich, ZH, Switzerland","locationCode":"5021","description":"\r\n\t<div>OBJECTIVES: Value-based healthcare have gained significant attention as a promising approach to improve patient outcomes and overcome reimbursement barriers while managing costs. This study aimed to explore the current state of VBCs, assess payer experiences and perceptions, and analyze future adoption. METHODS: Insights gathered from in-depth interviews (N=20) conducted with payers (public, private, national, regional) from Europe (Germany, Italy, The Netherlands, Sweden, and Spain), Latin America (Argentina, Brazil, Colombia, and Mexico), Asia-Pacific (APAC) (Australia, China, South Korea, and Taiwan), and the US were qualitatively analyzed. RESULTS: The findings highlight diverse experiences with VBCs across geographies. In Europe, payers expressed familiarity with VBCs and highlighted positive outcomes, such as improved patient outcomes and enhanced collaborations. However, they also emphasized the administrative burden, impeding broader adoption and leading to negotiations focused on simple price discounts. In Latin America and APAC, the experience varies with the maturity and the capacities of the healthcare system. Less mature markets are more familiar with financial-based agreements (FBAs) allowing for access equity and costs containment. Due to constrained data infrastructures, the adoption of outcome-based agreements (OBAs) remains limited despite acknowledged benefits. Whereas, in Australia, there is a trend to move away from OBAs due to the associated implementation burden and costs. The US payers showed a mixed interest in VBCs, with reservations about the need and the financial benefit. Overall, challenges such as data sharing, outcomes measures, trust, and complex financial flows, were identified as critical factors influencing future adoption of VBCs. While there is more interest in FBAs, there is still an opportunity for OBAs in specific disease areas with expensive drugs i.e., rare diseases CONCLUSIONS: VBC use is influenced mainly by financial and data capabilities. Stakeholders should address challenges while focusing on standardizing performance measures and promoting collaboration to foster wider adoption of VBCs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/sathi-c-et-al-qualitative-analysis-of-vbc-experience-perception-and-future-adoption-across-geographies-poster130133-pdf.pdf?sfvrsn=4c7ae473_0","title":"Sathi C et al Qualitative Analysis of VBC Experience Perception and Future Adoption Across Geographies Poster130133.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130133","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Analysis of the Change of the Added Benefit and Respective Reasons in Renewed Regular Benefit Assessments of Orphan Drugs Due to Exceeding the Sales Threshold in Germany","id":"49f47741-1193-4fcb-81ba-2e17b242b68d","sessionCode":"HTA29","topDisplay":"Brückel S1, Reindl S1, Tran TT2 1IGES Institut GmbH, Nuremberg, BY, Germany, 2IGES Institut GmbH, Berlin, Berlin, Germany","locationCode":"4060","description":"\r\n\t<div>OBJECTIVES: Orphan drugs (ODs) have privileges in the early benefit assessment (EBA) and the subsequent price negotiation (PN) in Germany. The added benefit (AB) is acknowledged by law, no appropriate comparator therapy (ACT) is defined. If the sales of the OD exceed a sales volume threshold (SVT) of 30m€ (50m€ until Nov 2022), the product must undergo a regular EBA. The objective of this study was to determine the change of the AB (cAB) and respective reasons after exceeding the SVT. METHODS: The results of all completed EBAs due to exceeding the SVT regardless the status of the PN by March 2023 were analyzed. The cAB and respective reasons were determined using qualitative text analysis. The analysis of cAB was carried out at subpopulation level. RESULTS: A total of 39 subpopulations were analyzed. In 5 subpopulations, there was an increase/quantification of the AB due to the submission of new evidence, whereas in 2 subpopulations the AB was no longer quantifiable. In 11 subpopulations the AB remained the same despite the submission of new evidence in 2 cases, and in 21 subpopulations the extent of AB decreased. In case of a decrease of AB, a RCT of the assessed drug was often available in the orphan procedure. However, that comparison was not adequate for deriving an AB in the exceeding procedure due to short study duration or compared to procedural demands in Germany inadequate ACT. CONCLUSIONS: The analysis showed that there are several obstacles that arise when an OD is being faced with a regular EBA. The most common obstacle was the lack of AMNOG-eligible trials. The in most cases available RCT were often not suitable because the ACT was not adequately implemented. Pharmaceutical companies should consider a possible exceeding of the SVT and the resulting requirements when planning pivotal studies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23brueckelhta29poster128851-pdf.pdf?sfvrsn=882d0954_0","title":"ISPOREurope23_Brueckel_HTA29_POSTER128851.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128851","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Efficiency Management of Immunization Programs in Peru, in the Context of the COVID-19 Pandemic: The Fully Liquid Acellular Hexavalent Vaccine","id":"a4a00d38-c1f7-44e5-92ac-2e7703d2ceb8","sessionCode":"EE135","topDisplay":"Mendoza MA1, Gutiérrez-Aguado A2, Upegui A3, Sarazu T4, Londono S5 1MK Producciones, Lima, Peru, 2MK Producciones, Lima, LIM, Peru, 3Sanofi, Bogota, Colombia, 4Sanofi, Lima, Peru, 5Sanofi, Bogota, CUN, Colombia","locationCode":"2072","description":"\r\n\t<div>OBJECTIVES: The World Bank and Pan American Health Organization, recommend the use of combined vaccines to improve the efficiency of National Immunization Programs (NIP). Studies suggest that the introduction of the fully liquid hexavalent vaccines and allows a more efficient resource allocation by freeing approximately 52% of the required storage capacity in comparison to the pentavalent vaccine + IPV scheme. This study aims to evaluate cold-chain optimization opportunities and the efficiency impact of introducing a fully liquid acellular hexavalent vaccine in the Peruvian NIP METHODS: The potential benefits associated with the introduction of the fully liquid acellular hexavalent vaccine were evaluated through a literature review and a cost analysis based on published literature, by comparing the total cost associated with the inclusion in comparison to the actual scheme (pentavalent vaccine + IPV). The analysis included vaccine acquisition, infrastructure, equipment, waste (final disposal), logistics (medical and office equipment), and home visit costs. Total costs were estimated for a target population of 475,000 infants under one year old. RESULTS: Total cold-chain costs per infant were estimated at USD$3.13 and USD$15.94 for the complete fully liquid acellular hexavalent and pentavalent vaccine schemes respectively. Therefore, once infrastructure, equipment, waste, logistics, and home visit costs are included in the analysis, the fully liquid acellular hexavalent vaccine represents potential savings of around 80% in comparison to the pentavalent vaccine + IPV scheme. For the analysis population, total savings could potentially reach USD$6,000,000. Additionally, freed storage capacity in the cold chain can be used for other vaccines. CONCLUSIONS: Adopting international recommendations helps guaranteeing the cold-chain availability of vaccines. This is particularly crucial, since the COVID-19 pandemic showed the need of an efficient cold-chain, for better attending national vaccination needs. The introduction of the fully liquid hexavalent vaccine allows a larger vaccination rate by decreasing the resources required and minimizing total costs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23londonoee135poster132307-pdf.pdf?sfvrsn=c75a81aa_0","title":"ISPOREurope23_Londono_EE135_POSTER132307.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132307","diseases":[{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Technology Assessment Requirements: Sensitive to Bacteriophage Products or Too Resistant to Change?","id":"88a43b57-64aa-4929-97f4-2ee69de8d162","sessionCode":"HTA3","topDisplay":"Strydom M1, Jouini A2, Jaros P1, Francois C3 1Putnam PHMR, London, UK, 2Putnam PHMR, Tunis, Tunis, Tunisia, 3Putnam PHMR, Paris, Paris, France","locationCode":"4039","description":"\r\n\t<div>OBJECTIVES: Antimicrobial resistance is a global threat. Phage Therapy Medicinal Products (PTMPs) can help address this crisis by providing an effective treatment against resistant bacteria, but a clear regulatory and market access framework is lacking. PTMPs can be prêt-à-porter (pre-formulated ‘fixed drugs’ with marketing authorisation [MA]) or sur-mesure (personalised magistral preparations). The aim is to determine if current HTA processes are suitable to deal with both types. METHODS: Methods guidance by HAS (France), G-BA (Germany), NICE (UK) and national policy documents were reviewed. Information was extracted on HTA processes and funding pathways. Eligibility to these were mapped to PTMP characteristics to determine fitness for purpose. Two analogues (larval debridement therapy [LDT], faecal microbiota transplant [FMT]) were analysed regarding HTA and funding. RESULTS: Each of the HTA bodies (HTABs) implemented antimicrobial-specific processes. G-BA exempts from HTA a product qualifying as a “Reserve Antibiotic”. HAS published bespoke evidence requirements for “last resort” antibiotics. NICE is piloting a new antimicrobial-specific process. The analogue review shows deviations between regulatory and funding positions. In all three countries, LDT and FMT are available. From a regulatory perspective both are ‘drugs’, yet only LDT is marketed as a drug (BioBag®), while FMT is available as a procedure. NICE assessed FMT (as a ‘Medical Technology’) but not LDT. Both are available on the NHS. HAS and G-BA assessed LDT but not FMT. LDT is routinely funded in Germany but not in France, following a negative HAS recommendation. In both Germany and France, FMT is only funded on a named-patient basis. CONCLUSIONS: Existing HTA frameworks focus on antibiotics, excluding other innovative antimicrobials. HTABs only assess drugs with MA, thus are not prepared to assess sur-mesure PTMPs. A clear regulatory and access pathway should be established at European level to facilitate adoption of PTMPs and sustained national funding pathways for sur-mesure PTMPs created.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/clement-francois2023health-technology-assessment-requirements-sensitive-to-bacteriophage-products-or-too-resistant-to-change132614-pdf.pdf?sfvrsn=8bf2fd28_0","title":"Clement Francois_2023_Health Technology Assessment Requirements Sensitive to Bacteriophage Products or Too Resistant to Change132614.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132614","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Role of EUnetHTA 21 in Promoting Patient Engagement","id":"8382d2eb-8667-4473-ba7e-2f359ed347d2","sessionCode":"PT5","topDisplay":"Alshaikheid M1, Dimassi M2, Omer U1, Borges S1, Doran R1 1Putnam PHMR, Newcastle Upon Tyne, Tyne and Wear, UK, 2Putnam PHMR, Soukra, 11, Tunisia","locationCode":"5A","description":"\r\n\t<div>OBJECTIVES: We aimed to evaluate patient engagement in the evolving European health technology assessment (HTA) process through exploring the current level of engagement and its impact, assessing the approaches taken by EUnetHTA to promote engagement, and identifying challenges and future perspectives. METHODS: A targeted review of patient engagement in the HTA process was conducted via analysis of EUnetHTA deliverables, guidance documents, methodologies, and updates from EUnetHTA and European Commission websites. RESULTS: Patient engagement in EUnetHTA initiatives is limited, from the perspective of stakeholders. However, EUnetHTA reported that patient input in early dialogue had valuable impact on engagement recommendations. In previous joint actions, EUnetHTA conducted 7 joint scientific consultations (JSCs), with 6 involving patients at the European level, and 2 joint clinical assessments (JCAs) for medical devices. The first published JCA mentioned that patients were consulted early in the scoping process. When establishing their current work plan, EUnetHTA and the European Medicines Agency prioritised development of methodologies for patient engagement in HTA. Deliverable D7.2/3 provides guidance for engaging patient representatives in HTA organisations. The European Regulation on HTA (HTAR) established a stakeholder network with 44 member organisations, including patient associations, and 2 observers. The EU4Patients project offers support by updating training content, designing an e-learning course, developing interactive training sessions for JCA and JSC, and implementing sustainability measures. Challenges remain, including lack of capacity and resources, expertise and training, alignment of organisations, conflict management, shared valuation of patient input, and pharmaceutical industry concerns regarding incorporation of stakeholder feedback. CONCLUSIONS: Current patient engagement in EUnetHTA initiatives remains limited, despite efforts from EUnetHTA. HTAR presents a unique opportunity to enhance engagement. To strengthen this framework and ensure efficient HTA, potential future measures could include promoting and allocating resources for patient training projects and establishing communication channels between national patient groups across Europe.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133125","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Matching Adjusted Indirect Comparison (MAIC) of Selpercatinib Vs Cabozantinib in RET Mutation-Positive Advanced Medullary Thyroid Cancer (MTC)","id":"a29e329f-5754-4392-add6-2f6dc439c80c","sessionCode":"CO21","topDisplay":"Jen MH1, Kiiskinen U2, Khanal M2, Han Y2, Hess L2, Tian W2, Vickers A3 1Eli Lilly and Company, Uxbridge, LON, UK, 2Eli Lilly and Company, Indianapolis, IN, USA, 3RTI Health Solutions, Manchester, LAN, UK","locationCode":"1015","description":"\r\n\t<div>OBJECTIVES: To estimate comparative effectiveness, in terms of objective response rate (ORR), progression free and overall survival (PFS, OS) using unanchored MAIC of single-arm LIBRETTO-001 (selpercatinib) and EXAM trials (cabozantinib vs placebo) in advanced/metastatic RET mutation-positive MTC. METHODS: Patient-level data from LIBRETTO-001 were weighted to match summary data from the cabozantinib arm of the EXAM trial. ORR and PFS were available for the RET mutation-positive cohort but OS only for the RET M918T subgroup in EXAM. Cohorts were balanced on all available baseline covariates (age, weight, performance status, sex, smoking status, prior tyrosine kinase inhibitor therapy, and RET M918T mutation status). Hazard ratios (HR) for PFS and OS, odds ratios (OR) for ORR, and related 95% CIs were estimated. Multiple imputation marginalization was performed as a sensitivity analysis for PFS and OS outcomes. RESULTS: A total of 295 who received selpercatinib (LIBRETTO-001) and 107 patients who received cabozantinib (EXAM) were included. Before weighting, ORR, PFS and OS comparisons suggested favoring selpercatinib. Weighting resulted in balanced distributions of baseline covariates. After weighting, ORR was 82.9% and 31.7% (OR=10.46[95%CI 6.24,17.55, p<.0001]) for selpercatinib vs cabozantinib, respectively. 12-month PFS rates are 89% and 54%, and HR=0.08 (95%CI:0.05,0.13, p<0.001) for selpercatinib vs cabozantinib. Respective 24-month OS rates are 92% and 66%, and HR=0.20 (95%CI: 0.13,0.32, p<0.001). CONCLUSIONS: In weighted comparison, selpercatinib demonstrated a significant improvement in ORR, PFS and OS versus cabozantinib. A limited number of covariates were available for adjusting selpercatinib data. Hence, there is a risk of unmeasured confounding that could influence these findings. Additional uncertainty is due to EXAM OS data in RET M918T subgroup while MAIC used covariates, including proportion of RET M918T, from RET mutation-positive patients. These findings should be interpreted with caution considering the limitations of an unanchored MAIC.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23jenco21poster131973-pdf.pdf?sfvrsn=e2c808b5_0","title":"ISPOREurope23_Jen_CO21_POSTER131973.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131973","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evaluation of the Economic Impact of Initial Diagnostic Modality Selection for Patients Suspected of Having Colorectal Cancer Liver Metastases or Hepatocellular Carcinoma in the UK","id":"e4ef5aa1-194e-483f-945d-2fbf300ba45a","sessionCode":"EE52","topDisplay":"Corbin A1, Blankenburg M2, Harris J3, Taylor S3, Ozgen Z4, Siddiqui I4, Elhamamy M2 1Wickenstones Ltd, Oxford, OXF, UK, 2Bayer AG Pharmaceuticals, Berlin, Berlin, Germany, 3Wickenstones Ltd, Carlow, County Carlow, Ireland, 4Bayer Public Limited Company, Reading, Berkshire, UK","locationCode":"2005","description":"\r\n\t<div>OBJECTIVES: Hepatocellular cancer (HCC) is the most common type of primary liver cancer; in addition, the liver is the most frequent site of metastatic disease for colorectal cancer. This study compared costs of diagnosis and treatment of HCC (primary liver cancer) and colorectal cancer liver metastasis (CRCLM; secondary liver cancer) in a UK setting, according to initial diagnostic imaging modality selection. METHODS: Modalities assessed included: gadoxetate disodium (ethoxylbenzyl-diethylenetriaminepentaacetic acid)-enhanced magnetic resonance imaging (EOB-MRI), extracellular contrast media enhanced-MRI (ECCM-MRI), multidetector computed tomography (MDCT) and contrast-enhanced ultrasound (CEUS). Individual decision tree models were developed to simulate the clinical pathway for HCC and CRCLM, from first diagnostic test to initial treatment decision, based on local clinical guidelines and validated by experts. Input values were derived from the literature as well as from interviews with local experts. RESULTS: Comparisons between the modalities demonstrated that initial selection of EOB-MRI delivered cost savings per patient versus alternative options. This result was consistent across HCC and CRCLM. Cost offsets with EOB-MRI were driven by a reduced requirement for follow-on procedures, both in terms of diagnosis and therapy, leading to reductions in costs related to contrast agents, scans, biopsies, and unnecessary treatments. Budget impact scenarios, where the proportionate use of different initial imaging modalities was varied, showed that increasing use of EOB-MRI was consistently associated with lowered costs. CONCLUSIONS: These findings demonstrate that, in the UK setting, initial selection of EOB-MRI in the diagnosis of primary and secondary liver cancers results in cost savings compared to other available options.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23harrisee52poster129521-pdf.pdf?sfvrsn=8df1bca2_0","title":"ISPOREurope23_Harris_EE52_POSTER129521.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129521","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Analysis of the Use of the Haute Autorité De Santé (HAS) Opinions by Hospital Pharmacists in France","id":"f0d1c283-7317-4301-83d3-2fc4cfcccad1","sessionCode":"EE124","topDisplay":"Moutier H1, Paubel P2, Fusier I3, Degrassat Theas A2 1University of Caen, Caen, Calvados, France, 2General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP) ; Law and Health Economics Department, Faculty of Pharmacy & Health Law Institute (INSERM UMR S1145) University of Paris, Paris, Ile-de-France, France, 3General Agency of Equipment and Health Products (AGEPS), Assistance Publique-Hôpitaux de Paris (AP-HP), PARIS, France","locationCode":"2055","description":"\r\n\t<div>OBJECTIVES: In France, the Haute Autorité de Santé (HAS) is in charge of the healthcare products assessment. For more than 10 years, in addition of the clinical assessment, economics studies are required by the HAS for the national evaluation of innovative healthcare products. For these products, HAS publishes transparency opinion and economic opinion based on clinical dossier and cost-effectiveness model (and budget impact model), respectively. This survey analyses the use of these HAS opinions by hospital pharmacists in France, in particular in the context of drugs’ referencing in hospitals. METHODS: This study was based on an electronic questionnaire distributed between December 2022 and February 2023 and shared with hospital pharmacists practicing in France. Results such as percentage of HAS opinions uses, information used from HAS opinions and hospital pharmacists’ needs were described in this study. RESULTS: In total, 89 pharmacists have responded to the survey. Among this sample, more than 76% use the clinical opinion from the HAS (Transparency dossier) and only 11% use the economic opinion from the HAS (cost-effectiveness analysis) in their practice. They declared to use HAS opinions for drugs’ referencing, to support health professionals’ discussions and/or to argue the choice or adaptation of therapeutic strategies. CONCLUSIONS: Even if the hospital pharmacists of this survey declared to be interested by economic issues, the economic opinions from the HAS are much less used than transparency opinion. This observation may reflect the difficulty of using national economic opinions by local payers such as hospital pharmacists in the context of healthcare product referencing. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132403","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Identifying Evidence Gaps for Hemophilia B by Developing a Factsheet Using Targeted Review of Literature: Shaping the Future Real‑World Evidence Studies","id":"b7f86507-9402-4e8b-b1e7-318c65ddfab3","sessionCode":"CO143","topDisplay":"Kaushik P, Arya S, Singh N, Nanda S, Grover R Quantify Research, Sahibzada Ajit Singh Nagar (Mohali), PB, India","locationCode":"1028","description":"\r\n\t<div>OBJECTIVES: Owing to the shrinking budget due to the global economic downturn, evidence from sources other than randomized controlled trials is being accepted, especially in the case of rare diseases. Factsheet is one such compilation of the available literature for a disease, based on targeted literature review. We aimed to develop a factsheet for hemophilia B for population in the United States (US) that will identify evidence gaps for shaping the future real‑world evidence (RWE) studies. METHODS: A targeted search was performed using Embase® on 21-April-2023, for hemophilia B in the US population. The categories for data-points were epidemiology, demographics, disease specific, comorbidities, mortality, treatment patterns, health care resource utilization (HCRU), quality of life (QoL) and disease management (hemophilia treatment centers [HTCs] and self-care). Title/abstract (TIAB) followed by full text screening was performed for inclusions and data-points were extracted from the included publications in the predesigned factsheet (Microsoft Excel). Publications/reports for extraction were prioritized based upon the most recent year of publication followed by the sample size. Ad-hoc google searches were performed for the data-points which were still missing. Percentage gaps were calculated by dividing the count of missing data-points by the total data-points evaluated for a particular category. RESULTS: A total of 151 records were retrieved. After TIAB and full text screening a total of 32 publications were included. In-depth analysis of publications led to data extraction from 12 publications/reports (9/3: Embase®/Google). All data-points related to the categories of demographics, comorbidities, and mortality were identified. However, gaps existed in data-points related to HTCs and self-care (100%), QoL (75%), epidemiology (62.9%), disease-specific (50%), treatment patterns, (28.5%) and HCRU (23.5%). CONCLUSIONS: The evidence indicates huge gaps (≥50%) in disease management, QoL, epidemiology, and disease-specific related data-points for hemophilia B in the US. These gaps can help in shaping and planning future RWE studies. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/co143-posterhemophilia-factsheetfinal132959-pdf.pdf?sfvrsn=efe8319_0","title":"CO143 Poster_Hemophilia factsheet_final132959.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132959","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Artificial Intelligence (AI) Tools for Outbreak Detection and Response: A Transnational Platform for Surveillance, Monitoring and Decision Support","id":"06250034-2431-4a9c-98b6-32421f7fd0af","sessionCode":"EPH51","topDisplay":"Génin M1, Botz J2, Coudeville L3, Eisenlauer M4, Fauvel T1, Huschka F4, Lambert N1, Wang D2, Bosch Castells V3, Commaille-Chapus C5, Frandji B6, Haberstroh M4, Robin JY5, Roehn P7, Sippel H4, Thiele P7, Thommes E8, Weber C4, Amzal B1, Fröhlich H2, Kannt A9, Mahé C3 1Quinten Health, Paris, Paris, France, 2Fraunhofer SCAI, Sankt Augustin, Germany, 3Sanofi, Lyon, France, 4umlaut consulting GmbH part of accenture, Aachen, Germany, 5Impact Healthcare, Paris, France, 6CompuGroup Medical, Nanterre, France, 7Docmetric GmbH, Koblenz, Germany, 8Sanofi Vaccines, Toronto, ON, Canada, 9Fraunhofer ITMP, Frankfurt, Germany","locationCode":"3050","description":"\r\n\t<div>OBJECTIVES: Numerous digital surveillance tools were developed during the COVID-19 pandemic to support public health decisions. Yet, those ad-hoc tools were essentially for short-term insights, more reactive than predictive, and not generalisable to be used to predict hospital capacities or shortages of medical supplies in real-time. To overcome these limitations, we developed, under a public-private consortium, a transnational predictive platform to detect the first signs of respiratory epidemics, monitor progression, and assist in defining and evaluating appropriate measures. METHODS: We developed AIOLOS, a web-based AI-powered monitoring and decision support tool, using integrative modeling and simulations informed by multiple data sources (wastewater, social media, mobility data). Trained for now on COVID-19 historical data in France and Germany at both regional and national levels, this platform aims at detecting early signs of a new viral epidemic, monitoring its spread, and informing public health decisions to optimize its social, public health and economic impact. RESULTS: The first project year was dedicated to the development, testing and calibration of models while engaging and aligning across partners and stakeholders. A first version of the tool was developed and dashboarded with visuals, delivering promising preliminary results , e.g. highlighting and quantifying the value of wastewater data in predicting pandemic waves, and the impact of social distancing and vaccination on epidemics. CONCLUSIONS: Improvements will be made in the coming year e.g. by integrating new data sources and partners, accounting for more pathogens and respiratory viruses and enabling real-time prediction updates. AIOLOS stands as a serious candidate to become an EU-wide public health decision support tool.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-numero-132618---aiolos-ispor132618-pdf.pdf?sfvrsn=37792866_0","title":"Poster numero 132618 - AIOLOS ISPOR132618.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132618","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Cost‑Effectiveness Analysis of Adjuvant Osimertinib in Patients with Resected EGFR Mutation‑Positive Non‑Small Cell Lung Cancer in Portugal","id":"7a51e4a8-a809-40c5-b9a3-33664559eff9","sessionCode":"EE72","topDisplay":"Andrade A1, Tavares AL2 1Astrazeneca, Barcarena, Oeiras, Portugal, 2Astrazeneca, Lisboa, Portugal","locationCode":"1094","description":"\r\n\t<div>OBJECTIVES: Osimertinib is a third-generation, irreversible, oral, EGFR tyrosine kinase inhibitor (TKI) that potently and selectively inhibits EGFR sensitizing mutations and the TKI resistance-conferring EGFR point mutation T790M. It is the standard of care for advanced EGFRm Non‑small Cell Lung Cancer (NSCLC) and was approved for the adjuvant treatment of adult patients with early-stage NSCLC. The aim of this analysis was to assess the cost-effectiveness of adjuvant osimertinib compared to active surveillance in the treatment of patients with resected EGFR mutation‑positive NSCLC, in the Portuguese setting. METHODS: A five-health-state transition model with time dependency was developed to estimate lifetime costs and survival of resected EGFRm patients treated with adjuvant osimertinib or active surveillance, with/without prior adjuvant chemotherapy, using a Portuguese National Health Service perspective. Transitions between health states were modeled using ADAURA (NCT02511106, DCO1 January-2020) and FLAURA (NCT02296125) trials data, Portuguese life tables, and real-world data (CancerLinQ Discovery). The model used a ‘cure’ assumption: patients remaining disease free for 5 years after treatment completion for resectable disease were considered ‘cured.’ EQ-5D-5L trial data was converted to utilities based on Portuguese tariffs. Healthcare resource utilization estimates were derived from Portuguese literature. RESULTS: Adjuvant osimertinib treatment was more effective than active surveillance leading to a mean 2.16 additional quality-adjusted life-years (QALYs) (8.53 vs 6.37) per patient. The modeled median percentage of patients alive at 10 years was 63.5% versus 41.3%, respectively. Osimertinib was associated with mean added costs of 30,514€ per patient and a cost/QALY (incremental cost-effectiveness ratio) of 14,130€ versus active surveillance. Model robustness was demonstrated through scenario analyses. CONCLUSIONS: In this cost-effectiveness assessment designed for the Portuguese setting, adjuvant osimertinib was considered cost-effective compared with active surveillance for patients with completely resected stage IB‒IIIA EGFRm NSCLC. The robustness of the model results was demonstrated with sensitivity analysis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23andradeee72poster128433-pdf.pdf?sfvrsn=f4dacf6_0","title":"ISPOREurope23_Andrade_EE72_POSTER128433.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128433","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Burden of Respiratory Syncytial Virus Infection in Germany: A Systematic Review","id":"9865510c-3a39-4041-94e8-3418a6ff09a7","sessionCode":"EPH14","topDisplay":"Poshtiban A, Wick M, Bangert M, Damm O Sanofi-Aventis Deutschland GmbH, Berlin, BE, Germany","locationCode":"3016","description":"\r\n\t<div>OBJECTIVES: Respiratory syncytial virus (RSV) is a major cause of acute lower respiratory infections and hospitalizations among infants, children, and the elderly. This systematic literature review aimed to summarize and quantify the epidemiological and economic burden of RSV infection at any age in Germany. METHODS: We conducted a systematic literature search in PubMed and Embase to identify full-text articles published from 2003 to 2023 and reporting data on the epidemiological or economic burden of RSV in Germany. Based on pre-specified eligibility criteria, data on incidence, rates of hospital and intensive care unit (ICU) admission, clinical manifestation, comorbidity profile, seasonality as well as health care resource use and costs were extracted. RESULTS: We included 36 articles in the review. The characteristics of the included studies were heterogenous regarding study population, setting, age groups and outcome measures. The majority of included studies focused on describing the disease burden in the pediatric population. Frequently reported epidemiological outcome measures were comorbidities (n=7), clinical manifestation (n=13), and seasonality (n=14). Twenty-eight articles stated RSV detection rates (across heterogenous study populations), ranging from 0.1 to 55.4%. All articles that reported the RSV detection rate across all age groups demonstrated the highest burden in infants and young children. Health care resource use of patients with RSV infections were analyzed in 18 articles, of which only one study quantified associated costs. ICU admission rates among RSV-related hospitalizations were reported in 9 articles, ranging from 5.1 to 10.1% among the overall population, and from 23 to 45% among children with a history of prematurity or underlying chronic diseases. CONCLUSIONS: This systematic review revealed that RSV causes substantial disease burden in infants, young children, and the elderly in Germany, highlighting the need for RSV prevention in these age groups. Further studies quantifying RSV-associated direct and indirect costs are needed.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poshtiban2023eph14burden-of-rsv-infection-in-germany130415-pdf.pdf?sfvrsn=e27632b7_0","title":"Poshtiban_2023_EPH14_Burden of RSV infection in Germany130415.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/poshtiban2023eph14burden-of-rsv-infection-in-germanysuppl130415-pdf.pdf?sfvrsn=ee7dea11_0","title":"Poshtiban_2023_EPH14_Burden of RSV infection in Germany_Suppl130415.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130415","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Multistakeholder Healthcare Cooperative: A New Paradigm for Healthcare Delivery","id":"bec49d3c-ad79-490d-8b86-3448ca28be48","sessionCode":"OP5","topDisplay":"Javadekar N1, Javadekar AN2 1MMRS-Maharashtra Medical Research Society,MMFHA Joshi hospital Pune, Erandawane, Pune, India, 2D.Y.Patil Medical College, Pimpri, Pune, Maharashtra, India","locationCode":"5079","description":"\r\n\t<div>OBJECTIVES: To analyse healthcare system with financial engineering perspective to optimise the solution . The healthcare delivery system in India is facing challenges to meet sustainable development goals by 2030. Government has a shortage of funds and 60% of the population pays from pocket for health care. This pushes many on the borders below the poverty line due to catastrophic out-of-pocket health expenditures.Healthcare demand and costs are rising due to the aging population and newer health technologies. Increased Expectations from medical treatment lead to frustration when the treatment fails to deliver desired outcomes, which leads to cases of violence against the medical community. METHODS: The healthcare delivery system was analyzed using a Financial engineering perspective with the objective of optimising the outcomes . Three major areas of financial engineering that are most relevant to the risk analysis/management needs of health care organizations are stochastic analysis and value at risk,portfolio optimisation and asset liability management,and distributed decision making and agency theory.Governments desire to maximize the health of the citizens at minimum costs, and people desire good health. Healthcare providers such as doctors and hospitals are assumed to be working towards maximizing consumption. Diagnostic centers and the pharma industry are purely profit-oriented. RESULTS: The multistakeholder system is a complex system and requires the meaningful cooperation of all the players to optimize the outcomes. Stakeholder management(STM) lies at the heart of FIR(fourth industrial revolution).Thus cooperative society may be a better model for delivery of health care in a complex multistakeholder environment with uncertain outcomes. CONCLUSIONS: Co-operatives in healthcare may offer solutions to the shortage of funds,lack of access, and also help deliver inclusive and patient-centered care. India has had a rich experience in the cooperative sector and there is a scope for introducing healthcare cooperatives as a novel model of healthcare delivery, to achieve universal health coverage by 2030.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"bb8fd992-de86-4d1a-8bec-f6e2c928db96","parentId":"00000000-0000-0000-0000-000000000000","title":"Organizational Practices","urlName":"organizational-practices"}],"categoryIds":["bb8fd992-de86-4d1a-8bec-f6e2c928db96"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-euro23-javadekar-op5133765-pdf.pdf?sfvrsn=5e2111f3_0","title":"ISPOR EURO23 Javadekar OP5133765.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133765","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost of Illness in Patient With Colorectal Cancer in México (2022)","id":"dfbdda40-f294-4dc2-a190-34b649271a47","sessionCode":"EPH27","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3030","description":"\r\n\t<div>OBJECTIVES: The purpose of this study was to estimate the total medical costs of colorectal cancer patients between symptom onset, seeking care, and initiation of treatment, and factors that interfere with these processes. Including 229 patients, where the direct costs of all patients in the gastroenterology area with a diagnosis of colorectal cancer. METHODS: Descriptive correlational study, including 229 patients, where the direct costs of all patients in the gastroenterology area with a diagnosis of colorectal cancer seen at INCAN from January to December 2022 were estimated. Data on hospital admissions, emergency department and outpatient visits, and drug prescriptions in 2022 following colorectal cancer diagnosis were obtained from administrative databases. The outcomes measured were the average actual costs per patient during this 12-month period, calculated and stratified by stage of disease at diagnosis, tumor histology, and tumor site. Costs are expressed in 2022 USD. RESULTS: Of 229 patients investigated the majority were male (59.8%), with mean age 58 years (IQR 49-69 years). The mean total time between symptom onset and treatment initiation was 3.64 months and the mean time between seeking medical care and diagnosis was 3.34 months. The average total medical care cost per patient was $18,055.56 USD. The costs of systemic treatment of advanced colorectal cancer were driven primarily by drug acquisition and administration costs. CONCLUSIONS: This study showed that the longer interval between seeking medical care and diagnosis was possibly caused by negative association between presenting symptoms and disease, causing more advanced stages of disease which are significantly more costly to manage. The present results may serve as a reference for future economic evaluations of treatment strategies for patients with colorectal cancer.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132309","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost of Illness (COI) Associated with Direct Antiviral Agents (DAAs) for the Treatment of Hepatitis C Viral (HCV) Infection in India: A Systematic Literature Review","id":"984f0cfc-5c24-462f-9c3f-35703def4f28","sessionCode":"EE123","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"2058","description":"\r\n\t<div>OBJECTIVES: Identification of costs associated with different DAAs regime through COI evaluation helps inform policy decisions and pharmacoeconomic analyses. This systematic literature review (SLR) aimed to identify real-world cost data related with HCV treatment. METHODS: A literature search on Embase, and MEDLINE (Ovid) from January 2010 to May 2023 was conducted, to find cost related studies for DAAs against HCV infection published in English language and focusing India. Additional grey literature search included Google scholar and conference abstracts (AASLD, EASL, APASL, INASL). Retrieved studies were screened by two reviewers and conflicts resolved by third reviewer. Quality of included studies was assessed by using Drummond, New-Castle Ottawa Scale, and JBI checklists. Direct costs included costs on treatment, diagnosis, food, and accommodation. Indirect cost included loss of work and other intangible costs. RESULTS: A total of 146 studies were retrieved and 13 studies were extracted. Five studies were observational, six pharmacoeconomic analyses, and two database studies. Across studies, dialysis (52%) was common reason for infection, and GT-3 (45.2%-63.4%) was frequent genotype. Mean drug cost/patient for 12 weeks with sofosbuvir+velpatasvir was INR43,447±21,247 in private and INR13,183±136 from state-government, with sofosbuvir+daclatasvir INR30,702±17,847 in private and INR4,019±849 from state-government, and with sofosbuvir+ledipasvir INR22,755±9,268 in private setting and INR9,576±0 from state-government. Mean diagnostic cost/patient was INR12,609±7,312 and INR2,687±16 in private and public setting, respectively. Overall, mean direct cost/patient with DAAs for 12 weeks was INR60,530±13,766 in private and INR27,119±17,304 in public setting. Mean indirect cost/patient was INR5,355±2,323 with HCV infection. No study for voxilaprevir and its combination was identified. CONCLUSIONS: This SLR showed significant difference between market price (private) and state-government price for DAAs. High cost of DAAs is still a hinderance in HCV infection eradication. There was significant heterogeneity in costs among studies, so there is need to conduct more real-world studies on costs associated with HCV infection.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129092","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing the Validity of Observational Filters for Systematic and Rapid Literature Reviews","id":"90f8051c-ab78-451f-9c81-3670bed06425","sessionCode":"SA16","topDisplay":"Smela B1, Świerk K1, Tusińska A1, Clay E2, Boyer L3, Toumi M3 1Assignity, Kraków, Poland, 2Clever-Access, Paris, France, 3Aix-Marseille University, Marseille, France","locationCode":"7023","description":"\r\n\t<div>OBJECTIVES: Search filters are predetermined combinations of phrases that have been developed to obtain a subset of records based on specific topics of interest. The objective was to create and test two filters supporting the identification of observational studies: one that could be used when running rapid reviews (RRs), containing only the main terms related to these types of studies (’basic’), and the second one, more sensitive, that could suit well systematic literature reviews (SLRs), named ‘comprehensive’. METHODS: The basic filter was created by combining keywords used in the observational search filters published by 3 identified sources: Scottish Intercollegiate Guidelines Network, British Medical Journal Knowledge Center, and OVID expert searches. In the comprehensive filter, keywords published by the Canadian Agency for Drugs and Technologies in Health were additionally included. The filers were tested using Cochrane’s SLRs as a ‘gold standard'. RESULTS: The performance of the filters was satisfactory – for the comprehensive filter, the coverage of available data was over 92%, while for the basic filter, which could be implemented in RRs aiming to synthesise key trends in a cost-efficient manner, the coverage was 77%, with most of the studies falling in the range between 84-96%. The use of the basic filter was associated with a significantly higher reduction of workload compared to the comprehensive filter – between 2 and 7 workdays. Studies were omitted mostly due to poor indexing and not using relevant keywords in abstracts. 63% of studies missed by the comprehensive filter were published prior to the year 2000. CONCLUSIONS: Effective evidence retrieval is critical for performing high-quality evidence synthesis to support healthcare decision-making. Implementing the methodological filters into the search strategy significantly reduces the workload, and therefore generates savings in time and costs. However, it is important to underline that restricting the strategy may lead to omitting relevant data sources.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133611","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Dupixam: An Algorithm to Identify Patients with Atopic Dermatitis Among Dupilumab-Treated Population Using the SNDS (SYSTEME NATIONAL DES DONNEES DE SANTE) in France","id":"371f0045-3ada-4583-9eb8-36b1f690db4b","sessionCode":"RWD26","topDisplay":"Allali N1, Vataire AL1, Thenié C1, Oger E2 1Sanofi, Gentilly, Paris, France, 2CHU Rennes, Rennes, Bretagne, France","locationCode":"7000","description":"\r\n\t<div>OBJECTIVES: Two studies were conducted to characterize French patients treated with dupilumab for moderate-to-severe atopic dermatitis (AD). MOVE was an observational, multicenter, transversal study conducted between 2019 and 2021 and included 628 patients treated with dupilumab for AD. DUPIXAM was an SNDS (exhaustive French claims database) study on patients initiating dupilumab 300mg between 2019-2020. Because dupilumab was also indicated in asthma and nasal polyps (CRSwNP), an algorithm was developed to identify patients with AD in DUPIXAM. MOVE was used to validate the DUPIXAM algorithm. METHODS: From the 3,900 patients aged 18+ with ≥1 dupilumab 300mg delivery, AD, asthma or CRSwNP markers were first identified (pharmacy, specialist consults, inpatient diagnosis coding) in the 10 years before dupilumab. A second markers screening in the single previous year allowed to extract moderate-to-severe AD patients among patients who presented both AD and asthma or CRSwNP markers. To determine whether patients had moderate-to-severe AD or severe asthma among patients with markers of both diseases in the previous year, systemic treatment was investigated: methotrexate, mycophenolate mofetil, azathioprine and cyclosporine were defined as AD-specific and mepolizumab, benralizumab and omalizumab as asthma-specific. Among patients presenting treatments with both omalizumab and cyclosporine, the frequency of AD-specific markers was investigated last. The linkage with MOVE allowed to validate the DUPIXAM algorithm. RESULTS: 3,216 patients were identified as moderate-to-severe AD: in the past 10 years, 846 presented markers of AD only; in the previous year, 1,399 other patients presented exclusively markers of AD, and 971 were identified through systemic treatments and frequency of AD markers. The linkage of DUPIXAM with MOVE showed a sensibility from the algorithm of 92,1%. Male patients with moderate-to-severe AD represented 52.8% of the population and were aged 45.2 ± 19.4, whereas female patients were aged 40.2 ± 17.6. CONCLUSIONS: The algorithm developed in DUPIXAM showed a good robustness and sensibility.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23allalirwd26poster130273-pdf.pdf?sfvrsn=3f2bb234_0","title":"ISPOREurope23_Allali_RWD26_POSTER130273.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130273","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Focus on Two Years of Economic Opinions on Solid Cancers: What Are the Specific Features?","id":"65947d6c-19fc-40ce-846a-375a74960599","sessionCode":"HTA12","topDisplay":"Sambuc C1, Boussahoua M2, Tehard B2, Midy F2, Chevalier J2, Roze S3 1VYOO Agency, Paris, 75, France, 2Vyoo Agency, Paris, 75, France, 3Vyoo Agency, VILLEURBANNE, 69, France","locationCode":"4040","description":"\r\n\t<div>OBJECTIVES: In France, the Commission for Economic and Public Health Evaluation (CEESP) provides an economic opinion of the incremental cost-effectiveness ratio (ICER) validity. Since 2016, due to the arrival of immunotherapies, treatment for solid tumor has taken a central position in marketing authorization and reimbursement. The many published opinions in this therapeutic area are likely to create anchoring and learning effects. The aim of this study is to analyze the CEESP opinions in oncology appraised over the last two years (2021-2022). METHODS: Using Vyoo Agency efficiency database, all CEESP opinions in oncology appraised between January 1st, 2021, and December 31st, 2022, were reviewed. An opinion is valid if there are no mention of major uncertainty or objection. RESULTS: Over the two years, 26 CEESP opinions concerned drugs (25) or medical devices (1) for a solid tumor on 12 different tumoral sites. They included neither major objection nor global uncertainty for 69% (18/26) of the dossiers. For those 18 dossiers, the average ICER is €153,245/QALY and ranges from €33,110/QALY to €300,000/QALY and one opinion concluded that the drug is dominated. Of the 26 economic opinions, 15 concerned an anti-PDL1, 13 of which have a validated ICER (87%). For these opinions, validated results vary from dominated to 300,000. The rate of opinions validating economic information for treatments based on another mechanism of action is much lower (45%). For these opinions, the ICER variation is between €169,780/QALY and €298,148/QALY. CONCLUSIONS: Over the two years considered for the analysis, CEESP has delivered 55 opinions, including 21 with a validated ICER (38%). Opinions on solid tumor comprised 47% of all opinions appraised during this period and they are characterized by a higher proportion of validated opinions (69%), especially regarding anti-PDL1 (87%). To conclude, it seems that the central place of oncology confers advantages to meet the CEESP expectations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023isporhta12130166-pdf.pdf?sfvrsn=a54cf755_0","title":"2023_ISPOR_HTA12130166.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130166","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Risk, Use, and Cost of Infection-Related Hospitalization of Individuals With Alzheimer’s Disease or Dementia","id":"fa60a910-96eb-45f7-ba68-37a838387640","sessionCode":"EE27","topDisplay":"Cheng JS1, Hsu JY1, Ku HP2 1Chang Gung University, Taoyuan, Taiwan, 2Chang Gung University, Tao-Yuan, Tao-Yuan, Taiwan","locationCode":"1072","description":"\r\n\t<div>OBJECTIVES: Individuals with Alzheimer’s disease or dementia require long-term and intensive care, leading to high medical expenditures and disease burden, which can be exacerbated by severe infections. However, little is known about the risk of severe infections among individuals with Alzheimer’s disease or dementia. Therefore, this study aimed to examine their patterns of severe infections, and identify factors associated with the risk, number, and cost of infection-related hospitalization. METHODS: Individuals with Alzheimer’s disease or dementia in 2019 were identified from the National Health Insurance (NHI) claims data, and 10,000 subjects were randomly selected from them for further analysis. Four types of infections were included: septicemia, bacterial infection, fungal infection, and viral infection. Logistic regression model was adopted to identify factors associated with the risk of infection-related hospitalization. Generalized linear models were used to examine factors associated with number and cost of infection-related hospitalization. RESULTS: The majority of the sample were more than 80 years old and female. 17% of them had infection-related hospitalizations in 2019. The most common infection type was bacterial infection, followed by septicemia. The average number and cost of the hospitalization were 0.27 and US$4,359, respectively. Older age, being male, being poor, higher Charlson Comorbidity Index score, worse continuity of care, and history of infection-related hospitalization were associated with a higher risk of infection-related hospitalization. Being male and history of infection-related hospital admission were correlated with higher number and cost of hospitalization. CONCLUSIONS: The findings of this study demonstrated that individuals with Alzheimer's disease or dementia were at a high risk of infection-related hospitalization. Age, sex, socioeconomic status, comorbidities, continuity of care, and history of infection-related hospitalization were associated with the risk. The information is of help to identify high-risk individuals and to plan preventive measures in the disease management of this patient group.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterpresentations-com-a0-ispor-europe-2023-20231103-final133921-pdf.pdf?sfvrsn=fec326bb_0","title":"PosterPresentations.com-A0-ISPOR Europe 2023 20231103 final133921.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133921","diseases":[{"id":"dc752772-538c-4933-b6a5-3b5b6d079673","parentId":"00000000-0000-0000-0000-000000000000","title":"Geriatrics","urlName":"Geriatrics"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Comparison of Healthcare Resource Utilization and Costs Between Patients with Chronic Lymphocytic Leukemia Treated with First-Line Ibrutinib or Acalabrutinib","id":"a4ce7099-da8b-4154-bd11-38cc205cc231","sessionCode":"EE70","topDisplay":"Rogers KA1, Qureshi ZP2, Ding Z2, Emond B3, Gogna P3, Lafeuille MH3, Bokun A2, Fradley M4 1Division of Hematology, The Ohio State University, Columbus, OH, USA, 2Janssen Scientific Affairs, LLC, Horsham, PA, USA, 3Analysis Group, Inc., Montreal, QC, Canada, 4Thalheimer Center for Cardio-Oncology, Abramson Cancer Center and Division of Cardiology, Hospital of the University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA, USA","locationCode":"2002","description":"\r\n\t<div>OBJECTIVES: To compare real-world healthcare resource utilization (HRU) and costs between patients with chronic lymphocytic leukemia (CLL) initiated on first-line (1L) ibrutinib or acalabrutinib. METHODS: Adults with CLL initiated on 1L ibrutinib or acalabrutinib monotherapy (index date) on or after acalabrutinib approval for CLL (11/21/2019) were analyzed using electronic medical records from the Acentrus database (11/21/2018-4/30/2022). Per-patient-per-month (PPPM) HRU and costs were evaluated during 1L therapy and compared between ibrutinib and acalabrutinib using multivariate regression models. RESULTS: Among 710 and 373 patients initiated on ibrutinib and acalabrutinib, respectively, mean age (ibrutinib vs. acalabrutinib: 71.5 vs. 72.4 years, P=0.159), sex (38.5% vs. 38.3%, P=0.971), and mean Quan-Charlson Comorbidity Index (3.1 vs. 3.0, P=0.597) were similar. Median duration of 1L was longer for ibrutinib (16.5 vs. 10.2 months, P<0.001). During 1L therapy, the mean number of inpatient days was similar for both cohorts (0.42 vs. 0.49 days PPPM, rate ratio [RR]=1.00, P=0.966), while the number of outpatient visits was significantly lower for ibrutinib compared to acalabrutinib (1.47 vs. 2.06 days PPPM, RR=0.76, P<0.001). Similar results were observed for CLL-related HRU (inpatient: RR=0.87, P=0.758; outpatient: RR=0.80, P=0.036). Mean total healthcare costs were significantly lower for ibrutinib compared to acalabrutinib (all-cause: $14,691 vs. $16,599 PPPM, mean monthly cost difference [MMCD]=-$1,355, P=0.004; CLL-related: $12,186 vs. $13,715 PPPM, MMCD=-$1,215, P=0.004). Results were consistent for the first 3 months (RR for outpatient=0.76, P<0.001; MMCD for total costs=-$810, P=0.120), 6 months (RR=0.77, P<0.001; MMCD=-$1,040, P=0.044), and 12 months (RR=0.76, P<0.001; MMCD=-$1,417, P<0.001), and among the subgroup with baseline atrial fibrillation (RR=0.53, P=0.044; MMCD=-$2,834, P=0.309). CONCLUSIONS: 1L CLL patients treated with ibrutinib had lower number of days with outpatient services and lower costs compared to acalabrutinib. These findings have important implications for optimal 1L BTKi selection. Additional research is warranted to understand reasons behind differences in HRU/costs between 1L ibrutinib and acalabrutinib.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dingee70poster131331-pdf.pdf?sfvrsn=534b6607_0","title":"ISPOREurope23_Ding_EE70_POSTER131331.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131331","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Antibiotics for Tuberculosis Patients at Pharmacies: Cases Using Standardized Patients in Pakistan","id":"0703f872-6339-4124-9875-38cceb038c64","sessionCode":"HSD16","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"4026","description":"\r\n\t<div>OBJECTIVES: Pakistan ranked 6th in antibiotic consumption and patients often receive antibiotics plus prescription-only drugs directly from pharmacies. Here in this study, we aimed to assess drug dispensing practices of pharmacies for presumed and confirmed tuberculosis in standardized patients. METHODS: In this cross-sectional study, carried out in three cities (Lahore, Rawalpindi and Sialkot), we adopted 2 standardized patient cases: first, a presumed TB patient presenting with 2–3 weeks of cough and fever (pulmonary tuberculosis symptoms (Case-1) and 2nd, a confirmed TB patient who was carrying microbiologically confirmed TB results (Case-2). Standardized patients were asked to present these cases once to sampled pharmacies. Ideal management for Cases-1 and Case-2 a priori as referral to a health-care provider without dispensing antibiotics or steroids or both. The differences in antibiotic use or steroid and number of medicines dispensed in referred and non-referred patients between Case-1 and Case-2 were mentioned using the descriptive statistics. RESULTS: Between 1 April, 2020, and, 31 July 2020, standardized patients completed 575 of 598 interactions among pharmacies in Lahore, Rawalpindi and Sialkot. We recorded ideal management in 115 (37.7%) of 305 Case-1 interactions and 130 (48.1%) of 270 in Case-2 interactions. Antibiotic dispensing was higher in Case-1 [71 of 305 instances (23.3%)] than in Case-2 interactions [27 (10.0%) of 270]. Anti-tuberculosis drugs were dispensed for 1 patient in Case-1 (0.3%) and 19 (7.0%) patients for Case-2 occasions CONCLUSIONS: Slightly higher than one third of pharmacies in Punjab, Pakistan correctly managed patients with presumed tuberculosis, but almost half of them correctly managed a case of confirmed tuberculosis. Pharmacies un-orthodoxically dispensed anti-tuberculosis drugs for Case-1 and Case-2. Presence of confirmed diagnosis is slightly changing the behavior in correct management of patients. Antibiotic misuse could lead to antibiotic resistance and we need to implement antimicrobial stewardship interventions.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128864","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Surge Capacity for Preserving Accessibility to Primary Healthcare Services during the COVID19 Pandemic in a Middle Income Country","id":"c587a8c3-38cf-49eb-8d83-395c14333cef","sessionCode":"EE149","topDisplay":"Singweratham N1, Phodha T2, Rochanathimoke O3, Techakehakij W4, Bunpean A5, Chotchoungchatchai S6, Vongmongkol V6 1Faculty of Public Health, Chiang Mai University, Muang District, 50, Thailand, 2Faculty of Pharmacy, ThammasatUniversity, Saimai, 10, Thailand, 3Faculty of Medicine, Bangkok Thonburi University, Bangkok, 10, Thailand, 4Lampang Hospital, Mueang Lampang District, Lampang, Thailand, 5Kanchanabhishek Institute of Medical and Public Health Technology, Faculty of Public Health and Allied Health Sciences, Praboromarajchanok Institute, Sai Noi District, Nonthaburi, Thailand, 6International Health Policy Program (IHPP), Ministry of Public Health, Mueang Nonthaburi District, Nonthaburi, Thailand","locationCode":"2077","description":"\r\n\t<div>OBJECTIVES: The number of confirmed COVID-19 cases in Thailand were almost 4 million, with 128 deaths from the disease and rising continuously. Tambon Health Promoting Hospitals (THPH), a primary care unit focusing on proactive problem solving at the individual and community levels continuously, were also affected by the COVID-19 pandemic. This study is to assess the impact of the COVID-19 pandemic on primary healthcare service unit costs at THPHs from the provider’s perspective in Thailand and to investigate the surge capacity management at THPHs during the COVID-19 pandemic. METHODS: This study is mixed methods including the quantitative part of costing analysis and the qualitative part of in-depth interviewing the healthcare personnel at the THPHs. Thirty-six THPHs included in this study were varied by size (S, M, and L) and location across 6 regions in Thailand. The average unit costs (total costs/ No. of utilization) of primary healthcare services were estimated by the activity-based costing technique using standard costing approach and discussed by the context of THPHs. Multivariate log-linear regressions were employed to investigate the impact of the COVID-19 pandemic on the average unit costs of primary healthcare services. Content analysis was used to understand the relevant reasons between the changes in costing and the surge capacity at the THPHs. RESULTS: Switching from a normal situation (in the year 2019) to the COVID-19 pandemic (in the year 2020 and 2021), this resulted in decrease in expected average unit costs of primary healthcare services at 53.98% (35%-132%) and 13.67% (39%-123%), respectively. Surge capacity of the THPHs needs the efficient networking between the healthcare team and the local government in the province regarding to the differences in THPH’s context. CONCLUSIONS: The severity of COVID-19 pandemic could impact the resources used and working process to preserve accessibility to primary healthcare services.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23phodhaee149poster130230-pdf.pdf?sfvrsn=6c7d96d6_0","title":"ISPOREurope23_Phodha_EE149_POSTER130230.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130230","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Budget Impact of Procalcitonin-Guided Antibiotic Stewardship Compared to Standard of Care for Patients with Suspected Sepsis Admitted to the Intensive Care Unit in Belgium","id":"f64824cf-ad26-47e0-bcca-3a43ff9607f4","sessionCode":"MT4","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"5031","description":"\r\n\t<div>OBJECTIVES: In Belgium, antibiotic resistance leads to approximately 530 deaths with a €24 million financial burden annually. This study estimated the impact of procalcitonin-guided antibiotic stewardship to reduce antibiotic consumption versus standard of care in patients with suspected sepsis. METHODS: A decision analytic tree modelled health and budget outcomes of procalcitonin-guided antibiotic stewardship programs for patients with suspected sepsis. A literature search, an expert survey with local clinical experts, and national database searches were conducted to obtain model input parameters. Main outcomes were total budget impact per patient, reduction in number of antibiotic resistance cases, and cost per antibiotic day avoided. To evaluate the impact of parameter uncertainty on the source data, a deterministic sensitivity analysis was performed. A scenario analysis was conducted to investigate budget impact when including parameters for reduction in length of stay in the intensive care unit and mechanical ventilation duration, in addition to base-case parameters. RESULTS: Based on model predictions, procalcitonin-guided antibiotic stewardship could reduce the number of antibiotic days by 66,868, resulting in €1.98 million savings towards antibiotic treatment. Antibiotic resistance cases could decrease by 7.7% (6.1% vs 9.2%) in the procalcitonin-guided setting compared with standard of care setting. The base-case budget impact suggests an investment of €1.90 per patient. The sensitivity analysis showed uncertainty, as the main drivers can alter potential cost savings. The scenario analysis indicated a saving of €1,405 per patient, with a reduction of 1.5 days in the intensive care unit (14.8 days vs 12.8 days), and a reduction of 22.7% (18.1–27.2%) in mechanical ventilation duration. The associated sensitivity analysis was shown to be robust in all parameters. CONCLUSIONS: Procalcitonin-guided antibiotic stewardship programs are associated with clinical benefits that positively influence antimicrobial resistance in Belgium. A small investment per patient to implement procalcitonin testing may lead to substantial savings.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23garnfeldtmt4poster128797-pdf.pdf?sfvrsn=18f0b19b_0","title":"ISPOREurope23_Garnfeldt_MT4_POSTER128797.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128797","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Utility Analyses of Glofitamab for the Treatment of Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma after Two or More Lines of Systemic Therapy in Italy","id":"5e87906c-1673-4135-b280-3a823aa4ab7a","sessionCode":"EE79","topDisplay":"Bellone M1, Ghislieri D2, Pradelli L1, Kokaliaris C3, Di Maio D3 1AdRes HEOR, Torino, TO, Italy, 2Roche Spa, Monza, Italy, 3F. Hoffman-La Roche, Basel, Basel-Stadt, Switzerland","locationCode":"1097","description":"\r\n\t<div>OBJECTIVES: To assess cost-utility of glofitamab for the treatment of adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy, from the Italian Health Service perspective. METHODS: A partitioned survival model of three mutually exclusive health-states – progression-free, post-progression and death – was developed to compare lifetime clinical outcomes and costs of patients treated with glofitamab and his main comparators: polatuzumab vedotin in association with rituximab and bendamustina (Pola-BR) or tafasitamab plus lenalidomide (Tafa+Lena), for patients non-eligible for CAR-T; tisagenlecleucel (Kym) or axicabtagene ciloleucel (Yesc) for the others. Progression-free survival (PFS) and overall (OS) were modelled independently for glofitamab and comparators using parametric survival curves obtained from inverse probability of treatment weighting (IPTW) or matching-adjusted indirect comparisons (MAIC). Health utilities values (for progression-free health-state on- and off-treatment and post-progression) were obtained by mapping EORTC QLQ-C30 scores to EQ-5D-3L values. Direct healthcare costs, including drug acquisition and administration, disease monitoring, adverse event management, and post-progression therapy were collected from Italian sources. Probabilistic sensitivity and scenario analysis evaluated the uncertainties on input parameters. RESULTS: Glofitamab generated additional QALYs at a lower cost when compared with Pola-BR (0.048; -€6,878), Tafa-Lena (0.829; -€386,565) and Kym (0.311; -€294,949), resulting economically dominant. Instead, when comparing glofitamab with Yesc, the incremental net monetary benefit was estimated in €202,829, proving that glofitamab was a cost-effective option at willingness-to-pay threshold of €40,000 per QALY. Model results were generally robust across scenario and sensitivity analyses tested. However, there are some areas of uncertainty relating to the limited follow up of glofitamab clinical trial, utility values and residual bias from the ITCs. CONCLUSIONS: Glofitamab can be considered a cost-effective option for Italian patients with 3L+ DLBCL, particularly for those who have exhausted currently available valid alternatives.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23belloneee79poster131765-pdf.pdf?sfvrsn=57d39e3d_0","title":"ISPOREurope23_Bellone_EE79_POSTER131765.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23belloneee79handout131765-pdf.pdf?sfvrsn=6b80f0e3_0","title":"ISPOREurope23_Bellone_EE79_HANDOUT131765.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131765","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring the Predictive Accuracy of Treatment Waning Methods: An Analysis of Pembrolizumab across Six Oncology Indications","id":"e6c7caaa-0d80-46c8-b8a3-3af6aafe74ac","sessionCode":"HTA7","topDisplay":"Harrington H1, Vasilyeva AV2, Micallef J1 1Costello Medical, London, UK, 2Costello Medical, Cambridge, UK","locationCode":"4051","description":"\r\n\t<div>OBJECTIVES: Treatment waning can substantially impact estimated overall survival (OS) in economic models and is a source of uncertainty in health technology assessments.1 We investigated the accuracy of four waning methods in predicting OS across six oncology indications, using pembrolizumab as a case study. METHODS: Six pembrolizumab clinical trials in different indications were identified that included two-year stopping rules, and where published OS data were available from both an early and more mature data cut-off (DCO) (median follow-up: 23–28.4 months and 44.5–69.9 months, respectively). For early DCOs, pembrolizumab and comparators were modelled via independent standard parametric extrapolation of OS data. Models were selected using goodness-of-fit and clinical expert estimates of plausible long-term survival from relevant National Institute for Health and Care Excellence appraisals. Four waning methods were applied to the extrapolated data: Methods 1A and 1B assumed full treatment effect until 5 years, after which all effect was lost relative to the comparator; Methods 2A and 2B linearly waned treatment effect between 2 and 5 years.1 Predicted life years (LYs) were calculated with each method, and with no waning, over the maximum follow-up in the more mature DCO, and compared with realised LYs over this period calculated directly from long-term Kaplan-Meier data. RESULTS: In most cases, all waning methods underestimated LYs compared to realised LYs. The predicted LYs aligned most closely with realised LY estimates when no waning was applied (mean difference: −3.9%). Of the waning methods, Method 1A was usually the most accurate and Method 2B was most often the least accurate compared to the realised LYs (mean difference: −4.0% and −6.8%, respectively). CONCLUSIONS: Treatment waning assumptions consistently underestimate OS when applied in combination with appropriate parametric extrapolations of short-term OS data, considering goodness-of-fit and clinical expert estimates of plausible long-term survival. REFERENCES: <ol> <li>Micallef J, Harrington H and van Hest N. ISPOR 2022</li> </ol></div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/treatment-waning131735-pdf.pdf?sfvrsn=d52d337e_0","title":"treatment-waning131735.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131735","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparison of Hospital Case Characteristics of Popliteal Artery Aneurysm Repair with ePTFE Covered Stent Grafts with a Heparin Bioactive Surface and Open Surgical Bypass: Highlights from the 2021 Hospital Dataset in Germany","id":"b0a96350-9542-45c2-8f97-3b0e172e0bb4","sessionCode":"MT12","topDisplay":"Iqbal K1, Roedig S2, Schumann E2 1WL Gore & Associates Ltd, Livingstone, WLN, UK, 2WL Gore & Associates GmBH, Putzbrunn, Germany","locationCode":"5036","description":"\r\n\t<div>OBJECTIVES: A popliteal artery aneurysm (PAA) is a bulging and weakness in the popliteal artery vessel wall. Treatment options include an endovascular repair (ER) using the GORE® VIABAHN® Endoprosthesis with PROPATEN Bioactive Surface and open surgical repair with autogenous conduit (OSR). Data reported in the national hospital dataset for ER and OSR were compared. METHODS: Data was extracted from the InEK DatenBrowser1 for 2021 using ICD code I72.4 Aneurysm and dissection of artery of lower extremity for the primary diagnosis, procedure codes 8.83b.e1 for the ER cohort, 5.393.53-55 and 5.930.00-.01 for the OSR cohort. Age, hospital stay duration, secondary diagnoses and DRG category were compared. RESULTS: There were 5,043 PAA cases reported for 2021, of these 1,471 (29.2%) were OSR and 391 (7.7%) were ER. Compared to OSR, the ER cohort had a higher proportion of patients aged over 65 years (76.5% versus 64.0%). The hospital stay was considerably lower for ER at 6.3 days versus 12.8 days. For secondary diagnoses, the ER cohort had fewer cases of ICD code I74.3 embolism and thrombosis of lower extremity arteries (12.8% versus 24.0%), T81.0 haemorrhage and hematoma as complication of surgery (10.8% versus 16.3%) and D62 acute post-haemorrhage anaemia (7.4% versus 19.8%). The weighted average DRG rate per case reported was 5,503€ for ER (excluding NUB payment) and 11,395€ for OSR. In the ER cohort 86% of cases were reimbursed within DRG F59 D-B (4,712€ –7,660€) and 94% of OSR cases were reimbursed within DRG F08 E-B (7,940€ – 21,260€). CONCLUSIONS: Hospital case data demonstrated that PAA repair with the GORE® VIABAHN® Device was used more frequently in older patients and required shorter hospital stay. It had fewer cases with secondary diagnoses of thrombosis, haemorrhage and hematoma. The cost to the health care payer was lower reflected in a smaller average DRG value.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128796","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Medication Adherence in Osteoporosis Treatments and the Impact in Patients' Quality of Life","id":"2d858e25-9caf-4e5d-bcce-3b4917cff6e9","sessionCode":"CO11","topDisplay":"Tsaousis P1, Theodorou P2, Hatzikou M2 1Orthopedic Surgeon, Edessa, Pella, Greece, 2Hellenic Open University, Patras, Greece","locationCode":"1010","description":"\r\n\t<div>OBJECTIVES: To investigate the degree of adherence to medication in women with osteoporosis and the impact in patient’s quality of life METHODS: A cross sectional study was performed from November 2022 till March 2023. The sample constituted by 75 osteoporosis patients living in the prefecture of Pella. In order to measure the adherence to treatment the Morisky Medication Adherence Scale (MMAS-8) used and for patient’s quality of life, the EQ-5D 5L and the EQ-VAS. Statistical analysis was performed with SPSS v26. RESULTS: Adherence to medication was rated high (81%) and the self-rated health index (EQ-vas) was also high (79.33%). Regarding the quality of life, patients reported that they had no or little stress – sadness (Μ=1.93+0.99), pain or discomfort (Μ=1.87+0.96) and problems in walking (Μ=1.81+1.06), while the majority had no problems at all in carrying out usual activities (Μ=1.67+0.98) and self-care (Μ=1.39+0.87). Women with higher levels of medication adherence showed higher quality of life (rho= 0.450, p<0.001) and self-rated health (rho= 0.337, p<0.001) while medication adherence, quality of life, and self-rated health were lower for older women [(rho= -0.321, p=0.005), (rho= -0.614, p<0.001) and (rho= -0.482, p<0.001) respectively] and those who had not attended school [(Η=9.30, p=0.026), (Η=18.52, p<0.001) and (Η=13.49, p=0.004) respectively], while quality of life was lower for those living alone (U=183.5, p=0.059). Additionally, quality of life and self-rated health were lower for women who are retired or unemployed [(Η=24.644, p<0.001) and (Η=15.916, p=0.003 respectively)] and experiencing many or some financial difficulties [(t-test=-3.872, p<0.001) and (t-test=-3.285, p=0.002 respectively)]. The most important predictive factor for all 3 parameters studied (medication compliance, quality of life and self-assessed health) was the low level of education. CONCLUSIONS: Osteoporosis is an important public health problem in Greece, especially in postmenopausal women and the factors affecting patient's compliance and QoL are very important for effective decision making strategies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hatzikouco11poster132446-pdf.pdf?sfvrsn=5076017c_0","title":"ISPOREUROPE23_HATZIKOU_CO11_POSTER132446.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132446","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Olaparib Plus Bevacizumab First-Line Maintenance in Ovarian Cancer Alongside the PAOLA-1 Trial","id":"5a2bc9fc-810e-433f-afd3-3b76e0a1875b","sessionCode":"EE63","topDisplay":"Perrier L1, Zarca K2, Cadet T1, Morelle M1, Cropet C1, Burges A3, Cecere SC4, Palacio I5, Polterauer S6, Yoshida H7, Vuylsteke P8, Colombo N9, Nøttrup TJ10, Abdeddaim C11, El-Balat A12, Cinieri S13, Herrero A14, Kaminski MC15, Pujade-Lauraine E16, Durand Zaleski I17, Ray-Coquard I18 1Centre Léon Bérard, Lyon, France, 2DRCI-URC Eco Ile-de-France (AP-HP), Assistance Publique-Hôpitaux de Paris, Paris, France, 3Klinikum der Universität München, München, Germany, 4Department of Urology and Gynecology, Division of Clinical Experimental Uro-Gynecological Oncology of The National Cancer Institute IRCCS Fondazione \"G. Pascale\" and MITO, Napoly, Italy, 5Hospital Central de Asturias, Oviedo, Spain, 6Department of Obstetrics and Gynecology, Medical University of Vienna, Austria, Vienna, Austria, 7Saitama Medical University International Medical Center, Saitama, Japan, 8CHU UCL Namur, UC Louvain Belgium, Namur, Belgium, 9Department of Medicine and Surgery University of Milan-Bicocca Gynecologic oncology Program, European Institute of Oncology IRCCS, Milan, Italy, 10Copenhagen University Hospital, Rigshospitalet, and NSGO, Denmark, Copenhagen, Denmark, 11Centre Oscar Lambret, Lille, France, 12Spital Uster, Frauenklinik, Klinikum der Johann Wolfgang Goethe-Universität Frankfurt, Klinik für Frauenheilkunde und Geburtshilfe, Uster, Switzerland, 13U.O.C. Oncologia Medica - Ospedale Senatore Antonio Perrino, Brindisi, Italy, 14Hospital Universitario Miguel Servet, Zaragoza, Spain, 15Institut de Cancérologie de Lorraine, Vandoeuvre-les-Nancy, France, 16ARCAGY Research, Paris, France, 17URCEco, AP-HP, Hôpital de l’Hôtel Dieu, F-75004, Paris, 75, France, 18Centre Léon Bérard, and University Claude Bernard Lyon I, Lyon and GINECO, France, Lyon, France","locationCode":"2013","description":"\r\n\t<div>OBJECTIVES: PAOLA-1 trial demonstrated a significant progression-free survival (PFS) and OS benefit when maintenance olaparib was added to bevacizumab in newly diagnosed advanced ovarian cancer patients with a positive tumor HRD test, leading to a FDA/EMA label in 2020. The aim of this study was to conduct a cost-effectiveness analysis (CEA) based on patient-level data from the PAOLA-1 trial in the HRD-positive population (n=387/806). METHODS: Patients were randomized 2:1 to olaparib plus bevacizumab or placebo plus bevacizumab. Hospital costs (€2022) were assessed from the French National Health perspective with a 60 month time horizon. Drug costs, including subsequent therapies, were drawn from the case report. Costs were discounted at 2.5% rate. Restricted mean survival time (RMST) were used. Incremental cost-effectiveness ratios (ICERs) in cost per progression free life year gained (PF-LYG) and in cost per life year gained (LYG) were calculated. Uncertainty around ICERs was captured by bootstrap. RESULTS: Total mean costs per patient were €112,510 (SD: 50,519) in the olaparib plus bevacizumab group and €70,517 (SD: 62,312) in the placebo plus bevacizumab group. Mean progression free survival were 3.375 and 2.058 years respectively, leading to an ICER of €31,885 per PF-LYG. Results were robust at 95%. Mean overall survival were 4.20 in the olaparib plus bevacizumab group (255/387) and 3.875 years in the placebo plus bevacizumab group (132/387). The corresponding ICER (€129,209 per LYG) may be over-estimated due to treatment crossover: >50% of HRD-positive patients received after progression subsequent PARPi in the placebo plus bevacizumab group. CONCLUSIONS: These results indicated that olaparib plus bevacizumab is deemed to be more effective and more costly as compared to placebo plus bevacizumab in the HRD-positive population. ICER is expected to reduce with further follow-up. ICER calculated using overall survival endpoint should be taken with caution, as crossover adjustment methods have not yet been applied.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor2023paola-ecoee63133079-pdf.pdf?sfvrsn=7e268121_0","title":"ISPOR2023_PAOLA ECO_EE63133079.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133079","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Digital Medical Services Delivered by Internet Hospitals via WeChat in China: A Qualitative Analytical Research","id":"2e787017-88cc-4239-ac92-3be0d4a98c39","sessionCode":"HSD24","topDisplay":"Lai Y1, Chen XY2, Li M3, Yao DN4, Shi HH5, Hu H6 1Guangzhou University of Chinese Medicine, Guangzhou, 44, China, 2Guangzhou University of Chinese Medicine, Guangzhou, Guangdong, China, 3Southeast University, Nanjing, Jiangsu, China, 4Nanjing Medical University, Nanjing, Jiangsu, China, 5Peking University, Beijing, Beijing, China, 6University of Macau, Macau SAR, Macau, China","locationCode":"4034","description":"\r\n\t<div>OBJECTIVES: The WeChat platform has become the main platform for Internet hospitals to provide digital medical services (DMSs) in China. However, little is known about the service model of DMSs delivered by internet hospitals via WeChat. This study aimed to explore the emerging service model of DMSs delivered by internet hospitals via WeChat. METHODS: This study applied a document research design to analyze DMSs of 25 leading internet hospitals in China. Materials were collected from their official WeChat platforms. Thematic analysis was conducted for data analysis. RESULTS: Themes highlighted four key dilemmas when delivering a service model of internet hospitals, including (i) professional service (medical service & pharmacy service); (ii) information service (convenient service, health science popularization & mapping); (iii) science & education service (clinical assistance & patient survey); (vi) online mall service (daily necessities). All the sample internet hospitals have delivered the medical service and convenient service. Both clinical assistance and daily necessities are delivered by only one sample internet hospital, respectively. Two sample internet hospitals conduct patient surveys to explore patient satisfaction to improve service quality. CONCLUSIONS: The healthcare industry is currently in search of innovative service models of internet hospitals in response to the unprecedented form of healthcare in China. However, the target customer, value offering and services provision of the service model design still remain debatable, the value of DMSs has not been fully realized. The service model of DMSs needs more distinguished innovations to provide personalized digital health and patient-centric services.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23laihsd24poster133240-pdf.pdf?sfvrsn=95ae4ea3_0","title":"ISPOREurope23_LAI_HSD24_POSTER133240.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133240","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Is the UK’S Ilap Process Delivering Its Intended Benefits? a Review of Access to Orbis Designated Oncology Products within the UK","id":"5daa7f08-052a-4116-8f04-3c0c92d9d3be","sessionCode":"HPR31","topDisplay":"Gaultney J, McDonald K, Mushtaq S IQVIA Ltd, London, UK","locationCode":"4001","description":"\r\n\t<div>OBJECTIVES: The UK’s Innovative Licensing Access Pathway (ILAP) provides manufacturers opportunity to engage early with UK stakeholders with the ambition to improve patient access to innovative medicines. Little is known about the benefits as there is no publicly available documentation on the products accepted into ILAP, though Orbis-designated oncology products must also qualify for ILAP. HTAs by NICE and SMC for Orbis-designated products were reviewed to assess if they receive the benefits of the ILAP scheme. METHODS: A search of an international database of extracted HTAs (HTA Accelerator) was conducted in June 2023 to identify NICE and SMC HTAs of products that received Orbis-designation. Data extractions included the evidence under assessment, agency critique, outcome, and time to recommendation. RESULTS: As of March 2023, 16 products received Orbis designation. The majority (n=9; 56%) were indicated for non-small cell lung cancer (NSCLC). From NICE, 58% received positive recommendations (+/-restrictions), 21% were negative and 21% did not submit. From SMC, 50% received positive recommendations (+/-restrictions), 14% were negative and 36% did not submit. In two instances, SMC and NICE appraisal decisions outcomes were divergent. Median and mean time to decision was 82 and 67 days longer for NICE compared to SMC, respectively [NICE: 244 or 268 days; SMC: 149 or 179 days]. Mean time to availability was 240 days in England vs 208 days in Scotland, which in comparison to the 2021 EFPIA Patient WAIT survey for oncology products, was similar for NICE (240 vs 241) but faster for SMC (208 vs 374). CONCLUSIONS: Timelines for HTA approval for products in Project Orbis are similar to non-Orbis oncology products in England, although faster in Scotland. Regulator and HTA decisions are not always aligned. Products in Project ORBIS are not necessarily representative of ILAP more generally but other information is currently not available.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/gaultney-et-alispor-poster-hpr31v1-029october-2023131739-pdf.pdf?sfvrsn=5888ee23_0","title":"Gaultney et al_ISPOR poster HPR31_v1.0_29October 2023131739.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131739","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Validity of Preference-Weighted Scores: A Case Study of the International Classification of Functioning, Disability, & Health","id":"fa6c89e1-ab94-4b01-bc73-3c868defd7d9","sessionCode":"PCR1","topDisplay":"Juhnke C1, Mühlbacher A2 1Hochschule Neubrandenburg, Neubrandenburg, Germany, 2Hochschule Neubrandenburg, Neubrandenburg, MV, Germany","locationCode":"6008","description":"\r\n\t<div>OBJECTIVES: Indices such as the International Classification of Functioning, Disability and Health (ICF) are commonly used to measure outcomes for decision making. As with other instruments, equal weight is assigned to each item without distinguishing relevance. From the patient's perspective, the validity of the outcomes may be questioned. The aim is to examine the extent to which a preference-weighted assessment of core elements of the ICF differs from unweighted assessments currently used in clinical decision making. METHODS: Three best-worst scaling experiments are used to assess ICF dimensions in terms of body function/movement, neglect/perception and activities. Stroke patients and citizens are recruited. The ICF \"tariff\" is created by converting the ICF generic percentage qualifiers for impairments into a unidimensional index. Preference weights for levels within each dimension are determined on a scale of 0 to 1, with the most desirable level rated 1. RESULTS: N=1112 participants were recruited through August/September 2022. There is evidence of divergent validity of preference-weighted and unweighted scores for the ICF, based on the intraclass correlation coefficient. Unweighted scores in the dimensions of body function, activity, and neglect differ significantly from preference-weighted scores from BWS experiments in general (e.g., ICC_movement: 0.864, ICC_neglect: 0.491) and for various hypothetical health states constructed on the basis of ICF states. CONCLUSIONS: Body functions have effects on activities, and these effect on health-related quality of life. This raises the question how the value is measured and considered in decision-making? The results reveal that ICF body functions or activities are not equally weighted by those affected. This fosters discussions on the differences of preference-weighted scores and simple additive models to develop a patient-centered classification of impairments and therapy goals. (The joint-project is funded by European Funds ESF, EFRE, ELER and the Ministery of Education, Science and Culture Mecklenburg-Vorpommern, Germany. Reference: ESF/14-BM-A55-0001/19-A01)</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/pcr1scoree-brain133632-pdf.pdf?sfvrsn=90b0ea9b_0","title":"PCR1_Score_E-BRAiN133632.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133632","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Use of Ferric Carboxymaltose for Pregnant and Postpartum Women in Brazil: Estimation of Target Poputation and Budget Impact Analysis from the Public Health Care System Perspective","id":"750b4a4c-66ee-4135-a44b-3cf569a04d0c","sessionCode":"EE113","topDisplay":"Campagnaro M1, Clemente V2, Pereira R3, Ramirez de Arellano Serna A4 1CSL Vifor, São Paulo, SP, Brazil, 2CSL Vifor, São Paulo, São Paulo, Brazil, 3CSLVifor, São Paulo, São Paulo, Brazil, 4CSL Vifor, Glattbrugg, ZH, Switzerland","locationCode":"2043","description":"\r\n\t<div>OBJECTIVES: From the Brazilian public health care system (SUS) perspective this study estimates the potential number of eligible patients with iron deficiency anemia (IDA) and the budgetary impact (BI) of pregnant and postpartum women (PPW). METHODS: A descriptive observational study, was carried out with data obtained from the Brazilian Institute of Geography and Statistics (IBGE) and data SUS platform, which were analyzed and statistically compared to survey the local Brazilian population and estimate its growth over 5 years. A BI analysis was performed considering different scenarios of increased utilization rates of FCM for patients with IDA (10%, 30%, 50%, 60% and 80%) over oral iron and low-dose intravenous iron (LD-IV) (90%, 70%, 50%, 40% and 20%). The time horizon was 5 years, between 2023 and 2027. RESULTS: we estimated a total of 156,413 eligible patients with IDA, amongst them 91,263 pregnant women and65.150 PPW. The introduction of FCM in the treatment of anemia due to IDA in pregnant women generates a BI cost-saving of BRL 623,447,445 in the first year, BRL 621,519,641 in the fifth year and accumulated cost-saving of BRL 3,112,131,041. For the PPW, it generates a cost-saving of BRL 466,253,736 in the first year, a cost-saving of BRL 464,812,001in the fifth year and accumulate cost-saving of BRL 2,327,449,950 over the LD-IV. CONCLUSIONS: 75% of the Brazilian population have access to SUS treatment, 91,263 pregnant women and 65,150 PPW will require iron supplementation. Intravenous iron, particularly FCM, has been shown to be cost saving in relation to LD-IV.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23campagnaroee113poster131294-pdf.pdf?sfvrsn=e2d0c178_0","title":"ISPOREurope23_Campagnaro_EE113_POSTER131294.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131294","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring the Link Between Institutional Country-Level Differences and Consideration of Additional Value Elements in HTAs: Results From a Pragmatic Review","id":"c110a8c1-328d-424a-98e5-3d378a15ff7d","sessionCode":"HTA43","topDisplay":"Radhakrishnan A1, Muir J2, Freitag A3, Mehra N4, Ozer Stillman I5, Sarri G6 1Cytel, Mississauga, ON, Canada, 2Cytel, Waltham, MA, USA, 3Cytel, London, LON, UK, 4Cytel, Amsterdam, NH, Netherlands, 5Takeda Pharmaceuticals, boston, MA, USA, 6Cytel, London, UK","locationCode":"5007","description":"\r\n\t<div>OBJECTIVES: Adapting the health technology assessment (HTA) process to include elements of value beyond clinical/economic benefits is a topic of much discussion. We aimed to expand this discussion by assessing whether the underlying culture and values embedded in the institutional context of a country, its healthcare system, and HTA process may influence the country’s predisposition toward higher acceptance of expanded value elements in HTAs. METHODS: A pragmatic review was conducted in Embase and MEDLINE (2013–2023) to identify English publications presenting country-level information, and variation in considerations or implementation of value elements (beyond clinical, economic) in HTA decision-making. Eligible publications included guideline reviews, HTA summaries, or previous reviews. Screening and data extraction was performed by two reviewers. Publications on individual countries were excluded. Results were narratively synthesized by presenting trends among countries with comparable socio-economic settings. RESULTS: Database searches returned 1,602 hits after de-duplication. With 75% of screening completed, seven records (guideline reviews, case studies, and consensus papers) were included. The most common value elements considered were productivity, health equity, and transportation with considerable variation among European countries. A higher number of value elements corresponded with sophistication of country-data infrastructure and HTA processes. All low- and middle-income countries emphasized health equity. Environmental impact was proposed mainly by HTA agencies in Canada and the United Kingdom whose processes are comparable. Social care aspects were more commonly considered in tax-based rather than insurance-based healthcare systems. CONCLUSIONS: HTA acceptance thresholds of additional value elements by country level varied widely. However, a growing trend was observed toward higher acceptance over time with a link between countries having universal care systems and higher income. Different trends in value considerations between national assessments may threaten the weight of European Union joint clinical assessments in HTAs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023radhakrishnanhta43poster133496-pdf.pdf?sfvrsn=e1d70b6e_0","title":"ISPOREurope2023_Radhakrishnan_HTA43_POSTER133496.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133496","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Impact of Advanced Training on Operational Efficiency in Robotic-Assisted Rectal Resection","id":"6ee7b3fa-9afe-4e1a-9cac-3e564b7eae9b","sessionCode":"RWD31","topDisplay":"D'Attilio D1, Yankovsky A1, Chacko A2, Lowery J1, Downs J1 1Intuitive Surgical, Palo Alto, CA, USA, 2Intuitive Surgical, Sunnyvale, CA, USA","locationCode":"7004","description":"\r\n\t<div>OBJECTIVES: While advanced training for robotic-assisted surgery (RAS) may be a vital component of increasing operational efficiency in RAS programs, there are no studies to our knowledge investigating its impact on operational efficiency. In this study, we extracted training and procedure data from da Vinci system logs to evaluate the impact of advanced training on procedure duration in robotic-assisted rectal resection. METHODS: We extracted procedure durations for rectal resections from da Vinci system logs and divided them into two groups: those performed by surgeons with basic training only and those performed by surgeons who had received advanced training in addition to basic (heretofore, “basic + advanced”). To evaluate procedure duration over time, we calculated the median (IQR) procedure duration for cases 1-10, 11-20, and then for every 20 cases up until the 200th case. We then compared the overall median (IQR) in the basic only and basic + advanced groups. A Kruskal-Wallis test was used to test for significance among groups. Visual analysis was performed using box-whisker plots. RESULTS: Procedure durations for 173,571 rectal resection cases were extracted from system logs (71,655 and 101,916 cases performed by basic only and basic + advanced groups, respectively). Both groups demonstrated a significant reduction in median (IQR) of procedure duration over time (p<0.001). The comparison of median (IQR) of both groups indicated that the basic + advanced group was significantly more efficient in terms of procedure duration (p<0.001). CONCLUSIONS: Results suggest that advanced training is correlated with reduced procedure duration and variability in RAS rectal resection. Similar analysis should be conducted to determine if advanced training shows the same impact in other procedure types.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023-rwd31-advanced-training-mp-procedure-time-10-23-2023-ay130550-pdf.pdf?sfvrsn=b94a29bc_0","title":"ISPOR EU 2023 RWD31 Advanced training MP procedure time 10.23.2023 AY130550.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130550","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cardiovascular and Renal Replacement Risks in Patients with Sub-Optimally Treated Anemia of Non-Dialysis-Dependent Chronic Kidney Disease","id":"21015bf0-f152-477e-9281-3eab946f7922","sessionCode":"EPH41","topDisplay":"Shaheen F1, Buessing M2, Lansang E3, Cheng LJ3, Smith Z2, Zuk E2, Lee SHY3, Hon-Chun Hsu P4 1Dr. Soliman Fakeeh Hospital, Jeddah, Makkah, Saudi Arabia, 2Medicus Economics, LLC, Milton, MA, USA, 3Astellas Pharma Singapore Pte. Ltd., Singapore, Singapore, 4University of the Witwatersrand, Johannesburg, South Africa","locationCode":"3042","description":"\r\n\t<div>OBJECTIVES: Patients with anemia (hemoglobin: <13.0 g/dL [males]; <12.0 g/dL [females]) of non–dialysis-dependent chronic kidney disease (NDD-CKD) are at elevated risk of renal failure and cardiovascular (CV) disease, and are often untreated. We aimed to estimate the risk of CV/renal replacement (RR) events in at-risk patients with sub-optimally treated anemia of NDD-CKD from South Africa, Saudi Arabia, and the United Arab Emirates (UAE) using a quantitative model. METHODS: CKD-specific risk equations were developed using individual data of stage 3–5 NDD-CKD patients with anemia recruited October 2002–December 2016 in the ongoing Salford Kidney Study using log-normal accelerated failure time models. Using these equations, our model projected the cumulative risks of RR (onset of dialysis/kidney transplant), CV (myocardial infarction [MI], unstable angina, coronary revascularization therapy, congestive cardiac failure, stroke, all-cause mortality [ACM]), and major adverse CV events (MACE; non-fatal MI/stroke/death due to CV event) over 1–15 years in at-risk population using country-specific population-level risk factors. Deterministic and probabilistic sensitivity analyses were performed to evaluate the model. RESULTS: The base-case results estimated that a substantial number of patients with sub-optimally treated anemia of NDD-CKD would require RR within 15 years (South Africa [205,351]; Saudi Arabia [366,937]; UAE [109,880]). A notable number of patients are estimated to be at risk of CV and ACM (South Africa [193,745]; Saudi Arabia [260,166]; UAE [77,907]) and MACE (South Africa [34,904]; Saudi Arabia [44,853]; UAE [13,431]) events over the same timeframe. CONCLUSIONS: The model projects a significant burden of illness associated with sub-optimally treated anemia of NDD-CKD in Saudi Arabia, South Africa, and the UAE over 15 years. However, these findings need to be validated with country-specific real-world data. These preliminary estimates may help physicians and policymakers to understand the magnitude of their at-risk population and the urgency of improving care for these patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/cv-risk-modelposterfinal-draft5oct23v1131037-pdf.pdf?sfvrsn=9d318b13_0","title":"CV risk model_poster_final draft_5Oct23_v1131037.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131037","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Behavioural Structured Expert Elicitation: A Case Study to Inform Hospitalisation Due to Respiratory Syncytial Virus in the UK","id":"10d8671c-3ad8-4c9f-9f49-3f71d60d05f3","sessionCode":"HTA30","topDisplay":"Schurer M1, Horscroft J2, Chetty M3, Hudson R4 1Lumanity Netherlands BV, Utrecht, Netherlands, 2Lumanity Netherlands BV, Utrecht, UT, Netherlands, 3Sanofi, Reading, UK, 4Sanofi, Reading, RDG, UK","locationCode":"4067","description":"\r\n\t<div>OBJECTIVES: Respiratory syncytial virus (RSV) is associated with high clinical and economic burden, especially in infants under one. Palivizumab and nirsevimab are licensed in this population for RSV prevention. Estimating the cost-effectiveness of these options is critical to inform reimbursement decisions. However, obtaining accurate numbers of RSV-driven hospitalizations and associated mortality – key drivers of cost effectiveness – is challenging due to coding practices and the impact of the COVID-19 pandemic. METHODS: Structured expert elicitation (SEE) is recommended by the UK National Institute for Health and Care Excellence to address uncertainty where empirical evidence is lacking. Several SEE frameworks exist, using different approaches to the elicitation and aggregation (e.g., behavioral and mathematical) of expert judgements. We used the Sheffield Elicitation Framework (SHELF), given its focus on expert discussion and consensus building, to elicit judgements from clinical experts from different backgrounds on the number of annual UK RSV-related hospitalizations across several subgroups of interest. RESULTS: Individual-level probability distributions were successfully elicited. The subsequent consensus workshop was critical for experts to share experiences and rationales for why the number of RSV-related hospitalizations may differ from estimates in published literature. Key points of discussion included that: not all RSV cases are laboratory-confirmed, as diagnosis does not alter treatment; false-negatives are more common than false-positives; literature was outdated; and, since the COVID pandemic, more sensitive tests (PCR) have become available and routine diagnostic testing has increased. After the workshop, group-level probability distributions were generated considering the collaboratively established multiple facets of uncertainty. CONCLUSIONS: While mathematical aggregation is an efficient method to address uncertainty of a large group of experts, behavioral aggregation using SHELF can be valuable to explore more complex quantities that require between-expert interaction with a smaller group of experts when it is expected that the knowledge of the whole is greater than its sum.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeuropeschurerhta30poster131172-pdf.pdf?sfvrsn=67fd20d8_0","title":"ISPOREurope_Schurer_HTA30_Poster131172.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131172","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Is NRDL Supporting Access or Hindering Access in China? A Time-Trend Analysis From 2017-2022","id":"1ff41eca-c30c-4d0e-93cc-405b76a4d59a","sessionCode":"HPR32","topDisplay":"Leong KW1, Wang C2, Macaulay R3 1Precision Advisors, London, LON, UK, 2Precision Advisors, London, UK, 3PRECISIONadvisors, Edinburgh, UK","locationCode":"3057","description":"\r\n\t<div>OBJECTIVES: The rapidly growing pharmaceutical market of China is an attractive market for many multinational companies (MNCs). As the main route for public reimbursement, inclusion in the National Reimbursement Drug List (NRDL) is key for pharmaceutical products to gain wide market access. Since 2017, NRDL’s scope has been expanded to cover innovative, patented drugs and has been updated in five consecutive years. This research examines NRDL outcomes over time. METHODS: NRDL outcomes (2017–2022) were identified and key information extracted (17-May-2023). RESULTS: The total number of medicines included in the NRDL increased by 15%, from 2571(2017) to 2967(2022). An average of 71 new medicines were added per year (range: 74[2021]–375[2017]), 52% of which were ‘Western’ medicines (i.e. non-traditional Chinese medicines). Average negotiation success rate was 80% (range: 65%[2019]–94%[2018]]). However, the average price cut negotiated was 56% and has risen from 44% in 2017 to 60% in 2022. Further, the proportion of negotiated medicines developed by MNCs dropped from 61% in 2017 to 32% in 2021. CONCLUSIONS: Broader inclusion and policy updates show China’s supportive attitude towards providing access for innovative products. However, steep price cuts have been demanded, with prices subject to further decrease upon NRDL renewals. The growing domestic competition layers in extra hurdles for MNCs. To optimize access, it is increasingly important for MNCs to validate price-access trade-offs before pursuing coverage via the NRDL – by assessing the likely level of price discounts required, volume/revenue potential and implications for launch sequencing – with a long-term vision and lifecycle management strategy in place. Beyond the NRDL, MNCs should also consider alternative routes to patient access, such as commercial health insurance, crowdfunding and direct-to-patient financing – particularly in competitive areas with strong domestic presence.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23macaulayhpr32poster132422-pdf.pdf?sfvrsn=2659f1ae_0","title":"ISPOREurope23_Macaulay_HPR32_POSTER132422.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132422","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Technology Assessment (HTA) of Clinical Care Pathways: Peritoneal Dialysis Versus Hemodialysis","id":"0e0a2f54-8787-48cd-81d8-41c5a1134816","sessionCode":"HTA31","topDisplay":"Antonini D1, Basile M2, Di Brino E2, Falasca G2, Fiore A2, Giorgio L2, Laurita R2, Oradei M2, Rumi F3, Cicchetti A2 1Graduate School of Health Economics and Management (ALTEMS), Università Cattolica del Sacro Cuore, Rome, RM, Italy, 2Graduate School of Health Economics and Management (ALTEMS), Università Cattolica del Sacro Cuore, Rome, Lazio, Italy, 3Graduate School of Health Economics and Management (ALTEMS), Università Cattolica del Sacro Cuore, Roma, RM, Italy","locationCode":"4068","description":"\r\n\t<div>OBJECTIVES: Chronic Kidney Disease (CKD) is a condition of altered renal function persisting for more than 3 months. The fifth and final stage of CKD, also known as end-stage renal disease (ESRD), occurs when there is a complete loss of renal function, so patients require renal replacement therapies. The project aims to compare, through a Health Technology Assessment (HTA), the clinical pathway of peritoneal dialysis (PD) with inpatient hemodialysis (HD). METHODS: The HTA report followed the EUnetHTA Core Model® (version 3.0) methodology. Through a literature review, epidemiological aspects of CKD, management of patients with stage 5 CKD, and technical, efficacy, and safety characteristics of PD and HD were identified. The report presents two analyses: a budget impact analysis (BIA), and a cost-utility analysis (CUA) based on a 4-cycle Markov model. The Activity Based Costing approach was used to develop both analyses, considering either the social or National Health Service (NHS) perspective. RESULTS: The literature review yielded 74 relevant studies. In Italy, in 2019, the incidence and prevalence level for CKD was identified as 16.2 and 81.1 per 100,000 population, respectively. A PD and HD utilization rate of 14.3% and 84.3% were also reported, respectively. Safety and efficacy analyses showed no significant differences between PD and HD. The BIA indicated potential savings per patient of € 274.87 at 5 years with an increased PD utilization. CUA’s findings showed that PD is the dominant management strategy compared with HD. These results emerged in both perspectives under analysis and were considered robust based on the sensitivity analyses conducted. CONCLUSIONS: Optimizing the use of PD over HD in stage 5 CKD patients in Italy could lead to improved resource allocation. From a societal perspective, this optimization could result in higher income levels, productivity savings, and improved quality of life for patients and their families.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23antoninihta31poster133585-pdf.pdf?sfvrsn=cc45e1d3_0","title":"ISPOREurope23_Antonini_HTA31_POSTER133585.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133585","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Challenges of Health Economic Modeling for Mental Health, Behavioral, and Neurodevelopmental Conditions","id":"a59972c0-31f7-4cbf-9e3f-427dd61bc758","sessionCode":"HTA4","topDisplay":"Sinha A1, Jones C2 1Mtech Access Ltd, Manchester, LAN, UK, 2Mtech Access Limited, Bicester, OXF, UK","locationCode":"4045","description":"\r\n\t<div>OBJECTIVES: This analysis pragmatically reviews prior health technology assessment (HTA) manufacturer submissions for treatments indicated for mental health, behavioral and neurodevelopmental conditions. It systematically appraises their methodological approaches, establishing key modelling challenges and potential solutions. METHODS: All submissions were identified from NICE’s website. A data extraction table was developed to identify key features from the submissions, including model structures and data sources for clinical, resource use and health-related quality of life (HRQoL) data. Key challenges and solutions were identified and evaluated. RESULTS: Eleven HTA submissions were identified from NICE’s website. Two were terminated, leaving nine for evaluation. HRQoL and resource use data were infrequently collected in the submissions’ underlying clinical trials, commonly addressed by utilizing alternative values from literature. External assessment groups (EAGs) typically acknowledged the necessity of this approach, but criticized where alternative sources represented a different population to that being modelled, or greatly predating the submissions. Five submissions modelled disease pathways beyond the acute phase of treatment, but did not source long-term clinical trial data. Where manufacturers extrapolated data from the acute phase of treatment to generate long-term data, this was well received by EAGs. Lastly, societal costs and caregiver burden were not included in the manufacturers’ base case analyses owing to insufficient data. CONCLUSIONS: The methodological challenges identified within this review include infrequent collection of HRQoL and resource use data and exclusion of societal costs. Data gaps for resource use and HRQoL are generally addressed with literature-derived values. Some models were restricted to shorter time horizons due to a lack of long-term clinical data, compromising their representation of the long-term nature of mental health disorders. Future research should focus on improving data collection practices and better integration of these solutions to strengthen the evidence base for treatments in this area.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023joneshta4poster131472-pdf.pdf?sfvrsn=b283267c_0","title":"ISPOREurope2023_Jones_HTA4_POSTER131472.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131472","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Effectiveness of Sensor Augmented Pump Therapy with Automated Insulin Suspension in the Management of Type 1 Diabetes (T1D) in Bulgaria","id":"6b0d0936-13bd-42e3-8e1c-430d6545a857","sessionCode":"EE14","topDisplay":"Ozdemir Saltik AZ1, Yu J2, Toth J3, Dacheva A4, Slavchev G4, Cohen O1 1Medtronic International Trading Sàrl, Tolochenaz, Switzerland, 2Medtronic International Trading Sàrl, Tolochenaz, VD, Switzerland, 3Medtronic, Zagreb, -, Croatia, 4HTA Ltd., Sofia, 22, Bulgaria","locationCode":"1064","description":"\r\n\t<div>OBJECTIVES: Sensor-augmented pump therapy (SAP) combines continuous glucose monitoring with continuous subcutaneous insulin infusion (CSII). SAP is costlier than CSII but provides additional clinical benefits over CSII alone. This study aims to determine whether SAP therapy with PLGM algorithm is cost-effective versus CSII in people living with T1D in Bulgaria. METHODS: A cost effectiveness analysis was performed using the validated IQVIA CORE Diabetes Model. Clinical and economic inputs were derived from published literature and expert opinion. Separate analyses were performed for hypoglycemic (baseline hypoglycemic event [HE] rate 90/100-patient-years) with baseline HbA1c 7.5% and hyperglycemic (baseline HE rate 10/100-patient-years) with baseline HbA1c 9% cohorts, using a patient-lifetime time horizon. Analyses considered the payer’s perspective. Future costs and clinical outcomes were discounted at 3.5% per annum. RESULTS: The between arm difference in HbA1c reduction was assumed to be -0.56% for hypoglycemic and -0.75% for hyperglycemic cohorts, favouring SAP, based on the formulae derived from a meta-analysis (Pickup JC. et al. BMJ. 2011;343:d3805). An 83% reduction of HE rates with SAP vs CSII was assumed for both cohorts (Choudhary P. et al. Diabetes Technol Ther. 2016 May;18(5):288-91). <ul> <li>SAP was projected to lead to an incremental gain of 2.14 quality-adjusted life years (QALYs) in the hypoglycemic cohort and of 1.13 QALYs in the hyperglycemic cohort.</li> <li>SAP usage increased the mean years of life free from complications, leading up to 3- and 5-years delay in complications onset versus CSII for hypoglycemic and hyperglycemic cohorts, respectively.</li> <li>Higher mean lifetime direct costs for SAP versus CSII resulted in projected incremental cost-effectiveness ratios of BGN40.206/QALY-gained for hypoglycemic and BGN74.403/QALY-gained for hyperglycemic cohorts.</li> <li>Extensive deterministic and probabilistic sensitivity analyses confirmed the robustness of results.</li> </ul> CONCLUSIONS: In Bulgaria, SAP therapy likely represents good value for money versus CSII in T1D management, particularly for the patients experiencing frequent and/or problematic hypoglycemic events.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23ozdemir-saltikee14poster128230-pdf.pdf?sfvrsn=6e496b18_0","title":"ISPOREurope23_Ozdemir Saltik_EE14_POSTER128230.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128230","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Care Resource Utilization and Cost in Parkinson's Disease Patients in a Real-World Setting in Quebec, Canada","id":"1933a6a0-6d96-41e9-8fa7-435ac1a83d55","sessionCode":"EE44","topDisplay":"Baribeau V1, Mohammed S2, Awan A2, Parison D2, Lachaine J1 1PeriPharm Inc, Montreal, QC, Canada, 2AbbVie, Montreal, QC, Canada","locationCode":"1089","description":"\r\n\t<div>OBJECTIVES: Parkinson’s Disease (PD) is among the most common neurodegenerative disorders and is related to severe disabilities. Few studies have estimated the economic impact of PD in a real-world setting. Moreover, the estimates vary widely due to differences in population and definition of PD used. The objective of this project is to analyze in a real-world setting, health care resource utilization (HCRU) of patients with a diagnosis of PD in Quebec, Canada. METHODS: From the Régie de l’Assurance Maladie du Québec (RAMQ) database, a sample of PD patients was selected if they had a diagnosis of PD (ICD-9 code 332.0 or ICD-10 code G20.x) and a PD medication (levodopa, dopamine agonist, catechol-o-methyl transferase inhibitor, monoamine-oxidase-B inhibitor and/or anticholinergic drug) at least once. Patients were matched by age group and sex to controls. RAMQ data were available for the period from January 2010 to December 2018. Annualized HCRU and costs (2021 adjusted) for all-cause of hospitalization, emergency department (ED) visits, outpatient visits and medication were calculated for PD patients and for the control group of non-PD patients. RESULTS: A total of 1,212 patients were included in the analysis. Average age was 74.3 years and 56.8% were male. The average yearly total HCRU cost per patient was 2.7 times higher in PD patients than in the age-and-sex matched control group without PD (CAN$17,405 [SD=22,369] vs. CAN$6,431 [SD=8,189], p<0.01). The difference in total cost was mainly due to higher inpatient cost (CAN$12,171 vs. CAN$3,388). Moreover, PD patients had more outpatient (9.3 [SD=8.3] vs. 6.9 [SD=6.6], p<0.01), and ED (1.7 [SD=2.3] vs. 0.8 [SD=1.2], p<0.01) visits than the matched control group. CONCLUSIONS: PD patients have a high burden of HCRU and cost compared to individuals without PD with the same age and sex, with inpatient cost as the major factor for the difference.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23lachaineee44poster129597-pdf.pdf?sfvrsn=6df581f5_0","title":"ISPOREurope23_Lachaine_EE44_POSTER129597.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129597","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"BoNT-As for Cervical Dystonia: Cost of Treatment and Response to Therapy in Canadian Patients","id":"95696258-3600-4a74-8f9a-43aa2248605d","sessionCode":"EE46","topDisplay":"Johnston K1, Griffin E1, Privolnev Y2 1Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada, 2Ipsen Biopharmaceuticals, Mississauga, ON, Canada","locationCode":"1088","description":"\r\n\t<div>OBJECTIVES: For adults with cervical dystonia (CD), treatment with botulinum neurotoxin type A (BoNT-A) can improve achievement of treatment goals. Differences across individual BoNT-A therapies with respect to acquisition cost, response rates, and dosing frequency can have implications for healthcare spending and patient outcomes. The objective of this analysis was to evaluate average expenditures per response obtained with abobotulinumtoxinA (aboBoNT-A) and onabotulinumtoxinA (onaBoNT-A) for CD in Canada. METHODS: A cost-effectiveness model was developed that incorporated data describing response rates in CD by BoNT-A therapy, health state utilities and health resource utilization by response status, and acquisition cost of BoNT-As in Canada. Response rates and dosing intervals were based on a prospective observational study comparing Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) scores for CD patients receiving aboBoNT-A (32.0%; 17.4 weeks) vs. onaBoNT-A (22.3%; 16.0 weeks). Health resource use by response status was based on a physician survey initially conducted in the United Kingdom and validated by Canadian physicians. Health state utilities by response status were based on published data reporting change from baseline in utility following BoNT-A treatment (0.60 vs. 0.76). Quality-adjusted life years (QALYs) were also adjusted for adverse events (AEs) associated with oral therapies that are utilized more frequently by BoNT-A non-responders. Probabilistic and one-way sensitivity analyses were conducted. RESULTS: Compared with onaBoNT-A, aboBoNT-A resulted in lower annual costs per patient for the management CD (savings of $286), and higher QALYs (increase of 0.02). Results were driven by differences in injection intervals and a higher treatment response rate for people receiving aboBoNT-A compared with onaBoNT-A. Total cost per responder was lower for patients receiving aboBoNT-A compared with onaBoNT-A (CD: $11,701 vs $18,074). Results were consistent across sensitivity analyses. CONCLUSIONS: With higher response rates and reduced costs, aboBoNT-A may be an optimal choice for treating cervical dystonia in Canada.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ip210130-ispor-eu-2023---dysport-cecd-poster-v1-0-for-printingupdated133011-pdf.pdf?sfvrsn=c8e217ad_0","title":"IP_210130 ISPOR EU 2023 - Dysport CE(CD) Poster v1.0 FOR PRINTING_updated133011.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133011","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Decision Modifiers Under Uncertainty: Comparison of Deterministic and Probabilistic Methods in Disease Severity Classification by NICE","id":"4ad14143-af8d-4d98-88a2-4457059606bd","sessionCode":"EE144","topDisplay":"Poole C1, O'Day K2, Cadarette S2, Oladapo T1, Gill M2, Li Q2, Wissinger E2, Carlton R2 1Xcenda (UK) Ltd, part of Cencora, London, UK, 2Xcenda, LLC, part of Cencora, Conshohocken, PA, USA","locationCode":"2078","description":"\r\n\t<div>OBJECTIVES: Replacement of the NICE end-of-life criterion decision modifier with a tiered disease severity criterion based on proportional/absolute QALY shortfall is a significant change in willingness-to-pay policy. Of 19 eligible NICE technology appraisals only 2 have achieved a QALY weighting (1 x 1.2 and 1 x 1.7) largely by deterministic analysis. We evaluated the extent to which NICE’s deterministic approach to disease severity classification could be impacted by parameter uncertainty. METHODS: A review screened 1294 abstracts for published between 2013 and 2019 of which 337 UK-based analyses with lifetime horizons were analysed. Among these, 20 studies (1.5%) reported quasi-lifetime QALYs with uncertainty (standard error [SE] or 95% confidence interval [95%CI]). Deterministic QALY shortfall was assessed using the UK population norms for life-expectancy and age-specific utility recommended by NICE, while probabilistic QALY shortfall was calculated for each weighting threshold (x1.0, x1.2, and x1.7) by using the SE/95%CI to estimate the probability that diseased cohorts would meet the criteria for a proportional or absolute QALY shortfall. RESULTS: Forty diseased cohorts representing usual care in 27 distinct diseases were identified from the selected studies with baseline ages ranging from newborn to 83 years. Most (n=33) were deterministically classified with no QALY weighting (x1.0), 3 cohorts (spinal injury and advanced heart failure) merited a x1.2 weighting, and 4 (inborn errors of metabolism) achieved a x1.7 weighting. Probabilistic classification showed only 5 cohorts where the likelihood of corresponding deterministic classification was less than 90%. CONCLUSIONS: In the available sample good alignment was observed between the deterministic and probabilistic approaches to QALY shortfall assessment. Few peer-reviewed cost-effectiveness studies using the UK reference case report lifetime QALYs with associated uncertainty, which will hamper a priori efforts to understand the likelihood of disease severity classification by NICE stakeholders.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133583","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Use and Cost in Metastatic Castration Resistant Prostate Cancer (mCRPC) in Finland","id":"5234a622-1d28-4f92-ac82-44594fe1c666","sessionCode":"EE84","topDisplay":"Rannikko A1, Ågesen T2, Hölsä O3, Tuominen S3, Ekman M4, Mattila R5 1University of Helsinki and Helsinki University Hospital, Helsinki, 18, Finland, 2AstraZeneca, Nordic, Oslo, Oslo, Norway, 3Medaffcon Oy, Espoo, 18, Finland, 4AstraZeneca, Nordic, Stockholm, Stockholm, Sweden, 5Medaffcon Oy, Espoo, Finland","locationCode":"2010","description":"\r\n\t<div>OBJECTIVES: There is limited real-world data on healthcare resource utilization (HCRU) for patients with metastatic castration resistant prostate cancer (mCRPC). The aim was to assess current specialized care HCRU and cost per patient with mCRPC in Finland. METHODS: All patients diagnosed with prostate cancer (ICD-10: C61) from 2013 to 2021 were identified from the Hospital districts of Helsinki and Uusimaa, and Southwest Finland, respectively, covering approximately 40% of the Finnish population. Specialized care data were collected from the electronic medical records of the respective hospital data lakes. HCRU-related costs were derived using publicly available unit costs. All visits, treatments, and procedures are included in the prices as national averages according to the specialty and type of visit. PC specificity was determined based on the recorded C61 diagnostic codes. RESULTS: We identified 31 307 prostate cancer patients, of whom 2 475 developed mCRPC during the study period, with a median age of 76 years (inter-quantile range 70-82). The number of outpatient contacts was 22.3 (95% CI:21.7-23.0) per patient year, of which 60% were PC-specific. Patients had 1.2 (95% CI:1.1-1.2) hospitalizations per year, with 5.0 (95% CI: 4.7-5.4) inpatient days, the majority of which (76%) were not documented as PC-related hospitalizations (76%). In addition, patients had 3.6 (95% CI:3.4-3.9) visits to radiotherapy per year, mostly driven by PC. All-cause costs per patient during the follow-up period were 19 277€ (95% CI:18 542-20 016€) for outpatient costs and 9 870€ (95% CI: 9 118-10 685€) for inpatient costs. No major differences were detected in costs between the first-line treatment and the entire follow-up period. CONCLUSIONS: Patients with mCRPC have a significant number of specialty care HCRU that are not directly associated with C61 diagnosis, indicating a significant burden of disease outside the confines of PC management and treatment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23mattilaee84poster129881-pdf.pdf?sfvrsn=c309fb94_0","title":"ISPOREurope23_Mattila_EE84_POSTER129881.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129881","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Psychometric Performance of the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Questionnaire Among Patients With Paroxysmal Nocturnal Hemoglobinuria","id":"6b0a7788-5898-4631-a922-447905ab4014","sessionCode":"PCR31","topDisplay":"Cella D1, Vallow S2, Bermann G3, McDonald J4, Ngerano G5, Linton S4, Arenson E4, Maitra S6, Lamoureux R7, Dickie G4 1Northwestern University, Evanston, IL, USA, 2Novartis Pharmaceuticals Corporation, New Hope, PA, USA, 3Novartis Pharma AG, Basel, Switzerland, 4Adelphi Values, Boston, MA, USA, 5Adelphi Values, Raleigh, NC, USA, 6Novartis Healthcare Pvt. Ltd., Hyderabad, Rangareddy, India, 7Adelphi Values, North Chelmsford, MA, USA","locationCode":"6033","description":"\r\n\t<div>OBJECTIVES: To evaluate the psychometric properties of the Functional Assessment of Chronic Illness Therapy—Fatigue (FACIT-Fatigue) in two Phase 3 trials: APPLY-PNH (randomized, active-controlled open-label) and APPOINT-PNH (single-arm, open-label) for patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare hemolytic disorder characterized by hemolysis, bone marrow failure, and thrombosis. Fatigue is a frequently reported symptom. METHODS: FACIT-Fatigue items were evaluated for item distribution and floor and ceiling effects. A bifactor model was conducted to test for essential unidimensionality and construct validity of a total score. Reliability analyses assessed both the internal consistency and stability over time of FACIT-Fatigue total score. Concurrent validity of the FACIT-Fatigue total score was assessed by examining correlations with a question on the patient’s global impression of fatigue severity (PGIS) and items from the EORTC-QLQ-C30 and EQ-5D-5L. Ability to distinguish between clinically distinct groups was assessed by PGIS level and hemoglobin change. Responsiveness to change was assessed by anchoring FACIT-F score to changes in EORTC-QLQ-C30 fatigue scores, PGIS level, and hemoglobin. Analyses were conducted separately by trial, except for the bi-factor model analysis, which used pooled data from APPOINT-PNH and APPLY-PNH to increase sample size. RESULTS: Analysis population for APPLY-PNH (N=95) was 68% female, median age 53.0 years; for APPOINT-PNH (N=40) it was 57% male, median age 38.5 years. Participants endorsed the full range of FACIT-Fatigue response options. A bifactor model supported the essential unidimensionality of FACIT-Fatigue scores which were both internally consistent and stable across time. Mean FACIT-Fatigue total score decreased with increased fatigue severity. Strong correlations were found between changes in FACIT-Fatigue total score with changes in scores for the other PRO measures, and weak-to-moderate correlations with change in hemoglobin. CONCLUSIONS: The FACIT-Fatigue was found to be reliable, construct-valid, and appropriate for use in measuring change in fatigue experienced by participants with PNH.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-pnh-facit-psychometric-poster-v70133095-pdf.pdf?sfvrsn=f3da6e55_0","title":"ISPOR EU PNH FACIT Psychometric Poster v7_0133095.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133095","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Adjusting for Switches to Multiple Treatments in Randomised Controlled Trials: A Comparison of Inverse Probability Weighting and Two-Stage Estimation Methods","id":"ce663463-9db1-4c48-8ecf-44a95b9566b2","sessionCode":"MSR9","topDisplay":"Bell Gorrod H1, White IR2, Mt-Isa S3, Hmissi A3, Vandormael K4, Cappoen N4, Latimer N5 1University of Sheffield, Sheffield, UK, 2University College London, London, UK, 3MSD, Zurich, Switzerland, 4MSD, Brussels, Belgium, 5University of Sheffield & Delta Hat Limited, Sheffield, DBY, Great Britain","locationCode":"5052","description":"\r\n\t<div>OBJECTIVES: Treatment switching commonly occurs in randomised controlled trials (RCTs). Participants may switch onto the comparator, or another treatment. Statistical adjustment methods have been used in health technology assessment to estimate outcomes that would have been observed in the absence of switching. The performance (e.g., bias, accuracy) of adjustment methods has been assessed in simulation studies, but these have focused on switching between randomised treatments. In practise, patients could switch onto a range of alternative treatments with different treatment effects. This study assesses the performance of adjustment methods in the presence of switching to multiple different treatments. METHODS: Survival data was simulated with patients in the control group able to switch onto multiple treatments. Inverse probability of censoring weights (IPCW) and Two-stage estimation (TSE) adjustment methods were applied, testing methods that (i) considered and adjusted for each type of switching separately; (ii) combined switchers and adjusted for switching without differentiating by treatment switched to. Adjusted results were compared to the simulated truth in 40 scenarios. Switch proportions, treatment effects, censoring proportions, and sample size were varied. RESULTS: IPCW and TSE applications that distinguished between the type of switch produced similar bias to applications which grouped all switches together. Modelling each type of switch separately did not result in more accurate adjustment analyses. TSE analyses performed well across all scenarios, while IPCW resulted in substantial bias in scenarios with high switching proportions. CONCLUSIONS: In situations where it is appropriate to adjust for switches to multiple treatments in RCTs, adjustment methods can be applied in different ways. In the scenarios tested, there was little advantage associated with adjusting for each type of switch separately, compared with applications that combined types of switch together. TSE produced low bias across all scenarios, whereas IPCW performed less consistently.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/switching-ispor-poster-final-with-code132006-pdf.pdf?sfvrsn=d0d1e6ba_0","title":"Switching ISPOR poster final with code132006.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132006","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Paternal Treatment on Birth Outcomes Among Individuals with Autoimmune Rheumatic Diseases","id":"b229d8f2-048d-4c5c-b8b0-452da93e97c8","sessionCode":"EPH30","topDisplay":"Shao YH Taipei Medical University, New Taipei City, New Taipei City, Taiwan","locationCode":"3032","description":"\r\n\t<div>OBJECTIVES: This study aimed to investigate the effects of conventional synthetic and biologic disease-modifying antirheumatic drugs (csDMARDs and bDMARDs) on birth outcomes in fathers with autoimmune rheumatic diseases (AIRDs). METHODS: We established a population-based birth cohort (n=35,049) comprising fathers diagnosed with AIRDs between 2004 and 2019. Data from the Maternal and Child Health Database, Taiwan Birth Certificate Registry, and Taiwan National Health Insurance Research Data were utilized. Paternal preconception exposure was defined as having a prescription between 39 and 60 weeks before conception. The primary outcomes assessed were congenital malformations, small-for-gestational age, and preterm births. The effects of variables on the associations of interest were evaluated using inverse probability of treatment-weighted logistic regression models. RESULTS: The results revealed that paternal exposure to csDMARDs or bDMARDs was associated with an elevated risk of preterm labor (odds ratio [OR]: 1.46; 95% confidence interval [CI]: 1.36-1.57) in their offspring. Paternal treatment with methotrexate (OR: 1.15; 95% CI: 1.04-1.26), cyclosporine (OR: 1.31; 95% CI: 1.09-1.57), and tumor necrosis factor inhibitors (TNFis) (OR: 1.16; 95% CI: 1.01-1.33) was associated with a higher risk of birth defects compared to their counterparts. Exposure to csDMARDs was significantly linked to increased risks of deformities in the central nervous system (OR: 1.27; 95% CI: 1.13-1.42), gastrointestinal system (OR: 1.29; 95% CI: 1.16-1.44), and musculoskeletal system (OR: 1.13; 95% CI: 1.05-1.22). Paternal preconception treatment with bDMARDs was correlated with oral cleft (OR: 1.97; 95% CI: 1.32-2.95), gastrointestinal system (OR: 1.59; 95% CI: 1.19-2.13), and musculoskeletal defects (OR: 1.56; 95% CI: 1.28-1.90). CONCLUSIONS: The study findings suggest that paternal exposure to csDMARDs and bDMARDs may contribute to adverse birth outcomes. These results underscore the importance of making informed decisions regarding medication use before planning a pregnancy, even for male patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129423","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Budget Impact of Patient Blood Management in the Cardiovascular Surgery Department of a Turkish Private Hospital","id":"b14b8060-8f68-45fb-885b-4589afdaae01","sessionCode":"EE11","topDisplay":"Tatar M1, Ramirez de Arellano Serna A2, Akdeniz CS3, Zeybey U3, Sahin S3, Ciftci C3 1Vitale Health Economics and Policy, London, UK, 2CSL Vifor, Glattbrugg, ZH, Switzerland, 3Demiroglu Bilim University, İstanbul, Turkey","locationCode":"1040","description":"\r\n\t<div>OBJECTIVES: The objective of this study is to predict the potential budget impact of implementing patient blood management (PBM) in the cardiovascular surgery department of a Turkish private hospital. METHODS: Two budget impact models were developed: one based on avoided complications (sepsis with pneumonia, sepsis without pneumonia, renal failure, myocardial infarction and stroke) of patients suffering from preoperative anemia. A second one based on avoided transfusions-related complications (sepsis, renal failure, myocardial infarction, stroke). Two hospital data sets were used in the analysis. The first data set covered overall data from 302 operations undertaken in the department between 2018-2022. These data were used in estimations for number of anemic patients, number of red blood cell transfusions (RBC) and length of stay in the hospital. The second data set covered the number of operations between 2020-2022. Recent meta-analysis results were used to estimate the number of avoided complications with the preoperative treatment of anemic patients and the number of avoided complications due to reduction in the use of RBC transfusions. Treatment costs of complications were estimated with the reimbursement prices of the Turkish healthcare payer (SSI). RESULTS: Preoperative treatment of anemia could have saved 4,189,802 TRY by avoiding anemia related complications. Total budget savings from reduction of RBC transfusions were estimated as 6,174,434 TRY. These are underestimations of real savings as the public prices of SSI were used in calculating the cost of treating complications. In addition to these, the hospital could have treated 137 more patients in the given period due to decreased length of stay. With these additional patients, the hospital could have earned additional 4.544.002 TRY with the floor prices of the Turkish Medical Association. CONCLUSIONS: PBM is a budget saving option for both the SSI and hospital. Adopting PBM programs in both public and private hospitals is recommended.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23tatare11posterv2129632-pdf.pdf?sfvrsn=924f929a_0","title":"ISPOREurope23_Tatar_E11_POSTERV2129632.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129632","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimated Spending on a Novel Gene Therapy for Treatment for Patients With Dystrophic Epidermolysis Bullosa","id":"0a6955fe-36a5-4c06-845b-4589c4246ca1","sessionCode":"EE88","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Novel gene therapies may offer important treatment opportunities for patients with rare genetic diseases such as dystrophic epidermolysis bullosa (DEB). DEB has a mild autosomal dominant variant and a severe autosomal recessive form that is associated with severe impacts to quality and length of life. Beremagene geperpavec (B-VEC) was FDA-approved in the United States (US) in May 2023 for both forms of DEB. While this therapy offers substantial benefits for autosomal recessive patients, there is greater uncertainty about its benefit in patients with autosomal dominant disease, who were largely excluded from the pre-approval clinical studies. We estimated expenditures on B-VEC therapy using different scenarios of the eligible population for treatment. METHODS: We estimated the number of prevalent and incident cases of DEB over a three-year period using US national registry data, and healthcare spending on B-VEC during the first three years of treatment assuming an annual cost of $300,000 per patient (consistent with the manufacturer-reported range). We also estimated lifetime total costs of treatment. RESULTS: For patients with autosomal dominant or autosomal recessive DEB treated with B-VEC in the first year, estimated spending in the US was $268 million (range: $178-$356 million). Over a three-year period estimated spending was $805 million (range: $536 million-$1.1 billion). If the indication were narrowed to only autosomal recessive patients, estimated three-year spending would be $399 million (range: $266-$533 million). Estimated lifetime total per patient costs for autosomal recessive disease and autosomal dominant disease amounted to $15 million (range: $10-$20 million) and $16 million (range $11-$22 million), respectively. CONCLUSIONS: B-VEC is an important new therapy for DEB, but the eligible population is a key factor in the ultimate costs borne by payers. The FDA should carefully consider whether it is appropriate to expand such products’ approvals beyond the populations in which they were tested.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133786","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Use and Costs Associated with Herpes Zoster/Post-Herpetic Neuralgia Episodes in Italy: A Retrospective Observational Study","id":"dc587410-ab6e-42c0-b17a-45a55c8f7b92","sessionCode":"EE142","topDisplay":"Zarkadoulas L1, Santacroce R2, Comparoni S2, Freguja R2, Veronesi C3, Dovizio M3, Degli Esposti L3 1GSK, Wavre, WBR, Belgium, 2GSK, Verona, VR, Italy, 3CliCon S.r.l. Società Benefit Health, Economics & Outcomes Research, Bologna, BO, Italy","locationCode":"2073","description":"\r\n\t<div>OBJECTIVES: Herpes Zoster (HZ) risk of infection increases with age and immunocompromised (IC) status due to underlying treatment or condition. HZ and post-herpetic neuralgia (PHN) generate considerable clinical and economic burdens. We estimated healthcare resource utilization and direct costs among HZ or/and PHN patients, in general adults, and adults with comorbidities, in Italy. METHODS: We conducted an observational retrospective analysis using reimbursement data from a pool of Italian local health authorities. Data between 01/2009 and 06/2022 were analyzed, covering approximately 20% of the Italian population. Adult patients were characterized and followed-up during at least one year 1-period before and after the index-date (first HZ/PHN hospitalization or HZ brivudine prescription). Direct healthcare costs were estimated in the overall adult HZ/PHN population and compared to non-HZ/PHN adults. Sub-group analyses were performed for older adults (≥50 years), IC adults, adults with chronic diseases, and adults with cancers. RESULTS: Among 193,259 HZ/PHN included adults (mean age 61.6 years), 145,923 were ≥50 years old; of them, 29.9% were IC and 76.1% had ≥1 chronic condition. Among the 3,618 patients aged ≥50 years included by hospitalization, the observed mean length of stay was of 18.3 days. During the 1-year follow-up, 18.8% of patients aged ≥50 years developed PHN complications. Total mean annual healthcare costs (adjusted for the 2022 inflation rate) following HZ/PHN diagnosis was estimated at 272M€; costs were observed to increase with age; 1,050€ (50-59 years), 1,382€ (60-64 years), 1,780€ (65-69 years), 2,327€ (70-79 years), 2,631€ (≥80 years), and mainly driven by drug prescriptions and all-cause hospitalizations. Among overall adults, higher healthcare costs were observed in HZ/PHN patients with IC condition, or with chronic diseases, or with cancers, when compared to their non-HZ/PHN counterparts. CONCLUSIONS: This real-world analysis showed that HZ poses a substantial economic burden in older adults and individuals with comorbidities, in Italy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23zarkadoulasee142poster131672-pdf.pdf?sfvrsn=8d0bab61_0","title":"ISPOREurope23_Zarkadoulas_EE142_POSTER131672.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23zarkadoulasee142handout131672-pdf.pdf?sfvrsn=9aec57f4_0","title":"ISPOREurope23_Zarkadoulas_EE142_HANDOUT131672.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131672","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Implication of Global Insights on Managed Entry Agreements for High-Cost Innovative Medicines in the South-East Asia Region","id":"6b339244-0d5a-4e98-a595-45c0204c59c7","sessionCode":"HPR39","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"4004","description":"\r\n\t<div>OBJECTIVES: Managed entry agreements (MEAs) are arrangements between manufacturers and healthcare payers/providers that facilitate the health technology assessment of innovative medicines, typically for oncology and rare diseases, that present financial and/or clinical uncertainties. MEAs aim to provide early access with broader coverage while minimizing the impact on healthcare budgets. MEAs are widely utilized, particularly in Central and Eastern Europe, with sporadic implementation in Asia (specifically South Korea and Taiwan), but not in Southeast Asia (SEA). This research aims to investigate the feasibility of implementing MEAs in SEA to enhance access to high-cost innovative medicines. METHODS: Qualitative surveys were conducted with eight experts, comprising payers and policymakers from six countries, namely Italy, the UK, Australia, Hong Kong, Taiwan, and South Korea. Nine questions were administered to gain insights into the drivers and barriers encountered during the implementation of innovative funding models in their respective countries, along with recommendations for SEA. Common themes that could inform the implementation of MEAs in SEA were identified. RESULTS: Key success factors for implementing MEAs in these countries included having strong political support, engaging relevant stakeholders, establishing robust data infrastructure, employing formal and comprehensive health technology assessment processes, ideally within a single-payer system. Experts emphasized the significance of formal appraisal mechanisms with clearly defined contractual terms, supplemented by additional alternative funding sources, to ensure the sustainability of access to high-cost drugs. Regarding the implementation of MEAs in SEA, experts suggested the following: 1) fostering intersectoral collaboration with stakeholder engagement and clear policy direction to cultivate expertise in MEAs, overcome bureaucratic hurdles, and address stakeholder capacity limitations; and 2) developing robust and standardized data infrastructure to enhance evidence generation for outcomes evaluation. CONCLUSIONS: By leveraging global and regional experiences, SEA can explore MEAs as valuable tools to facilitate patient access to effective innovative medicines while maintaining adequate control over healthcare budgets.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129555","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Systematic Literature Review on the Global Epidemiology of Angelman Syndrome","id":"785e232b-00cb-426d-aab5-471447965b4e","sessionCode":"EPH31","topDisplay":"Arregui Rementeria M1, Cadarette S2, Oladapo T3, Wissinger E4, Ruiz K4, Kistler K4 1Xcenda GmbH, part of Cencora, Castellon, Spain, 2Xcenda L.L.C., part of Cencora, Cary, NC, USA, 3Xcenda UK Limited, part of Cencora, London, UK, 4Xcenda L.L.C., part of Cencora, Conshohocken, PA, USA","locationCode":"3033","description":"\r\n\t<div>OBJECTIVES: Angelman syndrome (AS) is a rare genetic disorder caused by loss of UBE3A expression in neurons and characterized by generalized developmental delay, including severe intellectual disability. Treatment options are limited and focus on symptom management. It is unknown how many people are affected by AS. We aimed to determine the global prevalence of AS to help describe the full burden of this condition. METHODS: This systematic literature review (SLR) identified English-language population-based studies indexed in MEDLINE and EMBASE through March 2023 or presented at relevant congresses (2021-2023) reporting on AS prevalence. Screening and data extraction were conducted by 1 researcher; a second researcher conducted quality checks. If not specified in studies, prevalence was approximated using the reported number of cases and the population size of the relevant geography during the reporting period. RESULTS: Of 203 unique publications identified, 10 independent studies met eligibility criteria. Studies (mostly of retrospective design) were conducted in Europe (5 studies), Asia (3), North America (1), and Australia (1) and were heterogenous in terms of sample size, availability of genetic confirmation, time period, and reference population. The estimated AS prevalence per 10,000 people was 0.076 in Madrid, Spain (2013-2014), 0.075 in Japan (2009) and 0.163 in Australia (2003); prevalence per 10,000 adults was 0.212 in Marshfield, Wisconsin (2003); prevalence per 10,000 children was 0.219 in Saudi Arabia (2004-2005); and birth prevalence (per 10,000 live-births) was 0.252 in Denmark (1994–2014) and Finland (1990-2014), 0.468 in Estonia (2016), 0.448 in Hong Kong (1995-2015), and 0.248 in Western Australia (1953-2003). CONCLUSIONS: This SLR found that AS prevalence data are few and outdated. Prevalence estimates vary within reference populations, which may be due to variation in study years, geography or study methodology. Recent world-wide data on AS prevalence are needed to corroborate the prevalence estimates currently available.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131750","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"RSV Hospitalizations’ Burden in New-Born Patients and Potential Impact of Maternal Vaccination: A French Analysis","id":"c98dac11-63dc-4d8b-811b-47294d33f47f","sessionCode":"EPH13","topDisplay":"Fahfouhi Y1, Fiévez S2, Blanc E3, Marié L4, Densari Z4, Chillotti L5, Cancalon C6, Kernaleguen C6, Nuttens C7 1Pfizer, Paris, France, 2Pfizer SAS France, Paris, 75, France, 3Pfizer SAS France, Paris, France, 4stève consultants, Paris, France, 5stève consultants, Oullins, 69, France, 6Stève Consultants, Oullins, France, 7Pfizer, Paris, 75, France","locationCode":"3013","description":"\r\n\t<div>OBJECTIVES: Respiratory Syncytial Virus (RSV) infection is a major cause of hospitalizations for bronchiolitis in children in France. This study aims to estimate RSV-related hospitalizations in France for infants < 1 year of age and to measure the impact of annual maternal vaccination. METHODS: A retrospective, real-world study based on the French National hospital database (January 1st, 2015 to December 31st, 2021) was conducted. RSV-related hospitalizations were identified by the presence of one of the following ICD-10 diagnosis codes (J121, J205, J210, J219, J45 and R062). Descriptive statistics on patient characteristics, hospital stays, and associated costs were performed. To assess the impact of vaccination on the reduction of hospitalization, a Markov model combining the efficacy of maternal vaccination from the MATISSE trial and hospitalizations rates was developed. RESULTS: Overall, 300 951 hospitalizations and 261 459 patients were identified. Over the study period, 57% of the hospitalizations occurred during the RSV season (November to January) and 43% of stays occurred outside the epidemic seasons. In total, 46% of hospitalizations were identified in infants < 3 months of life and 27% in infants between 3 and 6 months of life. Almost 90% of hospitalizations involved full-term infants. One hospitalization out of 5 required a stay in intensive care unit and 5% required resuscitation support, with higher rate among the youngest patients. Mean hospitalization cost was estimated at 3 100 € for patients < 1 month and 2 207 € for those aged between 1 and 6 months. By applying the efficacy of maternal vaccination to the RSV-related hospitalizations, a reduction of 57% of hospitalisations was estimated, resulting in a cost saving of 84 million of euros. CONCLUSIONS: RSV infection represents a considerable burden in hospital setting in France, especially during the first months of life. Maternal vaccination suggests a significant benefit in decreasing this burden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/postervfisporp0207pfizerrsv133228-pdf.pdf?sfvrsn=935df872_0","title":"Poster_VF_ISPOR_P0207_Pfizer_RSV133228.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133228","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Rise of Undiagnosed Major Depressive Disorder Post-COVID-19 Pandemic in Europe","id":"798b7a0d-a663-44f4-8018-47ca89281472","sessionCode":"PCR9","topDisplay":"Annunziata K, Krupsky K, Lee L Oracle Life Sciences, Kansas City, MO, USA","locationCode":"6014","description":"\r\n\t<div>OBJECTIVES: To compare rates of diagnosed depression and undiagnosed depression among adults in the EU4 (France, Germany, Spain, and Italy) and the United Kingdom (UK), from pre-COVID-19 to post COVID-19 years using a representative general population survey. METHODS: Data from the 2011(N=57,512), 2018(N=62,000), and 2021(N=62,028) EU4+UK National Health and Wellness Survey, a nationally-representative, cross-sectional online survey of adults (aged ≥18), was analyzed. Respondents were classified as having major depressive syndrome (MDS) based on the Patient Health Questionnaire 9-item depression score (PHQ-9). MDS included 5 or more of the 9 depressive symptom criteria have been present at least “more than half the days” in the past 2 weeks, and 1 of the symptoms is depressed mood or anhedonia. Those who self-reported having depression that was diagnosed by a physician were classified as diagnosed. Those who did not self-report experiencing depression were classified as undiagnosed. Descriptive statistics were conducted. RESULTS: Diagnosed MDS in pre-COVID-19 years, 2011 and 2018 were 3.1%(n=1,799) and 3.7%(n=2,285), respectively. For those same years, those receiving a prescription treatment for depression were 65.1%(n=1,172) and 63.6%(n=1,454). In post COVID-19 2021, diagnosed MDS was 3.5%(n=2,175) and those on treatment was 61.8%(n=1,345). In 2011, using PHQ-9 to screen for MDS the undiagnosed rate was 3.5%(n=1,997). This rate increased to 4.2%(n=2,601) in 2018 and to 6.2%(n=3,834) in 2021. In 2021, among those with undiagnosed MDS, 70.1% had moderately-severe to severe depression. CONCLUSIONS: While rates of diagnosed MDS and treatment remained consistent from pre-COVID-19 to post COVID-19, the rates of undiagnosed MDS increased 1.5 times from the year just prior to COVID-19 to post COVID-19. These results reveal the underlying problem with MDS – many individuals are undiagnosed and untreated even as they experience severe depression. More than ever before, now in this post COVID-19 environment, access to mental health support and therapists are needed.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023posterdepcovidfinal133849-pdf.pdf?sfvrsn=f8f7a0bf_0","title":"ISPOR 2023_Poster_DepCOVID_FINAL133849.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133849","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Need for Economic Systems Modelling to Assess the True Value of Healthcare Innovations: An Application to Influenza Vaccinations","id":"e4108142-45f1-4ec7-868f-47e11418e4a5","sessionCode":"EE139","topDisplay":"Romanelli R1, Hafner M2 1RAND Europe, Cambridge, CAM, UK, 2RAND Europe, St Albans, UK","locationCode":"2067","description":"\r\n\t<div>OBJECTIVES: Public health challenges are often conceived as complex systems issues, characterized by interdependencies and diverging stakeholder priorities. The impact of healthcare interventions on complex systems varies based on numerous factors and not considering these system dynamics will lead to incomplete assessments of the value of new health technologies. METHODS: Taking a systems approach, we examine the value of seasonal influenza vaccinations in the UK by linking a dynamic disease transmission model with a computable general equilibrium (CGE) model representing the UK economy. CGE models reflect the full economic system including different domestic and international economic agents (e.g., households, firms, governments) with a detailed sectorial aggregation, including health and social care. The combined modelling approach between an epidemiological and economic model enables the assessment of clinical outcomes over time (e.g. number of infections, hospitalizations) and their economic and fiscal impacts (e.g. gross domestic product, government expenditures and tax income) over time. Analytically, different influenza vaccination scenarios are compared, varying by population groups (e.g., older adults, children). RESULTS: Applying a CGE modelling approach can help to quantify a wider set of value drivers of healthcare innovations. First, the modelling approach considers the entire economy as an interconnected system, considering both direct and indirect effects of an innovation and looks beyond the immediate impacts on the healthcare sector to understand all impacts across other sectors. Second, it can be used to understand the fiscal impacts of healthcare innovations and can illuminate their distributional effects, identifying which groups are likely to benefit or lose within the economic system. CONCLUSIONS: The use of CGE models in health economic evaluations and public health provides a holistic approach to understand and evaluate the complex fiscal and economic impacts of healthcare innovations. This systems approach is vital if we are to develop effective, sustainable, and equitable health interventions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130826","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Testing the Psychometric Properties of Several Euroqol Instruments for Measuring the Impact of COVID-19 in a Large Sample of Chinese Children and Their Parent Carers","id":"6873cff7-7be3-4378-b97f-4843e7e9f16e","sessionCode":"PCR26","topDisplay":"Zhou W1, Busschbach JV2, Herdman M3, Yang Z4 1Erasmus Medical Centre, Rotterdam, ZH, Netherlands, 2Erasmus University Rotterdam, Rotterdam, Netherlands, 3The Office of Health Economics, London, UK, 4Guizhou Medical University, Gui'an, 52, China","locationCode":"6027","description":"\r\n\t<div>OBJECTIVES: To test the Chinese EQ-5D-Y-3L value sets and EQ-TIPS in a large sample of young patients with COVID-19 in China, and to compare the differences between EQ-5D-5L and EQ-HWB-S in parent carers and identify the spillover effects of children's COVID-19 infection on their parents. METHODS: A longitudinal study was conducted on children aged 0-18 years with COVID-19 and their parents. Baseline and follow-up surveys were completed by 861 and 311 dyads, respectively. An additional 231 non-infected children were included as controls. Parents completed the EQ-TIPS, proxy EQ-5D-Y-3L, EQ-HWB-S, and EQ-5D-5L on a tablet. Children aged ≥6 years self-completed the EQ-5D-Y-3L. The analysis involved assessing known-groups validity, convergent validity, test-retest reliability, inter-rater agreement, and responsiveness of the EQ instruments. RESULTS: Children with COVID-19 and their parents, particularly those with more severe disease or poorer overall health assessment, exhibited higher EQ-TIPS Level Sum Score (LSS) and lower EQ-5D-Y/EQ-HWB-S/EQ-5D-5L index scores and EQ VAS. EQ-5D-5L captured a broader range of child health conditions compared to EQ-HWB-S. Moderate to strong correlations were found between overlapping dimensions of EQ-5D-5L and EQ-HWB-S. The EQ measures demonstrated good reliability, with ICCs ranging from 0.702 to 0.866 for test-retest agreement and 0.653 to 0.823 for inter-rater agreement. EQ-TIPS exhibited the highest responsiveness to COVID-19 recovery or improved OHA (ES: 1.21 - 1.75). Proxy EQ-5D-Y outperformed the self-completed version, while EQ-5D-5L performed slightly better than EQ-HWB-S in terms of responsiveness. Both EQ-HWB-S and EQ-5D-5L could estimate spillover effects associated with a child's health condition. CONCLUSIONS: The findings support the reliability, validity, and responsiveness of EQ-TIPS and EQ-5D-Y-3L in assessing health outcomes of young patients with COVID-19. EQ-HWB-S and EQ-5D-5L demonstrated strong performance, showing comparable construct validity and responsiveness. This highlights their potential for estimating the spillover effects related to a child's health condition in the context of COVID-19 infection.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-europe-2023-poster130227-pdf.pdf?sfvrsn=4f38fa27_0","title":"ISPOR Europe 2023-poster130227.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130227","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health-Related Quality of Life Multi-Instrument Comparison for the Use in Children Aged 2-4 Years Old","id":"7ddb5a4a-affa-4d64-9b8a-48ac3e90f20e","sessionCode":"MSR16","topDisplay":"van Heusden A1, Rivero-Arias O2, Herdman M3, Hiscock H4, Devlin N5, Dalziel K1 1The University of Melbourne, Melbourne, VIC, Australia, 2University of Oxford, Oxford, OXF, UK, 3Office of Health Economics, Mataró, B, Spain, 4Murdoch Children’s Research Institute, Melbourne, VIC, Australia, 5University of Melbourne, Melbourne, VIC, Australia","locationCode":"","description":"\r\n\t<div>OBJECTIVES: To test and compare the psychometric performance of the adapted EQ-5D-Y-3L/5L for use in children aged 2-4 years against CHU9D and HUI2/3 in children aged 2-4 years. METHODS: Survey data of children aged 2-4 years were obtained from the Australian Pediatric Multi-Instrument Comparison (P-MIC) study. Psychometric performance of the instruments was assessed by; feasibility, convergent validity (Spearman’s correlations), ceiling effects, known-group validity (Cohen’s D effect sizes for pre-specified groups), test re-test reliability (intra-class correlation coefficients), and responsiveness (standardized response mean effect sizes for changes in health) by health status using level summed scores. RESULTS: A total of 842 parents of children aged 2-4 years completed the survey with 33.1% of children having a special healthcare need. All instruments were easy to complete. High ceiling effects were observed for the adapted EQ-5D-Y-3L/5L and HUI2/3. The adapted EQ-5D-Y-3L had the largest Cohen’s D effect sizes for the majority of known groups analyses (54%) followed by CHU9D (38%). The adapted EQ-5D-Y-3L/5L had the largest interclass correlation coefficients for test re-test reliability (ICC:0.81,0.83). For responsiveness of improved health, the adapted EQ-5D-Y-3L, CHU9D, and HUI2/3 had the largest effect sizes (SRM:0.44-0.46). For responsiveness of worsened health, the adapted EQ-5D-Y-3L and HUI2/3 had the largest effect sizes (SRM:0.23). CONCLUSIONS: The adapted EQ-5D-Y-3L/5L and CHU9D showed overall good psychometric performance. The HUI2/3 performed poorly for almost all known-groups analyses compared to the other instruments. The adapted EQ-5D-Y-3L appears to have the best psychometric performance, based on tests performed, for the use in children aged 2-4 years.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130731","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Enhancing Prostate Cancer Care by Optimizing Patient Engagement with Digital Tools","id":"03c93bbc-7219-4740-ba98-48c1f184450a","sessionCode":"HSD8","topDisplay":"Tallentire CW1, Doogan E2, Povsic M1 1AMICULUM ACCESS, Bollington, Cheshire, UK, 2AMICULUM ACCESS, Bollington, CHE, UK","locationCode":"4020","description":"\r\n\t<div>OBJECTIVES: Digital tools can play an important role in personal monitoring and symptom management in prostate cancer (PC), which can reduce the strain on healthcare systems. This study aimed to explore patient engagement with digital tools and how to optimize their functionalities to better support PC care. METHODS: A literature review of real-world use of digital tools was conducted in PC care published within the last 5 years. The target database was Embase, supplemented with handsearching of PC congresses. The search strategy used keywords such as “telemedicine”, “app”, and “self-management”. RESULTS: Sixteen studies were included (1193 participants). Identified digital tools and their engagement rates (ie patients who used the digital tool/patients that were provided with the tool) were: apps (n=8, 43–88%), web-based platforms (n=7, 24–93%), wearable devices (n=4, 63–96%) and telephone (n=3, 71–93%). The most common functionality identified was symptom reporting (n=13), which was often combined with healthcare provider (HCP) monitoring (n=10). Other common functionalities included access to health/lifestyle records (N=8), direct communication with HCPs (n=10), digitized PC information (N=6), mental health support (N=6) and physical activity tracking (n=3). Functionality-specific engagement was 66–83%, the lowest being associated with HCP monitoring (range=24–93%) and the highest being associated with physical activity tracking (range=63–96%). Engagement rate was negatively correlated with patient age and disease stage. CONCLUSIONS: Digital tool engagement in PC was generally high and not tool-specific. Personal monitoring and symptom management showed the highest engagement – these are probably the most effective functionalities in enhancing PC care and should be implemented in future tools. With the advent of AI, there is great potential for improving outcomes and reducing healthcare burden across cancer care. Further research is required to determine the most effective digital tool design for older users and those with advanced disease.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dooganhsd8129826-pdf.pdf?sfvrsn=a177c1d2_0","title":"ISPOREurope23_Doogan_HSD8129826.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129826","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Can Models for Surrogate Endpoint Evaluation Be Used to Predict Generic Measures From Disease Specific Measures of Health Related Quality of Life Based on Summary Data?","id":"d860a05b-a89c-4ac2-a640-48d4fcf29022","sessionCode":"SA13","topDisplay":"Sadek A1, Cooper NJ2, Welton N1, Bujkiewicz S2 1University of Bristol, Bristol, UK, 2University of Leicester, Leicester, UK","locationCode":"7022","description":"\r\n\t<div>OBJECTIVES: Decisions on the cost-effectiveness of new treatments require estimates of treatment effects on generic Health related Quality of Life (HRQoL) outcomes. However, it is often the case that studies report disease specific scales, rather than the generic measure suitable for decision making, and this is especially the case in rare diseases. When there are multiple studies available, evidence synthesis of HRQoL outcomes can aid decision makers by fully reflecting the available evidence and reducing uncertainty. We aimed to explore the use of meta-analytic models for surrogate endpoint evaluation to obtain estimates of generic measures of HRQoL from disease specific measures. METHODS: We illustrate the methods using summary data from 25 RCTs reporting effectiveness of treatments in Ankylosing Spondylitis (AS) and non-radiographic axial Spondylarthritis (nr-axSpA). Target generic measures of HRQoL are mapped by predicting the missing treatment effects on the generic outcomes. We compare the following models: bivariate random meta-analysis (BRMA), Daniels-Hughes model (D&H), and BRMA in product normal formulation (BRMA PNF). The predicted and observed treatment effects on the generic outcomes are pooled and evaluated. RESULTS: We found associations between the generic 36-Item Short Form Survey Physical Component Summary (SF36-PCS) and the disease specific Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), the Mental Component Summary (SF36-MCS) and Functioning Index (BASFI). Cross validation confirmed the models’ prediction intervals to contain observed estimates for at least 90% of included studies. The treatment effect on BASDAI predicted the treatment effect on the SF36-PCS in all of the studies. BRMA PNF estimates were more precise than D&H estimates. Pooling estimates with BRMA PNF reduced uncertainty by 38% on SF36-PCS when compared to a univariate model. CONCLUSIONS: Methods for synthesis of surrogate endpoints predicted and increased precision in estimates of the generic HRQoL needed for HTA. BRMA PNF gave the most precise estimates.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/sporeurope23sadeksa13poster131783-pdf.pdf?sfvrsn=2e3dd77f_0","title":"SPOREurope23_Sadek_SA13_POSTER131783.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131783","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring the Effects of the COVID-19 Pandemic on Pneumococcal Disease in France: A Comprehensive Analysis of Public Health Impact and Economic Burden","id":"433cf255-bbb7-4978-a1b6-4a688a7879ba","sessionCode":"EPH16","topDisplay":"Carette J1, Massetti M2, Leleu H3 1Public Health Expertise, Paris, 75, France, 2Public Health Expertise, PARIS, 75, France, 3Public Health Expertise, Paris, France","locationCode":"3014","description":"\r\n\t<div>OBJECTIVES: Streptococcus pneumoniae represents a major public health issue in France, leading to many respiratory infections. The Covid-19 pandemic and measures to limit interindividual contacts have led to reduce viral infections (due to influenza and respiratory syncytial virus) and bacterial infections (due to meningococcus and pneumococcus) responsible for respiratory diseases. This study aims to analyse changes in the public health impact and economic burden of pneumococcal disease (PD) before and during the Covid-19 pandemic in France. METHODS: Based on the national epidemiological and demographic data and published data, the epidemiological burden associated with invasive pneumococcal disease (IPD) and non-invasive PD (including non bacteremic pneumococcal pneumonia (NBPP) and acute otitis media (AOM)) was estimated in children and adults, according to the serotype distribution. The economic impact associated with PD was valued using French national health insurance tariffs and published data. RESULTS: Before the pandemic, the burden of PD was estimated at 736 meningitis, 6,193 bacteraemia, 209,634 NBPP in the overall population, and 839,357 AOM in children in France. As the related management cost varies greatly depending on the nature and severity of the PD (from €11,216 per meningitis episode to €73 per AOM), the total economic burden of PD was estimated at over €1 million per year. However, the Covid-19 pandemic led to a drastic reduction in the incidence of IPD in France (by more than 40%), changed the bacterial ecology of PD and reduced its economic impact. CONCLUSIONS: We have developed a comprehensive analysis of the impact of the Covid-19 pandemic on the epidemiological and economic burden of PD in France. While the magnitude of this effect must be interpreted with caution as regards future changes in the global incidence of PD, its long-term impact on the PD’s serotype distribution must be carefully scrutinised.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133262","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Andexanet Alfa Versus Prothrombin Complex Concentrate Is Likely for the Treatment of Factor Xa Inhibitor–Related Major Bleeds in the Netherlands","id":"ebe02654-05a5-41a0-9650-4d0647cd194f","sessionCode":"EE4","topDisplay":"Lewis M1, Wensvoort N2, Heeks C1, Codling E1, Gray L1, Nekeman S2, Van Haalen H3 1FIECON Ltd, St Albans, UK, 2AstraZeneca, The Hague, Netherlands, 3AstraZeneca, Mölndal, O, Sweden","locationCode":"1058","description":"\r\n\t<div>OBJECTIVES: Factor Xa (FXa) inhibitors such as rivaroxaban and apixaban, commonly used for the prevention of thrombosis, are associated with an increased risk of major, potentially life-threatening bleeding. Andexanet alfa (AA) is indicated to reverse the anticoagulation effects of rivaroxaban or apixaban in such bleeding events. The EMA authorized AA for use throughout the EU in 2019, although cost-effectiveness data are scarce. The objective of this analysis was to assess the cost-effectiveness of AA, from a Dutch perspective. METHODS: A decision analytic model was used, comprising a decision tree in the short term and a Markov model in the long term. AA was compared to 4-factor prothrombin complex concentrate (4F-PCC) across rivaroxaban/apixaban users with life-threatening gastrointestinal, intracranial (ICH), or other major bleeds. The model was informed by the ANNEXA-4/ORANGE propensity score–matched comparison for 30-day mortality and by the ANNEXA-4/RETRACE indirect comparison for functional outcomes (for ICH). A lifetime horizon was applied, and a societal perspective was taken. Scenario analyses, including a healthcare perspective, were performed as well as one-way and probabilistic sensitivity analyses. Utility inputs and costs were sourced from literature and national list prices. Costs were inflated to 2023. RESULTS: Treatment with AA was associated with a QALY gain of 1.099 and incremental costs of €31,195, resulting in an ICER of €28,385/QALY. Under a willingness to pay threshold of €50,000/QALY, AA had an 87% probability of being cost-effective. Assuming a healthcare perspective resulted in an ICER of €29,929/QALY. Although results were most sensitive to long-term costs and utilities post-ICH, results were generally consistent across sensitivity and scenario analyses. CONCLUSIONS: While awaiting clinical trial (ANNEXA-I) results to confirm the incremental effectiveness of AA, this analysis indicates that AA is likely to be a cost-effective treatment option in patients with FXa inhibitor–related major bleeds in the Netherlands.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023-cost-effectiveness-posterfinal130452-pdf.pdf?sfvrsn=86025187_0","title":"ISPOR-EU 2023 Cost Effectiveness poster_FINAL130452.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130452","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Itch Intensity and Therapeutic Management of Pruritus in the Real-World Setting","id":"109132c1-5b7a-4d7e-85d0-4d13c6427c9c","sessionCode":"HSD18","topDisplay":"Rasouliyan L, Althoff A, Kumar V, Chang S, Long S, Mummert A OMNY Health, Atlanta, GA, USA","locationCode":"4028","description":"\r\n\t<div>OBJECTIVES: Pruritus is a cutaneous symptom but could be a manifestation of a wide range of systemic conditions making it difficult to manage. Itch intensity is a measure of symptom severity and can influence management. The objective of this research was to understand the influence of itch intensity on therapeutic management of pruritus in the real-world setting. <p class=\"paragraph\" style=\"margin: 0in; vertical-align: baseline; user-select: text; -webkit-user-drag: none; -webkit-tap-highlight-color: transparent; overflow-wrap: break-word; white-space: pre-wrap; font-kerning: none;\" paraid=\"1268927956\" paraeid=\"{3df29b07-a299-4cbf-b3f2-2c2bd89eba0f}{236}\">METHODS: Patients from 6 specialty dermatology networks within the OMNY Health Database with a 10-point itch intensity assessment associated with pruritus (ICD-10: L27*) from 2017-2023 were included. Percentages of patients with prescriptions for any of the following treatments were tabulated by itch intensity: topical treatments (corticosteroids [TCS], calcineurin inhibitors [TCI], capsaicin, menthol, <span style=\"user-select: text; -webkit-user-drag: none; -webkit-tap-highlight-color: transparent; background-image: var(--urlSpellingErrorV2, url(;\" img_16202958661688078460203_0=\"\" border-bottom:transparent=\"\" background-position-x:0=\"\" background-position-y:100=\"\">p<span style=\"user-select: text; -webkit-user-drag: none; -webkit-tap-highlight-color: transparent; background-image: var(--urlSpellingErrorV2, url(;\" img_16202958661688078460203_1=\"\" border-bottom:transparent=\"\" background-position-x:0=\"\" background-position-y:100=\"\">ramoxine/lidocaine/prilocaine, doxepin), systemic treatments (non-sedative antihistamines [NSAH], sedative antihistamines [SAH], opioid receptor antagonists [ORA], and selective serotonin reuptake inhibitors [SSRI], doxepin). <p class=\"paragraph\" style=\"margin: 0in; vertical-align: baseline; user-select: text; -webkit-user-drag: none; -webkit-tap-highlight-color: transparent; overflow-wrap: break-word; white-space: pre-wrap; font-kerning: none;\" paraid=\"253308581\" paraeid=\"{42f0a596-37fd-487a-b108-d4cd229aa691}{93}\">RESULTS: A total of 7,330 patients and 8,115 associated encounters were included. Distributions of gender (60% female), race (85% White, 8% Black, 7% Other), and age (57% > 60 years, 37% 21-60 years, 5% < 21 years) were tabulated. For 10-point itch intensity groups 0-1, 2-4, 5-7, and 8-10, prescriptions of topical treatments were 12%, 31%, 32%, and 25%, respectively, while prescriptions of systemic treatments were 9%, 22%, 33%, and 36%, respectively. Individual treatment classes followed similar patterns. TCS (45%) and SAH (18%) were the most prescribed topical and systemic treatments, respectively. Proportions of patients with prescriptions for topical menthol, NSAH, SAH, ORA, and systemic doxepin increased monotonically with itch intensity. Topical capsaicin, <span style=\"user-select: text; -webkit-user-drag: none; -webkit-tap-highlight-color: transparent; background-image: var(--urlSpellingErrorV2, url(;\" img_16202958661688078460203_2=\"\" border-bottom:transparent=\"\" background-position-x:0=\"\" background-position-y:100=\"\">pramoxine/lidocaine/prilocaine, and SSRI use were negligible over the study period. <p class=\"paragraph\" style=\"margin: 0in; vertical-align: baseline; user-select: text; -webkit-user-drag: none; -webkit-tap-highlight-color: transparent; overflow-wrap: break-word; white-space: pre-wrap; font-kerning: none;\" paraid=\"1285738505\" paraeid=\"{42f0a596-37fd-487a-b108-d4cd229aa691}{193}\">CONCLUSIONS: Results provide insights into real-world therapeutic management of pruritus as it relates to itch intensity. Symptom severity was strongly associated with greater prescriptions of most therapy classes. Future analyses would be helpful to understand the landscape of pruritus therapeutic management as it relates to itch intensity and symptom reduction. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023pruritus133850-pdf.pdf?sfvrsn=3f3a3d13_0","title":"ISPOR_EUROPE_2023_PRURITUS133850.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133850","diseases":[{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Novel Diabetes Subgroups Identified By Cluster Analyses: A Systematic Review and Meta-Analysis","id":"c69f354c-310c-4bac-9444-4dc8408ca85e","sessionCode":"EPH6","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3011","description":"\r\n\t<div>OBJECTIVES: Conventionally, diabetes is classified into Type-1 and-2 diabetes, but this classification offers limited insights into underlying mechanisms and heterogeneity within these types. Recently, a variety of novel diabetic patient clusters have been identified, using unsupervised machine-learning approaches. We aimed to identify all novel data-driven clusters of diabetic patients and to summarize patient characteristics and incidence of diabetic complications in each cluster. METHODS: A systematic review (PROSPERO CRD42023406046) of English-language journal articles was undertaken in PubMed, Embase, Scopus, and Web of Science from 1st January 2017 to 15th February 2023. References cited by included studies will be taken into account to identify publications before 2017 and potential missing literature. All studies were screened independently by two authors. Data extraction was conducted by one author and checked by another, using a pre-defined form. RESULTS: One hundred and sixteen studies were identified, among which fifty-two were related to of Ahlqvist (2018), including replication (36/52), validation (10/52) and both (6/52). The other 65 studies proposed new cluster definitions. Meta-analyses of patient characteristics (including age at diagnosis, BMI, HbA1c, HOMA2-B, HOMA2-IR) and incidence or relative risk of diabetic complications were performed for these 51 studies. The most common complications were nephropathy (15/52), retinopathy (15/52), neuropathy (12/52) and cardiovascular diseases (12/52). CONCLUSIONS: This review provided an overview of recent studies proposing novel diabetes subgroups based on clustering analysis. The cluster strategy proposed by Ahlqvist (2018) is the cornerstone of various follow-up studies. We found differences in data sources, patient population, algorithms and clustering variables, and outcomes of interests. The meta-analysis results provided summary estimates of centroids in each cluster, which enabled more reliable cluster classification in implementation. </div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130705","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Development of a Comprehensive Archive of Patient-Reported Outcome Measures (PROMS) for Clinical Research and Clinical Practice in Oncology","id":"7cfce105-39e0-483e-acad-4df4590d3363","sessionCode":"PCR17","topDisplay":"Malandrini FB1, Meregaglia M1, Pinto C2, Di Maio M3, Ciani O1 1SDA Bocconi School of Management, Milano, MI, Italy, 2Department of Medical Oncology, S. Maria Hospital - IRCCS, Reggio Emilia, Italy, Reggio Emilia, Italy, 3University of Turin, Torino, TO, Italy","locationCode":"6025","description":"\r\n\t<div>OBJECTIVES: Choosing the most adequate measure of patient-reported outcomes (PROs) in clinical trials, clinical practice, and post-authorization studies is not straightforward. This study aimed to develop a comprehensive archive of patient-reported outcome measures (PROMs) in oncology and identify their main characteristics and target outcome domains. METHODS: As part of the PRO4All project, we retrieved the available PROMs in oncology by searching facit.org, eortc.org, eprovide.mapi-trust.org, ema.europa.eu (European Public Assessment Reports), and published reviews. We developed a data extraction form to collect information on: PROM name, cancer area (based on ICD-10), type of questionnaire (i.e., self-reported, proxy-reported or caregiver’s report), questionnaire variant(s), recall period (e.g., last week) and number of items. Moreover, we assigned each item a specific domain according to a predefined 38-item taxonomy for outcome classification. RESULTS: A total of 308 PROMs were identified and fully analyzed. Over half (n=156, 50.6%) were cancer type-specific (e.g., breast cancer n=27, 8.8%), 132 (42.9%) were generic for cancer and 20 (6.5%) were intended for the general population but also recommended or used for cancer patients. 48 (15.6%) were variants of another questionnaire. The great majority of questionnaires (93.2%) were self-reported, 3.2% were proxy-reported (e.g., by parents), and 3.6% were related to caregiver’s status. In almost half of the cases (47.1%) the recall period was last week. The mean number of items per questionnaire was 22.5 (range: 1- 130). In total, 6921 items were assigned an outcome domain, which was emotional functioning/wellbeing in 21.5% of cases, physical functioning in 15.0%, general outcomes in 9.9% and delivery of care in 9.6%. CONCLUSIONS: This review study highlighted a significant heterogeneity of PROMs in oncology. The newly developed archive represents a useful tool for guiding researchers and practitioners in selecting the most suitable measures for cancer patients and fostering a patient-centered approach. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/malandrini-et-al-ispor-2023131837-pdf.pdf?sfvrsn=af79c4f7_0","title":"Malandrini et al., ISPOR 2023131837.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131837","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"What Is the Economic Impact of an Early Adoption of High Efficacy Treatments in Multiple Sclerosis? A Cost-Consequence Analysis From a Societal Perspective in the Portuguese Setting","id":"df0aaa59-1907-4e5d-a4a7-4e1f13c1da81","sessionCode":"EE50","topDisplay":"Gonçalves N, Esparteiro J, Barros M Novartis Portugal, Porto Salvo, Lisbon, Portugal","locationCode":"1090","description":"\r\n\t<div>OBJECTIVES: Multiple Sclerosis (MS) treatment paradigm has been challenged with the approval of high efficacy treatments (HET). There is increasing evidence that starting with HET early in the disease course can result in better long-term outcomes compared to escalation approach. Our objective is to estimate the economic impact of an early use of Ofatumumab (HET), compared to long term usage of moderate disease modifying therapies (DMT), in adult MS patients with active disease. METHODS: A cost-consequence analysis was performed using Markov state transition model, that simulated disease progression measured by Expanded Disability Status Scale (EDSS). Patients were assumed to remain on treatment until progression to EDSS 7. Efficacy, safety and health-state utility values were estimated from the ASCLEPIOS I/ II trials and network meta-analysis. Natural history was based on British Columbia MS registry and London Ontario database. Resource consumption was based on published literature, national microdata and expert opinion; unit costs were from official sources. Four scenarios with a time horizon of 10 years were simulated: two base scenarios evaluated Ofatumumab versus Dimethyl Fumarate (DMF) or Teriflunomide (standard DMT) without any treatment switches, and late switch scenario after 3 years of DMF or Teriflunomide treatment. RESULTS: In 10 years when Ofatumumab is initiated as first treatment option, 15.3% patients progressed to EDSS 7 or higher compared with 20.7% patients on DMF and 23.1% on Teriflunomide. Treatment switch after 3 years to Ofatumumab increases this rate to 18.1% in the scenario with DMF and 19.4% with Teriflunomide. Disease management cost for Ofatumumab when early started is 27.910€, for DMF 32.195€ and Teriflunomide 34.210€, representing a cost reduction in 10 years of 13% and 18%, respectively. CONCLUSIONS: At the end of 10 years, the early use of Ofatumumab demonstrated better clinical outcomes versus a delayed approach, accompanied by cost savings, in the Portuguese setting.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133411","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Comparative Study of Health-Related Quality-of-Life Outcomes in Patients Undergoing Total Hip Arthroplasty in the Hungarian Public and Private Healthcare","id":"6db94855-1a26-46bf-95af-4e32614749ba","sessionCode":"PCR10","topDisplay":"Kajos L1, Molics B1, Elmer D2, Csákvári T3, Pónusz-Kovács D1, Kovács B3, Boncz I3 1University of Pécs, Pécs, BA, Hungary, 2University of Pécs, Pécs, PE, Hungary, 3University of Pécs, Pécs, Hungary","locationCode":"6015","description":"\r\n\t<div>OBJECTIVES: The study aimed to compare the health-related quality-of-life outcomes of total hip arthroplasty patients in public and private healthcare in Hungary. METHODS: Patients were selected at the Department of Orthopedics, Clinical Centre of the University of Pécs and at the Da Vinci Private Clinic in Pécs (Hungary). In the selected public hospital, surgery is mainly performed with a traditional anterolateral approach, while in the private hospital it is performed with a minimally invasive technique and anterior approach. Patients completed the SF-36 and Oxford Hip Score (OHS) questionnaires before the surgery and 3 months later. RESULTS: The research involved 232 patients, 123 patients in public healthcare (45 male, 78 female, mean age: 67.24), 109 patients in private healthcare (52 male, 57 female, mean age: 63.77). The SF-36 Physical Health score (PCS) increased from 27.70 to 57.16 points for public hospitals patients and from 35.42 to 74.98 points for patients in the private hospital (p<0.001) by the 3rd month after surgery. The SF-36 Mental Health score (MCS) increased from 69.31 to 77.54 for public healthcare patients (p=0.001) and from 67.50 to 84.56 for private patients (p<0.001). The Oxford Hip Score showed an increase from 16.49 to 34.57 points for public patients and from 21.76 to 39.94 points for private patients (p<0.001). There was no difference in the improvement in OHS scores between the two healthcare sectors (p=0.949), but private patients showed greater improvement in SF-36 scores (PCS: p=0.001, MCS: p=0.007). CONCLUSIONS: When comparing quality-of-life scores, both healthcare sectors showed significant improvement at the 3rd month after total hip arthroplasty, but regarding the SF-36 questionnaire, the improvement was greater for private patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kajospcr10poster129862-pdf.pdf?sfvrsn=7286b549_0","title":"ISPOREurope23_Kajos_PCR10_POSTER129862.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129862","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Atezolizumab Plus Chemotherapy in Extensive-Stage Small-Cell Lung Cancer","id":"245b69b6-1658-4fad-a23f-4e9a4278ebcc","sessionCode":"CO19","topDisplay":"Domínguez L1, Carrión Madroñal I1, Marcos Rodríguez JA1, Valera-Rubio M2 1Hospital Universitario Virgen Macarena, Seville, Seville, Spain, 2Hospital Universitario Virgen Macarena, Sevilla, Spain","locationCode":"1022","description":"\r\n\t<div>OBJECTIVES: Atezolizumab in combination with carboplatin-etoposide is indicated for the first-line treatment of extensive-stage small-cell lung cancer(ES-SCLC). The aim of our study is to analyze the effectiveness and safety of atezolizumab combined with chemotherapy in ES-SCLC in a tertiary hospital, and to compare with the pivotal IMpower133 trial. METHODS: Retrospective observational study was conducted. We included patients treated with atezolizumab plus chemotherapy from January/2022-March/2023. Variables collected: sex, age, smoking-status, quality of life status according to the Eastern-Cooperative-Oncology-Group(ECOG) scale, location of metastases, comorbidities, duration of treatment, objective response rate(ORR) according to RECIST-v1.1 criteria(Response Evaluation Criteria in Solid Tumors), progression-free survival(PFS) and overall survival(OS) calculated by Kaplan-Meier method, and adverse events(AEs) according to Common Terminology Criteria for Adverse Events-v.5(CTCAE). Data obtained from the electronic medical record (Diraya®) and software Farmis_Oncofarm®, and processed with SPSS-Statistics-v.21. RESULTS: Fourteen patients(100% male) were included; median age of 62 years [Interquartile range (IQR):57-64]; 100% smokers. 79% had an ECOG:1 at baseline(14% ECOG:2;7% ECOG:0). The most frequent locations of metastases were: hepatic(57%), bone(50%) and renal(50%). The most frequent comorbidities were: arterial hypertension(43%), dyslipidemia(21%) and diabetes mellitus II(14%). Median duration of treatment was 5.2 months(IQR:4-7). At the date of analysis(13/04/2023), 7 patients are still on treatment. ORR was partial in 64% of patients(36% not evaluated). Median PFS and OS were 4.9(95%CI 2.1-7.8) and 5.8(95%CI 5.3-6.4) months, respectively. 93% of patients had some AE during treatment. Most frequent AEs were: anemia 77%(G3:38.5%), neutropenia 77%(G3-4:38.5-15.4%), asthenia 53.8%(G3:7.7%), thrombocytopenia 53.8%(G3:15.4%), elevated transaminases 30.8%(G3:0%), nausea 23%(G3:0%), and diarrhea 15.4%(G3:0%). No patient discontinued treatment due to AEs. CONCLUSIONS: Median OS and PFS were lower than that obtained in the pivotal IMpower133 trial. Although the majority of patients presented some AE, in no case were these AEs forced to discontinue treatment. Further studies with a larger sample size and longer follow-up period are needed to confirm these real-life results.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/co19atezo-ispor2023130626-pdf.pdf?sfvrsn=1866efb4_0","title":"CO19_Atezo-Ispor2023130626.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130626","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"How to Assess Nationwide Population-Based Clinical Benefit of Treatments Using Public Information? Immunotherapies Case Study","id":"968e24f4-8cd3-4b8d-83eb-4eaf94540b44","sessionCode":"CO17","topDisplay":"Grumberg V1, Cotte FE2, Gaudin AF2, Borget I3 1Paris Saclay University and Bristol Myers Squibb, PARIS 18, 75, France, 2Bristol Myers Squibb, Rueil-Malmaison, France, 3Institut Gustave Roussy and Paris Saclay University, Villejuif, France","locationCode":"1014","description":"\r\n\t<div>OBJECTIVES: New treatments’ benefit is evaluated, when they are marketed, by health authorities based on clinical trials results at individual patient level. However, treatments outcomes are rarely re-assessed in real-world practice and/or at the population level. The objectives are to present a methodology to assess population-based clinical benefit of treatments. METHODS: In France, the Haute Autorité de Santé (HAS) publishes medico-technical and cost-effectiveness (CE) assessments of all new indication of drugs. In CE assessments, extrapolated Kaplan-Meier survival curves over a lifetime horizon and utility values are available. The Hospital Reimbursement database (PMSI) allows to identify the number of patients initiating a given drug and to follow their use over time. The date of availability of each drug by indication is published in the Official gazette, as well as if it has benefited of early access. Once consolidated, this public dataset gives an opportunity to generate retrospective nationwide population-based estimation of drugs outcomes, expressed as deaths prevented (DP), life years (LY) and quality-adjusted life years (QALYs), by monthly incident cohorts. For this case study, savings obtained with immunotherapies versus their comparators were calculated from their introduction (2014) until Dec-2021. RESULTS: Between 2014 and 2021, 5 immunotherapies (avelumab, atezolizumab, durvalumab, nivolumab and pembrolizumab) were used in France in 21 indications in 8 tumor localizations. Based on the PMSI reports, we identified 132,924 patients who initiated an immunotherapy during the period. Using the extrapolated curves, we estimated 16,173 [13,803–17,141] deaths were prevented compared to previous standard of care by December 31st of 2021. Compared to standard treatments, immunotherapies allowed 37,318 [33,583–41,053] LYG and 27,710 [23,785–30,451] QALYs. We were able to calculate the results per drug, tumor localization and line. CONCLUSIONS: This methodology is an opportunity to estimate the clinical benefit of a drug (or a therapeutic class) at a nationwide population level.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-esmoclinical-benefit-icivf129378-pdf.pdf?sfvrsn=deb65c27_0","title":"Poster ESMO_clinical benefit ICI_VF129378.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129378","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization and Estimated Costs in Adult Pneumococcal Disease in Peru: Results from a Panel of Experts","id":"ddef585e-1995-4084-88c8-4eef67bc5f35","sessionCode":"EE137","topDisplay":"Figueroa J1, Valenzuela G1, Hirata Iha L2, Mezones-Holguin E3, Webster J4, Pungartnik P5, Zampirolli C5, Igansi CN5, Kano B6, Parellada C2 1MSD Peru, Market Access, Lima, Peru, 2MSD Brazil, Center for Observational and Real-world Evidence (CORE) Latin America, São Paulo, SP, Brazil, 3Universidad San Ignacio de Loyola, Centro de Excelencia en Investigaciones Económicas y Sociales en Salud, Lima, LIM, Peru, 4Merck & Co., Inc., Philadelphia, PA, USA, 5IQVIA, São Paulo, São Paulo, Brazil, 6IQVIA, São Paulo, SP, Brazil","locationCode":"2071","description":"\r\n\t<div>OBJECTIVES: Health interventions to reduce the clinical burden of pneumococcal disease (PD) could lead to substantial savings for the healthcare system. This study aimed to estimate the healthcare resource utilization (HCRU) and direct medical costs of PD in adults in Peru during 2022. METHODS: A panel of experts was developed with five local infectious disease or critical care specialists from Ministry of Health (MoH) institutions with experience in treating PD-related syndromes (pneumococcal pneumonia [PP], bacteremia, and meningitis) in adults. The HCRU by category (lab/imaging tests, professional services, procedures, medications, and length of hospital stay [LoS]) and frequency were collected using a discussion guide through online interviews. To estimate costs, a micro-costing approach from the payer perspective was applied for each PD (uncomplicated and complicated) considering the reported average use of each resource and its respective unit cost in the MoH national tariff. Costs were converted to United States dollars (exchange rate $1USD=3.65 soles in June 2023). RESULTS: The average LoS for PD ranged from 14.5 days for uncomplicated pneumonia to 55.5 days for complicated meningitis. For pneumonia, patients stayed an average of 4.6 days in the emergency room due to a shortage of hospital beds. The average cost per episode for outpatient pneumococcal pneumonia was $194, and for inpatient PP with and without complications was $2,038 and $10,450, respectively. For invasive pneumococcal disease, the most expensive average costs were bacteremia ($7,329), followed by complicated meningitis ($7,705), and uncomplicated meningitis ($5,778). The average cost for pneumococcal meningitis sequelae was $3,132. Among PD, the highest costs were driven by LoS, followed by lab/imaging tests, professional services, and medications. CONCLUSIONS: PD in adults was associated with a high utilization of resources, hospital occupancy rates, and direct medical costs. HCRU and cost data reflecting local practices are critical to conducting economic evaluations that may inform pneumococcal vaccination policies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23parelladaee137posterv2131084-pdf.pdf?sfvrsn=2ee401c5_0","title":"ISPOREurope23_Parellada_EE137_POSTERv2131084.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131084","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Early Access to Innovative Medicines: Decision Making and State-Change Analysis","id":"2574eb65-9d33-4d70-94bf-507581bc0ad5","sessionCode":"HPR27","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Requesting access to new hospital medicines through Early Access Programs (EAP) before mandatory health technology assessment is concluded by the Ministry of Health (MoH) has become increasingly popular in Portugal. We aimed to assess the likelihood of rejection/deferral decisions and state-changes over time, of new EAP requests in the period between November 2019 and February 2023. METHODS: An EAP state is a unique combination of the type of decision (rejected/deferred), exact therapeutic indication, number of patients allowed to enter, and cost implications due to the possibility of an initial free-of-charge period (EAP active with or without costs to the MoH). The odds of EAP rejection were analyzed through logistic regression, whereas the analysis of state-changes was based on descriptive statistics. RESULTS: Data was identified on 296 states of 189 unique new EAP, covering 133 distinct medicines. The majority of medicines (n=105, 78.9%) had only one EAP, while the remainder presented two (n=17; 12.8%) and three or more (n=11, 8.3%) EAP. For the 189 EAP overall, deferral rates were high (n=133, 70.4%). The likelihood of EAP deferral has been increasing over time: for every additional year during the observation period, the odds of EAP deferral were estimated to be 1.96 times higher than the year before (95% CI: [1.38;2.86]). State-changes were observed in over half of the 133 deferred EAP (n=68, 51.1%), the majority of which included at least a change from an “active without costs” to an “active with costs” state (n=60, 45.1%). CONCLUSIONS: The estimated increase in number of EAP deferred over time suggest that early access to innovative medicines in the Portuguese pharmaceutical market may be improving since 2019. Addressing equity concerns and anticipating the long-term policy implications stemming from the restrictive nature of EAP might become essential when meeting stakeholder expectations of broader access to innovation.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133841","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Uncertain About Uncertainty in Matching-Adjusted Indirect Comparisons (MAIC)? A Simulation Study to Compare Methods for Variance Estimation","id":"72abc2a1-76ae-42ed-8c3a-507a2efb754d","sessionCode":"MSR25","topDisplay":"Chandler C1, Proskorovsky I2 1Evidera, Waltham, MA, USA, 2Evidera, St-Laurent, QC, Canada","locationCode":"5065","description":"\r\n\t<div>OBJECTIVES: Matching-adjusted indirect comparison (MAIC) is the most common methodology considered in technology appraisals for pairwise comparisons that control for imbalances in baseline characteristics. The aim of this simulation study was to assess the performance of different methods for estimating the uncertainty around treatment effects derived via anchored MAIC. METHODS: Monte Carlo simulations (n=1,000 replications) were conducted for a total of 18 scenarios to investigate the impact of outcome type (binary, survival), sample size, and population overlap on variance estimation in MAICs. In each scenario, weighted logistic regression and Cox proportional hazards models were fitted for binary and survival outcomes using four different methods for variance estimation: 1) conventional estimators (CE) using raw weights; 2) CE using weights rescaled to the effective sample size (ESS); 3) robust sandwich estimators; and 4) bootstrapping. The performance of each method was evaluated on the basis of empirical coverage of 95% confidence intervals (CI) and the ratio of average estimated standard error (SE) versus empirical SE. RESULTS: The empirical coverage for CE + ESS-scaled weights (ranging from 94.5% to 96.4%) did not significantly deviate from the nominal confidence level in 17 of 18 scenarios. On the contrary, variance was underestimated by CE + raw weights (6 of 6 scenarios), bootstrapping (4/6), and sandwich estimators (3/6) in the scenarios with poor population overlap (~77% reduction in the ESS). The use of CE + raw weights underestimated variance in half of the scenarios with moderate overlap, while all other methods had unbiased results. All four methods provided accurate estimates of the variance in scenarios with strong overlap, with coverage and SE ratios between 94.2%-96.2% and 0.97-1.06. CONCLUSIONS: The extent of population overlap is an important consideration for variance estimation in MAICs. The use of CE + ESS-scaled weights produced SEs and CIs that were fairly precise across all scenarios.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/chandler-et-alispor-eu-2023final-posterv0-425oct2023128251-pdf.pdf?sfvrsn=1b075db1_0","title":"Chandler et al_ISPOR EU 2023_Final Poster_v0.4_25Oct2023128251.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128251","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Phacoemulsification Equipment in Surgical Throughput, Under the Perspective of a Greek Hospital","id":"85bb04af-012e-483a-81e5-50ede1328957","sessionCode":"EE130","topDisplay":"Papadopoulos M1, Hsiao CC2, Busutil R3 1Alcon Laboratories Greece, Marousi, A1, Greece, 2Alcon Vision LLC, Fort Worth, TX, USA, 3Alcon Healthcare, S.A., Sevilla, SE, Spain","locationCode":"2060","description":"\r\n\t<div>OBJECTIVES: Increased cost of healthcare is a common challenge which most countries face. The Greek government strives to ensure universal coverage and equitable access, focusing on cost containment policies and increased efficiencies. Cataract surgery is one of the most frequent surgical procedures at country level and phacoemulsification is the preferred technique. The objective of this analysis is to estimate the economic impact and efficiency of different phacoemulsification equipment, under the perspective of a Greek hospital. METHODS: A decision-analytic model was developed to analyze the aggregated impact on cataract surgery throughput of different phacoemulsification equipment features. Scenario 1 vs scenario 2 considered phacoemulsification equipment with the following variables: torsional vs longitudinal ultrasound movement (TvL), Active SentryTM vs. other handpiece (ASvO), active fluidics vs gravity-based infusion system (AFvGB) and Intrepid vs. Kelman phaco tip (IvK). Inputs were derived from the literature (one study per variable) and expert opinion. The model and underlying assumptions were validated by clinical experts. OR time was chosen as the key variable of efficiency. The model assumed a hospital performing 2,000 cataract procedures per-annum (grade 3 and 4 cataracts), with 100% adoption and equal acquisition costs for each one of the two scenarios. RESULTS: Savings in OR time (seconds) were estimated as 62 for TvL, 12.10 for ASvO, 41.71 for AFvGB, and 17.64 for IvK, accounting for 11% efficiency increase for the cataract procedure time between the two scenarios of the analysis. This could translate into an annual difference of 63,611€. CONCLUSIONS: This analysis highlights the relevance of phacoemulsification features such as ultrasound movement, handpiece, infusion system and phaco-tip, with the aim to achieve an efficient throughput for cataract surgery, with subsequent cost-savings for the hospital. Further research would be needed, to better estimate the contribution of phacoemulsification equipment for efficient cataract surgeries. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee130-impact-of-phacoemulsification-equipment-in-surgical-throughput-under-the-perspective-of-a-greek-hospital130966-pdf.pdf?sfvrsn=3179c342_0","title":"EE130 Impact of Phacoemulsification Equipment in Surgical Throughput, Under the Perspective of a Greek Hospital130966.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130966","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Treatment and Associated Outcomes of Type-2 Diabetes Mellitus Patients With a Cardiovascular Comorbidity and Comparison With Guideline Recommendations: A German Claims Data Analysis","id":"51bcb031-5c34-47a3-923d-512747b93778","sessionCode":"HSD3","topDisplay":"Gabler M1, Duerschmied D2, Grond M3, Lehrke M4, Martin S5, Tröbs SO6, Schultze M7, Kossack N8, Richter L9, Aberle J10 1Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim, Germany, 2Universitätsmedizin Mannheim, Mannheim, Germany, 3Kreisklinikum Siegen GmbH, Siegen, Germany, 4Universitätsklinikum Aachen (RWTH), Aachen, ., Germany, 5Westdeutsches Diabetes- und Gesundheitszentrum (WDGZ), Düsseldorf, ., Germany, 6Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim am Rhein, Germany, 7ZEG – Berlin Center for Epidemiology and Health Research GmbH, Berlin, Germany, 8WIG2 GmbH, Leipzig, SN, Germany, 9WIG2 GmbH (Wissenschaftliches Institut für Gesundheitsökonomie und Gesundheitssystemforschung) - Scientific Institute for Health Economics and Health System Research, Leipzig, Sachsen, Germany, 10Universitätsklinikum Hamburg-Eppendorf (UKE), Hamburg, Germany","locationCode":"4012","description":"\r\n\t<div>OBJECTIVES: Type-2 diabetes mellitus (T2DM) is often accompanied by cardiovascular (CV) comorbidities. With this common disease pattern, treatment options and guidelines are well established. Rather little is known about the real-world implementation of these guidelines in daily practice. The observational study presented here aims to characterize the CV and anti-diabetic (AD) treatment of T2DM patients with an incident CV disease throughout Germany, and analyze adherence to respective clinical guidelines with respect to specific outcomes such as mortality and (re-)hospitalization rates. METHODS: We used 2019 German claims data and selected (prevalent) T2DM patients with the following (incident) CV comorbidities: Ischemic stroke (IS), myocardial infarction (MI), heart failure (HF), coronary artery disease (CAD), and (combined) HF/CAD. Guideline adherence was defined as \"completely adherent\", \"partly adherent\" or \"non-adherent/untreated\". Propensity score matching was performed to adjust for confounding. RESULTS: CV-guideline adherence is increasing over time, with the share of completely adherent patients ranging from 29.6% (CAD/HF) to 57.5% (MI). The share of non-adherent patients is rather low, ranging from 1.8% (MI) to 14.2% (HF). One-year overall mortality was 9.2% in the patient group completely adherent to CV guidelines and 17.4% in the non-adherent patient group (p<0.0001). Similarly, one-year overall mortality was 2.6% in the patient group completely adherent to AD guidelines and 5.2% in the non-adherent patient group (p=0.0051). However, the rates for all-cause hospitalization were significantly higher in the patient group completely adherent to CV guidelines vs. non-adherent patients (66.0% vs. 62.7%, p=0.0024), most likely due to more intensive monitoring by physicians. CONCLUSIONS: Our findings reveal potentially preventable deaths, and potential for optimization, both from a patient’s perspective (i.e., fewer hospitalizations) and an economical perspective. Adherence to guidelines must be improved, which requires joint efforts: improving the dissemination and communication of guidelines on the one hand and having continuous medical education for practitioners on the other hand.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gablerhsd3poster133118-pdf.pdf?sfvrsn=39d9852e_0","title":"ISPOREurope23_Gabler_HSD3_POSTER133118.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133118","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Psoriasis Symptoms and Impacts Measure: Identifying Key Items Reflecting Core Symptoms of Patients with Plaque Psoriasis","id":"1a2c6839-9b37-45cd-beb8-5177883f2f35","sessionCode":"MSR85","topDisplay":"Warren RB1, Augustin M2, Warham R3, Lambert J4, Hoepken B5, Gottlieb AB6 1Dermatology Centre, Northern Care Alliance NHS Foundation Trust, Manchester, UK; NIHR Manchester Biomedical Research Centre, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK, 2Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf (UKE), Hamburg, Germany, 3Veramed, London, UK; UCB Pharma, Slough, UK, 4UCB Pharma, Colombes, France, 5UCB Pharma, Monheim, Germany, 6Department of Dermatology, The Icahn School of Medicine at Mount Sinai, New York, NY, USA","locationCode":"5046","description":"\r\n\t<div>OBJECTIVES: The 14-item Psoriasis Symptoms and Impacts Measure (P-SIM) is a novel patient-reported outcome tool which captures key symptoms and life impacts of psoriasis. This analysis aimed to identify a subset of P-SIM items representative of core symptoms experienced by patients with psoriasis, to allow frequent assessments to be quicker and less burdensome. METHODS: Findings from previous literature reviews and qualitative patient interviews were used to identify key items of interest to patients. Using observed case data pooled across all treatment arms at baseline and Week 16 of the BE VIVID (NCT03370133), BE SURE (NCT03412747) and BE READY (NCT03410992) phase 3 trials, inter-item correlations, principal component analysis (PCA) and exploratory factor analysis identified items with considerable overlap. Removed items were regressed on items retained, using the R-squared statistic to determine how much variability could be explained by retained items. RESULTS: Previous findings identified itching, skin pain, scaling, redness and burning as key items of interest for priority retention. Based on PCA, the 14 items were grouped: 11 symptom items; two impact items (embarrassment, choice of clothing); one fatigue item. Following overlap analysis between the 11 symptom items, cracking and dryness were retained; thickening (considerable overlap with scaling), irritation and sensitivity (both considerable overlap with burning and skin pain) and lesions (considerable overlap with redness) were removed. At baseline (N=1,227), regression analysis indicated the seven retained symptom items explained 84.5%, 87.7%, 86.9% and 86.7% of the variability of the removed thickening, irritation, sensitivity and lesions items, respectively. At Week 16 (N=1,126), these percentages increased to 94.2%, 94.3%, 92.2% and 92.6%. CONCLUSIONS: Seven P-SIM items representative of core symptoms experienced by patients with psoriasis were identified: itching, skin pain, scaling, redness, burning, cracking and dryness. This could inform development of a symptom short-form, allowing quick, valid and reliable assessment with minimized patient burden and inter-measure redundancy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23lambertmsr85poster128656-pdf.pdf?sfvrsn=8c769c28_0","title":"ISPOREurope23_Lambert_MSR85_POSTER128656.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128656","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Application of the Atlas Algorithm to Identify the Carboplatin+Gemcitabine Regimen in Patients with Metastatic Triple-Negative Breast Cancer (MTNBC) through the French Nationwide Hospital Discharge Database (PMSI)","id":"8c6c2d00-0845-49c7-9eb1-51f1e446b434","sessionCode":"MSR6","topDisplay":"Grenier B1, Lemeille P1, Lafon T1, Bensimon L2, Schmidt A1 1HEVA, Lyon, 69, France, 2MSD France, Puteaux, France","locationCode":"5057","description":"\r\n\t<div>OBJECTIVES: Metastatic triple-negative breast cancer (TNBC) corresponds to between 2,717 and 3,093 diagnoses in France every year according to the French National Authority for Health (HAS). The approval of Keytruda in combination with chemotherapy in patients with mTNBC raised the question of the applicability of its combination with carboplatin+gemcitabine (CG), used in the KEYNOTE-355 trial, to French clinical practice. The aim of this study was to quantify the use of the CG regimen in mTNBC patients before the approval of Keytruda in association with this protocol. METHODS: All newly hospitalized (without hospitalization in the 5 preceding years) patients with metastatic (C77, C78, C79) breast cancer (C50) between 01/01/2017 and 31/12/2020 were followed until the end of the study period (31/12/2020) or in-hospital death. To exclude non-TNBC patients, patients without any chemotherapy session (Z51.1) and/or with administration of an anti-HER2+ treatment or a treatment/procedure suggesting a RH+ status were excluded. In the remaining population, specific mTNBC management with CG in intra-DRG was sought using an algorithm (ATLAS) that compares the frequency of chemotherapy sessions with a theoretical regimens of 3 cycles (deemed sufficient to discriminate this protocol) of 21 days with a D0-D7-D21 injections regimen. RESULTS: Overall, between 2017 and 2020, ATLAS identified a CG regimen for more than 21 days in 1,358 patients and for more than 42 days in 1,032. Among all CG regimen identified, 14-16% were identified in 2017, 23% in 2018, 32-34% in 2019 and 29-30 % in 2020. CONCLUSIONS: This algorithm approximated the number of mTNBC patients treated with the CG protocol in France. The number of patients benefiting from this protocol and the increase in its use over time are consistent with the data reported by HAS in the efficiency opinion of Keytruda and the recent integration of this therapeutic option into management in France, respectively.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-msd-mtnbc-ispor-2023-v1r2-web130471-pdf.pdf?sfvrsn=e8fa10ff_0","title":"POSTER-MSD-mTNBC-ISPOR-2023-V1R2-WEB130471.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130471","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Icosapent Ethyl for Patients at High Cardiovascular Risk with Elevated Triglycerides in Greece","id":"eafbaa0e-74ab-47de-a4c5-523181f9645d","sessionCode":"EE9","topDisplay":"Stratopoulos A1, Kougioumtzoglou I2, Mortaki K3, Rigopoulos P3, Jakouloff D4 1Vianex S.A., Athens, A1, Greece, 2University of West Attica, Athens, Greece, 3Vianex S.A., Athens, Greece, 4Amarin Switzerland GmbH, Zug, Switzerland","locationCode":"1057","description":"\r\n\t<div>OBJECTIVES: Cardiovascular Disease (CVD) is responsible for more than 40% of total deaths in Greece. Despite the optimization of lipid-lowering treatments targeting LDL-C, patients keep experiencing CVD events, despite attaining LDL-C target, with elevated triglycerides (TG) being an independent marker for CVD risk. Icosapent ethyl (IPE) reduces the incidence of CVD events in adult statin-treated high-risk patients with elevated TG. This analysis targets to assess IPE`s economic value for the Greek Healthcare System. METHODS: A cost-utility model was designed in Microsoft Excel as a partitioned survival model with daily cycles, using individual patient data from REDUCE-IT trial (4.9 years follow-up). The model is already accepted by several regulatory organizations worldwide and was adapted for Greece. The population of the analysis was the ITT population of the trial. Costs and QALYs were calculated over a lifetime horizon, from a societal perspective. IPE + statin was compared to statin alone (Standard of Care, SoC), as per REDUCE-IT trial. Costs and utilities were linked to each event health state. Data inputs included drug acquisition, disease management, adverse event costs-disutilities, travel and caregiver costs and productivity losses. Costs and benefits were discounted by 3.5% and sensitivity analyses (deterministic and probabilistic) were performed to evaluate uncertainty. RESULTS: Τotal IPE therapy cost was 46,334€, compared to 34,990€ with SoC, whereas QALYs were 9.65 and 9.13 respectively. The ICER for the ITT population was 21,885€ and 16,226€ per QALY gained per QALY gained, in ITT and eCVD population respectively. In the cost-effectiveness acceptability curve, IPE had high probability of being cost-effective for the ITT and eCVD population. The robustness of the results was confirmed by various sensitivity analyses. CONCLUSIONS: Management of high-risk for CVD individuals with hypertriglyceridemia, with IPE + statin, is a cost-effective option for the Greek Healthcare System and the treatment`s value is even higher in patients with eCVD.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23stratopoulosee9poster130492-pdf.pdf?sfvrsn=91f00b96_0","title":"ISPOREurope23_Stratopoulos_EE9_Poster130492.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130492","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Perceived Financial Burden and Quality of Life Among Patients with Cancer","id":"f66451b7-a7ba-40bd-919a-529ed29178d4","sessionCode":"PT12","topDisplay":"Ruotsalainen J1, Purmonen T1, Korhonen MJ1, Aarnio EJ2 1Oriola, Espoo, Finland, 2University of Eastern Finland, Kuopio, Finland","locationCode":"3B","description":"\r\n\t<div>OBJECTIVES: Financial burden perceived by patients with cancer is often underappreciated in countries with universal coverage. This study aimed to characterize the association between perceived financial burden and health-related quality of life (HRQoL) among patients with cancer in Finland. METHODS: This was a cross-sectional survey of 504 patients with cancer enrolled trough community pharmacies and patient organizations between April 2019 and September 2020. Perceived financial burden was assessed with a question “Has cancer negatively impacted your financial situation?” (answer choices: yes, considerably; yes, to some extent; no; cannot say). HRQoL was measured with the generic 15D HRQoL instrument including the following dimensions: mobility, sight, breathing, sleeping, eating, speaking, secretion, daily activities, mentality, aliments and symptoms, depression, distress, vitality, and sexual activity. The association between perceived financial burden and 15D score was analyzed with linear regression adjusted for gender, time since diagnosis, cancer type and progression, need for assistance, net income, and private cancer insurance. RESULTS: Of all respondents, 79% were women; 58% had been diagnosed with cancer within the past two years and 8.3% over 10 years ago. Almost every second (47%) respondent perceived that cancer had negatively impacted their financial situation at least to some extent. The mean 15D score was 0.76 (standard deviation [SD]=0.11) in patients perceiving considerable burden (n=135), 0.82 (SD=0.10) in those with some burden (n=220) and 0.89 (SD=0.07) with no burden (n=132). Furthermore, perceived financial burden was inversely associated with the mean 15D score in multivariable regression model (beta coefficient for considerable vs. no burden: -0.09, p<0.0001; some vs. no burden: -0.05, p<0.0001) and with each dimension of 15D. CONCLUSIONS: Financial burden is a concern for a significant proportion of patients with cancer. Most importantly, the perceived financial burden seems to be associated with reduced HRQoL independent of the patient’s net income.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/perceived-financial-burden-and-quality-of-life-among-patients-with-cancerfinal129763-pdf.pdf?sfvrsn=88ab80ec_0","title":"Perceived financial burden and quality of life among patients with cancer_final129763.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129763","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Body Fatness Associations with Cancer: Retrospective Cohort Study from a Brazilian Health Management Organization (RWE)","id":"cdc683e2-65fc-4cfa-884a-52c26b117937","sessionCode":"RWD5","topDisplay":"Busch J, Dos Reis Neto JP CAPESESP, Rio de Janeiro, RJ, Brazil","locationCode":"6057","description":"\r\n\t<div>OBJECTIVES: Overweight and obesity are growing global health challenges. Body mass index (BMI) is the most frequently applied population-level measure for overall body fatness. Raised BMI is an important risk factor for cancer, which is another leading and rising health burden worldwide. About 4–8% of all cancers are attributed to obesity. This study analyzes the risk of development of cancer in overweight/obese individuals. METHODS: Design: Retrospective cohort study from 2005 to 2021. Population: Beneficiaries who completed an epidemiological survey with body mass index information at the time of joining the health plan. Source data: Electronic health records from the administrative database. Individuals were classified into two groups: non-overweight/obese (BMI≤24.9kg/m2) or overweight/obesity (BMI≥25kg/m2). Logistic regression models were used to examine the associations of BMI with risk of digestive system cancers, including esophageal, stomach, colorectal, liver, gallbladder, and pancreatic cancer, as well as kidney, thyroid, breast, endometrial, and ovarian, adjusting for age and sex. For significance, Chi-square tests (Fisher's Exact), when p<0.05. Confidence intervals (CI) 95%. RESULTS: A total of 8,840 individuals (average age 65±16.8 years) were analyzed. The prevalence of overweight/obesity was 53.4% (n=4,719, average age 67±15.1 years). When compared to non-overweight/obese (n=4,121, average age 62±18.2 years), overweight/obesity was associated with an odds ratio (OR)=1.44 of risk cancer (95%CI:1.18-1.76). When analyzed for specific causes of the 3 most frequent types (all p<0.05): digestive system cancers-OR=1.59 (1.06-2.38), breast–OR=1.39 (1.04-1.85) and thyroid–OR=1.72 (1.05-2.81). CONCLUSIONS: Obesity increases cancer risk and mortality. Our study reflects the strong association between obesity and risk of several cancers as showed in medical literature. Overweight and obesity prevalence are rising and is expected during next years an increase in cancers related with body fatness. Weight reducing programs should be urgently encouraged including improvement of diet and physical activity to better control weight and comorbidities, including cancer.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/bannera0body-fatness-associations-with-cancer-retrospective128122-pdf.pdf?sfvrsn=77bd2aa_0","title":"Banner_A0_BODY FATNESS ASSOCIATIONS WITH CANCER RETROSPECTIVE128122.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128122","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparison of Cost of Incident Rheumatoid Arthritis Patients in Turkey with Selected European Countries","id":"5289900b-b732-456d-8ede-52f768898cf8","sessionCode":"EE42","topDisplay":"Baser O1, Rodriguez N2, Rodchenko K3 1New York City College of Technology-CUNY, New York, NY, USA, 2Columbia Data Analytics, New York, NY, UNITED STATES, 3Columbia Data Analytics, New York, NY, USA","locationCode":"1085","description":"\r\n\t<div>OBJECTIVES: The aim of this study was to calculate annual costs of incident RA cases in Turkey as compared to published annual costs in France, Netherlands, Belgium and Germany. METHODS: Data for this retrospective analysis study was obtained from MEDULA, established under the 2007 Health Budget Law. All patients diagnosed with RA between ages 18 and 99 were identified using appropriate diagnosis codes from the International Classification of Diseases Tenth Revision Clinical Modification (ICD-10-CM). Patients were required to have a 1-year pre-index period (baseline period) and 1-year post-index period (follow-up period) with no RA diagnoses during the baseline period. To control for clinical characteristics, a comorbidity index score was calculated for each patient during the baseline. To estimate risk-adjusted total annual costs for incident cases, expected annual cost values based on patient demographic and clinical characteristics were estimated. Generalized linear models (GLMs) were used to estimate the expected annual cost values. RESULTS: Incident patients were aged 40-64 years and were 80.1% women. Nearly 35% of RA patients had at least one cardiovascular, diabetic, respiratory or allergy comorbid condition prior to diagnosis. The mean Elixhauser Comorbidity Index score was calculated as 5.31. Most patients were prescribed NSAIDs (89.6%) followed by disease-modifying anti-rheumatic drugs (DMARDs) (24.4%) and biologics (4.33%). Total annual costs for incident cases were close to €2,000, and 60% of the costs were due to pharmacy. CONCLUSIONS: In country-specific studies, RA expenditures were estimated at €4,000 in France; €5,028 in the Netherlands; €9,946 in Belgium; and €2,312 in Germany. The results suggest that the total annual cost of RA is lower in Turkey relative to the estimates in Europe. A significant portion of this cost was due to pharmaceutical expenditures.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23baseree42poster130653-pdf.pdf?sfvrsn=b2d86680_0","title":"ISPOREurope23_Baser_EE42_POSTER130653.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130653","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimating Undetected Medication Errors at Care Transitions: A Framework to Support Decision-Making","id":"bf42eaf7-9637-4c81-8525-53fef380d08e","sessionCode":"MSR20","topDisplay":"Gavan S1, Camacho E2, Keers R3, Chuter A2, Elliott R2 1Manchester Centre for Health Economics, Division of Population Health, Health Services Research and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK, UK, 2Manchester Centre for Health Economics, Division of Population Health, Health Services Research and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK, 3Division of Pharmacy & Optometry, The University of Manchester, Manchester, UK","locationCode":"5062","description":"\r\n\t<div>OBJECTIVES: Improving patient safety by reducing medication errors during prescribing at care transitions (for example, hospital admission or discharge) is a global policy priority. Standard medicines reconciliation is typically used to identify medication errors at care transitions. These procedures are imperfect and can miss potentially harmful medication errors. Decision-makers are now seeking evidence demonstrating the added-value of intervention strategies to reduce undetected medication errors at care transitions. To achieve this, analysts must first quantify the current prevalence of medication errors missed by standard medicines reconciliation. This quantity is challenging to estimate because undetected medication errors are unobservable. This study aims to demonstrate a framework enabling analysts to estimate the prevalence of undetected medication errors. METHODS: The framework defines total medication errors as the sum of medication errors detected and undetected by standard medicines reconciliation. Two data sources are required: (1) observational data on the performance of conventional medicines reconciliation to estimate the number of errors detected; (2) the relative risk reduction in medication errors detected by medicines reconciliation versus no medicines reconciliation. First, estimate the total medication errors: medication errors detected by medicines reconciliation divided by one minus the relative risk reduction. Then, estimate the total undetected medication errors: subtract the total detected medication errors (observed) from the total medication errors (estimated). A probabilistic UK-based case study illustrates the framework. RESULTS: Observational data report that 5,910/44,496 (13.3%) medication orders at admission had a medication error detected during medicines reconciliation (Ashcroft et al., 2015). A Cochrane meta-analysis estimated a relative risk reduction in medication errors of 0.13 following medicines reconciliation. Therefore, the estimated total medication errors is 15.3% of prescribed items. The total undetected medication errors is 1.98% of prescribed items. CONCLUSIONS: A robust understanding of undetected medication errors will help demonstrate the added-value of intervention strategies to improve patient safety at care transitions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131744","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Preparing for Successful Market Access and HTA Assessments in Europe","id":"8a823f6b-5e81-4e2b-b462-540c6209018f","sessionCode":"HTA67","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: A successful launch of a pharmaceutical product in across European markets hinges on careful and timely preparation to meet stakeholder requirements and expectations. All preparatory activities are interconnected; therefore, it is important to understand how activities should be timed relative to each other to avoid inefficiencies and gaps in information. This research explores the key activities and associated timelines to consider when preparing for successful market access and HTA assessments in Europe METHODS: A review of the literature was conducted to analyse key steps required to prepare a drug for European market access while a review of select EU market regulatory/HTA bodies decisions and publications, allowed for an analysis of historical examples of pharmaceutical products that underwent launch over the past 10 years. These insights were consolidated to generate a step-by-step timeline of activities required for successful launch in the EU, highlighting the opportunities and barriers of different European market archetypes. RESULTS: Successful launch planning has been categorized into five key phases each with an optimal timeframes relative to market entry; these include, (1) Landscape Analysis (48-months through 24-months pre-launch; with continuous updates), (2) Evidence Generation Planning (24-months pre-launch through 3-months post-launch), (3) Pricing Strategy (24-months through 6-months pre-launch), (4) Payer Engagement Preparation (24-months pre-launch through 3-months post-launch), and (5) HTA Preparation (18-months pre-launch through to launch). Each of these phases have been distilled further to outline critical activities for each preparation phase, highlight key learnings from historical product launches, and generate considerations for each European market archetype. CONCLUSIONS: Systematic and early planning for market access is essential to secure optimal price and access. Understanding the landscape, generating robust evidence, developing pricing strategies, engaging with payers, and preparing for HTA assessments are crucial steps in achieving successful market access in Europe. </div>\r\n\r\n","withdrawn":true,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133035","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Reasons for a Negative Pharmaceutical Benefits Advisory Committee (PBAC) Review in Rare Diseases in Australia: A Comprehensive Analysis","id":"24a75635-5063-4df1-9850-55cce200461b","sessionCode":"HTA23","topDisplay":"Mumford A1, Darlington O2, Lyttle SJ3, Shaw A3 1Initiate Consultancy, Northampton, UK, 2Initiate Consultancy, NA, UK, 3Initiate Consultancy, London, UK","locationCode":"4063","description":"\r\n\t<div>OBJECTIVES: The Pharmaceutical Benefits Advisory Committee (PBAC) plays a crucial role in evaluating the clinical and cost-effectiveness of medications for reimbursement in Australia. However, the process for rare diseases can pose unique challenges. The objective of this study was to identify and analyse the reasons for negative PBAC reviews in rare diseases, focusing on the objectives, methods, results, and conclusions of the assessment process. METHODS: This study involved a comprehensive review and analysis of publicly available PBAC meeting outcomes, guidelines, and relevant literature on orphan drug evaluations published between 2021 and 2023. The identified evaluations were examined to identify common reasons for negative reimbursement decisions by PBAC. RESULTS: The review identified 22 negative PBAC recommendations across 20 different orphan medications by PBAC between 2021 and 2023. The most frequent factors contributing to a negative outcome were high treatment costs (77%), uncertainties in estimating long-term clinical effectiveness (50%), limited clinical evidence (36%), and issues with economic modelling (32%). Additionally, the proposed positioning of treatments, variations in disease prevalence and heterogeneity, as well as limited patient populations and associated difficulties in conducting clinical trials contributed to negative outcomes. CONCLUSIONS: This study highlights the primary reasons for negative PBAC reviews for orphan medications, shedding light on the challenges faced in assessing the clinical and cost-effectiveness of treatments for rare diseases. It is essential to explore alternative evaluation methodologies that can help mitigate the unique challenges of obtaining reimbursement for treatments for rare diseases in Australia, such as adaptive pathways, real-world evidence, and patient-reported outcomes. Collaborative efforts among stakeholders, including pharmaceutical companies, patient advocacy groups, and regulatory bodies, are necessary to overcome these challenges and ensure timely access to effective treatments for patients with rare diseases.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/reasons-for-a-negative-pbac-orphan132314-pdf.pdf?sfvrsn=bc9d2430_0","title":"Reasons for a negative PBAC Orphan132314.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132314","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Evaluation of the NICE Early Value Assessment: What Is the Opportunity for Digital Therapeutics?","id":"24b90a50-10bd-482f-8fd1-562ee0ba79e9","sessionCode":"HTA5","topDisplay":"Macaulay R, Carr D, Moran V Precision Advisors, London, UK","locationCode":"4044","description":"\r\n\t<div>OBJECTIVES: To assess how digital therapeutics (DTx) have been evaluated by the National Institute for Care and Health Excellence (NICE) in the Early Value Assessment (EVA) process. METHODS: A targeted literature review was conducted on the NICE website for EVAs for digital therapies and the resulting health technology evaluations (HTE) published until 20-June-2023 were analysed for evidence submitted and reimbursement outcomes. RESULTS: DTx were grouped according to their indication and population in 3 HTEs. HTE3 evaluated 5 DTx for treating mild to moderate symptoms of anxiety or low mood in children or adolescents. The evidence base consisted of 7 studies and 2 conference abstracts, of which there was 1 randomized controlled trial (RCT). 4 DTx received conditional recommendations and 1 was not recommended. HTE8 evaluated 6 DTx for treating depression in adults. The evidence base consisted of 46 articles, of which there were 14 RCTs. 3 DTx received conditional recommendations and 3 were recommended for research only. HTE9 evaluated 11 DTx for treating anxiety disorders in adults. The evidence base consisted of 19 published studies, of which there were 4 RCTs. 12 recommendations were made: 6 conditional and 6 for research only. Overall, 16 DTx were given 27 recommendations, with 48% receiving conditional recommendations, 48% in research only, and 4% not recommended. Of the total evidence submitted, 32% of studies came from RCTs. CONCLUSIONS: The EVA process provides a route for DTx to be assessed early in evidence development and receive conditional reimbursement while additional confirmatory data is collected. This enables local patient access and may have positive effects in markets that look to NICE guidance. However, by submitting early and potentially less mature data, an ‘only in research’ recommendation is possible. Manufacturers need to consider this speed versus access trade-off in their product development journey. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporprecisionadvisorsan-evaluation-of-the-nice-early-value-assessment20oct23vf131663-pdf.pdf?sfvrsn=3becbf61_0","title":"ISPOR_PrecisionAdvisors_An evaluation of the NICE Early Value Assessment_20Oct23_vF131663.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131663","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Drivers of Positive Health Technology Assessment Outcomes for Oncology Drugs in Three Global Markets","id":"1fc26cb8-5aba-46a3-a181-57811ddc7fc7","sessionCode":"HTA11","topDisplay":"Norwood-Knutsson C1, Genane C2, Cesarec S1 1GlobalData, London, LON, UK, 2GlobalData, Le Blanc-Mesnil, France","locationCode":"4053","description":"\r\n\t<div>OBJECTIVES: To determine the key drivers and considerations influencing Health Technology Assessment (HTA) outcomes for oncology drugs in Australia, Canada, and the United Kingdom (UK), to provide insight into reimbursement pathways and alleviate uncertainties during the appraisal process across three national HTA agencies. METHODS: A comprehensive review of all HTA assessments conducted for marketed oncology drugs between January 2022 and June 2023 in respective markets was identified. By leveraging literature reviews of appraisal recommendations, common factors influencing HTA decision-making were assessed to categorize positive, negative, and neutral outcomes. RESULTS: Of the 150 HTA assessments identified, the most influential factors that impact HTA outcomes were clinical efficacy, economic implications, and disease-related factors. Australia (n=25) demonstrated the highest proportion of positive outcomes overall (76%) driven by economic cost-effectiveness, while clinical efficacy (55%) played a greater role in the UK (n=66). In Canada (n=59), clinical efficacy predominantly contributed to neutral (87%) and negative (100%) outcomes. 61% of cases irrespective of the outcome were attributed to clinical efficacy, which can be further segregated into the following categories, clinical evidence acceptability (67%), comparative efficacy (38%), and comparator choice (>1%). Among positive outcomes, clinical evidence acceptability was the most common sub-factor (53%), and insufficient/uncertain clinical evidence was a determining factor for the negative outcomes overall (50%). CONCLUSIONS: While HTA agencies exhibit divergent priorities within different markets, the study reveals commonalities in the factors forming the basis for decision-making, namely that the clinical dimension is still a key factor in assessments. Navigating the distinctive socio-political and population-specific needs of each market poses challenges for manufacturers to tailor strategies effectively. By accounting for HTA outcome drivers, stakeholders can better navigate the complex landscape more successfully to ensure improved access to oncology treatments.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/drivers-of-positive-health-technology-assessment-outcomes-for-oncology-drugs-in-three-global-marketscaitlin-norwood-knutsson131317-pdf.pdf?sfvrsn=e83ebce0_0","title":"Drivers of Positive Health Technology Assessment Outcomes for Oncology Drugs in Three Global Markets_Caitlin Norwood Knutsson131317.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131317","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Measuring Travel Distances to Victorian Cancer Care Facilities: Does Method Matter, and for Whom?","id":"616ac711-8d77-44b9-b0e6-580ab9dc6e89","sessionCode":"OP2","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Our study investigates the impact of an alternative methodology for analysing cancer patient travel distances for radiotherapy treatment in Victoria. Travel distances are often calculated using a simplistic method based on the straight-line distance between postcode centroids, which may not reflect true travel distance. Leveraging a state-wide retrospective linked dataset, we establish the difference in calculated travel distance between methods and its impact on eligibility for travel subsidies. METHODS: We analyse travel distances for patients diagnosed in Victoria between 2010 and 2019, undergoing radiotherapy in Victoria. We assess the absolute and relative differences in distances generated by two distinct methods: (1) straight-line distances between residence and facility postcodes centroids, (2) Google Maps API method calculating distances from residence postcode to facility postcode centroids, using the existing road networks. We then applied these distances to the Victorian Patient Transport Assistance Scheme 100km one-way travel subsidy threshold. RESULTS: Our study found that the straight-line method underestimated travel distances by ~15-20%. This underestimation was consistent overall and across remoteness levels. The calculated difference in means between methods was 8.92km (p<2.2x10-16). The maximum observed difference between the methods was 210km. Calculated straight-line distances had 0.38%, 17.1%, and 80.8% while the Google Maps method found 0.63%, 27.4% and 87.7% of metropolitan, inner regional and outer regional patients respectively reach the subsidy threshold. CONCLUSIONS: This study highlights the drawbacks of using the straight-line method to calculate travel distance for Victorian cancer patients. Underestimation of travel burden becomes more pronounced with longer travel distances with Victorians classified as ‘Outer Regional’ carrying the majority of the travel burden. Researchers and policymakers should carefully assess methodological choice and recognise the importance of granular population-wide research data to ensure equitable access to cancer care for all Victorians.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"bb8fd992-de86-4d1a-8bec-f6e2c928db96","parentId":"00000000-0000-0000-0000-000000000000","title":"Organizational Practices","urlName":"organizational-practices"}],"categoryIds":["bb8fd992-de86-4d1a-8bec-f6e2c928db96"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131365","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Summary of Economic Evaluations on Onco-Hematology in France By the Has: What Did We Learn?","id":"692d92fe-e071-4ad7-840f-584f91a0d63d","sessionCode":"HTA16","topDisplay":"Boussahoua M1, Sambuc C2, Tehard B1, Chevalier J1, Midy F1, Roze S3 1Vyoo Agency, Paris, 75, France, 2VYOO Agency, Paris, 75, France, 3Vyoo Agency, VILLEURBANNE, 69, France","locationCode":"4041","description":"\r\n\t<div>OBJECTIVES: In France, the Commission for Economic and Public Health Evaluation (CEESP) provides an economic opinion of the incremental cost-effectiveness ratio (ICER) validity. During the last few years, due to the arrival of innovative therapies such as CAR-T cells therapies, onco-hematology is taking an important place in this area. The aim is to analyze all the conclusions of the CEESP opinions in onco-hematology. METHODS: Using Vyoo Agency efficiency database, all CEESP opinions in onco-hematology appraised until May 25, 2023, were reviewed. We consider an opinion valid if there are no mention of major uncertainty or objection. As CEESP doctrine has evolved during the last 2 years, uncertainty was not a discriminant criterion before august 2021. So, the ICER as not validated only if CEESP conclusion is clearly stated that is not retained despite an acceptable methodology. RESULTS: Among the 34 opinions in onco-hematology, 33 were published (one was not published). These opinions were related to 10 indications, with 27% of them in chronic lymphocytic leukemia and 18% on Multiple Myeloma. Fourteen analyses were based on phase-2 trial with 12 opinions non-validated. The main reasons are related to the lack of robust data, estimation of treatment effect and/or the methodology of indirect comparison (including two-by-two comparison that do not allowed to provide the efficiency frontier. Among them, 7 opinions concerned CAR-Ts therapy and only 3 were validated by the CEESP. Among validated ICER, results range from €7,392/QALY to €568,465/QALY. CONCLUSIONS: Onco-hematology is characterized by a low proportion of validated economic opinions and an important variability of the results. These can be related to the specific methodological and clinical background of this area and the increase of CAR-T cells therapies. Indeed, the duration of the simulation, the treatment effect for one-shot therapy and the inclusion of all the comparators (single-arm trials) complicate the modeling.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023isporhta16130406-pdf.pdf?sfvrsn=5daf82ae_0","title":"2023_ISPOR_HTA16130406.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130406","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patient-Reported Outcomes (PROs) in German AMNOG-Assessments: Impact of Increasing the Response Threshold to 15 %","id":"2fd38de7-1640-42b5-ba1c-5880e4381ff3","sessionCode":"PT4","topDisplay":"Fischer-Huchzermeyer S, Blank P, Manegold I, Müller J, Billig S, Kulp W Xcenda GmbH, part of Cencora, Hannover, NI, Germany","locationCode":"4A","description":"\r\n\t<div>OBJECTIVES: Defining the Minimal Clinically Important Difference (MCID) for the evaluation of PROs is challenging. In March 2022, the Federal Joint Committee (G-BA) adopted a new response threshold of ≥ 15% of the scale range of the questionnaire for binary analyses of PROs. The aim of our study was to evaluate how the new response threshold affects the assessment of the added medical benefit of PROs in Germany. METHODS: The G-BA website was searched for benefit assessments published in the transition period 01/2021-03/2023. The search terms were limited to EQ-5D, SF36, FACT as these questionnaires represent the most common PROs affected by the adoption. Only assessments including data on both analyses, the previously accepted MCID and the newly introduced threshold of ≥ 15%, and that were methodologically accepted by the G-BA were considered. RESULTS: Overall, 23 of 129 screened assessments met the inclusion criteria. In 16 assessments, no significant PRO results could be demonstrated, regardless of the response threshold applied. For the remaining seven assessments, a significant treatment benefit was achieved with the previously accepted MID < 15%. However, in five of these seven assessments significant treatment benefit was also achieved with the new threshold of ≥ 15%. CONCLUSIONS: Overall, only in seven assessments PROs achieved a significant treatment benefit. This demonstrates how challenging it is to achieve an added medical benefit through PROs. This might be explained by the fact that the majority of assessments (16/23) covered oncology drugs for which maintaining health status or quality of life is already considered a treatment success. The new response threshold of ≥ 15% represents a higher hurdle to prove an added medical benefit based on PROs. Consequently, minor but yet patient relevant benefits might not be valued appropriately.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23fischer-huchzermeyerpt4poster129801-pdf.pdf?sfvrsn=747de223_0","title":"ISPOREurope23_Fischer-Huchzermeyer_PT4_POSTER129801.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129801","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Description of Portal Hypertension Treatment by Transjugular Intrahepatic Portosystemic Shunt: A French National Hospital Database Analysis","id":"7c95ae9f-a2de-45d6-89b3-58cf00539da1","sessionCode":"MT2","topDisplay":"de Léotoing L1, Tournier C2, Leboucher C2, Blein C2 1W.L. Gore & Associates, Paris, 75, France, 2Creativ-Ceutical, Lyon, France","locationCode":"5029","description":"\r\n\t<div>OBJECTIVES: Cirrhosis is the main cause of portal hypertension (PH), leading to ascites and/or variceal bleeding with a significant impact on patient’s survival. The main objectives of the study are to describe the hospital management of PH treatment by transjugular intrahepatic portosystemic shunt (TIPS) and estimate the burden of ascites and variceal bleeding recurrence. METHODS: This population-based retrospective cohort study was performed using the French exhaustive national hospital discharge database (PMSI). Patients who had hospitalization for a TIPS procedure according to the French procedures classification (CCAM) between January 1, 2018 and December 31, 2020 were included. Each patient included before December 31, 2019, was followed-up for one year after cohort entry date and additional hospitalizations for ascites or variceal bleeding treatments were retrieved, combining diagnosis and procedure codes: ascites and large volume paracentesis (LVP); variceal bleeding and endoscopic band ligation (EBL). RESULTS: 2,475 patients were hospitalized between 2018 and 2020 for a TIPS procedure. The mean age of patients was 58.7 ± 10.7 years old; 76.4% of them were males. 1,401 (56.6%) presented with ascites, and 1,280 (51.7%) with variceal bleeding. 81.7% were treated with a GORE® VIATORR® TIPS Endoprosthesis with Controlled Expansion. Mean length of stay (LOS) was 13.5 ± 21.2 days. Overall survival was 68.2% at one year and 56.0% at two years. Among the 1,690 patients included before December 31, 2019, 518 (20.9%) were re-hospitalized for LVP within 114 days on average and 103 (4.2%) for EBL within 215 days on average. CONCLUSIONS: This study confirms the interest of TIPS treatment on PH complications in France, with an impact on LVPs and EBLs. Given the forthcoming increase in cirrhosis, it highlights the need to refer more patients to earlier/preemptive TIPS in line with international guidelines, to reach expected survival.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23de-leotoingmt2poster129773-pdf.pdf?sfvrsn=7b60b141_0","title":"ISPOREurope23_DE LEOTOING_MT2_POSTER129773.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129773","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Capacity-Enhancing Innovation (CEI) and the Treatment of Severe Symptomatic Aortic Stenosis (sSAS) in England","id":"1573be01-a147-4bee-9dd2-59728c6e08f5","sessionCode":"RWD1","topDisplay":"Sarmah A1, Busca R1, Mamas MA2 1Edwards Lifesciences SA, Nyon, VD, Switzerland, 2Keele University, Keele, Newcastle, UK","locationCode":"6055","description":"\r\n\t<div>OBJECTIVES: Surgical aortic valve replacement (SAVR) has been the default treatment for sSAS, although transcatheter aortic valve implantation (TAVI) has emerged as an effective option since its introduction. CEI refers to the responsible adoption of new technologies that increase the ability of an organization to produce more, perform better, or operate at a higher level of efficiency. This study aims to quantify the role of TAVI as a CEI in reducing hospital length of stay (LoS) and its ability to expand treatment to more patients with sSAS. METHODS: This retrospective cohort study used secondary care patient-level Hospital Episode Statistics (HES) data for England, to identify two patient cohorts from inpatient records: SAVR cohort and TAVI cohort, between January 2017 and December 2022. Each admission was associated with the Healthcare Resource Groups (HRGs) to enable the differentiation between levels of care complexity (CC). The primary outcomes, average LoS in Intensive Care Unit (ICU), post-operation (post-op) period and for the entire admission were compared between the two cohorts. RESULTS: The HES database included 14165 TAVI and 11770 SAVR procedures across the 6 years study period. Compared to 2019, TAVI admissions increased by 26.3% in 2022 while SAVR admissions decreased by 30.3%. In 2022 the average admission LoS ranged between 4 and 10 days for TAVI and 9 and up to18 days for SAVR cases respectively for low and high CC groups. The average post op LoS ranged between 2 and 5 days for TAVI cases whilst 7 and up to 12 days for SAVR. ICU LoS was between 2 days for TAVI and 3 days for SAVR in low CC. CONCLUSIONS: TAVI represents a CEI for sSAS patients and given that clinical practice guidelines now suggest the inclusion of lower risk patients, we expect TAVI’s contribution to the healthcare system organization to grow.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-ceiispor-europe2023-30102023129199-pdf.pdf?sfvrsn=7c14e4dc_0","title":"Poster CEI_ISPOR Europe2023 30102023129199.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129199","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Utilizing Real-World Evidence to Estimate Treatment Costs Among Norwegian Patients with Myasthenia Gravis Treated with Intravenous Immunoglobulin (IVIG)","id":"1e6e7e07-6e35-4e50-b793-598c27e9f76a","sessionCode":"EE43","topDisplay":"Bugge C1, Engebretsen I1, Kristiansen IS2, Arneberg F3, Gilhus NE4 1Oslo Economics, Oslo, Norway, 2University of Oslo, Oslo, Norway, 3UCB, Oslo, Norway, 4University of Bergen, Bergen, Norway","locationCode":"1087","description":"\r\n\t<div>OBJECTIVES: Accurate estimation of costs for specific patient sub-populations is imperative for mathematical simulation models assessing cost-effectiveness of novel treatments. Economic evaluations often rely on guidelines and expert inputs, which may not adequately capture real-world treatment practices. This study aimed to utilize mandatory, nationwide patient-level registry data to estimate treatment costs of myasthenia gravis (MG), a rare neuromuscular disorder characterized by variable degrees of skeletal muscle weakness. METHODS: Incident MG patients were identified in the Norwegian Patient Registry based on the presence of at least two hospital encounters with an MG diagnosis code (ICD-10 G70.0) during 2010-2021, excluding patients recorded with MG in 2008 and 2009. Analyses were conducted for the subgroup of MG patients receiving intravenous immunoglobulin (IVIG) treatment, and MG patients not receiving IVIG treatment. Identified by procedure and ATC codes, IVIG treatment was defined as either a single in-patient visit where IVIG was administered or two out-patient episodes with an IVIG administration within a three-month interval. Annual direct treatment costs for each year following diagnosis were estimated using Diagnosis-Related Group (DRG) cost weights. RESULTS: Among the 1,083 incident MG patients, 153 (14.1%) were treated with IVIG. Patients undergoing IVIG treatment had 2.5-fold higher treatment costs during the first year after diagnosis (EUR 31,832 vs. EUR 12,706) and 3.1-fold higher costs during the second year after diagnosis (EUR 16,251 vs. EUR 5,284) compared to MG patients not receiving IVIG treatment. In the fifth year after diagnosis, IVIG-treated patients still had higher costs than those not receiving IVIG (EUR 8,533 vs. EUR 4,722). No significant differences in age or gender were observed between those with or without IVIG. CONCLUSIONS: IVIG treatment is a significant driver of direct medical costs associated with MG and changes in these costs should be accounted for when evaluating new interventions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-norwegian-mg-rwe-posterfinal301023131264-pdf.pdf?sfvrsn=9f23df7b_0","title":"ISPOR Norwegian MG RWE poster_final301023131264.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131264","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Comparative COVID-19 Vaccine Effectiveness of a Third Dose of mRNA-1273 Versus BNT162b2 Among Immunocompromised Adults in the US","id":"6c08cde3-60b2-4130-b696-59f1316b8615","sessionCode":"CO38","topDisplay":"Beck E, Sun T, Li L, Georgieva M, Van de Velde N Moderna, Inc., Cambridge, MA, USA","locationCode":"1036","description":"\r\n\t<div>OBJECTIVES: Moderately to severe immunocompromised (IC) individuals are among the most vulnerable for COVID-19. IC individuals comprise approximately 3% of the US population but account for an estimated 44% of breakthrough COVID-19 hospitalizations. Previous studies have shown that IC individuals were better protected with two doses of mRNA-1273 compared with 2 doses of BNT162b2 (primary series). The objective of this study was to compare the real-world effectiveness of the third dose of mRNA-1273 versus BNT162b2 in the US. METHODS: A retrospective cohort study using HealthVerity claims data from December 2020 to August 2022 was conducted. IC individuals who completed a primary vaccination series were followed from 14 days after receipt of their third dose of COVID-19 vaccine (mRNA-1273 or BNT162b2) until receipt of another COVID-19 vaccine, outcome(s) occurring, end of continuous enrollment or end of data cut. Inverse probability of treatment weighting (IPTW) was applied to adjust for baseline measurement confounding. Comparative vaccine effectiveness against medically attended COVID-19 infection and hospitalization was estimated based on rate differences (RDs) between the 2 vaccine groups. RESULTS: Preliminary results show that IC individuals who received mRNA-1273 as their third dose (n=52,943) had lower crude rates of medically attended COVID-19 (166 vs 179 per 1000 person-years) and COVID-19 hospitalization (7.9 vs 10.0 per 1000 person-years) compared with those who received BNT162b2 (n=60,084). After IPTW, people who received a third dose of mRNA-1273 had statistically lower rate of medically attended COVID-19 (RD=-7.5; 95% CI: -14.3, -0.6) and COVID-19 hospitalizations (RD=-2.3; 95% CI: -3.8, -0.8) compared with their BNT162b2 counterparts. CONCLUSIONS: Similar to prior experience with the primary series, these real-world effectiveness results demonstrate that IC individuals were better protected with a third dose of mRNA-1273 compared to BNT162b2. As COVID-19 disease burden remains high in the IC, these individuals should follow recommended vaccination guidance for the fall 2023/2024 season.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/icpopulationh2hrweposterisporeu2023132308-pdf.pdf?sfvrsn=37e9506_0","title":"IC_Population_H2H_RWE_Poster_ISPOR_EU_2023132308.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/icpopulationh2hrwepostersuppisporeu2023132308-pdf.pdf?sfvrsn=c7c103d0_0","title":"IC_Population_H2H_RWE_Poster_Supp_ISPOR_EU_2023132308.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132308","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical and Economic Impact of Point-of-Care (POC) Molecular Testing in Patients Presenting With Viral Respiratory Symptoms: A Systematic Literature Review (SLR)","id":"3db53659-8934-4530-a139-5abfe1b5c6d5","sessionCode":"MT10","topDisplay":"Hale B1, Mojebi A1, Wu P1, Keeping S1, Beaubrun A2 1Evidence Synthesis and Decision Modeling, PRECISIONheor, Vancouver, BC, Canada, 2Cepheid, Sunnyvale, CA, USA","locationCode":"5039","description":"\r\n\t<div>OBJECTIVES: Molecular tests, including polymerase chain reaction (PCR), can detect lower concentrations of viral material over a longer period of respiratory infection than antigen tests. Delays associated with central laboratory testing can result in hospital-acquired transmission, avoidable patient admission, and unnecessary use of antimicrobials may lead to increased cost of patient management. The aim of this study was to summarize comparisons of the clinical and economic outcomes associated with PoC molecular testing versus other diagnostic tests for viral respiratory infections. METHODS: An SLR conducted in April 2023 identified studies evaluating clinical and economic outcomes of molecular and antigen diagnostic tests for patients suspected of having a respiratory virus infection. RESULTS: The SLR included 232 studies, of which nine and 17 compared PoC molecular testing to other diagnostic tests for COVID-19 and influenza, respectively. Studies that tested for COVID-19 generally concluded that PoC molecular testing reduced median time to test result (0.18-3.1 hours vs. 4.29-26.4 hours; n=7 studies) and median length of emergency department stay (3.2-7.2 hours vs. 4.6-12.0 hours; n=4 studies) compared to laboratory PCR. PoC molecular testing was also found to decrease exposure time of uninfected patients and frequency of antiviral and antibacterial therapy, while improving patient flow and preventing healthcare-associated and hospital-acquired infections. A similar trend was observed in studies testing for influenza in that PoC molecular testing led to reduced time to test result and length of stay in hospital or emergency department compared to antigen tests or laboratory PCR, while reducing rates of hospitalization or emergency admission. CONCLUSIONS: Compared to other diagnostic tests, PoC molecular testing improves clinical outcomes, test turnaround time, and stewardship by decreasing unnecessary use of antibiotics and antivirals. It shifts cost away from more expensive settings by reducing length of stay at hospital and avoiding unnecessary exposure of patients to hospital-acquired infections.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23halemt10poster133618-pdf.pdf?sfvrsn=2c3e80b2_0","title":"ISPOREurope23_Hale_MT10_POSTER133618.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133618","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Mental Health Disparities for Young Adults with Arrest Histories in the United States","id":"d2359e23-71d2-4ea3-aa6a-5b44852fdd80","sessionCode":"EPH19","topDisplay":"Baser O1, Rodchenko K2, Zeng Y2 1City University of New York, New York, NY, USA, 2Columbia Data Analytics, New York, NY, USA","locationCode":"3020","description":"\r\n\t<div>OBJECTIVES: Over 4.53 million arrests were made in 2021 in the United States. People under 26 were more likely to be arrested than those who were older. Although mental health disparities have been found in the incarcerated population, the subject was not closely examined for young adults. This study explores the relationship between a history of arrest and mental health concerns in adults between 18 and 25 years old. METHODS: We analyzed secondary data using the 2021 National Survey on Drug Use and Health (NSDUH). The study used a subsample of 13,494 people aged 18 to 25, including 7330 women and 6164 men. The history of arrest was the independent variable. Depression, serious mental illness (SMI), substance use, suicidal ideation, and suicide attempt were the outcome variables. We performed five binary logistic regression models for each outcome variable, controlling for race/ethnicity, income, and education level. RESULTS: Of 13,494 respondents, 6.63% had a history of arrest. Among young women, a history of arrest was associated with significantly higher adjusted odds ratios for all mental health concerns. Most notably, a history of arrest increased the likelihood of substance use by a factor of 15.19, suicide attempts by 2.27, SMI by 1.79, suicide ideation by 1.75, and depression by 1.52. Among young men, a history of arrest was associated with increased adjusted odds ratios for substance use (AOR=13.37, p<0.001), suicidal ideation (AOR=1.45, p=0.011), and suicide attempt (AOR=1.82, p=0.044). CONCLUSIONS: We found a strong relationship between young people having a history of arrest and mental health concerns. Moreover, among young women, a history of arrest affected all mental health concerns, while it was associated with only substance use and suicide among young men. Providing arrestees with appropriate mental health care would benefit them and the criminal justice system by decreasing the odds of repeated arrests.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23samayoaeph19poster130644-pdf.pdf?sfvrsn=9428aa28_0","title":"ISPOREurope23_Samayoa_EPH19_POSTER130644.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130644","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Use of Patient Reported Outcomes in Key Trials Supporting Marketing Authorisation: 5-Year Analysis of Company Evidence Submitted for National Institute for Health and Care Excellence (NICE) Single Technology Appraisal (STA)","id":"3a59d730-0cef-4185-ba73-5b8e3a8c63f8","sessionCode":"PCR52","topDisplay":"Los A1, Goldsmith G2, Kilty RLH2, Brown A3, Phalguni A2 1Genesis Research, Darlington, UK, 2Genesis Research, Newcastle upon Tyne, Tyne and Wear, UK, 3Genesis Research, Whitley Bay, NT, UK","locationCode":"6049","description":"\r\n\t<div>OBJECTIVES: With growing interest in capturing patient experience during clinical trials, the use of PRO measures (PROMs) has been recommended by regulatory authorities. This study aimed to evaluate the use of PROMs in company evidence submitted for NICE STA in the last 5 years. METHODS: STAs published on the NICE website between April 1st 2018 and April 1st 2023 were reviewed. Information on disease indication, key trials design, and types of PROMs were collected and analysed. RESULTS: In total, 352 STAs were identified. After excluding appraisals which were terminated, replaced by newer guidance, or in development, 257 STAs were included . Of these 257 STAs , 140 (54.5%) were published in oncology, 20 (7.8%) in musculoskeletal, and 19 (7.4%) in neurological indications. PROMs were reported in 227 STAs (88.3%) and were limited to health-related quality of life (HRQoL) outcomes. 131 different PROMs were identified, including: (1) indication-specific QoL measures (n=76; 58.0%), (2) generic QoL measures for symptoms (e.g., pain), function (e.g., visual), or safety (e.g., adverse events) (n=30; 22.9%), and (3) generic QoL measures (n=25; 19.1%). The EuroQol-5 Dimensions (EQ-5D) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-core questionnaire (EORTC QLC-C30) were the most commonly used PROMs (n=144; 63.4% and n=81, 35.7%, respectively). We further analysed PROMs in oncology STAs. Across 140 STAs, 125 (89.3%) conducted PROM assessments, the majority of which used generic QoL measures (n=124; 99.2%), indication-specific measures were used in 63 STAs (50.4%), and generic measures for symptoms, function, or safety were reported in 15 STAs (12.0%). Most oncology STAs used a combination of generic and indication-specific PROMs (46.4%) or generic PROMs only (41.6%). CONCLUSIONS: The frequent use of PROMs in evidence submitted by company highlights the importance of QoL data in NICE STAs to support health technology assessment decision making.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-europe-pcr52-use-of-patient-reported-outcomesfinal130497-pdf.pdf?sfvrsn=c26aa4fc_0","title":"ISPOR Europe-PCR52-Use of Patient Reported Outcomes_Final130497.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130497","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Differing Approaches to the Health Economic Investigation of the Informal Care of People Living With Multiple Sclerosis: A Systematic Review and Meta-Analysis","id":"aa79aab8-91c7-4827-9bc2-5bcbc81e3b09","sessionCode":"EE45","topDisplay":"Adal T1, Roydhouse J2, van der Mei I1, Taylor BV1, Palmer AJ1, de Graaff B1, Henson G1, Campbell J1 1University of Tasmania, Hobart, TAS, Australia, 2Menzies Institute for Medical Research, Hobart, TAS, Australia","locationCode":"1092","description":"\r\n\t<div>OBJECTIVES: MS is a high-cost and high-burden disease that causes substantial health and economic impacts. Economic studies in MS often exclude the costs of Informal Care (IC) which will likely underestimate the cost-effectiveness of interventions aimed to slow, prevent, or halt MS-related disability progression. We aimed to estimate the annual per person cost of IC and identify cost differences between countries and MS-related disability severity levels. METHODS: This review was conducted in accordance with the PRISMA guidelines and a pre-determined study protocol (PROSPERO - CRD42023396457). Searches were conducted from four biomedical (PubMed, MEDLINE, EMBASE, and Scopus) and four economic databases. Two authors independently screened, assessed, and extracted data for narrative synthesis and meta-analysis. Data were stratified for country income level and Expanded Status Disability Scale (EDSS) of the person with MS where available. Summary statistics and meta-analyses of costs were converted to 2022 United States dollars (US$). RESULTS: N=6305 articles were identified from database searches. Notably, most studies evaluated the cost of IC from the perspective of the person living with MS, not the caregiver. Sixty were retained for narrative synthesis and n=49 for meta-analysis. Average monthly caregiving time was 60.1 hours. Globally, mean annual cost of IC per person were US$7,453 (95% CI $5987-9226) and the pooled mean cost from 49 studies was US$6,369(95% CI 5502-7236). The costs of IC were US$7,944 and US$2,284 in high-income and upper-middle-income countries, respectively. The costs for mild, moderate, and severe disability of the person with MS were US$1,109, US$6,580, and US$16,023, respectively. Overall, the costs of IC accounted for 15% of the total societal cost of MS, globally. CONCLUSIONS: The cost of IC contributes considerably to MS-related costs. Cost for IC increases with MS-related disability severity and country income level. Therefore, IC should be considered in cost estimation, policy development and decision-making.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132386","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Knowledge on HPV and Vaccination Among Undergraduates in Vojvodina","id":"fdef2968-044f-4cb9-b9fd-5c49c790f96b","sessionCode":"EPH34","topDisplay":"Lénárt Z1, Kozmann K1, Csákvári T1, Vajda R1, Fusz K1, Ferenczy M2, Boncz I1, Pakai A3 1University of Pécs, Pécs, Hungary, 2University of Pécs, Szombathely, ZA, Hungary, 3University of Pécs, Pécs, ZA, Hungary","locationCode":"3001","description":"\r\n\t<div>OBJECTIVES: Human papillomavirus (HPV) is the most common viral infection of the reproductive system. Although most HPV infections are mild, it is a risk factor of cervical cancer. The aim of this study is to assess knowledge about HPV and HPV vaccination, as well as screening uptake among university students. METHODS: We conducted a quantitative, cross-sectional study between November 2022 and February 2023 among university students from the Autonomous Province of Vojvodina, Serbia. Non-random, accidental sampling method was used to select full-time undergraduates of 18-25 years (N=102). A self-administered questionnaire was developed to measure sociodemographic data, as well as knowledge on HPV, HPV vaccines, cervical cancer and cervical cancer screening. Descriptive statistics, χ2 test and t test were calculated (p<0.05). RESULTS: The majority of respondents were women (78.4%), students of humanities (26.5%) and live in rural area (61.8%). Mean age of the sample was 21.59±1.91 years. Only 8.8% of the students received HPV vaccination. 46% of women have not attended cervical cancer screening before. Mean score of the knowledge test was 15.98±4.53 points. Regarding knowledge level, significant difference was found between sexes (p=0.02), but not in marital status (p=0.292) and type of residence (p=0.098). A higher proportion of students use the internet to gather information. The knowledge level of vaccinated persons is higher than that of unvaccinated persons (p=0.008). CONCLUSIONS: Our results will provide insights into students' knowledge, awareness and screening habits. The results of the survey will provide data for the development of further education programmes to increase the proportion of vaccinated individuals among students.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133751","diseases":[{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Drug Approvals for Advanced Breast Cancer in Brazil: Historical Perspective and Comparative Cost-Effectiveness Analysis","id":"58afbd7f-586a-461e-90dc-5c63535ad51f","sessionCode":"HPR8","topDisplay":"Goncalves V1, Nazareth Aguiar P2, Dos Reis Neto JP3, Santos M4, Busch J5, Katz A6, Gondo CM7, Paladini L8, Senra F9, Brust L10, Rocha J11, Stefani S12 1UNIMED Central RS, Porto Alegre, RS, Brazil, 2Grupo Oncoclínicas, São Paulo, Brazil, 3CAPESESP, Rio de Janeiro, RJ, Brazil, 4Sociedade Brasileira de Auditoria Médica, Sao Paulo, SP, Brazil, 5Souza Marques University, Rio de Janeiro, RJ, Brazil, 6UNIMED GUARULHOS, SÃO PAULO, S.P., Brazil, 7SEGUROS UNIMED, SÃO PAULO ,SP, Brazil, 8UNIMED Uberlândia, Uberlândia, MG, Brazil, 9UNIMED Araraquara, Araraquara, SP, Brazil, 10Centro Universitário Univates, Rio Grande do Sul, Brazil, 11Unimed Curitiba, Curitiba, PR, Brazil, 12UNIMED Central RS, Porto Alegre, Brazil","locationCode":"3065","description":"\r\n\t<div>OBJECTIVES: We sought to evaluate the clinical and economic characteristics of cancer drugs approved for the treatment of advanced breast cancer (ABC) and understand the factors limiting incorporation, access to innovative drugs and improvement in clinical outcomes for the disease in Brazil. METHODS: By using the Brazilian regulatory agency (ANVISA) database of the last 15 years, we identified all drugs which were first-in-class or with newly approved indications for the treatment of ABC in Brazil. After that, we evaluated the expected benefit and quality of life data from each drug approved based on secondary data and the listed price at the time of the approval, for a comparative cost-effectiveness analysis between all agents and the recently recommended national willingness-to-pay threshold (WTP), established by CONITEC (Brazilian Health Technologic Assessment Agency) for incorporation in the Brazilian Public Health System (SUS). RESULTS: Twenty-one first-in-class or newer indications were identified in the ANVISA database. Fifty-seven percent of the drug approvals were based only on progression-free survival data (PFS) and not on overall survival, mostly with modest benefit (median PFS = 5.4 months), with some notable exceptions, such as the anti-HER2 agents Pertuzumab, Trastuzumab-Deruxtecan for HER2-low disease and the CDK (cyclin-dependent kinase) inhibitors Abemaciclib and Ribociclib, all of which provide clinically relevant expected survival gain. The cost-effectiveness analysis showed us that all drugs evaluated were highly above the CONITEC recommended WTP for incorporation (1 to 3 GDP-per capita or 7,507.16 – 22.521,48 US dollars). CONCLUSIONS: Our study adds on the topic of cancer treatment affordability in middle and lower-income countries and underscore the necessity to negotiate fair pricing for cancer medicines in these regions, prioritizing high value drugs and also establish a more realistic WTP threshold, in order to provide broader access to much needed innovative cancer medications, especially in the public health system perspective.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-ispor-2023132998-pdf.pdf?sfvrsn=fb5c215_0","title":"POSTER ISPOR 2023132998.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132998","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimating Lifetime Benefits of Optimizing Secondary Preventive Treatment for Atherosclerotic Cardiovascular Disease","id":"35afeb13-4fac-4899-94a9-5d8113abfea8","sessionCode":"MSR1","topDisplay":"Gill J, Miracolo A, Politopoulou K, Jayawardana S, Apostolou EA, Carter AW, Kanavos P London School of Economics and Political Science, London, UK","locationCode":"5047","description":"\r\n\t<div>OBJECTIVES: Cardiovascular disease (CVD) is the primary cause of mortality globally, costing the EU economy more than €200 billion annually. Whilst medical guidelines for the secondary prevention of CVD recommend thresholds for risk factor treatment, there is insufficient achievement of therapeutic goals. We targeted a literature gap to estimate lifetime benefits achievable via optimization of secondary preventive treatment of atherosclerotic CVD (ASCVD) patients, providing an estimation of anticipated benefits in population health (country level). METHODS: The simulation exercise used an existing analytical framework and the SMART-REACH survival model. A literature review identified CVD risk factors and ASCVD prevalence in EU4, Denmark, Poland, and the UK. A multivariable regression, which modelled treatment scenarios, was developed, giving baseline (risk free) event-free survival at 1-year. The model was replicated to generate coefficients for all risk-factors – 1-year survival estimates were based on all the risk factor inputs. RESULTS: Increasing ‘treatment coverage’ of hypertension and hyperlipidaemia from 43% to 70% and quitting smoking in the ASCVD population could lead to 38,288 life years gained per year across 7 countries. Broken down as: hypertension: 20, 215.1, 338.9, 352.7, 132.5, 161.7 and 225.7; hyperlipidaemia: 174.3, 1901.1, 2955.2, 2035.8, 1157.6, 713 and 717.4; smoking cessation: 424.6, 3916.7, 7193.7, 6421.3, 2821.2, 2362.5 and 4047.9 in Denmark, France, Germany, Italy, Poland, Spain and the UK. Enhancing diabetes treatment coverage could save an additional 56,066 years. In all countries, the combined effect of treatment (hypertension, hyperlipidaemia & diabetes) saves more life-years than quitting smoking, highlighting the importance of identifying and treating patients with hypertension, high cholesterol and diabetes. CONCLUSIONS: This unique approach develops estimates that can feed into strategies for research and policy for secondary prevention of CVD in Europe given the thousands of annual life years that could be accrued and the impact on quality-adjusted life expectancy, productivity and other dimensions of value.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gillmsr1poster130444-pdf.pdf?sfvrsn=b663f14b_0","title":"ISPOREurope23_Gill_MSR1_POSTER130444.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130444","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Potential Budget Impact of Negative Pressure Wound Therapy with Instillation and Dwell Time Versus Control Therapies for Wound Management in Mexico","id":"da7ca7d9-a565-45c3-a71d-5d87a3862644","sessionCode":"MT11","topDisplay":"Campos D1, Vega Hernandez D2, Palka-Santini M3, Siabro V4, Topachevskyi O5 13M Health Care, Pavas, Costa Rica, 23M Health Care, Ciudad de México, MEX, Mexico, 33M Deutschland GmbH, Neuss, NW, Germany, 4Digital Health Outcomes, Kiev, Ukraine, 5Digital Health Outcomes, Kyiv, Ukraine","locationCode":"5035","description":"\r\n\t<div>OBJECTIVES: This study assessed the potential budget impact of adopting negative pressure wound therapy with instillation and dwell time (NPWTi-d) versus commonly used alternative therapies (control) for the adjunctive management of complex chronic and acute wounds in Mexico. METHODS: The per patient budget impact of implementing NPWTi-d was estimated from a public hospital perspective using a health economic model. Clinical parameters were derived from a published systematic review/meta-analysis of 13 comparative studies comprising 720 patients. The number of wound-related operating room visits was 1.77 for NPWTi-d and2.69 for the control group. Length of therapy was 9.88 versus 21.80 for NPWTi-d and control, respectively. Overall costs were estimated as the sum of three main components: cost of hospital stay for duration of therapy, cost of therapy (device, dressings, canisters and/or instilled solutions), and cost of operating room (OR) associated with excisional debridement. One way sensitivity analysis (OWSA), using a +/-20% value variation for all model parameters and breakeven point analysis were used to support the model predictions. Local costs of the control therapies and materials cost were used for the calculation. The hospital costs used were from the groups related to the diagnosis (GRD-IMSS) 2017. The effect of length of therapy variation for NPWTi-d was explored through a breakeven point analysis. RESULTS: The estimated total cost reduction using NPWTi-d was $15,246 (40.7% ) for Mexico. OWSA shows that the main cost-drivers are the OR debridement events and the OR debridement costs. Variations of a +/-20% around the input values do not impact the potential savings trend. A breakeven point analysis shows that the NPWTi-d length of therapy could be increased by up to 2.96 times for Mexico. CONCLUSIONS: The use of NPWTi-d could have a positive impact on hospital budgets for all public hospitals on Mexico.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023-ispor-europepotential-budget-impact-of-npwt-with-instillation-in-mexico-17oct2023-129331-pdf.pdf?sfvrsn=1d8decba_0","title":"2023 ISPOR Europe_Potential budget impact of NPWT with Instillation in Mexico (17oct2023) 129331.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129331","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Development of a De Novo Model to Assess the Cost-Effectiveness of a New Treatment Option for Patients With Primary Immunoglobulin a Nephropathy (IgAN)","id":"6aa92dbe-6414-4fcf-acfe-5da2b1cb8930","sessionCode":"EE119","topDisplay":"Davies N1, Dickinson O1, Downward L2, Pitcher D2, Gale D3, Ioannou P1, Kipentzoglou K4, Crabtree M5 1Mtech Access Ltd, Bicester, UK, 2UK Renal Registry, Bristol, BST, UK, 3University College London, London, England, UK, 4Britannia Pharmaceuticals Ltd, Reading, England, UK, 5STADA, Reading, RDG, UK","locationCode":"2049","description":"\r\n\t<div>OBJECTIVES: Immunoglobulin A Nephropathy (IgAN) is a rare progressive chronic kidney disease (CKD). Patients with IgAN are typically younger, less comorbid, and may experience more rapid disease progression than patients with CKD. The nature of disease progression necessitates surrogate endpoints to assess long-term efficacy of IgAN treatments on late-stage disease progression. The first disease-specific therapy for IgAN received marketing authorisation in the UK in 2023 (Nefecon®, targeted-release budesonide). No IgAN-specific UK cost-effectiveness analyses have been published. A UK-specific cost-effectiveness model was developed to assess treatments for IgAN. METHODS: A systematic review of IgAN economic literature informed the model structure, assumptions, and data sources for a UK-specific model. IgAN real-world evidence (RWE) from the UK National Registry of Rare Kidney Diseases (RaDaR) was used to address key evidence gaps. RESULTS: A de novo Markov cohort model, including eight health states and an absorbing mortality state, was developed. The model’s health states were primarily defined by CKD state (eGFR levels, a clinically-accepted measure of kidney function). Patient change in eGFR obtained from clinical trial data informed the transitions between CKD 1–4. A Kaplan-Meier curve estimating the probability of progressing to end-stage renal disease or mortality over time was obtained from UK RaDaR and extrapolated to estimate long-term transitions from CKD 4 to CKD 5. For the intervention arm, a hazard ratio calculated using published data linking relative changes in 1-year eGFR slopes to long-term clinical outcomes was applied to the Kaplan-Meier curve. Standardised mortality ratios from UK RaDaR further informed the risk of mortality from all CKD stages and dialysis. CONCLUSIONS: Model strengths include the use of clinical trial data, UK-specific RWE, and expert validation. Uncertainty regarding the representativeness of CKD data for patients with IgAN, small patient samples, and use of surrogate endpoints for disease progression may warrant further exploration.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23crabtreeee119poster132901-pdf.pdf?sfvrsn=3dd8a0f4_0","title":"ISPOREurope23_Crabtree_EE119_POSTER132901.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132901","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Emerging Biomarkers in Metastatic Castration Resistant Prostate Cancer As Evaluated in Early Phase Clinical Trials: A Targeted Literature Review","id":"c9f16e01-4e02-4330-96a0-5df2cf94b857","sessionCode":"SA7","topDisplay":"Lee L1, Jones M2, Timmel EE3, Masten L3, Liu L4, Andreu-Vieyra C5, Serfass L6 1Pfizer, Inc., New York, NY, USA, 2Evidera, Inc., Toronto, ON, Canada, 3Evidera, Inc., Waltham, MA, USA, 4Pfizer, Inc., San Diego, CA, USA, 5Pfizer, Inc., Collegeville, PA, USA, 6Pfizer, Inc., Paris, France","locationCode":"7013","description":"\r\n\t<div>OBJECTIVES: A targeted literature review was conducted to investigate the most recent emerging trends in biomarkers evaluated in metastatic castration resistant prostate cancer (mCRPC) clinical trials. METHODS: Electronic databases (Embase, MEDLINE, Cochrane Library), ClinicalTrials.gov, and key English-language conference proceedings (2019 – 2022) were reviewed. Phase 1 or 2 trials of systemic treatments that utilized biomarker data in correlative analyses of pharmacokinetic, efficacy, and/or safety outcomes in mCRPC were included. Biomarker data was descriptively compared between trials published in two time periods (Period 1: 1/1/19 – 12/31/20, Period 2: 1/1/21 – 11/30/22). RESULTS: A total of 55 trials were included (n=30 in Period 1, n=25 in Period 2). Androgen receptor (AR) aberrations were the most commonly reported among all biomarkers in both Periods (43% and 48%, respectively). Comparing Period 1 and 2, there was an increase in the use of biomarkers related to DNA damage and repair (DDR) deficiencies (30% to 48%, respectively), phosphoinositide 3-kinase (PI3K)/protein kinase B (AKT) pathway activation (17% to 32%, respectively), and tumor protein 53 (TP53) mutations (10% to 28%, respectively). Across all studies, biomarkers related to tumor mutational burden, microsatellite instability status, or immune markers were studied mostly in association with immune modulators. None of 55 trials correlated biomarkers to pharmacokinetic or safety outcomes, but 51% reported survival outcomes, which were primarily correlated to AR and DDR deficiencies. Of these, 64% quantified this association statistically. CONCLUSIONS: While AR pathway biomarkers remained the most common in early mCRPC trials across both time periods, the use of DDR, PI3K/AKT, and TP53 biomarkers increased from Period 1 to 2. Survival outcomes correlated to AR and other biomarkers were also reported in over half of the early mCRPC trials.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/biomaker-tlr-ispor-eufinal130708-pdf.pdf?sfvrsn=4601439b_0","title":"Biomaker TLR ISPOR EU_Final130708.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130708","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization Among Patients with Transfusion-Dependent Beta-Thalassemia in France","id":"57b8c216-3ac9-4f23-ac7e-5e175ea727db","sessionCode":"EE109","topDisplay":"Baldwin J1, Udeze C1, Li N1, Boulmerka L2, Dahal L1, Pesce G3, Quignot N4, Jiang H3, Galacteros F5 1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Formerly of Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 3Certara France, Paris, France, 4Certara France, Paris, 75, France, 5Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, Créteil, France","locationCode":"2046","description":"\r\n\t<div>OBJECTIVES: Patients with transfusion-dependent β-thalassemia (TDT) have reduced or absent β-globin production and require regular red blood cell transfusions (RBCTs) for survival. This study describes the healthcare resource utilization (HCRU) associated with TDT in France. METHODS: This longitudinal, retrospective cohort study utilized the French National Health Data System database (SNDS, système national des données de santé). Patients were identified by having an inpatient claim or registration in the long-term condition database (ALD, affection longue durée) with a diagnosis of β-thalassemia between January 1, 2012, and March 1, 2019. Eligible patients with TDT were required to have ≥8 RBCTs/year in any 2 consecutive years. Patients were required to have data for ≥1 year before and after their index date (i.e., the eighth transfusion in the second year of 2 consecutive years). Patients were followed from index until death or study period end (March 1, 2020). Demographics were assessed at index. The rates of RBCTs and HCRU were summarized per patient per year (PPPY) during the follow-up period. RESULTS: A total of 331 patients with TDT were included. The mean age of patients was 26.1 years, and 164 (49.5%) patients were female. The mean length of follow-up was 4.9 years. Patients with TDT averaged 13.5 RBCTs PPPY during the follow-up period. Patients averaged 14.8 inpatient hospital admissions PPPY; of these, 13.8 were day cases and 1.0 were overnight stays lasting ≥1 day. Patients also averaged 3.3 emergency room visits, 6.3 outpatient visits, and 16.9 outpatient prescriptions (all PPPY). CONCLUSIONS: Patients with TDT in France require substantial HCRU associated with their care. HCRU is driven by frequent RBCTs, inpatient hospital admissions, and significant utilization rates of emergency room visits, outpatient visits, and prescriptions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23udezeee109posterv2129397-pdf.pdf?sfvrsn=dd5bd2bd_0","title":"ISPOREurope23_Udeze_EE109_POSTER_V2129397.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129397","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"CAR-T Cell Therapies' Pathway: Evolution of Management and of Costing Methodology in the French Cost-Effectiveness Analyses (CEA) Landscape","id":"259c833b-b7a9-4ae4-ad54-5e335fb601d0","sessionCode":"EE127","topDisplay":"Sellami R1, Sivignon M2, Doghri O1, Supiot R1 1Putnam PHMR, Paris, France, 2Putnam PHMR, Lyon, 69, France","locationCode":"2059","description":"\r\n\t<div>OBJECTIVES: The number of CAR-T cell therapies accessing the market is witnessing a great increase, inducing a regulatory landscape evolution. Practices adopted to identify and collect relevant cost inputs are evolving accordingly. Objectives of this analysis are to review relevant literature sources, enumerate CAR-T cell therapy steps and gather approaches used to estimate cost inputs of CAR-T cell therapies’ related CEA in the French health-economics landscape. METHODS: Available French efficiency opinions of CAR-T cell therapies in oncology and recent French publications on CAR-T cell pathway management were reviewed. Costing approaches were identified, compared, and enriched with requests addressed by the French HTA and with recent relevant items identified in the economic official updates. RESULTS: Seven efficiency opinions, three full papers and two posters were reviewed. CAR-T pathway steps associated to costs included: CAR-T eligibility, leukapheresis, bridging and lymphodepletive chemotherapies’ administration, CAR-T infusion, hospital-stay and post-infusion follow-up, hospital discharge either to patient-hotel or to rehabilitation center, management of adverse events specific to CAR-T (AESC). Leukapheresis was costed using Disease Related Group (GHM) 28Z16Z. Inpatient and outpatient chemotherapy administration were costed using GHM 28Z07Z and 17M06 respectively. CAR-T infusion was costed using GHM 27Z03 initially then 17M15. A new act FGLF671 specific to CAR-T infusion was introduced in 2022. Cost of CAR-T storage and of hospitals’ training were considered from collective perspective only. A 15000€ complementary technical fee was introduced in 2021 and updated in 2023. While grade 3-4 adverse events were usually included in CEAs, when available, grade 1-2 AESC and their associated costs were requested to be included. CONCLUSIONS: This review helped elaborating a costing approach of CAR-T patients’ management in France considering the relevant publications available and reflecting the costs evolution over time. It remains obvious that real-world studies are the most accurate sources to document the costs related to CAR-T patients’ management.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/marine-sivignon2023-car-t-cell-therapies-pathway-evolution-of-management-and-of-costing-methodology-in-the-french-cost-effectiveness-analyses-cea-landscape131111-pdf.pdf?sfvrsn=5a6a54e5_0","title":"Marine Sivignon_2023_ CAR-T Cell Therapies' Pathway Evolution of Management and of Costing Methodology in the French Cost-Effectiveness Analyses (CEA) Landscape131111.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131111","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Review of NICE Submissions for Medical Technologies in the UK: Key Trends over the Last 2 Years","id":"d14bf07b-e03f-43f7-863c-5e97fb0353ec","sessionCode":"MT16","topDisplay":"Ioannou P, Gillard S Mtech Access Ltd, Bicester, UK","locationCode":"5042","description":"\r\n\t<div>OBJECTIVES: To review submissions to the Medical Technologies Evaluation Programme (MTEP) of NICE over the past 2 years to assess the proportion of devices receiving positive recommendations and to identify the key factors leading to a negative recommendation. METHODS: The NICE database was searched for published medical technologies guidance from June 2021 to June 2023 (searches conducted 23rd June 2023). We interrogated the submissions and extracted key information including device name and characteristics, indication, recommendation, and key criticisms from the NICE committee meeting. RESULTS: 26 MTEP guidance documents were published over the 2-year period; 17 were new guidance documents and 9 were guidance documents that had been updated following a review process. 2 submissions were for digital apps, 7 submissions focused on devices for identification or prevention of disease, and the remaining 17 focused on devices to treat a condition or aid a procedure. Of the 26 MTEP decisions, 13 devices were recommended in full (50%), 5 were recommended in patient subgroups or recommended with a requirement for ongoing research (19%) and 8 were not recommended (31%). Lack of good quality evidence was cited as the main reason for a negative recommendation as the patient and healthcare benefits were uncertain and could not be proven. Additional trials or the collection of real-world evidence was recommended by NICE in these situations. For the 9 devices where guidance was updated, 1 device was not recommended, 1 device was recommended in a subgroup, and 7 devices were fully recommended. CONCLUSIONS: To increase the chances of positive recommendation following MTEP, medical device manufacturers need to carefully consider the level of evidence available for their device ahead of submission. An evidence review and gap analysis, which can be facilitated via the Medtech Early Assessment tool (META), is recommended.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/persefoni-ioannou-maria-dimitrova-and-samantha-gillard132003-pdf.pdf?sfvrsn=6aeaca5a_0","title":"Persefoni Ioannou, Maria Dimitrova and Samantha Gillard132003.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132003","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Are the Hurdles Too High for Access to Gene Therapies in the UK?","id":"335d7779-f5e8-4d15-a3e6-5ee7ad672339","sessionCode":"HTA24","topDisplay":"Sideris E1, Holdgate O2, Patel A2, Rouse P2, Guest S2 1Roche Products Ltd, Herts, UK, 2Roche Products Ltd, Welwyn Garden City, HRT, UK","locationCode":"4064","description":"\r\n\t<div>OBJECTIVES: Gene therapies have the potential to deliver significant benefit to patients as well as to the NHS in the UK. However, access and reimbursement for gene therapies is inherently challenging. Given their one-off nature and high research & development and manufacturing costs, gene therapies are associated with a complex evidence base, low patient numbers, uncertainty around long-term outcomes (in some cases multiple decades), variability in patient outcomes and issues around affordability and budget impact. In this work we aimed to explore whether access to gene therapies in a cost-effectiveness market like the UK is feasible via the standard health technology assessment (HTA) routes. METHODS: We undertook a review of the UK reimbursement decisions for gene therapies over the last 5 years (2008-2023). In addition, we performed a review of NHS England ‘smart deals’ since 2018. We sought to identify hurdles in reimbursement. RESULTS: There are currently five gene therapies reimbursed in the UK, all of which have benefited from the flexibilities of the NICE highly specialized technology (HST) process and the SMC ultra-orphan process. A sixth gene therapy was assessed from NICE through the STA route but was not recommended. Our review of NHS England ‘smart deals’ revealed most of these were interim funding arrangements, population health arrangements and portfolio deals - and not innovative payment mechanisms linked to outcomes - with much of the detail not in the public domain due to confidentiality agreements. CONCLUSIONS: All gene therapies recommended within the UK required specialized ultra-orphan routes for HTA in combination with tailored commercial agreements. In the absence of a gene therapy specific HTA route, assessing via NICE HST and SMC ultra-orphan processes are paramount to bringing these innovations to the UK population.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23siderishta24poster130594-pdf.pdf?sfvrsn=5a8698bf_0","title":"ISPOREurope23_Sideris_HTA24_POSTER130594.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130594","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Modelling Challenges in Geographic Atrophy","id":"15df3cd9-3a1d-4e16-b803-5f714a8cc36d","sessionCode":"MSR18","topDisplay":"Intorcia M1, Hill S2, Longworth L3, Sarda SP1, Oluboyede Y4, Moor H2 1Apellis Pharmaceuticals, Waltham, MA, USA, 2Putnam PHMR Ltd., London, LON, UK, 3Putnam PHMR Ltd., London, UK, 4Putnam PHMR Ltd., Newcastle Upon Tyne, NT, UK","locationCode":"5060","description":"\r\n\t<div>OBJECTIVES: Geographic atrophy (GA) is the advanced form of dry age-related macular degeneration (AMD) and manifests as central visual loss and impairs health-related quality-of-life. Our objective was to review existing literature for guidance on developing cost-effectiveness (CE) models for GA. METHODS: A targeted literature review was conducted to identify clinical trial reports, commentary papers, and methodological articles on GA populations and economic models of populations with either GA or similar conditions (e.g., wet AMD; restricted to publications since 2020 in wet AMD). We searched MEDLINE, Embase, and PubMed and conducted a hand-search of health technology appraisals (HTAs) published by the National Institute for Health and Care Excellence (NICE). RESULTS: No GA models were identified. Seventeen studies were included, among them were six wet AMD modelling studies, three NICE HTAs, and one methodological article. All included modelling studies used Markov state-transition cohort models. Several identified limitations of the wet AMD models were their lack of applicability to GA. Limitations were also related to model structure and input parameters. Intrinsic clinical differences between GA and wet AMD, such as high levels of heterogeneity in prognostic factors for visual function, a more gradual disease progression than wet AMD, and limited possibility of vision improvement, limit the applicability of existing model structures to the GA population. CE models for the GA population are needed. Given the nature of GA disease progression, key challenges of developing robust CE models include extrapolation of long-term clinical and cost outcomes beyond clinical trials and establishing and validating links between structural and visual function outcomes and health-related utilities. CONCLUSIONS: There are no existing GA CE models. Existing wet AMD CE models have limited applicability to GA. The lack of real-world data for GA presents a daunting challenge to develop a long-term CE model.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132199","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Construct Validity of a New Migraine Satisfaction with Treatment Questionnaire","id":"89ba967f-3a7f-4494-a194-5fe6a6ee49d9","sessionCode":"PCR12","topDisplay":"Ruiz MA1, Moya MC2, Soto J3, Rejas J1, Gago-Veiga A4, González-García N5, Díaz de Terán J6, Heredia-Rodríguez P4, Camiña J7, García-Azorín D8, Giné-Ciprés E9, González-Quintanilla V10, Torres-Ferrús M9, Armada B3 1Universidad Autónoma de Madrid, Madrid, Madrid, Spain, 2Pfizer Spain, Alcobendas, Madrid, Spain, 3Pfizer Spain, Madrid, Madrid, Spain, 4University Hospital La Princesa, Madrid, Madrid, Spain, 5Hospital Clínico San Carlos, Madrid, Madrid, Spain, 6University Hospital La Paz, Madrid, Madrid, Spain, 7University Hospital Son Espases, Baleares, Baleares, Spain, 8Hospital Clínico Universitario de Valladolid, Valladolid, Spain, 9University Hospital Vall d'Hebron, Barcelona, Cataluña, Spain, 10University Hospital Marqués de Valdecilla, Santander, Cantabria, Spain","locationCode":"6018","description":"\r\n\t<div>OBJECTIVES: The aim of this study is to test the content validity of a new questionnaire measuring satisfaction with Treatment in migraine patients, the MISSAT-Q. METHODS: After thorough scientific literature review and grounded in the SATMED-Q satisfaction model, a total of 35 items were produced to build up the questionnaire. Eight dimensions were defined to structure the questionnaire: Treatment effectiveness in crisis and in prevention, side effects, convenience, impact on Health-Related-Quality-of-Life (HRQoL), monitoring, emotions, and overall satisfaction. An expert panel composed by 3 clinicians, 1 nurse, 3 pharma-economists, 1 expert patient and 1 methodologist supervised item generating process. Thirteen content-specialists in migraine (8 clinicians, 2 nurses, 2 health-outcomes-research specialists and 1 psychologist) valued each of the proposed items in all 8 dimensions defined by the questionnaire (1=measured, 0=unsure, -1=not measured). Item-Domain congruence was measured using the Hambleton-Rovinelli (1976) procedure for assessing item validity by inter-rater agreement. RESULTS: All items obtained a positive score in the theoretical intended dimension, ranging between 0.52 and 0.92 (88%≥0.70). Most items obtained lower and negative scores in all other dimension. The following pattern was observed: The overall satisfaction dimension tended to obtain low positive scores for most items. Similarly, HRQoL dimension tended to obtain moderate positive scores on most items, particularly for side-effect, prevention and emotion items. Content-specialists agreed on the assignment of items to their construct dimension as the main measurement dimension. Albeit two dimensions gathered some minor positive values from other theoretic dimension: overall satisfaction, prevention and emotions. This result was expected since the treatment satisfaction construct assumes dimensions to be conceptually correlated. CONCLUSIONS: Results from the item-Domain congruence task evidence on the validity of most items proposed to measure the Migraine Treatment Satisfaction concept structure, pointing out particular items which may be problematic in further validation steps, like confirmatory factor analysis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor2023misatqpcr12129605-pdf.pdf?sfvrsn=e9389978_0","title":"ISPOR_2023_MISATQ_PCR12129605.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129605","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Temporary Soft Tissue Coverage in Burn Patients - Which Materials Are Used - and By Whom? Analysis of German Billing Data","id":"f8a83d50-de18-47b9-8a1a-6069f71ef1bc","sessionCode":"EPH44","topDisplay":"Wahler S1, Koll C2, Müller A3, Seyed-Abbaszadeh P4 1St. Bernward GmbH, Hamburg, Germany, 2Diabetes Praxis Blankenese, Hamburg, Germany, 3Analytic Services GmbH, Munich, Germany, 4Hamburg University, Hamburg, Germany","locationCode":"3044","description":"\r\n\t<div>OBJECTIVES: Numerous materials are available for temporary soft tissue coverage in burn patients. The German system classifies eight different groups. On the basis of billing data, we analyzed what materials were used preferentially in 2021 and whether age group or gender differences can be found. METHODS: Evaluation of the billings of all German hospitals about materials of temporary soft tissue coverage exclusively for burn wounds with data from the Institute for the Hospital Remuneration System and the Federal Statistical Office. RESULTS: In 2021, 12,764 temporary soft tissue covers were coded for burn wounds. Of these, 7,263 (56.9%) in children <18y and 1,446 (11.3%) were in adults >60y. Share of used materials was (in parentheses: In patients <18y and >60y): hydrolytically absorbable membrane 62.0% (63.0%; 53.5%), alloplastic materials 28.5% (30.0%; 31.5%), allogeneic material 4.0% (1.9%; 9.1%), xenogeneic material 3.0% (2.4%; 3.0%), allogeneic skin substitute 1.6% (1.6%; 1.6%); activated keratinocytes 0.0% (0.0%; 0.1%); combination of different materials 0.3% (0.2%; 0.8%); and other materials 0.5% (0.8%; 0.4%). Average age of the patients was 22.7y (median 10y). For the individual materials, the results were (median in parentheses): Hydrolytically resorbable membrane 21.6y (9y), alloplastic materials 22.3y (6y), allogeneic material 39.8y (42y), xenogeneic material 28.3y (22y), allogeneic skin substitute 22.4y (7y); activated keratinocytes 37.6y (35y); combination of different materials 33.1y (23y); and other materials 11.9y (3y). Coverages in male patients accounted for 60.6% of all coverages. Males were also preferred in combinations (69.4%) and allogeneic materials (66.6%). Other materials were used preferably in female patients (61.8%). CONCLUSIONS: Analysis of temporary coverage for burns using billing data is feasible. Half of all such applications were coded in children younger than 10 years in 2021. Hydrolytically absorbable membranes are preferred in all age groups and genders. This analysis can only reflect preferences of practitioners, independent of clinical evidence and efficacy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130263","diseases":[{"id":"3f994852-a0d4-4706-9665-e4d867006d9a","parentId":"00000000-0000-0000-0000-000000000000","title":"Injury & Trauma","urlName":"injury-trauma"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Methodological Aspects of a COVID-19 Vaccine Discrete Choice Experiment Survey in Canada, Germany, UK, and US General Populations","id":"b2279bf9-d0c3-430c-bb1e-6096abcc5150","sessionCode":"MSR21","topDisplay":"Sri Bhashyam S1, Lewis HB2, Rousculp M3, Shane LG4, d la Cruz M5, Galinsky J2, Demchuk K6, Lehmann C7, Waite N8, Lazarus J9, Bonanni P10, Salisbury D11 1ICON Plc, Reading, RDG, UK, 2ICON Plc, Reading, Reading, UK, 3Novavax Inc., Gaithersburg, MD, USA, 4L.G. Shane, New York, NY, USA, 5ICON Plc, Raleigh, NC, USA, 6ICON Plc, Burlington, Canada, 7University of Cologne, Albertus-Magnus-Platz, 50923 Köln, Germany, 8University of Waterloo, Waterloo, ON, Canada, 9ISGlobal Barcelona Institute for Global Health, Barcelona, Barcelona, Spain, 10University of Florence, Italy, 50121 Firenze, Florence, Italy, 11Programme for Global Health, Royal Institute of International Affairs, Chatham House, London, UK","locationCode":"5063","description":"\r\n\t<div>OBJECTIVES: COVID-19 vaccine preferences can influence vaccine coverage. Discrete choice experiments (DCEs) can be used to elicit tradeoffs. DCE development requires evidence-based attribute selection and validation of understanding with target audiences. An online DCE survey was developed to assess preferences and systematic tradeoffs for COVID-19 vaccines in Canada, Germany, the UK, USA, and Italy. METHODS: First, a targeted literature review and interviews with country-level experts were conducted to inform the attributes and levels and the DCE survey. Secondly, pretest interviews were conducted in Canada, Germany, the UK, and USA (March 2023) to evaluate respondents’ understanding of the survey and ability to make tradeoffs as expected. Thirdly, a soft launch of the survey was conducted in Canada, the UK, and USA (April‒May 2023) to evaluate the experimental design. Self-reported antivaccinationists were excluded. RESULTS: Six pretest interviews were completed in each country (N=24). Mean age was 43.7 years; 50% were women. Participants’ top four priorities were vaccine protection against COVID-19, serious side effects, protection against severe COVID-19, and common side effects, followed by vaccine type and timing of COVID-19/influenza vaccines. These interviews validated the importance and understanding of key attributes driving people’s choices, and feedback was used to improve attribute description clarity. The soft launch of the survey included 259 respondents; 9.7% were partially vaccinated/unvaccinated and 90.3% were fully vaccinated. Mean age was 69.3 (SD=8.59) years; 60% were men. No changes to the experimental design were deemed necessary. Full survey results will be available in autumn 2023. CONCLUSIONS: This study highlights the importance of key vaccine attributes driving people’s choices and evaluates people’s characteristics that may influence vaccine preferences. The DCE will be fielded in Canada, Germany, the UK, USA, and Italy (up to 2,500 participants) to increase the understanding of COVID-19 vaccine preference and hesitancy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23rousculpmsr21poster132392-pdf.pdf?sfvrsn=d2b9e21b_0","title":"ISPOREurope23_Rousculp_MSR21_Poster132392.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132392","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Relationship Between the Directness of the Evidence and Identification of Added Therapeutic Value: An Analysis of HTA Procedures","id":"46c8c80b-6a5b-4c7b-8a41-60b556bfecc5","sessionCode":"HTA46","topDisplay":"Ribeiro C, Penedones A, Mendes D, Alves C University of Coimbra, Coimbra, Portugal","locationCode":"4079","description":"\r\n\t<div>OBJECTIVES: Relative effectiveness assessment procedures are a critical step of HTA. Some evaluation procedures may need to resort on adjusted indirect comparisons, such as network meta-analysis (NMA), to assess the therapeutic value of the pharmacological intervention. The aim of this study is to assess if there is a relationship between the directness of evidence supporting the relative effectiveness assessments and the identification of added therapeutic value (ATV). METHODS: Relative effectiveness assessments produced between 2018 and 2021 by 6 HTA bodies (NICE [England and Wales], SMC [Scotland], HAS [France], AEMPS [Spain], SiNATS [Portugal] and IQWIG [Germany]) were reviewed. Data on the pharmacological intervention, comparator(s), data sources and conclusions of therapeutic value was retrieved. Direct evidence reflects evaluations informed by the same comparator(s) and studies that integrated the clinical development program of the pharmacological intervention, representing direct links to assess the health outcomes. Indirect evidence represents the opposite. The qui-square test was applied to explore a possible relationship between the directness of the evidence and the identification of ATV. RESULTS: Of the 987 relative effectiveness assessment procedures analyzed, 717 were exclusively supported by the direct evidence. Of those, 531 (74.06%) concluded for ATV, 63 (8.79%) for equivalence, 13 (1.81%) were inconclusive, and 110 (15.34%) did not recognize therapeutic value. A total of 270 procedures were informed by indirect evidence. Of those, 199 (73.70%) concluded for ATV, 31 (11.48%) for equivalence, 12 (4.44%) were inconclusive, and 28 (10.37%) did not recognize therapeutic value. The chi-square test did not find a relationship between the directness of the evidence and identification of ATV, except for IQWIG reports only (indirect evidence was related with less chance of ATV identification). CONCLUSIONS: These results suggest that there is no relationship between the directness of the evidence supporting the relative effectiveness assessment procedures and the identification of ATV.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/alvesposter-2ispor129479-pdf.pdf?sfvrsn=f6d0323f_0","title":"Alves_Poster 2_ISPOR129479.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129479","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Quality of Life of Moroccan Children with Rare Diseases: Evidence from Real-World Data","id":"52fac747-1fc7-43ad-8e9d-60d20d13404d","sessionCode":"PCR249","topDisplay":"El Hani M1, Ratbi I2, Jdioui W2, Zerkaoui M2, Rifai L2, Cherrah Y1, Thimou Izgua A3, Serragui S1 1Pharmacoepidemiology & Pharmacoeconomics Research Team, Laboratory of Pharmacology and Toxicology, Faculty of Medicine and Pharmacy, University Mohammed V of Rabat, Rabat, Morocco, 2Unity of Medical Genetics, Children’s Hospital, Ibn Sina University Hospital, University Mohammed V of Rabat, Rabat, Morocco, 3Center of consultations and external explorations, Children’s Hospital, Ibn Sina University Hospital, Rabat, Morocco","locationCode":"7025","description":"\r\n\t<div>OBJECTIVES: Rare diseases have a substantial impact on the Quality of Life (QoL) of patients, yet there is limited knowledge about the QoL of Moroccan patients with rare diseases. Our objective was to evaluate the influence of rare diseases on the overall QoL of Moroccan children with such conditions. METHODS: We conducted a prospective descriptive study from June 17th, 2023, to july 19th, 2023. The study population consisted of children with rare diseases who sought genetic consultation at the Center of Consultations and External Explorations of the Children's Hospital at Ibn Sina University Hospital in Rabat, Morocco. The study sample included children who met the inclusion criteria and whose parents provided informed consent. Socio-demographic data were collected, and QoL was assessed using the PedsQL TM 4.0 Generic Core Scales. RESULTS: The study included a total of 16 patients aged 2 to 18 years. The mean age of the participants were 2.31±1.25 years, with 43,8% falling into the toddler age group. Out of the total patients, 62.5% (n=10) were male. Additionally, 43.8% (n=7) of the children were consanguineous. 11 parents completed the PedsQL as proxy-reporters for their children. The patients exhibited lower scores in physical (mean: 49.80), emotional (mean: 63.43), social (65.31), and school functioning (mean: 31.42). When comparing QoL scores between girls and boys, boys had lower scores on all PedsQL subscales except for emotional functioning. CONCLUSIONS: QoL is recognized as a crucial measure of well-being. This study provided evidence of notable declines in QoL among children with rare diseases. These findings should assist health policymakers in providing targeted and improved support for children affected by rare diseases.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23el-hanipcr249poster131316-pdf.pdf?sfvrsn=3779a209_0","title":"ISPOREUROPE23_EL HANI_PCR249_POSTER131316.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131316","diseases":[{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Hospital Capacity Tool to Assess the Potential Impact of Using a Fixed Dose Combination of Pertuzumab/Trastuzumab for Subcutaneous Injection Versus Intravenous Pertuzumab/Trastuzumab in HER2+ Breast Cancer","id":"66e691e4-5c9d-416f-a839-610c8400a9d2","sessionCode":"HSD11","topDisplay":"Cunha A Roche Farmacêutica Química Lda., Amadora, Lisboa, Portugal","locationCode":"4019","description":"\r\n\t<div>OBJECTIVES: Globally, the cancer burden continues to grow, exerting a tremendous strain on individuals and healthcare systems. The aim of this study is to simulate the use of a capacity tool to calculate the impact of using a fixed-dose combination of pertuzumab and trastuzumab for subcutaneous injection (PH FDC SC) versus the intravenous options (PIV + HIV) on the hospital and patients. METHODS: We have developed a Microsoft Excel® tool and inputted information about a hypothetical hospital (number of patients, infusion chairs and working hours). We used data from the literature to input the active time spent by HCPs on drug preparation, administration and monitoring and the total amount of time spent by patients for administration and observation at the hospital. RESULTS: In this scenario, we observed that the time spent by HCPs to prepare the medication and to administer and monitor all patients during one year of treatment is about 2 times greater with PIV + HIV compared to PH FDC SC. The use of PH FDC SC can save 5,8% of the hospital’s total capacity and allows to treat more patients per infusion chair per day. Furthermore, patients treated with PH FDC SC can stay around 90% less time in the hospital compared to patients receiving PIV + HIV. CONCLUSIONS: This capacity tool demonstrates the impact of using different treatment options for HER2+ breast cancer on the hospital capacity and human resources customized to each hospital reality. The simulation demonstrated that PH FDC SC has potential to provide substantial timesavings for the pharmacy, the nurses and the patients due to its simpler preparation and considerably shorter duration of administration and observation periods. In conclusion, this capacity tool can inform hospitals about the value of different treatments besides efficacy and safety, demonstrating the impact on the hospital day-to-day management and on patients’ time.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23cunhahsd11poster129659-pdf.pdf?sfvrsn=b77bf50b_0","title":"ISPOREurope23_Cunha_HSD11_POSTER129659.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129659","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Simulation Study to Examine the Influence of Varied Standard Deviation of Covariates across Trials on Population Adjusted Indirect Treatment Comparisons","id":"064943ad-57e4-4beb-bda8-623ebe47279d","sessionCode":"MSR31","topDisplay":"Essink E1, Tremblay G2, Groff M3 1Cytel, Rotterdam, ZH, Netherlands, 2Cytel, Waltham, MA, USA, 3Cytel Canada Inc., Guelph, ON, Canada","locationCode":"5072","description":"\r\n\t<div>OBJECTIVES: This study assessed the influence of varying standard deviations across trials on the performance of Simulated Treatment Comparisons (STC) and Matching Adjusted Indirect Comparison (MAIC) methods. STC and MAIC compare treatment efficacy in health technology assessments when direct head-to-head trials are unavailable. These methods account for baseline characteristic differences, but the degree of imbalance can affect the outcomes regression model. METHODS: Patient level data were simulated using ‘wakefield’ package in R. The baseline characteristics (study size, mean age, standard deviation of age) and association parameters were predetermined. The standard deviation of age in the comparators trial was set to 25%, 50%, 100%, 150% and 200% that of the standard deviation of age in the index trial. Additionally, since the effect modification strength may impact the investigated relationship, the effect modification strength was increased using increments of -0.025. Each combination of standard deviation difference and effect modification strength underwent 1000 iterations. Bias was estimated as the log-odds of treatment difference calculated using STC or MAIC minus the true value, and coverage determined if the 95% confidence intervals contained the true treatment difference. Results were summarized using linear regression and means. RESULTS: For all standard deviation differences of age considered, STC outperformed MAIC both in terms of bias and coverage. No linear trends were observed in increased bias with higher standard deviation age in the comparator trial relative to the index trial (STC, p=0.900; MAIC, p=0.380). Linear trends were also absent in decreased coverage with increased standard deviation age in the comparator trial relative to the index trial (STC, p=0.659; MAIC, p=0.962). CONCLUSIONS: The performance of STC and MAIC are unaffected by increasing the standard deviation of a given covariate in the comparator trial relative to the standard deviation of the same covariate in the index trial.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133589","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"First Interim Analysis of Patient-Reported Outcomes in View Study: A Multicenter, Single-Arm, Prospective, Non-Interventional Study of Vonoprazan in Real-World Clinical Practice in China","id":"599cf666-a14e-4ba4-a831-629fc94e021b","sessionCode":"PCR5","topDisplay":"Xiao Y1, Wang Q2, Li G3, Nail A4, Song Q5, Xie L6, Chen M7 1The First Affiliated Hospital, Sun Yat-Sen University,, Guangzhou, Guangdong, China, 2Qilu Hospital of Shandong University, Qingdao, Shandong, China, 3The Affiliated Hospital of Hangzhou Normal University,, Hangzhou, Zhejiang, China, 4Takeda Pharmaceutical Company, Cambridge, MA, USA, 5Takeda Pharmaceutical Company, Shanghai, China, 6Takeda Pharmaceutical Company, Beijing, China, 7The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China","locationCode":"6012","description":"\r\n\t<div>OBJECTIVES: To evaluate changes in the quality of sleep and life of Chinese patients diagnosed with reflux esophagitis (RE) when treated with vonoprazan in real-world clinical practice. METHODS: VIEW is a multicenter, single-arm, prospective, observational study (NCT04501627). Enrolled patients were ≥18 years and were treated with 20 mg vonoprazan QD for 4 weeks (8 weeks if insufficient healing). The effectiveness of vonoprazan (overall and for patients ≥65 years) was assessed using changes from baseline to week-4 in PRO scores: Pittsburgh Sleep Quality Index (PSQI, for sleep quality and patterns); the EuroQol 5-Dimension 5-Level (EQ-5D-5L, for quality of life); and the EQ-Visual Analogue Scale (EQ-VAS; for health status). Negative differences in PSQI and positive differences in EQ-5D-5L and EQ-VAS scores represent an improvement. RESULTS: Of 1012 patients enrolled, 419 patients had a PSQI score at both baseline and week-4, and the mean±SD (95% CI) of the differences in the two scores was -0.6±2.27 (-0.83, -0.40). For 63 patients ≥65 years, the mean±SD difference was -0.5±2.47(-1.15, 0.10). The percentage (95% CI) of patients with poor sleep quality (global score >5) in the overall population decreased from 43.2% (39.01, 47.45) at baseline to 32.3% (27.88, 36.88) at week-4. In the ≥65 years age-group, this percentage decreased from 51.9% (40.36, 63.29) to 45.5% (33.14, 58.19). For 451 patients with EQ-5D-5L index scores at both baseline and week-4, the mean±SD of the differences was 0.028±0.0732 (0.0217, 0.0353). For 70 patients ≥65 years, the mean±SD of the differences was 0.041±0.072 (0.0242, 0.0587). For 452 patients with EQ-VAS scores at both baseline and week-4, the mean±SD of the differences was 5.1±11.52 (4.06, 6.19), while in 70 patients ≥65 years, the mean±SD of the differences was 8.5±14.69 (4.97, 11.97). CONCLUSIONS: Vonoprazan treatment in real-world clinical practice improved both sleep quality and quality of life of Chinese patients diagnosed with RE.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/takedaview-studyispor-euposter-draft20231020128584-pdf.pdf?sfvrsn=38b6e5f_0","title":"Takeda_VIEW study_ISPOR EU_poster_ Draft20231020128584.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128584","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Access to Orphan Pediatric and Adolescent Medicinal Products in Greece Over 4 Years (2018-2021)","id":"dfbebd31-c90d-4ae5-a30c-62b911e0b0d1","sessionCode":"HTA22","topDisplay":"Margetis A1, Kani C1, Chantzaras A1, Koutsiouris V2, Bacopoulou F1 1Health Technology Assessment and Reimbursement Committee, Hellenic Ministry of Health, Athens, Greece, 2Hellenic Ministry of Health, Athens, Attiki, Greece","locationCode":"4059","description":"\r\n\t<div>OBJECTIVES: Equal and timely access across Europe remains a long-standing challenge for orphan pediatric medicinal products, despite the existence of specific regulatory framework in the EU. The aim of the present study was to monitor access to orphan medicinal products (MPs) for children and adolescents after the establishment of the national HTA and Reimbursement Committee (January 2018-December 2021) in Greece. METHODS: Data were collected from the European Medicines Agency (EMA) and the Greek Ministry of Health websites, as well as from the Health Technology Assessment (HTA) Committee’s database. The dataset included MPs with a clear reference in their Summary of Product Characteristic (SPC) to pediatric and/or adolescent use. Data on the orphan status and the therapeutic category (ATC3) of the active substances were also collected. Median times for MPs with a pediatric/adolescent indication and according to their orphan status were calculated based on their marketing authorization date, their dossier submission to the Greek HTA Committee and their inclusion in the Positive Reimbursement List. RESULTS: Over the 4-year study period, from 54 MPs with orphan pediatric/adolescent indication submitted to EMA, 13 with orphan designation were submitted to the national HTA and Reimbursement Committee. The median time (25th – 75th percentile) from marketing authorization to national HTA submission was 365 (246-508) days for pediatric/adolescent MPs in general and 393 (203-521) days for orphan pediatric/adolescent MPs specifically. The majority of pediatric/adolescent MPs referred to immunosuppressants (ATC3: L04A) while orphans pertained to other respiratory system MPs (ATC3: R07A). CONCLUSIONS: Delays in access to pediatric/adolescent MPs may be enhanced by the presence of orphan status. Forthcoming changes in the European pharmaceutical legislation should consider reforms and incentives for pediatric/adolescent therapies. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bacopoulouhta22access-to-orphan-pediatrics133610-pdf.pdf?sfvrsn=b2a6c5d6_0","title":"ISPOREurope23_BACOPOULOU_HTA22_ACCESS TO ORPHAN PEDIATRICS133610.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133610","diseases":[{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Implicit Devaluation of Health Benefits Over Time in NICE Decision-Making Criteria: Assessing NICE’s Static Willingness to Pay Threshold in the Context of Inflation.","id":"0b149f17-69f9-44e0-8acc-63383dac405e","sessionCode":"EE152","topDisplay":"Percival C1, Worbes Cerezo M2 1Janssen-Cilag Ltd, High Wycombe, UK, 2Janssen-Cilag Ltd, High Wycombe, LON, UK","locationCode":"3000","description":"\r\n\t<div>OBJECTIVES: In 2004, The National Institute for Health and Care Excellence (NICE) acknowledged it had a willingness to pay (WTP) range of £20,000 to £30,000 per quality-adjusted life-year (QALY) gained. For almost 20 years, this threshold has remained unchanged despite inflation, resulting in a real term decline in the WTP for health benefits. This differs to other valuations of health used in UK cost-benefit analyses and has eroded incentives to launch novel technologies in the UK over time. We aimed to review and compare the application of inflation to valuations of health in UK economic evaluations and document the real terms decline in NICE’s WTP for a QALY since 2004. METHODS: A hand search of grey literature and UK government department and non-departmental public body websites was conducted to identify valuations of health used in UK economic evaluations and applied inflation adjustments. Inflation data were extracted from the Office for Budget Responsibility’s March 2023 Economic and fiscal outlook publication, and the Office for National Statistics. RESULTS: Health benefit valuations were identified for five UK government departments and non-departmental public bodies. NICE is the only identified institution that has not adjusted its valuation of health benefits for historical inflation. Importantly, from 2004 to 2022, the real value of the NICE threshold has declined by at least 30% across multiple inflation measures. CONCLUSIONS: Explicit methods for updating WTP thresholds should be established to avoid devaluations of health benefits over time due to inflation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133091","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Predicting Post-AMNOG Price for a New Product Launch in Germany","id":"a8859dd5-e472-44d5-8c54-634984d3090a","sessionCode":"HTA20","topDisplay":"Jha P1, Shah S2, Ma A3 1Eversana, Pune, MH, India, 2Eversana, Mumbai, MH, India, 3Eversana, Yardley, PA, USA","locationCode":"4056","description":"\r\n\t<div>OBJECTIVES: In Germany all new innovative medicines are subject to an early benefit assessment by the German Federal Joint Committee (G-BA) with subsequent price negotiation and optional arbitration. This study aims to explore the various data-based modeling techniques to predict post-AMNOG (Arzneimittelmarkt-Neuordnungsgeset) annual cost of treatment (COT) for oncology products. METHODS: The modeling approach utilizes historical pricing data and G-BA ratings available within Eversana’s NAVLIN database. The oncology dataset includes 321 data-points spanning 40 products and 71 indications launched from 2018-2023, that have undergone the AMNOG process and have an annual COT. Initially, a One-Way Analysis of Variance (ANOVA) was conducted on G-BA ratings with respect to annual COT. Next, machine learning predictive algorithms – including four different types of regression methods, k-nearest neighbors (KNN) and decision trees – were tested on standardized data. Models were cross-validated and trained on historical data from 2018-2020, and further tested on product launches starting 2021. Accuracy and reliability of models was assessed using adjusted R-squared (R-sq), Mean Absolute Error (MAE), Root Mean Squared Error (RMSE) and Mean Absolute Percentage Error (MAPE). RESULTS: ANOVA test established a relationship between G-BA and post-AMNOG COT (F-value 2.2107 and p-value 0.0419). On training dataset, Decision Tree model provided the best adjusted R-sq (0.901) and lowest MAPE (10.6%) scores. The gamma regression model had the lowest comparative performance based on all metrics. On test-data (2021-2022), the Huber regression model performed the best with adjusted R-sq of 0.987 and MAPE of 8.9%. The gamma model had the lowest performance, while KNN and decision tree models also performed poorly. CONCLUSIONS: The Huber Regression model displayed best performance in predicting post-AMNOG price for products launched in 2021 -22. This flexible yet rigorous framework can be modified to include more independent variables, understand their effect on launch prices and evaluate algorithms for predictive modeling of COT.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/navlin---ispor---predicting-post-amnog-price-for-a-new-product-launch-in-germany-final132171-pdf.pdf?sfvrsn=a5f0ec75_0","title":"NAVLIN - ISPOR - Predicting Post-AMNOG Price for a New Product Launch in Germany (Final)132171.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132171","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Using Automated Data Extraction Methods to Understand the Evolution of Insulin Prescribing in the UK between 2021 and 2022","id":"258b1a5d-80a1-4f49-bba4-64188ccf4bdb","sessionCode":"HSD2","topDisplay":"Qureshi A, Lucion D, Smith A, Buxton J, Scott C, Wright J Valid Insight, Macclesfield, UK","locationCode":"4013","description":"\r\n\t<div>OBJECTIVES: The landscape of insulin prescribing in the UK faces challenges due to the strain on the NHS from COVID-19, disruptions in the global supply chain, and the growing numbers of people with diabetes This research aims to evaluate insulin prescribing patterns in the UK from January 2021 to December 2022 using automated data extraction. METHODS: We utilized data from the English Prescribing Dataset (EPD), to analyze insulin prescription data over a two-year period spanning from January 2021 to December 2022. Due to the volume of data contained within the EPD, we employed an automated process to extract pertinent insulin prescriptions from the dataset. For a prescription to be classified as relevant, the data needed to include either the 'insulin type' or 'insulin product of interest' within the prescription record. RESULTS: Between January 2021 and December 2022, there was an overall increase in insulin prescription across the UK (11.8%). Across regions, the Midlands had the highest total quantity of insulin products prescribed (20.64%) followed by North East and Yorkshire (16.95%). In the UK, insulin aspart was the most prescribed product (32.0%), suggesting the largest market share within the insulin landscape. CONCLUSIONS: The trends in insulin prescribing can be attributed to several reasons including the potential backlog of undiagnosed patients due to COVID-19 and the rise post-COVID-19. Leveraging automated extraction methods and analyzing comprehensive prescription datasets provides payers with insights to allow informed decision-making regarding resource allocation to improve diabetes care and patient access.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23lucionhsd2poster132019-pdf.pdf?sfvrsn=796ec036_0","title":"ISPOREurope23_Lucion_HSD2_POSTER132019.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132019","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Inventory-Facilitated Goal Setting Interview Conducted by Disease Experts Yields a Broader Range of Goal Areas","id":"c77650ca-8c5f-4399-b089-642a125c348d","sessionCode":"CO37","topDisplay":"Cheema K1, St. Jean C2, Stanley J3, Cummine J1, Hodgetts W1, Chapman CA3, Rockwood K4, Howlett SE4, Sevinc G3 1University of Alberta, Edmonton, AB, Canada, 2University of Alberta, Stratford, AB, Canada, 3Ardea Outcomes, Halifax, NS, Canada, 4Dalhousie University, Halifax, NS, Canada","locationCode":"1001","description":"\r\n\t<div>OBJECTIVES: Goal attainment scaling (GAS) quantifies the impact of an intervention on personal goals. GAS interviewers work with patients/caregivers to set goals that are meaningful, specific, attainable, and relevant. Although interviewer expertise in the condition and use of a goal inventory are thought to improve goal setting, their impact has not been systematically investigated. Here we assessed the impacts of interviewer expertise and the use of a goal inventory on the number of goals, goal type, time to set goals, and goal quality in individuals with dyslexia. METHODS: Seventeen individuals were interviewed by two interviewers; one with expertise in dyslexia (n=9) and one without (n=8) to set 3 to 5 goals. Nine participants were provided with an inventory of 30 goal areas mapped to reading, writing, emotions and “self-advocacy and social” domains and had the option to add goals de novo. The remaining participants were not provided with an inventory. Goal quality was assessed by an independent assessor. RESULTS: While all participants set between 3 and 5 goals, the expert interviewer set more goals than the non-expert (36 vs. 27 goals). In addition, those provided with a goal inventory set more goals than those without (35 vs. 28 goals). They also set more diverse goals with 9 (25.7%) from self-advocacy and social domains compared to 2 (7.1%) without the inventory. Mean time to complete goal setting was similar for expert and non-experts (35±13.2 vs. 33±6.6 minutes). The time to complete was also similar with and without a goal inventory (36±12.2 vs. 32±8.3 minutes). Most goals (62/63) were rated as acceptable or excellent, but the expert set more goals rated as excellent (29/36) than the non-expert (6/27). CONCLUSIONS: The use of expert interviewers and a goal inventory to facilitate the goal-setting process can help clinicians and individuals identify a broader range of goal areas.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sevincco37poster-133004-pdf.pdf?sfvrsn=78b37054_0","title":"ISPOREurope23_Sevinc_CO37_POSTER 133004.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133004","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"End-of-Life Cost and Characteristics of Acute Myeloid Leukemia Decedents: A National Study From 2011-2020","id":"62a2d56e-0231-457e-b7c3-643bdd93e771","sessionCode":"EE76","topDisplay":"Han H1, Suh HS2 1Institute of Regulatory Innovation through Science, Kyung Hee University, Seongnamsi, South Korea, 2Department of Pharmacy, College of Pharmacy, Kyung Hee University, Seoul, Korea, Republic of (South)","locationCode":"2019","description":"\r\n\t<div>OBJECTIVES: This study aims to identify the economic burden associated with end-of-life care in Acute Myeloid Leukemia (AML) patients, a hematologic malignancy recognized for its substantial end-of-life costs. METHODS: This longitudinal retrospective cohort study, employing the Health insurance Review and Assessment Service databases from January 2008 to August 2020, representing over 98% of the Korean population. The incident AML patients were defined with the first diagnosis of AML (ICD-10 code: C92-95 excluding C92.1, C92.2, C92.3, C94.4, C94.5, C94.6) between 2010 and 2020, with no AML history during the 2-year washout period. The AML decedents were defined with the coded death in the insurance claims. The study defined end-of-life as the healthcare resource utilization cost in the last year of life from 2011 to 2020, and any patients without claim history for the last year of life were excluded RESULTS: A total of 12,104 AML decedents (mean age: 62.30 years) were identified, including 7,016 male patients (57.79%). Among those, 1,255 patients (10.34%) received hematopoietic-stem-cell-transplantation (HSCT), and the average Charlson Comorbidity Index (CCI) was 4.19. 54.51% of the population received treatment in tertiary hospitals, and 92.81% of population were health insurance payers. The others were under Medicaid or the Veterans’ scheme. The average end-of-life cost was $50,763 per patient, including hospitalization, emergency visits, outpatient visits, and prescribed drug, with out-of-pocket payment constituting 6.29% of the total cost. The patient with HSCT history was 2.15 times higher end-of-life costs than those without. Over the decade, the end-of-life cost saw an upward trend, increasing on average by 5% per year. In 2020, the most recent year of the study, the highest end-of-life cost reached $62,425 per patient. CONCLUSIONS: End-of-life costs were increasing about 50% over the decade. Notably, it was found that patients with AML who underwent hematopoietic stem cell transplantation (HSCT) experienced significantly higher end-of-life costs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee76session1ispor-eu2023hyunjinhan132840-pdf.pdf?sfvrsn=913b515e_0","title":"EE76_Session1_ISPOR EU_2023_Hyunjin_Han132840.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132840","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Teduglutide for Treating Short-Bowel Syndrome in Adults: A Meta-Analysis of Real-World Evidence Compared to Clinical Trial Data","id":"faa17d87-ffb8-4edd-9c8b-644028d5b599","sessionCode":"CO6","topDisplay":"Eaton J1, Harvey R2, Cain P3, Dallongeville A4, Campbell-Hill S5 1Takeda UK Ltd, London, UK, 2Cabourn Statistics, Manchester, UK, 3Takeda, UK Health Economics, London, UK, 4Takeda UK Ltd, London, London, UK, 5Takeda Pharmaceuticals UK, London, London, UK","locationCode":"1007","description":"\r\n\t<div>OBJECTIVES: Teduglutide is a treatment that allows patients with short bowel syndrome and type 3 intestinal failure (SBS-IF) to reduce their dependence on intravenous nutrition and fluids (parenteral support; PS). A pivotal randomized controlled trial (RCT) of teduglutide showed 63% of patients experienced a ≥20% reduction in weekly PS volume at 24 weeks. Individual real-world evidence (RWE) studies have reported greater and more rapid PS reductions than clinical trials, but whether this is a general trend is unknown. We tested the hypothesis that clinical outcomes with teduglutide are better in RWE than in the pivotal RCT. METHODS: A systematic literature review was conducted on 21st May 2021 to identify RWE. Inclusion criteria were adults with SBS-IF receiving teduglutide per label; abstract-only publications were excluded. Outcomes of interest were clinical response (≥20% reduction in weekly PS volume) and PS independence (100% reduction in weekly PS volume), each at 6 and 12 months of teduglutide treatment. Data were pooled and a Wald-type test was then used to compare the percentage of patients achieving these outcomes in pooled RWE vs pivotal RCT. RESULTS: Eight RWE studies were identified. There was no significant difference in rates of clinical response between RWE and RCT at either time point: 6 months: 79.8% vs 69.2%, p=0.380; 12 months: 78.3% vs 91.7%, p=0.053). However, more patients gained PS independence in RWE vs RCT at both time points: 6 months: 24.6% vs 0%, p<0.001; 12 months: 30.5% vs 5.6%, p<0.001. These trends were maintained when outlying RWE results were removed in sensitivity analyses. CONCLUSIONS: Rates of clinical response with teduglutide appear to be similar between RWE and RCT, but more patients appear to gain independence from PS in RWE than observed in the RCT. This may result from PS weaning protocols that were enforced in the RCT.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23eatonco6poster129748-pdf.pdf?sfvrsn=76e5cf19_0","title":"ISPOREurope23_Eaton_CO6_POSTER129748.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129748","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Unequal Access of Innovative Drug Products Across Canada","id":"57a3abb3-9e86-4436-bc6e-6473d08177b4","sessionCode":"HTA56","topDisplay":"Feener S, Barbeau M Bausch Health, Canada Inc., Laval, QC, Canada","locationCode":"5006","description":"\r\n\t<div>OBJECTIVES: In Canada, Health Technology Assessment (HTA) reviews are performed by the Canadian Agency for Drugs and Technologies in Health (CADTH) and the institute national d’excellence en santé et en services sociaux (INESSS), and then the file is sent to the pan-Canadian Pharmaceutical Alliance (pCPA) for negotiations of confidential reimbursement listing agreements. The decision to publicly fund drugs belongs to respective public drug plans, which can lead to unequal access across the country. The objective of this analysis was to assess if there are discrepancies in the listing status across the public drug plans for the latest innovative dermatology, metabolic, central nervous system (CNS), and respiratory products on the Canadian market. METHODS: Dermatology, metabolic, CNS, and respiratory products that reached a pan-Canadian Pharmaceutical Alliance (pCPA) agreement between January 1, 2016 and June 28, 2023 were collected. The listing status of the products on the public drug plans were recorded. An analysis of the percentage listed across drug plans was calculated using all products that had signed a pCPA Letter of Intent (LOI). RESULTS: There were 11 innovative dermatology products, 10 CNS, 8 respiratory, and 14 metabolic products with a signed pCPA agreement. Percent listing ranged from 27% of dermatology products (British Columbia) to 100% of dermatology and CNS products (several provinces). The median percent listing was 100%, 90%, 88%, and 79% for dermatology, CNS, respiratory and metabolic products respectively. The biggest gap in percent listings is in dermatology, where the Canadian median was 100% versus 27% in British Columbia. CONCLUSIONS: The analysis shows that there is unequal and unequitable access to innovative dermatology products in British Columbia in comparison to the rest of Canada and to other therapeutic areas.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23feenerhta56poster132395-pdf.pdf?sfvrsn=116adf80_0","title":"ISPOREurope23_Feener_HTA56_POSTER132395.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132395","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"DiGA in Germany: Achievable Prices After Price Negotiation or a Decision of the Arbitration Board","id":"9b64f5af-45fa-4d89-97bd-658f23a89c5f","sessionCode":"HPR40","topDisplay":"Meyer-Harms R1, Koppenhöfer S1, Pütz DC2 1Ecker + Ecker GmbH, Hamburg, SH, Germany, 2Ecker + Ecker GmbH, Hamburg, Germany","locationCode":"4009","description":"\r\n\t<div>OBJECTIVES: In Germany, people insured with the statutory health insurance are entitled to treatment with digital health applications (DiGA). The reimbursement prices of DiGA are negotiated with the National Association of Health Insurance Funds (GKV-SV) or decided by the arbitration board if the negotiation fails. Here, we surveyed the prices of DiGA after price negotiation or arbitral award and analyzed whether differences of prices can be explained by positive health care effect, indication or whether the price was negotiated or determined by an arbitral award. METHODS: The DiGA directory of the BfArM and arbitral awards were screened. Selected DiGA were grouped by indication, positive healthcare effect and whether the price resulted from a successful negotiation or was set by an arbitral award. Then price levels of these groups were compared. RESULTS: Currently (June 2023), reimbursement prices for 14 DiGA are available, 7 of which were decided by the arbitration board. Out of these 14 DiGA, 10 are indicated for physiological diseases and one each for a neurological disorder, a muscular disorder, a metabolic disease, and tinnitus. The manufacturer's price for these DiGA varies between 204 € and 744 € and averages at 491 €, while the reimbursement price varies between 189 € and 243 € and averages at 221 €. The average reimbursement price determined by the arbitration board is 218 €. There was no detectable correlation of the prices with any of the factors defined. CONCLUSIONS: Negotiated prices did not differ significantly from those determined by the arbitration board. Similarly, indication and positive health care had no influence on the reimbursement price. However, most available prices are for DiGA in psychological indications and implement some kind of psychotherapy. Although the price corridor for DiGA seems fixed and rather narrow, how new indications and innovative therapeutic approaches influence attainable prices remains to be seen.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131428","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Follow the Money: Reporting Public Contributions and Incentives on Research and Development Programs for Agenzia Italiana Del Farmaco (AIFA; ITALIAN MEDICINES AGENCY)","id":"66a6527e-d729-4ac6-8ef3-6611898175e6","sessionCode":"HPR33","topDisplay":"Pourrahmat MM1, Maposa P1, del Aguila M2, Fazeli MS1 1Evidinno Outcome Research Inc., Vancouver, BC, Canada, 2Evidinno Outcome Research Inc., Danville, CA, USA","locationCode":"4000","description":"\r\n\t<div>OBJECTIVES: In October 2020, Italy adopted the Pricing and Reimbursement Decree from AIFA. Pharmaceutical companies seeking reimbursement must provide information about public contributions and incentives received on research and development programs. We identified sources of publicly available information regarding public contributions and incentives for research and development of four medicinal products treating acute myeloid leukemia, multiple myeloma, non-Hodgkin lymphoma, and primary myelofibrosis. METHODS: A search identified databases with a public repository on contributions and incentives granted by bodies governed by public law. Publicly funded research databases included (region): Consiglio Nazionale delle Ricerche, AIFA, Governo italiano, and Ministero della Salute (Italy), L'Agence nationale de la recherche and scanR (France), Deutsche Forschungsgemeinschaft (Germany), Ministry for Science and Innovation, IIb Sant Pau, Carlos III Health Institute (Spain), Research and Innovation (United Kingdom), Community Research and Development Information Service (European Union [EU]), RePORT (United States [US]), and Grants and Contribution Dataset (Canada). Across all databases identified above, various terms for drug names, disease area, drug class, manufacturer/sponsor names, and names/registration numbers of pivotal trials were searched. Additional grey literature searches of US and EU clinical trial registry databases identified sponsors of relevant clinical trials. Targeted online searches identified relevant news articles, publications, and press releases. Only information related to public contributions, grants, subsidies, tax rebates as well as other forms of sponsorship were captured. RESULTS: Information related to the nature of contributions, geographical origin and disburser was available; however, information pertaining to contributions and intellectual property rights or profit-sharing schemes were not uniformly found. Information was most available from the US, particularly for studies funded by governmental agencies. Italy had some funding information, while other EU countries and the United Kingdom reported projects but not funding. CONCLUSIONS: While difficult and time-consuming, it is possible to identify and provide this information in reimbursement dossiers submitted in Italy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23del-aguilahpr33poster132463-pdf.pdf?sfvrsn=76bac693_0","title":"ISPOREurope23_del Aguila_HPR33_POSTER132463.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132463","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Epidemiology and Patient Characteristics of Pemphigus Vulgaris in Italy Based on Administrative Database","id":"db291e17-3cff-4cfe-9274-66ded71a85bb","sessionCode":"EPH39","topDisplay":"De Ruyck F1, Degli Esposti L2, Giacomini E3, Leogrande M2, Wittlin B1, Iannazzo S1, Agalarov Y1, Blein C4 1argenx BVBA, Ghent, Belgium, 2CliCon S.r.l. Società Benefit Health, Economics & Outcomes Research, Bologna, BO, Italy, 3CliCon S.r.l. Società Benefit Health, Economics & Outcomes Research, Bologna, Italy, 4argenx BVBA, Zwijnaarde, Belgium","locationCode":"3039","description":"\r\n\t<div>OBJECTIVES: Pemphigus diseases are life-threatening, chronic, autoimmune blistering diseases (AIBDs). This research aims to explore pemphigus vulgaris (PV) epidemiology in Italy, using real world database. METHODS: This retrospective cohort study included patients with PV in administrative database Local Health Units from January 2010 to December 2020. Eligible patients require at least one hospitalization discharge diagnosis at primary or secondary level for pemphigus (ICD-9-CM: 694.4); an exemption code for PV (code: RL0030) and a continuous enrolment for at least 12 months. We considered the index date as the first match with inclusion criteria. Prevalence and Incidence were calculated on an integrated database including both in- and out-patients, geographically distributed and with demographic characteristics representative of the Italian population, covering 6 million health-assisted subjects. RESULTS: From the claims database, we identified 537 patients with an exemption code for PV. Among 28% (n=149) had at least one hospitalization discharge diagnosis for PV and 72% (n=388) had no hospitalization. In the first sub-cohort, 44.3% were men, mean (SD) age at index date was 55.8 (13.0), mean (SD) years of follow-up were 5.7 (2.8) , and the mean (SD) CCI index was 0.7 (0.9) . In 2020 prevalence and incidence were respectively 22 and 2 cases per million. The incidence is quite stable over time and prevalence increases of +38%. In the second sub-cohort 38,7% were men, mean (SD) age at index date was 56.1 (13.7), mean (SD) years of follow-up were 5.5 (2.6) and the mean (SD) CCI index was 0.6 (0.8) . In 2020 prevalence and incidence were respectively 41 and 4 cases per million. The prevalence increases with 32% over the time. CONCLUSIONS: In 2020, the overall incidence of Pemphigus vulgaris is estimated at 6 cases per million with a predominance in women. The findings are matching the previous literature (Micali et al., 1998).</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132832","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Characterization and Trends of Non-Hypothesis Evaluating Treatment Effectiveness (Non-HETE) Studies","id":"b39c9856-bf57-46a6-891f-675a547f6254","sessionCode":"RWD34","topDisplay":"Sheth S1, Willke R2, Crown W2, Hanisch M3, Buikema A2, Barrette E2, Cziraky MJ2, Gautier S4, Alfonso-Cristancho R2 1Center for Health Outcomes, Policy & Economics, Ernest Mario School of Pharmacy, School of Public Health, Rutgers University, Livingston, NJ, USA, 2ISPOR, Lawrenceville, NJ, USA, 3ISPOR, Rahway, NJ, USA, 4ISPOR, Ringwood, NJ, USA","locationCode":"7009","description":"\r\n\t<div>OBJECTIVES: Hypothesis Evaluating Treatment Effectiveness (HETE) studies are increasing however characterization of the design, conduct and reporting of non-HETE studies is limited. The ISPOR Institutional Council RWE Work Group is keen to identifying opportunities for improving the reporting, transparency, and reproducibility of non-HETE studies. This study aims to provide a comprehensive qualitative and quantitative characterization of non-HETE studies by examining registered studies on ClinicalTrials.gov. METHODS: Clinical study metadata were cross-sectionally extracted from <a href=\"https://linkprotect.cudasvc.com/url?a=https%3a%2f%2fClinicalTrials.gov&c=E,1,b3mivzpx2OdvybtVVEzSL43w8wv37ti4xKi9TOawL75SyGVtDqPtz9QqjNuxcqd_WJ9Q7WtKxoLDEL-NaHnRQ63c9iOaTFm6sagu31fZp00,&typo=1&ancr_add=1\">ClinicalTrials.gov</a> on March 02, 2023 using the search delimiters “Observational” and “Retrospective”. Additional filters were for studies “completed” and with “study protocol documents”. Included studies contained data dating back to 1977. Regional distribution, participant demographics, data sources, study duration, treatment types, and methodology were analyzed. RESULTS: 339 unique studies were extracted. The number of studies increased on average by 59.7% annually from 2004 to 2022. The majority of studies were sponsored by academia/providers (53%), industry (44%), Government entities and/or other sponsors (3%). Geographically, the majority of studies were conducted in Europe (39%) and North America (33%). Data sources varied, with health records data (40%) and claims databases (35%) being the most common. The average study duration decreased over time (187.1 months in 2004 to 1.76 months in 2022), while the average sample size per study increased (408 subjects in 2002 to over 50,000 in 2021). CONCLUSIONS: The analysis provided insights into treatment types and the heterogeneity of methods. Trends of registered non-HETE studies showed increasing number of studies worldwide, with a mix of funding and higher participation from different regions. Increasing availability of electronic records and larger databases seem to reflect the increased number of studies, higher sample sizes and lower study duration. Heterogeneity in data sources, methods and reporting highlight the need for enhanced guidance. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133640","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Trends in Northern Ireland HTA Guidance Adoption: NICE Shows Dominance over SMC and AWMSG","id":"ad86f107-658f-460f-adb2-67df5cb8e0d8","sessionCode":"HTA68","topDisplay":"Hill K, Harries M, Wakeman E Ipsen Ltd, Slough, Berkshire, UK","locationCode":"5011","description":"\r\n\t<div>OBJECTIVES: The Strategic Planning and Performance Group (SGGP) of the Department of Health in Northern Ireland (NI) support timely introduction of medicines, ensuring that people in NI benefit from medical advances. It has a Managed Entry process based on adoption of HTA decisions from other UK bodies. This research aimed to identify trends of adoption, analysing the speed and variation of HTA guidance uptake. METHODS: The analysis was performed on SGGP recommendations between 21st January 2021 and 12th May 2023, reviewed by two independent reviewers to identify the disease area, which HTA body the guidance was adopted from, the time between adoption and original publication and whether the guidance was negative or positive. The relationship between these variables was further investigated to determine if there were patterns of adoption via a qualitative synthesis. RESULTS: 271 appraisals were identified: 181 (67%) were adopted from NICE, in line with the SGGP board that decisions made by NICE are adopted as policy, 78 (29%) recommendations were adopted from SMC and 12 (4%) from AWMSG. Of the total appraisals, 46% were in oncology with 77% guidance adopted from NICE and 21% from SMC in this disease area. Negative recommendations were also adopted, accounting for 23% of all adopted guidance since January 2021; 56% of these negative recommendations were adopted from SMC. When comparing negative decisions, 18% of appraisals had differing guidance across the HTA bodies. The majority of guidance is adopted within 6 months of initial publication from the correlating HTA body, with only 6% of technologies not adopted within this timeframe. CONCLUSIONS: NI adopts NICE positive guidance as policy, but defaults to SMC for negative recommendations when neither HTA body have received submissions from companies. Patient access in NI to technologies recommended by NICE, SMC and AWMSG generally occurs within 6 months of their recommendation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023hillhta68poster130692-pdf.pdf?sfvrsn=88a4ddb0_0","title":"ISPOREurope2023_Hill_HTA68_POSTER130692.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130692","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Optimizing Aortic Stenosis Management: Evaluating the Clinical and Economic Impact of Transcatheter and Surgical Valve Replacement in Taiwan","id":"2a22abdd-4706-45c2-a159-67ec129fe8d4","sessionCode":"EE7","topDisplay":"Fu YH1, Yeh CF2, Lin MS2, Chang CJ3, Lin FJ3 1Graduate Institute of Clinical Pharmacy, College of Medicine, National Taiwan University, Baltimore, MD, USA, 2Division of Cardiology and Cardiovascular Center, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan, 3Graduate Institute of Clinical Pharmacy, College of Medicine, National Taiwan University, Taipei, Taiwan","locationCode":"1051","description":"\r\n\t<div>OBJECTIVES: The comparative effectiveness of transcatheter aortic valve replacement (TAVR) versus surgical aortic valve replacement (SAVR) in real-world settings for patients with aortic stenosis (AS) in Taiwan remains uncertain. This study aimed to assess the clinical and economic outcomes during and after the index admission for aortic valve replacement (AVR) in Taiwan. METHODS: Using data from Taiwan’s National Health Insurance Research Database, AS patients who underwent TAVR or SAVR between January 2016 and December 2018 were identified. A 1:1 propensity score (PS) matched cohort was constructed and followed up for two years from the index admission for AVR. Clinical outcomes were compared using restricted mean survival time (RMST) analysis, and economic implications were assessed through incremental cost-effectiveness ratio (ICER) calculations. RESULTS: A total of 455 patients underwent TAVR, while 832 patients received SAVR between 2016 and 2018. Among the 235 PS-matched pairs, patients who underwent TAVR experienced shorter median hospitalization periods, including total length of stay (15 vs. 20 days) and ICU stay (2 vs. 5 days), compared to patients who received SAVR. Furthermore, patients with TAVR demonstrated lower all-cause mortality rates, with a 2-year RMST difference of 0.087 years. However, patients receiving TAVR exhibited higher healthcare resource utilization both during and after the index admission. Overall, the ICER at 2 years was estimated to be NT$ 4,366,908 per life-year (three times Taiwan’s gross domestic product per capita: NT$ 2,533,455 in 2020). Subgroup analysis suggested that TAVR was more cost-effective for patients with a logistic EuroSCORE of ≥10 (ICER: NT$ 1,994,038 per life-year), while SAVR emerged as the dominant option for patients with a logistic EuroSCORE of <10. CONCLUSIONS: TAVR demonstrated favorable clinical outcomes with shorter hospitalization periods and reduced all-cause mortality. However, considering its cost-effectiveness, TAVR appeared to be a viable option primarily for patients with a higher cardiac operative risk.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeu2023ee7fin129735-pdf.pdf?sfvrsn=29b51785_0","title":"ISPOREU2023_EE7_fin129735.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129735","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patient Preferences for Treatment Attributes and Endpoints in Neoadjuvant Therapy for Early-Stage Breast Cancer","id":"a0144b29-a9a4-41e7-8d48-6836757b66cd","sessionCode":"PCR23","topDisplay":"Batchelder L1, Guéroult-Accolas L2, Anastasaki E3, Dunton K4, Lüftner D5, Oswald C6, Ryan J7, Schmitt D8, Steinerova V9, Varghese D10, Johal S10 1IQVIA, Swindon, WIL, UK, 2Mon Réseau Cancer du Sein (Patients en Réseau), Paris, France, 3IQVIA, London, UK, 4Daiichi Sankyo Europe GmbH, Uxbridge, LON, UK, 5Humboldt University, Berlin, Germany, 6IQVIA, Cambridge, CAM, UK, 7AstraZeneca, Cambridge, CAM, UK, 8PATH - Patients' Tumor Bank of Hope, Munich, Germany, 9IQVIA, Amsterdam, North Holland, Netherlands, 10AstraZeneca, Cambridge, UK","locationCode":"6020","description":"\r\n\t<div>OBJECTIVES: Typically, health technology assessment (HTA) focuses on overall survival (OS) as the key clinical endpoint when making decisions on new treatments in oncology. However, capturing OS in early-stage breast cancer (eBC) is not always possible, as longer follow-ups are required. Therefore, this study assessed patient preferences for treatment attributes and endpoints in neoadjuvant therapy where mature and non-confounded OS is unlikely to be available. METHODS: An online survey was developed with healthcare professionals and patient advisory groups and completed by HER2+ eBC patients in Germany, France, Italy and Spain. Patients were presented with 15 discrete choice experiment (DCE) tasks and for each task, asked to choose between two hypothetical treatment profiles, plus an opt-out option. Each DCE profile included 5 attributes with accompanied definitions: OS at 5-years (%) (5-levels), disease-free survival (DFS) at 5-years (%) (5-levels), pathological complete response (pCR) (%) (4-levels), impact of side effects on quality of life (QoL) (3-levels), and ability to receive breast conserving surgery (2-levels). Data was analysed using multinomial logit regressions. RESULTS: 334 HER2+ eBC patients completed the survey. Patients placed most importance on increases in pCR from 25% to 100% (coefficient = 0.55, odds ratio = 1.73, p-value < 0.001), followed by increases in DFS from ‘data unknown’ at 5-years to 95% (coefficient = 0.39, odds ratio = 1.48, p-value < 0.001). Based on the preference weights, pCR (31%) was most important relative to all other attributes, followed by DFS (24%), and OS (22%). CONCLUSIONS: pCR and DFS were considered important by HER2+ eBC patients. These findings are in-line with EMA and FDA reviews that accept pCR as a clinically valid endpoint. It is important HTA bodies and payers consider patient preferences and the relative importance placed on different treatment attributes and endpoints in their decision-making.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ebc-patient-preferenceispor-posterfinal131795-pdf.pdf?sfvrsn=ff70e7ac_0","title":"eBC patient preference_ISPOR Poster_FINAL131795.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131795","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Drivers for Innovation in Digital Therapeutics in Germany","id":"38f79f2c-7df7-4895-a66b-68a424416862","sessionCode":"MT15","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Digital therapeutics (DTx, DiGAs) are a new class of healthcare services. Introduced in Germany in 2019 three key measures have been defined by the Federal Ministry of Health to spur innovation in this field: fast track procedure, preliminary listing, and free pricing for the first 12 months. METHODS: For this analysis the legal procedures behind these measures are discussed and reviewed using in-depth interviews with payers and DTx-companies in Germany from February – April 2023. RESULTS: Interestingly, “fast track” is commonly accepted as an important measure, it is not defined in the respective law. The only official reference is made by BfArM, which is responsible for regulation and making reimbursement decisions for DTx. For most interviewed, fast track refers to predetermined timelines (3 months) for review of the initial application. However, in practice they can be circumvent by questions to the developer which lead to delay or voluntary withdrawel. Preliminary listing was a major achievement in DTx-legislation. However, preliminary listing is limited to one year and requires data generation in parallel. Challenges occur, once there are study delays or the preliminary listing fails becoming permanent. In both cases DTx-developer face huge clawbacks, which already led to bankruptcy. Once this is a potential scenario, prescriber will be reluctant to move forward, not knowing, if the product will be available in the future. The same goes for investors. So preliminary listing does also have drawbacks. Free pricing for the first 12 months received heavy critique from payers but not from industry. However, once the first 12 months elapsed and a new price is not already decided, adequate accruals are needed. CONCLUSIONS: Preliminary listing sounds promising to drive innovation in DTx. But, after careful analysis, the overall benefit is less clear.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131425","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring Heterogeneity in Biosimilars Dynamics in Italy: Results From Three Chronic Therapeutic Areas","id":"1aeacf66-a4eb-40ef-8709-68ded33339bf","sessionCode":"HPR17","topDisplay":"Tettamanti A, Berruto F, Bosio V, Tucci C, Urbinati D IQVIA, Milan, Italy","locationCode":"3072","description":"\r\n\t<div>OBJECTIVES: This study aims to assess the potential NHS savings due to biosimilars adoption and the related market dynamics of originators and other patent-protected molecules within the same therapeutic area. METHODS: 18 biosimilar products and 7 originators in 3 therapeutic areas were selected for the analysis (14 TNF inhibitors - TNFi, 6 insulins and 5 low molecular weight heparins - LMWH). To investigate the consumption of biosimilars and the impact of the purchasing price on expenditure trends within each molecule and therapeutic area, retail and hospital sales data were obtained from IQVIA proprietary database from 2018 to 2022, in terms of values (net acquisition prices) and volume (days of therapy). RESULTS: The analyses led to different results in the 3 therapeutic areas. The consumption of biosimilars increased between 2018 and 2022 reaching a peak of 95% among TNFi molecules, generating a 20% net costs saving in the TNFi class and a 2% reduction considering the whole therapeutic areas, even with several recent launches. Conversely, biosimilar insulins reached a maximum of 15% of consumption, without influencing acquisition costs within the therapeutic class. In the LMWH class biosimilars gained a 77% of consumption compared to biologics, while variations in the total expenditure were not observed for the therapeutic class. CONCLUSIONS: Biosimilars’ market dynamics greatly vary according to molecule and therapeutic area. While the number of biosimilar products doesn’t seem to affect their uptake, intra-molecule competition may represent the main driver for costs reduction. Indirect competition outcomes seem influenced by specific market variables (e.g., distribution channel, prescriber perception and local policies). In conclusion, with increasing biologics facing patent expiry, there are still opportunities to maximize biosimilars uptake, potentially resulting in a positive effect on NHS budget.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23urbinatihpr17posterv2133066-pdf.pdf?sfvrsn=9034de87_0","title":"ISPOREurope23_Urbinati_HPR17_POSTERV2133066.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133066","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Concept Elicitation Interviews to Refine a Conceptual Model of the Patient Experience of Amyotrophic Lateral Sclerosis (ALS)","id":"69ea5548-7bad-423c-9c0c-690092076414","sessionCode":"PT3","topDisplay":"Al-zubeidi T1, Makin H2, Minor C3, Smolkina E4, Hakimi-Hawken N4 1Clarivate, London, UK, 2Clarivate, London, LON, UK, 3Sanofi, Cambridge, MA, USA, 4Sanofi, Amsterdam, Netherlands","locationCode":"3A","description":"\r\n\t<div>OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a rare, incurable neurodegenerative disease characterized by motor neuron loss resulting in weakness, disability, and eventually death. This study aimed to conduct qualitative interviews with people living with ALS exploring the signs, symptoms and impacts of ALS and refine a conceptual model (CM) for ALS. METHODS: This study was a cross-sectional, non-interventional, qualitative study comprising of concept elicitation interviews with adult participants with a clinical diagnosis of ALS. Interviews were conducted by trained interviewers using a semi-structured interview guide. Interview transcripts were analyzed by directed content methods using ATLAS.ti software by trained researchers. Data collection and analysis was completed when data saturation was achieved. RESULTS: Fifteen adult participants were interviewed. Twenty-two signs/symptoms were reported by participants, mostly spontaneously. Most frequently reported signs/symptoms of ALS were physical weakness (n=12/15, 80.0%), changes to speech/difficulty speaking/talking (n=11, 73.3%), respiratory/breathing issues (n=7/15, 46.7%), fatigue/tiredness (n=7/15, 46.7%), decrease in fine and gross motor control (n=7/15, 46.7%). The most frequently bothersome symptoms reported by participants included changes to their speech and talking, muscle spasms, twitches and cramps, breathing difficulties, and fatigue/exhaustion/lack of energy. Participants reported that ALS impacted many aspects of their physical functioning including difficulty walking, difficulty climbing stairs, difficulty eating. Participants experienced increased falls and needed to use mobility aids, due to reduced/loss of mobility. In addition to difficulty walking, most frequently reported bothersome impacts included emotional and mood impacts and loss of independence. A conceptual model, drafted after a review of existing literature/online blogs/forums was updated following patient interviews. CONCLUSIONS: ALS is a debilitating and rapidly progressing disease with high unmet need and devastating impacts on all aspects of patients’ lives with severe impacts on physical and emotional wellbeing. The conceptual model emerging from this study can be used to support the choice of existing disease-specific instruments in clinical studies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23tamarapt3poster132764-pdf.pdf?sfvrsn=17fa690_0","title":"ISPOREurope23_Tamara_PT3_POSTER132764.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132764","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Characterising the Economic Burden Associated With Obstructive Hypertrophic Cardiomyopathy: Healthcare Resource Use and Costs in England","id":"e4d08c90-bb4c-42e7-a81c-691c01203d41","sessionCode":"EE1","topDisplay":"Osman F1, Zema CL2, Hurst M3, Sandler B4, Brellier F4, Utuama O2, Kirichek O5, Houghton J5, Lemmer T4, Tome Esteban M6 1University Hospital Coventry & Warwickshire NHS Trust, Coventry, UK, 2Bristol Myers Squibb, Princeton, NJ, USA, 3Bristol Myers Squibb, Uxbridge, LON, UK, 4Bristol Myers Squibb, Uxbridge, UK, 5Health Economics & Outcomes Research Ltd, Cardiff, UK, 6St George’s Hospital NHS Foundation Trust & St. George’s, University of London, London, UK","locationCode":"1053","description":"\r\n\t<div>OBJECTIVES: Obstructive hypertrophic cardiomyopathy (HCM) is associated with high disease burden, including an increased risk of complications such as arrhythmias and heart failure. This is likely to result in high healthcare resource use (HCRU) and consequent costs. This study aimed to characterise the economic burden associated with obstructive HCM in England. METHODS: This retrospective study used electronic health records from the primary care Clinical Practice Research Datalink (CPRD) GOLD and Aurum datasets linked to secondary care Hospital Episode Statistics (HES) databases in England. Patients were aged ≥18 years at index date (diagnosis of obstructive HCM between April 2009 and October 2020) with ≥1 year of follow-up. HCRU was captured from activity counts for primary and secondary care services with costs obtained from the National Schedule of NHS costs annual report (2019-20), adjusted to 2021 costs. HCRU and costs were estimated per patient-year (PPY) and stratified by severity measured by New York Heart Association (NYHA) class. NYHA class was assigned using an algorithm based on prescriptions and symptoms. RESULTS: A total of 3,730 patients met eligibility criteria, contributing 19,352 patient-years. At baseline, 26%, 34%, 38% and 3% were NYHA class I-IV, respectively, while at end of follow-up this distribution was 6%, 47%, 44% and 4%. Total HRU costs irrespective of NYHA were £3,893 PPY with the majority (£2,071 PPY) from secondary inpatient care. PPY costs increased with higher (worse) NYHA class, from £2,747 (NYHA class I) to £6,990 (NYHA class IV). For patients in NYHA class II-IV, non-elective care was the primary driver of inpatient costs, whereas, elective care for NYHA class I patients. CONCLUSIONS: HCRU and costs increased with higher (worse) NYHA class across primary and secondary care. These data indicate that the health economic burden of obstructive HCM in England is considerable, particularly in patients with more severe disease.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23osmanee1poster133501-pdf.pdf?sfvrsn=893d6cb3_0","title":"ISPOREurope23_Osman_EE1_POSTER133501.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133501","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Using Ketoanalogues of Essential Aminoacids in Patients With Chronic Kidney Disease in Kazakhstan","id":"442f87bf-6e16-411a-bd0e-693bcdf1ce58","sessionCode":"EE115","topDisplay":"Avdeyev A1, Gulyaev A2, Akhmedullin R3 1Medical Center Hospital of the President’s Affairs Administration of the Republic of Kazakhstan, Nur-Sultan, AKM, Kazakhstan, 2Nazarbayev University, Astana, Astana, Kazakhstan, 3Medical Center Hospital of the President’s Affairs Administration of the Republic of Kazakhstan, Astana, AKM, Kazakhstan","locationCode":"2048","description":"\r\n\t<div>OBJECTIVES: This study aims to assess the clinical and economic impact of ketoanalogues of essential aminoacids (Ketosteril) in the management of CKD in the context of the Kazakhstani healthcare system. METHODS: Our analysis incorporated annual direct costs per patient in the management of CKD in adult patients comparing Ketosteril with a placebo. We conducted an economic evaluation focusing on the cost-effectiveness and one-way sensitivity analyses up to 90%. The change in QALYs was used as the primary endpoint of measure. RESULTS: The findings of the cost-effectiveness analysis unveiled a compelling cost-effectiveness advantage of Ketosteril over the placebo, in the CKD management. Ketosteril resulted in a better ratio of the main cost-effectiveness ratio (CER) parameters and a negative incremental cost-effectiveness ratio (ICER). For the QALY, the CER was USD3218 versus USD6788 and the ICER was –USD6779. Our findings were confirmed with the sensitivity analysis. CONCLUSIONS: Our findings provide strong evidence supporting effectiveness and safety profile of using ketoanalogues of essential aminoacids for individuals with CKD. These crucial understandings have promoted its adoption into numerous global therapeutic practices. Furthermore, Ketosteril's advantageous pharmacoeconomic characteristics suggest its recommendation for the management of CKD in Kazakhstan.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023avdeyev-ee115-ketosteril132949-pdf.pdf?sfvrsn=3c680f0f_0","title":"ISPOR EU 2023_Avdeyev EE115 Ketosteril132949.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132949","diseases":[{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Applications for Tafamidis (Vyndaqel®) Under a Managed Access Protcol in Ireland","id":"0b179812-ec89-44d2-ae28-694ba6564d07","sessionCode":"HPR14","topDisplay":"Lucey S1, Gorry C1, Clarke S2, Barry M2 1HSE Medicines Management Programme, Dublin, Ireland, 2HSE Medicines Management Programme, Dublin, Dublin, Ireland","locationCode":"3055","description":"\r\n\t<div>OBJECTIVES: Vyndaqel® (tafamidis) 61 mg capsules were reimbursed in Ireland under the High Tech Arrangement effective 1 March 2022, for the treatment of wild-type (wt) or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Reimbursement is subject to a Health Service Executive (HSE)-Managed Access Protocol (MAP). This study provides an overview of applications for tafamidis (Vyndaqel®) reimbursement in the first year of the protocol. METHODS: All applications submitted to the HSE-Medicines Management Programme (MMP) between 1 March 2022 and 28 February 2023 were reviewed. Data was compiled and analysed in Microsoft Excel™. RESULTS: A total of 104 applications were received. The majority of applications (84.6%) were for males (n=88), with 15.4% applications for female patients (n=16). The average age of applicants was 76.4 years (range 48-89 years). Of the 104 patients, 20 (19.2%) had a confirmed hereditary diagnosis (hATTR-CM), and 79 (76%) had a confirmed diagnosis of ATTR-CMwt. Genotype was not available for five patients (4.8%). The diagnosis of ATTR-CM was established by biopsy in 29 patients and by nuclear scintigraphy in 74 patients. Further information in relation to diagnosis was still outstanding for one patient. Reimbursement was approved for 87.5% of applications received. Further information relating to 10 applications (9.6%) was still outstanding at the end of year one. Three applications (2.9%) were not approved as they did not meet the reimbursement criteria. Reimbursement approval was withdrawn for two patients, where the prescriber wished to switch the patient to alternative treatment (patisiran (Onpattro®)). The number of applications received in year 1 is double what was estimated in the Health Technology Assessment (HTA) conducted by the National Centre for Pharmacoeconomics. CONCLUSIONS: The managed access protocol ensures drug reimbursement is in line with the criteria agreed by the HSE. Data collected may serve to inform future HTA and reimbursement decisions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23luceyhpr14poster132370-pdf.pdf?sfvrsn=ded86029_0","title":"ISPOREurope23_Lucey_HPR14_POSTER132370.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132370","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Literature Review of the Humanistic Burden Associated with Geographic Atrophy","id":"5f18ed80-9727-43f4-976d-69640b36262c","sessionCode":"PCR38","topDisplay":"Aggarwal P1, Mathur S2, Gupta J2, Siddiqui MK3 1EBM Health Consultants, Delhi, DL, India, 2EBM Health Consultants, New Delhi, India, 3EBM Health Consultants, New Delhi, DL, India","locationCode":"6005","description":"\r\n\t<div>OBJECTIVES: The objective of the present review is to provide a comprehensive analysis of the humanistic burden associated with geographic atrophy (GA), the advanced stage of dry age-related macular degeneration that leads to vision loss. METHODS: A comprehensive literature search was performed across Medline and Embase databases. Eligibility criteria were predefined, and studies published in English between January 2013 and June 2023 were included in the analysis. RESULTS: Our systematic literature review identified 22 unique studies, conducted primarily in North America and Europe, with sample size ranging from 8 to 259 participants. The mean age for included participants ranged from 64 to 82 years. In these studies, seven different disease-specific scales were used to measure health-related quality of life (HRQoL). Among these scales, the NEI-VFQ scale was the most frequently used, appearing in 55% of the studies, followed by the Functional Reading Independence (FRI) Index (9% of studies). The key symptoms and concepts assessed were near activities, distance activities, general vision, near vision, dependency, mental health, social functioning, reading speed, functional reading independence, anxiety/depression, fear, difficulty driving, and impaired ability to recognise faces. In two comparative studies, it was found that those with GA had poorer vision-related functioning and lower HRQoL than patients without GA. Patients in the GA group had significantly lower NEI-VFQ-25 composite scores compared to those in the non-GA group (p<0.001). Furthermore, patients with GA had significantly lower scores in NEI-VFQ-25 subscales related to near activities, distance activities, social functioning, and mental health compared to patients without GA (p<0.001). CONCLUSIONS: These findings underscore the significant impact of GA on patients' visual functioning and HRQoL. The insights gained from this literature review provide a valuable foundation for further research and contribute to the understanding of the humanistic burden associated with GA.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/day-1-isporgahumanistic-final131844-pdf.pdf?sfvrsn=724e92af_0","title":"Day 1 ISPOR_GA_Humanistic Final131844.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131844","diseases":[{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evolving Impact of the COVID-19 Pandemic in Chronic Dialysis Recipients According to Wave Sub-Periods","id":"566b6377-a263-428e-8471-6a0645d10db1","sessionCode":"EPH40","topDisplay":"Leye E1, El Karaoui K2, Delory T3, Lapidus N4, Hejblum G5 1Sorbonne Université, INSERM, Institut Pierre Louis d’Épidémiologie et de Santé Publique, Paris, 75, France, 2Sorbonne Université, INSERM U1155, AP-HP, Hôpital Tenon, Service de Néphrologie, Paris, France, 3Sorbonne Université, INSERM, Institut Pierre Louis d’Épidémiologie et de Santé Publique, Centre Hospitalier Annecy-Genevois, French Institute for Demographic Studies (INED), Mortality, Health and Epidemiology Unit, Epagny Metz-Tessy, France, 4Sorbonne Université, INSERM, Institut Pierre Louis d’Épidémiologie et de Santé Publique, AP-HP, Hôpital Saint-Antoine, Unité de Santé Publique, Paris, France, 5Sorbonne Université, INSERM, Institut Pierre Louis d’Épidémiologie et de Santé Publique, Paris, France","locationCode":"3041","description":"\r\n\t<div>OBJECTIVES: A national French cohort study was devised for investigating the impact of the pandemic waves and between-wave sub-periods on the survival of chronic dialysis recipients (CDR). The association between vaccination roll-out and mortality was also investigated. METHODS: Using the French national health claims database, incident persons with end stage kidney disease between 2015 and 2020, except those who received a pre-emptive kidney transplant were included and followed-up from their first dialysis date to December 31, 2022. The survival of CDR during the pre-pandemic and pandemic periods were estimated and compared to controls (matched on sex, age, comorbidities and region), using Cox models with time-dependent covariates. RESULTS: The 60,481 CDR and 120,962 matched controls totalized 600,175 person-years followed-up. Global mortality in CDR was higher than in controls (hazard ratio (HR) [95% confidence interval]: 3.12 [3.06-3.18]). Compared to the pre-pandemic period, the pandemic first (March 01 to May 18, 2020) and second wave (September 07, 2020 to May 10, 2021) sub-periods were associated with a higher risk of death (HR 1.18 [1.13-1.24], 1.10 [1.07-1.13], respectively), while the risk of death was similar during the third wave (November 21, 2021 to April 25, 2022, HR: 0.97 [0.93-1.02). In contrast, the first, second and third between-wave sub-periods were associated with a lower risk of death (HR: 0.88 [0.84-0.91], 0.87 [0.84-0.90] and 0.81[0.78-0.84] respectively). Sub-period evolving trends were similar in CDR and controls. Adherence to COVID-19 vaccine booster was low, although increasing doses were associated with decreasing risks of death (HR: 0.68 [0.66-0.69] to 0.35 [0.30-0.41] for 2 to 5 doses, respectively). CONCLUSIONS: During the pandemic period in France, excess mortality in CDR decreased with time, and likely characterized with a between-wave-related harvesting pattern similar in CDR and controls. The study advocates research on candidate policies for increasing adherence to vaccine booster doses.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023leyeeph40poster131137-pdf.pdf?sfvrsn=80c3b1ca_0","title":"ISPOREurope2023_Leye_EPH40_POSTER131137.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131137","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Consumer Preferences for Attributes of Influenza Vaccines in the United States: Results from a Discrete-Choice Experiment Study","id":"f8e5321e-d9e1-4ed3-ac5a-6a1e0c2c783e","sessionCode":"PCR47","topDisplay":"Poulos C1, Leach CA1, Kent C1, Rudin D2, Buck P3, Ghaswalla P2 1RTI Health Solutions, Research Triangle Park, NC, USA, 2Moderna, Inc., Cambridge, MA, USA, 3Moderna, Inc., Philadelphia, PA, USA","locationCode":"6047","description":"\r\n\t<div>OBJECTIVES: Quantify preferences for influenza vaccine attributes among consumers in the United States (US). METHODS: Using a discrete-choice experiment, US adults were presented with a series of experimentally designed pairs of hypothetical influenza vaccines comprising five attributes with varying levels: absolute vaccine efficacy (AVE) (15%-60% of influenza infections prevented), hospitalization prevention (same as most other vaccines, more than most other vaccines), durability (protection fades within 6 months, constant protection for at least 6 months), and side effects (risk of moderate-to-severe injection site reactions [ISR] [0%-50%] and risk of flu-like symptoms [FLS] due to the vaccine [0%-60%]). In each choice question, respondents selected the preferred vaccine or “no flu vaccine.” Random-parameters logit analysis results were used to calculate conditional relative attribute importance (CRI) out of 100% and maximum acceptable risks of vaccine side effects in exchange for improvements in vaccine efficacy. RESULTS: The sample included 400 respondents aged 18-64 years and 201 respondents aged ≥ 65 years. Half had at least one non-age risk factor for severe influenza. On average, consumers preferred influenza vaccination to opting out of vaccination. Consumers placed greatest importance on avoiding risk of FLS (CRI, 40%), followed by increasing AVE (CRI, 36%) and avoiding ISR risk (CRI, 21%). On average, hospitalization prevention and durability had no influence on vaccine choice. Consumers were less tolerant of FLS than ISR. For an improvement in AVE from 15% to 25%, consumers would be willing to accept a 34% risk of ISR or a 19% risk of FLS. For an improvement in AVE from 25% to 50%, consumers would be willing to accept a 31% risk of ISR or a 16% risk of FLS. CONCLUSIONS: Adults in the US were willing to accept increases in vaccine-related risks for improved AVE, and they were more tolerant of ISR risks than FLS risks.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23poulospcr47posterv2130020-pdf.pdf?sfvrsn=49964fe1_0","title":"ISPOREurope23_Poulos_PCR47_POSTERV2130020.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130020","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Challenges of Identifying Health Utility Data for Patients With Penta-Refractory Multiple Myeloma to Inform HTA Reimbursement Discussion for Newer Treatment Options","id":"7e01b060-8a99-455a-9053-6a7b3b2205d4","sessionCode":"HTA18","topDisplay":"Hibbs R1, Bianco M2, Noble-Longster J1, Stainer L3, Cooper C4, Strickson AJ1 1Tolley Health Economics Ltd., Buxton, Derbyshire, UK, 2Menarini Stemline, Pomezia, RM, Italy, 3Tolley Health Economics Ltd., Buxton, DBY, UK, 4Independent Researcher, London, UK","locationCode":"4057","description":"\r\n\t<div>OBJECTIVES: Selinexor plus dexamethasone (Sd) is the first regimen licensed for penta-refractory multiple myeloma (PR-MM), seeking reimbursement in Europe through Health Technology Assessment (HTA). To inform cost-effectiveness analyses (CEA) for HTA, robust data in a relevant population is required, including health state utility values (HSUVs) representative of health-related quality of life (HRQoL) outcomes of interventions versus standard of care. METHODS: A systematic review was conducted to identify economic evaluations, costs and resource use, and health utility data, in patients with relapsed and/or refractory MM (RRMM), including PR-MM. RESULTS: Bibliographic database searching with additional handsearching identified 62 records reporting utility values mostly in earlier lines of RRMM (2L/3L/4L). Additional studies were identified reporting HRQoL data from measures mappable to EQ-5D, however, there were no published studies reporting utility values for a PR-MM population. Two records were identified from the phase 2b STORM trial of Sd, in a penta-exposed population (PE-MM) with ≥50% PR-MM, reporting HRQoL data using FACT-G/MM/MM-TOI for which mapping to EQ-5D-3L/5L is required to elicit utility values. As a potential proxy to PR-MM, a CEA of belantamab mafodotin (belamaf) versus Sd in a 5L+ triple-class refractory (TCR) population applied HSUVs from DREAMM-2 (4L+ triple-class exposed [TCE]) obtained by mapping EORTC-QLQ-C30/MY-20 data to EQ-5D-3L. These mapped utilities were referenced in a second belamaf CEA (5L+ TCE). A third CEA of treatments in heavily pre-treated RRMM applied published HSUVs from MM-003 in double-class refractory participants who had received a median of five prior lines. The pre-progression HSUV reported across all three records was 0.73. CONCLUSIONS: Although robust data is required for CEA informing HTA of newer interventions, studies only reported HRQoL data specifically for patients with PE-MM and nothing specifically was for PR-MM. Furthermore, reported HRQoL data were derived from tools other than EQ-5D, necessitating mapping exercises to obtain HSUVs for CEA.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23stainerhta18poster133116-pdf.pdf?sfvrsn=4887b8a1_0","title":"ISPOREurope23_Stainer_HTA18_POSTER133116.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133116","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Regional Differences in the Use of Ige Testing in Primary Care: The Case of Sweden","id":"683fd7c6-cf5d-4dce-acdf-6abba5f4f91d","sessionCode":"HSD21","topDisplay":"Pousette A1, Fajutrao L2, Ekebom A3, Hjelmgren J4, Dahl Å5 1The Swedish Institute for Health Economics (IHE), Stockholm, AB, Sweden, 2Thermo Fisher Scientific, Stockholm, Sweden, 3Swedish Museum of Natural History, Stockholm, Sweden, 4The Swedish Institute for Health Economics (IHE), Lund, M, Sweden, 5University of Gothenburg, Gothenburg, Sweden","locationCode":"4030","description":"\r\n\t<div>OBJECTIVES: Airborne allergy is a public health concern contributing to significant costs for the Swedish healthcare system. Clinicians usually use IgE testing to guide the diagnosis and treatment of allergies. Substantial differences in IgE testing between healthcare regions are observed. Variations in pollen levels are believed to be one driving factor. In this study, we investigated other factors like measures imposed by regional decision makers governing the provision of primary care and incentives of IgE testing to explain these IgE test utilization differences. METHODS: A two-step approach was applied: 1) A statistical OLS regression model was used to evaluate the relationship between the number of IgE tests per capita and annual regional pollen levels (14 / 21 regions), identifying regions with large deviations from actual and expected test levels. 2) Differences in various measures (e.g., allergy treatment guidelines, reimbursement models, and other regulative aspects) involved in the governance of primary care provision were evaluated for their influence in IgE testing in selected regions. RESULTS: A clear statistical relationship between annual regional pollen levels and performance of IgE tests (R2 adjusted=0.51) was demonstrated. Among regions with the highest deviations from expected levels, significant variations in allergy testing recommendations in local treatment guidelines were noted. In addition, results indicated that IgE test utilization seemed to be also influenced by economic incentives to primary care centers. CONCLUSIONS: Large interregional differences in the performance of allergy tests exist in Sweden. These differences cannot only be explained by annual pollen levels, but also by differences in the regional management and governance of primary care services delivery.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-poster-ige-testingfinal27oct129912-pdf.pdf?sfvrsn=3471a320_0","title":"ISPOR poster_ IgE Testing_FINAL_27_oct129912.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129912","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Italian Healthcare System Competences in View of the New European HTA Regulation","id":"a6728ec3-1433-485a-8993-6ba09b1c3b6a","sessionCode":"HTA70","topDisplay":"Belfiore M, Cavazzani V, Chinelli M, Laimeche M Roche S.p.A., Monza, MB, Italy","locationCode":"5000","description":"\r\n\t<div>OBJECTIVES: Regulation (EU) 2021/2282 on Health Technology Assessment (HTA), which enters into force from 2025, aims to harmonize the clinical evaluation, relating to the HTA procedures of the Member States, in order to accelerate and homogenize access to medicines and medical devices in the EU. For an effective implementation of the Regulation, HTA competencies will play a key role in each involved country. METHODS: A literature research was performed to identify key gaps related to the new HTA Regulation in order to build an HTA competencies model.Therefore, an online survey on a sample of 133 Italian representatives among pharmaceutical companies (49), HTA bodies (19), universities (30) and scientific societies (35) was developed to map the relevance and readiness in regards to the identified competencies on a scale from 0 to 5. The survey was submitted thanks to the collaboration of ISPOR Italy-Rome chapter. RESULTS: The prioritized competencies were: clinical effectiveness, health economics, ethics and patient involvement, digital, communication and management.The survey showed that the relevance of the identified skills scored higher than the respective internal readiness. All the actors ranked clinical effectiveness high (it was scored at least 4). Ethics and patient involvement was ranked high by both the pharma industry and the HTA body in relevance although presenting the higher gap between relevance and readiness (4.3 against 3.5 for the HTA body and 4 against 3.4 for the pharma companies). CONCLUSIONS: Given these trends, it is possible to conclude that a higher investment in skills, both soft and hard ones, is necessary. It is also important to raise awareness about the ones that lack most with reference to the current Italian healthcare system. An exchange between pharmaceutical companies, HTA bodies, universities and scientific societies, should be encouraged by promoting multidisciplinary training plans, in order to foster the collaboration among public and private parties.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeuropebelfiorehta70poster131511-pdf.pdf?sfvrsn=b887ec6_0","title":"ISPOREurope_Belfiore_HTA70_Poster131511.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131511","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Germany: Leading the Way for Digital Health Technology Assessment in Europe","id":"5335c396-ab82-41ac-a4b5-6bd860d2d526","sessionCode":"HPR25","topDisplay":"Patel M1, Ng E1, Desai A2 1Red Nucleus, London, LON, UK, 2Red Nucleus, San Francisco, CA, USA","locationCode":"3077","description":"\r\n\t<div>OBJECTIVES: In 2018 Germany were ranked 17th out of 18 countries for their level of digitisation of healthcare delivery, as reported by the SmartHealthSystems study. Just 2 years later the German government introduced the Digital Healthcare Act, stimulating the implementation of the DiGA system (Digitale Gesundheitsanwendung), in combination with use of electronic health records and e-prescribing. These changes have propelled the German method of leveraging Digital Health Technologies (DHTs) to the forefront of European healthcare digitization, such that several other European countries are modelling their approach on Germany’s methodology. Here, we explore the key success factors of the German program, and how other EU markets are adapting their national frameworks based on this. METHODS: We analysed the changes brought in by the Digital Healthcare Act, and the impact this had on the use of DHTs, compared to DHT use prior to introduction of the act. Through scrutiny of governmental policy we also assessed how other EU markets structured and implemented their DHT assessment systems to compare similarities and differences between their and Germany’s frameworks. RESULTS: The Germany DiGA system has been replicated, or modeled upon, by several EU countries, including France with their PECAN model, Belgium, Spain and several of the Nordics markets. Small differences exist between systems, such as the classes of DHTs eligible for DiGA vs. PECAN. There are also discussions to utilize the German DiGA approach towards harmonizing market access for reimbursable medical apps across Europe. CONCLUSIONS: Germany’s DiGA fast track process for DHTs is considered best-in-class and well suited for European health systems. This required a substantial change in legislation, but decisive and focused action has resulted in 1000’s of patients now benefiting from using DHTs, as well as allowing for high levels of innovation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23patelhpr25poster133471-pdf.pdf?sfvrsn=bd4562a9_0","title":"ISPOREurope23_Patel_HPR25_POSTER133471.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133471","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"ARV Treatment and Care for People Living with HIV: Cost-Benefit Analysis","id":"23438f43-6883-42fd-8ea1-6c50f12f09bb","sessionCode":"EE32","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1075","description":"\r\n\t<div>OBJECTIVES: According to the latest statistics from the World Health Organization, more than 37.7 million people are living with HIV at the end of 2020 and about two thirds of those infected are in the African region and 680,000 people have died from HIV-related causes. HIV/AIDS is part of the list of serious or disabling illnesses requiring long-term care. According to the IHME, the death toll exceeds 860,000 and the disease burden estimated at more than 47 million years of life lost or DALYs. METHODS: Aware of this problem, Morocco has made great strides in the fight against HIV-AIDS, thanks to the strong political will that has placed the response to HIV among the national health priorities. Since the introduction of ARVs in Morocco in 1998, the Ministry of Health and Social Protection has been providing free care for PLWHA, with additional support from the Global Fund. This has generated significant economic costs, making it an important public health issue. The aim of this study is to evaluate the return on investment of ARV treatment and total care for PLWHA.In this study, we sought to measure the cost-benefit ratio (CBR) of an investment to be made and the expected economic benefits. RESULTS: The care package for PLWH includes antiretroviral treatment, drugs for certain opportunistic infections, HIV laboratory tests, immuno virological follow-up and consultation. The total cost of care for PLWHA amounts to US$ 5,246,387.23. We were able to identify the number of deaths prevented thanks to ARVs. The monetary value gained from the 1,098 deaths averted is US$70,225,363. The return on investment is 13.38. CONCLUSIONS: This return on investment could be higher if we add the gains due to savings in social costs, as well as savings in indirect costs such as lost productivity due to absenteeism.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131075","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Budget Impact of Stepping Off Patients from Long-Term Inappropriate Proton Pump Inhibitors (PPIs) Use to Episodic Alginate Treatment","id":"c4a903e3-a790-49b6-8f30-6c56d637ba61","sessionCode":"EE24","topDisplay":"Wray J1, Aluko P2, Sharma Y3, Guerra I3, Coyle C1, Plehhova K1 1Reckitt, Hull, Kingston upon Hull, UK, 2Reckitt, Newcastle upon Tyne, NBL, UK, 3IQVIA, London, UK","locationCode":"1070","description":"\r\n\t<div>OBJECTIVES: Prolonged use of proton pump inhibitors (PPIs) has been associated with adverse effects, including increased risk of infections, kidney diseases, and bone fractures. Current treatment guidelines recommend 4-8 weeks of PPIs in patients with gastro-oesophageal reflux disease (GORD) and dyspepsia. Patients are often offered longer-term maintenance therapy, however NICE guideline recommends gradual step-down. Nevertheless, studies have shown that patients are placed on PPIs for an extended period. This study investigates the economic impact of stepping off patients on long-term PPIs to episodic alginate in the management of GORD and dyspepsia from NHS England payer perspective. METHODS: A budget impact model (BIM) with a 5-year time horizon was developed to estimate the economic impact of stepping down patients from long-term PPIs to episodic alginate. The model included real world data from NHS primary care of adult population identified as having inappropriate long-term PPI usage. Sensitivity analyses were performed to assess the impact of uncertainty around input parameters. RESULTS: Over a 5-year period, switching 20% of patients on long-term PPIs to alginates led to a cumulative cost savings of approximately £21 million. Irrespective of the adverse events, there were cost savings in patients treated with alginates episodically. The cost savings increased with each year of switching, from £2.9 million in year 1 to £5.8 million in year 5. When parameter values were varied by +/-25% in the sensitivity analysis, the net budget continued to be cost-savings in patients switched to alginate. CONCLUSIONS: Switching patients from inappropriate long-term PPI to episodic alginate therapy can help reduce the burden on NHS England by 3.56% over 5 years for patients on inappropriate long term PPI. It is important for prescribers to consider the potential cost saving implications which may give room for reallocation of resources without detrimental impact to patient health outcomes.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23wrayee24poster128101-pdf.pdf?sfvrsn=60169daa_0","title":"ISPOREurope23_Wray_EE24_POSTER128101.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128101","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Database Findings From Previous NICE Evaluations in Renal Cell Carcinoma to Support a Pathway Approach to Technology Appraisal","id":"0f98fa91-e50b-4246-a7ae-6cb671e1e13d","sessionCode":"HTA15","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"4055","description":"\r\n\t<div>OBJECTIVES: The range, complexity and number of National Institute for Health and Care Excellence (NICE) technology appraisal (TA) guidance is increasing each year and the total is expected to reach over 1,000 in 2024. A database was constructed to record key issues and decision-making assumptions from published NICE TAs to facilitate knowledge sharing across NICE, committee members and the wider HTA community. Renal cell carcinoma (RCC) was selected for initial extraction to support NICE’s pathway approach to technology appraisal, which aims to develop a core disease pathway model to improve consistency and reduce duplication. METHODS: A database was designed and constructed in Microsoft Excel®. Manual searches of the NICE website were performed. NICE TA recommendations issued between March 2000 and December 2022 were reviewed. Data were extracted from RCC appraisal documents published before December 2022. RESULTS: 15 of 854 published NICE TAs considered RCC. 11 of 15 were single technology appraisals, 2 managed access reviews, 1 multiple technology appraisal and 1 terminated. Clinical data was primarily informed by Phase 3 open-label studies and only the 3 most recent appraisals were supplemented with real-world data. Economic models were partitioned survival (72%) or state transition (28%). Differences across appraisals included model structure and health states, utility modelling and approaches to model long-term effects. The most common key issues related to generalisability, clinical evidence, long-term extrapolations and end-of-life criteria. CONCLUSIONS: The database presents a summary of trends and themes that could be used to inform committee discussion in future RCC appraisals. Findings strengthen the rationale for the development of a pathway appraisal approach by highlighting key similarities and differences across RCC appraisals that could be improved on with a single pathway model. Following this initial extraction, further database development and extractions are planned for other conditions in the TA program.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133460","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Valuable Perspective on Medical Devices – Assesing the Organizational Impact of Health Technologies in Decision-Making","id":"b0fdd540-7e0e-4794-939b-6e3671070038","sessionCode":"HTA39","topDisplay":"Springborg A1, Simonsen K2 1The Danish Health Technology Council, Aalborg, 81, Denmark, 2The Danish Health Technology Council, Aalborg Ø, Denmark, Denmark","locationCode":"4074","description":"\r\n\t<div>OBJECTIVES: The primary outcomes of health technologies are often found within clinical effectiveness and safety. Some medical devices differ by having primarily organizational impacts such as facilitating workflows. This abstract presents a case study on two Health Technology Assessments (HTA) of medical devices with primary outcomes on the organizational aspects, illustrating the importance of evaluating medical devices from a wider perspective. METHODS: In 2023 the Danish Health Technology Council (DHTC) published HTAs on handheld ultrasound devices in emergency departments and intelligent beds in intensive care units. DHTC assess health technologies through four perspectives: Clinical effectiveness, Patient’s perspective, Organizational implications and Health economics. The Organizational implications of the investigated technologies were identified through surveys and interviews with health care professionals. By comparing the two HTAs, we identified which factors may present common facilitators or barriers to the realization of the value of these health technologies. RESULTS: Three factors were identified as common for the organizational value of the technologies investigated: compatibility, availability, and the attitudes of health care professionals. Compatibility with regards to the physical surroundings in the clinical setting and integrated health systems appears crucial for new medical devices to be implemented into existing systems and workflows. Availability, including the number of devices and location of the health technologies impacts access as well as health care professional usability and routine. Lastly, attitudes towards health technologies and the perceived quality of the devices were identified as critical for the willingness to utilize new technologies. These factors are conditional on local structures and important to address, ensuring that the potentials of technologies are realized in clinical practice. CONCLUSIONS: This case study found that organizational implications of health technologies may be crucial for realization of the value of medical devices, making the organizational aspect a valuable perspective in decision-making and implementation of technologies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23springborghta39poster132552-pdf.pdf?sfvrsn=19a212f8_0","title":"ISPOREurope23_Springborg_HTA39_POSTER132552.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132552","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Price, Spending, & Utilization of Oral Second-Generation Antipsychotics & the Impact of Generic Entry on Market Shares: Trend Analysis of Medicaid Database from 1991 to 2022","id":"995b2c33-827b-4cfa-b302-6e9298919950","sessionCode":"EE122","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"2054","description":"\r\n\t<div>OBJECTIVES: Analyze the trend in price, spending, and utilization of orally administered SGAPs over the past 30 years utilizing Medicaid claims database and compares the market share of brands versus generics. METHODS: Descriptive analysis utilizing the Medicaid claims database of all oral SGAPs approved since 1991 as well as injectable Aristada. NDC codes were used to identify and extract SGAPs. Brand and generic were defined using NDC1 code. Price per prescription was calculated as the total reimbursement divided by the number of prescriptions. Price was defined as the average prescription reimbursement. Spending was defined as total reimbursement for all SGAPs and for each brand and generic. The number of prescriptions were measured to assess the utilization rate. Values were adjusted for inflation using CPI medical. RESULTS: Total of 22 SGAPs were included of which 8 were generic. Estimated total reimbursement for all SGAPs was $ 223 billion of which market share of brand versus generic was 94% to %7. Brand Risperdal had the highest reimbursement amount which was 29% of the total expenditure while generic Asenapine had the lowest, the trend showed gradual increase in reimbursement until 2005 following fluctuations which was projected to be continued until 2025.The average price per prescription was $298. Brand Invega had the highest average price per prescription of $2940 while generic quetiapine had the lowest with $42.the price rate continued to increase over the year for brands unlike generics. Over 661 million prescriptions and 19.5 million units. Generic prescriptions market share increased to 84% by 2022 while brands decreased to %16. CONCLUSIONS: The spending on SGAPs increased tremendously over the past 30 years as more brands and generics were introduced to the market. Generic SGAPs had an impact on controlling the cost burden and there was a shift toward prescribing more generic than brand.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128193","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Humanistic and Economic Burden of Multiple System Atrophy: A Systematic Review","id":"7aa3a03c-6732-4187-9bc0-6ef8848d84c5","sessionCode":"PCR11","topDisplay":"Aggarwal P1, Kaur H1, Mathur S1, Gupta J1, Siddiqui MK2 1EBM Health Consultants, New Delhi, India, 2EBM Health Consultants, New Delhi, DL, India","locationCode":"6004","description":"\r\n\t<div>OBJECTIVES: Multiple system atrophy (MSA) is a rare and progressive neurological disorder affecting various bodily systems. Our systematic literature review focused on evaluating the overall humanistic and economic burden associated with MSA and identify any existing gaps in the current evidence. METHODS: A comprehensive literature search was performed across Medline and Embase databases. Studies published in the English language between January 2013 and June 2023 reporting the humanistic and economic burden of MSA were included using pre-defined eligibility criteria. RESULTS: Of the 2357 studies identified, a total of 43 studies focused on humanistic burden and three studies examining economic burden of MSA were included. Health-related quality of life (HRQoL) was assessed using both generic and MSA-specific instruments, with UMSARS (n=27 studies), UPDRS (n=8), PDQ (n=7) scales being used more commonly across the included studies. The findings from these studies suggest that individuals with MSA-P (parkinsonian subtype) experience more severe motor impairment, hyposmia (reduced sense of smell), depression, anxiety, cognitive impairment, and lower HRQoL compared to those with MSA-C (cerebellar subtype). One study indicated that non-motor symptoms are more prevalent and severe in MSA-P patients, particularly in terms of mood/apathy and gastrointestinal symptoms. Most of the studies examining the economic burden of MSA were conducted in North America and Europe. These studies revealed that the average annual cost of care per diagnosed MSA patient was US$9405, with only US$1782 directly attributed to MSA-related encounters. One study reported resource utilisation, indicating that most participants, 96% and 70.2%, had visited >1 family physician and emergency department, respectively. Additionally, 62% of individuals had been hospitalised multiple times, and 25.5% spent more than 30 days in the hospital. CONCLUSIONS: These findings highlight the considerable humanistic and economic challenges faced by individuals living with MSA. Further research is needed to better quantify the economic burden associated with MSA.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/day-1-ispormsaecon-and-hum-final132933-pdf.pdf?sfvrsn=562052cc_0","title":"Day 1 ISPOR_MSA_Econ and Hum Final132933.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132933","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Treatment Pathways and Outcomes of Advanced Ovarian Cancer Patients Who Are Underrepresented in Trials","id":"6151ddb2-9036-4327-ab83-6f888cb9af92","sessionCode":"RWD13","topDisplay":"Picariello F, Wallis J, Luhar S, Carpenter L Arcturis Data, Oxford, UK","locationCode":"6067","description":"\r\n\t<div>OBJECTIVES: The aim was to address low external validity concerns of trial evidence by examining treatment pathways and outcomes of ovarian cancer patients in the UK, focusing on underrepresented subgroups. METHODS: This retrospective study used de-identified electronic health records for advanced ovarian cancer patients diagnosed between 2015 and 2023 from UK NHS partners collated as part of the Arcturis Data Platform. The following subgroups were defined: age >65, Black, Asian and minority ethnic (BAME) groups, Index of Multiple Deprivation (IMD) deciles 1-3, ECOG score ≥2, moderate to severe comorbidity based on Charlson Comorbidity Index (CCI) ≥3, and no cytoreductive surgery. For each subgroup, treatment sequencing and survival were assessed. RESULTS: From a total of 1,030 advanced ovarian cancer patients, there were 606 patients aged >65, 18 BAME patients, 193 patients living in socioeconomically deprived/disadvantaged areas, 103 patients with an ECOG score ≥2, 43 patients with moderate/severe level of comorbidities, and 422 patients without cytoreductive surgery. First-line therapy rates ranged 50-98.7%, lower among patients with more comorbidities. Carboplatin and paclitaxel was the most common first-line regimen. Maintenance therapy rates varied 0%-50%, with bevacizumab as the preferred regimen. Approximately half received second-line therapy, with limited second-line maintenance. Median time to next treatment (TTNT) from first-line therapy initiation ranged 4.5-15 months, shortest among patients without cytoreductive surgery. Median TTNT from first-line maintenance initiation fluctuated around 7-10 months across most subgroups. Median time to death generally fluctuated between 20-30 months from first-line therapy and maintenance therapy initiation. Shorter survival (12-17 months) was observed among sicker patients based on comorbidities, ECOG, and cytoreductive surgery. CONCLUSIONS: This study sheds light on treatment pathways and outcomes of underrepresented ovarian cancer patients, particularly highlighting a potential unmet need among patients with higher comorbidity burden and no cytoreductive surgery. Findings should be interpreted with caution given the small sample sizes of some subgroups.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeuropr2023picariellorwd13poster130089-pdf.pdf?sfvrsn=b662d3de_0","title":"ISPOREuropr2023_Picariello_RWD13_Poster130089.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130089","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Status Changes of Refugees from North Korea After Defection: Using National-Wide Big Data in South Korea","id":"147b2aab-d534-46be-9fdf-6fec32612eb5","sessionCode":"RWD35","topDisplay":"Lee J1, Choi M2, Cha Y3 1National Health Insurance Service, Wonju, 42, South Korea, 2National Health Insurance Service, Seoul, 42, South Korea, 3National Health Insurance Service, seoul, 42, South Korea","locationCode":"7008","description":"\r\n\t<div>OBJECTIVES: The number of North Korea Refugees(NKRs) in South Korea is steadily increasing but previous studies on NKRs' health status have been limited. This study examines the health status of NKRs living in Sough Korea and identify how does it change according to adaptation process. METHODS: We conducted a cross-sectional study and longitudinal study by using National-wide big data targeting all North Korean refugees(NKRs) in South Korea from 2002 to 2019. We examined healthcare expenditure, frequency of healthcare utilization, and health status outcomes such as body mass index(BMI), waist measurements, and health-related factors(smoking, alcohol consumption, and regular exercise). To compare NKRs and South Koreans, we utilized the National Health Insurance Statistical Yearbook for South Koreans, employing the same categorization as used for NKRs. RESULTS: The results of examining the key variables are as follows. Examining the latest available data from 2019, there were 38,565 NKRs in South Korea. Females had higher healthcare utilization than males, following a similar pattern of utilization with a slight increase in the second year after entry and a decrease in the third year. Per capita healthcare expenditure increased by 4.8% annually. Male NKRs had a smoking rate of 53.2%, significantly higher (17.9 percentage points) than the 35.3% among South Koreans. The prevalence of 'mental and behavioral disorders' among NKRs was over double that of South Koreans. CONCLUSIONS: The findings will contribute to a better understanding of the healthcare needs of this vulnerable population and inform targeted interventions to improve their overall well-being. Our findings provide empirical evidence regarding health status NKRs in South Korea. This study suggests that pamphlets, manuals of health screening program should be developed. There is a need to improve the existing data on refugee's health status, needs and access to healthcare to be able to provide optimal healthcare tailored to the needs of refugees.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23jungmyun131574poster131574-pdf.pdf?sfvrsn=681f665c_0","title":"ISPOREurope23_JUNGMYUN_131574_POSTER131574.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131574","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Scoping Mock-Up: What Can We Learn From an EU-4 Experiment for EU HTA?","id":"40b3c71a-f69b-40bf-9d5a-70140cc64448","sessionCode":"HTA58","topDisplay":"Ecker T1, Le Mao J2, Prada M3, Lizán L4, Brozek A1 1Ecker + Ecker GmbH, Hamburg, Germany, 2CEMKA, Bourg La Reine, France, 3Intexo SB, Rome, RM, Italy, 4Outcomes'10, Castellón de la Plana, Castellón de la Plana, Spain","locationCode":"5004","description":"\r\n\t<div>OBJECTIVES: Scoping defines each HTA institute’s setting of the research question with its components Population, Intervention, Control, Outcomes (PICO). The scoping process will be key for the subsequent EU HTA. The future scoping process was simulated within our European network. METHODS: Mock-up was done for Onasemnogene abeparvovec (Zolgensma®), as ATMPs are within the first wave of EU HTA, ATMPs pose special methodological challenges within HTA, and this was a widely discussed product launch. Scoping consists of 2 phases, survey and consolidation. Survey was done considering actual assessment and reimbursement restrictions of the respective countries. Written feedback from Italy, Spain, France, and Germany was checked for consistency and follow-up questions asked for clarification. Consolidation was performed by TE and AB. RESULTS: Assessment and reimbursement are not completely separate processes in all 4 countries. Germany was the only country for which the complete label was relevant for reimbursement. France excluded symptomatic patients with SMA type 3. Italy and Spain only included symptomatic patients with SMA type 1 and presymptomatic patients with up to 2 copies of the SMN2 gene. For the symptomatic patients Italy and Spain defined restrictions regarding weight and age of patients. In addition, Spain also set an age restriction for the presymptomatic population. Nusinersen was considered a relevant comparator in all countries at some point of the evaluation, whereas risdiplam was only considered by France during reassessment which occurred later. Initially, Germany did not define a comparator. Not all outcomes have had the same relevance in every jurisdiction, but there seemed to be reasonable consensus. CONCLUSIONS: In summary, whereas intervention, control, and outcomes mostly converge, no two countries aligned on the reimbursed population. Due to the special situation final assessment was determined by the data and was to some extent independent of a predefined PICO scheme.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23brozekhta58poster131341-pdf.pdf?sfvrsn=c5b352bb_0","title":"ISPOREurope23_Brozek_HTA58_POSTER131341.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131341","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Overview of Systematic Reviews of Economic Evaluations on Cancer Screening: Landscape, Quality, and Recommendations","id":"8b965b84-b9f2-4e12-8203-701c36e12735","sessionCode":"EE60","topDisplay":"Tickell LA, Rabie H, Lim KK King's College London, London, UK","locationCode":"1047","description":"\r\n\t<div>OBJECTIVES: Numerous systematic reviews of economic evaluations (SREEs) have been performed to examine the value for money of cancer screening. However, the evidence and the quality of these SREEs has not been systematically assessed. This study aimed to review and to summarize the evidence in SREEs of cancer screening, their methodological quality as well as policy and research recommendations, to examine how well they can inform decisions. METHODS: We performed systematic searches on four bibliographic databases (Medline, Embase, EconLit, and the NHS Economic Evaluation Database) in June 2022. Two independent researchers screened the titles / abstracts, followed by full texts. We included SREEs on cancer screening published within 2012-2022. We examined the SREEs on the extent of reporting and evaluated their methodological quality using the AMSTAR-2 checklist. RESULTS: Of 766 unique articles screened, 30 SREEs were included. The SREEs examined screening for breast (23%), colorectal (23%), gastric (10%), liver cancers (10%) and 5 other cancers, using imaging methods (80%), laboratory testing (57%) or physical examinations (13%) in the general population (60%), hospital (10%), primary care (7%) or community (3%). While most SREEs reported economic evaluation methodologies, <50% reported screening uptake / adherence (37%) and none discussed equity issues. AMSTAR-2 ratings ranged 31-85%; most SREEs had duplicate screenings of articles (87%) but only a minority (6.7%) reported the funding sources of the included EEs. While majority SREEs were able to conclude on the relative cost-effectiveness of cancer screening, they also recommended further studies to address the uncertainty of evidence and lack of generalizability of findings to other settings. CONCLUSIONS: SREEs of cancer screening ranged widely in completeness of reporting and methodological quality. In addition, they may not adequately address all policy concerns e.g., implications on equity. Future SREEs and EEs should strive to address these gaps.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee60-ispor-europe-2023-tickell132021-pdf.pdf?sfvrsn=2efd1b2a_0","title":"EE60 ISPOR Europe 2023 Tickell132021.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/ee60-ispor-europe-2023-tickell-appendix132021-pdf.pdf?sfvrsn=f638941e_0","title":"EE60 ISPOR Europe 2023 Tickell Appendix132021.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132021","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Hepatocellular Carcinoma Patient Pathway in Portugal – From Diagnosis to Treatment – Results From Opal Study","id":"54f56c8e-9d00-47c3-a305-70b0c04f4cb6","sessionCode":"HSD12","topDisplay":"Moital I1, Fonseca F2, Pinto AR2, Bernardo F3 1Astrazeneca, Lisbon, Portugal, 2Astrazeneca, Lisbon, Lisbon, Portugal, 3Astrazeneca, Queluz de Baixo, Portugal","locationCode":"4018","description":"\r\n\t<div>OBJECTIVES: Hepatocellular Carcinoma (HCC) represents approximately 75% of all primary liver cancers. Main cause of HCC is cirrhosis resulting from alcohol misuse, hepatitis B (HBV) or C (HCV) and fatty liver disease. Although a decrease in HCC related to HCV and HBV is being observed, an increase of fatty liver disease as cause of HCC is increasing. Changes in major HCC etiology may increase diagnostic challenges, namely due to lack disease awareness, lack surveillance programs and involvement of new players (medical specialties). Complexity of disease treatment is also increasing with new treatment options, including immunotherapy not only for advanced disease but also for early stages. This analysis of OPAL-PT aims to develop an organized patient pathway registry that enables the Portuguese process of care understanding, including characterization of initial symptoms, type of healthcare units and medical specialties involved from diagnosis to treatment. METHODS: Real world, multi-centre, cohort, longitudinal study of patients with newly diagnosed HCC from January 1st, 2018 until December 31st, 2021. A building blocks framework was used to develop an organized patient pathway registry and collect clinical data from the electronic medical records for process of care characterization. Data collection is ongoing in 8 sites (minimum sample size of 250 patients). RESULTS: The building blocks structure developed was based on clinical experience of 8 sites. It consists a user-adaptive interface that allow physicians to select a personalized patient pathway. The framework includes 3 essential elements: Diagnosis & Disease characterization, Healthcare Services and Adverse Event and 9 drag and drop elements that assist the adaptative treatment sequency. CONCLUSIONS: The generated per hospital patient pathway allows the understanding of the different institutions process of care and improve HCC comprehensive care coordination. These results may lead to HCC management improvement and support future perspectives for multidisciplinary management and clinical practice optimization.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bernardo2hsd12poster133719-pdf.pdf?sfvrsn=f2075c5c_0","title":"ISPOREurope23_Bernardo_2_HSD12_POSTER133719.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133719","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Ocean (OP-103): Patients with Relapsed/Refractory Multiple Myeloma Treated with Melflufen Plus Dexamethasone or Pomalidomide Plus Dexamethasone - a Resource Utilization Analysis of Adverse Events Leading to Hospitalizations","id":"e407369f-1302-4318-aeac-712894b14bdf","sessionCode":"EE73","topDisplay":"Hellem Schjesvold F1, Ludwig H2, Delimpasi S3, Robak P4, Mateos MV5, Sandberg A6, Obermüller J6, Norin S6, Richardson PG7, Sonneveld P8 1Oslo University Hospital; University of Oslo, Oslo, Norway, 2Wilhelminen Cancer Research Institute, Vienna, Austria, 3Evangelismos Hospital, Athens, Greece, 4University Hospital Ostrava, Ostrava, Czech Republic, 5University Hospital of Salamanca/IBSAL/CIC/CIBERONC, Zamora, ZA, Spain, 6Oncopeptides AB, Stockholm, Sweden, 7Dana-Farber Cancer Institute, Boston, MA, USA, 8Erasmus MC Cancer Institute, Rotterdam, Netherlands","locationCode":"2014","description":"\r\n\t<div>OBJECTIVES: This analysis evaluates resource utilization in the OCEAN study of patients with relapsed/refractory multiple myeloma (RRMM) treated with melphalan flufenamide (melflufen) plus dexamethasone (dex) or pomalidomide (pom) plus dex, by looking at the frequency of treatment-emergent adverse events (TEAEs) leading to hospitalization. METHODS: Patients who received ≥1 dose of study treatment (safety population) were analyzed for the proportion of patients in each treatment arm with TEAEs leading to hospitalization >24 hours, and number of TEAEs leading to hospitalization >24 hours per treatment year. RESULTS: There were 226 (99.1%) and 241 (98.0%) patients in the melflufen+dex and pom+dex arms experiencing 3919 and 2306 TEAEs, respectively. The most common type of TEAE was hematologic, occurring in 93.4% and 64.6% of patients, respectively. In total, 131 and 166 TEAEs in 30.7% and 35.0% of patients led to hospitalization, respectively. The most common type of TEAEs leading to hospitalization were infections and infestations in 12.7% and 19.5% of patients, respectively, including infective pneumonias, occurring in 5.7% and 9.8%. Hematologic TEAEs led to hospitalization in 7.5% and 3.7% of patients, respectively. Other TEAEs that led to hospitalization included injury, poisoning, and procedural complications in 3.9% and 3.7% and cardiac disorders in 2.2% and 4.5% of patients, respectively. Hospitalizing TEAEs occurred at frequencies of 0.86 and 1.12 per treatment year in the melflufen+dex and pom+dex arms, respectively. Infections and infestations occurred at frequencies of 0.27 and 0.41 per treatment year in the melflufen+dex and pom+dex arms, respectively. CONCLUSIONS: Inpatient services for TEAEs had limited use in the OCEAN study. The most common reason for hospitalization was infections in both arms. Few of the hematologic TEAEs required hospitalization. Overall, hospitalizing TEAEs were less frequent with melflufen+dex compared with pom+dex.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23schjesvoldee73poster130622-pdf.pdf?sfvrsn=45cc7562_0","title":"ISPOREurope23_Schjesvold_EE73_POSTER130622.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130622","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Socio-Economic Value of Adult Immunization: Results from a Comprehensive Literature Review of Four Immunization Programs in Ten Countries","id":"5cda4374-cead-4bd3-8df7-7136a37c96eb","sessionCode":"EE136","topDisplay":"El Banhawi H1, Bell E1, Chowdhury S1, Brassel S2, Steuten L1 1Office of Health Economics, London, LON, UK, 2Office of Health Economics, London, UK","locationCode":"2065","description":"\r\n\t<div>OBJECTIVES: To assess and summarize the current evidence base for the broad socio-economic value of influenza, pneumococcal, herpes zoster, and respiratory syncytial virus (RSV) adult immunization programs in ten countries (Australia, Brazil, France, Germany, Italy, Japan, Poland, South Africa, Thailand, and the USA). METHODS: We undertook a structured literature review of evidence published in the last 5 years. Results were mapped onto a previously published framework including 11 value elements that collectively represent the broader socio-economic value of vaccination. For each individual program and country, we then visualized the quality/availability of evidence as well as the value of the vaccination program on each value element. RESULTS: There is strong evidence demonstrating the value of vaccination in terms of mortality, morbidity, cost-offsets, and productivity. A nascent evidence base also shows some positive effects with respect to social equity value and value in preventing antimicrobial resistance. Evidence indicates that increased uptake and coverage of existing vaccination programs, as well as expanding the sub-populations of adults included in immunization programs, could realize further benefits, and further increase cost-effectiveness. However, key gaps remain, including the effects of vaccination programs on carers and the enablement value of vaccines on other interventions. CONCLUSIONS: adult vaccination programs generate value across a broad range of socio-economic outcomes and expanding coverage of adult vaccination programs would further increase that. Gaps in the current evidence base also suggest that the literature currently underestimates the value of adult vaccination programs, and researchers and HTA practitioners should prioritize research into under-recognized, 'broad' dimensions of the value of adult vaccination programs such as social equity.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131147","diseases":[{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Adelphi Adherence Questionnaire (ADAQ©): Psychometric Validation of a New Patient Self-Report Measure of Medication Adherence Across Multiple Disease Areas","id":"bcc900dd-9c4a-4c86-831f-716c8c55800b","sessionCode":"PCR50","topDisplay":"Clarke N1, Bentley S1, Stochl J1, Higgins V2, Arbuckle R1, Piercy J2 1Adelphi Values Ltd, Bollington, Cheshire, UK, 2Adelphi Real World, Bollington, UK","locationCode":"6048","description":"\r\n\t<div>OBJECTIVES: The Adelphi Adherence Questionnaire (ADAQ©) is a patient-reported 13-item generic medication adherence instrument, developed with qualitative patient/clinician input. Measurement properties of the ADAQ© were evaluated across multiple disease areas, modes of administration, and treatment regimens. METHODS: Data were drawn from the Adelphi Real World Disease Specific Programmes (cross sectional surveys conducted in the USA) in Atopic Dermatitis (N=180), Heart Failure (N=544), Human Immunodeficiency Virus (HIV; N=247), Multiple Sclerosis (N=1337), and Osteoarthritis (OA; N=723). Item response distributions and inter-item polychoric correlations were examined. Latent variable modelling was used to evaluate the ADAQ© measurement model across disease areas (including exploratory and confirmatory factor analysis, bifactor modelling, exploratory graph analysis, Item Response Theory, and Mokken scaling). Reliability was estimated using coefficients alpha and omega. Convergent validity was evaluated using Spearman’s and polyserial correlations with the Adherence to Refills and Medications Scale (ARMS) and clinician-reported adherence/compliance where available. RESULTS: Consistent with previous research, potential item-level ceiling effects were observed across disease areas (i.e., large proportions of patients selecting the most adherent response for an item). Latent variable modelling consistently indicated that an 11-item scoring of the ADAQ© provided the best model fit across studies, with bifactor modelling suggesting the appropriateness of an essentially unidimensional measurement model. A scoring algorithm (the unweighted average of items 1 to 11) represents the overall ADAQ score, with items 12 and 13 providing additional information. Reliability coefficients omega hierarchical and alpha indicated the ADAQ© has excellent reliability (>0.9) across all disease areas. Convergent validity was demonstrated with the ADAQ© scores showing moderate-to-strong correlations with ARMS scores (0.591 in HIV to 0.774 in OA), and moderate correlations with clinician-reported adherence/compliance (all >|0.3|). CONCLUSIONS: Evidence across multiple disease areas supports use of an 11-item ADAQ© score as a unidimensional, reliable, and valid measure of patient-reported adherence to medication.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23arbucklepcr50poster133442-pdf.pdf?sfvrsn=6ee5ec46_0","title":"ISPOREurope23_Arbuckle_PCR50_POSTER133442.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133442","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Uncovering the Influence of Smoking on Health Care Processes for Adult Lung Cancer Patients in Chile: Insights From a Multi-Perspective Qualitative Study","id":"ffd8f2ec-aa0b-457e-a977-721457569d64","sessionCode":"PCR16","topDisplay":"Campaña Castillo C1, Cabieses B2, Obach A3, Vezzani F3, Espinoza MA2 1Universidad del Desarrollo, Santiago, RM, Chile, 2Centre for Cancer Prevention and Control (CECAN), FONDAP 152220002., Santiago, santiago, Chile, 3Universidad del Desarrollo, Santiago, Chile","locationCode":"6003","description":"\r\n\t<div>OBJECTIVES: To explore the perceived impact of smoking on the healthcare processes of adults with lung cancer in Chile, according to patients, health professionals and civil society leaders. METHODS: Qualitative research with a case-study design. Online semi-structured interviews were conducted with multiple relevant actors: adult patients with lung cancer (n=18), health professionals (n=8), and civil society leaders (n=1). Thematic analysis was conducted following the stages of the therapeutic trajectory of patients (TTP) and UDD´s scientific ethics committee approved this study. RESULTS: In general, participants perceived negative impacts of smoking in relation to lung cancer. At the beginning of the TTP, lung cancer patients who were smokers perceived being unaware of the country's smoking cessation programs, while others failed in their attempts to quit smoking. Misleading perceptions about the harmful effects of tobacco depending on the hours of smoking were identified. At the diagnosis stage, a perception of responsibility for having cancer as a smoker was reported. Some smoking patients only reduced tobacco consumption after diagnosis of lung cancer, which they explained as due to lack of information. Other patients experienced trying to quit smoking without professional follow-up and failed. It was also perceived that the language used by health professionals when informing the diagnosis was complex and violent, using phrases like \"you asked for it\". According to health professionals and leaders, tobacco consumer patients were less adherent and had worse outcomes at the treatment stage. High investment treatment for cancer was perceived by participants, but low investment in smoking cessation strategies. CONCLUSIONS: Participants perceived negative impacts of smoking in relation to lung cancer at different stages of the process. Stigma and discrimination against smoking patients was perceived, as well as the need to invest in smoking cessation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132190","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"How Is Real-World Evidence (RWE) Being Used in Health Technology Assessment (HTA) Decisions? Qualitative Insights from Comparisons of Three RWE Study Types Across Multiple Jurisdictions","id":"adb609e0-d63b-4cba-8c12-7282f0e8ddd7","sessionCode":"HTA72","topDisplay":"Hanisch M1, Johnson CE2, Marsico M2, Ramus C3 1Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA, 2Merck & Co., Inc., Upper Gwynedd, PA, USA, 3IQVIA, Washington, DC, USA","locationCode":"5009","description":"\r\n\t<div>OBJECTIVES: The role of RWE in HTA decisions is not well understood. We investigated the role of three RWE study types (external control, patient-reported outcome [PRO], retrospective/database) in HTA decisions, including commonly cited HTA critiques of RWE. METHODS: IQVIA’s HTA Accelerator® was used to identify published HTA decisions and develop 49 cases across 17 products. Publicly available reports were reviewed/coded for each case; synthesized findings included the submission contexts, purposes of submitted RWE studies, and whether the RWE studies were deemed fit-for-purpose. Twelve hour-long expert interviews added context to the case review findings. RESULTS: Many RWE study types have potential roles in HTA decisions, but we found that acceptance of RWE was context-dependent and varied across countries and by the type of scientific question being answered. In our analysis, external control studies contributed to decisions in only 33% of the cases, but did so more frequently than PRO (4%) or other retrospective (0%) studies. Critiques regarding RWE were related to generalizability (inconsistent standard of care, not generalizable to clinical practice, low patient numbers), data quality (completeness/accuracy, mis-defined outcome variables, insufficiently described data), confounding (confounding unmeasured/unjustified, naive comparisons), and data analysis (incorrect adjustment methods, missing/unclear analytical methods). HTA concerns stressed the importance of using country-specific data, aligning to current clinical practice, and submitting complete/sufficiently described RWE. CONCLUSIONS: Use of RWE in HTA decisions largely depends on study type, specific context of the submission, and meeting HTA expectations regarding confounding, generalizability, and data quality/analysis. While HTA policies regarding RWE highlight these shared basic concerns, RWE planning for HTA is encumbered by a need for greater alignment on specific issues including acceptable study designs and data fitness-for-purpose. Our findings suggest that early and iterative engagement between sponsors and HTA bodies is crucial to ensure that HTA decisions are informed by the highest-quality RWE.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hanischhta72poster130211-pdf.pdf?sfvrsn=9ef87df_0","title":"ISPOREurope23_Hanisch_HTA72_POSTER130211.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130211","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health-Related Quality of Life in the German Early Benefit Assessment—An Update","id":"23d5f7d6-fb64-4229-b87f-72e64847ccbc","sessionCode":"HTA69","topDisplay":"Kramer L1, Thaa B1, Moos M2, Esser M1 1co.value, Berlin, Germany, 2Pharm-Analytics GmbH, Hamburg, Germany","locationCode":"5013","description":"\r\n\t<div>OBJECTIVES: Health-related quality of life (HRQoL) is generally defined as the personal well-being in important areas of life including physical, mental, social, family-, and work-related factors. The inclusion of HRQoL data is regularly requested and a deciding factor in the German early benefit assessment. This research aims at defining the relevance of HRQoL in early benefit assessment by analyzing benefit assessments between 2011 and 2022. METHODS: Using the existing AMNOG database from Pharm-Analytics, all early benefit assessments since 01/01/2011 until 03/11/2022 were analyzed. All G-BA resolutions were assessed for consideration of HRQoL data, taking the disease area and the extent of additional benefit into account. RESULTS: Between 2011 and 2022 HRQoL data were considered by the G-BA in 352 of 793 completed benefit assessments (44%). Regarding disease areas, HRQoL data were considered most often in oncology in absolute numbers (n = 178), but proportionally most often in diseases of the urogenital system (5/8, 63%). Overall, HRQoL data were considered in 9 out of 12 assessments (75%) with the highest possible additional benefit (major additional benefit) but only 19% of assessments (66/345) without additional benefit. Most G-BA assessments in which HRQoL data were not taken into account show no additional benefit (279/431, 65%). Conversely, assessments in which HRQoL data were considered display a more equal distribution among the awarded benefit categories. CONCLUSIONS: Benefit assessments in which the G-BA considers HRQoL data correlate with a better outcome and a higher additional benefit than assessments in which HRQoL data are not taken into account. However, HRQoL data do not automatically lead to a benefit as the high methodical requirements posed by the G-BA need to be met. Overall, HRQoL data can be supportive for benefit assessment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kramerhta69poster131766-pdf.pdf?sfvrsn=4cce0b09_0","title":"ISPOREurope23_Kramer_HTA69_POSTER131766.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131766","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Unmet Medical Needs and Treatment Preferences for Systemic Treatments in Patients with Moderate to Severe Psoriasis: A Cross-Sectional Survey Study in China","id":"1197ce91-54f4-4d62-97e6-7308daa3426d","sessionCode":"PCR40","topDisplay":"Kuang Y1, Li Y2, Lv C3, Li M4, Zhang Z5, Chen Y6, Chen W7, Wang X8, Ba L8, Zhong Y9, Feldman SR10, Chen X1 1Xiangya Hospital of Central South University, Changsha, Hunan, China, 2The Second Affiliated Hospital of Harbin Medical University, Harbin, Heilongjiang, China, 3Dalian Dermatological Diseases Hospital, Dalian, Liaoning, China, 4Dushu Lake Hospital of Suzhou University, Suzhou, Jiangsu, China, 5The 8th Affiliated Hospital of Zhongshan University, Shenzhen, Guangdong, China, 6Changsha Normin Health Technology Ltd, Changsha, Hunan, China, 7Normin Health Consulting Ltd, Mississauga, ON, Canada, 8Bristol Myers Squibb-China, Shanghai, Shanghai, China, 9Bristol Myers Squibb, Lawrenceville, NJ, USA, 10Wake Forest School of Medicine, Winston-Salem, NC, USA","locationCode":"6038","description":"\r\n\t<div>OBJECTIVES: To assess the unmet needs and treatment preferences among Chinese patients with moderate to severe psoriasis (msPsO). METHODS: 300 msPsO patients previously treated with systemic therapies were surveyed in five Chinese tertiary hospitals. The unmet needs defined as treatment effectiveness, safety, convenience, and challenges were assessed for oral drugs (acitretin, methotrexate, cyclosporine, apremilast) and biologics (adalimumab, infliximab, etanercept, secukinumab, ixekizumab, ustekinumab, guselkumab). Treatment preferences were evaluated by a choice-based conjoint (CBC) questionnaire with seven medication attributes for administration route, frequency, effectiveness, and safety. Descriptive statistics were employed to summarize the data; conjoint simulation analyses were conducted to assess the treatment preference. RESULTS: Mean age of these patients was 43 years old, 63% were males, and 41% were with severe psoriasis. The treatment persistence of oral drugs and biologics were 2.7-6.2 months and 9.5-17.0 months, respectively. The impacts of treatment side effects were reduced quality of life (38%) for oral drugs and increased expenditure related to managing adverse events (AE) for biologics (29%). The most frequently reported treatment inconveniences included regular lab tests to monitor AE of oral drugs (other than apremilast) (49%) and injection preparation for biologics (27.8%). The primary treatment discontinuation reasons were unsatisfactory effectiveness (77%) for oral drugs and loss of efficacy over time (61%) for biologics. The reported treatment challenges with biologics also contained administration assistance needs from healthcare providers (68%), limited access (47%), injection pain (32%), needle fear (31%), injection preparation (28%), and paradoxical skin reactions (6-21%). When attributes were comparable, oral drugs were preferred over subcutaneous injection drugs for moderate (64% vs. 36%) and severe psoriasis (61% vs. 39%). CONCLUSIONS: This study highlights the unmet needs of current msPsO treatments for effectiveness, safety, convenience, and challenges in Chinese patients. For treatments with comparable medication attributes, Chinese patients with msPsO prefer oral treatment over injection treatment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23chenpcr40poster132460-pdf.pdf?sfvrsn=ef0156e5_0","title":"ISPOREurope23_Chen_PCR40_POSTER132460.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132460","diseases":[{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Oral Health behind the Bars: Oral Health Seeking Behavior Among Jail Prisoners in Khyber Pakhtunkhwa, Pakistan","id":"7f27a4ec-f9c9-4c84-998a-739acb416ead","sessionCode":"EPH36","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3037","description":"\r\n\t<div>OBJECTIVES: To determine the oral health care-seeking behavior and its associated factors among prison inmates METHODS: This cross-sectional study was conducted at Central Prisoner Jail, Peshawar Khyber Pakhtunkhwa, Pakistan from November 2021 to April 2022. A consecutive sampling technique was used from both convicted and under-trial prisoners by using a pre-tested WHO Basic Oral Health Survey 2013 tool. Our outcome variable was “Visit to a dentist in the last 12 months (Never/Once or more than once). Chi square test was used for association with all other explanatory variables while multivariable logistic regression was performed to adjust for potential confounders. RESULTS: A total of 225 participants were recruited to the study with a mean (SD) age of 32.9 (11.4). More than two-thirds 200(88.9%)of the participants were males. One third of the sample never visit to the dentist75(33.3). The frequency of dental visits was higher in males as compared to females, n=132 (66.0%,p-value =0.54). Participants who completed college/university education and never visited the dentist in the last 12 months constituted a smaller proportion (17.6%) compared to those who visited the dentist once or more then once n=28(82.4%,p-value = 0.003).Individuals who were using toothbrush were most frequently visiting to the dentist n=130(72.6%=p value=0.001) as compare to never visitor. Multivariate logistic regression analysis revealed that Participants who experienced teeth pain or discomfort having 0.42 times lower odds of visiting the dentist compared to those who did not experience any pain or discomfort [AOR 0.42 (95% CI 0.17-0.80),p= 0.04].Similarly, Participants who do not use any denture have 4.06 times higher odds[AOR 4.06(95% CI 1.76-9.36),p= 0.001] of visiting the dentist compared to those who use a denture. CONCLUSIONS: Prisoners who were experiencing teeth pain or discomfort and not using dentures were the strong predicators with lower dental visit frequency to seek oral health care behavior by jail prisoners <quillbot-extension-portal></quillbot-extension-portal> <quillbot-extension-portal></quillbot-extension-portal> <quillbot-extension-portal></quillbot-extension-portal></div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131282","diseases":[{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Prevalence and Comorbidities of Prurigo Nodularis – A Danish Register-Based Study From 1995 to 2021","id":"8d1e54c2-2e4c-4478-bb81-746aab5ea53b","sessionCode":"EPH55","topDisplay":"Ibler KS1, Torpet M2, Lyngsie PJ3, Olsen J4, Münster M4, Elberling J5 1Zealand University Roskilde, Roskilde, Denmark, 2Sanofi, Copenhagen Oe, Denmark, 3Sanofi, Copenhagen, Denmark, 4EY, Frederiksberg, Denmark, 5Herlev and Gentofte Hospital, Hellerup, Denmark","locationCode":"3052","description":"\r\n\t<div>OBJECTIVES: Prurigo Nodularis (PN), or chronic nodular prurigo, is a rare chronic skin disease, characterised by multiple itchy, firm nodules, usually with a symmetrically distribution on the torso and the extensor sides of the extremities. PN severely affects the quality of life. The prevalence across countries has been estimated between 8,4-210 per 100.000. We aimed to estimate the prevalence of PN and explore the occurrence as well as the chronology of associated comorbidities at the hospital setting in Denmark. METHODS: Patients with PN were identified using the ICD10-code L28.1 (PN) as their primary or secondary diagnosis in the Danish National Patient Register (DNPR) between 1995 and 2021. The comorbidities such as diabetes, asthma and chronic obstructive pulmonary disease (COPD), identified before and/or after the first diagnosis with PN, were identified in the DNPR. RESULTS: During the study period, 1,207 Danish citizens with PN diagnosis were identified (mean age at diagnosis: 59 years, 58% were female). Ultimo 2021, the number was 830 patients (total Danish population 5,900,000) giving a prevalence of 14 per 100.000. A total of 437 (35%) had no associated comorbidities before being diagnosed with PN. After diagnosis, 358 (28%) patients did not have any diagnosed comorbidities. We found that 640 (53%) PN patients were diagnosed with minimum one comorbidity before being diagnosed with PN, while 67% of the PN patients were diagnosed with minimum one comorbidity after diagnosis. Thus, PN is associated with many comorbidities and the three most prevalent were found to be COPD, asthma and type 2 diabetes. CONCLUSIONS: Using the ICD10-code L28.1 the prevalence of PN is somewhat lower than expected. One explanation could be that a great fraction of patients with PN are categorised with other primary diagnosis such as AD. Two thirds of the identified PN patients had other diseases.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23olseneph55poster133571-pdf.pdf?sfvrsn=bcb17c82_0","title":"ISPOREurope23_Olsen_EPH55_POSTER133571.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133571","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patient and Caregiver Perceptions of Systemic Anticancer Treatments in Advanced Bladder Cancer (aBC): Results of a Social Media Listening (SML) Study Conducted in 5 European Countries (Eu5)","id":"9d8e1e15-932c-416c-a8f6-748373e140dc","sessionCode":"PCR24","topDisplay":"Schück S1, Loussikian P1, Mebarki A1, Malaab J1, Foulquié P1, Talmatkadi M1, Kearney M2 1Kap Code, Paris, France, 2Merck Healthcare KGaA, Darmstadt, HE, Germany","locationCode":"6022","description":"\r\n\t<div>OBJECTIVES: Although recent therapeutic advances in aBC have been made, little is known about patient and caregiver perceptions of different systemic anticancer treatments. To further explore this, we conducted an SML study to assess patient and caregiver perceptions of chemotherapy and immunotherapy (IO) for aBC using social media–posted data. METHODS: This retrospective, real-world study evaluated written public posts on geolocated social media in Eu5 (France, Germany, Spain, Italy, and the UK) posted from October 2017 to October 2022. Natural language processing methods were used to filter out irrelevant content and identify posts from patients and caregivers. Posts mentioning any line of chemotherapy or IO were qualitatively analyzed by 2 researchers to classify treatment perceptions (positive, negative, mixed, or without perception). RESULTS: 1,670 posts from 1,396 users discussing aBC in 98 public online sources were identified (699 posts from 546 patients, 971 posts from 850 caregivers). Half of patients were male (272 [49.8%]) and most caregivers were female (474 [55.8%]); average age was 52.8 and 35.2 years, respectively. Among posts mentioning chemotherapy (n=553), 58.6% did not express any opinion; in posts that did, negative perceptions (23.2%) were more commonly reported than positive (9.2%). Perceived chemotherapy benefits, as identified in 69 posts, were effectiveness (35.4%), tumor reduction (14.5%), and treatment tolerance (13.0%). In posts mentioning IO and expressing an opinion (n=52/115), 21.7% expressed a positive perception and 9.6% negative. Among those mentioning perceived benefits of IO (n=20/52), 50% cited its effectiveness. CONCLUSIONS: This qualitative study provides valuable insights on how to improve aBC treatment experience among patients and caregivers. Strengthening support for patients receiving chemotherapy may help them and their caregivers manage side effects and improve their perceptions. This could enable more patients to achieve disease control and become eligible to receive avelumab first-line maintenance treatment to obtain survival benefits.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kearneypcr24posterv2129057-pdf.pdf?sfvrsn=9368300_0","title":"ISPOREurope23_Kearney_PCR24_POSTERv2129057.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129057","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Cost Effectiveness of Pharmaceutical Management for High-Risk Older Persons Living in Residential Housing","id":"9fa014ea-84d7-45ca-b7d5-74f3f58e561a","sessionCode":"EPH1","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3004","description":"\r\n\t<div>OBJECTIVES: The cost-effectiveness of community based cardiovascular disease (CVD) risk-reduction interventions for older adults in residential settings is not well established. Support and Services at Home (SASH) is an intervention designed to reduce modifiable CVD risk factors through improved medication management and behavioral interventions. This program has previously demonstrated success, but the cost-effectiveness is unknown. Community based interventions for older, high-risk population are potentially attractive complements to healthcare delivery a lower cost relative to medical care and may be attractive in rural settings with limited clinical resources. METHODS: SASH performed screenings and provided ongoing participant support within predominantly rural communities. We calculated quality-adjusted life years (QALYs) gained and program costs using a Markov model. Transition probabilities were calculated using Framingham risk equations or derived from the literature using the observed mean reduction in 10-year CVD risk score over of 24 months follow-up. Program cost-effectiveness was calculated for both at-risk (abnormal baseline CVD risk factors) and overall program populations. Costs and benefits were discounted at r=3%. RESULTS: The base-case scenario evaluating a 75-year-old male participant revealed an incremental cost savings of $4320 and a gain of 0.33 QALYs associated with the intervention. Cost savings were greater in at-risk populations. The economic dominance of the model was robust in multiple sensitivity analyses. CONCLUSIONS: This interventions targeted at high-risk community-based residential older individuals to reduce CVD risk is cost-effective. They key elements were the improved use of medications and reductions in smoking rates. This suggests that population-based public health programs may have the potential to complement primary care preventative services to improve health and reduce the burden of traditional medical care.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133938","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Trade-Off between Comparators Comprehensiveness and Methodological Robustness in Cost-Utility Studies Reviewed By Has in France","id":"53cfd260-dee4-4214-8739-758af5625de6","sessionCode":"EE151","topDisplay":"Kirion J, Alaoui E, Robert J, Baffert S Cemka, Bourg-la-Reine, France","locationCode":"2074","description":"\r\n\t<div>OBJECTIVES: In the absence of direct comparisons between alternative healthcare interventions within cost-utility studies, the French National Authority for Health (HAS) recommends performing network meta-analysis (NMA) to estimate their relative effectiveness. NMA is not always feasible whenever a common comparator between the selected alternatives is lacking. Several indirect pairwise comparisons, such as MAIC and propensity score, can then be performed. The aim of our study was to evaluate how these approaches were accepted in HAS opinions about dossiers currently published. METHODS: A retrospective analysis of the HAS opinions published until December 31, 2022 was carried out. RESULTS: 79 dossiers were analyzed from 2020 to 2022. To date, the HAS has not accepted the validity of pairwise comparison methods to assess the efficiency of a new treatment. In the opinions that included several indirect comparisons instead of a NMA, beyond the remarks on the heterogeneity of the studies that questioned the possibility of conducting valid indirect comparisons, the impossibility of constructing an efficiency frontier and therefore of concluding on the efficiency of the assessed product was underlined. In evaluations based on pairwise comparisons, two ICERs will be estimated making it difficult to draw any final conclusion. The HAS suggested checking whether one of the analyses could be considered more relevant and able to support the main analysis. CONCLUSIONS: The use of pairwise comparisons to obtain a multi-option efficiency frontier was not validated so far by the HAS. In these cases, the HAS recommends that only one comparison be retained to respect methodological validity, despite lack of comparators comprehensiveness in the base case analysis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23alaoui132544poster132544-pdf.pdf?sfvrsn=46d5f39d_0","title":"ISPOREurope23_ALAOUI_132544_POSTER132544.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132544","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Identifying Predictors of Response Following Discontinuation of JAKi Therapy in Patients with Rheumatoid Arthritis","id":"99eeb5e0-c4ac-4898-bbd2-765b0563bcb5","sessionCode":"RWD9","topDisplay":"De Cock D1, Durez P2, Lenaerts J3, Westhovens R4, Verschueren P4 1VUB, Jette, Belgium, 2Cliniques Universitaires Saint-Luc, Brussel, Brussel, Belgium, 3Reuma-Instituut Hasselt, Hasselt, Limburg, Belgium, 4University Hospitals Leuven, Leuven, Vlaams Brabant, Belgium","locationCode":"6062","description":"\r\n\t<div>OBJECTIVES: Previous research from the real-world evidence TARDIS register indicated that IL6 inhibitors showed superior clinical response after JAKi cessation compared to other b/tsDMARDs in rheumatoid arthritis (RA). However, baseline differences in demographic and clinical variables, as well as prior exposure to advanced therapies, were observed among the treatment groups, potentially influencing the findings. Therefore, the objective was to identify factors that could predict clinical responses. METHODS: A total of 193 RA patients with complete baseline and follow-up data were included in the analysis. Patients were categorized based on the subsequent therapy they received: TNFi, Abatacept, IL6 inhibition, or JAKi. Linear and logistic regression models were constructed, with changes in Disease Activity Score 28 (DAS28) between baseline and first follow-up, DAS28 remission (DAS28 < 2.6), and DAS28 low disease activity (LDA, DAS28 ≤ 3.2) as dependent variables. Various demographic and clinical variables were incorporated into the models, and backward elimination was performed to identify statistically significant predictors. RESULTS: The results showed that 33%(63/193) of patients initiated TNFi therapy, 11%(20/193) received Abatacept, 18%(34/193) underwent IL6 inhibition, and 39%(76/193) were prescribed another JAKi therapy. Remission and LDA were achieved in 42%(81/193) and 63%(122/193) of patients, respectively. The analysis revealed that baseline Health Assessment Questionnaire (HAQ) score and not receiving abatacept were common predictors for achieving remission and LDA. Factors positively associated with a greater change in DAS28 included baseline Patient Global Assessment (PGA)(Relative Risk(RR)0.02(0.01,0.03)), tender joint count (TJC)(RR(0.06(0.01,0.12)), swollen joint count (SJC) (RR0.10(0.03,0.16)), and C-reactive protein (CRP) (RR0.02(0.01,0.03)). Conversely, baseline HAQ score (RR-0.60(-0.88,-0.31)) and not receiving abatacept (RR-0.70(-1.25,-0.15)) were negatively correlated with DAS28 change in the adjusted multivariate linear regression model. CONCLUSIONS: Results indicated that, after adjustment, patients with poorer functionality and those receiving abatacept compared to other b/tsDMARDs exhibited less favorable short-term clinical outcomes. Further investigation is necessary due to the relatively small sample size.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterispor202320230611131131-pdf.pdf?sfvrsn=d81385f6_0","title":"Poster_ISPOR2023_20230611131131.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131131","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Impact of Co-Locating GP-Led Minor Injury Clinic on Pediatric Emergency Department","id":"eb654c19-4cd7-4860-99bc-76a050ae1145","sessionCode":"HSD13","topDisplay":"Pak A The University of Queensland, St Lucia, QLD, Australia","locationCode":"4023","description":"\r\n\t<div>BACKGROUND: Co-locating a GP clinic proximate to a paediatric ED could potentially alleviate overcrowding, reduce waiting times for patients with minor illnesses, and create a more efficient pathway for appropriate care. In 2002, the Queensland Children’s Hospital (QCH), in partnership with UQ Health Care (UQHC), initiated a trial to establish Paediatric Minor Injury and Illness Clinic (P-MIIC) proximate to QCH’s ED. OBJECTIVES: To evaluate the impact of implementing P-MIIC from the perspectives of patients and clinical staff. METHODS: Comparison of patient experience was conducted between P-MIIC patients and ‘GP appropriate’ patients who were treated in the ED. Data were collected through a validated patient experience survey. The analysis included the evaluation of the uptake of services, length of stay and waiting time to treatment. De-identified administrative patient data (QCH=12,166; P-MIIC = 971) was used for analysis. During the trial, P-MIIC was closed due to staffing shortages for a two-week period. We utilised this exogenous interruption to analyse the impact on QCH ED operations. RESULTS: P-MIIC patients exhibited more positive responses than QCH ED patients with over 90% of P-MIIC patients selecting the highest rating for all experience measures. During its operational hours, P-MIIC provided support to QCH ED and attended to 9% to 24% of 'GP appropriate' QCH ED presentations, depending on the time of day. With P-MIIC in operations, both QCH ED median total and waiting times were lower by 19 (10%) and 11 (11%) minutes respectively. CONCLUSIONS: The implementation of P-MIIC positively impacted patient experiences and reduced waiting times in QCH ED. The utilisation of P-MIIC varied, and its absence during busy periods contributed to increased waiting times in ED. These results highlight the potential benefits of incorporating a primary care model like P-MIIC in a hospital setting to enhance patient experiences and improve efficiency in emergency care.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23pakhsd13poster133003-pdf.pdf?sfvrsn=3e2e7947_0","title":"ISPOREurope23_Pak_HSD13_POSTER133003.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133003","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"EQ-5D-5L Valuation Studies: Do the Effects of Dimensional Health Problems Interact with Each Other?","id":"98dd2bbf-2d18-402e-9204-76b96dd5a73b","sessionCode":"MSR23","topDisplay":"Vasan Thakumar A, Luo N National University of Singapore, Singapore, Singapore, Singapore","locationCode":"5074","description":"\r\n\t<div>OBJECTIVES: Studies have shown the disutility of having multiple health problems is usually smaller than the disutility sum of individual component health problems. However, existing model specifications for predicting utility values of the multi-dimensional EQ-5D health states do not reflect this phenomenon. This study aimed to evaluate the two-way interaction effects between health dimensions on modelling of EQ-5D values. METHODS: We tested 10 two-way dimensional interactions with 16 EQ-5D-5L valuation datasets, each derived from a different country/district using the same study protocol. We generated interaction terms by treating dimensional problem levels of EQ-5D-5L health states as continuous variables, and added them into a 20-parameter (incremental) main-effects model for predicting composite time trade-off (cTTO) values. Model performance was assessed in terms of: i) parameter statistical significance; ii) predicted health-state values’ logical consistency; and iii) out-of-sample prediction accuracy using mean square error (MSQE), and Pearson’s R (R), also compared with that of the main-effects model. RESULTS: In 15 countries, there was minimally one significant interaction term, with the median (interquartile) of significant interaction terms being 3 (1 to 4). In 9 countries, including interactions increased the number of significant main-effect terms. Most significant interactions occurred between the pain/discomfort and anxiety/depression dimensions (n= 9 countries) and interactions mainly exhibited diminished joint disutility. Incorporating interaction terms resulted in lower MSQE (mean reduction: 24.6%; SE: 5.74) with higher R (mean increase: 0.6%; SE: 0.20) in 13 of 15 countries. Additionally, logically inconsistent predictions for health-state pairs with dominance relationship was minimal, ranging from 0% to 0.48%. CONCLUSIONS: Significant dimensional interaction effects existed in EQ-5D-5L health-state valuation. The interactions existed, behaved as expected, and improved data modelling in most countries studied. Future valuation studies for multi-dimensional health descriptive systems such as EQ-5D should consider exploring interaction effects between health dimensions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23vasanthakumarmsr23posterv2130739-pdf.pdf?sfvrsn=53423ffc_0","title":"ISPOREurope23_VasanThakumar_MSR23_POSTERV2130739.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130739","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Difelikefalin for the Treatment of Moderate to Severe Chronic Kidney Disease-Associated Pruritus (CKD-AP) in Adult Patients Receiving in-Centre Haemodialysis","id":"98428061-2d96-488f-b8fd-76c33a50e4c3","sessionCode":"EE120","topDisplay":"Collins C1, Edmonds T2, Taylor I3, Mumford A4, Darlington O5, Schaufler T6, Soro M7, Baxter G6 1Initiate Consultancy, Alderton, NTH, UK, 2Initiate Consultancy, Nottingham, NGM, UK, 3Initiate Consultancy, Fowey, UK, 4Initiate Consultancy, Northampton, UK, 5Initiate Consultancy, NA, UK, 6CSL Vifor, Glattbrugg, ZH, Switzerland, 7CSL Vifor, Rome, Italy","locationCode":"2050","description":"\r\n\t<div>OBJECTIVES: Chronic kidney disease associated pruritus (CKD-aP) is linked to poor health-related quality of life (HRQoL), as well as increased risks of adverse health outcomes. Difelikefalin was demonstrated to be an efficacious treatment for moderate to severe CKD-aP in two phase III trials: KALM-1 and KALM-2. The objective of this study was to estimate the cost-effectiveness of difelikefalin in addition to best supportive care (BSC) for the treatment of adults with moderate to severe CKD-aP undergoing in-centre haemodialysis, from a UK payer perspective. METHODS: A lifetime Markov model comprising five health states defined by itch severity was constructed to estimate costs and outcomes for patients treated with difelikefalin plus BSC or BSC alone. CKD-aP progression was modelled based on 5-D Itch scale severity measurements from the KALM trials. Mortality and transplant rates were modelled using time-dependent probabilities from the UK Renal Registry. Relative risk of hospitalisation and mortality were applied based on itch severity, informed by the Dialysis Outcomes and Practice Patterns Study (DOPPS). Utility estimates and costs were sourced from literature and the NHS cost collection, and discounted at 3.5% annually. RESULTS: Treatment with difelikefalin with BSC compared with BSC alone was associated with increased life expectancy (0.11 years per person) and increased quality adjusted life years (QALYs, 0.26 per person), at an incremental cost of £7,814 per person. Benefits were driven by reductions in itch severity and consequently improved HRQoL and reduced mortality risk, along with reductions in healthcare resource use and concomitant medications. Difelikefalin was estimated to be cost-effective at a willingness-to-pay threshold of £30,000/QALY at a cost of £31.90/vial. CONCLUSIONS: Difelikefalin plus BSC is a cost-effective treatment for CKD-aP versus BSC alone, with the potential to ameliorate the significant burden CKD-aP imposes on patients in the UK.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee120---csl-vifor-initiate-ispor-poster132533-pdf.pdf?sfvrsn=4d03790d_0","title":"EE120 - CSL Vifor Initiate ISPOR Poster132533.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132533","diseases":[{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Risk of Death Before and After Metastasis in High-Risk Localized and Locally Advanced Prostate Cancer Patients Undergoing Radical Prostatectomy or Radiotherapy as Primary Treatment in the United States: A Retrospective Study","id":"445f2104-e78d-4553-9eb1-772c4ecbf042","sessionCode":"RWD19","topDisplay":"Freedland S1, Fernandes L2, De Solda F3, Buyukkaramikli N4, Labson D3, Yang L3, Pan F3, Mir MC5 1Cedars-Sinai Medical Center, Los Angeles, CA, USA, 2Janssen Pharmaceutica N.V., Beerse, Belgium, 3Janssen Global Services LLC, Raritan, NJ, USA, 4Janssen Pharmaceutica N.V., Beerse, Antwerp, Belgium, 5IMED Robotic Surgery Unit, Valencia, V, Spain","locationCode":"6068","description":"\r\n\t<div>OBJECTIVES: It is well-known that prostate cancer (PC) death rates increase after metastasis, but the magnitude of the change is unclear, especially with recent novel therapies in metastatic PC. We examined changes in mortality after metastasis in high-risk localised and locally advanced PC (HR-LPC/LAPC) undergoing primary treatment with radical prostatectomy (RP) or radiotherapy with/without androgen deprivation therapy (RT±ADT). METHODS: ConcertAI Patient360TM, which provides data from predominantly medical oncologists, was queried from January 2000 – October 2022 for men with HR-LPC/LAPC who underwent primary treatment with either RP or RT±ADT. Pre-/post-metastasis survival was analysed using Kaplan-Meier estimation to generate pre-/post-metastasis 5-year mortality rates. 5-year Standardised Mortality Ratios (SMR) were computed as the ratio of observed 5-year mortality rate in HR-LPC/LAPC patients receiving RP or RT±ADT to the age-adjusted mortality rate for the general male population in the United States. RESULTS: Among 5,008 patients identified as HR-LPC/LAPC patients, 1,696 underwent RP and 686 RT±ADT. Patients undergoing RP were younger (median age at RP=63 vs RT±ADT=69). During follow-up (median=7.5 years), around half of the patients developed metastases in both groups. In RP patients, the relative risk of death increased after metastasis by 8-fold [95% Confidence Interval (CI) 6.16,9.93] (5-year pre-metastasis SMR: 0.50 [95% CI 0.39,0.62]; and 5-year post-metastasis SMR: 3.86 [95% CI 3.62,4.11]). In the RT±ADT group, the increase was 5-fold [95% CI 3.97,6.84] (5-year pre-metastasis SMR: 0.72 [95% CI 0.55,0.91]; and 5-year post-metastasis SMR: 3.71 [95% CI 3.32,4.07]). CONCLUSIONS: This retrospective study of patients seen predominantly by medical oncologists demonstrated that mortality risk in HR-LPC/LAPC patients significantly increased after metastasis, suggesting therapies delaying disease progression in this setting may improve overall survival. Since study data comes predominantly from oncology practices likely explaining the bias of very high metastases rates, future research can consider alternative data sources (i.e., data from urology practices) to further investigate this topic.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23freedlandrwd19poster128051-pdf.pdf?sfvrsn=428b39bf_0","title":"ISPOREurope23_Freedland_RWD19_POSTER128051.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128051","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Development of an Algorithm to Identify the Type of Diabetes in the French Administrative Health Care Database “Système National Des Données De Santé” (SNDS)","id":"df852c78-0f02-4f61-ad6c-77b81ae8fbfd","sessionCode":"MSR2","topDisplay":"Bretin O1, Casarotto E1, Bessou A1, Maurel F1, Serusclat P2, Joubert M3, Fagherazzi G4, Berteau C5, Pouyet A6, Maillard C7 1IQVIA France, Courbevoie, France, 2Groupe Hospitalier Mutualiste Les Portes du Sud, Venissieux, France, 3CHU de Caen, Caen, France, 4Paris south Paris Saclay University, Villejuif, France, 5Roche Diabetes Care France, Meylan, 38, France, 6TIMKL, Montbonnot Saint Martin, France, 7IQVIA Opérations France, La défense, France","locationCode":"5048","description":"\r\n\t<div>OBJECTIVES: The French administrative health care database (SNDS), covering 99% of the French population, is a powerful tool for epidemiological and pharmacoeconomic studies on diabetes. However, its lack of clinical information makes it difficult to accurately identify the type of diabetes. The objective was to develop an accurate machine learning algorithm to determine the type of diabetes in the SNDS, validated thanks to a linkage with primary care clinical data. METHODS: Electronic medical records (EMR) of a network of French general practitioners (GP) were probabilistically linked with the SNDS. This linkage allowed to constitute a population of diabetic patients whose type of diabetes was retrieved from GP consultations. About 200 predictors were derived from SNDS data to help discriminate between type-1 diabetes (T1D) and type-2 diabetes (T2D). Various machine learning algorithms (penalized logistic regressions, RandomForest, XGBoost) were trained and optimized by a 10-fold cross-validation procedure on the training set. The best model was selected for its ability to predict T1D on the test set, via the F1-score metric. Its performance was benchmarked against already-published algorithms applied to the test set. RESULTS: A cohort of 40,774 people with diabetes was constituted, including 39,122 (95.9%) T2D and 1,652 (4.1%) T1D. A LASSO penalized regression obtained the best performance (F1: 0.79 (T1D); precisions: 84.6% (T1D), 98.9% (T2D); sensitivities: 73.8% (T1D), 99.4% (T2D)), outperforming the Charbonnel’s decision tree (F1: 0.66) and Fuentes’s best retrained logistic regression (F1: 0.59). CONCLUSIONS: Thanks to an innovative linkage between SNDS and EMR, we have developed a high-performance classification model that outperforms existing published algorithms to identify the type of diabetes in a large medico-administrative database. It can be reused by the scientific community to conduct epidemiological and pharmacoeconomic studies on each type of diabetes in the French population.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/epicodiabposterisporiqviavf132649-pdf.pdf?sfvrsn=82354d2a_0","title":"Epicodiab_Poster_ISPOR_IQVIA_VF132649.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132649","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Demonstrating Similar or Greater Health Benefits Based on Indirect Evidence: A Review of NICE Evaluations That Include a Cost-Comparison Approach","id":"53f5b3dc-696e-4c71-9def-78af61dff140","sessionCode":"HTA34","topDisplay":"Haycock M1, Willows LP2, Wickstead R1 1Costello Medical, London, UK, 2Costello Medical, Cambridge, UK","locationCode":"4072","description":"\r\n\t<div>OBJECTIVES: The National Institute for Health and Care Excellence (NICE) cost-comparison pathway (formerly fast-track appraisal pathway) is an expedited reimbursement route for interventions that demonstrate similar/greater health benefits, at similar/lower costs, than technologies already recommended for the same indication. Indirect treatment comparisons (ITCs) are increasingly used to meet these criteria but can be associated with high uncertainty. We reviewed all NICE evaluations using cost-comparison analyses to understand the indirect evidence used and accepted by NICE to demonstrate similar/greater health benefits. METHODS: NICE evaluations containing cost-comparison analyses with final guidance published between April 2017 and May 2023 were identified. Details of the evidence used to demonstrate similar/greater health benefits for the intervention were extracted, including the ITC methodology and volume of evidence presented. RESULTS: 19 cost-comparison and 11 single-technology appraisal (STA) evaluations with cost-comparison analyses were identified. 26/30 (86.7%) demonstrated similar/greater health benefits between the intervention and at least one relevant comparator using an ITC; 20/26 (76.9%) of these used a network meta-analysis. Considering the primary clinical endpoint, 3/26 (11.5%) demonstrated statistically significant superiority for the intervention versus comparator(s), 9/26 (34.6%) demonstrated no statistically significant difference, and 5/26 (19.2%) described ‘comparable’ results. Results were redacted for the remainder (34.6%). 2/26 (7.7%) evaluations demonstrated equal efficacy using one endpoint, 5/26 (19.2%) using two, 3/26 (11.5%) using three, and 16/36 (61.5%) using four or more. 18/26 (69.2%) and 2/26 (7.7%) evaluations also demonstrated similar/greater safety and health-related quality of life outcomes through an ITC, respectively. CONCLUSIONS: There was considerable variation in the indirect evidence used and accepted to demonstrate similar/greater benefits for the intervention. A greater understanding of the threshold of evidence required to support these criteria is needed, particularly with the introduction of NICE’s proportionate approach, which may lead to increasing use of the cost-comparison pathway to expedite patient access whilst reducing required resource.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/cost-comparison131666-pdf.pdf?sfvrsn=f55c922f_0","title":"cost-comparison131666.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131666","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Analysis of Public Health Insurance Expenditures in Turkiye By SGK between 2020-2021-2022","id":"cd46aa7d-2159-4e96-bbcf-78e4b2d40ee8","sessionCode":"HPR34","topDisplay":"Erdoğan A1, Kurnaz M1, Okcun S1, Kockaya G2 1ECONiX Research, Istanbul, Turkey, 2ECONiX Research, Samsun, 55, Turkey","locationCode":"4005","description":"\r\n\t<div>OBJECTIVES: Policy of implementing low prices for pharmaceuticals and medical devices aims to ensure affordability and accessibility of healthcare products for the population. The aim of this study is to analyze the change in the budgets of the Social Security Institution (SGK) and General Health Insurance (GHI) in Turkiye between 2020-2021-2022 and the factors affecting this change. METHODS: The data for this study were obtained from official sources, including the SGK and the Ministry of Treasury and Finance, specifically the data published for the year 2022. The analysis was conducted using Microsoft Excel. Key variables examined in the study included the number of applications to state, university, and private hospitals, the number of invoices, the number of prescriptions, the total SGK budget, and the actual health expenditures for the years 2020, 2021, and 2022. Changes in these variables over time were analyzed. RESULTS: According to the analysis, number of applications increased by 20% in 2021 compared to 2020, while amount of application invoices increased by 38%. In 2021, number of prescriptions increased by 19% and amount of prescription invoices increased by 26% compared to 2020. Number of applications increased by 25% in 2022 compared to 2021, while amount of application invoices increased by 57%. In 2022, number of prescriptions increased by 13% and amount of prescription invoices increased by 68% compared to 2021. When health expenditures and collected premiums by SGK are examined, it is seen that there were 27 billion TL, 13 billion TL and 30 billion TL budget surplus in 2020, 2021 and 2022, respectively. CONCLUSIONS: The analysis demonstrates that the SGK has experienced budget surpluses in recent years, with the highest surplus observed in 2022. This can be attributed to the low-price policy implemented for pharmaceuticals and medical devices, which has resulted in cost savings for the SGK.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/public-health-insurance130923-pdf.pdf?sfvrsn=64b0bbb3_0","title":"Public Health Insurance130923.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130923","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Facilitating and Accelerating Patient-Centered Outcomes Measures Awareness and Use in Clinical Research in Rare Diseases","id":"ed497af8-7951-4af9-bee0-7a26eb447bc6","sessionCode":"PCR28","topDisplay":"Desvignes-Gleizes C1, Mc Donough G2, Serriere-Lanneau V2, Sherafat-Kazemzadeh R3, Rath A2, Bothorel S3 1Mapi Research Trust, Lyon, 69, France, 2Inserm | US14 - Orphanet, Paris, France, 3Mapi Research Trust, Lyon, France","locationCode":"6028","description":"\r\n\t<div>OBJECTIVES: Since 2017, Mapi Research Trust and Orphanet have joined forces and aligned their missions to support a common goal, the dissemination of knowledge and use of Patient-Centered Outcomes Measures (PCOMs) in rare disease (RD) research and healthcare management. The lack of awareness of PCOMs in RD is a strong limitation in patient centric clinical research. However, the development of disease-specific PCOMs for more than 7,000 RD is not a realistic objective. For this reason, we worked at increasing the visibility of existing PCOMs and optimizing their use in RD clinical research and care. METHODS: On one hand, Mapi Research Trust aligned its nomenclature (MeSH classification) used in its Patient-Centered Outcomes Measures database PROQOLID™ with Orphanet alignments of RD nomenclature (ORPHA Code). This alignment allows the identification in PROQOLID™ of PCOMs developed in RDs. In each RD-PCOM page a link to the related Orphanet disease page was added. On the other hand, Orphanet identified RDs for which a PCOM was available in PROQOLID™ and applicable at group of disorders, disorders and subtypes level of Orphanet classification. On each page of identified RDs, a link to existing RD-PCOMs in PROQOLID™ was added. RESULTS: In PROQOLID™, 537 PCOMs, including 380 Patient-Reported Outcome Measures have been identified and an Orphanet disease page link has been added. On the other hand, in Orphanet database, 685 disease pages were enriched with a link to existing RD-PCOMs in PROQOLID™. CONCLUSIONS: Mapi Research Trust and Orphanet joint their effort in connecting their Rare Disease and PCOMs databases allowing more visibility of existing RD-PCOMs and improving their use in RD clinical care and research.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023orphanet-mrt-db-connectionvf131044-pdf.pdf?sfvrsn=2fa569ea_0","title":"ISPOR EU 2023_Orphanet-MRT DB connection_VF131044.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131044","diseases":[{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Tofacitinib for the Treatment of Active Ankylosing Spondylitis in Greece","id":"1529e80e-b6f3-4e11-87d4-7ab0ebab9608","sessionCode":"EE40","topDisplay":"Gourzoulidis G1, Solakidi A2, Psarra M3, Nikitopoulou E2, Tzanetakos C3 1Health Through Evidence, Athens, A1, Greece, 2Pfizer Hellas, Athens, Greece, 3Health Through Evidence, Athens, Greece","locationCode":"1042","description":"\r\n\t<div>OBJECTIVES: The aim of the present study was to evaluate the cost-effectiveness of tofacitinib compared to currently marketed biologic treatment in patients with active ankylosing spondylitis (AS) who have responded inadequately to conventional therapy (biologic- naïve population) or previous biologic therapy (biologic-experienced population) in Greece. METHODS: A published model comprising a decision tree and a 3-state Markov model was adapted from a public payer perspective over a lifetime horizon. Adalimumab and secukinumab, having the highest market shares among biologics for the treatment of AS in Greece (standard practice) were selected as comparators in the analysis. Clinical parameters captured treatment response defined per ASAS20, short-term and long-term changes in Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) and Bath Ankylosing Spondylitis Functional Index (BASFI) scores long-term biologic treatment discontinuation, and adverse events. Efficacy, safety data and utility values were elicited from published literature. Direct costs pertaining to drug acquisition, monitoring, adverse events and disease management costs were considered in the analysis (€,2022). Model outcomes were patients’ quality-adjusted life years (QALY), total costs and incremental cost-effectiveness ratio (ICER).All future outcomes were discounted at 3.5% per annum.A probabilistic sensitivity analysis (PSA) was conducted to account for model uncertainty. RESULTS: In biologic-naïve population, compared to adalimumab, tofacitinib produced an estimated 0.06 additional QALYs (10.67 vs 10.73), at additional costs of €2,403 (€147,096 vs €149,500) resulting in an ICER of €41,378 per QALY gained. In biologic-experienced population, the total cost per patient for tofacitinib and secukinumab was estimated to be €151,371 and €145,757 respectively. In terms of health outcomes, tofacitinib was associated with 0.13, increment in QALYs compared with secukinumab resulting in an ICER of €42,784 per QALY gained. PSA confirmed the deterministic results for both populations. CONCLUSIONS: Tofacitinib was estimated to be a cost-effective option for the treatment of active AS in Greece for both biologic-naive and biologic-experienced patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/tofacitinibasispor23final-130491-pdf.pdf?sfvrsn=1b0683b5_0","title":"Tofacitinib_AS_ISPOR23_Final 130491.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130491","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Existing Practices for the Identification, Selection, or Assessment of Registries and Real-Word Data for Regulatory/HTA Purposes: A More-EUROPA Project Systematic Review","id":"45a3e8c0-6138-4e3c-b156-7ac63c83ad46","sessionCode":"MSR30","topDisplay":"Zebachi S1, Moreira J1, Tanniou J1, Godbillot P1, Tang P1, Amzal B2 1Quinten Health, Paris, France, 2Quinten Health, Paris 05 Panthéon, France","locationCode":"5064","description":"\r\n\t<div>OBJECTIVES: Real-world data (RWD), particularly patient registries, represent a potential useful source of information and may be required at various stages of the drug lifecycle. Different questions may be asked throughout the regulatory and/or HTA decision-making process, including concerns related drug safety, reimbursement, cost-effectiveness... The selection of an appropriate registry plays a critical role in RWD-based research, as registry features directly impact the quality of statistical analyses and the resulting evidence. In light of this, the More-EUROPA project, a five-year public-private program involving 7 EU countries, aims to identify and propose practices for a more efficient use of RWD in the development, registration, and assessment of medicinal products in Europe. Specifically, one main objective is to develop a screening tool to timely identify suitable registries on a ‘fit-for-purpose’ approach. METHODS: A literature review was conducted to investigate the current practices regarding the identification, selection, and assessment of registries and RWD sources on a fit-for-purpose setting, mainly from a regulatory/HTA decision-making perspective. Articles published between 2013 and 2023 were identified through MEDLINE, Web of Science and Scopus databases, employing free-text search terms related to registries and RWD in the context of fit-for-use approaches. RESULTS: A total of 819 articles were initially identified, out of which 765 were excluded during primary and secondary screening, resulting in a final set of 54 articles for analysis. Findings indicate a growing presence of guidance provided by regulatory agencies and HTA bodies. However, apart from a few specific repositories allowing the identification (e.g., ENCePP) or framework tools for assessment of registries quality (e.g., REQueST), no comprehensive tool integrating the dimensions of identification, selection, and assessment has been identified in the literature. CONCLUSIONS: These results underline the need to create an integrative tool allowing to standardize the identification and quality assessment of registries.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/postemsr30133370-pdf.pdf?sfvrsn=b2effa51_0","title":"Poste_MSR30133370.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133370","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Knowledge, Attitudes and Practices of Patients Towards Generics in Greece","id":"17347c46-a8c1-437d-9434-7b169846f04b","sessionCode":"PCR54","topDisplay":"Souliotis K1, Ollandezos M2, Golna C3, Maragou F4, Golnas P3, Dedes N4 1University of Peloponnese, Corinth, Peloponnese, Greece, 2PANHELLENIC ASSOCIATION OF PHARMACEUTICAL INDUSTRY, ATHENS, Greece, 3Health Policy Institute, Maroussi, Attika, Greece, 4Greek Patients' Association, Athens, Attika, Greece","locationCode":"6051","description":"\r\n\t<div>OBJECTIVES: To assess knowledge, attitudes, and practices towards generic pharmaceuticals (generics) amongst patients, members of Patient Associations (PAs) in Greece. METHODS: An online survey elicited knowledge, attitudes, and practices regarding generics in Greece. Participants were registered members of PAs in Greece and received an email invitation containing a link to the survey by the Greek Patients’ Association. All responses were anonymous. RESULTS: A total of 100 patients participated in the survey. Of them, 63% were women and 76% were 35 - 64 years old. 84% held a bachelor’s degree or higher. 67% were currently employed. 96% knew of generics and 91% knew they contain the same active substance as originators. 82% knew that generics have the same effectiveness as originators, and 45% that generics are as safe as their originator. 62% perceived generics to be manufactured in high-quality facilities. 90% considered generics important, irrespective of personal or family income, and 91% suggested patients should be free to select between generics and originators. In line with this, 75% perceived that social insurance should not mandate use of generics. Almost half (49%) of respondents perceived a switch from originator to generic to have no impact on clinical outcomes. 72% had ever been prescribed a generic, 58% had switched treatment from originator to generic, mostly on doctor’s recommendation, and 63% would be willing to switch to a generic in the future. CONCLUSIONS: Members of PAs in Greece exhibit high levels of knowledge, acceptance, and use of generics. Further strengthening public awareness of safety and quality assurance processes for the manufacturing of generics together with additional financial incentives may contribute to increasing their voluntary uptake.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23souliotispcr54poster130241-pdf.pdf?sfvrsn=a37fbb37_0","title":"ISPOREurope23_Souliotis_PCR54_POSTER130241.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130241","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Added Benefit and Revenues of Oncology Drugs Approved By the European Medicines Agency between 1995 and 2020","id":"86ebd9b5-44d0-482c-8cde-7bb52074c1e2","sessionCode":"HPR10","topDisplay":"Brinkhuis F1, Goettsch W2, Mantel-Teeuwisse AK3, Bloem LT3 1Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, UT, Netherlands, 2National Health Care Institute (ZIN), Diemen, Netherlands, 3Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, Netherlands","locationCode":"3064","description":"\r\n\t<div>OBJECTIVES: The rising costs, expedited approvals, and negative added benefit ratings of oncology drugs have raised concerns about the alignment of regulatory and reimbursement incentives. This study aimed to evaluate the association between added benefit and revenues of oncology drugs, and to examine potential disparities in added benefit and revenues among different approval pathways of the European Medicines Agency (EMA) for these drugs. METHODS: We identified oncology drugs and their indications approved by the EMA (1995-2020) and retrieved corresponding added benefit ratings published by seven organizations, including health technology assessment agencies from the United States, France, Germany, and Italy, along with two medical oncology societies and a medical journal. Revenue data were extracted from publicly available financial reports. We standardized the obtained added benefit ratings, compared cumulative revenues to estimated R&D costs, and explored the association between added benefit and revenues. In subgroup analyses, we distinguished between standard marketing authorization (SMA), conditional marketing authorization (CMA), and authorization under exceptional circumstances (AEC). RESULTS: For 166 indications, 458 added benefit ratings were collected; 189 (41%) were negative/non-quantifiable. The median time to offset median R&D costs was three years; 90% of drugs recovered these costs within eight years. Drugs with higher added benefit ratings generally had greater revenues. Negative/non-quantifiable added benefit ratings were more frequent for CMAs and AECs than for SMAs (RR 1.53, 95%-CI 1.23-1.89). CMAs generated lower revenues and took longer to offset R&D costs than SMAs (four versus three years). CONCLUSIONS: While revenues seemingly align with added benefit, most oncology drugs recover R&D costs within a few years despite providing little added benefit. This is particularly true for drugs approved through CMA, which inherently lack comprehensive evidence. Policymakers should evaluate whether current regulatory and reimbursement incentives effectively promote the best drugs for high-need patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/finalposterispor2nov132543-pdf.pdf?sfvrsn=215353af_0","title":"FINAL_Poster_ISPOR_2nov132543.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132543","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Economic Evaluation of the Personalized Peer Support Program (P-SUP) for Patients with Type 2 Diabetes and/or Coronary Artery Disease","id":"bc0fa179-599f-4e69-9327-7bde0560c891","sessionCode":"EE20","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Disease Management Programs (DMPs) provide standardized, evidence-based patient care. In Germany, DMPs have been shown to improve quality and patient-centeredness of care. Regarding self-management support there is still room for improvement in comparison with international standards. The P-SUP program supports patients with type 2 diabetes (T2DM) and/or coronary artery disease (CAD) in conducting successful self-management through a multimodal program: (1) physical activity meetings in peer support groups, paired with educational lectures on physical exercise, nutrition or disease management; (2) a specially designed online platform; (3) personalized feedback for patients on medical outcomes from their general physicians; and (4) regular telephone coaching for vulnerable patients to increase intrinsic motivation and activation. METHODS: The aim of the health economic evaluation of the program is to analyze its cost-effectiveness from the perspective of statutory health insurance. Measured outcomes include health care services utilization and its cost, quality of life, and demographical characteristics. Secondary analyses will focus on the potential heterogeneity of effects based on subgroup analysis. RESULTS: Intervention costs are collected from project accounting data. Routine data from statutory health insurances and project data are imported and merged for the purpose of analysis. Deterministic and probabilistic sensitivity analyses will be conducted to account for uncertainty. Preliminary results will be available at the time of the presentation. CONCLUSIONS: P-SUP goes beyond existing designs by representing a complex intervention that integrates several components into one program, targeting both peer support effects and the individual needs of the patients. The project is funded by the Innovations Fund of the German General Joint Committee. Funding number: 01NVF18033</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130533","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Role of HTA Agencies in Relation to Medical Technologies: A Discourse Analysis","id":"80c6ef30-6765-4521-9a64-7c0d115a2d8b","sessionCode":"MT9","topDisplay":"Michels R1, de Graaff B2, Abrishami P2, Delnoij D3 1Erasmus School of Health Policy and Management, The Hague, ZH, Netherlands, 2Erasmus School of Health Policy and Management, Rotterdam, ZH, Netherlands, 3National Health Care Institute, Diemen, Netherlands","locationCode":"5037","description":"\r\n\t<div>OBJECTIVES: Conventional means of governing by HTA agencies appear to not always be fully fit or sufficient to assess innovative technologies. HTA agencies need to adapt to innovative medical technologies, while also relating to broader societal developments, such as a growing stress on the sustainability of healthcare systems. Whereas some of the earliest literature on HTA describes assessments of various medical technologies, in more recent years the applicability of existing HTA methodology to medical technologies is disputed in scientific literature. In this literature review, we explore different academic discourses around the role of HTA agencies in relation to medical technologies. METHODS: We review literature on the role of HTA agencies regarding medical technologies and make use of a discourse analysis approach to explore the language in the selected literature. Discourse analysis is a useful tool for analyzing different modes of framing an issue, and for demarcating dominant ways in doing so. Through a systematic search, we identified 858 articles in academic literature. After inclusion and exclusion, we included 10% of articles. These articles were coded inductively using Atlas.Ti software. RESULTS: We describe the different academic discourses around the role of HTA agencies regarding medical technology. We emphasize how medical technologies are framed as both similar and different from pharmaceuticals, leading to different views in the literature on if, and how, HTA is useful in governing medical technologies. We also note how these discourses have evolved over time in relation to the role of HTA agencies. CONCLUSIONS: This study reports on the academic discourses on the role of HTA agencies regarding medical technologies, including key metaphors, key stakeholders, and key publications. We relate these discourses to broader sociopolitical trends over time, such as safety scandals in the medical technology market and changing regulations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132940","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Nesting a Decision Tree into a Markov Model Structure: An Alternative Way of Tracking Movement in a Diagnostic Model","id":"a7e4014c-8728-4838-85a0-7d1c93c8e0fa","sessionCode":"MSR8","topDisplay":"Afonso D, Davies H, Avey B, Mealing S York Health Economics Consortium, York, UK","locationCode":"5054","description":"\r\n\t<div>OBJECTIVES: To conceptualize a cohort-level cost-effectiveness model involving multiple screening sessions over a lifetime where, at the point of every screening session, individuals can be redistributed across health states based on their screening results. METHODS: A targeted literature review of previous screening models was conducted to assess previous approaches to modelling multiple screening sessions over a lifetime. A method was conceptualized that involved allocating people into certain health states via a decision tree and then nesting the same decision tree probabilities within Markov model cycles at certain points in the time horizon (in this case, when screening sessions are due). This method was applied to an economic model using literature-based evidence. RESULTS: In the economic model the cohort enters a decision tree and is split by disease status. The screening test and subsequent diagnostic test (in this case, taking adherence, sensitivity and specificity into account) move people into ‘undiagnosed’, ‘diagnosed’, or ‘no disease’ health states, at which point they enter the Markov model. People with undiagnosed disease can naturally progress or be diagnosed by symptom presentation. People with no disease have a probability of getting the disease. At the point of screening in the Markov model (the frequency of which can be determined by the user), people transition through the decision tree probabilities and are redistributed across the undiagnosed and diagnosed health states, before transitioning to the next cycle in the Markov model. CONCLUSIONS: This flexible method can be used when building health economic models to sufficiently track the diagnosis of a naturally progressing disease at the point of screening as well as symptom presentation. This could also be applied to other types of models where part-way through the time horizon several pathway options become available during one model cycle.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23afonsomrs8poster129586-pdf.pdf?sfvrsn=3e7a3e0d_0","title":"ISPOREurope23_Afonso_MRS8_POSTER129586.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129586","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical Effectiveness of Cochlear Implants in Children: A Systematic Review and Metanalysis","id":"671965ba-cf48-46c5-bc8b-7d423f770c24","sessionCode":"CO33","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1032","description":"\r\n\t<div>OBJECTIVES: A surgically implanted medical device that by passes the damaged part of the inner ear to electrically stimulate the remaining neural fibers of the auditory nerve. Electrical current stimulates the remaining auditory nerve fibers in the damaged inner ear to generate sensations of hearing. The objectives of this study were to analyze cochlear implantation evidence to conduct a systematic review to determine the clinical efficacy of Cochlear implantation in patients requiring implantation with outcomes of improved hearing and quality of life. METHODS: A comprehensive search was performed at various electronic databases using PubMed, Web of Science, Google Scholar, and Cochrane. A PRISMA method is used to curate and collocate the studies. Quantitative data were pooled into statistical meta-analyses using the Cochrane Review Manager (RevMan) to compare clinical efficacy with and without Cochlear Implants. RESULTS: A total of 3234 articles were obtained and after evaluation, 14 articles were accepted that answered the research questions and met the criteria for systematic review and meta-analysis. Therefore, the outcomes of metanalysis are improved hearing with subgroups speech hearing, spatial hearing and hearing quality at 95% CI with the overall treatment effect (mean difference= -2.65) and other outcome quality of life with subgroups physical wellbeing, emotional wellbeing, self-esteem, family, friends and school at 95% CI with overall treatment effect (mean difference = -5.04) and thus showing a statistically significant effect favoring with cochlear implants. CONCLUSIONS: In this review we found that cochlear implants played an important role in patients’ improved hearing and quality of life. Therefore, this review hypothesizes that children with hearing loss or deafness patients receives treatment with or without cochlear implants may improve hearing and quality of life. This adds to the confidence that cochlear implants will become more prevalent in the next few years.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129204","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Matching-Adjusted Indirect Comparison of the Efficacy of Bimekizumab and Secukinumab at 52 Weeks for the Treatment of Psoriatic Arthritis","id":"78f10c44-4355-41ab-8da1-7d83005c79fc","sessionCode":"CO10","topDisplay":"Mease PJ1, Warren RB2, Nash P3, Grouin JM4, Willems D5, Taieb V6, Eells J7, McInnes I8 1Swedish Medical Center and Providence St. Joseph Health, University of Washington, Seattle, WA, USA, 2Dermatology Centre, Northern Care Alliance NHS Foundation Trust, Manchester, UK; NIHR Manchester Biomedical Research Centre, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK, 3University of Queensland, Brisbane, Australia, 4University of Rouin, Rouen, Normandy, France, 5UCB Pharma, Brussels, Belgium, 6UCB Pharma, Colombes, France, 7UCB Pharma, Slough, UK, 8Glasgow Biomedical Research Centre, Glasgow, UK","locationCode":"1011","description":"\r\n\t<div>OBJECTIVES: To compare the efficacy of bimekizumab 160mg and secukinumab 150-mg (SEC150) and 300-mg (SEC300) at 52 weeks (Wk52) for the treatment of psoriatic arthritis (PsA) using matching-adjusted indirect comparisons (MAICs). METHODS: Relevant trials were systematically identified. Individual patient data from BE OPTIMAL (N=431) and BE COMPLETE (N=260) were matched to aggregated data from biologic disease-modifying anti-rheumatic drug-naïve (bDMARD-n) and tumor necrosis factor inhibitor-experienced (TNFi-exp) subgroups from FUTURE 2 for SEC150 and SEC300 (bDMARD-n: N=63/37; TNFi-exp: N=67/33). To account for cross-trial differences in baseline characteristics, logistic regression for age, sex, Health Assessment Questionnaire-Disability Index, percentage with psoriasis affecting ≥3% body surface area, swollen and tender joint counts, and methotrexate use was used; adjustment variables were selected based on expert consensus (n=5). Unanchored comparisons of recalculated bimekizumab and SEC Wk52 outcomes for American College of Rheumatology (ACR) 20/50/70 and minimal disease activity (MDA) index non-responder imputation outcomes were reported as odds ratios (ORs) with 95% confidence intervals (CIs). RESULTS: In bDMARD-n patients, bimekizumab (effective sample size [ESS]=236) had a significantly greater likelihood of ACR70 response at Wk52 than SEC150 (OR [95% CI]: 2.39 [1.26, 4.53; p=0.008] and SEC300 (2.03 [1.11, 3.72; p=0.021]). In TNFi-exp patients at Wk52, bimekizumab (ESS=146) had a significantly greater likelihood of response than SEC150 for ACR20 (3.50 [1.64–7.49]), ACR50 (3.32 [1.41, 7.80; p=0.001]), ACR70 (2.95 [1.08, 8.07; p=0.035]) and MDA (3.52 [1.38, 8.99; p=0.009]), and greater than SEC300 for ACR50 (2.44 [1.06, 5.65; p=0.037]) and MDA (2.92 [1.20, 7.09; p=0.018]). All other analyses were non-significant. CONCLUSIONS: The MAICs suggested bimekizumab to have greater likelihood of long-term response than SEC150 and SEC300 on most outcomes in patients with PsA, regardless of their prior bDMARD exposure. These results are consistent with a recent NMA where bimekizumab ranked higher than SEC for ACR and MDA outcomes at Wk16 to 24. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23willemsco10poster129984-pdf.pdf?sfvrsn=e77c902a_0","title":"ISPOREurope23_Willems_CO10_POSTER129984.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129984","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Review of Migraine-Related Pros in FDA Labelling Claims (2018-2023)","id":"2060f0e5-8e59-4d0c-b16f-7e274e4512a1","sessionCode":"PCR43","topDisplay":"Zupan Z1, Doma R2, Heinrich M3, Rudell K4 1University of Belgrade, Belgrade, Serbia, 2Parexel International, Durham, NC, USA, 3Parexel International, London, London, UK, 4Parexel International, LONDON, LON, UK","locationCode":"6042","description":"\r\n\t<div>OBJECTIVES: The objective of this review was to characterize the inclusion of migraine patient-reported outcomes (PROs) in FDA approvals subsequent the FDA’s release in 2018 of the Patient Focused Drug Development (PFDD) guidance. METHODS: The PROLABELS database was searched over a 5-year period between June 2018 and June 2023 for migraine disorders with label claims referencing migraine-specific PROs. The PROLABELS database is a commercially available database for Clinical Outcome Assessment (COA) endpoint strategies that are used to demonstrate the efficacy and safety of drugs or devices in the market approval process. RESULTS: Eleven drugs with migraine PROs in the label were identified. The primary and secondary endpoints were most often assessed using daily diaries measuring the number of monthly migraine days (n=5, 45%) or headache pain occurring two hours post dosing (n=5, 45%), use of rescue medication (n=3, 27%), most bothersome symptom freedom at two hours post-dosage (n=5, 45%), pain relief 2hrs post dose (n=5, 45%), migraine associated symptoms (n=4, 36%), headache severity diary (n=1, 1%), and activities of daily living and motor function due to migraine (n=1, 1%). Other validated PRO questionnaires included the labels assessed the impact of migraine (n=2, 18%), and migraine quality of life (n =1, 1%). CONCLUSIONS: Only 11 migraine-related therapies were approved by FDA in the 5-year period since the FDA released its PFDD guidance that incorporated migraine-specific PROs in the product label. Data used to assess endpoints were most often gathered using daily dairies. However, only two migraine-related diaries included in label claims have been published to date. Future work should focus on further development of diaries for migraine symptoms meeting the labelling claims criteria. The availability of such diaries to the scientific community could facilitate timely access to effective migraine treatments.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131856","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Indirect Cost Burden for Individuals Living with Transfusion-Dependent Beta-Thalassemia in Europe","id":"a4c8614e-f2d7-45f8-96e4-7e50894fe5b1","sessionCode":"EE111","topDisplay":"Lilly L1, Drahos J1, Boateng-Kuffour A1, Calvert M2, Levine L3, Dongha N4, Li N1, Pakbaz Z5, Shah F6, Ainsworth N7, Udeze C1, Martin A7 1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2University of Birmingham, Birmingham, UK, 3Independent Consultant, Petaluma, CA, USA, 4Independent Consultant, London, UK, 5University of California Irvine School of Medicine, Orange, CA, USA, 6NHS Blood and Transplant, London, UK, 7QC Medica, Liverpool, UK","locationCode":"2044","description":"\r\n\t<div>OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is associated with substantial direct cost burden; however, the indirect cost burden for individuals living with TDT has not been well characterized, particularly in Europe. This study describes indirect costs incurred among individuals living with TDT in Europe. METHODS: A quantitative survey was conducted with individuals with TDT in Europe (France, Germany, Italy, and United Kingdom) and the United States who self-reported ≥8 red blood cell transfusions/year in each of the 2 years before enrollment. This analysis focused on data from the included European countries. Health-related activities and self-reported out-of-pocket costs were assessed using bespoke survey items. Health-related activities were costed using the proxy good method. Monthly out-of-pocket costs were summed and annualized. The Work Productivity and Activity Impairment (WPAI) questionnaire was used to assess workplace absenteeism and presenteeism. Workplace absenteeism and presenteeism costs were determined based on the average annual salaries in each country per Organization for Economic Co-operation and Development (OECD) average wages indicator. RESULTS: A total of 61 individuals in Europe completed the survey. Individuals reported conducting health-related activities a mean of 22 hours/month, resulting in an annual cost estimate of $6,345. Mean out-of-pocket costs were $222/month, resulting in annual costs of $2,670. Based on the WPAI, approximately 50% of European individuals were employed (n = 31/61). Among them, mean absenteeism and presenteeism rates were 28.5% and 43.5%, respectively, resulting in an annual loss of $13,972 due to absenteeism and $21,326 due to presenteeism for each employed individual with TDT. CONCLUSIONS: Individuals with TDT living in Europe had substantial indirect cost burden due to health-related activities, out-of-pocket costs, and workplace absenteeism and presenteeism.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23liee111posterv2129842-pdf.pdf?sfvrsn=31c087c1_0","title":"ISPOREurope23_Li_EE111_POSTER_V2129842.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129842","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"What Is a Highly Specialised Technology (HST)? The Revised NICE HST Criteria in Practice","id":"69c9d56b-853c-4a46-a54f-7e9ec5a9f088","sessionCode":"HTA25","topDisplay":"Haria K1, Choy JY2, Kaut J3, Griffiths A4, Newell I3, Whalen JD5, Tutein Nolthenius J5 1Costello Medical, London, LON, UK, 2Costello Medical, Singapore, Singapore, 3Costello Medical, London, UK, 4Costello Medical, Cambridge, UK, 5Pharming Group N.V., Leiden, Netherlands","locationCode":"4066","description":"\r\n\t<div>OBJECTIVES: The National Institute for Health and Care Excellence (NICE) updated their criteria for the HST Program in February 2022: (1) disease prevalence <1:50,000; (2) <300 treatment-eligible patients in England; (3) disease significantly impacts length/quality of life; and (4) no satisfactory treatment options exist or technology offers significant benefit over existing treatment. Judgements on the eligibility of technologies for the HST Program are naturally subjective, and NICE states that these criteria were designed to facilitate consistent and predictable decision-making. This research aims to explore NICE’s application of the revised HST criteria. METHODS: The NICE website was searched for ongoing and published technology appraisals (TAs) and HSTs with invitations to participate post-February 2022. Published HST checklists were identified and reviewed to determine how NICE evaluated candidate technologies against the HST criteria. RESULTS: Eleven published HST checklists were identified; of these, only one appraisal was subsequently routed to HST having met all four criteria. Criterion 1 was satisfied by 6/11 appraisals and criterion 2 by a different set of 6/11 appraisals. In most appraisals (10/11), NICE based their decisions for criterion 1 on the prevalence of the full population with the broad disease, instead of the licensed indication. 5/11 appraisals did not meet criterion 3; of these, impacts on length/quality of life were considered significant for only a proportion of the population (2/5 appraisals) or were deemed unclear/not significant (3/5 appraisals). 3/11 appraisals met criterion 4; there were considered to be no satisfactory treatments available for all three conditions. 0/11 interventions were considered to offer significant additional benefit over existing treatments. CONCLUSIONS: Clarifying the HST criteria wording and providing greater detail on what may be considered ‘sufficient’ evidence may improve predictability in decision-making, allowing manufacturers to better assess the suitability of their treatments for HST, thereby offering efficiencies to all stakeholders.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23tuteinnoltheniushta25poster130972-pdf.pdf?sfvrsn=c7e1b9e_0","title":"ISPOREurope23_TuteinNolthenius_HTA25_POSTER130972.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130972","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of Moderate and Severe Hemophilia A and B in Spain, Real-World Evidence Insights from the Cost of Hemophilia in Europe: A Socio-Economic Survey II (CHESS II) Study","id":"c4c16d72-8266-4290-ba25-7eb2eca7c701","sessionCode":"RWD22","topDisplay":"Peral C1, De Lossada Juste A1, Lwoff N2, Espinoza Cámac N3, Martinez N4, Casado MÁ5, Burke T6, Kane A7, Alvir J8, Thakkar S9, Ferri Grazzi E10 1Pfizer S.L.U., MADRID, M, Spain, 2Pfizer, S.L.U, Alcobendas, Madrid, Spain, 3Pharmacoeconomics & Outcomes Research Iberia (PORIB), Madrid, Madrid, Spain, 4A former PORIB employee, Madrid, Spain, 5Pharmacoeconomics & Outcomes Research Iberia (PORIB), Madrid, Spain, 6HCD Economics, Daresbury, Warrington, WRT, UK, 7Pfizer, Inc., New York, NY, USA, 8Pfizer Inc, New York, NY, USA, 9Pfizer Inc., New York, NY, USA, 10HCD Economics, Daresbury, , CHW, UK","locationCode":"6076","description":"\r\n\t<div>OBJECTIVES: Hemophilia is a congenital bleeding disorder characterized by a deficiency or absence of factor VIII in hemophilia A (HA), or IX in hemophilia B (HB), which leads to prolonged spontaneous or traumatic bleeding events and can be classified as mild (>5-40% of normal), moderate (1-5%), or severe (<1%) depending on endogenous FVIII/IX levels. In Spain, little data exists on the societal economic burden of hemophilia. The aim of this analysis is to assess direct and indirect cost associated with moderate and severe HA and HB in Spain. METHODS: CHESS II, a 12-month retrospective, cross-sectional, burden-of-illness study was carried out during 2018-2020, in adult HA/HB patients of any severity in eight European countries. Data on Spanish non-inhibitor HA/HB moderate/severe patients were analyzed descriptively. Aggregate annual costs were calculated using publicly available information from Spanish official sources (€,2022). RESULTS: Of the 341 patients in the Spanish CHESS II cohort, 228 met the inclusion criteria: 181 had HA (37%[n=66] moderate; 63%[n=115] severe), and 107 HB (26%[n=28] moderate; 74%[n=79] severe). Mean (SD) age and weight ranged 40.4-40.8 (14.3;14.3) and 74.8-77.6 (9.7;11.8), respectively. Direct cost was higher in HB and in severe patients in both cohorts, with €17,251 (moderate HA), €17,796 (moderate HB), €116,767 (severe HA) and €206,996 (severe HB). Overall, FVIII/FIX costs represented 87% of direct cost and were the main cost driver, particularly in the severe cohorts. Mean indirect cost per patient was €4,089 (moderate HA), €797 (moderate HB), €8,633 (severe HA) and €8,049 (severe HB). Finally, the average total cost (direct and indirect) for moderate and severe patients was €91,017 (HA) and €163,924 (HB). CONCLUSIONS: Hemophilia in Spain is associated with a substantial cost burden both from the healthcare system and societal perspective, predominantly in severe patients. Additional research is warranted to further explore the economic burden associated with elevated treatment- and indirect- cost.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23peralrwd22poster129354-pdf.pdf?sfvrsn=ba6cab6b_0","title":"ISPOREurope23_Peral_RWD22_POSTER129354.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129354","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"X-Linked Retinitis Pigmentosa Impacts Patients’ Independence, Work Status, and Quality of Life: Insights from the Cross-Sectional Explore Xlrp-1.2 Physician Survey","id":"8bb80eb5-c42e-4387-9e43-7eb9ecbd2a67","sessionCode":"HSD14","topDisplay":"Denee T1, Lee J2, Fartaes A3, Ampeh K4, Pungor K5 1Janssen-Cilag, Breda, Netherlands, 2Janssen-Cilag A/S, Birkerod, Denmark, 3IQVIA, Milan, Italy, 4IQVIA, London, UK, 5Janssen-Cilag GmbH, Neuss, Germany","locationCode":"4024","description":"\r\n\t<div>OBJECTIVES: To understand unmet needs in managing X-linked retinitis pigmentosa (XLRP) and evaluate its impact on patients’ independence/autonomy, work status, and quality of life (QoL). METHODS: In the EXPLORE XLRP-1.2 survey, retina specialists/ophthalmologists with experience managing XLRP (n=15) and geneticists (n=3) in Austria, Belgium, Denmark, Finland, the Netherlands, Norway, Sweden, and Switzerland were interviewed to elicit insights on disease manifestations and impacts in their patients with XLRP (n=47). RESULTS: Most patients with XLRP were male (70%) and aged 18–49 years (75%); 17% lived alone (53% lived with family) and 19% were employed (32% were students; 11% were retired/on disability). Furthermore, 34% of patients were ‘completely independent’, while 40% were ‘somewhat or completely dependent’ on family/friends. Independence decreased with XLRP progression: 79% of early-stage patients (defined by nyctalopia/night blindness) were ‘completely autonomous’, and 91% of late-stage patients (with central visual impairment/blindness) were ‘completely or somewhat dependent’. Specialist visits involved travel to a different city for 49% of patients, usually requiring support from family members. With declining independence, patients were less active in the workplace, especially those ‘completely dependent’ on friends/family. However, healthcare providers were unaware of independence and employment status for 36% of patients. Social/emotional/financial support was offered to 47% of patients, varying by country. Despite the impact of XLRP on patients’ lives, only 28% of specialists reported monitoring QoL; most only monitor XLRP progression, manage symptoms, and provide visual aids. Clinical experts agreed that lack of treatment options and a standard XLRP management plan are key unmet needs; better access to resources/information, emotional support, and genetic counselling are also needed. CONCLUSIONS: Patients with XLRP require considerable assistance; as XLRP progresses, patients’ independence declines and they are less active in the workplace. Unmet needs in XLRP management include new treatment options, improved access to resources/information, and routine monitoring of QoL.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/hab80336-janssen-ispor-eu-xlrp-1-2-qol-poster-841x1189mmfinal130701-pdf.pdf?sfvrsn=4bbe979c_0","title":"HAB80336 Janssen ISPOR EU XLRP 1.2 QoL Poster 841x1189mm_FINAL130701.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130701","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization (HCRU), Effectiveness and Safety of Trifluridine/Tipiracil in Treatment of Refractory Metastatic Colorectal Cancer in a Portuguese Comprehensive Cancer Center (PCCC)","id":"c77f7d9b-2ebf-47e9-83d6-7f09434ffb66","sessionCode":"CO22","topDisplay":"Redondo P1, Brito da Silva J2, de Paiva Agostinho C2, Banha R2, Silva AS3, Faustino C2, Marques D2 1IPO Porto, Porto, Portugal, 2Instituto Português de Oncologia do Porto, FG, E.P.E., Porto, 13, Portugal, 3IPO Porto, Porto, 13, Portugal","locationCode":"1019","description":"\r\n\t<div>OBJECTIVES: Trifluridine/Tipiracil(FDT-TPI) is a drug approved for refractory metastatic colorectal cancer(mCRC) treatment. This study aim to assessed HCRU and FDT-TPI clinical outcomes in a real-world(RW) setting. METHODS: Retrospective cohort study with all mCRC patients(pts) who started FDT-TPI as palliative treatment before 01/11/2022, with follow-up until 30/04/2023. Data was collected from medical/administrative records. Clinical characteristics and HCRU were evaluated using descriptive statistics. Kaplan-Meier method was used for survival analysis. HCRU outcomes included: outpatient and emergency room visits; hospitalizations; complementary diagnostic and therapeutic procedures (CDTs). RESULTS: The cohort included 196 pts, 65%male, with a median age of 63/yo(23-85). Majority had an ECOG1(62%) and 47% had ≥3 metastatic sites at treatment initiation. Treatment median cycles was 3. Median PFS was 3.0 months and OS was 6.4 months. In a subgroup analysis stratified by number of metastatic sites (1vs2vs ≥3), median OS was superior in pts with fewer sites involved (9.6 vs 6.8 vs 5.2 months). Disease control rate was 10%. Median time to worsening of ECOG ≥2 was 5.3 months. Grade>=3 adverse events(AE) occurred in 43% of the pts, the most common were neutropenia(31%) and anemia(8%). No toxic deaths were documented. AE led to dose reductions in 24% of the pts and treatment delays in 55%. There were a median of 7 medical appointments and 1 CTscans performed during treatment. Proportion of pts admitted in emergency was 54,4%; 29,2% were hospitalized. The mean cost per patient of the prescribed drug was 8280,06€. CONCLUSIONS: Given the high prevalence of mCRC pts, the treatment is likely to result in potential high budget impact for hospitals. Our study showed more frequent dose reduction, worse disease control rate and a safety profile with fewer grade>=3 AE reported compared to clinical trials. Survival benefits and time to worsening of performance status were comparable to the ones reported.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/pcn20posterisporeurope2023finalv2133381-pdf.pdf?sfvrsn=38eb470_0","title":"PCN20_Poster_ISPOR_Europe_2023_final_v2133381.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133381","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Applying Transportability Methods to Real-World Data: A Scoping Review","id":"2657f6eb-4a49-4c39-b11e-7f3e5991e1e6","sessionCode":"RWD30","topDisplay":"Wang H1, Tikhonovsky N2, Gupta VA3, Thompson A1, Ramagopalan S1, Duffield S4 1Lane Clark & Peacock, London, UK, 2Lane Clark & Peacock, London, LON, UK, 3National Institute for Health and Care Excellence, Manchester, UK, 4National Institute for Health and Care Excellence, Liverpool, UK","locationCode":"7003","description":"\r\n\t<div>OBJECTIVES: Randomised controlled trials (RCTs) are the cornerstone of evidence generation for decisions on whether to approve or reimburse a treatment. However, real-world data (RWD) is emerging as an additional evidence source for health technology assessment (HTA) agencies. For example, in rare diseases and indications, international RWD may be the best information available but may be geographically distinct or otherwise not representative of the target population. This can create uncertainty about validity of the results. Applying transportability methods to a suitable data source can help to overcome this uncertainty by providing an estimate of what the treatment effect would have been had the original study been conducted in the target population by adjusting for differences between the two populations. Here we conducted a scoping review to identify and describe studies applying transportability methods to RWD. METHODS: PubMed/Medline, Cochrane and Google Scholar were searched using predefined terms and reference lists scanned for articles in English applying transportability methods in real-world contexts, either from RCT to RWD or from RWD to RWD. Data were extracted on the transportability scenario considered, transportability method used and therapeutic area examined. RESULTS: A total of 13 studies were included. Most (9 out of 13) transported a treatment effect from an RCT to a target population identified in RWD. Weighting-based approaches were the dominant adjustment method employed. The included studies cut across several therapeutic areas, including HIV, breast cancer and schizophreniform disorder. CONCLUSIONS: The evidence base on real-world applications of transportability methods is limited. More examples of transporting results from RWD studies are required to support the use of these methods for decision making in HTA. In the meantime, HTA agencies require guidance to assess and manage submissions that utilise different sample and target populations to prevent unnecessary delay in decisions and, ultimately, treatment access.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23tikhonovskyrwd30poster132784-pdf.pdf?sfvrsn=5c48a6a1_0","title":"ISPOREurope23_Tikhonovsky_RWD30_POSTER132784.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132784","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Feasibility of Systematic Goal Quality Assurance During Goal Attainment Scaling in a Rare Disease","id":"8880ab02-83c6-4776-9845-7f5a6ee60142","sessionCode":"CO27","topDisplay":"Chapman CA1, George M1, Knox K1, Rockwood K2, Howlett SE2, Sevinc G1 1Ardea Outcomes, Halifax, NS, Canada, 2Dalhousie University, Halifax, NS, Canada","locationCode":"1000","description":"\r\n\t<div>OBJECTIVES: Goal Attainment Scaling (GAS) is a patient-centric outcome measure that quantifies the impact of an intervention on individualized goals. Clinicians work with patients and/or caregivers to identify goals and build goal scales unique to each patient and clinically meaningful. Here we investigated the feasibility of using a goal quality assessment inventory to ensure a standardized approach to high-quality goal development across patients with heterogeneous symptoms. METHODS: Four clinician experts were trained in setting psychometrically adequate goals with caregivers of individuals with SCN2A-associated developmental and epileptic encephalopathies. The inventory was developed to be used in real-time goal quality assurance within a fast-paced clinical trial environment and included items (n=20) such as language specificity, outcome orientation, patient-friendly language etc. Two assessors used the inventory to independently evaluate all the goal scales. Interrater reliability (IRR) was evaluated with Cohen’s kappa (95% confidence intervals, CI) and Scott's Pi. Percentage agreement between the two raters was also calculated. RESULTS: Ten participants (Mean age =8.2 years, SD=5.62, range 3.4-20.4; Nmale=8) and their caregivers took part in goal-setting interviews with four GAS raters. Across all goals (n=30), Cohen's Kappa was 0.395 [CI 0.275 0.515] and Scott’s Pi was 0.319, suggesting fair IRR. The overall percent agreement between raters was 88.8%. CONCLUSIONS: There is a critical need to standardize implementation of personalized endpoints like goal attainment scaling to ensure consistency across sites/patients. We recommend implementation of a two-step goal setting process and real-time goal quality assessment using the inventory, providing GAS interviewers with feedback tailored to each scale which can be used to finalize goals with patients before treatment administration. Although additional studies are needed, this work suggests that goal quality assessment inventories may be a helpful component in a standardized approach to goal-setting in clinical development.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sevincco27poster-133273-pdf.pdf?sfvrsn=b2d7b6b8_0","title":"ISPOREurope23_Sevinc_CO27_POSTER 133273.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133273","diseases":[{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Cost-Utility Analysis of the MyPal eHealth Application, an ePro Intervention Aiming to Foster Palliative Care of Cancer Patients: Results From a Randomized Clinical Trial in 4 European Countries","id":"9d664bd8-efed-4d2a-881d-7f9dbe36db4c","sessionCode":"EE82","topDisplay":"Naoum P1, Athanasakis K2, Radova L3, Bonotis P4, Scarfo L5, Doubek M6, Ghia P5, Kazantzaki E7, Pontikoglou C7, Pospisilova S3, Rosenquist R8, Ekström Smedby K8, Pavi E1 1University of West Attica, Athens, Attica, Greece, 2University of West Attica, Athens, Greece, 3Masaryk University, Brno, Brno, Czech Republic, 4Centre for Research and Technology Hellas, Thessaloniki, Thessaloniki, Greece, 5Università Vita Salute San Raffaele and IRCCS Ospedale San Raffaele, Milano, Milano, Italy, 6University Hospital Brno and Faculty of Medicine, Masaryk University, Brno, Brno, Czech Republic, 7General University Hospital of Heraklion, Heraklion, Heraklion, Greece, 8Karolinska University Hospital, Stockholm, Sweden","locationCode":"2012","description":"\r\n\t<div>OBJECTIVES: To assess cost-effectiveness of MyPal, an eHealth intervention that aims to foster palliative care for oncologic patients. METHODS: A randomized clinical trial (RCT) was conducted in 4 European countries (Czech Republic, Greece, Italy, Sweden). Participants consisted of adult patients with chronic lymphocytic leukaemia/small lymphocytic lymphoma (CLL/SLL) or myelodysplastic syndrome (MDS). Intervention arm patients used the MyPal intervention and wore a smart wristband, while control arm patients were offered general palliative care. Health-related quality of life was assessed with the EQ-5D-3L tool, at baseline and once every 4 weeks for total follow-up of 6 months. Costs included healthcare resource use, healthcare professional (HCP) time and the wristband. Cost data were acquired from all 4 participating countries with 2022 as the reference year. A multinational analysis of costs (expressed as PPPs) was performed, under the third-party payer perspective. The Incremental Cost-Utility Ratio (ICUR) was estimated and a non-parametric bootstrapping analysis was conducted. RESULTS: Overall, 171 patients participated in the RCT; however, only 66 - 31 in the intervention and 35 in the control arm - had complete EQ-5D and cost data and, thus, were included in the analysis. The mean 6-month QALYs were estimated at 0.403 (95%CI: 0.380, 0.425) for intervention arm patients and 0.388 (95%CI: 0.366, 0.409) for control arm patients. Total mean costs were 1,261.94 PPPs and 377.52 PPPs, respectively, for intervention and control arm patients. Intervention arm costs were significantly affected by the time for training HCPs and addressing technical issues. ICUR was 57,522.09 PPPs/QALY, while the bootstrapping analysis showed that, in 82.4% of the pairs, MyPal was both more effective and more costly than usual care. CONCLUSIONS: MyPal intervention incurred higher costs than standard palliative care; still there is potential for better cost-effectiveness if HCP training and technical solutions are standardized and managed more effectively, notwithstanding the pandemic effect.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23naoumee82poster133493-pdf.pdf?sfvrsn=a052757e_0","title":"ISPOREurope23_Naoum_EE82_POSTER133493.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133493","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring the Relationship Between Hemochromatosis and Fracture Risk: A Population-Based Matched Cohort Study Stratified on Ferritin Levels","id":"7fa13c31-1588-4870-9cb6-7fd4efe6cacb","sessionCode":"EPH38","topDisplay":"Martinez-De la Torre A1, Burkard T2, Hofbauer LC3, Steinbicker AU4, Rauner M3, Burden AM5 1ETH Zurich, Zurich, Zurich, Switzerland, 2University of Oxford, Oxford, Oxford, UK, 3Technische Universität Dresden, Dresden, Dresden, Germany, 4University Hospital Frankfurt, Goethe University, Frankfurt, Frankfurt, Germany, 5University of Toronto, Toronto, ON, Canada","locationCode":"3040","description":"\r\n\t<div>OBJECTIVES: Patients with hereditary hemochromatosis, beta-thalassemia, and sickle-cell disease have an elevated risk of fracture. However, the extent that this is due to iron overload remains unclear. We aimed to assess the risk of fractures among patients with iron overload (i.e., serum ferritin values >1000ug/L) and among those with a diagnosis of hemochromatosis, beta-thalassemia, or sickle-cell disease without iron overload, compared to matched controls. METHODS: We used primary care data from the UK from the IQVIA Medical Research Database that incorporates data from THIN, a Cegedim Database of anonymized electronic health records. Patients aged >18 years with an incident diagnosis of hemochromatosis, beta-thalassemia, or sickle-cell disease or with an incident record of elevated serum ferritin values (>1000ug/L) were identified between 2010-2022. Each case was matched with up to 10 controls. We used time-varying Cox proportional hazard models adjusted for confounders to estimate hazard ratios (HRs) and 95% confidence intervals (CIs) for the risk of major osteoporotic fracture associated stratified by incident diagnosis vs evidenced iron overload. RESULTS: We identified 6754 patients with incident diagnosis and 13,510 with iron overload, who were matched to 79,256 and 113,700 controls, respectively. Incident diagnosis patients had an average age of 63.2 and 34.3% were female, while high ferritin value patients had an average age of 47 and 48.7% were female. We observed that patients with an incident diagnosis had no elevated risk of fractures (HR 0.99, 95% CI 0.84 – 1.16), but patients with high ferritin value had a statistically significant increased risk for fractures (HR 1.91, 95% CI 1.73 – 2.10). CONCLUSIONS: In this population-based cohort study we found that patients with high ferritin value had a 91% increased risk of suffering a fracture. Our findings highlight the importance of considering ferritin levels in the assessment of fracture risk in hemochromatosis patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor2023poster129460-pdf.pdf?sfvrsn=c6dc9474_0","title":"ISPOR2023_poster129460.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129460","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Inequity in Access to Breast Reconstruction Techniques in Belgian Health System: Is it Justified?","id":"6c6ea544-e01f-46b1-a2cc-8025c0d2823f","sessionCode":"MT14","topDisplay":"Marbaix S1, Feijen M2, Peperstraete A3, Hamdi M4 1University of Mons - Umons, Mons, Hainaut, Belgium, 2Zuyderland Medisch Centrum, Maastricht, Limburg, Netherlands, 3AZ Klina, Brasschaat, Antwerpen, Belgium, 4UZ Brussel, Brussels, Brussels, Belgium","locationCode":"5041","description":"\r\n\t<div>OBJECTIVES: Breast cancer patients who have faced mastectomy can undergo autologous or alloplastic breast reconstruction. In contrast to other European countries, the Belgian healthcare system allocates distinct budget according to the chosen technique: the autologous breast reconstruction is rewarded based on the updated surgery skills and annual inflation while the budget allocated to innovative breast implants remains at the level of 10 years old implants. This situation jeopardizes access to innovation. This research aims to evaluate the cost-effectiveness analysis of a new type of implant for breast reconstruction. METHODS: A decision tree has been built to evaluate the cost-effectiveness of the innovative lightweight polyurethane coated breast implant compared to the traditional textured/smooth implant. Because of its specific coating and filling, this new implant reduces the risk of re-hospitalization due to complications commonly observed with the textured/smooth implants. The innovative borosilicate microsphere filling also reduces the weight of the implant which can be beneficial for the remaining skin and soft tissues after breast amputation, reducing pain and improving aesthetics. Successful breast reconstruction means both better physical and mental health recovery. RESULTS: From a national payer perspective, the lightweight polyurethane coated breast implant is cost-saving compared to the traditional textured/smooth implants, as it offers long term healthcare savings due to fewer re-hospitalizations. Fewer complications and improved esthetical outcomes also impact positively patients’ quality of life. CONCLUSIONS: The present cost-effectiveness analysis suggests that from a national payer perspective, allocating a higher budget to an innovative implant such as the lightweight polyurethane coated breast implant can be justified.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23marbaixmt14poster128104-pdf.pdf?sfvrsn=42b6776b_0","title":"ISPOREurope23_Marbaix_MT14_POSTER128104.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128104","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Harnessing the Potential of Natural-Language Processing and Interconnected Data Streams for Complex Diseases in the Hospital Setting: Lupus Case Study in France (LUPUS REAL)","id":"49a813cc-a04f-44a9-ba49-80e94ed443b1","sessionCode":"PT9","topDisplay":"Jouve M1, Lagonotte E2, Valette M3, Sano B4, Ricci JF5, Vuiblet V6 1Sancare, Paris, 75, France, 2URCA, Reims, France, 3Sancare, Paris, Paris, France, 4Alira Health, Paris, France, 5Alira Heatlh, Basel, BS, Switzerland, 6CHU de Reims, Reims, Grand Est, France","locationCode":"6B","description":"\r\n\t<div>OBJECTIVES: Efficiently identifying and characterizing with precision patients suffering from a heterogeneous condition with diverse manifestations is complex with traditional data sources. This study assesses the ability of an interconnected hospital-specific natural language processing (NLP)-powered solution (Realli) to identify and characterize patients with lupus in France. METHODS: Upon Ethics and Scientific Committee approval, we searched native hospital patient electronic medical records (“lupus” or “lupique” text strings) and hospital claims (ICD-10-CM L93.X or M32.X). RESULTS: Between January 2018 and March 2023, we identified 191 adult patients with lupus (mean age 48.1 years; 88% females), with 95.8%, 20.4% and 4.2% of patients who presented with lupus erythematous, nephritic, and cutaneous, respectively and not mutually exclusive (ICD codes, text strings). Biomarker values on lupus-specific or not, such as anti-DNA antibodies, anti-RNP antibodies, Smith antibodies, and C-reactive protein were retrieved in 63.9%, 4.2%, 12.6%, and 54.5% of patients, while disease activity index (i.e., SLEDAI or BILAG) was reported in 9.4% patients. Most prevalent comorbidities were cardiovascular (56.5%), metabolic (22.5%) and psychiatric (16.8%) disorders and infections (8.9%). In addition, renal impairment was identified in 29.8% (all renal ICD-10 codes) and 24.1% (ICD-10 N17.X to N19.X only or creatinine clearance levels <60 mL/min/1.73 m2). Among those latter, 52% required dialysis. Lastly, 26.7% and 15.2% of patients presented with antiphospholipid antibody syndrome or Gougerot-Sjögren syndrome, respectively. Patients received (not concomitantly) cyclophosphamide (4.7%), immunosuppressants (6.8%), glucocorticoids (47.7%), hydroxychloroquine (73.8%), belimumab (14.7%), and/or rituximab (8.4%). Over the study period, patients were hospitalized on average 7 times (median 3). CONCLUSIONS: A NLP-powered solution connected to multidata hospital sources is effective in characterizing complex pathologies, with a breadth and granularity of information not available in traditional RWE data sources. Learning from this single-center study will be deployed to multihospital systems in France to test the robustness of the findings.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23jouvept9poster133083-pdf.pdf?sfvrsn=fc5f8598_0","title":"ISPOREurope23_Jouve_PT9_POSTER133083.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133083","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Socioeconomic Burden of Obesity – Increased Rate and Duration of Sick Leave in Patients Living with Obesity: A Cross-Sectional Claims Data Analysis in Germany","id":"cc143e13-5c0f-4cda-87ec-817a352b6641","sessionCode":"RWD4","topDisplay":"Timpel P1, John N2, Kossack N2, Seitz L3, Verket M4, Müller-Wieland D4, Häckl D2 1WIG2 GmbH, Leipzig, Germany, 2WIG2 GmbH, Leipzig, Saxony, Germany, 3Novo Nordisk Pharma GmbH, Mainz, Germany, 4University Hospital Aachen, Aachen, Germany","locationCode":"6058","description":"\r\n\t<div>OBJECTIVES: Available evidence on the association of obesity and workplace productivity loss is scarce and fails to estimate the impact across individual obesity classes. Therefore, this observational study aims to explore sick leave in patients living with obesity stratified by obesity class. METHODS: This is a cross-sectional, observational study of routinely collected claims data of the statutory health insurance (SHI) system in Germany. Individuals generally eligible for sick payments between 2016 and 2021 were included. Individuals living with obesity, identified by the presence of diagnoses for obesity, were selected and stratified into four subgroups, namely BMI classes I, II, III and not specified obesity class based on the observed diagnosis for obesity (ICD-10-GM E66). Results were compared to patients without obesity. RESULTS: Of the available 3,227,677 individuals in the database (2021), 124,617 met the inclusion criteria (e.g. obesity, minimum age of 5 years and eligibility for sick leave). In 2021, higher rates of patients with sick leave (68.3% vs 51.7%) and elevated average number of days on sick leave were found for those living with obesity (40.5 days, 95% CI [40.1, 40.9]) compared to patients without obesity (18.9 days, 95% CI [18.8, 19.0]). Average number of days on sick leave was highest for obesity class III (47.9), followed by classes II (44.2), class I (42.3), and not specified obesity (33.3). Similar results were observed between 2016-2020. CONCLUSIONS: The results suggest that rate and duration of sick leave increase with obesity class. As this might have an impact on productivity losses and societal health burden, structured obesity prevention and treatment should be implemented more intensively.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23timpelrwd4poster129723-pdf.pdf?sfvrsn=1e326a6f_0","title":"ISPOREurope23_Timpel_RWD4_POSTER129723.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129723","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Oncolytic Therapies in China: Laying the Groundwork for a Successful Launch","id":"23027871-aef7-4a53-ae46-81a690f1c72c","sessionCode":"HPR11","topDisplay":"Jiang L1, Wang X2, Shi Y3, Wang S4, Yu Z4 1Clarivate, Washington, DC, USA, 2Georgetown University, Washington, DC, USA, 3Clarivate, Boston, MA, USA, 4Clarivate, Philadelphia, PA, USA","locationCode":"3067","description":"\r\n\t<div>OBJECTIVES: To determine the policy changes and market access implications and best practices of oncology products in China. The aim of this study is to determine how to best position oncology drug launches given the frequency of updates in the National Reimbursement Drug List (NRDL) to be more inclusive of innovative western oncolytic medicine that can be accessed within China. This study will also help form access strategies of oncolytic products based on examples. METHODS: Through secondary research, we will look at the new policy changes (updates directly from the Chinese Government). Through qualitative research with key stakeholders, we will interpret the evolving market landscape and understand the implications of pricing and reimbursement of oncology products in China. Lastly, we will perform quantitative research analyzing the price changes pre-and post- NRDL negotiation of oncology products. RESULTS: Our research compared key differences of the overall oncology market in China and other western markets. Additionally, the research identified key opportunities and challenges in the evolving market access landscape of oncolytic therapies in China which can be used to formulate a successful market access strategy. Lastly, the research will generate recommendations for western pharmaceutical companies on product/indication prioritization, key factors influencing access strategies via public reimbursement and private insurance reimbursement. CONCLUSIONS: The Chinese government emphasize on improving access of oncolytic therapies as demonstrated in NRDL negotiations in recent years. As a result, oncolytic therapies have relative advantages compared with other therapeutic areas in terms of demonstrating a perceived stronger value proposition by the central government. Successful planning and implementation of new oncolytic product launch strategies in China requires in-depth and agile understanding of the market to precisely position the product and seize the opportunities.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/oct252023-final-ispor-euoncolytic-therapies-in-china-laying-the-groundwork-for-a-successful-launch133545-pdf.pdf?sfvrsn=22cfd3bc_0","title":"OCT252023 FINAL_ ISPOR EU__Oncolytic Therapies in China Laying the Groundwork for a Successful Launch133545.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133545","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Review and Evaluation of Health Equity Considerations in Health Technology Assessments in Cystic Fibrosis","id":"d7d9fda0-8ef7-412d-974e-81ab0232e3ab","sessionCode":"HPR15","topDisplay":"Lee LY1, Sheth S2, Valliant S3, Khan Z1 1Center for Health Outcomes, Policy & Economics, Ernest Mario School of Pharmacy, School of Public Health, Rutgers University, Piscataway, NJ, USA, 2Center for Health Outcomes, Policy & Economics, Ernest Mario School of Pharmacy, School of Public Health, Rutgers University, Livingston, NJ, USA, 3Center for Health Outcomes, Policy & Economics, Ernest Mario School of Pharmacy, School of Public Health, Rutgers University, Perkasie, PA, USA","locationCode":"3070","description":"\r\n\t<div>OBJECTIVES: A gap exists between considerations of health equity and the implementation into health technology assessment (HTA) frameworks. This review aims to describe the extent of health equity incorporation within global HTA evaluation of cystic fibrosis medications. METHODS: National HTA bodies were identified using the International Network of Agencies for Health Technology Assessment (INAHTA) List and independent assessment bodies (European Medicine Agency and Institute for Clinical and Economic Review). Included HTA bodies had public reports in English on cystic fibrosis medications, identified using following search terms: cystic fibrosis, lumacaftor, elexacaftor, tezacaftor, and ivacaftor. Included submissions were reviewed utilizing the Framework for Equity by Benkhalti et al. and the health equity key questions within each HTA evaluations were descriptively reported. Some considerations included: stakeholders involvement, outcome measures and methodological approach. RESULTS: Of the 33 HTA bodies identified, 12 had at least one report on cystic fibrosis medication. Three HTA bodies were included in the analysis, representing United States, European Union, and Canada. Canada and US included patient engagement and advocacy considerations. There was variable to no consideration of medication access and its impact on outcomes. No health equity methodological considerations were identified. Among the identified HTA reports, there were significant gaps in the level of incorporation and implementation of health equity considerations. Limitations include the exclusion of non-English reports, significantly reducing the sample size. CONCLUSIONS: The study underscores the need to address gaps in the application and standardization of health equity principles and frameworks. This challenge is further exacerbated by variabilities amongst different countries in terms of their health equity guidance, if any exists. By shedding light on the challenges and opportunities specific to cystic fibrosis, this study provides valuable insights for enhancing the integration of health equity in HTA assessments, especially in disease areas with high unmet needs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23leehpr15poster132333-pdf.pdf?sfvrsn=6d74ebac_0","title":"ISPOREurope23_Lee_HPR15_POSTER132333.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132333","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Pharmacoeconomic Evaluation of National Immunization Program Realisation in Algeria","id":"5985b293-11a5-4707-bb99-81aedb41153a","sessionCode":"EE134","topDisplay":"Laichour A1, Kihel M1, Aissaoui A1, Olivera G2 1Sanofi, Algiers, Algeria, 2Sanofi, Lyon, 69, France","locationCode":"2070","description":"\r\n\t<div>OBJECTIVES: The objective of this study was to estimate the pharmacoeconomic impact, of the switch from the current immunization schedule with a tetravalent vaccine (DTwP-Hib) +HBV+IPV to a new schedule with an aP hexavalent (DTaP-HBV-HiB-IPV) vaccine in Algeria from Ministry of Health (MoH) and societal perspective. METHODS: A cost minimization analysis was designed with a 1-year time horizon for all vaccination eligible cohorts in Algeria. Demographic data references were taken from the national register, current immunization schedule and the new schedule from the MoH decree. The costs included were vaccines acquisition, adverse events management, vaccines administration, vaccines wastage, transporting children to vaccination centers, vaccines supply chain, loss of parents’ productivity. All costs were reported in euros and collected locally. A one-way sensitivity analysis was performed. RESULTS: The overall cost associated to the national immunization program was found to increase by +26 M€ (+58%) per year versus to the current schedule. Except for vaccine acquisition costs, which would increase, we found -19 M€ savings per year in the other costs considered as listed above. From the sensitivity analysis the cost drivers were found to be vaccine prices, the probability of use of medication for adverse events, the price of children transport. CONCLUSIONS: The implementation of an aP hexavalent vaccine in Algeria likely to be a sustainable alternative to the current immunization program, due to the savings on the management of adverse events and programmatic costs, In addition to the cost-savings, the introduction of an aP hexavalent vaccine, offering convenience and reducing the number of visits by 36%, may contribute to VCR increase and thus control of targeted diseases. Future studies may provide insights on the VCR, epidemiological and economic impact of this implementation in Algeria. KEY WORDS: Vaccine, Hexavalent vaccine, Cost minimization, Immunization schedule, VCR.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23laichouree134poster132369-pdf.pdf?sfvrsn=69e32aa5_0","title":"ISPOREurope23_Laichour_EE134_POSTER132369.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132369","diseases":[{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Graphical Representation of Real-World Data for Analysing Infection Outbreaks Within a Tertiary Hospital Setting: A Data-Driven Approach","id":"7215d5c5-55ad-4183-978d-81bcf4a2644b","sessionCode":"RWD7","topDisplay":"Gualda Gea J1, Barón-Miras LE2, Vilella A2, Torá-Rocamora I2, Torralbo B2, Fortes I2, Valls S2, Rodriguez L2, Parejo R2, Santana G2, Martinez JA3, Bertran MJ2 1Hospital Clinic de Barcelona, Barcelona, Barelona, Spain, 2Hospital Clinic de Barcelona, Barcelona, Barcelona, Spain, 3Hospital Clínic Barcelona, Barcelona, Barcelona, Spain","locationCode":"6060","description":"\r\n\t<div>OBJECTIVES: This study aimed to explore the use of semi-automated graphical representations in analysing and understanding infection outbreaks caused by multi-drug resistant microorganism within a hospital setting. METHODS: Comprehensive data on inpatient interactions and contacts were collected from hospital information systems (HIS). Code for graph displaying was developed to easily perform representations with raw HIS extractions, without needing further data depuration. Three graph types were employed: (1) a patient-patient contact network diagram to study the interaction between patients, (2) a patient-ward contact network diagram to analyse the patient interaction with different wards, and (3) a time-scale graph representing the length of stay within a ward of cases, marking their infection onset and the period of coincidence with their contacts in that ward. All graphics were performed using R language. RESULTS: The adapted graphical representations provided a comprehensive visual overview of the patient contact networks within the hospital. The case-patient contact network diagram helped identify clusters of patients, shedding light on potential routes of infection transmission. The case-ward contact network diagram facilitated understanding of the distribution of cases across different wards and connect clusters and the graphical representation of contact duration within a ward show the variations in contact time among patients. These graphical representations significantly contribute to visualizing infection outbreak dynamics and enable targeted interventions for effective outbreak control. CONCLUSIONS: The utilization of graphical representations based on real-world data proved effective in visualizing patient contact networks and analysing infection outbreaks within a hospital setting. The graphical representation provided valuable insights into de dynamics of infection transmission, empowering healthcare professionals to implement timely targeted interventions for effective outbreak control. This data-driven approach using case-patient and case-wards networks, along contact duration visualization, enhances the understanding and management of infection outbreaks caused by multi-drug resistant microorganism within hospital. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterispor133807-pdf.pdf?sfvrsn=ee289df2_0","title":"poster_ISPOR133807.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133807","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Longitudinal Analysis of Diagnostic Procedures for Sleep Disorders in the German In-Patient Sector","id":"3803f782-beba-4994-b235-81cb1eecd43a","sessionCode":"HSD7","topDisplay":"Braun M1, Stockhoff M2, Schoebel C3 1University Hospital Essen, Bonn, NW, Germany, 2Onera Health, Eindhoven, Noord-Brabant, Netherlands, 3University Hospital Essen, Essen, NW, Germany","locationCode":"4017","description":"\r\n\t<div>OBJECTIVES: The healthcare landscape in Germany is undergoing rapid changes due to multiple factors, not only but at least partially accelerated by the SARS-CoV-2 pandemic. Factors relevant to in-patient sleep medicine providers are on the one hand increased demand for sleep diagnostics and technological developments; and on the other hand political pressure towards more outpatient care; shortages of staff; and increased economic pressure for hospitals. Aim of this research is to analyze changes of in-patient sleep procedures over the past fifteen years in light of these changes. METHODS: Data on claims related to the sleep tests was obtained from the public databases at the Federal Statistics Bureau (Destatis) and the German Hospital Institute (InEK). Relevant cases were identified with OPS procedure codes, which must be used to receive reimbursement for services provided in hospitals. RESULTS: Over the period 2008-2022, inpatient sleep diagnostic procedures decreased in total by 29.8%, with Polysomnography cases declining by 42.6%, and vigilance test by 41.8%. Polygraphy utilization grew by 15.5% overall, though this number declined too after peaking in 2014. The compound annual growth rate was -2.5% for all tests; -3.9% for Polysomnography; +1.4% for Polygraphy and -3.8% for vigilance tests respectively. The largest reduction in volume occurred in the year 2020 (-27.1%; -30.7%; -21.5%; and -32.3%). During the entire period, overall procedure volume grew only in the years 2014 and 2022 (5.3% and 7.7%). CONCLUSIONS: Utilization of in-patient sleep diagnostic services declined substantially in Germany over the past fifteen years, after peaking in 2014. A decrease in procedures occurred already before the SARS-CoV-2 pandemic, but significantly accelerated during the years 2020-2021. Diagnostic tests increased moderately again in the year 2022 when hospitals largely re-started in-patient services. Reasons for the decline of in-patient sleep diagnostic services could not be established from this analysis and will be subject of further studies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128284","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Aztreonam-Avibactam (ATM-AVI) Versus Meropenem for the Treatment of Serious Infections Due to Gram-Negative Bacteria for Which There Are Limited Treatment Options (LTO) in Italy","id":"60e1851a-d7d8-4247-9d5a-82e1131f2435","sessionCode":"EE34","topDisplay":"Bao X1, Woodcock F1, Di Virgilio R2, Kantecki M3, Chow J4, Gheorghe M5 1Source Health Economics, London, LON, UK, 2Pfizer Italia, Rome, Italy, 3Pfizer, Paris, France, 4Pfizer Global, Collegeville, PA, USA, 5Pfizer SRL, Bucharest, București – Ilfov, Romania","locationCode":"1076","description":"\r\n\t<div>OBJECTIVES: Aztreonam-Avibactam (ATM-AVI) is a combination therapy including the monobactam aztreonam (ATM) and the non-β-lactam β-lactamase inhibitor avibactam (AVI). A Phase 3 randomised clinical trial (REVISIT) investigated ATM-AVI for the treatment of serious Gram-negative infections (GNI) including MBL-producing pathogens for which there are limited treatment options (LTO). This analysis evaluates the cost-effectiveness of ATM-AVI ± metronidazole versus meropenem ± colistin for the treatment of complicated intra-abdominal infections (cIAI) and hospital-acquired pneumonia/ventilator-associated pneumonia (HAP/VAP) in the Italian setting. Uncertainty in the model was assessed using probabilistic sensitivity analysis, one-way deterministic sensitivity analysis, and scenario analyses. METHODS: A cost-effectiveness analysis used a decision tree model that simulates the clinical pathway, followed by a Markov model to capture lifetime impacts on cured patients was used. The model adopted Italian National Health System perspective with a 3% discount rate. Treatment response rates were taken from REVISIT, with separate analyses for the cIAI and HAP/VAP populations. The impact of resistant pathogens from recent literature was included as an additional factor RESULTS: The key drivers of the model are the response rates from REVISIT and the impact of resistant pathogens which influence QALY gains from survival in the long-term model. For cIAI, treatment with ATM-AVI ± metronidazole versus meropenem ± colistin leads to a gain of 0.23 LYs, 0.21 QALYs, and incremental costs of €3,970, generating an ICER of €18,997/QALY. For the HAP/VAP, there was a gain of 0.46 LYs, 0.42 QALYs, and incremental costs of €4,480, generating an ICER of €10,725/QALY. For both cIAI and HAP/VAP indications, the ICER is well below the willingness-to-pay threshold of €30,000/QALY accepted in Italy. CONCLUSIONS: The introduction of ATM-AVI will lead to improved outcomes for patients with cIAI and HAP/VAP, with a minimal cost impact in the Italian setting, meaning that ATM-AVI is a cost-effective strategy compared to meropenem.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023-cem-atm-avipdf131965-pdf.pdf?sfvrsn=628ce83f_0","title":"ISPOR EU 2023 CEM ATM AVIpdf131965.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131965","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Bulgaria National Stroke Program: Prevention Strategies","id":"b9ec64f3-f9a8-4cce-a230-8472352ef3de","sessionCode":"HPR1","topDisplay":"Dacheva A1, Georgiev A2, Vutova Y2, Slavchev G3, Djambazov S4 1Medtronic International Trading Sàrl, Sofia, 23, Bulgaria, 2HTA Ltd., Sofia, 22, Bulgaria, 3Medtronic International Trading Sàrl, Sofia, 22, Bulgaria, 4Medical University Pleven, Sofia, 23, Bulgaria","locationCode":"3059","description":"\r\n\t<div>OBJECTIVES: This abstract aims to analyze the strategies for stroke prevention in Bulgaria employed by the National Stroke Program including public education, lifestyle modification programs, control of risk factors, and primary prevention through pharmacological interventions in high-risk groups. The objective is to assess the effectiveness and potential impact of these strategies in the context of stroke prevention in Bulgaria. METHODS: A comprehensive analysis of the strategies for stroke prevention in Bulgaria is conducted based on a review of relevant literature and existing evidence. The methods involve evaluating the applicability, feasibility, and evidence-based effectiveness of each strategy within the Bulgarian context. RESULTS: Public education campaigns are identified as a crucial strategy for raising awareness about stroke risk factors and early signs among the population. These campaigns can contribute to early recognition of symptoms and prompt medical intervention. Lifestyle modification programs focusing on balanced nutrition, regular physical activity, and smoking cessation are also highlighted as essential components of stroke prevention. The control of risk factors, including hypertension, diabetes, and high cholesterol, is found to be crucial in reducing stroke risk. Lastly, primary prevention through pharmacological interventions in high-risk groups, such as anticoagulant therapy or carotid endarterectomy, is identified as an effective approach. CONCLUSIONS: The analyzed strategies for stroke prevention in Bulgaria, including public education, lifestyle modification programs, control of risk factors, and primary prevention through pharmacological interventions, offer a comprehensive approach to reducing the burden of stroke. Implementing these strategies requires collaboration among healthcare professionals, policymakers, and the public to ensure effective implementation, widespread adoption, and long-term sustainability. By addressing stroke prevention through these multifaceted strategies, Bulgaria has the potential to significantly improve population health outcomes and reduce the incidence of stroke.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dachevahpr1poster128943-pdf.pdf?sfvrsn=40608977_0","title":"ISPOREurope23_Dacheva_HPR1_POSTER128943.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128943","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Knowledge, Attitude, and Practice of Pharmacy Professionals Against Dispensing Antibiotics Without Prescription in Ethiopia","id":"5e9a019f-3695-4740-8d53-8472dbe84195","sessionCode":"HSD5","topDisplay":"Haile K1, Yabeyu A2 1University of Gondar, Gondar, 1, Ethiopia, 2Ambo University, addis ababa, Ethiopia","locationCode":"4015","description":"\r\n\t<div>OBJECTIVES: To assess the knowledge, attitude and practice of community pharmacists and pharmacy assistants towards Dispensing Antibiotics and Without Prescription (DAWP) METHODS: A cross-sectional study was conducted, and data was collected from community pharmacists and pharmacy assistants during the eighth national pharmacist’s day, which was held on December 4, 2021, in Addis Ababa, Ethiopia. A pre-tested self-administered questionnaire comprise of four sections (socio-demographic characteristics, and knowledge, attitude and practice towards DAWP was used. The data was analyzed using SPSS version 26 and descriptive statistics (mean, percentage, standard deviation) were computed. Binary logistic regression was used to predict determinates of DAWP. RESULTS: A total of 175 community pharmacy professional were invited in the study, with 158 (111 pharmacists and 47 pharmacy assistants) completing the survey for 90.3% response rate. Most of the participants (86.7%) were aware that DAWP is illegal in Ethiopia. Despite their knowledge, the extent of DAWP was found to be 67.7%. The most common reason given by study participants for DAWP was that most patients do not want to consult prescribers unless the infection appears serious (53.2%). Pharmacy professionals with educational background of masters and above (AOR= 0.354, 95% CI: 0.013–0.744, P= 0.045), and with two to five years of working in community pharmacy (AOR= 0.745, 95% CI: 0.595–0.933, P= 0.010) had a lower tendency to DAWP, respectively CONCLUSIONS: Despite majority of pharmacy professional are aware that DAWP is illegal and contributes to antibiotic resistance, they commonly DAWP for common cold and diarrheal diseases. Strict enforcement of existing antibiotic supply policies, and ongoing educational support for community pharmacy professionals on the judicious use of antibiotics is recommended.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hailehsd5posterv3132330-pdf.pdf?sfvrsn=a715547f_0","title":"ISPOREurope23_Haile_HSD5_POSTERV3132330.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132330","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Еconomic Evaluation of Intravascular Ultrasound (IVUS) for Imaging in Patients with Coronary Artery Disease in Bulgaria","id":"3321f14d-b8cb-44cd-8484-847fd20819e9","sessionCode":"EE8","topDisplay":"Dacheva A1, Slavchev G2, Seitaridou Y3, Vutova Y4, Djambazov S5, Vekov T6 1Medtronic International Trading Sàrl, Sofia, 23, Bulgaria, 2Medtronic International Trading Sàrl, Sofia, 22, Bulgaria, 3Faculty of Pharmacy, Medical University - Sofia, Sofia, 22, Bulgaria, 4HTA Ltd., Sofia, 22, Bulgaria, 5Medical University Pleven, Sofia, 23, Bulgaria, 6Medical University Pleven, Pleven, Bulgaria","locationCode":"1037","description":"\r\n\t<div>OBJECTIVES: Intravascular ultrasound (IVUS) is an imaging modality used primarily in interventional cardiology to characterize lesion morphology, quantify plaque significance, guide stent sizing, assess stent implantation, and identify procedural complications. The primary objective of this study was to perform an economic assessment of IVUS for imaging in patients with coronary artery disease in Bulgaria. METHODS: The economic method chosen for the evaluation of IVUS is a cost-utility analysis. In addition, a cost-effectiveness analysis was also conducted. Health benefits for patients in the applied model are measured as quality-adjusted life years (QALYs) and life-years added (LYs). A model evaluating the cost-effectiveness of IVUS-guided percutaneous coronary intervention (PCI) versus PCI with the classic angiography-guided strategy was build. The duration of the time horizon is up to a lifetime. Costs and health benefits are discounted with 3,5%. The cost estimate reflects the payer's perspective (NHIF). A probabilistic and one-way sensitivity analysis was performed. RESULTS: The results show that IVUS is cost-effective in STEMI patients compared to angiography guided PCI (ICUR = BGN 8,757.12/QALY and ICER = BGN 4,722.67/LYs), with a favorable cost-effectiveness threshold of BGN 60,636.00. Calculated ICUR and ICER values for IVUS in UA/NSTEMI patients (ICUR = 16,238.27 BGN/QALY and ICER = 9,006.28 BGN. /LYs) relative to alternative medical activity (CA-led PCI) are located in the second quadrant of the cost-effectiveness graph (south-east - lower cost at higher health benefit values) below the conventionally accepted threshold for favorable cost-effectiveness of BGN 60,636.00/QALY. CONCLUSIONS: Routine use of IVUS has been shown to result in fewer serious cardiovascular events within the first year, a significant reduction in the cost of drug-eluting stent placement, a significant reduction in costs due to avoidance of stent edge apposition and dissection, in-stent restenosis (ISR), stent thrombosis, etc.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dachevaee8poster128976-pdf.pdf?sfvrsn=31e9f15b_0","title":"ISPOREurope23_Dacheva_EE8_POSTER128976.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128976","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Identifying Oncology Effectiveness Endpoints Using Administrative Secondary Databases: An Example Utilising Hospital Episode Statistics in England","id":"59feea9a-d0db-4843-b6d0-84f5128b9030","sessionCode":"RWD16","topDisplay":"Rolfe C1, Alexander M2, Wilkes E2, Heaton D3 1OPEN Health, Marlow, BKM, UK, 2OPEN Health, London, UK, 3OPEN Health, Runcorn, HAL, UK","locationCode":"6074","description":"\r\n\t<div>OBJECTIVES: Administrative databases collect electronic health records data that can have a secondary use for pharmaco-epidemiological research and support generation of real-world evidence. In oncology, overall survival and progression-free survival are often used as endpoints to evaluate treatment effectiveness, but administrative databases generally don’t record progression markers (e.g. repeat stage recording, laboratory measures, response to treatment). As such proxy measures are required. Here, we outline our methodology for developing proxy measures for a study of melanoma using Hospital Episode Statistics in England. METHODS: Stages of diagnoses and effectiveness endpoints were defined with clinical experts’ input from diagnoses coded using the International Classification of Diseases version 10 (ICD-10 diagnostic) and procedures and treatment administration coded using the Office of Population Censuses Surveys Classification of Surgical Operations and Procedures, 4th Revision (OPCS-4). RESULTS: Using ICD-10 diagnosis codes for melanoma diagnosis and presence of metastases lymph node removal procedure codes and groups of chemotherapy administration codes), it was possible to determine stage 0, stage I/II, III and IV Stages of melanoma, and resectable versus unresectable melanoma. The following proxy-effectiveness endpoints were also definable: time-to-next treatment (time from start of systemic treatment to date of initiation of next OPCS treatment combination), progression-free survival (reaching disease next stage, the presence of a new metastasis, or death; a sensitivity analysis also used start of a new OPCS treatment combination as an indicator of progression), recurrence-free survival (with recurrence being defined as new metastasis or a new lymph node removal surgery or a new cutaneous melanoma diagnosis >30 days after last recorded diagnosis) and overall survival (using absence of a record of death). CONCLUSIONS: Derivation of oncology effectiveness endpoints using diagnoses, treatments and procedures recorded in HES is possible; nevertheless, data granularity remains lower than data that would be collected via primary data collection methods such as chart review.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23rolferwd16poster131870-pdf.pdf?sfvrsn=fa78c9bd_0","title":"ISPOREurope23_Rolfe_RWD16_POSTER131870.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131870","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Bridging the Gap: Exploring the PICO Vs Estimand Frameworks in EU Health Technology Assessment (HTA)","id":"e3986691-a55b-466d-806a-84fe346cc231","sessionCode":"HTA71","topDisplay":"Sieverding M1, Allignol A2 1Veramed, Berlin, BE, Germany, 2Daiichi Sankyo Europe, Darmstadt, Germany","locationCode":"5018","description":"\r\n\t<div>OBJECTIVES: This poster aims to provide a comprehensive overview of the PICO and Estimand frameworks within the EU HTA process. We want to identify challenges and pitfalls in translating between them as well as discussing strategies to optimize evidence generation and mitigate risks associated with mismatches between frameworks. METHODS: The poster presents a detailed analysis of the Estimand and PICO frameworks and their application in the EU HTA process. It includes a thorough review of relevant literature, guidelines, and regulatory requirements. Practical examples and case studies are utilized to illustrate the challenges and consequences of misspecification when translating between Estimands and PICOs. RESULTS: The analysis highlights the key attributes of the Estimand framework, including treatment, population, variable, population-level summary, and intercurrent events. It also elucidates the components of the PICO framework, focusing on population, intervention, comparator, and outcomes. The poster emphasizes the lack of explicit guidance in the PICO framework regarding the handling of intercurrent events, which poses challenges in evidence generation. The case studies presented demonstrate the potential consequences of misspecification and error in preplanning when translating between Estimands and PICOs. The importance of considering real-world treatment scenarios, including appropriate comparators, and employing methods such as indirect treatment comparisons is discussed as strategies to overcome these challenges. CONCLUSIONS: Clear communication and collaboration between regulatory and market access teams are essential to align objectives and optimize evidence generation in the EU HTA process. Decision makers should proactively address mismatches between Estimands and PICOs during trial design to mitigate risks and ensure high-quality evidence. By understanding the nuances and implications of the PICO and Estimand frameworks, key decision makers can navigate the evolving EU HTA landscape more effectively. This knowledge is relevant to make informed decisions, enhance the quality and relevance of evidence, and ultimately improve patient access to innovative therapies in the European Union.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-europe-posteronline131673-pdf.pdf?sfvrsn=10c4ee6a_0","title":"ISPOR Europe Poster_Online131673.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131673","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparative Analysis of Coding Schemas for Assessing Major Bleeding Risk Between Non-Vitamin K Antagonist Oral Anticoagulants and Warfarin","id":"04787a8c-9735-4865-8943-85e49ef5c6fa","sessionCode":"CO36","topDisplay":"Chien HT1, Chao TF2, Wang R3, Lin SY4, Lin FJ5 1Graduate Institute of Clinical Pharmacy, College of Medicine, National Taiwan University, Taipei, TPQ, Taiwan, 2Division of Cardiology, Department of Medicine, Taipei Veterans General Hospital, Taipei, Taiwan, 3Daiichi Sankyo Inc, Basking Ridge, NJ, USA, 4Department of Pharmacy, National Taiwan University Hospital, Taipei, Taiwan, 5Graduate Institute of Clinical Pharmacy, College of Medicine, National Taiwan University, Taipei, Taiwan","locationCode":"1035","description":"\r\n\t<div>OBJECTIVES: This study aimed to investigate the impact of utilizing different diagnosis coding schemas on evaluating the major bleeding risk associated with non-vitamin K antagonist oral anticoagulants (NOACs) and warfarin. METHODS: A retrospective cohort study was conducted using Taiwan's National Health Insurance Research Database to identify atrial fibrillation patients newly prescribed NOACs or warfarin between June 2012 and June 2019. The primary outcome was major bleeding events, including gastrointestinal bleeding (GIB), intracranial hemorrhage (ICH), and other major bleeding (OMB). Different coding schemas proposed by Cunningham et al., the FDA, and Yao et al. for defining major bleeding were compared. The impact of including or excluding patients with a bleeding history was also explored. Propensity score matching was performed to ensure covariate balance. Incidence rates and hazard ratios (HR) were estimated to evaluate major bleeding risks between NOACs and warfarin. RESULTS: After matching, both NOAC and warfarin groups comprised 20,704 patients. Across coding schemas, NOACs consistently showed significantly lower rates of all major bleeding (HR range: 0.73~0.76) and ICH (HR range: 0.43~0.63) compared to warfarin, while GIB rates were similar between the treatment groups (HR range: 0.87 to 0.90; p-value all >0.05). The number of events varied based on the coding stringency, with Cunningham et al. algorithm yielding the highest, followed by the FDA and YAO et al. For ICH, focusing on the primary diagnosis or considering only spontaneous cases (excluding traumatic ICH) resulted in a greater risk reduction associated with NOAC. Risk estimates remained consistent when excluding patients with a bleeding history. CONCLUSIONS: In general, different coding schemas had minimal impact on comparing major bleeding between NOACs and warfarin, except for ICH. Relying solely on the primary diagnosis and considering only spontaneous ICH yielded a greater risk difference in ICH favoring NOACs over warfarin.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hsiutingco36posterv2129138-pdf.pdf?sfvrsn=4866dd4d_0","title":"ISPOREurope23_HsiuTing_CO36_PosterV2129138.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129138","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Single-Inhaler Triple Therapy (FF/UMEC/VI) in COPD Using the Fulfil Trial: China Medical Insurance System Perspective","id":"8590b397-d686-41c0-a5d2-85f96db0be76","sessionCode":"EE103","topDisplay":"Noorduyn SG1, Wang X2, Martin A3, Cai R4, Tang Z5, Bointon D4, Sun A5, Ismaila A6 1Value Evidence and Outcomes, R&D Global Medical, GSK, Mississauga, ON, Canada, 2ICON Health Economics, ICON plc, Stockholm, Sweden, 3Value Evidence and Outcomes, GSK, Brentford, UK, 4ICON Health Economics, ICON plc, Amsterdam, Netherlands, 5Value Evidence and Outcomes, GSK, Shanghai, China, 6Value Evidence and Outcomes, GSK, Collegeville, PA, USA","locationCode":"2037","description":"\r\n\t<div>OBJECTIVES: To assess the cost-effectiveness of single-inhaler fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) versus budesonide/formoterol (BUD/FOR) for patients with symptomatic COPD and a history of exacerbations from a China medical insurance system perspective based on data from the FULFIL trial (NCT02345161). METHODS: This study adapted a previously published hybrid decision tree/Markov economic model (Schroeder et al. Respiratory Medicine. 2019). The initial decision tree replicated the outcomes in 24 weeks of the FULFIL trial. Outputs from the decision tree formed the starting position of the Markov model, which comprised six health states based on COPD severity and the presence/absence of recent exacerbation experience. The Markov was parameterized based on data from the three-year TORCH (The Towards a Revolution in COPD Health) study. Health state utilities were based on a cross-sectional study in China, and the disutilities of exacerbation events were sourced from published literature. China healthcare resource unit costs and drug costs were applied, with costs (2022 CNY ¥) and health outcomes discounted at 5% annually. The analysis was deterministic with a lifetime horizon. Scenario, one-way and probabilistic sensitivity analyses were performed to test the robustness of key assumptions and input parameter values. RESULTS: Compared with BUD/FOR, FF/UMEC/VI provided an additional 0.678 life years (LYs) and 0.703 quality-adjusted life years (QALYs), with a cost saving of ¥11,493. Thus, FF/UMEC/VI was a dominant treatment versus BUD/FOR, and remained dominant across all scenario and one-way sensitivity analyses. In the probabilistic sensitivity analysis, FF/UMEC/VI was a dominant treatment option across all simulations and thus the probability that FF/UMEC/VI was cost-effective versus BUD/FOR was 100% at any willingness-to-pay threshold. CONCLUSIONS: FF/UMEC/VI single-inhaler triple therapy improved health outcomes and was a dominant treatment compared with BUD/FOR for patients with symptomatic COPD in China. FUNDING: GSK (217637)</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23noorduynee103poster130476-pdf.pdf?sfvrsn=3b64f7a2_0","title":"ISPOREurope23_Noorduyn_EE103_POSTER130476.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130476","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Postpartum Hemorrhage: A Look into Colombia's Health System","id":"779ffe72-a2ef-4431-855b-861f549618ec","sessionCode":"EPH33","topDisplay":"Casas-Ramirez D1, Perez CA2, Moreno S1 1Novo Nordisk, Bogotá, CUN, Colombia, 2Novo Nordisk, Bogota, Colombia","locationCode":"3035","description":"\r\n\t<div>OBJECTIVES: Postpartum hemorrhage (PPH) is the principal cause of maternal mortality worldwide. Globally, PPH causes 20% of maternal deaths and was responsible for 8% of maternal deaths in developed countries and 20% of maternal deaths in developing regions during 2003-2009. The objective of this analysis is to generate evidence related to the situation of postpartum hemorrhage in Colombia to support decision-making in public health METHODS: A retrospective analysis of data was carried out regarding the ICD-10 O721, code related to immediate postpartum hemorrhage (IPP) between 2019 and 2022 in Colombia. The medicines administered were extracted from the drug prescription registry (MIPRES). Hospitals and procedures were taken from the Health Benefits Information System (RIPS). Mortality records were obtained from the Vital Statistics Death Registry (SISPRO). Sociodemographic Information was obtained from the Public Health Surveillance System (SIVIGILA) RESULTS: On average, 250 cases of HPP occur annually, mostly in the departments of Valle del Cauca (18%), Antioquia (15%), and Cauca (10%) with a mortality rate of 9%. 35% of the cases are affiliated with the contributory regime, 58% to the subsidized regime, and the remaining 7% to another type of affiliation. Tranexamic acid is the standard of care and is reimbursed by the National Benefit Plan; the second therapeutics option is reimbursed by maximum budget and corresponds to uterotonics (mainly misoprostol) which represent 95% of the medicines used in this segment. The final option is a mechanical intervention such as a hysterectomy that is rarely required (1%) CONCLUSIONS: PPH in Colombia is one of the principal causes of preventable maternity morbidity and mortality. It is essential to create knowledge of the event and the development of specific strategies according to local needs, to reduce the incidence in the country and raise awareness among the population</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23casaseph33poster133844-pdf.pdf?sfvrsn=8bfeff83_0","title":"ISPOREurope23_Casas_EPH33_POSTER133844.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133844","diseases":[{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Surrogate Endpoints Used in NICE Technology Appraisals for Oncology and Non-Oncology Indications, 2022–23","id":"c6b57733-813b-459b-b94a-86dabeaa7675","sessionCode":"HTA64","topDisplay":"Heptinstall A, Adkins E Maverex Ltd, Newcastle upon Tyne, Tyne and Wear, UK","locationCode":"4078","description":"\r\n\t<div>OBJECTIVES: Surrogate endpoints may be used to predict patient-relevant clinical outcomes when it would be unethical or impractical to conduct a trial to assess the final patient-relevant outcome (e.g., requiring unattainably long follow-up, or when the clinical benefit of improving the surrogate endpoint is well established). The use of surrogate endpoints in health technology assessment (HTA) is increasing, particularly in oncology. While this can allow faster access to new treatments, it increases uncertainty for decision-makers and payers. Consequently, the National Institute for Health and Care Excellence (NICE) is working with international organisations to develop guidance on their use in cost-effectiveness analysis. We aimed to understand the frequency and type of surrogate primary efficacy endpoints currently being used in NICE decision-making. METHODS: HTAs published on the NICE website from 01.06.2022–31.05.2023 were reviewed. The use of surrogate endpoints as primary efficacy outcomes, therapeutic area, and recommendation decision were recorded. RESULTS: Of the 76 HTAs reviewed, 68% included at least one surrogate primary efficacy endpoint. Six (8%) were not recommended by NICE, of which five used surrogates. The most common surrogates were progression-free survival (31%) and measures of response rate (31%). For HTAs in oncology indications, surrogates were used in 82% (32/39), mainly progression-free survival (50% [16/32]), response rate (28% [9/32]), and other survival outcomes such as disease- or event-free survival (25% [8/32]) (with two randomised controlled trials having dual-primary endpoints). For non-oncology indications, surrogates were used in 54% (20/37), mainly response rate (35% [7/20]). CONCLUSIONS: Surrogate primary efficacy endpoints are frequently used and widely accepted by NICE when making a recommendation in both oncology and non-oncology indications. The introduction of clear guidance on their use in HTA will help to ensure a consistent approach, reduce uncertainty in decision-making, and allow fairer access to new treatments.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131918","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Overcrowding and Boarding Time: Emergency Department Performance and Impacting Factors","id":"e46efe91-4cda-42f1-b3c5-86e8aa58ace6","sessionCode":"OP6","topDisplay":"Foglia E1, Asperti F1, Bellavia D1, Schettini F1, Bellini R2, Boverio R2, Gualco C2, Zanelli C2, Porazzi E1 1LIUC University, Castellanza, VA, Italy, 2Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo, Alessandria, Italy, Italy","locationCode":"5077","description":"\r\n\t<div>OBJECTIVES: Higher boarding times (BTs) usually affect the Emergency Departments’ (EDs) activities, resulting in a lower care quality level for patients, waiting for an inpatient bed. This study aims to evaluate the different EDs performance indicators affecting BT. METHODS: Real-life data were collected in an ED in Northern Italy, considering the years 2019-2022, and referring to 189,976 accesses (134,199 adults and 55,777 pediatrics). The following KPIs were assessed: time between ED access and the first visit, BT, ED overall stay, ED repeated access within 72 hours. Both the National Emergency Department Overcrowding Scale (NEDOCS) and the Emergency Department Working Index (EDWIN) were adopted. All measures were compared to the national standards. A bivariate correlation was performed to explain endogenous and exogenous factors, that could impact on the patients’ BT. RESULTS: Time between ED access and the first visit results over the national standards, for all the patients, except for white and green patients (referring to pediatric department). Repeated accesses within 72 hours increased for white and green patients; a decreasing trend was registered for red and yellow patients, especially in the children’s pediatric department. Stratifying the accesses and evaluating the NEDOCS index, in the pediatric department overcrowding situations were demonstrated only for the light-blue and green patients. The bivariate analyses revealed that time band (β=-0.057; p-value=0.000), patient's code (β=-0.028; p-value=0.005), and ED organizational assets (β=0.039; p-value=0.000) could affect the BT, with an increase during the night and for the less complex codes. CONCLUSIONS: BT resulted higher at night, as a consequence of the reduced number of recovered patients, increasing the overcrowding. The overall process time is enlarged over the years 2020-2021, also due to the COVID-19 spread; better results emerged in 2022. EDWIN and NEDOCS indexes could be considered as relevant indicators, providing more comprehensive information for understanding EDs dynamics and improving EDs management.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"bb8fd992-de86-4d1a-8bec-f6e2c928db96","parentId":"00000000-0000-0000-0000-000000000000","title":"Organizational Practices","urlName":"organizational-practices"}],"categoryIds":["bb8fd992-de86-4d1a-8bec-f6e2c928db96"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bellaviaop6poster131501-pdf.pdf?sfvrsn=f2dbd563_0","title":"ISPOREurope23_Bellavia_OP6_POSTER131501.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131501","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Insurer and Patient Costs for Repeat Breast Surgery After Initial Lumpectomy for Breast Cancer","id":"8b5bea1f-cbbd-4c17-83a3-8724fa972a16","sessionCode":"EE67","topDisplay":"Wing S1, Liu Y2, Zheng F2, Selber JC3 1Intuitive Surgical, Portland, OR, USA, 2Intuitive Surgical, Sunnyvale, CA, USA, 3Corewell Health East, Grand Rapids, MI, USA","locationCode":"2021","description":"\r\n\t<div>OBJECTIVES: ~20-25% of patients who undergo a primary lumpectomy for the treatment of breast cancer require a reoperation due to adverse outcomes like positive surgical margins or cancer recurrence. We analyze the economic impact of patients who require repeat breast tissue resection as part of their treatment. METHODS: We used the Merative MarketScan Research Database, containing patient-level data on individuals with commercial or Medicare Supplemental insurance coverage in the United States. From 2016-2021, we identified women who received an index lumpectomy for the treatment of primary breast cancer and identified their healthcare encounters 1 year post-operatively, including any repeat lumpectomies or mastectomies. We aggregated total healthcare expenditures and out-of-pocket costs among patients with at least 3 years of continous enrollment in their insurance plan. Patients with greater than 3 breast excision/resection procedures were excluded. RESULTS: Among 9,409 patients with a primary lumpectomy, 25% (n=2,315) underwent a secondary breast surgery, of which 75% (n=1,730) was a repeat lumpectomy and 25% (n=585) was a mastectomy. Median time to second procure was 3 weeks (IQR: 1.3-7.7) and 5% (n=438) underwent a 3rd procedure. Lower age and smoking, but not high BMI, were associated with increased risk of reoperation, as well as risk of mastectomy instead of lumpectomy as a secondary procedure. Median healthcare expenditure for primary lumpectomy plus one year follow up was $38,811 USD ($695 out-of-pocket). Among patients with a secondary procedure, median healthcare expenditure from primary lumpectomy plus one year follow up was $43,761 ($980 out-of-pocket) for repeat lumpectomy and $63,782 ($777 out-of-pocket) for subsequent mastectomy patients. CONCLUSIONS: While lumpectomy is the most common surgical therapy for early-stage breast cancer, it often is not a definitive procedure, which can result in large added financial and operational burdens. Patient risk stratification and intraoperative adjuncts are needed to minimize risk of reoperation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23wingee67poster133796-pdf.pdf?sfvrsn=7739155_0","title":"ISPOREurope23_Wing_EE67_POSTER133796.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133796","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Progressive Pulmonary Fibrosis: A Modelling Analysis of Long-Term Progression Based on Inbuild","id":"cd34b28d-1e11-4437-a06a-873144943775","sessionCode":"CO31","topDisplay":"Raspin C1, Ramon A2, Baldwin M3, Diamantopoulos A1, Valenzuela C4, Cottin V5 1Symmetron Limited, London, UK, 2Boehringer Ingelheim España S.A., Sant Cugat del Vallès, Spain, 3Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, 4Pulmonology Department, Universidad Autónoma de Madrid, Madrid, Spain, 5National Coordinating Reference Center for Rare Pulmonary Diseases, Louis Pradel Hospital, University Claude Bernard, Lyon, France","locationCode":"1030","description":"\r\n\t<div>OBJECTIVES: Some patients with interstitial lung disease (ILD) develop a progressive fibrosing phenotype (PF-ILD) or progressive pulmonary fibrosis (PPF). Nintedanib has been shown in clinical trials to delay the progression of idiopathic pulmonary fibrosis (IPF) and non-IPF PPF. Whilst some studies have analyzed progression for up to 2 years in patients, little is known about progression in PPF past this timepoint. The objective of our analysis was to model disease progression or death over the lifetime of PPF patients. METHODS: This analysis used data from patients with non-IPF PPF in the phase 3 INBUILD trial. Disease progression was defined as a ≥10% relative decline in forced vital capacity (FVC) within 12 months. Change in FVC was analyzed using regression analysis with independent models for nintedanib and placebo, plus a general model that included all patient data and a treatment coefficient. Survival analysis was conducted on the time-to-progression estimates from INBUILD as an alternative to the FVC regression analysis. The prediction of outcomes related to disease progression and mortality were simulated and expressed as time to disease progression using an individual patient-level simulation model. RESULTS: The parameters in the regression models included baseline FVC, age, criteria for progressive ILD, acute exacerbation and interaction effects between time and fixed factors. All parameters were statistically significant at the 5% level with the exception of acute exacerbation. The incremental time to disease progression or death for nintedanib over placebo was 1.56 years using the independent models and 2.89 years using the general model. In the alternative time-to-progression analyses, the best fitting survival models were exponential, log-normal and Weibull, and the incremental time to disease progression or death ranged from 0.52 to 0.99 years. CONCLUSIONS: This modelling suggested that nintedanib could delay long-term disease progression or death in patients with PPF.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23raspinco31poster130596-pdf.pdf?sfvrsn=dab4e7e2_0","title":"ISPOREurope23_Raspin_CO31_POSTER130596.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130596","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Leave or Stay? Influencing Factors of Intention to Leave or Stay in Healthcare Profession After Coronavirus Pandemic Among Frontline Nurses","id":"be5bd9af-4c66-446d-a4e3-876c93b2181a","sessionCode":"EPH53","topDisplay":"Bogdán P1, Zrínyi M2, Verzár Z1, Haitham K1, Kozmann K1, Boncz I1, Szabó L1, Pakai A2 1University of Pécs, Pécs, Hungary, 2University of Pécs, Pécs, ZA, Hungary","locationCode":"3002","description":"\r\n\t<div>OBJECTIVES: Aim of our study was to determine the factors that leads healthcare workers to stay or leave their profession after every difficulty that pandemic caused. METHODS: The study was conducted between October 2022 and November 2023 at the University of Pécs, Clinical Centre – Regional coronavirus treatment centrum, however participants were recruited all over the country. Non-random, professional, purposive expert sampling was used to involve healthcare workers with at least 3 months of experience, working with patients on coronavirus department. Data collection was carried out using online questionnaire, which included sociodemographic, self-edited questions, the \"Motivation at Work Scale” and the “Impact of Event Scale - Revised.” In descriptive statistics we calculated means, standard deviation, minimum, maximum score, absolute and relative frequency. Microsoft Office Excel 2016 and SPSSv2.5 was used for statistical calculations such as Pearson correlation, ANOVA, Chi square test, independent sample t-test (p<0.05). RESULTS: On the “IES-R” scale, average score was 36.71±16.35 with 0 point lowest and 73 point highest score. On the “MAWS” scale average score was 69.53±21.04 with 18 point lowest and 119 point highest score. Correlation was not found between “IES-R” and “MAWS” scores (p>0.05). There is a positive correlation between intention to stay and “MAWS” scores (r=0.332; p<0.05). We found negative correlation between intention to leave and “IES-R” scores. (r=-0.198; p<0.05). There is no connection between age, work experience and “MAWS” scores. We measured significantly higher scores on “MAWS” scale in the group that currently carries out university studies (76.53±20.97), compared to the group with no university studies (67.56±20) (p<0.05). CONCLUSIONS: Intention to leave is highly influenced by the negative effect of working on a COVID hospital department. Therefore, mental support is a crucial component to decrease “loss” of healthcare workers. Higher educational level influenced work motivation positively.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133725","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Vaccine Preparation Time, Cost, and Preference Comparison Between Pre-Filled Syringe Formulations and Vaccines That Require Reconstitution: A Targeted Literature Review","id":"dff20557-aa55-49e7-b6a4-879934f765e1","sessionCode":"HSD22","topDisplay":"Mehta D1, Kimball-Carroll SM2, Krivelyova A3, Lai KZH4, Van de Velde N1 1Moderna Inc., Cambridge, MA, USA, 2ICON Plc., Burlington, ON, Canada, 3ICON Plc., London, UK, 4ICON Clinical Research, Toronto, ON, Canada","locationCode":"4031","description":"\r\n\t<div>OBJECTIVES: The targeted literature review (TLR) aimed to evaluate vaccine preparation time, associated labor costs and preference of healthcare practitioners for vaccines that require reconstitution (VRR) vs vaccines that are supplied as a pre-filled syringe (PFS). METHODS: A TLR was conducted in Embase and MEDLINE to identify studies that measured time or costs associated with the two vaccine formulations. We supplemented the list by searching the references and citations referenced by included studies. Eligibility criteria were developed based on formulation and outcomes. Costs in USD dollars were extracted. RESULTS: Of 344 studies screened, 9 were eligible, including 2 time and motion, 2 with labor costs, 1 interview-based assessment of time and cost, and 8 with preference data. From 3 studies, time associated with vaccine administration averaged 30, 33 and 36 seconds for PFS and 66, 70.5 and 75 seconds for VRR. Considering median pharmacist pay of $61.8/hr the labor cost for administering PFS is $0.62/dose and $1.29 for administering VRR. Assuming similar annual vaccination rate for flu of 69.8% this translates into $26.1 million projected labor cost savings for PFS vaccines. Similar administration costs were reported in other countries, with a mean cost/dose for PFS of $0.82 and $1.64 for VRR in Belgium, and $3.56 and $6.23, respectively, in Malaysia. Studies that reported preference found that PFS was the preferred formulation, with main reasons being ease of administration and reduced immunization errors (number of VRR errors was 47 vs. 10 for PFS; out of total 57 immunization errors in 192 preparations). CONCLUSIONS: Compared with VRR, PFS vaccines require roughly half the time for administration, half the mean cost in labor and are associated with a reduced number of errors. Vaccine programs that select PFS formulations might have the potential for significant savings due to reduced labor and error-related costs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/vaccines-pfs-vs-reconstitution-tlr-poster132334-pdf.pdf?sfvrsn=4b7fcb43_0","title":"Vaccines PFS vs Reconstitution TLR Poster132334.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/vaccines-pfs-vs-reconstitution-tlr-poster-supp132334-pdf.pdf?sfvrsn=b2b3be52_0","title":"Vaccines PFS vs Reconstitution TLR Poster Supp132334.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132334","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Cost-Effectiveness of Tradition Chinese Medicine Icaritin Versus Cinobufotalin in Patients with Unresectable Advanced Hepatocellular Carcinoma in China","id":"cffd6987-c9e3-466c-aad3-887d73726e9f","sessionCode":"EE77","topDisplay":"Guo J1, Xuan J2, Zhuo Y3 1Shanghai Centennial Scientific Ltd, Toronto, ON, Canada, 2Health Economic Research Institute, School of Pharmacy, Sun Yat-sen University, Guangzhou, Guangdong, China, 3Health Economic Research Institute, School of Pharmacy, Sun Yat-Sen University, Guangzhou, Guangdong, China","locationCode":"2004","description":"\r\n\t<div>OBJECTIVES: To evaluated the cost-effectiveness of patients with unresectable advanced hepatocellular carcinoma using traditional Chinese medicine Icaritin versus Cinobufotaline in China from societal perspective. METHODS: A partitional survival model was conducted strictly followed by China Guidelines for Pharmacoeconomic Evaluations to evaluate the incremental cost-effectiveness ratio (ICER) of two medicine with a lifetime horizon. To fit parametric models, a log-normal was generated for Cinobufotaline’s overall survival (OS) and exponential distribution for Icaritin’s OS, an exponential distribution for progression free survival of both groups. The efficacy data input was collected from clinical trial SNG1705ICR-1. The utility data, probability of treatment related adverse events were collected from the literature. Cost inputs were derived from public database and the literature. Model robustness was assessed via one-way sensitivity and probabilistic sensitivity analyses. RESULTS: Comparing with Cinobufotaline, Icaritin had a higher cost (¥192,462 versus ¥5,351) and longer life year (1.26 versus 0.77), quality-adjusted life years (QALY) gained of 0.46 (1.06 versus 0.60) over a lifetime horizon. Key drivers were the lower probability of adverse events and better clinical efficacy of Icaritin, the ICER of the baseline result was ¥358,709 per QALY gained. CONCLUSIONS: Overall, from the societal perspective, Icaritin is likely to be a cost-effective option compared with Cinobufotaline for Chinese unresectable advanced hepatocellular carcinoma patients s. Meanwhile, inclusion of broader evidence on clinical efficacy using first-line therapy from real-world studies among Chinese population is suggested, which could solidate the economic evidence and further improve the use of Icaritin.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster---lcc-1-2130279-pdf.pdf?sfvrsn=2095742_0","title":"Poster - LCC 1.2130279.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130279","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Burden of Informal Caregivers of People Diagnosed With Myasthenia Gravis","id":"631c2a1a-3eee-4b51-81d2-887f83f08794","sessionCode":"PCR13","topDisplay":"Dewilde S1, Tollenaar NH2, De Ruyck F3, Phillips G4, Paci S5 1Services in Health Economics (SHE), Brussels, VBR, Belgium, 2Services in Health Economics (SHE), Sint-Pieters-Woluwe, Brussels Capital Region, Belgium, 3Argenx BVBA, Olsene, Belgium, 4Argenx US Inc., Boston, MA, USA, 5Argenx BVBA, Ghent, East Flanders, Belgium","locationCode":"6017","description":"\r\n\t<div>OBJECTIVES: Informal caregivers play a crucial role in supporting individuals with Myasthenia Gravis (MG), but their burden remains understudied. This analysis aimed to assess the burden experienced by informal caregivers of MG patients and explore its association with disease severity. METHODS: Pairs of adult MG patients and their informal caregivers from Germany, Italy, Spain and the UK contributed data to the observational digital MyRealWorld-MG study, and these data were combined with a paper survey among patients and caregivers in France. Patients were categorized based on their MG-ADL score as mild (0-4), moderate (5-9) or severe (>=10), whilst the caregiving burden was evaluated using the Zarit Burden Interview (ZBI-22). RESULTS: The study included 69 patients and their caregivers, who were predominantly spouses (84%). Mean ages of caregivers (53.7) and patients (50.7) were similar, but only 39% of caregivers were female, compared to 75% of patients. The mean daily caregiving hours were 5.1 (SD 6.5), with 20% of caregivers providing care for 15-49 hours/week and 30% for 50+ hours/week. Approximately 18% of caregivers reported feeling frequently or nearly always stressed, while 44% reported frequent or near-constant fear about the future. Additionally, 36% felt that their patient highly depended on them, and 30% reported their patient made them feel like they were the only one they could depend on. Financial strain was also evident, with 26% of caregivers frequently or nearly always feeling that they lacked sufficient funds to care for the MG patient alongside other expenses. The mean ZBI score was 24.3 (SD 15.0). Caregivers of patients with moderate or severe MG had significantly higher ZBI total scores (28.3 and 27.1, respectively) compared to caregivers of patients with mild MG (18.7). CONCLUSIONS: This analysis highlights the considerable burden experienced by informal caregivers of individuals with MG, as reflected by high ZBI scores across multiple domains.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023---poster-1-pcr13132641-pdf.pdf?sfvrsn=479a8538_0","title":"ISPOR EU 2023 - Poster 1 (PCR13)132641.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132641","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring the Impact of End-of-Life Designation on Access to Oncology Therapies in the United Kingdom","id":"f2ee5f7b-d275-4376-b6ad-89662108a4bd","sessionCode":"HTA10","topDisplay":"Azariadi A1, Georgallis M2, Howell A2 1Fortrea, London, LON, UK, 2Fortrea, London, London, UK","locationCode":"4047","description":"\r\n\t<div>OBJECTIVES: To examine the differences between Scottish Medicines Consortium (SMC) and National Institute for Health and Care Excellence (NICE) criteria for assessing oncology therapies with end-of-life (EOL) designation and understand how EOL status might impact decision-making. METHODS: Appraisals for oncology therapies assessed by SMC under the EOL process, published May, 2020–April, 2023, were reviewed and compared with NICE reports of the same treatments. The process was repeated for appraisals assessed by NICE during the same timeframe and cross-referenced to equivalent SMC appraisals. A data extraction tool captured decision details, EOL status, reimbursement restrictions/requirements, and how the Patient and Clinician Engagement meeting and Cancer Drugs Fund could affect recommendations. RESULTS: During the timeframe studied, SMC assessed 35 oncology therapies and NICE assessed 29 oncology therapies meeting their respective EOL criteria. There were differences in EOL status between agencies, with only 66% of therapies assessed by SMC also meeting NICE’s EOL criteria. Regardless, the outcome of the majority of appraisals were aligned. Reimbursement was granted in 87% and 91% of appraisals by SMC and NICE, respectively, with decision-making aligning in 87% of recommendations. Among medicines with divergent outcomes, the agencies reported similar concerns about evidence submitted, including the degree of certainty around the clinical/cost-effectiveness and application of EOL criteria. CONCLUSIONS: SMC and NICE criteria for EOL designation differ, hence oncology therapies granted EOL status do not always overlap. However, nearly all appraisals identified resulted in reimbursement, meaning equitable access in Scotland and England. In rare cases of divergent outcomes, the reasons for opposing recommendations and contribution level of EOL designation in decision-making remain unclear. With lack of uniformity in assessment criteria or detailed reporting on how decisions are reached, increased transparency on how different factors are weighted, particularly when reimbursement is not approved, could help inform future submissions for oncology therapies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23azariadihta10poster130393-pdf.pdf?sfvrsn=9bd0e91f_0","title":"ISPOREurope23_Azariadi_HTA10_POSTER130393.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130393","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Costs of Home Mechanical Ventilation and Oxygen Therapy in Russia: Real-World Data","id":"7694f67f-8d9b-4fd5-a5b0-89ed39667bb6","sessionCode":"EE100","topDisplay":"Krysanova V1, Krysanov I2, Ermakova V3, Makarova E4, Kurkin DV2 1Medical Institute of Continuing Education, BIOTECH University, Moscow, Russian Federation, 2A.I. Yevdokimov Moscow State University of Medicine and Dentistry, Moscow, MOW, Russian Federation, 3Sechenov First Moscow State Medical University, Moscow, Russian Federation, 4A.I. Yevdokimov Moscow State University of Medicine and Dentistry, Santiago de Compostela, C, Spain","locationCode":"2036","description":"\r\n\t<div>OBJECTIVES: The aim of the study was to assess the socio-economic burden of managing patients with chronic diseases accompanied by chronic respiratory failure (CRF) and requiring long-term home mechanical ventilation (non-invasive or invasive) and/or oxygen therapy in Russia. METHODS: The work consisted of two parts: an observational study of real-world clinical practice (2021-2022) and, based on the data obtained, economic model for a long-term home ventilation. The first part of the work included 65 patients (mean age 61 years, proportion of men 56.9%, n=37) with diseases accompanied by CRF (COPD, obesity hypoventilation syndrome, congenital kyphoscoliosis, consequences of spinal injury, amyotrophic lateral sclerosis, lymphangioleiomyomatosis, histiocytosis, idiopathic pulmonary fibrosis, post-COVID syndrome, etc.), receiving different types of home ventilation during 9 months. The total economic burden associated with the management of patients requiring long-term home ventilation and oxygen therapy was determined as the sum of all types of analyzed direct (medical and non-medical) and indirect costs, the data for the calculation were collected based on medical records and patients’ diaries. RESULTS: The total costs for all included patients during 9 months amounted to €250,398.95 excluding the cost of necessary medical equipment and consumables. Direct medical expenses accounted for the largest share of costs (€176,373.99), direct non-medical costs amounted to €65 845,76., and indirect - €8,179.2. The weighted average costs per 1 patient with a chronic disease requiring long-term home mechanical ventilation and oxygen therapy amounted to €8,248.47 within 1 year, excluding the necessary medical equipment and consumables. Direct medical costs amount to €6,695.73, direct non-medical costs – €1,382.69, indirect - €170.05 (rate for June, 2023). CONCLUSIONS: The analysis is the first experience in assessing the costs of long-term home mechanical ventilation and oxygen therapy in patients with various chronic diseases in real-world clinical practice, which demonstrates the high economic burden on the healthcare system, society and the patient.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23makarovaee100poster128534-pdf.pdf?sfvrsn=9c412c56_0","title":"ISPOREurope23_Makarova_EE100_POSTER128534.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128534","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Preparing Healthcare Systems for Broader Cell & Gene Therapy (CGT) Access: What Needs to Change for Pharma, Healthcare Systems, and Patients so All Benefit From CGTs?","id":"c8cac2f2-c81f-476b-becb-8ac73b560a22","sessionCode":"HPR23","topDisplay":"Green N Eradigm Consulting, London, UK","locationCode":"3076","description":"\r\n\t<div><span class=\"NormalTextRun SCXW192017173 BCX0\" data-ccp-charstyle=\"eop\" data-ccp-charstyle-defn=\"{"ObjectId":"8f707132-e184-4aeb-b0f6-a8084f16756c|79","ClassId":1073872969,"Properties":[469775450,"eop",201340122,"1",134233614,"true",469778129,"eop",335572020,"1",469778324,"Default Paragraph Font"]}\">OBJECTIVES: Cell & gene therapies (CGTs) have heralded a new era of medicine, promising potentially curative treatments for patients with acute, chronic, and often fatal diseases. However, discrepancies between the high prices set by pharma and healthcare systems’ willingness-to-pay has restricted access and somewhat dampened the broadening potential of CGTs. In the current healthcare environment, the majority of emerging CGTs will struggle to establish a foothold, as currently it is simply not designed to accommodate this step-change in innovation. Through this research, we aimed to explore concerted motions that pharma and healthcare systems can action to unify and co-create win-win situations, opening access to more patients globally. METHODS: A literature review was conducted to evaluate key drivers behind positive and negative CGT access decisions, from the perspectives of both healthcare system and pharma. RESULTS: Our research highlighted a breadth of factors driving negative access such as pharma’s withdrawal from the HTA process, insufficient evidence generation or, at times, an apparent lack of communication between parties, leading to HTA misalignment. With these insights, we developed a framework with which pharma and healthcare systems can each meet their respective objectives. The framework details three key areas which pharma and healthcare systems must find mutual ground to streamline processes and extend access to those in need: the clinical evidence required to support CGTs, the value recognition of these innovative therapies, and the pricing models that could plausibly mitigate existing healthcare expenditure challenges. CONCLUSIONS: Evolving evidence expectations, increasing collaboration between key stakeholders, and flexibility in adopting new pricing frameworks are vital steps to achieving access and unlocking the maximal value of CGTs. As CGTs broaden their applicability into more prevalent diseases, such as solid tumors and Alzheimer’s disease, the time is now to find solutions to access barriers that exist today for CGTs. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23greenhpr23poster132845-pdf.pdf?sfvrsn=fc6ea527_0","title":"ISPOREurope23_Green_HPR23_POSTER132845.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132845","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Cost-Effectiveness of Publicly Reimbursed Multi-Gene Panel Sequencing to Inform Therapeutic Decisions for Advanced Non-Small Cell Lung Cancer in British Columbia, Canada","id":"3cfac48e-3d49-4663-8e8e-8b9a299901f0","sessionCode":"EE57","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Multi-gene panel sequencing streamlines treatment selection for advanced non-small cell lung cancer (NSCLC). Implementation continues to be uneven across jurisdictions, in part due to uncertain clinical and economic impacts compared to single-gene testing. In British Columbia (BC), Canada, the public healthcare system reimbursed a multi-gene panel in September 2016. This study determined the population-level cost-effectiveness of publicly reimbursed multi-gene panel sequencing compared to single-gene testing for advanced NSCLC. METHODS: Our population-based retrospective study design used patient-level linked administrative health databases. We considered adult BC residents with a panel-eligible lung cancer diagnosis between September 2016 and December 2018. We conducted 1:1 genetic algorithm matching of recipients receiving multi-gene panel sequencing to controls receiving single-gene testing, maximizing balance on observed demographic and clinical characteristics. Following matching, we estimated mean three-year survival time and costs (public healthcare payer perspective; 2021 CAD) and calculated the incremental net monetary benefit (INMB) for life-years gained (LYG) at conventional willingness-to-pay thresholds using inverse probability of censoring weighted linear regression and nonparametric bootstrapping. RESULTS: We matched 858 panel-eligible advanced NSCLC patients to controls, achieving good balance for the 16 included covariates. Average test turnaround times were 18.6 days for multi-gene panel sequencing and 7.0 days for single-gene testing. After matching, mean incremental costs were -$1,413 (95%CI: -$31,424, $29,757) and mean incremental LYG were 0.00 (95%CI: -0.60, 0.04). Cost reductions were driven by systemic therapy cost savings (∆C: -$1,995; 95%CI: -$6,138, $2,148). The INMB at $100,000/LYG was $1,303 (95%CI: –$31,450, $32,272), with a 31% probability of being cost-effective. CONCLUSIONS: In Canada, there is uneven reimbursement of panel-based sequencing in cancer control. We used population-based real-world data to examine value for money for multi-gene versus single-gene testing for advanced NSCLC. We found no statistically significant differences in survival or expenditures associated with multi-gene panel implementation.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129835","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Catheter Ablation to Treat Cardiac Arrhythmia in Germany – Trends Based on German Hospital Data","id":"708a1d34-2729-4144-91aa-8c9c2d5f830f","sessionCode":"RWD2","topDisplay":"Theil J, Meise D Xcenda GmbH, part of Cencora, Hannover, NI, Germany","locationCode":"6056","description":"\r\n\t<div>OBJECTIVES: Catheter ablation is indicated in patients with irregular or rapid heartbeats due to e.g., ventricular tachycardia, atrial fibrillation, or atrial flutter. The study objective was to investigate the utilization of catheter ablation to treat cardiac arrhythmia in Germany. METHODS: This retrospective data analysis was based on German hospital data of the Federal Statistical Office of Germany (DESTATIS) and the Institute for the Hospital Remuneration System (InEK) of the years 2005 to 2021. Catheter ablation for the treatment of cardiac arrhythmia was identified by the OPS code 8-835. The analysis included the OPS code frequency per year and the distribution of age, sex, and methods of ablation using the fifth digit of the OPS code. RESULTS: The number of OPS codes for catheter ablations increased from 24,242 in 2005 to 230,270 in 2021. The number rose from 2005 to 2017 by 15% on average per year and markedly from 2017 to 2018 by 40%. Afterwards, the increase was 11% to 12% per year except for a 1%-increase from 2019 to 2020. Generally, more men than women received ablation treatment with a sex ratio ranging between 1.4:1 and 1.6:1. More than 75% of patients were 55 years or older. The proportion of conventional radiofrequency ablations decreased from 67% in 2005 to 10% in 2021 whereas the proportion of cooled radiofrequency ablations increased from 14% in 2005 to 31% in 2021. Ablation using three-dimensional electro anatomical mapping techniques rose from 12% in 2005 to 25% in 2021. Cryoablation was first performed in 2010 and constituted 12% of performed ablations in 2021. CONCLUSIONS: The utilization of catheter ablation to treat cardiac arrhythmia has proliferated over the past 17 years in Germany. The sex ratio remained stable. Methods of ablation have evolved and most treatments in 2021 utilized the cooled radiofrequency ablation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023-ispor-eucardiac-ablationqc-approvedgraphics-approvedfinal131691-pdf.pdf?sfvrsn=2dd84bc7_0","title":"2023 ISPOR EU_Cardiac ablation_QC Approved_Graphics Approved_FINAL131691.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131691","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Validation of Dynamic Transmission Model for Varicella Immunization Program in Germany","id":"43c2d637-03cf-44ef-b588-8d32b970ff56","sessionCode":"EPH46","topDisplay":"Żerda I1, Fundament T2, Chełmikowski F2, Stanisz T2, Kłębczyk W2, Clay E3, Aballea S4 1Assignity, Krakow, MA, Poland, 2Assignity, Krakow, Poland, 3Clever-Access, Paris, France, 4InovIntell, Marseille, 13, France","locationCode":"3049","description":"\r\n\t<div>OBJECTIVES: Dynamic transmission models (DTMs) play a crucial role in designing and evaluating the public health impact of vaccination programs. In the case of varicella vaccines, DTMs have been used to address concerns regarding a potential increase in varicella infection age and herpes zoster incidence following the implementation of universal varicella vaccination (UVV) in young children. This study aimed to validate the dynamic modelling predictions using actual data from a country with a long-term UVV program. METHODS: A newly developed compartmental, age-stratified DTM was utilized to estimate the public health impact of UVV program in Germany, considering the settings and timeframe of the existing UVV program introduced in 2004. Country-specific varicella seroprevalence during the pre-vaccination period, social contact patterns, and disease characteristics were obtained from the literature. The national statistics provided information on the year of vaccine introduction, the strategy employed, and the observed coverage rates. Vaccine characteristics were based on clinical trials. The model-predicted change in varicella incidence was compared to the observed data following vaccination introduction in the country. RESULTS: The incidence rate predicted by the DTM following UVV introduction in Germany reproduced well the trend observed in reported data, i.e. decrease by over 50% after 4 and by over 80% after 7 years post-vaccination. Validation against the data observed after 10 years from the vaccine introduction showed that in long-term the model tends to be conservative, predicting varicella incidence higher than observed. CONCLUSIONS: The validation results provide strong support for the credibility of the DTM as a method for assessing the impact of varicella vaccination programs implemented in selected countries. In contrast, a static model using the same settings significantly underestimated the impact of these vaccination programs. However, it is important to acknowledge the influence of population heterogeneity and vaccination settings on the reliability of the model.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133080","diseases":[{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A 2013-2019 Comparative Study of Patient Safety Culture at 16 Hospitals in Shimane, Japan","id":"027f085b-2b74-4077-8d79-8db018f5a2ee","sessionCode":"HSD27","topDisplay":"Endo S1, Hirose M2 1Shimane University Hospital, Izumo, 32, Japan, 2Kobe University Graduate School of Medicine, Izumo, 32, Japan","locationCode":"4032","description":"\r\n\t<div>OBJECTIVES: This study aims to examine the difference of patient safety culture between 2013 and 2019 at 16 hospitals in a rural prefecture of Japan. METHODS: Questionnaire surveys were conducted at 16 hospitals (average number of beds:238 [50 to 600]; average number of staff:377 [49 to 1,700]) in 2013 and 2019 with using a Hospital Survey on Patient Safety Culture (HSOPSC) sheet developed by the United States Agency for Healthcare Research and Quality (AHRQ). It consists of 12 factors (42 items) based on four categories (communication, organizational activity and understanding, education/learning and number of reports). RESULTS: The numbers of valid respondents were 2977 (doctors: 215, nurses: 1638, clinical staff: 493, office workers: 364) in 2013 and 3451 (doctors: 235, nurses: 1797, clinical staff: 618, office workers: 492) in 2019, respectively. The average positive response rates (PRRs) of 12 dimensions in 2019 was 53.1 and increased by 0.3%, compared with that in 2013. Out of 12 dimensions, PRRs of 8 dimensions increased and three of them including D 1: Communication openness, D 8: comprehensive understanding of safety, and D 10: Supervisor/manager expectations and actions promoting safety extremely changed. On the contrary, D 3: Frequency of events reported, D 4: Hands-off and transitions, and D 12: Teamwork within units declined. The 12-dimension average PRR by occupation increased by 4.3% and PRRs for physicians and clinical staff increased by 2.4%, respectively. The PRRs for Nurses and clerical staff were unchanged. CONCLUSIONS: Compared PRRs between 2013 and 2019, the average PRRs of dimensions related to 'organization' increased, meanwhile the PRRs of dimensions related to 'communication' decreased. Improving the working environment is therefore likely to create a patient safety culture.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-europe-2023-psc-in-japan-endo-s-2023-11-13-rev133180-pdf.pdf?sfvrsn=eab2dce8_0","title":"ISPOR Europe 2023. PSC in Japan (Endo S) 2023.11.13. rev133180.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133180","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Burden of Disease and Cost of Illness of Age Related Macular Degeneration and Diabetic Macular Edema in Portugal","id":"c5233f1e-d665-4912-b937-8e1744fac0b7","sessionCode":"EPH37","topDisplay":"Gouveia M1, Cabral D2, Silva R3, Carneiro Â4, Alarcão J5, Cafe A5, Canhão H6, Rodrigues A6, Sousa RD6 1Católica Lisbon School of Business and Economics, Lisboa, 11, Portugal, 2Nova Medical School, Universidade Nova de Lisboa, Lisboa, Portugal, 3Faculdade de Medicina, Universidade de Coimbra, Coimbra, Portugal, 4Faculdade Medicina, Universidade do Porto, Porto, Portugal, 5Roche Farmacêutica Química, Lda, Amadora, Portugal, 6Nova Medical School, Lisbon, Portugal","locationCode":"3038","description":"\r\n\t<div>OBJECTIVES: This study aims to estimate the burden and costs associated with Wet Age-Related Macular Degeneration (wAMD) and Diabetic Macular Edema (DME) in Portugal in 2018. The objective is to provide a clear assessment of the impact of these ophthalmological diseases on population health and the financial implications for the healthcare system and society as a whole. METHODS: The burden of disease was quantified using disability-adjusted life years (DALY). Mortality was not attributed to wAMD and DME, and the burden was solely based on the resulting disabilities. Costs of AMD and DME were estimated using multiple data sources, including Hospital Morbidity Database (DRG), official Portuguese Statistics on population and wages, epidemiological information from the literature, and expert opinions. RESULTS: The estimated prevalence of wAMD and DME was 18,068 and 44,109 cases, respectively. The burden of disease attributed to wAMD was 954 DALYs and 1,906 DALYs for DME. DALYs due to wAMD are projected to increase 36.6% over the next 20 years. During the same period, DME DALYs are expected to rise 10.1%. The direct annual costs associated with wAMD and DME in 2018 were estimated at €30.79million and €28.76million, respectively. While productivity costs related to wAMD were assumed to be negligible, the estimated productivity costs for DME amounted to €39million, surpassing the direct costs. Combining productivity losses and direct costs for DME yields a social cost of €67.8million. CONCLUSIONS: Considering the substantial number of affected individuals, AMD and DME in Portugal pose a considerable disease burden on the population and generate significant direct costs to the healthcare system. The burden and costs are anticipated to rise in the future due to demographic changes associated with an aging population. This study provides valuable insight into the consequences of AMD and DME, emphasizing the need to prioritize and define healthcare policies based on these findings.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23cafeeph37poster131661-pdf.pdf?sfvrsn=ad5ab15e_0","title":"ISPOREurope23_Cafe_EPH37_POSTER131661.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131661","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"England's Population Health Agreement for Inclisiran – Evidence of Very Limited Prescribing Uptake","id":"c44d3b9f-d9ec-43e7-b9a7-8ed377f4e5d2","sessionCode":"HSD1","topDisplay":"Wang G1, Macaulay R2 1Precision Advisors, Esher, UK, 2PRECISIONadvisors, Edinburgh, UK","locationCode":"4011","description":"\r\n\t<div>OBJECTIVES: In September 2021, following NICE’s restricted recommendation for inclisiran (TA733), NHS England announced its first ‘population health agreement’. This enabled the treatment of up to 300,000 patients with primary hypercholesterolaemia or mixed dyslipidaemia, potentially avoiding 55,000 myocardial infarctions and saving 30,000 lives. This unprecedented deal enabled twice-yearly subcutaneous injections to be provided by GPs in primary care at a reimbursed-price of £55 per injection vs. list-price £1,987.36, with a large-scale primary prevention trial to collect CV outcomes in 40,000 UK patients (ORION-17). This research evaluated the success of this agreement in terms of inclisiran uptake/prescribing. METHODS: Inclisiran uptake data from primary care prescribing (<a href=\"https://openprescribing.net/\">https://openprescribing.net/</a>) was identified between December 2021 and March 2023 by the seven NHS England commissioning regions (26-JUNE-2023). RESULTS: Only 5,931 injections have been prescribed in total. In 2021, only 50 injections were prescribed. In 2022, 3,156 injections were prescribed, with the South-West leading with 1,245 injections, compared to the lowest, 141 injections in London. Although annual uptake is increasing, this trend continues in the first three months of 2023, with South-West having 831 injections and again the lowest in London of 126 injections. Assuming two injections per patient per annum and without accounting for the initiation phase of two injections in the first three months, less than 3,000 patients have been treated in total. This represents ~1% of the eligible population quoted. CONCLUSIONS: Despite the company committing to provide inclisiran at a substantially discounted price and initiating a large-scale UK CV outcomes trial, only ~1% of the eligible patient population appear to have been treated 1.5 years since this ‘population health agreement’ was set up. Further, in March 2023, the company announced they would not be going ahead with the ORION-17 trial. This highlights the multiple, substantial barriers facing companies in UK market access despite competitive and innovative approaches.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23macaulayhsd1poster133818-pdf.pdf?sfvrsn=481361ea_0","title":"ISPOREurope23_Macaulay_HSD1_POSTER133818.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133818","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Efficacy of the Dupilumab 200 Mg Dose Every 2 Weeks in Children with Moderate-to-Severe Asthma during Voyage and Excursion","id":"a288cbc8-b849-4518-b723-8ef2feb74d7b","sessionCode":"CO26","topDisplay":"Phipatanakul W1, Vogelberg C2, Bacharier LB3, Dell SD4, Altincatal A5, Gall R6, Ledanois O7, Sacks H6, Jacob-Nara JA8, Deniz Y6, Rowe PJ8 1Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, 2University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany, 3Monroe Carell Jr Children’s Hospital at Vanderbilt University Medical Center, Nashville, TN, USA, 4BC Children’s Hospital, Vancouver, BC, Canada, 5Sanofi, Cambridge, MA, USA, 6Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA, 7Sanofi, Paris, France, 8Sanofi, Bridgewater, NJ, USA","locationCode":"1025","description":"\r\n\t<div>OBJECTIVES: In VOYAGE (NCT02948959), 6–11-year-old children with uncontrolled, moderate-to-severe asthma received dupilumab 100mg q2w (body weight ≤30kg at baseline) or 200mg q2w (>30kg at baseline) for 52 weeks. In the 52-week open-label extension EXCURSION study (NCT03560466), children (>30kg) received dupilumab 200mg q2w. Dupilumab reduced exacerbation rates (AER), improved percent-predicted FEV1 and was generally well tolerated. Pharmacokinetic/pharmacodynamic VOYAGE data showed comparable dose exposure between both regimens. This analysis assessed whether dupilumab 200mg q2w efficacy is comparable with the overall analysis. METHODS: This post hoc analysis of VOYAGE and EXCURSION includes children who received dupilumab 200mg or placebo q2w for 52 weeks in VOYAGE and dupilumab 200mg q2w for 52 weeks in EXCURSION, although the study was not powered to assess efficacy with this dose separately. Endpoints: unadjusted AER; change from VOYAGE baseline in ppFEV1 at Weeks 12 and 52. RESULTS: Of 408 children, 158 received dupilumab 200mg q2w in both VOYAGE and EXCURSION (dupilumab/dupilumab), 105 children received placebo in VOYAGE and dupilumab 200mg q2w in EXCURSION (placebo/dupilumab). In VOYAGE, unadjusted AER was lower in dupilumab vs placebo groups (0.392 vs 0.608). In EXCURSION, further reductions were observed (0.129 dupilumab/dupilumab, 0.136 placebo/dupilumab). In VOYAGE, dupilumab improved ppFEV1 at Week 12 by mean (SD) of 11.3 (16.4) percentage points, and 13.2 (18.4) points by Week 52; in the placebo arm, marginal improvements were observed at Week 12 (3.5 [11.4] percentage points) and Week 52 (2.6 [13.6] points). In EXCURSION, dupilumab sustained improvements in ppFEV1 in the dupilumab/dupilumab arm (change from VOYAGE baseline at Week 12: 10.9 [17.7]; Week 52: 12.2 [17.8] percentage points) and improved ppFEV1 in the placebo/dupilumab arm (Week 12: 7.2 [13.6] percentage points), sustained to Week 52 (7.6 [16.1] points). CONCLUSIONS: In patients >30 kg, the dupilumab 200mg dose achieved effective asthma control, in children with uncontrolled, moderate-to-severe asthma.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23phipatanakulco26poster131888-pdf.pdf?sfvrsn=b1cd1500_0","title":"ISPOREurope23_Phipatanakul_CO26_POSTER131888.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131888","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Insights into Stroke Patients: Assessing Knowledge, Awareness, and Perception of Stroke Symptoms and Cardiovascular Risk Factors for Enhanced Care","id":"112a3c86-4eb8-463c-8d2a-8f0e75436ce3","sessionCode":"CO2","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1003","description":"\r\n\t<div>OBJECTIVES: This study aims to quantify stroke knowledge and health-related behaviors in people with recent stroke, and their relation with cardiovascular disease risk. METHODS: The patients enrolled in the study were subjected to a comprehensive clinical history and physical examination. An analytical methodology was employed to evaluate the risk factors, clinico-radiological profile, & outcome in patients with acute cardiovascular/cerebrovascular disease. The data collected from 600 selected subjects underwent internal comparison & statistical analysis using descriptive and inferential statistics, in accordance with the study's formulated objectives. Stroke survivors received a detailed and validated questionnaire. RESULTS: The findings of the study demonstrate that a considerable percentage (62%) of Stroke patients with limited educational attainment and lower income levels displayed lack of knowledge and awareness regarding stroke, encompassing its correlated risk factors and warning signs. Older patients between the ages of 45 and 65, characterized by elevated socioeconomic status, including higher levels of education and income, along with those possessing a familial history of stroke, exhibited significantly higher levels of awareness. Furthermore, hypertension, diabetes mellitus, and smoking emerged as the most frequently reported recognized risk factors for stroke. CONCLUSIONS: Among the identified risk factors for stroke, hypertension, diabetes mellitus, and smoking emerged as the most commonly reported known contributors. However, a noteworthy aspect revealed in this study was the limited awareness among the subjects regarding their elevated susceptibility to stroke. Particularly, the individuals included in the study demonstrated a significant lack of knowledge regarding their increased risk for stroke. Therefore, there exists a pressing need for comprehensive and intensive health education interventions to enhance stroke awareness, particularly among the most vulnerable population segments. Remarkably, the findings also highlighted the alarmingly low level of awareness among the participants concerning diabetes mellitus as a risk factor for cardiovascular stroke.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131448","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessment of Target Muscle Reinnervation/Regenerative Peripheral Nerve Interface: Systematic Review","id":"4d398ef6-517a-4c2b-a6f5-8f1051b6bcf5","sessionCode":"HTA41","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"4077","description":"\r\n\t<div>OBJECTIVES: The objective of this assessment was to determine the safety and effectiveness of the technique for treating and preventing neuromas, residual limb pain, and phantom pain by connecting severed nerves to the nerves of the surrounding muscles or wrapping them with adjacent muscle sections for: i) patients undergoing lower and upper extremity amputation, ii) patients who have undergone lower and upper extremity amputation, and iii) patients with sensory nerve injury caused by surgical incisions and scars. METHODS: The safety and effectiveness of the technique was assessed via a systematic literature review. A literature search was conducted using five domestic databases, including KoreaMed, and Ovid-MEDLINE, Ovid-EMBASE, and the Cochrane Library. The search strategy and manual search generated 570 results. RESULTS: The safety and effectiveness of the technique was assessed using 11 studies selected. It cannot be concluded that the technique is comparable to the conventional procedure based on the currently available evidence. The effectiveness was assessed for the following: 1) For the prevention of neuromas, one study reported no cases of symptomatic neuroma in the intervention group. 2) For pain, phantom pain was less prevalent in the intervention group. Residual limb pain was also lower in the intervention group than in the comparison group in patients who underwent lower limb amputation or transtibial amputatio. Only one study was found regarding the prevention of neuromas, and although patient reports showed equal or greater effectiveness compared to existing techniques for phantom pain and stump pain, there is inadequate literature evidence for objective indices. CONCLUSIONS: Further research is needed to determine the safety and effectiveness for treating and preventing neuromas, residual limb pain, and phantom pain in patients undergoing lower and upper extremity amputation, patients who have undergone lower and upper extremity amputation, and patients with sensory nerve injury caused by surgical incisions and scars.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128303","diseases":[{"id":"3f994852-a0d4-4706-9665-e4d867006d9a","parentId":"00000000-0000-0000-0000-000000000000","title":"Injury & Trauma","urlName":"injury-trauma"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Amyotrophic Lateral Sclerosis on Work and Daily Living: Results from a Real-World Survey","id":"45e66aab-5b8e-4598-a18b-8f5971947633","sessionCode":"PCR15","topDisplay":"Stenson K1, Mellor J2, Wright J2, Earl L2, Ball N2, Iqbal H2, Thomas O2, Sethi N3 1Biogen, Weymouth, MA, USA, 2Adelphi Real World, Bollington, UK, 3ALS Therapy Development Institute, Watertown, MA, USA","locationCode":"6019","description":"\r\n\t<div>OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a rare, degenerative neuromuscular disease, leading to progressive loss of muscle function, and ultimately death. The debilitating nature of the condition negatively impacts the working and living arrangements of people with ALS (pALS) and their caregivers (cALS). METHODS: The Adelphi ALS Disease Specific Programme (DSP™) is a cross-sectional survey of neurologists, pALS, and cALS in France, Germany, Italy, Spain, the UK, and the USA (Jul 2020–Mar 2021). Neurologists completed questionnaires on consulting pALS demographics, including employment status and living arrangements. These same pALS and their cALS, if applicable, were invited to complete questionnaires from their perspectives. RESULTS: 142 neurologists reported data on 880 pALS; 135 pALS and 82 cALS provided self-reported data. 46% of pALS reported they had changed their working arrangements due to ALS, with 91% of these pALS having either reduced hours or stopping work entirely. Neurologists reported that changes in employment status occurred on average 16.1 months after symptom onset, and the most frequently cited reasons were impaired mobility (74%), increased muscle weakness (35%) and impaired communication (23%). 37% of cALS reported a change to their working arrangements to care for the pALS, with 50% of these cALS having either reduced hours or stopping work entirely. The support activities cALS most reported were “preparing meals/cooking” (72%), “shopping” (68%), and “cleaning/housework” (68%). A high frequency of home modifications was reported, and in 14% of cases, moving to a more ALS friendly house. ~50% of pALS and cALS agreed that ALS was a significant financial burden. CONCLUSIONS: ALS-related impacts to working arrangements may lead to loss of income for ALS families even as changes to living arrangements become necessary. Substantial caregiver burden is common as responsibilities shift with disease progression. These impacts to work and daily living highlight the high level of unmet need in ALS.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-euadelphi-als-work-and-living-changes-poster-v6-0-03-10-23final130502-pdf.pdf?sfvrsn=26f16389_0","title":"ISPOR EU_Adelphi ALS Work and Living changes Poster v6.0 03.10.23_final130502.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130502","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Effectiveness Analysis of Pembrolizumab as First-Line Treatment for Persistent, Recurrent, or Metastatic Cervical Cancer in Greece","id":"dd0c7230-1588-4836-a9b3-8fbefefb0460","sessionCode":"EE66","topDisplay":"Yfantopoulos N1, Gountas I2, Swami S3, Skroumpelos A4, Karokis A4 1MSD Greece, Alimos, A1, Greece, 2MSD Greece, Athens, Greece, 3MSD (UK) Limited, London, UK, 4MSD Greece, Alimos, Attica, Greece","locationCode":"2022","description":"\r\n\t<div>OBJECTIVES: In 2020, the World Health Assembly announced a global comprehensive plan to eliminate cervical cancer, involving vaccination, screening, and treatment-rate targets. Pembrolizumab in combination with - paclitaxel, cisplatin, carboplatin, and bevacizumab - is reimbursed for first-line treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS>=1) based on the results of KEYNOTE-826 trial. The present study aims to assess cost-effectiveness of Pembrolizumab plus SoC versus SoC alone in Greece in this setting. METHODS: A semi-Markov State Transition model with three health states (progression-free, progressed disease and death) was adapted to Greece, adopting payer’s perspective with a 40 years’ time-horizon. Clinical, safety and quality of life data were drawn from KEYNOTE-826 trial (22 months follow-up data). Primary outcomes were patients’ life years gained (LYg), quality-adjusted life years (QALYs), total costs and incremental cost-effectiveness ratios (ICER) per QALY. Both costs and QALYs were discounted at 3.0% per annum. Α one-way sensitivity analysis (OWSA) was undertaken to examine the most influential parameters on the results and a probabilistic sensitivity analysis (PSA) was conducted to account for uncertainty in the model. RESULTS: The model showed that, over a lifetime horizon, total average cost per patient with pembrolizumab plus SoC was estimated at €157,368 whereas the cost of SoC alone was €73,360. Pembrolizumab with SoC was more effective than SoC providing 6.104 LYs versus 2.991 LYg which translated into 3.412 and 1.799 QALYs respectively; overall incremental LYG were 3.113 and the incremental QALYs were 1.613. The ICER was estimated at €52,081/QALY which is below the national cost-effectiveness threshold for Greece(3x GDP capita, €52,770/QALY). The OWSA and PSA results confirmed the robustness of the model. CONCLUSIONS: The present modeling study suggests that pembrolizumab in combination with SoC is a cost-effective intervention compared to SoC that improves health outcomes and the efficient allocation of resources in Greece.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023-1l-cervical-cancer-132988-pdf.pdf?sfvrsn=9a1d2da5_0","title":"2023 1L Cervical Cancer 132988.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132988","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Formulation of PCR-Test Screening for COVID-19 Regarding Multiple Factors in Epidemic","id":"c7c14a80-8881-4fde-bccb-8fdb804a6530","sessionCode":"EE28","topDisplay":"Kamae I1, Kobayashi M2, Watanabe R3 1The University of Tokyo, Tokyo, 13, Japan, 2CRECON Medical Assessment Inc., Tokyo, Japan, 3Kanagawa University of Human Services, Yokosuka, Kanagawa, Japan","locationCode":"1077","description":"\r\n\t<div>OBJECTIVES: Multiple factors such as virulence of a virus, diagnostic accuracy, vaccinations, and immune evasion would affect the wave formation of COVID-19 pandemic. Regarding those factors, this study formulates cost-effectiveness of PCR-test screening in community. METHODS: The incremental cost-effectiveness ratio (ICER) of PCR-test vs. no test was developed in theory from the public payer’s perspective. It defined a numerator with the incremental costs of PCR-test screening, while a denominator with the Quality-adjusted Life Years (QALYs) saved by prevention of viral transmission. The factors included: 1)k: level of transmission, 2)Rt: effective reproduction number, 3)θ: immunity protection rate in individuals, 4)ε: evasion rate from vaccines, and 5)Sn: test sensitivity, 6)M: the number of cases tested, and 7)D+: the number of infected cases in M. Example computations were also conducted. RESULTS: Let Ct, Cd, Cid be a test-kit cost, direct costs, and indirect costs, respectively, for PCR-test screening. Then a numerator of ICER was given with M (Ct+Cd+Cid), and a denominator with the expression of (Q)D＋Sn{1－Rtk (1–θ(1-ε))k} / {1－Rt (1–θ(1-ε))}, where Q means the QALY lost in assumed ten days of illness with expected utility U, i.e., Q = 10(1-U)/365. As an example, the formula was numerically computed with PCR-test sensitivity of 85% in a practical setting in Japan with Ct+Cd+Cid =$214(￥30,000), M=5000, D+=1000, U=0.667 (using 0.2 assigned to severely ill and 0 to death), Rt=3, k=2, θ=70%, ε=60%. Then the ICER was $43,782/QALY (￥6,129,476). Sensitivity analyses revealed k, Rt, and the proportion of D+ in M is quite sensitive to worsening the cost-effectiveness. CONCLUSIONS: Our formula can estimate the cost-effectiveness of COVID screening in various phases of pandemic, and contribute to assess the value of screening tests to prepare for the next pandemic X.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133394","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Onasemnogene Abeparvovec-Xioi (Zolgensma®) and Best Supportive Care Treatment for Spinal Muscular Atrophy I in the Netherlands With Early-Treatment Scenario","id":"94bd2470-9adb-4bb8-b32e-90936574c67c","sessionCode":"EE89","topDisplay":"Meijer A1, Omar Alsaleh AJ2 1Bologna University, Amsterdam, Netherlands, 2Bologna University, Bologna, Italy","locationCode":"2028","description":"\r\n\t<div>OBJECTIVES: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder and is considered one of the most common genetic causes of infant mortality. Onasemnogene Abeparvovec-xioi (OA) is a gene therapy showing promising health outcomes for SMA treatment, with a list price of approximately €2,000,000. This study aims to provide a cost-effectiveness analysis in the Netherlands for the treatment of SMA type I with OA with newly published data. METHODS: A Markov model with five health states was replicated to analyze the costs and outcomes for patients diagnosed with SMA Type I. The analysis was conducted from a societal perspective in the Netherlands over a time period of 99 years. Both one-way and probabilistic sensitivity analyses were conducted to assess the uncertainties associated with the model's parameters. A scenario analysis was performed to evaluate the potential benefits of early treatment. RESULTS: Base-case incremental cost-effectiveness ratio (ICER) of OA was €257,717 per quality-adjusted life year (QALY) compared to best supportive care (BSC). The ICER of OA from the early treatment scenario was €127,107 compared to BSC. Both are above the Dutch willingness-to-pay (WTP) reference value of €80,000. Key drivers influencing the ICERs were the costs of OA treatment and utility and cost values of ‘sitting independently’ health state. CONCLUSIONS: Based on this model, treatment with OA supports the notion that it offers significant improvements in disease progression, motor skills, and quality of life compared to BSC. A reduced ICER of almost 27% is observed compared to the HTA conducted by the Dutch Health Care Institute. However, it is not cost-effective under the Dutch WTP threshold. Limited availability of clinical trial data, characterized by small sample sizes and short follow-up periods, causes great uncertainty. Decision-makers should find a suitable balance between these uncertainties and the cost they are willing to pay for the treatment of rare diseases.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023meijeree89posterv2132069-pdf.pdf?sfvrsn=780fe133_0","title":"ISPOREurope2023_Meijer_EE89_POSTERV2132069.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023meijeree89handout132069-pdf.pdf?sfvrsn=8febcd8a_0","title":"ISPOREurope2023_Meijer_EE89_HANDOUT132069.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132069","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Burden of Disease and Cost of Permanent Productivity Loss of Cardiovascular Disease (CVD) in South America (SA)","id":"7238db38-8d1b-4323-8341-918311b70336","sessionCode":"EPH4","topDisplay":"Bandeira TFGDS1, Mosegui G2, Vianna CMM3, Antoñanzas F4, Gil AJ4 1Universidade Federal Fluminense, Niterói, Rio de Janeiro, Brazil, 2Universidade Federal Fluminense, Rio de Janeiro, Brazil, 3Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brazil, 4University of La Rioja, Logrono, Spain","locationCode":"3007","description":"\r\n\t<div>OBJECTIVES: Describe the burden of disease and estimate the burden of permanent productivity losses caused by CVD in South American countries. METHODS: This is an exploratory, population-based study. A proxy of the human capital approach (HCA) was used to estimate the cost of permanent productivity losses associated with CVD. To calculate this cost, the sum of the years of productive life lost for each death was multiplied by the proportion in the workforce and the employment rate, and then by the annual minimum wage or purchasing power parity (PPP) in United States dollars (US$) for each country in the economically active age groups. Separate calculations were done for men and women. Epidemiologic measurements by standardized age group among the 12 countries were captured through the Institute for Health Metrics and Evaluation (IHME) website that uses the Burden Study Global Disease, Injury and Risk Factors (GBD) 2019 assessment. Correlation analyses were performed. RESULTS: The total number of deaths from CVD in 2019 was 171.757 and the years of productive life lost were 2.040.974,21 years. The total cost of permanent productivity loss was about US$ 3,7 billion based on annual minimum wage and US$ 8 billion in PPP, representing 0.11% of the region’s gross domestic product. The cost per death was US$ 22.904. The cost of productivity losses differed substantially between countries and by sex. There is a correlation between sociodemographic and economic health indicators and the standardized rates of incidence and DALY in the CVD. CONCLUSIONS: CVD impose a significant economic burden on SA in terms of health and productivity. Characterization of the economic costs of these diseases can support governments in the allocation of resources to develop policies and interventions to reduce their burden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23moseguieph4poster128548-pdf.pdf?sfvrsn=ec36ff70_0","title":"ISPOREurope23_Mosegui_EPH4_POSTER128548.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128548","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Daratumumab in Combination with Lenalidomide and Dexamethasone Improves Survival in Newly Diagnosed Transplant-Ineligible Multiple Myeloma: A Parametric Network Meta-Analysis in a United Kingdom Setting","id":"f71efe3b-932e-4b19-9f65-92959deb2154","sessionCode":"CO24","topDisplay":"van Beekhuizen S1, Bird A2, Freitag A3, Yuan Z4, Ming T5 1Cytel Inc., Rotterdam, ZH, Netherlands, 2Janssen, High Wycombe, Buckinghamshire, UK, 3Cytel, London, LON, UK, 4Cytel, Rotterdam, ZH, Netherlands, 5Janssen-Cilag Limited, High Wycombe, Buckinghamshire, UK","locationCode":"1023","description":"\r\n\t<div>OBJECTIVES: Daratumumab in combination with lenalidomide and dexamethasone (DRd) is indicated for patients with newly diagnosed transplant-ineligible multiple myeloma. Previous hazard ratio network meta-analyses (NMA) have shown DRd to be clinically superior to other treatments. Proportional hazard assumption (PHA) violations in the network, however, required the exploration of advanced NMA methods. This research aimed to conduct a parametric NMA (PNMA) to evaluate overall survival (OS) and progression-free survival (PFS) for DRd compared with relevant treatments in the United Kingdom (UK) setting. METHODS: Data on DRd and relevant UK comparators (lenalidomide[continuous]-dexamethasone [Rdc], melphalan-prednisone-thalidomide [MPT], bortezomib-melphalan-prednisone [VMP]) were identified through a systematic literature review. Pseudo-individual patient-level data were obtained by using the Guyot algorithm to reconstruct data from digitized published Kaplan-Meier curves. The PNMA distributions included Weibull, exponential, log-normal, Gompertz, log-logistic, gamma, and generalized gamma. The models were compared based on leave-one-out information criteria and mean survival (95% confidence interval [CI]). MAIA (DRd) was used as the reference trial. RESULTS: The evidence network consisted of six and seven trials for OS and PFS, respectively (MAIA, FIRST, VISTA, IFM99-06, IFM01/01, Sacchi 2011, TMSG [PFS only]). The PHA was violated in one (PFS) and two (OS) trials. The gamma (PFS) and Gompertz (OS) distributions showed the best statistical fit. Treatment with DRd resulted in the longest OS at 11.6 years (95% CI: 7.2, 36.1), followed by Rdc (5.7 [4.9, 7.0]), MPT (4.9 [4.0, 6.3]), and VMP (4.4 [3.3, 6.3]). DRd also showed the longest PFS (8.0 years [6.7, 9.8]), followed by Rdc (4.0 [3.5, 4.7]), VMP (2.7 [1.8, 4.0]), and MPT (2.6 [2.1, 3.2]). These results aligned with previously published indirect treatment comparisons. CONCLUSIONS: PNMA was considered an appropriate method to address PHA violations in the network. The method confirmed the hazard ratio NMA results that demonstrated DRd improves survival compared with other treatments.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23beekhuizenco24poster129468-pdf.pdf?sfvrsn=72bfbf89_0","title":"ISPOREurope23_Beekhuizen_CO24_POSTER129468.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129468","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Are There Any Common Denominators for Early Access Programs in Europe?","id":"3a3aeabc-c348-42d5-bb95-92d8dd619e52","sessionCode":"HPR43","topDisplay":"Ecker T1, Le Mao J2, Prada M3, Aceituno Mata S4, Moeller B5, Huraskin D1 1Ecker + Ecker GmbH, Hamburg, Germany, 2CEMKA, Bourg La Reine, France, 3Intexo SB, Rome, RM, Italy, 4Outcomes'10, Castellón de la Plana, CS, Spain, 5Krammer, Wrbka & Partner GmbH, Vienna, Austria","locationCode":"4003","description":"\r\n\t<div>OBJECTIVES: Early access programs (EAP) are needed, as long as pharmaceuticals are not approved or commercially available in a certain jurisdiction. They address a patient need and can be a relevant strategy for market access. Legal background is Article 83 of EU Regulation 726/2004. This analysis sets out to explore if and which common denominators exist for EAP within the EU and what are the potential implications for market access. METHODS: For this analysis current state of EAP regulation and products under EAP for Spain, Italy, France, Germany, and Austria have been reported by respective country experts, as of June 2023. Focus is on program on cohorts. RESULTS: There are two main EAP for patient cohorts without therapeutic alternatives in Italy. In compassionate use programs (CUP), the product is provided for free by the pharmaceutical company (currently 81 products). Law 648/1996 reimburses the use of certain drugs based on a price negotiated with the NHS (currently 150). All programs require approval from AIFA and require a therapeutic protocol. In Spain early access is limited to case-by-case decisions; programs for cohorts do not exist. Like Italy, France has an extensive EAP. Decision is based on a submission by the pharmaceutical company showing medical need, efficacy and safety and presumption of innovation. Product use is reimbursed and price during EAP is determined by the pharmaceutical company; therapeutic protocol and data collection are mandatory with 74 products currently under EAP. Contrary to France, in Austria and Germany CUPs are not reimbursed. They are also in time, but therapeutic protocol and data collection again are mandatory. 28 products are currently under CUP in Germany and 3 in Austria. CONCLUSIONS: Even though EAPs are regulated by European law, each country follows different approaches towards EAP. There is no such concept as a common EU EAP strategy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23huraskinhpr43poster131333-pdf.pdf?sfvrsn=7bd018c1_0","title":"ISPOREurope23_Huraskin_HPR43_POSTER131333.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131333","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Characterization of Demography and Comorbidity in Newly Diagnosed Multiple Myeloma Patients in the UK","id":"36a3e22d-9733-4fa4-8d53-93e69726addf","sessionCode":"SA4","topDisplay":"Tunaru F1, O'Reilly J2, Perkins A3, Wallis J1, Carpenter L1 1Arcturis Data, Oxford, UK, 2Arcturis Data, Oxford, OXF, UK, 3Arcturis Data, Kidlington, UK","locationCode":"7016","description":"\r\n\t<div>OBJECTIVES: Patient comorbidity remains a challenge for determining optimal therapy for Multiple Myeloma (MM). We look towards a real-world data cohort of adult newly diagnosed MM (NDMM) patients to characterize demography and comorbidity in this group and assess the effects of comorbidity on overall survival. METHODS: This retrospective study used de-identified electronic health records for Multiple Myeloma patients diagnosed between 2013 and 2023 from UK NHS partners collated as part of the Arcturis Data Platform. NDMM patients were identified as having those with a first primary diagnosis code for MM (ICD-10: C90.0). Patients with any malignancy occurring up to 6 months prior to MM diagnosis were excluded. Charlson Comorbidity Index (CCI) at time of MM diagnosis was based on all comorbid diagnoses in the 3 years prior to MM diagnosis. A Cox regression model was used to assess the impact of CCI at diagnosis on 8-year overall survival when adjusting for age and sex. RESULTS: A total of 1294 NDMM were identified, with mean age of 69 years (SD 12); with race including 67.9% White, 3.6% Black, 3.2% Mixed and 2.4% Asian. 783 (60.5%) patients were male, and the mean deprivation index was 7.57 (standard deviation 2.31). The most common comorbidity was hypertension (36.2%), followed by acute renal failure (20.4%). Most patients had a CCI of 0 (64.7%), 26.0% were mildly comorbid (CCI 1-2), 9% were moderately/severely comorbid (CC1 3+). After adjusting for age and sex, relative to patients with no comorbidities, those with mild CCI indicated a non-significant increase in risk of mortality (HR 1.25; 95% CI 0.99-1.57), whilst those with severe CCI indicated a significant increase in risk of mortality (HR 1.57; 95% CI 1.15-2.14). CONCLUSIONS: Multi-morbid MM patients continue to be at increased risk of poorer outcomes and further research to understand optimal management remains an area of unmet clinical need. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/filipaisporcomorbiditymmposterfinal133124-pdf.pdf?sfvrsn=fece3336_0","title":"Filipa_ISPOR_Comorbidity_MM_Poster_final133124.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133124","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Incidence of Postpartum Mental and Behavioral Disorders in Japan","id":"dde1574e-145a-4cc3-96bd-9460e85be2d7","sessionCode":"EPH18","topDisplay":"Iwasaki K1, Ha C1, Takeshima T2, Sato Y3, Hiroi S3, Hatakama A4, Igarashi A5 1Milliman, Inc., Chiyoda-ku, Tokyo, Japan, 2Milliman, Inc., Tokyo, Japan, 3Shionogi & Co., Ltd., Osaka, Japan, 4DeSC Healthcare, Tokyo, Japan, 5Yokohama City University School of Medicine, Yokohama, Kanagagawa, Japan","locationCode":"3019","description":"\r\n\t<div>OBJECTIVES: Postpartum depression (PPD) develops in a critical stage of a woman’s life and the likelihood of depressive symptoms could be twice as high as other periods of life. PPD is often left untreated and can continue for a long time. Negative impacts are expected on the subsequent family planning and the parenting behavior of the affected family. Thus, we assessed the incidence of postpartum mental and behavioral disorders, which can accelerate the already-falling birthrate in Japan. METHODS: DeSC database including data of employee-based health insurance (EHI) for employees and their families and the citizens’ health insurance (NHI) for non-/self-employed workers was used. Since eutocia is not covered by the compulsory insurance and cannot be directly identified with claims, a childbirth was identified if a woman aged 15–49 years at childbirth had a matched ID with a newborn from April 2014 through May 2022. The cumulative incidence rate (CIR) of mental and behavioral disorders (F00–F99) were compared between the birth group and the non-birth control matched for sex, birthday month, insurance type, and the first and last month of the observable period using Logrank test. The monthly incidence from childbirth was also examined. RESULTS: The birth group included 23,236 women (29% NHI, 3% EHI, 69% EHI as family members). The CIR was significantly higher in the birth group (p<0.0001). A total of 4,917 women developed mental and behavioral disorders in the birth group. The incidence was smaller during six months antepartum and larger during three months postpartum. CONCLUSIONS: The incidence of mental and behavioral disorders was higher after the childbirth than before, suggesting the importance to pay attention to maternal mental health especially during three months after the childbirth even if no signs of PPD were observed before the childbirth.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeu2023eph18poster132624-pdf.pdf?sfvrsn=d9992261_0","title":"ISPOREU2023_EPH18_poster132624.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132624","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of the NEON Intervention for People With Psychosis and Non-Psychosis Mental Health Problems","id":"0a9744ac-f3b5-4c62-8829-94830d99140a","sessionCode":"EE38","topDisplay":"Gavan S1, Paterson L2, Rennick-Egglestone S3, Slade M3, Newby C4, Elliott R2 1Manchester Centre for Health Economics, Division of Population Health, Health Services Research and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK, UK, 2Manchester Centre for Health Economics, Division of Population Health, Health Services Research and Primary Care, School of Health Sciences, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK, 3School of Health Sciences, Institute of Mental Health, University of Nottingham, Nottingham, UK, 4School of Medicine, Institute of Mental Health, University of Nottingham, Nottingham, UK","locationCode":"1083","description":"\r\n\t<div>OBJECTIVES: The Narrative Experiences Online (NEON) Intervention is a digital health intervention to improve quality of life for people with mental health problems by providing online access to mental health recovery narratives. The NEON Intervention incorporates an automated algorithmic system to generate personalised recommendations of narratives, or users can browse the collection of narratives themselves. The aim of this study was to determine the cost-effectiveness of the NEON Intervention for people with psychosis and non-psychosis mental health problems in England. METHODS: A within-trial cost-effectiveness analysis of the NEON Intervention compared with current practice was performed alongside two definitive randomised controlled trials for people with psychosis (NEON Trial: ISRCTN11152837) and non-psychosis (NEON-O Trial: ISRCTN63197153) mental health problems. The time horizon was 12 months. The perspective was the National Health Service (NHS) England. Participants included those who had, and had not, used primary and/or secondary mental health services. Health outcomes were expressed as quality-adjusted life years (QALYs). Delivery costs of the digital health intervention were included. Participant-reported downstream health and social care costs were collected. Total QALYs and total costs for both arms were estimated by generalised linear models with multiple imputation adjusting for baseline characteristics. Sampling uncertainty was handled by bootstrapping. The primary outcome was the incremental cost-effectiveness ratio (ICER). A threshold of £20,000-£30,000 per QALY gained was used to determine cost-effectiveness. RESULTS: The NEON Trial comprised 739 participants. The NEON-O Trial comprised 1,023 participants. For the NEON Trial, the ICER was £110,501 (incremental cost: £1,177; incremental QALY: 0.0107; probability of cost-effectiveness at £30,000 per QALY gained: 3.9%). For the NEON-O Trial, the ICER was £12,526 (incremental cost: £178; incremental QALY: 0.0142; probability of cost-effectiveness at £30,000 per QALY gained: 88.2%). CONCLUSIONS: The NEON Intervention improved QALYs for both trial populations and is a cost-effective digital health intervention for people with non-psychosis mental health problems.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133076","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing the Risk of Bias in Clinical Trials for Health Technology Assessments: Should Existing Tools Reflect the ICH E9(R1) Addendum on Estimands and Sensitivity Analyses?","id":"91000b68-728c-456b-b89d-955a0b9ea450","sessionCode":"HTA50","topDisplay":"Poythress J1, Morga A2 1Astellas Pharma Global Development, Inc., USA, Chicago, IL, USA, 2Astellas Pharma Europe Ltd, Addlestone , UK","locationCode":"5017","description":"\r\n\t<div>OBJECTIVES: For randomized, interventional studies, the EUnetHTA 21 guideline on Validity of Clinical Studies recommends the Risk of Bias 2 (RoB 2) tool developed by the Cochrane group for assessing risk of bias as part of the Joint Clinical Assessment (JCA). In this study, we look at the extent to which the existing RoB 2 tool aligns with the principles of the ICH E9(R1) Addendum and identify relevant gaps. METHODS: We critically review the Cochrane RoB 2 tool from the perspective of the ICH E9(R1) Addendum. We examine each signaling question and ask whether the question can be answered appropriately for a trial in which the endpoint was defined in terms of an estimand as described in ICH E9(R1). We identify areas where misalignment between the terminology used by the RoB 2 tool and ICH E9(R1) could cause confusion, leading to an inappropriate conclusion with respect to risk of bias or conflicting conclusions across different assessors. RESULTS: The Cochrane RoB 2 tool does not easily accommodate assessment of endpoints in which the treatment effect of interest corresponds to hypothetical or while-on-treatment strategies for handling intercurrent events. For endpoints defined using a composite strategy, appropriate use of the RoB 2 depends on the subjective interpretation of the user, potentially resulting in conflicting conclusions among different assessors. Although the treatment policy and principal stratum strategies are most aligned with the two choices of treatment effects offered to users of the RoB 2 tool (i.e., ITT and per-protocol), nuances in the different concepts represented by ITT vs. treatment policy and per-protocol vs. principal stratum could still result in misapplication of the tool. CONCLUSIONS: It is critical that tools for assessing risk of bias, such as RoB 2, are updated to reflect the relevant ICH guidelines that inform the design, conduct and interpretation of clinical studies. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23morgahta50poster132303-pdf.pdf?sfvrsn=58959a06_0","title":"ISPOREurope23_Morga_HTA50_POSTER132303.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132303","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Efficiency and the Opportunity for Expanded Treatment Access with Incremental Increases in Biosimilar Adalimumab and Tocilizumab Use: A European Perspective","id":"694a7264-601c-4880-a5e9-95de3aae99dd","sessionCode":"EE121","topDisplay":"Shastri K1, Clarke K2, Ainslie-Garcia M2, Ferko N2 1Fresenius Kabi SwissBioSim GmbH, Morris Plains, NJ, USA, 2EVERSANA, Burlington, ON, Canada","locationCode":"2053","description":"\r\n\t<div>OBJECTIVES: Biosimilars can help to improve patient access through providing cost-effective treatment options. This study assessed the savings and expanded patient access that could be realized from incremental increased use of adalimumab biosimilars, and the introduction of a tocilizumab biosimilar. METHODS: Separate ex-ante analyses were conducted for France, Germany, Italy, Spain, and the United Kingdom (UK), parameterized using country-specific list prices, unit volumes annually, and market shares for each therapy. Discounting scenarios of 20%, 30%, and 40% were tested for tocilizumab. Outputs included direct cost-savings associated with drug acquisition, and the incremental number of patients that could be treated on a budget-neutral basis. Two biosimilar conversion scenarios were tested. RESULTS: Current-day use of adalimumab biosimilars ranged from 46% in France to 85% in the UK. Gain in total biosimilar market share yielded savings of €8.4 million in the UK (93% market share) to €102 million in Germany (88% market share), equivalent to 877 and 10,611 additional patients that could be treated under a budget-neutral basis. Exclusive use of adalimumab biosimilars could yield €15.8 million (1,644 additional patients) in near-saturated markets such as the UK, to €204 million in Germany (21,223 additional patients). Introduction of biosimilar tocilizumab provided savings of €15.7-€57.2 million in the most conservative scenario (20% discount at current day adalimumab biosimilar shares), equivalent to treating an additional 12%-21% of patients. With exclusive use of tocilizumab biosimilars at a 40% discount, this increased to €38.4-€150 million (67% increase in patient access for all countries). CONCLUSIONS: This study demonstrates the benefits that can be realized through increased biosimilar adoption, not only in an untapped tocilizumab market, but also through incremental increases in well-established markets such as adalimumab. As healthcare budgets continue to face downwards pressure globally, strategies to increase biosimilar uptake could prove useful to help manage financial constraints.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023cost-efficiency-of-ada-and-tcz-biosimilars-poster129969-pdf.pdf?sfvrsn=eecfc42d_0","title":"ISPOR EU 2023_Cost efficiency of ADA and TCZ biosimilars Poster129969.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129969","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Identification & Use of Prognostic Variables (PVs)/Treatment Effect Modifiers (TEMs) in Indirect Treatment Comparisons (ITCs) By Systematic Literature Review (SLR): Case Study of Chimeric Antigen Receptor (CAR) T-Cell Therapies","id":"d08455cb-a62a-4f69-822c-95ec0c240f79","sessionCode":"CO14","topDisplay":"Igbelina CD1, Campden R2, Grieve S3, Thakur D4 1Cytel Inc., Vancouver, BC, Canada, 2Cytel Inc., Calgary, AB, Canada, 3Cytel Inc., Montreal, QC, Canada, 4Cytel Inc., Toronto, ON, Canada","locationCode":"1017","description":"\r\n\t<div>OBJECTIVES: ITCs estimate relative treatment effects not compared in head-to-head trials. According to the assumption of transitivity, trials must be comparable on factors including PVs/TEMs; however, these are not always consistently identified. METHODS: An SLR was conducted across three databases (Embase, MEDLINE, CDSR) to identify ITCs including CAR T-cell therapies. Data on the indication, intervention, identification, PVs/TEMs, and sensitivity analyses were summarized. Abstracts were considered if no associated full-text articles were identified. RESULTS: 687 publications were identified; 30 ITCs (14 abstracts, 16 articles) on seven hematological malignancies met the inclusion criteria. Five therapies (with overlap between studies) were evaluated: tisagenlecleucel (12), axicabtagene (9), ciltacabtagene (9), lisocabtagene (8), and idecabtagene (5). Most abstracts (71% of 14) did not report how PVs/TEMs were identified, while full-text publications included varied descriptions. PVs/TEMs were identified by statistical ranking (31%), literature review and clinical experts (19%), not reported (19%), clinical experts (13%), or literature review (6%). There was heterogeneity in PVs/TEMs within the same indication and among ITCs evaluating the same CAR-Ts. Common variables between studies included age (22), ECOG status (13), and prior stem-cell transplant (12). Among seven ITCs in RRMM, two ITCs adjusted for sex, and one ITC each adjusted for creatine clearance, extramedullary disease, and race. There was no overlap in PVs/TEMs among seven ITCs in DLBCL (including two ITCs comparing lisocabtagene to tisagenlecleucel; four ITCs comparing axicabtagene to lisocabtagene). Sensitivity analysis may account for the variable use of different PVs/TEMs between studies; however, less than half of the ITCs (13/30) reported sensitivity analysis. CONCLUSIONS: This case study identified a lack of transparency in the identification of PVs/TEMs and inconsistencies in the ways PVs/TEMs are incorporated into ITCs. There is a need for guidance on the identification of PVs/TEMs which would reduce bias in the ITC results and increase confidence in decision-making.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23thakurco14poster133649-pdf.pdf?sfvrsn=8be50702_0","title":"ISPOREurope23_Thakur_CO14_POSTER133649.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133649","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Technology Assessment (HTA) Case Studies: Comparing Acceptability of Real-World Evidence (RWE) in Appraisals for Oncology Medicines","id":"5de9d749-13ac-4527-ba2e-961843454779","sessionCode":"HTA8","topDisplay":"Zong J1, Pan X2, Rojubally A3, Jiao X1, Bruno A1, Gdovin Bergeson J4 1Bayer Healthcare Pharmaceuticals, Inc., Whippany, NJ, USA, 2Bayer Healthcare Pharmaceuticals, Inc., Boston, MA, USA, 3Franklin Pharmaceutical Consulting, Surrey, BC, Canada, 4Franklin Pharmaceutical Consulting, Columbia, SC, USA","locationCode":"4050","description":"\r\n\t<div><span class=\"NormalTextRun SCXW227764482 BCX8\" data-ccp-parastyle=\"Table Paragraph\" data-ccp-parastyle-defn=\"{"ObjectId":"f20d6368-bbb0-430c-af6f-b215b3316dab|17","ClassId":1073872969,"Properties":[469775450,"Table Paragraph",201340122,"2",134234082,"true",134233614,"true",469778129,"TableParagraph",335572020,"1",469777841,"Arial Black",469777842,"Arial Black",469777843,"Arial Black",469777844,"Arial Black",469769226,"Arial Black",335559685,"107",134245417,"false",469778324,"Normal"]}\">OBJECTIVES: RWE is increasingly utilized in HTA appraisals. Clear guidance on RWE to be accepted by HTA bodies is lacking. This review compared RWE acceptability in HTA decision-making in recent appraisals for oncology medicines. METHODS: A search of Technology Appraisals published by NICE, Benefit Assessments published by G-BA and Opinions on Medicinal Products reports published by HAS from January to December 2022 was conducted. Oncology medicine appraisals which included RWE were identified and RWE acceptability was classified as (primary evidence, supportive evidence, not adequate for decision making, not addressed, or other) by reviewer’s assessment. Medicines reviewed by more than one HTA body were selected for comparative assessment of RWE acceptability. RESULTS: RWE was referenced in 16 reports by NICE, 14 by G-BA, and 10 by HAS. Six oncology medicines referencing RWE were selected as case studies: amivantamab, sotorasib and tepotinib (NICE and G-BA); avapritinib, axicabtagene-ciloleucel and tisagenlecleucel (HAS and G-BA). NICE acceptability of RWE was in alignment with G-BA's for amivantamab (RWE not adequate for decision-making) and divergent for tepotinib and sotorasib (NICE: RWE as supportive evidence and G-BA: RWE not adequate for decision-making). HAS and G-BA were in alignment for tisagenlecleucel and axicabtagene-ciloleucel (RWE not adequate for decision-making) and diverged for avapritinib (HAS: RWE as supportive evidence and G-BA: RWE not adequate for decision-making). RWE acceptability by NICE as supportive evidence is promising. HAS and G-BA’s evaluation of RWE suggests emphasis on data quality, selection biases, and confounding factors related to RWE use. CONCLUSIONS: There is discrepancy in the assessment of RWE by the 3 HTAs in recent oncology appraisals. Demands for quick and uniform access of new oncology therapies warrant harmonization of RWE relevance across HTAs, and development of comprehensive consensus guidelines with all stakeholders to standardize best practices for RWE studies and assessment. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23zonghta8poster132157-pdf.pdf?sfvrsn=6f50fbdc_0","title":"ISPOREurope23_Zong_HTA8_POSTER132157.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132157","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Optimizing Funding Priorities to Maximize the Impact: Valuable Health Research Approach","id":"9b124399-b137-4a71-bb2d-96306061e5e7","sessionCode":"EE145","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: We aimed to refine a funder’s research priorities by explicitly evaluating the prospect of benefits from new evidence, using an approach that considers the best available evidence, patients’ and clinicians’ values, research cost, and lost opportunities to study participants. METHODS: A Norwegian funder, the Kavli Trust Programmes on Health Research set funding priorities by assessing evidence from systematic reviews. One of the 2022 priorities was non-pharmacological interventions for children and adolescents with attention-deficit/hyperactivity disorder (ADHD). We evaluated five ADHD interventions with a systematic review summarising the effectiveness on core/overall ADHD symptoms: social skills training, mindfulness-based intervention, mind-body exercise, cognitive intervention, and physical activity intervention. We conducted cumulative meta-analyses using these systematic review results. To simulate how evidence from a new randomized controlled trial (RCT) might impact a clinical decision, we revised the cost-effectiveness analysis used for developing the 2018 NICE guideline for treating children and adolescents with ADHD. Then, we calculated a new RCT’s expected net benefit of sampling (ENBS) for two hypothetical budgets of £100,000 and £200,000. RESULTS: Funding an RCT of social skills training intervention with a budget of £100,000 and £200,000, had the maximum potential impact with the ENBS of £1,057,068 and £1,626,598, respectively. Also, an RCT of cognitive intervention might be valuable because the ENBS was £136,683 and £646,563 for the corresponding budgets. However, the evidence from an RCT might have little or no value for other interventions because the ENBS was -£89,592 and £10,719 for physical activity intervention, -£98.891 and -£94.101 for mind-body exercise, and -£117,976 and -£246.273 for mindfulness-based intervention. CONCLUSIONS: We demonstrated that, by implementing the valuable research approach, funders could enhance the research priority-setting process and maximise the chance of generating evidence that might benefit patients, clinicians, and policymakers. Ultimately, this approach might help address waste and increase the value of health research. </div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133398","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of ESMO-Recommended Lenvatinib and Pembrolizumab Regimen as Second-Line Treatment for Advanced Endometrial Carcinoma in Taiwan: A Comparison With NCCN Recommendations","id":"821479b2-c4fe-4c1a-bcde-97a9a3bd310a","sessionCode":"EE55","topDisplay":"Chueh CH1, Ho PK2, Chiang ST2, Wen YW3, Shiu MN2, Liu JH4, Li WH4, Tsai YW2 1National Yang Ming Chiao Tung University, Taipei city, Taiwan, 2National Yang Ming Chiao Tung University, Taipei, Taiwan, 3Chang Gung University, Taoyuan, Taiwan, 4Cheng Hsin General Hospital, Taipei, Taiwan","locationCode":"1045","description":"\r\n\t<div>BACKGROUND: The European Society for Medical Oncology (ESMO) and the National Comprehensive Cancer Network (NCCN) provided different recommendations for the second-line treatment of advanced endometrial carcinoma (EC). ESMO recommends the combination of lenvatinib and pembrolizumab (LP) for all patients, while the NCCN suggests LP for patients with proficient mismatch repair (pMMR) and pembrolizumab for those with deficient mismatch repair (dMMR). OBJECTIVES: This study aims to evaluate the cost-effectiveness of the universal LP regimen recommended by ESMO as a second-line treatment for advanced EC, compared to the biomarker-based treatment regimen proposed by the NCCN, within the context of Taiwan's National Health Insurance (NHI). METHODS: A 20-year partitioned survival model was employed using the efficacy results from the KEYNOTE-775 and KEYNOTE-158 trials. The prices of LP referred to the fee schedule set by Taiwan NHI. Direct medical costs were estimated based on NHI claim data. Health state utilities and disutilities associated with adverse events were derived from relevant literature sources. The willingness-to-pay threshold was set as three times the GDP per capita in 2022 (equivalent to NT$2,925,582). Quality-adjusted life-years (QALYs) and costs were discounted at a rate of 3%. Deterministic and probabilistic sensitivity analyses were conducted to assess the uncertainty. RESULTS: The ESMO-recommended universal LP regimen, when compared to the NCCN-recommended treatment regimen, led to a cost reduction of NT$821,018 but a decrease of 0.6 QALYs. The analysis revealed a negative net monetary benefit (-NT$937,915) and an exceedingly low acceptance probability (2%). The time horizon, health state utilities, and medication cost of lenvatinib emerged as the primary sources of uncertainty influencing the results. CONCLUSIONS: Within the framework of Taiwan's NHI, the NCCN's biomarker-based regimen emerges as a more cost-effective treatment option for patients with advanced EC compared to ESMO's recommendation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23chiangee55poster133218-pdf.pdf?sfvrsn=28493139_0","title":"ISPOREurope23_Chiang_EE55_POSTER133218.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133218","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"What Do Patients Value? Development of a Preference-Based Health Utility Score for Chronic Obstructive Pulmonary Disease (COPD)","id":"b5c36cc6-d6e1-4e12-8f55-986f1569c6a7","sessionCode":"PT6","topDisplay":"Jones B1, Cook N2, Ryan M3 1Novartis Pharma AG, Basel, Switzerland, 2Novartis Pharma AG, Basel, Basel Stadt, Switzerland, 3Health Economics Research Unit, University of Aberdeen, Aberdeen, Scotland, UK","locationCode":"6A","description":"\r\n\t<div>OBJECTIVES: There is increasing interest in the integration of quantitative evidence from patient preference studies into HTA decision-making with a question being how this can best be achieved. We generated a health utility score for patients with COPD and considered its use within HTAs. METHODS: Based on prior qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance and urinary incontinence. A Discrete Choice Experiment (DCE) based on these six symptoms was employed with 1050 COPD patients from 5 countries (US, UK, France, Australia, Japan). The random parameter logit regression technique was used to estimate utility scores for all COPD health states. The relationship between patients’ COPD health utility score, self-perceived severity of their COPD and EQ-5D-3L utility score was assessed, with data stratified according to patient COPD disease severity and comorbidity subgroups. RESULTS: The COPD health utility model had face validity, with utility scores negatively correlated with patients’ self-perceived COPD severity. Correlation between the COPD health utility scores and EQ-5D-3L values was only moderate (Spearman correlation, 0.52). This was largely explained by patients with EQ-5D-3L scores <0 exhibiting a range of comorbidities beyond their COPD which impacted their EQ-5D-3L scores, whereas the COPD health utility score was impacted less by the comorbid conditions. CONCLUSIONS: A disease-focused measure of utility offers benefit in clinical trials of new therapies and in generating utility data in support of HTA submissions. Our COPD utility measure, derived from the DCE, provides a patient-centered health utility score, which is more sensitive to the COPD health of the individual and less sensitive to comorbidities. The instrument should be considered alongside more generic instruments for use when valuing new COPD therapies and using utility data in submissions to licensing and reimbursement agencies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023jonespt6poster129887-pdf.pdf?sfvrsn=dfe319aa_0","title":"ISPOREurope_2023_Jones_PT6_POSTER129887.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129887","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Association between Health-Related Quality of Life and Requirement for Non-Professional Care in Adults with Rare Diseases: A Real-World Study","id":"2b685150-2ceb-4d50-848e-98ba9975f8b5","sessionCode":"PCR27","topDisplay":"Johnson B1, Piercy J2, de Courcy J3, Castellano G3, Iqbal H2, Gibson G3, Williams A4 1Kyowa Kirin International, West Drayton, LON, UK, 2Adelphi Real World, Bollington, UK, 3Adelphi Real World, Bollington, Cheshire, UK, 4Kyowa Kirin International, Marlow, Buckinghamshire, UK","locationCode":"6030","description":"\r\n\t<div>OBJECTIVES: Rare diseases are often associated with a considerable burden for patients and their caregivers. Non-professional care is common, typically provided by family and friends. This study assessed whether patients’ health-related quality of life (HRQoL) is associated with the requirement for non-professional care across five rare diseases. METHODS: We analysed real-world data from Adelphi Disease Specific Programmes for five rare diseases: Amyotrophic Lateral Sclerosis (ALS), Graft-Versus-Host Disease (GVHD), Huntington’s Disease (HD), Myasthenia Gravis (MG), Progressive Supranuclear Palsy (PSP) in the USA, France, Germany, Italy, Spain, UK; July 2017 to March 2021. Physicians provided cross-sectional data on patients’ demographics and non-professional care requirements whilst patients self-reported HRQoL via the EQ-5D. Multivariable logistic regression assessed the relationship between patients’ EQ-5D utility and presence of a non-professional caregiver, controlling for patients’ characteristics (age, sex, geographic location, and disease). RESULTS: The analysis included 823 patients with a mean (standard deviation [SD]) age of 55.8 (14.5) years; 58.6% were male; 23.0% from the USA. 12.0%, 28.6%, 5.7%, 37.9%, and 15.8% of patients had ALS, GVHD, HD, MG, and PSP, respectively. Patients had a mean (SD) EQ-5D utility of 0.64 (0.24). Non-professional caregivers were reported for 49.8% of patients (range 37.8% MG – 60.8% PSP). Multivariable regression showed a significant (p<0.001) association between EQ-5D utility and presence of a non-professional caregiver (odds ratio 0.037; 95% confidence intervals 0.014-0.097), indicating that an increase of 0.1 in EQ-5D utility is associated with a 28% decrease in the odds of receiving non-professional care. Additionally, older patients, and those with GVHD (using MG as a reference category), were significantly more likely to receive non-professional care. CONCLUSIONS: Results showed a significant, negative association between patients’ EQ-5D utility and requirement for non-professional care, indicating that improvements to HRQoL in individuals with rare diseases may lead to a reduced burden on family and friends.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23johnsonpcr27poster130480-pdf.pdf?sfvrsn=594fa2a0_0","title":"ISPOREurope23_Johnson_PCR27_POSTER130480.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130480","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing the Definition and Outcomes of Innovative Drugs by Health Technology Assessment Bodies in England, France, and Italy (Q1 2023)","id":"dd10ec87-f36a-41b0-8d3f-98d9c2d9cc84","sessionCode":"HTA37","topDisplay":"Roberts G1, Taylor I2, Greenwood M1, Lewis H1 1Initiate Consultancy, London, London, UK, 2Initiate Consultancy, London, UK","locationCode":"4073","description":"\r\n\t<div>OBJECTIVES: Innovation is a critical consideration for pharmaceutical companies and health technology assessment (HTA) bodies. A medicine being recognised as innovative can result in financial rewards and accelerated approval processes for the manufacturer. However, the definition and interpretation of ‘innovation’ differs across jurisdictions, potentially leading to inconsistencies in the recognition and reward of innovative medicines. This study investigates how various HTA bodies define and measure innovation, as well as whether there are any benefits or trends for medicines classified as ‘innovative’. METHODS: Data was collected from the websites of three HTA bodies during the first quarter of 2023 (January to March): NICE (England), HAS (France), and AIFA (Italy). An extensive review of HTA and innovation reports was carried out to determine which medicines were classified 'innovative' and identify any prevailing trends regarding the types of innovative medicines gaining reimbursement. RESULTS: Out of a total of 76 publicly available appraisals, 26 (30.3%) technologies were recognised as ‘innovative’ by either NICE (8/23 [34.8%]), HAS (16/37 [43%]), or AIFA (3/16 [18.7%]). The majority of assessments classified as innovative by HAS pertained to orphan drugs (9/16 [56.25%]), while NICE and AIFA appraisals each only classified one orphan drug as innovative. The granting of innovative status was often based on whether drugs represented a significant advancement in treatment or addressed conditions with substantial unmet needs. Most innovative products were for oncology treatment (17/28 [62.96%]), with only one categorised under each of the other therapeutic areas in each country. CONCLUSIONS: This study demonstrates the varying rates of recognition for innovative medicines by highlighting how different HTA bodies define and measure innovation. The findings emphasise the importance of harmonising the definition and assessment of innovation across jurisdictions to ensure consistency in the recognition and reward of innovative medicines if innovation is to be used as a criterion for appraisal.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/assessing-the-definition-of-innovation-and-the-outcomes-of-innovative-drugs-in-england-france-and-italy-in-q1-2023132365-pdf.pdf?sfvrsn=45f12127_0","title":"Assessing the definition of innovation and the outcomes of innovative drugs in England, France, and Italy in Q1 2023132365.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132365","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Progressive Disease Milestones and Survival in Duchenne Muscular Dystrophy (DMD): A Model-Based Synthesis for Extrapolating Lifetime Treatment Effects From Clinical Trial Results","id":"e713e04e-e2ca-4607-b632-9a1a7052e39a","sessionCode":"PCR29","topDisplay":"Smith E1, Posner N2, Signorovitch J3, Johnson M3, Merla V2, Gomez-Lievano A3, Zhang A3, Sharma A3, Inês M4, Cappelleri J2, Cislo P2 1Duke University, Durham, NC, USA, 2Pfizer Inc., New York, NY, USA, 3Analysis Group, Inc., Boston, MA, USA, 4Pfizer Inc, Porto Salvo, 11, Portugal","locationCode":"6031","description":"\r\n\t<div>OBJECTIVES: Randomized controlled trials of gene therapies in Duchenne muscular dystrophy (DMD) have studied effects on ambulatory function limited to one year using the North Star Ambulatory Assessment (NSAA). However, the projected lifetime effects of these therapies will need to be considered in health economic evaluations. Drawing on natural history data, we developed a model-based synthesis for projecting plausible effects on longer-term disease milestones, including mortality, based on 1-year outcomes from clinical trials. METHODS: A quantitative natural history (NH) model of progressive milestones was developed to link 1-year changes in NSAA total score to mortality though intermediate associations with loss of ambulation (LoA) and need for mechanical ventilation. Median time between milestones were estimated from a targeted literature review and a Kaplan-Meier analysis of the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG) database. RESULTS: The NH model estimated the median age of LoA at 11.0 years and median time from LoA to first use of mechanical ventilation at 9.4 years, consistent with published estimates. A literature review yielded a median time of 7.0 years from ventilation to death. The model-based median age at death was 27.4 years, consistent with published medians of 27.0-36.2 years. Two scenarios were developed for projecting plausible lifetime treatment effects: 1) a ‘fixed-shift’ effect, where age-at-event for all milestones is delayed by the same number of years, and 2) a ‘persistent slowing’ effect in which milestones are delayed based on a constant deceleration of disease progression. CONCLUSIONS: Lifetime effects of novel therapies for DMD will ultimately be determined based on real-world evidence over several decades. In the near-term, the NH model presented here, which incorporates NH data, published evidence and plausible assumptions, can complement and supplement the lifetime projections used in health economic evaluations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23smithpcr29poster133787-pdf.pdf?sfvrsn=a7c19127_0","title":"ISPOREurope23_Smith_PCR29_POSTER_133787.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133787","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Cost-Effectiveness of Asfotase Alfa for Treatment of Patients with HPP in England","id":"57dcd03c-27a2-4a3e-9bd6-9a7f5fb229ba","sessionCode":"EE93","topDisplay":"Dawson H1, OConnell T2, Fang S3, Kirkwood K4, Crowell M5, Saraf Y1 1Alexion, AstraZeneca Rare Disease, London, England, UK, 2Medicus Economics, LLC, Cambridge, MA, USA, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4Parexel, New York, NY, USA, 5Medicus Economics, LLC, Boston, MA, USA","locationCode":"2031","description":"\r\n\t<div>OBJECTIVES: In 2017, a managed access agreement (MAA) was developed establishing coverage in England of asfotase alfa, an enzyme-replacement therapy for paediatric-onset hypophosphatasia, an ultra-rare condition associated with significant mortality in infants and morbidity in children and adults. The MAA specified an outcomes-based agreement, conditioning payments on realization of benefits that were modelled in a cost-effectiveness analysis (CEA) of asfotase alfa vs best supportive care. Such benefits included (i) for patients starting treatment at ages <2 years, invasive-ventilation free survival at age 2 and (ii) for patients starting treatment at ages ≥2, quality-adjusted life year (QALY) gains based on health-utility improvements (PedsQL for ages 2-<18 years and EQ-5D-5L for ages ≥18 years). In this analysis, benefits observed under the MAA were compared against cost-effective benefit levels from the original CEA, to assess the real-world cost-effectiveness of asfotase alfa. METHODS: Actual outcomes observed under the MAA were compared to expected outcomes from the original CEA, grouping patients by baseline-age (years) cohorts (0-1, 2-4, 5-12, 13-<18, and adult). RESULTS: The MAA enrolled 54 patients from 2017-2022, of whom 2 did not receive treatment and 4 had HPP-unrelated issues preventing analysis of QALY gains. Ten patients started the MAA at ages <2; all 8 who turned age 2 during the MAA were invasive-ventilation free. QALY gains were analyzed in the 38 patients who started treatment at ages ≥2 and the 8 who turned 2 during the MAA. Median follow-up was 27, 45, 54, and 24 months for the 0-1, 2-4, 5-12, and adult baseline-age cohorts. No patients were included for the 13-17 cohort. Median actual-minus-expected QALY gains across the cohorts ranged from -0.13 to +0.48. Overall, 89% of expected QALY gains were realized. CONCLUSIONS: Benefits of asfotase alfa observed under the MAA were consistent with previous CEA, indicating real-world cost effectiveness.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sarafee93poster131813-pdf.pdf?sfvrsn=bd7158bb_0","title":"ISPOREurope23_Saraf_EE93_POSTER131813.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131813","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Mean Time on Triptan Treatment Among Danish Migraine Patients Before Discontinuing Treatment—A Register-Based Study","id":"d41f1d61-47e9-482a-9c7c-9a85a9b80037","sessionCode":"EPH22","topDisplay":"Ashina M1, Hansen T2, Hansen JM3, Hauberg D4, Lønberg US5, Steiner TJ6 1Copenhagen University Hospital - Rigshospitalet, Copenhagen, Capital Region, Denmark, 2Copenhagen University Hospital - Rigshospitalet, Glostrup, Denmark, 3Private Neurology Practice, Slagelse, Region Zealand, Denmark, 4Pfizer, Ballerup, 84, Denmark, 5Pfizer, Ballerup, Capital Region, Denmark, 6Norwegian University of Science and Technology, Trondheim, Trøndelag, Norway","locationCode":"3024","description":"\r\n\t<div>OBJECTIVES: To determine the correlation between number of distinct triptans tried and mean total time on triptan treatment (TToTT) among Danish migraine patients, using routinely collected data. METHODS: From the Danish National Prescription Register we identified patients (≥18 years) who had redeemed at least one triptan prescription between 1998 and 2019 and subsequently discontinued treatment with triptans (defined as no further triptan prescription redemptions for at least a two-year period). They were divided into seven groups corresponding to the number of distinct triptans tried (range 1-7) before discontinuation. Mean TToTT was calculated for each group. RESULTS: N=211,026 acquired at least one triptan during the study period. The majority tried only one distinct triptan (n=172,668), with a mean TToTT of 1.5 years, but most had limited exposure to triptans (median TToTT=0.0 years). Significantly longer means were observed between groups treated with 2 vs. 3 (p<0.001) and 3 vs. 4 (p<0.001) distinct triptans (6.6, 9.2 and 10.7 years for 2, 3 and 4 triptans respectively). However, no further increase in mean TToTT was observed when comparing groups treated with 4 vs. 5, 5 vs. 6 or 6 vs. 7 distinct triptans. CONCLUSIONS: The study shows that mean TToTT among patients who eventually discontinued triptans altogether increased with the number of distinct triptans tried up until 4 triptans, with no further increase with >4 distinct triptans. Further research is needed to determine whether triptan discontinuation is due to inadequate symptom relief, poor tolerability or for other reasons, and to establish the likelihood of response to other triptans after failure (for whatever reason) of one or more.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hanseneph22132903-pdf.pdf?sfvrsn=4b74645e_0","title":"ISPOREurope23_Hansen_EPH22132903.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132903","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Standardized Contrasts Across Trials in Individual Patient Data Meta-Analysis With Time-to-Event Outcomes","id":"7216362a-7b76-4376-bb96-9ad246fe68f2","sessionCode":"MSR22","topDisplay":"Gasparini A1, Crowther M2, Schaffer JM3 1Red Door Analytics, Bromma, AB, Sweden, 2Red Door Analytics, Stockholm, Stockholms län, Sweden, 3Baylor Scott & White, The Heart Hospital, Plano, TX, USA","locationCode":"5067","description":"\r\n\t<div>OBJECTIVES: Randomised controlled clinical trials evaluating an intervention on time-to-event outcomes are common, with individual patient data (IPD) often combined in IPD meta-analyses (IPD-MA) to summarise the overall treatment effect across trials. This work develops and extends existing methodology for benchmarking performance and time-to-event outcomes across trials and other hierarchical units within a formal causal framework. METHODS: A common approach to IPD-MA is to analyse the pooled data with mixed-effects models, directly modelling and accounting for heterogeneity between trials while focussing on the fixed effects for inference. Nonetheless, mixed-effects models are also used for benchmarking purposes, e.g., focussing on the random effects to quantify the performance of hierarchical units (such as different trials or hospitals within regions). With this work, we extend previous work on linear mixed models (with a continuous outcome) to the settings of time-to-event outcomes. Specifically, we frame multilevel survival models within a formal potential outcomes framework and define relevant estimands, including contrasts across hierarchical units. We also define estimators for all newly-defined quantities, based on regression standardisation, including algorithms to estimate their standard errors. RESULTS: We illustrate the methodology in practice using data from an IPD meta-analysis of prognostic studies in breast cancer, where we provide fair (under certain assumptions) comparisons of survival probabilities across trials, including contrasts comparing, e.g., the best versus an average trial. Our approach is akin to direct standardisation, where we estimate how the overall study population would fare under the performance of each trial. Moreover, these measures can evaluate what the survival outcome from a trial would be if study participants had a different covariate distribution. CONCLUSIONS: The newly-developed methodology can be used to benchmark time-to-event outcomes with fair and unbiased comparisons (i.e., across time or geographical regions) between hierarchical units, such as different trials in IPD-MA. <div> <div></div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gasparinimsr22poster132626-pdf.pdf?sfvrsn=36ab2ab3_0","title":"ISPOREurope23_Gasparini_MSR22_POSTER132626.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132626","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Expected Value of Perfect Implementation of Icosapent Ethyl (Vazkepa) in Sweden","id":"490cf4cf-24e7-462a-9ab9-9b25cd625b79","sessionCode":"EE6","topDisplay":"Eklund M1, Bernfort L2, Levin LÅ2 1Linköping University, Linkoping, E, Sweden, 2Linköping University, Linkoping, Sweden","locationCode":"1050","description":"\r\n\t<div>OBJECTIVES: Icosapent ethyl group (Vazkepa) reduces the risk of cardiovascular (CV) disease recurrence in statin-treated patients with elevated levels of triglycerides. Clinical practice variations and uneven implementation of effective and cost-effective healthcare technologies are a key challenge for healthcare systems which may lead to suboptimal treatment and in turn efficiency losses, increased costs, and health losses. This study aims to subcategorize the expected value of perfect implementation (EVPIM) to estimate the absolute and relative value of eliminating slow, low, and delayed implementation of Vazkepa in Sweden and by Swedish regions. METHODS: This study estimates the effects of optimal (perfect) and actual implementation in Sweden from Swedish hospitals after incident events in a five-year perspective by combining cost-effectiveness evidence with epidemiology from publicly available data from Swedeheart registry. The implementation analysis is based on the EVPIM framework by Fenwick et al. which estimates the value of increasing implementation from the current level up to a perfect level. RESULTS: EVPIM estimates the absolute and relative value of eliminating different parts of the suboptimal implementation. Preliminary results show that 84 % of the suboptimal implementation is represented by low implementation, and 16 % by slow implementation. The total EVPIM estimates are 936 QALYs and SEK 468 million, equal to a cost of SEK 18 960 per patient. Thus, it is estimated that by eliminating low and slow implementation at least, 1 869 CV events may be avoided over a five-year period, including 159 deaths, 145 strokes, and 480 myocardial infarctions. CONCLUSIONS: An EVPIM estimate of SEK 468 million implies that implementation strategies up to this value are cost-effective. The results also show the health loss that may be avoided by eliminating the suboptimal implementation strategies. This may help decision makers identify what part of the suboptimal implementation is contributing to the largest share of the total EVPIM.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23eklundee6poster130784-pdf.pdf?sfvrsn=7cca9ea8_0","title":"ISPOREurope23_Eklund_EE6_POSTER130784.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130784","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patient Preference for Paroxysmal Nocturnal Haemoglobinuria Treatments: Analysis From the Q-Methodology of Treatment Attributes","id":"cd776c19-1a82-4970-a246-9b4b8b9558e7","sessionCode":"PCR32","topDisplay":"Fishman J1, Wilson K2, Middleton S3, Ralph L4, Salcines J3 1Apellis Pharmaceuticals, Inc., Waltham, MA, USA, 2Swedish Orphan Biovitrum AB, Stockholm, Sweden, 3L.E.K Consulting, London, UK, 4L.E.K. Consulting, San Francisco, CA, USA","locationCode":"6034","description":"\r\n\t<div>OBJECTIVES: Collect quantitative data on drivers of patient treatment preferences in paroxysmal nocturnal haemoglobinuria (PNH). METHODS: PNH patients (n=156, U.S., n=117, EU and Canada n=39) completed a questionnaire ranking attractiveness of PNH treatment aspects, including product and clinical features, considering available treatments. A Q-methodology was used to collect the data and utility scores were computed as average ranks, on a -4 to +4 scale. RESULTS: Fatigue levels and blood transfusions, together with dosing frequency had the highest preference among patients. Unlikeliness to feel fatigued at all was preferred (+2.13) compared to somewhat fatigued (-0.21) or very much fatigued (-1.64). This correlated with fatigue symptoms reported by patients as impactful on quality of life and mobility. Treatments that meant no annual blood transfusions required were preferred (+1.97) vs requirement of 1, 2, 3 or 4+ transfusions annually while taking the drug (-0.61, -1.46, -2.02 and -2.74 respectively). Administration every eight weeks was most valued (+2.62), vs every two weeks (+0.69), weekly (-0.38) or twice per week (-1.40). Route of administration was less impactful (+0.07 for subcutaneous pump; +0.08 for subcutaneous auto-injector; +0.44 for intravenous), however, aspects like mobility during administration (+1.74) and ability to self-administer at home or work (+1.17) were preferred. A needle hidden during administration was valued (+0.48) vs one visible (-0.02) or larger (+0.08). Scores were directionally similar for patients in Europe and Canada vs U.S., but less emphasis was given to no blood transfusion requirement (+1.31 and +2.19, respectively) and no fatigue (+1.97 and +2.19, respectively); reduced dosing (+2.77 and +2.57, respectively) and self-administration (+1.56 and +1.03, respectively) had a slightly importance outside of U.S. CONCLUSIONS: Results suggest that the most important PNH treatment attributes are improvement in fatigue and reduced number of blood transfusions required, along with less frequent dosing. Mobility during administration, self-administration and non-visible needles are also preferred.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/3002patient-prefsv5132258-pdf.pdf?sfvrsn=1132aa82_0","title":"3002_Patient Prefs_v5132258.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132258","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Health Return on Investment (ROI) Assessor: Evaluating Novel Cardiovascular Disease (CVD) Prevention Approaches in Estonia","id":"b8d1445f-346f-45ec-86a7-9bad12ba7ce9","sessionCode":"EE5","topDisplay":"Hernandez-Villafuerte K1, Schmitt M2, Fries J2 1WifOR Institute, Darmstadt , HE, Germany, 2WifOR Institute, Darmstadt, Hessen, Germany","locationCode":"1052","description":"\r\n\t<div>OBJECTIVES: Worldwide cardiovascular disease (CVD) morbidity and mortality trends induces high direct and indirect costs. We propose an innovative approach entailing a societal and macroeconomic perspective to assess value creation of health investments to reduce CVD burden and promote economic growth within the country: the Health ROI Assessor framework. It is operationalized in a measurement tool and applied to a real-world prevention program in Estonia. The intervention provides a tailored solution to reduce low-density lipoprotein cholesterol and related CVD risk at the population level (age 30 to 79, without CVD history). METHODS: The framework includes two pillars: 1. The Health Economy (HE) Footprint estimates direct effects within the HE (Gross Value-Added and corresponding job creation) and spillovers to other economic sectors (indirect/induced effects); 2. The Human Capital Effect evaluates avoided productivity losses related to better health outcomes. Model inputs include age-, gender- and risk-specific previously estimated avoided CV events (i.e., Markov Model implemented in a separate project), which could be derived from the Estonian intervention program. Labor-market interaction connects both pillars via labor-force supply/demand adjustments. Time series analysis allows us to build a dynamic framework of health investments. The implementation is based on health and economic data from official statistics. RESULTS: Results for Estonia suggest that each additional €1 Mio invested in CVD prevention creates €2.1 Mio of value in the HE and along the supply chain and 46 additional jobs within the first three years. The Human Capital Effect pillar indicates that the 350 non-fatal and 93 fatal avoided CVD events predicted for the first ten years translate into 0.9 million hours of gained productivity, representing €32.2 Mio (€18.8 Mio paid and €13.4 Mio unpaid work). CONCLUSIONS: Our results support policy decisions to improve preventive resource allocation, make healthcare systems sustainable and incentivize economic growth.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hernandez-villafuerteee5poster129130-pdf.pdf?sfvrsn=9d501c3d_0","title":"ISPOREurope23_Hernandez-Villafuerte_EE5_POSTER129130.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129130","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Review of Approved Managed Entry Agreements in Sweden","id":"fc227d8e-fdb6-49dc-9095-9c14c53beef8","sessionCode":"HPR42","topDisplay":"Pandey K1, Sharma A2, Singh B3, Pandey S1, Tripathi N1 1Heorlytics, SAS Nagar Mohali, PB, India, 2Pharmacoevidence, SAS Nagar Mohali, PB, India, 3Pharmacoevidence, Mohali, PB, India","locationCode":"4007","description":"\r\n\t<div>OBJECTIVES: Drug manufacturers' entry management process or Managed entry agreement (MEA) aims to reduce costs and address uncertainty in benefits from new health technologies. This Mid-path approach secures stakeholders' interests, promoting innovation and Pareto improvement in society's welfare by involving payers, manufacturers, and patients. This study analyzes the Swedish MEA market nature and highlights important observations for payers and manufacturers. METHODS: The study includes insights into Five financial-based MEAs for various events. We reviewed five indications specific MEAs, including Hepatitis C, Prostate Cancer, Heart failure, Hereditary Optic Neuropathy, and Cystic Fibrosis. RESULTS: Most agreements were signed at the national level to prevent regional inequality. To the most extent, Sweden's reimbursement decision relies on incremental cost-effectiveness ratio (ICER), resulting in a higher proportion of financial-based agreements focusing on cost containment rather than performance-based ones. In addition to MEAs for prescribed drugs in the market, there are also agreements for requisition drugs. While prescribed drugs had a market of 34 billion SEK (approximately 3.4 billion Euros) in 2021, local agreements were 2.7 billion SEK, and requisition drugs had 10 billion SEK. Sweden's MEAs focus on biosimilars and other therapeutic classes rather than innovative drugs. Among potential hurdles, the administrative burden is the main barrier to outcome-based MEAs, as more than the availability of nurses are needed, given the unwillingness of outsiders from payers and hospitals to collect patient data. CONCLUSIONS: MEAs are tools for achieving patient access to new medicines and health technologies through quicker coverage decisions while managing uncertainty about products' performance and budget impact. Coverage with Evidence Development has also proven burdensome for payers in Sweden and has often left them discouraged. Disease-specific and Product-specific contracts are crucial factors in the success of MEAs in Sweden. Financial-based agreements are more than other types of agreements in Sweden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/mea-poster133972-pdf.pdf?sfvrsn=80c7309c_0","title":"MEA poster133972.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133972","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Artificial Intelligence (AI) Based Telemedicine for Cost-Effective Cataract Surgery Follow-up at NHS: An Economic Evaluation","id":"ddf0713a-7a74-494d-9972-9c535aa1c2b0","sessionCode":"EE106","topDisplay":"Bajre M1, Lim E2, Pennington ND3, Rose J4 1Oxford Academic Health Science Network, Oxford, UK, 2Imperial College Healthcare NHS Trust, London, UK, 3Ufonia Limited, Oxford, UK, 4Oxford Academic Health Science Network, Oxford, OXF, Great Britain","locationCode":"2038","description":"\r\n\t<div>OBJECTIVES: A real-world economic evaluation was performed to compare the direct staff cost savings from the use of Artificial intelligence (AI) based telemedicine for post-operative cataract follow-up versus the standard face-to-face-led follow-up for the patients at Imperial College Healthcare NHS Foundation Trust (ICH). METHODS: The study models the impact of using an autonomous voice conversational agent developed to improve clinical efficiency for cataract surgery follow-up. The study evaluates the implementation of AI based voice telemedicine conversation delivered to patients via automated telephone calls for post-operative follow-up when compared to face-to-face follow-up by clinicians in the cataract post-operative standard care. AI telemedicine reduces the planned face-to-face visits and allows staff resources to be allocated to perform other tasks within the ophthalmology setting. A decision-analytic model was developed and tested for the real-world data provided for 97 patients for this analysis from ICH. Only staff costs from the face-to-face follow-up pathway were compared with the staff cost involved when AI based telemedicine was used. Moreover, follow-up of all the post-operative cataract patients by telemedicine may decrease unplanned eye casualty visits leading to improved patient management and resulting in further reduction in the cost of care. RESULTS: The economic analysis result indicated that there was an average staff cost saving of £35.18 per patient in the AI based telemedicine pathway when compared to the standard care pathway of face-to-face patient follow-up. The advantage of AI based telemedicine was in avoiding face-to-face clinicians’ appointment times for non-complicated cases and avoiding unnecessary hospital visits at ICH. Additionally, it also helps with reduced direct face-to-face visits with clinicians leading to reduce infection control at hospitals. CONCLUSIONS: This study concluded that the use of AI based telemedicine for post-operative cataract follow-up at ICH was cost-effective.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bajreee106poster128965-pdf.pdf?sfvrsn=856757bb_0","title":"ISPOREurope23_Bajre_EE106_POSTER128965.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128965","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"How Safe Are New Drugs? Analysis of US FDA Safety Regulatory Actions, 1980-2021","id":"dc500a64-66eb-432d-b6e1-9c7d34802b2c","sessionCode":"EPH43","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3043","description":"\r\n\t<div>OBJECTIVES: The USFDA's expedited pathways for drug development and review have enabled the rapid introduction of new drugs to the market, but have also reduced the available evidence on their safety and efficacy. This study assessed trends in USFDA safety actions, including market discontinuations, risk evaluation and mitigation strategies (REMS), boxed warnings, medication guides, and patient information, for new drugs approved by the USFDA between 1980 and 2021. It also evaluated the association between expedited review pathways and USFDA safety regulatory actions, and assessed the effect of the Prescription Drug User Fee Act (PDUFA) and the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) on USFDA safety regulatory actions. METHODS: Data on new molecular entities, new therapeutic biologics, and gene and cell therapies approved between 1980 and 2021 were extracted from the USFDA website. Descriptive analysis, restricted cubic spline non-parametric linear regression, and logistic regression were performed. RESULTS: From 1980 to 2021, the USFDA approved 1,310 new drugs. A total of 343 (31.8%) new molecular entities, 87 (39.7%) biologics, and 6 (50.0%) cell and gene therapies had boxed warnings. Market discontinuation affected 198 (15.1%) products, including 39 (3.0%) that were withdrawn for safety reasons. PDUFA increased patient information by 30% per year, while FDASIA increased patient safety information by more than 50% per year, medication guides by 15% per year, and REMS by 4%. Boxed warnings decreased by 11% per year after FDASIA. CONCLUSIONS: Safety withdrawals and REMS affected a relatively low number of drugs. Safety-related boxed warnings, medication guides, and patient information were included in the labels of one-fourth of the new drugs. The safety information directed to health care professionals decreased, while the safety information directed to patients increased during the study period.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132417","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evaluation of Vascular Access Modality for Hemodialysis Patients: Analysis of Real-World Data in Italy","id":"38023cfc-cbcd-4458-914f-9c8377b2f739","sessionCode":"HSD19","topDisplay":"Monteverde Spencer GT1, Dovizio M2, Iacolare B2, Andretta M3, Bacca M4, Bartolini F5, Barbieri A6, Ciaccia A7, Chinellato A8, Costantini A9, De Vita F10, Gentile S11, Mancini D4, Mensurati M12, Moscogiuri R13, Mosele E14, Pagliaro R15, Petragnani N10, Re D16, Santoleri F9, Martoni M1, Di Stasi F1, Degli Esposti L2 1W. L. Gore & Associati S.r.l., Verona, Italy, 2CliCon S.r.l. Società Benefit Health, Economics & Outcomes Research, Bologna, BO, Italy, 3Azienda ULSS 8 Berica, Vicenza, Italy, 4ASL Brindisi, Brindisi, Italy, 5USL Umbria 2, Terni, Italy, 6ASL Vercelli, Vercelli, Italy, 7Servizio Farmaceutico Territoriale ASL Foggia, Foggia, Italy, 8Azienda ULSS 3 Serenissima, Mestre (VE), Italy, 9ASL Pescara, Pescara, Italy, 10UOC Farmacia Ospedaliera, ASL Lanciano Vasto Chieti, Chieti, Italy, 11Direzione Generale per la Salute Regione Molise, Campobasso, Italy, 12ASL Roma 3, Roma, Italy, 13ASL Taranto, Taranto, Italy, 14UOC Assistenza Farmaceutica Territoriale Azienda ULSS 7 Pedemontana, Bassano del Grappa (VI), Italy, 15ASL Roma 5, Tivoli, Italy, 16ASL Teramo, Roseto degli Abruzzi, Italy","locationCode":"4029","description":"\r\n\t<div>OBJECTIVES: The vascular access type has implications on successful hemodialysis. This real-world analysis investigated the vascular access dialysis types: central venous catheter (CVC), arteriovenous fistula (AVF) and arteriovenous graft (AVG) in an Italian clinical practice setting. METHODS: A retrospective analysis was conducted on administrative databases of a sample of healthcare entities (geographically distributed across Italy), covering almost 10% of Italian population. Between 01/2009-08/2022, patients with ≥1 record of hemodialysis (procedure code 39.95 and/or hospitalization code V56.0) were included. The first hemodialysis record was the index-date; the vascular access type (CVC, AVF, AVG) was evaluated before and after the index-date. RESULTS: 3,451 hemodialysis patients were included; 61.7% males, mean age of 68.7 years. Among them, 48.6% had a single vascular access implantation: 33.1% had CVC only, 14.7% AVF only and 0.8% AVG only. Considering patients with ≥1 vascular access type, 24.9% were implanted with both CVC and AVF [14.5% (N = 502) started with CVC followed by AVF (after a mean of 218 days), 10.4% (N = 360) started with AVF followed by CVC (after a mean of 664 days)] and 0.8% (N = 26) had CVC-AVF-AVG pathway (after a mean of 169 and 439 days, respectively). Among AVG users (N=191, 5.5%), 47% (N = 90) started with AVG and 53% (N = 101) had AVG after an AVF or CVC. Among patients who switched vascular access, the mean duration (days) on the first vascular access was 533 ± 768 for CVC, 913±936 for AVF and 688 ± 846 for AVG. CONCLUSIONS: This real-world analysis shows vascular access types used in hemodialysis patients in Italy. Almost 50% of patients had a single vascular access implantation, almost 25% alternated CVC/AVF and 5.5% had AVG. These results could be informative on the management of hemodialysis patients in clinical practice in Italy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/231229550-en-postera0-av-access-italy-fnl-mr131022-pdf.pdf?sfvrsn=ee6fc221_0","title":"231229550-EN-Poster_A0-AV Access Italy-FNL-MR131022.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131022","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Transmission Dynamics and Vaccination Strategies for Chikungunya Virus (CHIKV): An Outbreak Simulation Study in Rome, Italy","id":"c7b0a13c-18b4-4e7c-8e66-9d7a2d0f119d","sessionCode":"EPH15","topDisplay":"Tiozzo G1, Sloof B2, Hofstra H3, Vondeling GT4, de Roo A5 1University Medical Center Groningen, Groningen, GR, Netherlands, 2University Medical Center Groningen, Groningen, Groningen, Netherlands, 3Asc Academics, Groningen, GR, Netherlands, 4Valneva, Vienna, Austria, 5Valneva, Vienna, 9, Austria","locationCode":"3015","description":"\r\n\t<div>OBJECTIVES: CHIKV is transmitted by Aedes aegypti and Aedes albopictus. The disease is characterized by high morbidity and debilitating long-term sequalae. The global spread of CHIKV has been facilitated by viral adaptation to new vectors and expansion of mosquito habitats due to climate change, posing increased outbreak risks in temperate regions after importation of CHIKV. This study assessed the transmission dynamics and effectiveness of vaccination as an intervention strategy in the event of an CHIKV outbreak. METHODS: A human-mosquito SEIR model was employed to replicate the transmission dynamics of the 2017 CHIKV outbreak in Anzio and simulate a hypothetical outbreak in Rome. The model incorporated mosquito population dynamics, accounting for the mosquito life-cycle stages and temperature dependencies. The primary outcome measure was the number of infections in two scenarios: with and without vaccination. Different levels of vaccine coverage were tested to evaluate their impact on infection rates. RESULTS: Without intervention and given the current environmental circumstances, a local CHIKV outbreak in Rome could affect 15.05% of the population, resulting in 413,607 infections. To illustrate the potential impact of climate change, an increase of 1-degree Celsius was applied in the model, leading to an infection rate of 81.38% among the local population. Assuming the vaccine effectiveness of 98.9% based on phase 3 seroprotection data, vaccination with VLA1553 could prevent two-thirds of infections with a coverage rate of 20%. Increasing the coverage to 40% resulted in a 90% reduction in infections. The timing of vaccine deployment and the time-to-immunity were considered in the analysis. CONCLUSIONS: The impact of a potential CHIKV outbreak in Rome showed to be considerable, especially in a future climate change scenario. The study highlights that in the event of a CHIKV outbreak, intervention with vaccination deployed in a timely manner would be highly effective in preventing a significant disease burden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23tiozzoeph15poster132600-pdf.pdf?sfvrsn=91486637_0","title":"ISPOREurope23_Tiozzo_EPH15_POSTER132600.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132600","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Technology Assessment of New Drugs in Spain: Understanding the Evaluators’ Evidence Needs","id":"7ff1e629-cb91-40dc-a780-9e67c74b14e9","sessionCode":"HPR28","topDisplay":"Carmo M1, Aguilar Sugrañes L2, Aguirre J2, Pinel M3, Domínguez J2, Callejo D3 1IQVIA, Oeiras, Portugal, 2IQVIA, Barcelona, Barcelona, Spain, 3IQVIA, Madrid, Madrid, Spain","locationCode":"3079","description":"\r\n\t<div>OBJECTIVES: To describe the health technology assessment (HTA) of drugs authorized by the European Medicines Agency (EMA) in Spain and their potential relation to the drugs’ reimbursement status. METHODS: A list of human medicines centrally authorized from 01/2018-12/2021 was retrieved from the EMA website. The date of the Therapeutic Positioning Report (“IPT” from Spanish initials) publication was extracted from the Spanish Agency of Medicines and Medical Devices (AEMPS) website. IQVIA’s HTA Accelerator database contains HTA publications from over forty countries and was used to extract detailed IPT data, when available, categorizing the evaluators remarks by type and as positive or negative. A product was considered reimbursed when included in the national reimbursement list as of 20th June 2023 (Nomenclátor). RESULTS: A total of 168 new medicines were authorized by the EMA during the study period, of which 125 (74.4%) had an IPT published in Spain by 20th June 2023. The proportion of drugs with an IPT was similar for orphan and non-orphan drugs (75.4% vs. 73.8%), and higher in oncology (84.8%). One in five (20.9%) products without an IPT were reimbursed versus 82.4% of products with an IPT. A sample of 89 (71.2%) IPTs was available in IQVIA’s HTA Accelerator, of which 79.8% were from reimbursed products. On average, 1.9 positive and 2.1 negative aspects were mentioned in the IPTs of reimbursed products versus 1.4 and 2.4, respectively, on IPTs of non-reimbursed products. Increased risk of adverse events was the most stated negative aspect of the drug, present in 11.7% of the analyzed IPTs (and in 18.2% of non-reimbursed products). Superiority versus placebo in the primary endpoint was the most common positive aspect (in 28.8% of reimbursed products’ IPTs vs. 10.0% of those non-reimbursed). CONCLUSIONS: Incorporating the evidence needs of evaluators in the study designs may support reimbursement decisions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23carmo-hpr28posterv4133115-pdf.pdf?sfvrsn=e858e7d6_0","title":"ISPOREurope23_Carmo_ HPR28_POSTER_v4133115.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133115","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Literature Review (SLR) of the Clinical Efficacy and Safety Evidence Associated with Treatment of Patients with Higher-Risk Myelodysplastic Syndrome (HR-MDS)","id":"f3021fc3-9ec9-4551-9ce1-9e7c6152090d","sessionCode":"CO25","topDisplay":"Papadakis-Sali A1, Bobrowska A2, Shaw S2, Radford M3, Sabate Estrella EJ3, Asukai Y4 1Gilead Sciences, Uxbridge, UK, 2Costello Medical, Cambridge, UK, 3Gilead Sciences, Foster City, CA, USA, 4Gilead Sciences, London, LON, UK","locationCode":"1013","description":"\r\n\t<div>OBJECTIVES: HR-MDS is associated with poor survival outcomes due to cytopenias and progression to acute myeloid leukemia (AML). This SLR aimed to identify clinical efficacy and safety evidence associated with HR-MDS treatments. METHODS: MEDLINE, Embase, Cochrane Databases and the Database of Abstracts of Reviews of Effects were searched in October 2022, supplemented by hand-searches of grey literature and SLR bibliographies. Eligible articles reported interventional or observational studies of pharmacological management of HR-MDS. Study quality was assessed using the ROBINS-I and University of York's Centre for Reviews and Dissemination checklists. RESULTS: Of 8,821 records retrieved, 59 unique studies were included. Most assessed hypomethylating agents (HMA) monotherapies (n=34, 23 assessing azacitidine), and HMA+venetoclax (n=10, 5 assessing azacitidine+venetoclax). Median overall survival (OS) (n=17) ranged from 7.3 (best supportive care [BSC], which includes antibiotics, erythropoietin treatment, and transfusions) to 28.2 months (azacitidine+venetoclax). The only statistical improvement in median OS between treatments was azacitidine compared with BSC (16.9 versus 7.3 months, p=0.039, n=1). Median progression-free survival (n=4) ranged from 1.05 (azacitidine) to 17.5 months (azacitidine+venetoclax). Objective response rate (n=23) ranged from 0% (BSC) to 93.3% (decitabine+low-dose cytarabine). Median time to AML progression (n=5) ranged from 2 (BSC) to 19.3 months (HMA), with decitabine significantly delaying time to progression compared with BSC (p=0.03). 33.3–50% of patients became transfusion independent following azacitidine treatment, compared with 25–46.5% following azacitidine+venetoclax and 58% following magrolimab+azacitidine (n=5). 0% (intensive chemotherapy and sabatolimab+HMA) to 14.4% (azacitidine) of patients discontinued treatment due to adverse events (n=5). CONCLUSIONS: Clinical and safety outcomes for patients are poor and there remains an unmet medical need. HMA monotherapy and combination therapies were associated with better outcomes compared with BSC and chemotherapy, with combinations demonstrating the greatest benefit. A quantitative treatment comparison would be valuable to capture the absolute and relative benefits of HR-MDS treatments.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23papadakis-salico25poster129782-pdf.pdf?sfvrsn=d8d399e_0","title":"ISPOREurope23_Papadakis-Sali_CO25_POSTER129782.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129782","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Costs of Proton Radiotherapy and Photon Radiotherapy for Non-Small Cell Lung Cancer in the Netherlands Using a Time-Driven Activity-Based Costing Approach","id":"d68ecac0-3c2e-496c-bcc0-9eca3e172e4e","sessionCode":"EE54","topDisplay":"Sugden B1, Witlox W1, Ramaekers B1, Joore M1, De Ruysscher D2 1Maastricht University Medical Centre+, Maastricht, LI, Netherlands, 2Maastro Clinic, Maastricht University Medical Centre+, Maastricht, Limburg, Netherlands","locationCode":"2018","description":"\r\n\t<div>OBJECTIVES: For non-small cell lung cancer (NSCLC), proton radiotherapy (PT) may provide superior clinical benefit compared with photon radiotherapy (XRT) but is also more costly. Information regarding costs (and comparisons thereof) of PT and XRT is limited, particularly from a societal perspective. Hence, this study aimed to perform a cost analysis of PT versus XRT for the treatment of NSCLC in a combined radiotherapy centre in the Netherlands. METHODS: The Dutch Cost Guide was followed to estimate costs from first referral to end of treatment for PT and XRT. Costs were calculated probabilistically using Monte Carlo simulations (10,000 iterations) and separated into three categories: healthcare costs, patient/family costs, and productivity losses. Direct healthcare personnel costs were estimated using a time-driven activity-based costing approach, allowing for planning costs to be incorporated independently from number of fractions (20 and 30 fraction schedules). The impact of taking a healthcare perspective and reduced PT fractionation time were explored in scenario analyses. RESULTS: Treatment planning and preparation costs per patient were €2,075 for PT and €1,985 for XRT. For 20 fraction schedules (societal perspective (healthcare perspective)), costs per fraction were €1,637 (€812) for PT and €687 (€126) for XRT giving total treatment costs of €34,821 (€18,308) for PT and €15,722 (€4,497) for XRT. For 30 fraction schedules, costs per fraction were €1,362 (€811) for PT and €499 (€125) for XRT giving total treatment costs of €42,923 (€18,297) for PT and €16,963 (€4,487) for XRT. The greatest incremental cost driver for PT versus XRT was non-personnel healthcare costs (equipment, buildings, overheads). Reducing PT fractionation time reduced cost disparities by 49-54%. CONCLUSIONS: Through a detailed micro-costing approach, this cost analysis highlights cost disparities between PT and XRT treatment modalities and serves to inform future cost-effectiveness analyses. Scenario analyses reveal the potential for narrowing cost differences through reducing PT fractionation time. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023-poster-presentationsugdenmumc-1133306-pdf.pdf?sfvrsn=86e1678_0","title":"ISPOR 2023 Poster presentation_SUGDEN_MUMC (1)133306.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133306","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Defining Severity Category Thresholds to Guide Psoriasis Symptoms and Impacts Measure Score Interpretation Using Pooled Data from Three Phase 3 Trials in Plaque Psoriasis","id":"27b2ec16-7482-4a48-a782-9ede9bcb3aa3","sessionCode":"MSR52","topDisplay":"Augustin M1, Gottlieb AB2, Warham R3, Lambert J4, Hoepken B5, Warren RB6 1Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf (UKE), Hamburg, Germany, 2Department of Dermatology, The Icahn School of Medicine at Mount Sinai, New York, NY, USA, 3Veramed, London, UK; UCB Pharma, Slough, UK, 4UCB Pharma, Colombes, France, 5UCB Pharma, Monheim, Germany, 6Dermatology Centre, Northern Care Alliance NHS Foundation Trust, Manchester, UK; NIHR Manchester Biomedical Research Centre, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK","locationCode":"5045","description":"\r\n\t<div>OBJECTIVES: Plaque psoriasis can substantially reduce patients’ health-related quality of life. The 14-item Psoriasis Symptoms and Impacts Measure (P-SIM) is a novel, well-designed, fit-for-purpose patient-reported outcome tool which captures key symptoms and life impacts of psoriasis. This analysis aimed to estimate severity category thresholds (meaningful score regions) for a subset of seven P-SIM items identified as representative of core symptoms experienced by patients with psoriasis. METHODS: Data were pooled across all treatment arms from the BE VIVID (NCT03370133), BE SURE (NCT03412747) and BE READY (NCT03410992) phase 3 trials. P-SIM items were scored daily over 16 weeks on a numeric rating scale from 0 (no symptom) to 10 (very severe symptom) and averaged weekly. Seven P-SIM items representative of core symptoms (itching, skin pain, scaling, redness, burning, cracking, dryness) were included for severity threshold analysis. To determine severity thresholds, best cut points, corresponding with maximum Youden index values from receiver operating characteristic curves using Dermatology Life Quality Index (DLQI) Item 1 (skin symptoms) as an anchor, were used. Random sampling of baseline and Week 16 data (observed case) was used, based on availability of both P‑SIM and DLQI Item 1 data at the respective timepoint; for each included patient, only one of the two timepoints was considered. RESULTS: Data from 1,345 (baseline: N=717; Week 16: N=628) patients were included in the analysis. P-SIM item score severity thresholds corresponding to ‘none or minimal’/’mild’/’moderate’/’severe’ symptoms were: itching, 0–1.2/>1.2–4.3/>4.3–6.2/>6.2–10; skin pain, 0–1.2/>1.2–3.4/>3.4–6.2/>6.2–10; scaling, 0–1.8/>1.8–4.6/>4.6–6.2/>6.2–10; redness, 0–1.2/>1.2–4.6/>4.6–6.9/>6.9–10; burning, 0–1.2/>1.2–3.4/>3.4–6.1/>6.1–10; cracking, 0–1.1/>1.1–3.3/>3.3–5.4/>5.4–10; dryness, 0–1.5/>1.5–5.0/>5.0–6.9/>6.9–10. CONCLUSIONS: These severity thresholds, with assigned verbal descriptors, could support with the interpretation of P‑SIM item scores and assessment of treatment efficacy in patients with psoriasis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23lambertmsr52poster130862-pdf.pdf?sfvrsn=908c03_0","title":"ISPOREurope23_Lambert_MSR52_POSTER130862.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130862","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Impact of Leber’s Heredity Optic Neuropathy (LHON) on Quality of Life (QoL): A Systematic Literature Review (SLR)","id":"8c9c9a80-e57b-4ee3-bdf2-9ee570f455e8","sessionCode":"PCR37","topDisplay":"Mumford A1, Mumford J2, Lewis H2, Gibson J2, Coyle S2 1Initiate Consultancy, Northampton, UK, 2Initiate Consultancy, London, London, UK","locationCode":"6039","description":"\r\n\t<div>OBJECTIVES: LHON is a rare, autosomal dominant, genetic disorder which causes severe vision loss or blindness, with an average onset between 10-30 years. The most common symptoms of LHON are vision loss and reduced visual acuity, often in both eyes. There is currently no cure, although some treatments are able to slow or halt the progression of the disease. This systematic literature review (SLR) was conducted to gather further insight into how QoL is measured in LOHN, and the impacts of LOHN on patients’ QoL. METHODS: Embase, Medline Econlit, and EBMR (Cochrane) databases were searched on 6th June 2023 for unrestricted literature, using standard quality of life and utility search strings. RESULTS: 30 papers were identified from the initial search. Following abstract and full paper review, r9 papers concerning 5 studies were included: 1 study regarding the potential therapy Idebenone and 2 studies regarding lenadogene nolparvovec, a gene therapy. The papers identified a variety of disease-related QoL burdens, including impacts on daily living, assistance, interpersonal relationships, career/finances, mental health/wellbeing. Management of risk factors that are considered as triggers to disease (smoking/alcohol intake) were also highlighted. Impacts on QoL were not limited to those with sight loss, extending additionally to families and asymptomatic carriers. It was also found that patients who were newly diagnosed, or diagnosed later in life, suffered the greatest QoL burden. The QoL measures reported were VF-14, visual function questionnaire-25 (VFQ-25), energy levels evaluated via self-reported VAS scale, and depression scores using the Beck Depression Inventory (BDI-I). Each, although established QoL measures, lack specificity to LHON. CONCLUSIONS: There is limited published literature relating to LHON and QoL, and no QoL measure specifically tailored to LHON. The impact LHON has on QoL reaches across all aspects of daily life, emphasising the importance of developing new therapies in this area.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/qol-in-lhon-v0-1132362-pdf.pdf?sfvrsn=e43eb23e_0","title":"QoL in LHON v0.1132362.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132362","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"What Do the Students Think About Food Safety Knowledge at the University of Pécs?","id":"cbad5e76-2357-4994-92b7-9f7fb79e8b5d","sessionCode":"EPH54","topDisplay":"Keczeli V1, Gubicskóné Kisbenedek A1, Kóró M2, Tisza B1, Czeglédiné Asztalos Á3, Tóth V1, Szántóri P3, Boncz I1, Verzár Z1 1University of Pécs, Pécs, Hungary, 2Institute of Human Nutrition and Dietetics, University of Pécs, Pécs, Hungary, 3University of Pécs, Institute of Human Nutrition and Dietetics, Pécs, Hungary","locationCode":"3003","description":"\r\n\t<div>OBJECTIVES: Foodborne illnesses representing various medical conditions, which are occurred by the consumption of food or water contaminated with bacteria and/or their toxins, parasites, viruses or chemicals. The foodborne diseases are still dominating globally. The aim of the study is to evaluate the food safety awareness of the students of the University of Pécs by exploring their level of theoretical knowledge. METHODS: 459 students of the University of Pécs participated a cross sectional study between 26/11/2022 and 10/12/2022. The online, anonymous questionnaire consisted of 3 parts: sociodemographic data, food handling practices and a sample of questions about the-oretical knowledge in food safety. SPSS 27.0 Statistics software was used to process the data using descriptive statistics, T-test, ANOVA, Kruskal-Wallis tests (p≤ 0.05). RESULTS: There was a significant difference among the current education and food hygiene knowledge (p=0.017), that participants with higher educational level seems to have more awareness with food safety. Students with “older age” seems to be more sensible for food hygiene rules p<0.01, not only the age but also the educational attainment (p=0.002) or the different social status (p=0.001) seems to be playing a crucial role in food safety awareness, as students, who have a student job have usually higher levels of food hygiene knowledge. Those who have children, indicating a better awareness (p=0.002). The results show that this theoretical knowledge stay theoretical: even they know, that they have to do, they don’t care about it in the practice (p=0.001). CONCLUSIONS: Current study focused on undergraduate students, with the aim to use conclusions for developing targeted educational materials in order to reduce the incidence and complications of foodborne illness. The result of current study is for great interest to change inappropriate beliefs and inadequate practices connected to food habits, so has its importance regarding health promotion and primer prevention.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23keczelieph54poster133801-pdf.pdf?sfvrsn=f54efd0d_0","title":"ISPOREurope23_Keczeli_EPH54_POSTER133801.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133801","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Obesity on Health-Related Quality of Life, Sleep, and Work – a Multinational Survey","id":"f50afb56-54d4-492b-a032-9fe471e8adf1","sessionCode":"PCR4","topDisplay":"Khare S1, Redig J2, Higgins V3, Leith A4, Mortimer A3, Tahbaz A5, Artime E6 1Eli Lilly and Company, Woking, SRY, UK, 2Eli Lilly and Company, Stockholm, Sweden, 3Adelphi Real World, Bollington, UK, 4Adelphi Real World, Bollington, CHE, UK, 5Eli Lilly and Company, Bracknell, UK, 6Eli Lilly and Company, ALCOBENDAS - MADRID , M, Spain","locationCode":"6010","description":"\r\n\t<div>OBJECTIVES: To describe the impact of obesity on health-related quality of life (HRQoL), sleep and work delineated by BMI and the presence of comorbidities. METHODS: Data were drawn from the Adelphi Obesity Disease Specific Programme™, a cross-sectional survey of physicians and the people with obesity (PwO) whom they manage on a weight management programme or using anti-obesity medications, conducted in Brazil, Canada, China, Japan, Saudi Arabia and the United Arab Emirates from April–December 2022. Physicians reported demographics and clinical characteristics for their consecutive 3 to 8 qualifying PwO. PwO were then invited to complete a questionnaire containing SF-36v2.0 Health Survey (scores range from 0 - 100, with higher scores indicating better HRQoL), Jenkins Sleep Evaluation Questionnaire (JSEQ; where 0 indicated no sleep problems to 20 indicating most sleep problems) and Work Productivity and Activity Impairment Questionnaire (higher scores indicating greater impairment and less productivity). PwO who completed all 3 patient-reported outcome measures were then grouped by current BMI (kg/m2) i) <30/<28 in China/<25 in Japan [B0] and ii) ≥30/≥28 in China/≥25 in Japan [B1], and by presence of comorbidities i) none [C0] and ii) ≥1 [C1]. Analyses were descriptive. RESULTS: Of a total 1,434 PwO (n/%: 243/17% were B0 and 1191/83% B1; 262/18% were C0 and 1172/82% C1), mean±SD age was 40.5±12.5 years, 57% female, 71% employed. Mean±SD BMI at diagnosis was 34.2±8.4, current 32.2±7.6. Mean±SD SF-36v2.0 component summary scores: physical, 50.0±7.9 (B0), 46.8±8.4 (B1); 50.3±7.3 (C0), 46.7±8.5 (C1); mental, 44.9±9.1 (B0), 44.4±10.1 (B1); 46.4±8.7 (C0), 44.1±10.1 (C1). Mean±SD JSEQ scores: 4.9±4.3 (B0), 5.0±4.1 (B1); 4.3±3.9 (C0), 5.2±4.2 (C1)). Mean±SD overall work impairment scores: 61.5%±11.1 (B0), 65.7%±12.2 (B1); 61.7%±11.4 (C0), 65.8%±12.2 (C1). CONCLUSIONS: Our results suggest obesity and comorbidity presence have an impact on HRQoL and work. Future awareness of this when managing PwO could help optimise outcomes.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kharepcr4poster130896-pdf.pdf?sfvrsn=9c01bc0b_0","title":"ISPOREurope23_Khare_PCR4_POSTER130896.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130896","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"IDegAsp Versus Continuation of Prior Therapies in India: Long-Term Cost-Effectiveness Analyses Based on Arise","id":"545a9781-0301-48df-9bdd-9fec4ff48432","sessionCode":"EE21","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1060","description":"\r\n\t<div>OBJECTIVES: The real-world ARISE study has associated the coformulation of insulin degludec and insulin aspart (IDegAsp) with significant improvements in glycated haemoglobin (HbA1c) and body weight, factors associated with the incidence of long-term complications in type 2 diabetes, compared with a mix of prior therapies. The present study aimed to evaluate the long-term cost-effectiveness of IDegAsp versus continuing prior therapies in India. METHODS: Clinical and cost outcomes were projected over patients’ lifetimes using the IQVIA Core Diabetes Model (v9.5+). Country-specific baseline characteristics and changes in HbA1c and body weight, as well as overall hypoglycaemic event rates, were taken from ARISE. Costs were expressed from a societal perspective in India, accounted in 2021 Indian rupees (INR). Outcomes were annually discounted at 5%. Immediate IDegAsp treatment was evaluated versus a 3-year delay. RESULTS: In India, immediate IDegAsp was associated with life expectancy improvements of 0.05 years, and quality-adjusted life expectancy improvements of 0.12 quality-adjusted life years (QALYs), versus a 3-year delay. Combined costs in India, were estimated to INR 10,221 higher with immediate IDegAsp treatment compared with a 3-year delay, with higher treatment costs partially offset by cost savings from avoidance of diabetes-related complications and fewer days of lost productivity. IDegAsp was associated with incremental cost-effectiveness ratio of INR 87,206 per QALY gained in India compared with a 3-year delay. CONCLUSIONS: Based on willingness-to-pay threshold of INR 200,000 per QALY gained, immediate IDegAsp therapy is likely to represent a cost-effective treatment for type 2 diabetes in India.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeuropegarcia-urangaee21poster130613-pdf.pdf?sfvrsn=41f8fe17_0","title":"ISPOREurope_Garcia Uranga_EE21_POSTER130613.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130613","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Societal Burden of Non-Alcoholic Steatohepatitis (NASH) in Denmark - Real-World Evidence from National Registries","id":"80fae779-57f8-408c-9026-a0e12c5fda08","sessionCode":"EE17","topDisplay":"Rudolfsen JH1, Gluud LL2, Grønbæk H3, Jensen M4, Vyberg M5, Olsen J1, Poulsen PB6, Hovelsø N6, Gregersen N6, Thomsen AB6, Jepsen P3 1EY, Frederiksberg, Denmark, 2Copenhagen University Hospital Hvidovre, Copenhagen, Denmark, 3Aarhus University Hospital, Aarhus, Aarhus, Denmark, 4University of Copenhagen, Copenhagen, Denmark, 5Copenhagen University Hvidovre, Copenhagen, Denmark, 6Pfizer Denmark, Ballerup, Denmark","locationCode":"1065","description":"\r\n\t<div>OBJECTIVES: Non-alcoholic steatohepatitis (NASH) is associated with increased risk of cirrhosis, hepatocellular carcinoma, cardiovascular disease, and type 2 diabetes, but evidence on both healthcare and societal burden of NASH is lacking. The present study examined healthcare and societal costs related to patients with non-alcoholic fatty liver disease (NAFLD), including NASH. METHODS: We used real-world data from the comprehensive nationwide Danish registries to identify all patients with a hospital diagnosis and biopsy-confirmed NAFLD (≥18 years) from 1997–2021. Patients were classified as having non-cirrhotic NASH (with or without fibrosis), simple steatosis or cirrhosis. All patient groups were matched 1:5 with liver-disease free reference groups. Healthcare and homecare costs, production loss, sick leave, unemployment and early retirement were investigated 5 years before diagnosis until 11 years after, i.e. 16 years. Healthcare and societal excess costs due to NASH/NAFLD were calculated as the difference between patients and the matching reference group. RESULTS: 3,712 NAFLD patients were identified (NASH=1,030; simple steatosis=1,540 and cirrhosis=1,142). Highest average total healthcare costs were found in the year leading up to diagnosis: cirrhosis €12,949 (6.2-fold higher than reference group), NASH € 6,318 (4.1-fold higher) and simple steatosis € 4,716 (3.1-fold higher). For cirrhosis patients, inpatient contacts accounted for 68% of the excess costs, whereas outpatient contacts accounted for 49% of NASH patients’ costs. Excess costs of NASH in the years after diagnosis were around 70% of excess costs in the year leading up to diagnosis. NASH patients’ income was statistically significantly lower than the reference groups’ income. Higher risk of early retirement due to disability was also found (HR: 4.37; 95% CI: 3.17–6.02). CONCLUSIONS: The burden on society caused by NASH/NAFLD was considerable, even for the early stages of the disease. Moreover, patients experienced adverse effects with reduced incomes as well as early retirement due to disability.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee17---ispor-nash-poster-18oct2023130240-pdf.pdf?sfvrsn=9212876_0","title":"EE17 - ISPOR NASH Poster 18Oct2023130240.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130240","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing Cancer Related Premature Mortality and Productivity Loss in Lung, Melanoma, and Breast Cancers in European Regions: Temporal Trends in 2010-2019","id":"93ebdc4b-7934-4855-b8c8-a11349b76efa","sessionCode":"EPH26","topDisplay":"Brandtmuller A1, Oliver E2, Weston G3, Bencina G4, Hughes R5 1MSD Pharma Hungary Ltd, Budapest, Hungary, 2Adelphi Values PROVE, Bollington, Cheshire, UK, 3Adelphi Values PROVE, Bollington, UK, 4MSD, Center for Observational and Real World Evidence (CORE), Zagreb, 01, Croatia, 5Adelphi Values PROVE, Bollington, CHE, UK","locationCode":"3026","description":"\r\n\t<div>OBJECTIVES: This study estimated the mortality burden and the cost of lost productivity due to premature deaths in melanoma, lung, and breast cancers in Central-Eastern Europe (CEE), Northern, Western, and Southern Europe (SE). We estimated the years of productive life lost (YPLL) due to premature mortality and the present value of lost productivity (PVFLP) associated with these cancer types between 2010-2019. METHODS: The human capital approach was used to estimate YPLL due to premature deaths from lung, melanoma, and breast cancers (ICD-10 code: C33-34, C43, and C50, respectively). PVFLP was calculated using age-specific mortality, wages, and employment rates in European countries. Data was sourced from the WHO, Eurostat, and the World Bank. RESULTS: The number of deaths due to melanoma, lung and breast cancer remained relatively stable in the European regions, with some increase in breast cancer (CEE: 19%), and melanoma deaths (CEE: 16%, SE: 13%) comparing 2019 to 2010. YPLL/death due to lung cancer and melanoma decreased in 2019 versus 2010 across all European regions (25%-42% decrease in lung cancer, and 30%-37% decrease in melanoma). YPLL/death in breast cancer decreased by 18%-21%. PVFLP decreased in all European regions for each cancer, in a range of 25%-37% for melanoma (SE-CEE), 22%-42% for lung (SE-CEE), and 11%-21% in breast cancer (SE-CEE). In Europe, the decrease in PVFLP in 2019 compared to 2010 was €2,995M for lung, €295M for melanoma, and €466M for breast cancer, with an overall reduction of productivity cost of €3,756M in these cancer types. CONCLUSIONS: Our study illustrates a trend toward lower productivity costs from 2010-2019. This finding is likely driven by deaths occurring at an older age that can be effect of aging population, but also suggesting that advances in cancer prevention and the treatment landscape have extended the life of cancer patients, yielding less productivity loss.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/eph26---assessing-cancer-related-premature-mortality-and-productivity-loss-in-lung-melanoma-and-breast-cancers-in-european-regions-temporal-trends-in-2010-2019132281-pdf.pdf?sfvrsn=3ed8ce9b_0","title":"EPH26 - Assessing Cancer Related Premature Mortality and Productivity Loss in Lung Melanoma and Breast Cancers in European regions Temporal Trends in 2010-2019132281.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132281","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Originator and Generic Medicines in Armenian Pharmacy Outlets","id":"2d4b6834-482c-437f-a90e-a17d70f419a9","sessionCode":"HPR19","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: In Armenia the great majority of patients purchases most of medicines out of pocket and has to select among products which have very different price. The objective of this study was evaluating the prices of medicines available for outpatients in pharmacy outlets in Armenia. METHODS: Data on availability and patient prices for 63 tracer medicines were collected from 30 pharmacy outlets in different regions of Armenia. For each tracer medicine (with indicated specific dosage form and strength) all the products authorized in Armenia were included in the Survey form. RESULTS: Almost all (n=62) the selected tracer medicines were available on the pharmaceutical market. Originator brands (OB) for 10 and generics for 62 of 63 tracer medicines were available in at least two pharmacy outlets. For 3 medicines (Ceftriaxone, Fluconazole and Amlodipine) the originator brand and more than ten generics were available on the pharmaceutical market. In all the cases when OBs were available, they were higher priced than lowest priced generics (LPGs). Using matched medicine pairs (OBs:LPGs ratio for 10 medicines), the price of OBs were on average 6.5 times the price of LPGs. For one medicine (Ceftriaxone) OB was more than ten times more expensive than LPG. For two medicines (Fluconazole and Amoxicillin / Clavulanic Acid) highest priced generics (HPGs) were more expensive than OBs. For Fluconazole HPG was ten times more expensive than LPG. CONCLUSIONS: Lowest priced generic equivalents are much more available than originator brands. Originator brands are more expensive than the lowest priced generics, however in some cases are less expensive than the highest priced generics. Changes in national reimbursement policy and regulation are necessary. Recommendations are drafted. As community pharmacists have to provide detailed information about all the equivalent products available at pharmacy outlets, continuing professional education of pharmacists on national regulation could decrease spending on medicines.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133431","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"97c2e725-3c74-4625-997f-a72378c5a557","parentId":"00000000-0000-0000-0000-000000000000","title":"Generics","urlName":"Generics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Are Digital Health Therapeutics Reshaping the HTA Framework for Health Technologies?","id":"82dc666a-a500-4882-bcdc-a1d2ec33d2ef","sessionCode":"HTA33","topDisplay":"Tutulea E1, Barchanska M2, Perez-Kempner L3 1Parexel International, Bucharest, B, Romania, 2Parexel International, Krakow, Poland, 3Parexel International, Lebrija, SE, Spain","locationCode":"4071","description":"\r\n\t<div>OBJECTIVES: With the emergence of digital health therapeutics (DHTs) there has been a growing awareness of the impact of the patient's experience on health technologies, reflected in new health technology assessment (HTA) criteria. This research aims to gain insights into how HTA authorities consider patient-related insights for drugs vs. DHTs and whether the new HTA framework for DHTs will impact the assessment of drugs moving forward. METHODS: A total of 9 innovative drugs approved via accelerated pathway in Europe between 2018 and 2022 were identified from the European Medicines Agency database. HTA evaluations for the selected drugs were retrieved from HTA agencies in 3 countries (i.e., UK, France and Germany). Subsequently, DHTs reimbursed in these countries up to 2022 were identified, and their HTA evaluations were analysed. Narratives on patient-related insights, including Patient Related Outcomes (PROs), for both drugs and DHTs were analysed. RESULTS: Overall, 10/20 HTA reports for innovative drugs included patient groups/experts consulted during the appraisal and 16/20 HTAs included Quality of Life (QoL)/PRO data. In the rationale for the decision, a total of 17/20 HTAs included QoL or other patient-centric claims, including 13 QoL arguments and 9 other patient-centric arguments. Conversely, all HTA evaluations for digital therapeutics (42/42) included assessment of patient-related insights, including a mix of DHT functionality (41/42), good data practices (41/42), PRO/QoL (34/42), health literacy (6/42), improvement of productivity and daily functioning (4/42). CONCLUSIONS: While QoL/PRO remain important outcomes in the assessment of novel technologies, DHTs are shifting the HTA paradigm leading to the incorporation of new patient-related insights that place patients’ ability to use and understand technologies in direct relationship with clinical outcomes at the centre of the HTA. With the expansion of the DHTs, the HTA framework will continue to evolve, possibly reshaping how traditional drugs are being assessed.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/hta33ispor-eupatient-centric-draft-posterfinal132049-pdf.pdf?sfvrsn=2fa3aa43_0","title":"HTA33_ISPOR EU_Patient-centric draft poster_FINAL132049.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132049","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Budget Impact Model for the Swiss Healthcare System: Assessing Potential Budget Savings of an Adapted Fall-2023-COVID-19 Vaccination Strategy","id":"72126c84-5dc8-4330-9576-a24b6110b89b","sessionCode":"EE141","topDisplay":"Certejan T1, Joshi K2, Wahler S3, Beck E2 1Moderna, Inc., Basel, BS, Switzerland, 2Moderna, Inc., Cambridge, MA, USA, 3St. Bernward GmbH, Hamburg, Germany","locationCode":"2064","description":"\r\n\t<div>OBJECTIVES: The COVID-19 pandemic has caused significant challenges to healthcare systems worldwide, also for Switzerland. Vaccination campaigns have been pivotal in curbing the spread of the disease, and booster vaccinations have emerged as potentially enhancing protection. This study aims to evaluate the budget impact of implementing an adapted Fall-2023-COVID-19 vaccination strategy for individuals aged 65+ years, and immunocompromised individuals aged 18+ in the Swiss healthcare system and assess its potential cost savings. METHODS: A budget impact model was developed to estimate the financial implications of introducing an adapted Moderna mRNA Fall-2023-COVID-19 vaccination strategy compared to the absence of such a strategy. The model incorporated data on clinical vaccine effectiveness, disease burden, healthcare resource utilization, and costs (outpatient, hospitalizations, death, long COVID). Cost per vaccine dose was assumed to be 80 CHF. The analysis was conducted from the perspective of the Swiss healthcare system over a one-year time horizon. RESULTS: The budget impact model revealed that a vaccination strategy with adapted Moderna Fall-2023 COVID-19 versus a no vaccination strategy would avoid 65,059 symptomatic infections. Furthermore, 5,574 hospitalizations, including 518 ICU cases with necessary ventilation and 1,048 deaths could be prevented. The overall costs for Covid-19 care could be estimated with CHF 1,264m without a program and with vaccination program CHF 1,221m, a cost reduction by 3.4%. Main areas of savings were reduced short term hospitalization cost with CHF 124m and total post-infection cost, including long covid, with CHF 39m. The 65–74-year-old population cohort showed the highest savings at CHF 36m. CONCLUSIONS: Vaccinating the Swiss population aged 65+ years and immunocompromised individuals aged 18+ years with the Fall-2023-COVID-19 Moderna vaccine versus no vaccination campaign will prevent hospitalizations, ICU stays, long COVID, and deaths as well as associated treatment costs. The overall health care budget spending can be reduced by such a vaccination strategy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/swissbudgetimpactmodelposterisporeu2023132856-pdf.pdf?sfvrsn=9ac47656_0","title":"Swiss_Budget_Impact_Model_Poster_ISPOR_EU_2023132856.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132856","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Effectiveness of the Non-Face-to-Face Comprehensive Elderly Care Application “Smart Silver Care” for Community-Dwelling Elderly: A Randomized Controlled Trial","id":"5fdb1fd6-dfb7-4486-aa7b-a36cca734c1d","sessionCode":"CO7","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1008","description":"\r\n\t<div>OBJECTIVES: This study aims to evaluate the effectiveness of the non-face- to-face comprehensive elderly care application “Smart Silver Care”. METHODS: This study was designed as a randomized controlled trial (RCT). Sixty community-dwelling elderly individuals were randomly assigned to experimental and control groups in a 1:1 ratio. Participants joined the “Smart Silver Care” intervention using a tablet and smartwatch for the various programs we provided. The participants performed five tasks five days a week, consisting of physical, emotional, and cognitive programs. Participants could communicate with the researchers in real-time from their homes, and the researchers could remotely supervise their performance. RESULTS: We found positive effects on relevant scales testing risk of falls (ABC: p=0.028; TUG, t=0.001). However, there was no time × group interaction between the experimental and control groups on the relevant scales testing depression and quality of life (SGDS-k: p=0.225; EQ-5D-5L, p=0.172) While the SGDS-K and EQ-5D-5L did not show statistical significance, we found improvement trends in the experimental group. CONCLUSIONS: The findings of this study show that Smart Silver Care significantly reduced the risk of falls in community-dwelling elderly, and a qualitative evaluation confirmed that it could be conveniently used by the elderly. Thus, “Smart Silver Care” offers a feasible intervention for improving the quality of life of the elderly, including physical aspects.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129870","diseases":[{"id":"dc752772-538c-4933-b6a5-3b5b6d079673","parentId":"00000000-0000-0000-0000-000000000000","title":"Geriatrics","urlName":"Geriatrics"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"When Should the Conduct of RWE Studies Be Prioritized for Reimbursement: Insights from Canadian and Global Stakeholders","id":"8d458aca-8ebf-4fa4-98bf-a438237e2b6b","sessionCode":"HTA32","topDisplay":"Boss J1, Shulak L2, Rao T2, Tam C1, Sullivan S3 1Evidera Evidence Synthesis, Modeling and Communication, London, LON, UK, 2Evidera Evidence Synthesis, Modeling and Communication, St-Laurent, QC, Canada, 3Evidera Evidence Synthesis, Modeling and Communication, Ivry-sur-Seine Cedex, Ile de France, France","locationCode":"4069","description":"\r\n\t<div>OBJECTIVES: Given the ongoing importance of RWE both in international and Canadian healthcare decision-making (such as influencing HTA reimbursement decisions), we set out to collect insights from stakeholders on when generating RWE should be prioritized. This builds on previous work where we identified CADTH reimbursement reviews from 2017-2021 that demonstrated the influence of RWE on recommendations. METHODS: To better understand the process for deciding when a RWE study should be prioritized, educational workshops were held providing examples of how RWE has previously been used in reimbursement decision-making. Stakeholders included industry partners, HTA decision-makers, payers, and patient advocates. As a subsequent step, qualitative data are being collected from stakeholders in 1:1 or small-group sessions to obtain insights on RWE experiences both in Canada and other global markets. Analyses of RWE in CADTH reimbursement reviews were also updated with 2022 data. RESULTS: Background information on the RWE environment and previous findings of our research were shared with stakeholders. Perspectives are being obtained on the following topics: (1) what factors have influenced decisions to conduct RWE studies in the past, (2) when does RWE generated in other countries influence this decision, (3) when does including a RWE study optimize impact on reimbursement. Approximately 28% of initial CADTH submissions included RWE in 2022; similar trends were observed as in previous work for top therapeutic areas and use in rare diseases. CONCLUSIONS: Given the recent publication of frameworks by global HTA/regulatory agencies that focus on RWE methodology, there is a need to further support practical implementation of RWE. A framework that prioritizes when to generate RWE and that could influence decision-making would provide value and could be tailored to different countries’ RWE environment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bosshta32poster129949-pdf.pdf?sfvrsn=4368cac1_0","title":"ISPOREurope23_Boss_HTA32_POSTER129949.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129949","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Review on the Safety and Effectiveness of the Active Middle Ear Implant in the Treatment of Conductive and Mixed Hearing Loss","id":"ba8d8445-6f60-44f9-b979-a50e339cceb1","sessionCode":"SA11","topDisplay":"Park E1, Han HR1, Shin C2, Lee W2 1National Evidence-based Healthcare Collaborating Agency, Seoul, Korea, Republic of (South), 2National Evidence-based healthcare Collaborating Agency, Seoul, Korea, Republic of (South)","locationCode":"7019","description":"\r\n\t<div>OBJECTIVES: An active middle ear implant (aMEI) was evaluated by the New Health Technology Assessment Committee in Korea in 2012 as a safe and effective technique for sensorineural hearing loss. However, for conductive hearing loss (CHL) and mixed hearing loss (MHL) it was determined a technique requiring further research due to insufficiency evidence. This systematic review aimed to evaluate safety and effectiveness of aMEI in CHL and MHL patients who do not expected achieve sufficient hearing gain with conventional treatment options. METHODS: Ovid-MEDLINE, Ovid-EMBASE, Cochrane library and five Korean databases were searched on 27 January 2022. The risk of bias was assessed using the SIGN methodology checklist. The entire process was conducted in collaboration with otolaryngology experts. RESULTS: Of the total 1,270 retrieved articles, 14 articles were selected. Three cohort studies compared aMEI to bone conduction hearing implants (BCHI) and twelve case-crossover studies compared aMEI with cHA in the same patient. The hearing thresholds in the range of 500 to 8000 Hz were significantly lower aMEI than cHA. The meta-analysis result based on five studies showed a significantly greater functional gain in aMEI compared to BCHI or cHA (Mean difference = 9.57 dB, 95% CI = 8.35, 10.78, I2 = 0%). In four studies, speech discrimination was significantly better with aMEI compared to cHA. Patient satisfaction was higher with aMEI compared to cHA, but no differences with BCHI. Adverse events such as device dislocation, mild dizziness, wound infection, and facial palsy, were reported. However, these cases were rare and fully recovered with treatment. Additionally, there was no post-operative residual hearing loss. CONCLUSIONS: aMEI has been proven to be a safe and effective option for hearing rehabilitation in CHL and MHL patients who do not achieve sufficient hearing gain with conventional treatments or unable to use cHA due to anatomical limitations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/-sa11-2023-ispor-europeeunji-parkamei128615-pdf.pdf?sfvrsn=5d01c7df_0","title":"[SA11] 2023 ISPOR Europe_Eunji Park_aMEI128615.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128615","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Intracardiac Echocardiography Operational Experience of Chinese Interventional Cardiologists in Conducting Radio-Frequency Catheter Ablation for Cardiac Arrhythmias: A Cross-Sectional Survey Study","id":"7ae24668-93bb-4912-b7d4-a56bd0ec914d","sessionCode":"MT3","topDisplay":"Pu X1, Chen S1, Zeng R1, Hu H1, Kang Y1, Fu H1, Chen W2 1West China Hospital of Sichuan University, Chengdu, Sichuan, China, 2Xiangya Hospital of Central South University, Mississauga, ON, Canada","locationCode":"5028","description":"\r\n\t<div>OBJECTIVES: To understand the use of Intracardiac echocardiography (ICE)-related confidence, values, and satisfaction of Chinese interventional cardiologists (IC). METHODS: From August 2022 to October 2022, 100 IC with experience in using ICE to guide radio-frequency catheter ablation (RFCA) from hospitals across China participated in this survey regarding their experience with ICE (confidence, values, and satisfaction). The collected survey information was summarized by descriptive statistical methods. Multiple logistic and generalized linear regression analyses were conducted to explore the influential factors on recognized values and satisfaction of Chinese IC. RESULTS: The included IC had an average age of 40.9 years and mainly worked in Tier-I (35.0%) and Tier-II cities (63.0%). Sixty-one IC had conducted more than 100 ICE-guided RFCA procedures. Compared to no ICE-guidance RFCA, IC who proceeded ICE-guided RFCA have significantly higher confidence scores in performing transseptal puncture (9.6 vs. 8.0, p<0.001) and cardiac tamponade monitoring (9.6 vs. 7.0, p<0.001). The widely recognized values of ICE among IC included detecting thrombus in the left atrium preoperatively (92.0%), optimizing transseptal puncture process (91.0%), reducing the risk of intraoperative complications (91.0%), reducing or eliminating intraoperative radiation exposure (89.0%), and avoiding transesophageal echocardiography (85.0%). Included IC was generally satisfied with using ICE to guide RFCA (4.3 out of 5 scores). The cumulative number of performed ICE-guided RFCA (≥100 vs. <100) was significantly correlated to the improved satisfaction score on the operation convenience of ICE (coefficient 0.101, p=0.025) and recognized ICE values for reducing or eliminating radiation exposure [odds ratio (OR) 5.113, p= 0.039] and saving ablation time (OR 3.794, p=0.048). CONCLUSIONS: Chinese IC report that ICE-guided RFCA can significantly improve confidence in conducting transseptal puncture and cardiac tamponade monitoring. The values of ICE-guided RFCA are widely recognized and the Chinese IC with more ICE experience are likely to have higher satisfaction.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23chenmt3poster131376-pdf.pdf?sfvrsn=b201199e_0","title":"ISPOREurope23_Chen_MT3_POSTER131376.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131376","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Effectiveness of Romosozumab Followed by Alendronate Versus Teriparatide in the Treatment of Severe Osteoporosis in Greece","id":"20558a70-21a8-4a39-8b98-a5834fab05d3","sessionCode":"EE41","topDisplay":"Golnas P1, Relakis J2, Kountouris V2, Golna C1, Makras P3, Willems D4, Souliotis K5 1Health Policy Institute, Maroussi, Attika, Greece, 2UCB Pharma, Athens, Attika, Greece, 3251 Hellenic Air Force & VA General Hospital, Athens, Attika, Greece, 4UCB Pharma, Brussels, Belgium, 5University of Peloponnese, Corinth, Peloponnese, Greece","locationCode":"1086","description":"\r\n\t<div>OBJECTIVES: Romosozumab, a monoclonal antibody for osteoporosis treatment, inhibits sclerostin, increasing bone formation and decreasing bone resorption. We evaluated the incremental cost-effectiveness of treatment with romosozumab for 12 months (mo) followed by alendronate for 12 mo compared with teriparatide for 24 mo in patients with severe osteoporosis [T-score <-2.5 and fragility fracture(s)] in Greece. METHODS: A Markov model developed in Microsoft Excel was localized to the Greek setting from the third-party payer (EOPYY) perspective. Clinical inputs were derived from the ARCH trial, a systematic literature review, and a network meta-analysis. Cost inputs were obtained from Greek public sources. The model included five health states: at risk of fracture, hip fracture, vertebral fracture, non-hip-non-vertebral fracture, and dead. All patients entered the model in the at risk of fracture state and were followed for a lifetime horizon to account for short and long-term cost and health effect consequences. Both quality-adjusted-life-years (QALY) and costs were discounted at an annual rate of 3.5%. Cost-effectiveness was reported in terms of costs per QALY gained, in 2023 Euros (€). RESULTS: Compared with teriparatide, romosozumab followed by alendronate yielded 0.054 additional QALYs and 0.015 additional life-years. Number of patients needed to treat (NNT) to avoid one hip fracture in a lifetime horizon was 31 for romosozumab + alendronate versus 86 for teriparatide. Fracture-related medical costs were lower in the romosozumab + alendronate arm (-€298). Total incremental cost was €1,956 and cost per QALY was €36,222, which is below the perceived willingness-to-pay threshold of approximately 3 times the per capita GDP of Greece (€51,219). Model robustness and base case assumptions were validated with univariate and probabilistic sensitivity analyses. CONCLUSIONS: According to our analysis, treatment with romosozumab (12 mo) followed by alendronate (12 mo) was cost-effective compared to teriparatide (24mo) in postmenopausal women with severe osteoporosis in Greece.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23souliotisee41poster131433-pdf.pdf?sfvrsn=26ac2957_0","title":"ISPOREurope23_Souliotis_EE41_POSTER131433.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131433","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Drug Utilization and Evaluation of Antibiotics in Pre and Post Operative Obstetrics Ward at a Tertiary Care Hospital","id":"4c0efb27-b2f0-409c-a4de-a61fba7462e4","sessionCode":"SA3","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"7012","description":"\r\n\t<div>OBJECTIVES: The study was to evaluate the drug utilization pattern of antibiotics used in the Obstetrics patients in pre and post-operative at the tertiary care hospital. The objective was to assess the Drug Utilization and evaluation of Antibiotics using DDD (Defined Daily Dose) and to evaluate Prescribing pattern by Anatomical Therapeutic Classification (ATC) and to determine WHO prescribing indicator. The study highlights on the number of antibiotics prescribed in different type of surgeries and explains the relation of diet and education status of the participants. METHODS: A prospective study find of 120 prescriptions of women attending the inpatient Department of Obstetrics and Gynecology, with 160 bedded unit of Geetanjali Hospital, Udaipur which is 1200 bedded hospital which supply tertiary health care for the population of Udaipur district and close by areas during the length of February’22 to July’22 was conducted. RESULTS: The average no. of antibiotics used per prescription was 2.93. The most commonly prescribed antibiotic was a combination of Ceftriaxone and Sulbactam (302 gms), whose DDD was found to be 151 gms. And on further evaluation DDD/100 HOSPITALIZED DAYS was found to be 83.88 gms followed by Ceftriaxone 56.5 grams and 31.38 grams, whereas, Cefuroxime axetil was used in the minimum amount i.e., 1 gram whose DDD was found to be 0.5 gram and DDD/100 BED DAYS was 1.11 gram respectively.. Along with antibiotics, antihistamines, narcotics, PPIs, multivitamins, anti-HTN, antithyroid, antiplatelet and benzodiazepines were prescribed. CONCLUSIONS: Third generation cephalosporins, i.e., ceftriaxone was the most frequently prescribed antibiotics and every antibiotic given was of brand name. All the antibiotics which were prescribed during the study that were according to the essential drugs list. All though the present study provides valuable insight about the overall pattern of drug use profile in pre and post-operative patients in the obstetrics unit.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129257","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Effectiveness of Implantable Cardioverter Defibrillator Therapy Versus Drug Therapy in Patients for 1.5 Primary Prevention in China: An Analysis Using the Improve SCA Study","id":"80855179-b307-41f5-b942-a6712ce2c521","sessionCode":"EE10","topDisplay":"Sun H1, Fu J2, Song Y3 1Fudan University, Shanghai, Shanghai, China, 2Medtronic (Shanghai) Management Co., Ltd., Shanghai, China, 3Medtronic Greater China, Beijing, China","locationCode":"1056","description":"\r\n\t<div>OBJECTIVES: The Improve SCA study has identified a cohort of patients called 1.5 primary prevention (1.5PP) based on PP population with the presence of certain risk factors, and the results showed a 49% relative risk reduction in all-cause mortality among those ICD implanted 1.5PP patients. In this study, we assessed the cost-effectiveness of ICD compared to drug therapy among 1.5PP patients from the Chinese payer perspective. METHODS: A Markov model, using a 1-month cycle over a lifetime horizon, was developed to describe different health states of patients after treatment with ICD and drug therapy. The disease-specific clinical inputs and utilities were obtained from the Improve SCA study and current literature. The costs were based on the tender price published by government and the results of cost surveys of fifteen local clinicians from different demographic parts of China. For each therapy, we modelled the total costs and quality-adjusted life-years (QALYs), and then calculated incremental the cost-effectiveness ratio (ICER). According to the WHO recommendation, therapies were evaluated with the willingness-to-pay (WTP) threshold of $37,260 (3 times China’s GDP per capita) per QALY gained. RESULTS: In the base case of lifetime horizon, the total discounted costs and QALYs for ICD therapy were $68,313 and 7.92 QALYs, whereas drug therapy was associated with the cost of $31,258 and 6.09 QALYs. The ICER was $20,239/QALY, below 2 times China’s GDP per capita. Therefore, ICD therapy is a cost-effective strategy compared to drug therapy for the 1.5PP patients. This result was validated by sensitivity analysis, in which ICD therapy remained the cost-effective option. CONCLUSIONS: Compared to drug therapy, ICD therapy is cost-effective in the 1.5PP population in China.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-icd-1-5pp-poster--1027-final132068-pdf.pdf?sfvrsn=cb8d917f_0","title":"ISPOR-ICD-1.5PP-Poster -1027 final132068.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132068","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Use of Care Pathway Analysis to Inform Health Economic Models and Evidence Generation Requirements: Practical Applications and Examples from Two Projects","id":"b26e8b7f-7287-40e0-8221-a70f8b9a5e35","sessionCode":"SA8","topDisplay":"Gregg E, Sanderson A, Graziadio S York Health Economics Consortium, York, UK","locationCode":"7017","description":"\r\n\t<div>OBJECTIVES: The care pathway is the journey that a patient with a specific medical condition takes during an episode of healthcare. Care pathway analysis (CPA) is a methodology that facilitates the identification and mapping (using a flow diagram) of medical decisions within the current pathway for a certain condition. The objective of this work is to raise awareness about CPA and situations where this methodology is useful in health economics and outcomes research. METHODS: Initially, CPA involves a pragmatic review to identify and synthesise national and international guidelines that describe the care pathway for the condition of interest. This is followed by a qualitative evaluation using semi-structured interviews and thematic analysis. Interviews with experts (clinicians and/or other relevant stakeholders) are undertaken to understand where (and why) real-world practices differ from the published guidance. These are used to validate the care pathway and to capture variations in clinical practice among clinicians/countries. RESULTS: CPA can be used to optimise the structure of economic models and inform evidence generation requirements. Practical applications from two CPA projects will be presented: the first focussed on a new treatment for cataracts and the second on adult-onset Still’s disease. For diagnostics and devices, CPA is useful in the early stages of product development. In this situation, CPA can help identify the added value of the new technology and its optimal positioning within the care pathway, alongside potential barriers/facilitators for adoption. In drug evaluations, CPA is more useful in situations where there is a lack of robust data. For example, in rare diseases where clinical guidelines are often limited, and care pathways can be characterised by high variability. CONCLUSIONS: CPA can support the development of economic models and is particularly useful in certain situations; for example, for new diagnostics and devices and in drug evaluations for rare diseases.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23graziadiosa8poster131990-pdf.pdf?sfvrsn=cbc52ffa_0","title":"ISPOREurope23_Graziadio__SA8_POSTER131990.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131990","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Deprescribing of Proton Pump Inhibitors in Elderly Patients: A Cost-Effectiveness Analysis","id":"8461f69c-32c5-4923-9353-a73aff26d71a","sessionCode":"EE26","topDisplay":"Xie M, You J Chinese University of Hong Kong, Hong Kong, N.T., China","locationCode":"1071","description":"\r\n\t<div>OBJECTIVES: Proton pump inhibitors (PPIs) are indicated for treating gastroesophageal reflux disease, a chronic gastrointestinal disorder commonly occurred in the elderly population. However, overutilization of PPIs increases the healthcare expenditure and risk of adverse drug events in elderly patients. Interventions for deprescribing inappropriate use of PPIs were reported to achieve high success rates. This study aimed to examine the cost-effectiveness of pharmacist-led deprescribing of PPIs in the elderly population from the perspective of public healthcare provider in Hong Kong. METHODS: A decision tree analytical model was designed to simulate clinical and economic outcomes of pharmacist-led deprescribing of PPIs and standard care in elderly patients aged 65 and above. The model time horizon was 1 year. Model parameters were retrieved from published literature and public data. Primary outcomes were direct medical costs and quality-adjusted life-years (QALYs) loss. Base-case analysis and sensitivity analyses were performed. RESULTS: In the base-case analysis, the pharmacist-led deprescribing saved 0.0249 QALYs and reduced direct medical cost by HKD 1,835 (USD 1=HKD 7.8) when compared to the standard care. In one-way sensitivity analysis, the base-case results were robust to variation of all model parameters. In probabilistic sensitivity analysis, QALYs and cost saved by pharmacist-led deprescribing were 0.0288 (95% CI: 0.0286 – 0.0290, p <0.01) and HKD1,969 (95%CI: 1,964 – 1,974, p <0.01), respectively. The pharmacist-led deprescribing was found to save QALYs at lower costs in 100% of 10,000 Monte Carlo simulations. CONCLUSIONS: Pharmacist-led deprescribing of PPIs appeared to save both QALYs and cost, with a high probability to be accepted as the cost-effective option for deprescribing inappropriate PPI usage in the elderly, from the perspective of public healthcare provider in Hong Kong.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130330","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"EQ-5D Utilities for Patients With Nonalcoholic Steatohepatitis (NASH): A Comparison of Historical Utilities to a Recent Real-World Multi-Country Patient Survey","id":"42c7623e-e887-46c1-bf4b-a7e0d3bb9663","sessionCode":"PCR3","topDisplay":"Fishman J1, Higgins V2, Piercy J2, Pike J2 1Madrigal Pharmaceuticals, Inc., Smyrna, GA, USA, 2Adelphi Real World, Bollington, UK","locationCode":"6011","description":"\r\n\t<div>OBJECTIVES: To describe EQ-5D utilities previously reported in the literature and compare them to real-world EQ-5D utilities for NASH patients from a multi-country patient survey. METHODS: A structured literature review (SLR) of EQ-5D utilities in NASH patients was conducted. Mean EQ-5D utilities from these studies were compared to data from the Adelphi NASH Disease Specific Programme (DSP)™, a real-world, cross-sectional survey of patients with NASH in France, Germany, Italy, Spain, UK, and USA conducted in Q1 2018 and Q1 2019, respectively. After applying matched inclusion/exclusion criteria to the DSP population, matching-adjusted indirect comparison analysis was used to balance DSP data with each of the SLR studies on age, sex, comorbidities, and fibrosis stage. EQ-5D utilities were compared using t-tests. Analysis was repeated using relevant country-specific EQ-5D scoring tariffs. RESULTS: Ten studies with varied recruitment criteria, patient demographics and clinical characteristics were identified in the SLR. Reported mean (SD) utilities ranged from 0.67 (SD not reported) to 0.83 (0.14). Utilities from studies requiring biopsy-confirmed NASH diagnosis ranged from 0.70 (SD not reported) to 0.83 (0.14). Utilities from studies not requiring biopsy-confirmed diagnosis ranged from 0.67 (SD not reported) to 0.83 (0.21). In balanced comparisons, DSP mean utilities ranged from 0.63 (0.18) to 0.91 (0.14) and were often higher or lower (p<0.05) than corresponding SLR utilities. The statistical significance and difference range (from -0.20 to 0.19) were sometimes dependent on the country-specific scoring tariff. CONCLUSIONS: There is a range of utilities reported both in the literature and balanced DSP data. These differences may be due to inclusion/exclusion criteria, sampling methodology or the country-specific EQ-5D tariff. Therefore, these factors warrant consideration when making cost-effectiveness decisions based on estimated EQ-5D utilities, highlighting the importance of country-specific EQ-5D tariffs and of conducting sensitivity analysis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23piercypcr3poster132129-pdf.pdf?sfvrsn=bc071b73_0","title":"ISPOREurope23_Piercy_PCR3_POSTER132129.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132129","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Using Foundationone Liquid CDx in Patients With Advanced Non-Small Cell Lung Cancer in Whom Tissue-Based Testing Is Not Feasible","id":"2cf329bf-dee0-44e3-9d73-a878bb38fe09","sessionCode":"EE71","topDisplay":"Isla D1, Alvarez-Alvarez R2, Arnal M3, Arriola E4, Azkarate A5, Azkona E6, García-Campelo R7, Garrido P8, Nadal E9, Ortega AL10, Carcedo D11, Villacampa A11, Lozano V12, Córcoles F12, Lavara J12, Bernabé R13 1Hospital Clínico Universitario Lozano Blesa, Zaragoza, Spain, 2Hospital Universitario Gregorio Marañón, Madrid, Spain, 3Hospital Provincial de Castellón, Castellón, Spain, 4Hospital del Mar, Barcelona, Spain, 5Hospital Universitario Son Espases, Palma de Mallorca, Spain, 6Hospital Universitario de Cruces, Bilbao, Spain, 7Hospital Universitario A Coruña, A Coruña, Spain, 8Hospital Universitario Ramón y Cajal, Madrid, Spain, 9ICO - Institut Català d'Oncologia (Duran i Reynals), Hospitalet De Llobregat, Spain, 10Hospital Universitario de Jaen, Jaen, Spain, 11Hygeia Consulting, Madrid, M, Spain, 12Roche Farma S.A, Madrid, Spain, 13Hospital Universitario Virgen del Rocío, Sevilla, Spain","locationCode":"2020","description":"\r\n\t<div>OBJECTIVES: To assess the cost-effectiveness of using FoundationOne Liquid CDx (F1LCDx) in liquid biopsy compared to no-molecular diagnosis (no-mDx) among patients with advanced non-small cell lung cancer (NSCLC) where a tissue diagnosis is not possible, from the perspective of the Spanish National Health System. METHODS: A joint model (decision-tree and partitioned-survival models) was developed to compare both costs and effects of performing a molecular diagnostic from a liquid biopsy with F1LCDx versus no-mDx over a lifetime horizon, so a 3% discount rate was applied for both cost and health outcomes. Treatment and utilities data were obtained from the literature and survival curves were extrapolated using exponential models. Only direct costs were considered (€2023). All the assumptions and inputs used in the model were validated by a panel of 11 oncologists from different Spanish centers. To assess uncertainty, several sensitivity analyses were performed. RESULTS: In a hypothetical cohort of 1,000 patients with advanced NSCLC in whom tissue-based testing cannot be performed, if F1LCDx is used in liquid biopsy 392 oncogenic biomarker alterations would be detected, 166 patients could receive reimbursed targeted therapies and 53 patients could be enrolled in clinical trials. No alterations are found if no-mDx is done. Over the lifetime horizon, using F1LCDx in the 1,000-patients cohort resulted in a gain in quality-adjusted life-years (QALYs) of 360.54 and in 449.48 life years (LYs), while it involved an incremental cost of 13,237,529€. Therefore, the resulting ratios were 36,716€ per QALY gained and 29,451€ per LY gained. Sensitivity analyses were consistent with the base case results. CONCLUSIONS: In patients where it is not possible to obtain a tissue sample, molecular diagnosis is not available at initiation of treatment. Our analysis shows that using F1LCDx in these patients is a cost-effective strategy in Spain.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23villacampaee71poster133197-pdf.pdf?sfvrsn=156c6343_0","title":"ISPOREurope23_Villacampa_EE71_POSTER133197.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133197","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Immunoglobulin Use in France 2015 to 2022–Real-World Data From the French Claims Database","id":"6815a549-a0a2-4123-8705-a8b2868fca22","sessionCode":"RWD36","topDisplay":"Darlington M1, Paulin L1, Ngaleu Siaha BF1, Arnaud NO2, Durand Zaleski I3 1DRCI-URC Eco Ile-de-France (AP-HP), Assistance Publique-Hôpitaux de Paris, Paris, 75, France, 2DRCI-URC Eco Ile-de-France (AP-HP), Assistance Publique-Hôpitaux de Paris, paris, France, 3Professor of Public Health Medicine and Doctor of Economics. Director of URCEco, Paris, Ile-de-France, France","locationCode":"7005","description":"\r\n\t<div>OBJECTIVES: Europe faces shortages in the supply of life saving medicines such as immunoglobulins (Igs) and this can worsen in times of crisis such as Covid-19. To understand the full picture of Ig use in Europe, exhaustive national data at a patient level is required. This research explores France as an example using its centralised national claims database (SNDS). METHODS: An exhaustive list of the Ig products used in France from 2015 to 2022 was established. An algorithm to extract hospital and ambulatory claims information from the SNDS was developed for all patients delivered Igs. The diagnosis (ICD10), diagnostic related group (for hospitalisations), dosage, volume (kg) and type of immunoglobulin (IV/SC) were extracted for each patient-prescription. The data was aggregated by family of disease according to the European Medicines Agency guidelines. RESULTS: This is an exploratory study and first results show that between 2015 and 2019, the number of patients administered Ig increased in France on average by 4% per year but descended by 6% in 2020. A decreasing trend was observed in administration in hospitals compensated in part by an increase of usage in the ambulatory setting. CONCLUSIONS: Accessing Ig dispensation data is cumbersome and impossible in many countries due to a lack of centralised databases and data protection laws. The French SNDS database is possibly unique in Europe in collecting and making available pseudonymised health information collected by public bodies. Certain organisations with a public service mission, listed by decree, have permanent access to SNDS data. Other countries could benefit from the French experience for detailed evidence based analysis of Ig usage.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23darlingtonrwd36poster-v1132066-pdf.pdf?sfvrsn=929e2ca6_0","title":"ISPOREurope23_DARLINGTON_RWD36_Poster v1132066.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132066","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Changes in Prescribing of Systemic Cancer Therapies in England: A Nationwide Analysis of Community and Hospital Medicines Utilisation","id":"edd2beb9-4985-4694-92cc-a97688c861d7","sessionCode":"EPH24","topDisplay":"Selle Arocha L1, Beattie A2, Mitchell G2, Robinson DE2, Pearson-Stuttard J1, Bray BD2 1Health Analytics, Lane Clark & Peacock LLP, London, LON, UK, 2Health Analytics, Lane Clark & Peacock LLP, London, UK","locationCode":"3028","description":"\r\n\t<div>OBJECTIVES: Oncology is one of the fastest moving areas of clinical practice, with 89 new oncology therapies receiving both EU and US marketing authorisation from 2010-2019. We aimed to use recently available open national data sources to describe the changes in utilisation of systemic anticancer therapies in England between 2019 and 2022. METHODS: We used data between January 2019 and December 2022 from two national datasets in England: The Secondary Care Medicines Dataset containing processed pharmacy stock control data from all hospitals in England, and the English Prescribing Dataset containing information on prescriptions issued by general practitioners. Systemic anticancer therapy (SACT) treatments were classified based on their administration route, mechanism of action and inferred indication (breast, prostate, lung, colorectal and melanoma). Utilisation patterns and trends were described as population adjusted rates (total quantity per person/month). RESULTS: During this time period we identified ~1.1 billion prescription items for SACT. Average monthly prescription rates were 24.5 for breast, 13.4 for lung, 9.0 for colorectal, 3.8 for prostate and 0.8 for melanoma per 100 people. There was a small (16%) drop in rates between 2019 and May 2020, corresponding to the early phase of the COVID19 pandemic but monthly rates subsequently rose to 46.4 per 100 people by December 2022 (30% higher than the 2019 average). Whilst cytotoxic agents remained the most used SACT in hospitals, between 2019 and December 2022 antibody-drug conjugates saw the highest percentage increase (219%) followed by hormonal agents (96%). All cancer types studied showed an increase in prescription rates after the pandemic. CONCLUSIONS: Newly available open data sources make it possible to track trends in oncology medicines utilisation in England with a high degree of granularity and short data lag. Antibody drug conjugates are now the fastest growing type of SACT according to mechanism of action.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sellearochaeph24poster132133-pdf.pdf?sfvrsn=d17d54ca_0","title":"ISPOREurope23_SelleArocha_EPH24_POSTER132133.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132133","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Examining Guidance and Key Principles for Conducting Living Systematic Reviews: A Methods Review","id":"775063ce-03f5-427c-b834-aa2542e53361","sessionCode":"MSR28","topDisplay":"Diamond M1, Valbuena-Fajardo J2, Appiah K2, Rizzo M3 1Cytel, Leiden, ZH, Netherlands, 2Cytel, Rotterdam, Netherlands, 3Cytel, London, London, UK","locationCode":"5071","description":"\r\n\t<div>OBJECTIVES: Living health technology assessment (HTA) and living systematic reviews (LSR) have gained increasing interest since the coronavirus disease 2019 pandemic. Given the rise in use of such methodology, this study aimed to identify best practice guidance and any associated challenges in conducting LSRs. METHODS: A comprehensive literature review was conducted in January 2023 in Embase and MEDLINE (via Ovid). The websites of key organisations were also searched to identify papers reporting on best practice recommendations on LSR methodology. Studies were selected by a single reviewer. Included studies were extracted in a piloted template with data collected on each step of the LSR process, as well as any user experience on perceived challenges. RESULTS: Common themes across the identified reports included the need for a protocol following existing reporting standards with adaptations to include pre-specification on which research questions require a living approach; the timing and frequency of updates; and adaptations to the analytic techniques and reporting to accommodate frequent updates. Searches were recommended to be broad and adaptive to consider changes in relevant interventions. Searches were recommended to occur following the release of publications from key congresses. Themes for optimisation of the LSR included online digital data extraction for automated presentation and visualisation of data. (Semi-) automation of tasks was consistently recommended to meet the timely demand of LSRs. Some challenges included resource implications of frequent updates, as well as re-thinking how to disseminate results as traditional methods such as peer-reviewed journals are less timely. CONCLUSIONS: The need for LSRs is evident, however, alignment on methodology and reporting standards is required. Prior to a consensus on standards for LSRs, stakeholders (academia, industry, guideline development groups and HTA agencies) must agree on the suitability of using (semi-) automated tools to realise and maintain the demand of LSRs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23diamondmsr28poster133573-pdf.pdf?sfvrsn=ceb48441_0","title":"ISPOREurope23_Diamond_MSR28_POSTER133573.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133573","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Diagnosis Patterns in Breast Cancer Women in Brazil: Inequities Among Public and Private Settings in São Paulo State","id":"cbefb64c-e4d4-4f65-917e-aa789ffef43b","sessionCode":"EPH23","topDisplay":"Julian G1, Santos HW1, Piton L2, Lazaridis E1 1Pfizer, São Paulo, São Paulo, Brazil, 2Pfizer Brasil LTDA, São Paulo, SP, Brazil","locationCode":"3027","description":"\r\n\t<div>OBJECTIVES: Breast cancer diagnosis and treatment evolved a lot in the last two decades, with an increased importance to screening programs and introduction of hallmark novel treatment strategies. In this context, it is crucial to evaluate diagnosis patterns in and clinical characteristics changes through time. In Brazil, inequities in public and private healthcare settings exist in both diagnosis and treatment, therefore, we aim to evaluate diagnosis patterns disparities among both healthcare perspectives in São Paulo state, Brazil METHODS: This is a descriptive analysis using real-world data from FOSP São Paulo state registry. We included all female breast cancer cases from 2000-2020 with healthcare coverage information available. We evaluated differences of demographic and clinical characteristics among public and private healthcare setting patients through the period RESULTS: In the study period, we identified 88,532 eligible cases of breast cancer, of which 71,051 (80.2%) were from public and 17,481(19.8%) from private perspective. Patients mean age at diagnosis were 56.0y±13.5, 56.2y±13.4 and 55.1y±13.5 for overall, public and private, respectively. The proportion of diagnosis at metastatic stage was larger in public (10.6%) when compared to private setting (4.3%), and this proportion almost doubled through decades in both perspectives, from 6.7% in 2000-2010 to 11.5% in 2011-2020 in the public setting, and 2.4% to 4.7% in the private setting CONCLUSIONS: Breast cancer inequities in diagnosis are huge among public and private settings in Brazil, which leads to important differences in the outcomes. In addition, an increase on the proportion of metastatic disease is notable in both settings, which reinforces the importance of awareness campaigns and screening.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023-breast-cancer-poster133149-pdf.pdf?sfvrsn=c026a3e1_0","title":"ISPOR 2023 Breast Cancer Poster133149.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133149","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of 20-Valent Pneumococcal Conjugate Vaccine in Singaporean Adults Aged ≥18 Years","id":"1f72b422-efda-4cf0-a878-acb0e7b33f3f","sessionCode":"EE138","topDisplay":"Averin A1, Oh SX2, Vietri J3, Atwood M1, Hariharan D1, Huang L4 1Policy Analysis Inc. (PAI), Boston, MA, USA, 2Pfizer Private Limited, Singapore, Singapore, 3Pfizer, Inc., Collegeville, PA, USA, 4Pfizer, Inc., Newtown, PA, USA","locationCode":"2068","description":"\r\n\t<div>OBJECTIVES: The Singapore Ministry of Health (MOH) recommends pneumococcal vaccination—13-valent pneumococcal conjugate vaccine (PCV13) or 23-valent pneumococcal polysaccharide vaccine (PPSV23)—in adults aged 18-64 years with chronic medical (“at-risk”; 1 dose PPSV23) or immunocompromising (“high-risk”) conditions (PCV13+PPSV23, revaccination with PPSV23 5 years later), and all adults aged ≥65 years (PCV13+PPSV23). With the recent licensure of 20-valent PCV (PCV20), we conducted a cost-effectiveness analysis (CEA). METHODS: A probabilistic cohort model was developed to project the lifetime risks and costs of invasive pneumococcal disease (IPD), all-cause nonbacteremic pneumonia (NBP), and the expected impact of vaccination. Clinical outcomes were projected on an annual basis based on current epidemiology and age/risk profile. Economic costs were estimated based on cases and corresponding medical costs (discounting, 3%/year). Costs of vaccine and administration were tallied at time of receipt. Cost per quality-adjusted life year (QALY) gained with PCV20 (vs. current recommendations) was assessed among at-risk adults aged 18-64 years, high-risk adults aged 18-64 years, and all adults aged ≥65 years, separately and collectively; a healthcare system perspective was used. RESULTS: PCV20 was estimated to reduce IPD cases by 42, NBP cases by 2,982, deaths by 207 in at-risk/high-risk population aged 18-64 years and all adults aged ≥65 years (N=1.6 million). Estimated overall net costs (including vaccination and medical costs) were lower by SGD 0.7 million, making PCV20 dominant versus current strategies. In the age- and risk-specific subgroups, replacing PCV13 with PCV20 was dominant among high-risk adults aged 18-64 years and all adults ≥65 years; among at-risk adults aged 18-64 years, cost/QALY was SGD 3,329. CONCLUSIONS: CEA suggests that use of PCV20—in lieu of current recommendations by the Singapore MOH among at-/high-risk adults aged 18-64 years and all adults aged 65-99 years—would yield overall cost savings and represent a cost-effective use of scarce healthcare resources.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-poster-cleancea-of-pcv20-among-sg-adultsfinal-png7857131295-pdf.pdf?sfvrsn=4031b76d_0","title":"ISPOR Poster (clean)_CEA of PCV20 Among SG Adults_Final (PNG7857)131295.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131295","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-of-Illness of Skin Cancer: A Systematic Literature Review","id":"6161c2a3-0bb7-4a09-b045-ad11d5132837","sessionCode":"SA6","topDisplay":"Meertens A1, Van Coile L1, Van Iseghem T2, Brochez L1, Verhaeghe N2, Hoorens I1 1Department of Dermatology, Ghent University Hospital, Ghent, Belgium, 2Department of Public Health and Primary Care, Interuniversity Centre for Health Economics Research (I-CHER), Ghent University, Ghent, Belgium","locationCode":"7015","description":"\r\n\t<div>OBJECTIVES: A systematic literature review was conducted to provide an overview of the methodological approaches applied in melanoma skin cancer (MSC) and non-melanoma skin cancer (NMSC) cost-of-illness (COI) studies and to identify the main cost drivers. METHODS: This review was conducted using the PRISMA guidelines. Pubmed, Embase and Web of Science were searched using search concepts related to skin cancer and COI. Three authors independently screened the records on eligibility criteria. A checklist for COI studies was used to assess the quality of the articles. RESULTS: A total of 40 studies were included in this review. The majority of the studies (n=31) only focused on MSC, a few (n=3) on NMSC and six studies examined both. The study’s perspective was described in 23 articles and only 5 studies reported the epidemiological approach, indicating variation in study quality. Direct costs were estimated in all studies, while indirect costs were only estimated in 7 studies. Included cost items differed across studies. For MSC both direct and indirect costs increased as the disease stages progressed. In advanced stage MSC, systemic therapy demonstrated to be the highest cost item (44-96% of costs), while diagnosis (34%), inpatient costs (26-37%) and outpatient costs (38-44%) were the predominant cost items in NMSC. Heterogeneity was observed in cost estimates across studies, mainly due to differences in study population characteristics and methodological approaches. CONCLUSIONS: This systematic review provides an overview of the methodological approaches in skin cancer COI studies. For advanced stage MSC, systemic therapy was the main cost drivers, indicating the need for effective treatment strategies and potential impact of targeted therapies on reducing the economic burden. For NMSC, the main cost drivers were diagnosis and in- and outpatient costs, indicating the importance of prevention and early detection. The heterogeneity in the included studies underscores the necessity for harmonized costing methodologies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-ispor-congres-annick-meertens129157-pdf.pdf?sfvrsn=a90cc734_0","title":"Poster ISPOR congres Annick Meertens129157.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129157","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Review and Analysis of Economic Model Characteristics According to Published HTA Conclusions in Ukraine","id":"3904c74d-a4e2-4ee8-b62c-ad55d5c26520","sessionCode":"HTA66","topDisplay":"Ioltukhovskyi I1, Khmelovska M1, Serediuk V2, Piniazhko O1, Babenko M3 1HTA Department of the State Expert Center of the Ministry of Health of Ukraine, Kyiv, Ukraine, 2HTA Department of the State Expert Center of the Ministry of Health of Ukraine, Kiev, Ukraine, 3State Expert Center of the Ministry of Health of Ukraine, Kyiv, Ukraine","locationCode":"5020","description":"\r\n\t<div>OBJECTIVES: Since 2021 and up to June 2023 there were 30 HTA conclusions published by the HTA Department of State Expert Center of the Ministry of Health of Ukraine. According to the methodologic recommendations and national legislation, an economic model is required to be presented in the company submission dossier for the HTA procedure. Therefore, the main objective is to define and analyse the most common economic model designs and characteristics such as type of pharmacoeconomic analysis, model types and sensitivity analysis types. METHODS: The review is aimed at nonconfidential data of HTA conclusions published by the HTA Department of “State Expert Center of the Ministry of Health of Ukraine”. RESULTS: Among 30 economic models, most conducted only cost-utility analysis (14) or both cost-effectiveness and cost-utility analyses (14). One (1) model conducted cost-minimization analysis and one (1) only cost-effectiveness analysis. The vast majority of economic models were designed as a Markov model (26). Two (2) models were designed as mixed decision tree / Markov models. There were three sensitivity analyses types conducted in economic models: only one-way SA (16), only multi-way SA (6), both one-way and probabilistic SA (4). CONCLUSIONS: The most common economic evaluation type that is used by applicants in Ukraine is cost-utility analysis. Nearly all of the models simulate Markov cohorts and most of them conducted one-way sensitivity analysis. A few pharmacoeconomic models were global and were adapted by the applicants due to local characteristics of the health system of Ukraine.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterispor2023hta-conclusionsoct30132930-pdf.pdf?sfvrsn=5ca70953_0","title":"Poster_ISPOR_2023_HTA conclusions_oct30132930.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132930","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Work and Activity Impairment in Individuals With Chronic Kidney Disease According to Renin-Angiotensin-Aldosterone System Inhibitor (RAASi) Use","id":"27b9d53b-293a-4944-8922-adc3ed9fd378","sessionCode":"PCR44","topDisplay":"Ofori-Asenso R1, Palmer E1, Chen T2, Khan I3, Rao N4, Fishbane S5, Kashihara N6, Kanda E6 1AstraZeneca, Cambridge, Cambridgeshire, UK, 2AstraZeneca, Gaithersburg, MD, USA, 3AstraZeneca, New York, NY, USA, 4AstraZeneca, Cambridge, CAM, UK, 5Zucker School of Medicine, Hempstead, NY, USA, 6Kawasaki Medical School, Kurashiki, Okayama, Japan","locationCode":"6043","description":"\r\n\t<div>OBJECTIVES: The use of RAASis in individuals with chronic kidney disease (CKD) has been associated with improved health outcomes, including delayed estimated glomerular filtration rate decline and lowered risk of cardiovascular complications. However, limited data exist describing the impact of RAASi use on productivity and activity of individuals living with CKD. In this study, we compared the work productivity and activity levels of patients in the DISCOVER CKD cohort with or without RAASi use at baseline. METHODS: DISCOVER CKD (NCT04034992) is a noninterventional cohort study characterising contemporary real-world management of CKD to provide insights into the current gaps in CKD treatment. The study includes both retrospective and prospective cohorts, integrating primary and secondary data collection. The prospective phase recruited patients from Sweden, USA, UK, Italy, Japan and Spain. The impact of disease on paid and unpaid work and daily activities of life was assessed using the Work Productivity and Activity Impairment CKD (WPAI-CKD) questionnaire, which employed a 7-day recall period. Domain scores were compared between individuals with and without RAASi treatment at baseline using an analysis of covariance model with adjustment for age, sex, country, CKD stage, comorbidity and number of other medications. P<0.05 was considered statistically significant. The study received ethics approval and all patients provided informed consent. RESULTS: In total, 236 RAASi users and 182 non-RAASi users completed the WPAI-CKD questionnaire. The results indicated that individuals receiving RAASi therapy experienced lower percentages of missed work time (difference: –5.5; P=0.017), work impairment (difference: –7.5; P=0.025), activity impairment (difference: –8.9; P<0.001) and overall work productivity loss (difference: –10.3; P=0.009) compared with patients not using RAASis. CONCLUSIONS: These results show an association between RAASi use and increased work productivity and activity levels in individuals with CKD, suggesting that working capability can be further improved through enhanced CKD management.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23ofori-asensopcr44poster133017-pdf.pdf?sfvrsn=c096590_0","title":"ISPOREurope23_Ofori-Asenso_PCR44_POSTER133017.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133017","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing Feasibility of Socially Assistive Humanoid Robots in Assisting in Nursing Routine","id":"78cf7cf9-9347-4894-8a4a-aec1d9d2dfee","sessionCode":"MT8","topDisplay":"Mlakar I1, Flis V1, Smrke U1, Roj IR2, Arioz U1, Šafran V1, Plohl N1 1University of Maribor, Maribor, Slovenia, 2University Medical Centre Maribor, Maribor, 070, Slovenia","locationCode":"5038","description":"\r\n\t<div>OBJECTIVES: There is an estimate an estimated global shortfall of 5.9 million nurses. Furthermore, the contribution of nurses in revolutionizing the way healthcare is delivered in hospitals is significant and often the digitalization represents a major source of stress. he objective of this study was to experimentally asses the feasibility of including socially assistive humanoid robots in nursing and care routine during hospitalization. METHODS: Socially assistive humanoid robotic units have a potential to significantly improve the landscape of nursing and care routine, for both nursing staff and patients. However, too often the staff must use a workaround to overcome the poor usability of an implementation of the system. Namely, oversimplification in design of functionalities, without nursing staff , leads to unhelpful features, creating inconveniences and frustrations. The study presented was carried out to understand if and how the implementation of socially assistive humanoid robot Frida, co-designed with nursing staff, could be integrated into nursing routine at University Clinical Center Maribor. We demonstrated functionalities in a live setting with 21 nurses and present video-recordings from the live demonstrations to further 82 nursers as an online survey. The participants were asked to evaluate acceptance of robot (adapted the Acceptability e-scale), trust in robot’s activities (the ECA trust questionnaire) and ethical acceptability of the solution (the EAS Scale). RESULTS: All measured constructs were generally positively associated, with most of the bivariate associations also being statistically significant (at least at the .05 level). There is positive association between acceptability, and aspects of trust, as well as ethical acceptability for use. CONCLUSIONS: Overall, the stakeholders perceive the solution as easy to use, useful and expressing benevolent and credible behavior. The stakeholders also believe that presented use in nursing and care and the simulated behavior of the robot are ethically acceptable.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporposter130790130790-pdf.pdf?sfvrsn=47bc95fb_0","title":"ISPOR_Poster_130790130790.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130790","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Prevalence of Polypharmacy in the Statutorily Insured German Population","id":"c370f2fd-b920-4dcc-b3b6-af5acbd2a00b","sessionCode":"EPH3","topDisplay":"Wilke K1, Junker S2, Hahn P1, Mueller S3, Fuchs A4 1IPAM e.V., University of Wismar, Wismar, MV, Germany, 2Cytel Inc., Berlin, Germany, 3Cytel Inc., Berlin, Berlin, Germany, 4AOK PLUS, Dresden, Saxony, Germany","locationCode":"3005","description":"\r\n\t<div>OBJECTIVES: Polypharmacy (concomitant use of multiple drugs) is common in older and multi-morbid patients and has been shown to increase the risk for drug-drug interactions, prescription of potentially inappropriate medication, and lacking adherence, finally leading to poor treatment efficacy. So far, little is known about the actual extent of polypharmacy in the elderly German population. Therefore, this study aimed to describe the prevalence and characteristics of patients experiencing polypharmacy in Germany. METHODS: The analysis was based on a subset (10%) of an anonymized German claims dataset representative of the statutorily insured German population concerning age and sex. (Excessive) polypharmacy was defined as the prescription of (≥10) ≥5 distinct drugs (excluding topical preparations/vaccinations/diagnostics/additives to intravenous solutions) in ≥2 quarters of 2019. The occurrence of polypharmacy, patient characteristics, and treatment use were assessed in 2019. Results were additionally reported for subgroups of patients aged ≥65 years and patients ≥65 years old diagnosed with cardiovascular disease (CVD). RESULTS: Out of 308,148 individuals analyzed, prevalence of (excessive) polypharmacy was found to be (1.5%) 12.7% in 2019. The mean age of the identified polypharmacy cases was 70.4 years (median: 73), and 54.8% were female; 78.0%/82.8% also indicated polypharmacy in 2018/2020. Among patients aged ≥65 years/and with CVD, rates of (excessive) polypharmacy were considerably higher, with (5.1%) 40.0%/(5.9%) 45.3%. On average, individuals experiencing polypharmacy used 10.1 distinct drugs (median: 9) and redeemed a mean of 32.0 prescriptions (median: 28) in 2019. The most commonly used medication classes were agents acting on the renin-angiotensin system (79.7% of patients), beta-blockers (67.0%), and drugs for acid-related disorders (52.2%). CONCLUSIONS: A substantial proportion of the insured German population is affected by polypharmacy, which is especially high in persons ≥65 years with cardiovascular comorbidities. In light of the significant burden of multi-medication, cases of polypharmacy should be monitored closely in clinical practice.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23junkereph3poster133429-pdf.pdf?sfvrsn=3c1c097f_0","title":"ISPOREurope23_Junker_EPH3_POSTER133429.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133429","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-Life Costs of Patients With Diffuse Large B-Cell Lymphoma (DLBCL) Treated by CAR T-Cells in France Between 2017 and 2020 (EpiCART Study)","id":"269b4c4a-4f58-43d5-9ec3-b037db30401a","sessionCode":"EE59","topDisplay":"Borget I1, Berthet M2, Grenier B3, Baccam E3, Zang A2, Lauvray P2, Cartron G4 1Gustave Roussy Cancer Centre and University Paris-Saclay, Villejuif, 92, France, 2Gilead Sciences, Boulogne-Billancourt, 75, France, 3HEVA, Lyon, 69, France, 4CHU Montpellier, Montpellier, Hérault, France","locationCode":"1098","description":"\r\n\t<div>OBJECTIVES: CAR-T-cell therapies showed efficacy for the treatment of various lymphoma subtypes, including DLBCL. The objective of the EpiCART study was to assess the costs of CAR-T-cell-treated DLBCL patients in France. METHODS: EpiCART is an observational retrospective study performed using the French Nationwide claims database (SNDS), which includes both community and hospital data. All adult patients treated with CAR-T-cells for DLBCL between January 1, 2018 and December 31, 2020 were included. Costs were evaluated in €2022 from the French National Health Insurance’s perspective from 2 months before to 1 year after CAR-T-cell infusion, excluding the cost of CAR-T-cells and using a Bang and Tsiatis model (to account for censored data). Costs of adverse events were defined as the costs of Intensive Care Units 1 year following the discharge post-CAR-T-cell infusion stay. RESULTS: Over the study period, 528 DLBCL patients received a CAR-T-cell treatment (axicabtagene ciloleucel or tisagenlecleucel). Median (IQR) age was 63.0 (16.2) years and patients were men (61%). Average duration of CAR-T stay was 25.3 (13.3) days, including 7.0 (4.1) days post-infusion. Mean cost of total treatment pathway was €90,957 per patient, including €12,400 in the 2 months prior to the CAR-T-cell stay, €31,494 during the CAR-T-cell stay, and €49,207 in the year following. Cost of adverse events occurring in 157 patients was €25,594 per patient on average. CONCLUSIONS: Results are consistent with available literature, with a mean cost per patient of €90,957 (excluding CAR-T-cell costs, but including all other patients' costs such as subsequent therapies and EoL costs), representing a total cost of care in line with other severe hemopathies. This must be put into perspective with the subsequent savings inherent to patients with an increased survival/complete remission compared to former standard of care. These results reflect French real-world practices and provide new information on healthcare consumptions outside of the CAR-T-cell stay.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-gilead-epicart-ispor-2023-v1r3-web132187-pdf.pdf?sfvrsn=19509a14_0","title":"POSTER-Gilead-EpiCART-ISPOR-2023-V1R3-WEB132187.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132187","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Characterization of Time-to-Treatment-Discontinuation (TTTD) as a Proxy for Progression-Free Survival (PFS) in Real-World Evidence (RWE) Based Analyses of Relapsed-Refractory Multiple Myeloma (RRMM)","id":"9b99bd13-0341-4128-b757-b05b8176acf8","sessionCode":"RWD18","topDisplay":"O'Reilly J1, Perkins A2, Tunaru F3, Wallis J3, Carpenter L3 1Arcturis Data, Oxford, OXF, UK, 2Arcturis Data, Kidlington, UK, 3Arcturis Data, Oxford, UK","locationCode":"6072","description":"\r\n\t<div>OBJECTIVES: RWE is playing an increasingly important role in assessing the comparative effectiveness and cost effectiveness of novel therapeutics in multiple myeloma (MM). The International Myeloma Working Group (IMWG) define disease progression based on longitudinal changes in laboratory markers, however many RWE databases that have been used as part of regulatory filings have relied on approximate measures of progression-free survival (PFS), such as Time to Treatment Discontinuation (TTTD), due to the lack of laboratory data. The Arcturis Data Platform contains comprehensive therapy and laboratory data, allowing for characterisation of the difference in real-world PFS (rwPFS) and TTTD. METHODS: This retrospective study used de-identified electronic health records for MM patients diagnosed between 2000 and 2023 from UK NHS partners collated as part of the Arcturis Data Platform. TTTD and rwPFS were used to calculate the delay between progression and treatment discontinuation for each patient. Time-heterogeneity of the accuracy of TTTD was investigated by obtaining distributions of TTTD percentage error stratified by quartiles of progression time. RESULTS: From a total of 6,749 MM patients, a cohort of 184 RWE RRMM patients treated with either daratumumab or pomalidomide was constructed that matched patients from the GEN501 and SIRIUS trials. The median delay between progression and TTTD is 22 days (-14, 91), with the percentage error of TTTD being greatest amongst patients with the shortest time to progression. This effect decreased in both magnitude and variance amongst patients with longer times to progression. CONCLUSIONS: TTTD has been used as a proxy for PFS in cost and comparative-effectiveness analyses as part of regulatory assessment. This study shows that in RRMM, the delay between TTTD and rwPFS can be large, and the relationship is not time-homogenous. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23oreillyrwd18poster132255-pdf.pdf?sfvrsn=9e143826_0","title":"ISPOREurope23_OREILLY_RWD18_POSTER132255.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132255","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing the Cost-Effectiveness of Introducing Benralizumab in the Treatment of Severe Uncontrolled Eosinophilic Asthma Patients in Prince Sultan Military Medical City (PSMMC) - Kingdom of Saudi Arabia (KSA)","id":"a270837c-ebcf-4fdd-9096-b06363e918db","sessionCode":"EE101","topDisplay":"Idrees M1, Aldossari H1, Abushal M1, Ahmed A1, Tarek N2, Alsaqaaby M3, Dhopte P4, Basu S5, Alharbi B1 1Prince Sultan Military Medical City, Riyadh, Saudi Arabia, 2IQVIA, Cairo, Egypt, 3IQVIA, Riyadh, 01, Saudi Arabia, 4IQVIA, New Delhi, Delhi, India, 5IQVIA-India, New Delhi, Delhi, India","locationCode":"2034","description":"\r\n\t<div>OBJECTIVES: Benralizumab is a humanized monoclonal antibody. It is indicated for adult patients with severe uncontrolled eosinophilic asthma despite baseline standard therapy. This study aims to assess the cost-effectiveness of introducing Benralizumab as an add-on therapy for the indicated patients in PSMMC. METHODS: A three-state excel based Markov model was adapted using 3 Phase III trials (ZONDA, SIROCCO and CALIMA) to assess the incremental cost-effectiveness ratio of Benralizumab vs. standard of care (SOC) alone and vs. Omalizumab. Health outcomes were estimated in terms of quality adjusted life years (QALYs) over a lifetime-horizon. Patients with severe uncontrolled asthma were defined as those on chronic oral corticosteroids (OCS) use or ≥3 exacerbations; baseline eosinophil count of ≥300 cells/µL in the previous year. Key model inputs included rates of exacerbations, treatment response rates, OCS-sparing, and cost-components. RESULTS: Benralizumab demonstrated a cost-effectiveness ratio of SAR 372,674 ($ 99,380) / QALY gain vs SOC, while it has shown to be a dominant option when compared to SOC in addition to Omalizumab, generating incremental 0.1 QALYs per patient at an incremental cost of SAR -268,551 ($ -71,614). Benralizumab was associated with a lower number of asthma exacerbations equals to 4.73 exacerbations Vs. SOC alone, generating an ICER of SAR 52,871 ($ 14,098) per exacerbation avoided. The increment in cost is mainly derived by total treatment costs which includes medications, administration, adverse events management and add-on costs like physician visits and Laboratory tests. The one-way sensitivity analysis showed that day-to-day utility was the most influential parameter when comparing Benralizumab to SOC alone and SOC plus Omalizumab. The cost-effectiveness acceptability curve illustrated a probability near 0% of Omalizumab being cost-effective when compared to Benralizumab. CONCLUSIONS: Benralizumab is a dominant option when compared to Omalizumab, generating higher QALYs per patient at lower costs over a life-time horizon.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/fasenra-ispor-postersubmission130360-pdf.pdf?sfvrsn=fa918590_0","title":"Fasenra ISPOR Poster_Submission130360.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130360","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Innovative Direct-to-Physician Retrospective Chart Review Approach: Synthesized Learnings from Completed and Ongoing","id":"bee5b682-e6a2-42a7-b324-b10904d0c036","sessionCode":"SA5","topDisplay":"Brett N1, Kent C2, Kiure A3, Capart P4, Rouleau A4 1PPD, part of Thermo Fisher Scientific, Saint Laurent, QC, Canada, 2PPD, part of Thermo Fisher Scientific, Miami, FL, USA, 3PPD, part of Thermo Fisher Scientific, Boston, MA, USA, 4PPD, part of Thermo Fisher Scientific, Paris, Île-de-France, France","locationCode":"7014","description":"\r\n\t<div>OBJECTIVES: Site-based medical chart review studies collect retrospective real-world evidence (RWE) when existing data sources are not available/relevant. Challenges include timescale inflexibilities, site burden and cost. A direct-to-physician chart review methodology may address these challenges. However, it is important to understand considerations and use-case scenarios for this approach. The objective was to describe recently undertaken direct-to-physician studies to inform researchers on appropriate use-cases, study design considerations, and share synthesized metrics and learnings. METHODS: A review of 4 US-based direct-to-physician studies (2 completed, 2 ongoing) was undertaken to evaluate objectives, population, outcome measures/variables, and key milestone metrics. RESULTS: Of the 4 historical cohort studies, 2 were natural history studies, 1 was a drug utilization study and 1 was a drug effectiveness/safety study. 1 study investigated patients with a rare pediatric disease and 3 investigated patients who failed treatment lines for oncology indications. Data collected included patient characteristics, treatment patterns, clinical outcomes, adverse events and healthcare resource utilization. Patient follow-up was ≤2-3 years. Studies received ethics approval ≤1 month of submission, and the 2 completed studies had high-quality efficient data collection timeframes (2-3 months), with low missingness of patient characteristics (< 5%), expectedly varied visit frequency among patients, and descriptive outcome analyses. Key considerations for direct-to-physician studies include sample size (≤approximately 150-200 patients), patients are treated by the physicians’ network, and variables are expected to be found in treating physician’s medical records. Direct-to-physician study limitations include, few countries where direct-to-physician studies can be conducted, limited breadth of therapeutic areas, and somewhat restricted chart abstraction time. CONCLUSIONS: In our experience, direct-to-physician retrospective chart review design was time-efficient for generating RWE to inform rare disease/condition management or to understand drug utilization and benefit-risk. When deciding whether to perform a direct-to-physician study, it is imperative to examine the methodologic considerations to ensure appropriateness of design in the selected country.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/brett-direct-to-physician-chart-review-ispor-eu-2023-posterfinal-30oct2023129629-pdf.pdf?sfvrsn=53c7ecbb_0","title":"Brett-Direct to Physician Chart Review ISPOR EU 2023 poster_FInal _30Oct2023129629.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129629","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Burden of Illness of Peripheral Arterial Disease (PAD): A Review of Reviews","id":"995d8eaa-9833-4fa4-813c-b19d9d42c723","sessionCode":"EPH2","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3008","description":"\r\n\t<div>OBJECTIVES: Peripheral arterial disease (PAD) is a common cluster of circulatory diseases characterized by narrowed arteries. Narrowed arteries reduce blood flow to the limbs, often leading to pain, functional impairment, and reduced quality of life. PAD is estimated to affect over 230 million people worldwide, indicating a significant global health concern. The objective of the review was to identify published reviews of studies reporting data on the burden of illness data of PAD. METHODS: A literature search of Pubmed was conducted on the 13th of June, 2023. The search was restricted to literature or systematic reviews published within ten years of the search date to include only current and relevant reviews. The search used terms considered both the study type and disease. Studies were included in the first stage by review of titles and abstracts. Studies deemed suitable for inclusion in the review were then assessed by review of the full-text article. RESULTS: The search yielded a total of 75 publications. Of these, 17 were included after the first stage review. A further eleven studies were excluded after the second stage review. The review identified five reviews of the cost-of-illness and one study of patient-reported outcomes in PAD. CONCLUSIONS: The burden of illness related to peripheral arterial disease (PAD) extends beyond direct healthcare costs, significantly affecting patients' quality of life and societal productivity. However, there is a conspicuous gap in the literature, with a limited number of comprehensive reviews evaluating the entire burden of illness associated with PAD. A deeper understanding of the burden associated with PAD will provide critical insights into the cost-effectiveness of various diagnostic and therapeutic strategies, helping to prioritize interventions that can deliver optimal health outcomes.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129739","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of CDK 4/6I for the Treatment of Postmenopausal Patients With Advanced Breast Cancer HR-Positive, HER2-Negative From the Point of View of the Payer of the Public Health System in Panama","id":"fc381488-e68b-40db-984a-b2e186514f01","sessionCode":"EE81","topDisplay":"Castillo O1, Sinta G2 1Instituto Oncologico Nacional, Panama , 8, Panama, 2Novartis, PANAMA, 8, Panama","locationCode":"2000","description":"\r\n\t<div>OBJECTIVES: The aim of this study is to estimate the cost-effectiveness of ribociclib + letrozole, Abemaciclib + letrozole or ANAS (Anastrozole) versus palbociclib + letrozole in the treatment of HR-positive, HER2-negative advanced breast cancer patients in the first line of treatment from the perspective of the National Cancer Institute of Panama (ION). METHODS: We developed a cost-effectiveness model studying the area under the curve that considers the health states of progression-free survival, progression and death. The time horizon was 20 years with monthly cycles, the average age of the cohort was 62 years and the discount rate for both effects and costs was 3% per-year. Efficacy of treatments was obtained from the MONALEESA-2, MONARCH-2, and PALOMA-2 clinical trial studies, through a Matching-adjusted indirect treatment comparison (MAIC). The identification, quantification and valuation of resources represented the Panamian purchase portal and the ION. Costs of drugs, management of the disease, and adverse events were considered. RESULTS: The total expected QALYs for ribociclib + letrozole was 4,121 QALY, on the other hand the total expected QALYs for Abemaciclib + letrozole was 3.891 QALY and for palbociclib + letrozole 3,688 QALY. Ribociclib + letrozole showed dominance over palbociclib + letrozole and Abemaciclib + letrozole. CONCLUSIONS: Ribociclib + letrozole reports an average incremental saving of US$73,498 and an average incremental gain of 0,433 QALY compared versus Palbociclib + letrozole, and an average incremental saving of US$45,425 and an average incremental gain of 0,14 QALY compared versus Abemaciclib + letrozole.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/abstract-cdk-4-6i-mbc-ispor-eurpoa-2023-final-002133173-pdf.pdf?sfvrsn=b27af22e_0","title":"Abstract CDK 4-6i mBC ISPOR Eurpoa 2023 final (002)133173.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/abstract-cost-effectiveness-model-in-panama133173-pdf.pdf?sfvrsn=c2f38d38_0","title":"Abstract Cost-effectiveness model in Panama133173.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133173","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Mapping Facilities, Equipment and Trained Personnel of Radiotherapy in Greece","id":"a85f94e6-b3a7-4961-9779-b41c6f2bf699","sessionCode":"MT5","topDisplay":"Vozikis A1, Pissakas G2, Kapetanakis G3, Apostolidis K4 1University of Piraeus, Piraeus, Greece, 2The Alexandra Hospital, ATHENS, A1, Greece, 3The All.Can Greece Initiative, Athens, Attica, Greece, 4The All.Can Greece Initiative, Brussels, VLI, Belgium","locationCode":"5033","description":"\r\n\t<div>OBJECTIVES: Radiotherapy demonstrates an important role in achieving high quality cancer treatment, either as monotherapy or in an adjuvant or neoadjuvant role. The present study provides a detailed and transparent mapping of the radiotherapy sector in Greece, both in terms of facilities and radiotherapy equipment, as well as in terms of staffing of the existing departments. METHODS: Updated data referring to the facilities, trained personnel and equipment of Radiotherapy in Greece were drawn from the National Organization for the Provision of Health Services (EOPYY), the Oncology Centres, from radiation oncologists and other experts in the field, were obtained on May 2023. RESULTS: According to the data provided to us, there are 57 radiotherapy units in operation in Greece, of which 32 units belong to the public sector and 25 to the private sector and are installed in 30 public and private hospitals. Regarding the human resources of the Radiotherapy Departments, 114 doctors, 63 Radiophysicists and 144 Technologists are employed. CONCLUSIONS: With a total of 57 radiotherapy machines available in the country, and a ratio of 0.54376 units per 100,000 inhabitants, Greece does not meet the recent recommendations of COCIR \"golden rule\" - target of 7 machines per million inhabitants. In terms of staffing and staff mix, the level is well below European standards and guidelines for both the private and public sectors. Understaffing is one of the reasons that while in the rest of Europe and the US radiotherapy is the main treatment for more than 60% of cancer patients, this percentage in Greece is estimated to be around 30%. As a result, the health care system in Greece is forced to pay for more expensive treatments (e.g. surgery and extensive chemotherapy), which in many cases are less effective.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23vozikismt5poster131943-pdf.pdf?sfvrsn=a305572c_0","title":"ISPOREurope23_Vozikis_MT5_POSTER131943.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131943","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Humanistic and Economic Burden of Illness in Progressive Fibrosing Idiopathic Lung Disease: A Targeted Literature Review","id":"6eebddc6-bd9f-46ff-94c7-b42cc840a0e7","sessionCode":"PCR34","topDisplay":"Jozsa IG1, Penaloza C2, Marjenberg Z3 1Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Mainz-Bingen, Germany, 2Boehringer Ingelheim International GmbH, Ingelheim, RP, Germany, 3Maverex Ltd, Newcastle-upon-Tyne, Tyne and Wear, UK","locationCode":"6036","description":"\r\n\t<div>OBJECTIVES: Progressive fibrosing lung diseases (PF-ILDs), including idiopathic pulmonary fibrosis (IPF), share common pathophysiologic characteristics and are associated with substantial morbidity and mortality. Antifibrotic (AF) therapy aims to manage symptoms and slow disease progression, thereby improving morbidity and life expectancy. However, uptake and adherence is variable. An understanding of the burden of PF-ILD in the era of AF therapy is required to inform payers of clinical unmet needs. This targeted literature review aims to identify the humanistic (health-related quality of life [HRQoL]) and economic burden of PF-ILD and IPF. METHODS: Electronic databases (Embase, MEDLINE, the Cochrane Library) were searched. Studies published in English between 2017–2022 evaluating HRQoL, healthcare resource use, or cost impact in adult patients with a diagnosis of IPF or PF-ILD and their caregivers were included. RESULTS: Twenty-five studies were included: fourteen reporting humanistic burden and eleven reporting economic burden, with the majority reporting data on IPF. IPF led to impairment in patient HRQoL as assessed by multiple disease-specific and generic patient-reported outcome instruments, particularly for mobility, activity, pain, anxiety, and depression domains. Increasing disease severity and comorbidities were associated with significant worsening of HRQoL, increased risk of hospitalization, and higher healthcare costs. Hospitalization rates ranged from 21.7–78.5%, with high levels of primary care, specialist care and medication resource use. For one-third of patients, ability to work was impaired. Delayed diagnosis and delayed initiation of AF treatment was associated with increased healthcare costs. CONCLUSIONS: IPF is associated with a substantial humanistic and economic burden driven by disease severity and comorbidities. Slowing disease progression with early AF treatment initiation whilst managing symptoms may reduce the overall burden of disease. Additional real-world evidence (RWE) assessing burden in treatment-naive versus AF-treated patients and further RWE in patients with non-IPF PF-ILD are required to inform treatment decisions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23jozsapcr34poster130883-pdf.pdf?sfvrsn=309f5908_0","title":"ISPOREurope23_Jozsa_PCR34_POSTER130883.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130883","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Benefits and Challenges of Different Qualitative Approaches in Rare Disease: A Case Study in Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) Using a Narrative Exercise and Qualitative Interviews","id":"dea652c9-940a-478b-a190-b437ebc026af","sessionCode":"PCR30","topDisplay":"Skrobanski H1, Matter E2, Acaster S1, Hitchcock I3, Whalen JD3, Tutein Nolthenius J3, Crocker-Buque A3, Harrington A4, Vandenberghe D3, Munro E3, Williams K1 1Acaster Lloyd Consulting Ltd, London, LON, UK, 2Acaster Lloyd Consulting Ltd, London, UK, 3Pharming Group N.V, Leiden, South Holland, Netherlands, 4Pharming Healthcare Inc, Warren, NJ, USA","locationCode":"6032","description":"\r\n\t<div>OBJECTIVES: Qualitative research allows for the in-depth exploration of the impact of disease from a patient or caregiver perspective. Data collection can be challenging in rare diseases due to limited knowledge of the disease and the small pool of potential participants. This study explored the benefits and challenges of using multiple approaches to qualitative data collection using a case study in activated phosphoinositide 3-kinase delta syndrome (APDS). METHODS: Two qualitative approaches were used to explore the impact of disease in APDS: a narrative exercise and qualitative interviews. First, participants were asked to write or audio-record an unstructured narrative account of their experience with APDS. In the interviews, participants were probed on the topics reported in the narrative exercise and asked semi-structured questions about their experience. Data were analyzed using content and thematic analysis. RESULTS: Four individuals with APDS and five caregivers participated in the study. All participants provided written narrative accounts approximately 1-4 pages long. Topics reported included the arduous journey to diagnosis, and brief descriptions of clinical manifestations, symptoms, treatments, and health-related quality of life (HRQoL) impacts. Interviews elicited similar topics, but provided a more detailed exploration of symptomatic clinical manifestations (e.g., breathing and swallowing difficulty associated with enlarged tonsils), HRQoL impacts (e.g., hearing loss impacting on school) and treatments (e.g., time burden). CONCLUSIONS: This study highlights the benefits of using multiple qualitative approaches in rare diseases. The narrative accounts identified new concepts that were important to individuals with APDS and their caregivers. The semi-structured qualitative interviews enabled details on these concepts to be expanded on and clarified. However, although all participants completed both components, a challenge could include increased participant burden due to the additional time required for both approaches which may have impacted recruitment. Therefore, it is important to remain flexible to participation across methods when recruiting. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23skrobanskipcr30poster132636-pdf.pdf?sfvrsn=fe440b40_0","title":"ISPOREurope23_Skrobanski_PCR30_POSTER132636.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132636","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Perceived Barriers to HIV Prevention and Detection Among Adolescents and Young People, According to Civil Organization Leaders and Health Professionals in Chile","id":"0083c926-a1a9-4ffa-bd1c-b520e59bf6fb","sessionCode":"PCR8","topDisplay":"Chandia S1, Obach A1, Sadler M1, Carreño-Calderon A2, Cabieses B1, Roberts A1, Campaña Castillo C3 1Universidad del Desarrollo, Santiago, Chile, 2Universidad del Desarrollo, Santiago, Chile, Chile, 3Universidad del Desarrollo, Santiago, RM, Chile","locationCode":"6002","description":"\r\n\t<div>OBJECTIVES: To identify the barriers that Chilean adolescents and young people face in relation to the prevention and detection of HIV, according to the perception of expert academics and health authorities of the Metropolitan Region today. METHODS: Qualitative study. 15 semi-structured interviews were conducted with health professionals specialized in HIV or with experience in working with young people on HIV issues and representatives of civil society organizations. Participants recruitment took place following a snowballing technique, thematic analysis was performed and UDD´s ethical approval was obtained. This study was carried out by the Center for Global Intercultural Health /CeSGI- ICIM.UDD and the project FONISSA19I0091. RESULTS: HIV prevention and detection in young people and adolescents was marked by barriers in the health system, including administrative barriers and those of health professionals and the low health literacy delivered to young people. The most prominent perceived barrier among young people was around the use of condoms, the conceptualization of HIV infection and the effective use they give to health centers. Another of the barriers mentioned is the lack of effective prevention and HIV detection updated to the concerns and needs of today's youth. Finally, in health professionals, heteronormative and risk approaches continue to prevail in sexual health care, focused on the reproductive spheres of sexuality. CONCLUSIONS: We found relevant perceived barriers in relation to the prevention and detection of HIV among adolescent and young people in Chile, from both the population and the health systems sides. Effective prevention and detection of HIV in these particular groups require of a better understanding of their beliefs, experiences and practices from healthcare systems.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132331","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Economic Evaluation of a Combined Early Detection Strategy to Prevent Acute Myocardial Infarction","id":"a6a58075-a390-4c15-81da-b5e38516fd48","sessionCode":"HTA1","topDisplay":"Oude Wolcherink M1, Pouwels X1, Van der Harst P2, Doggen CJ3, Koffijberg E1 1University of Twente, Enschede, OV, Netherlands, 2University Medical Centre Utrecht, Department of Cardiology, Utrecht, Utrecht, Netherlands, 3University of Twente, Enschede, Netherlands","locationCode":"4042","description":"\r\n\t<div>OBJECTIVES: Early detection of coronary artery disease (CAD) by computed tomography coronary artery calcium scoring (CT-CACS) followed by optimal medical treatment could prevent cardiovascular events (CVEs). However, optimal medical treatment alone may not be sufficient to prevent CVEs in individuals with severe coronary artery calcifications. Therefore, cardiac magnetic resonance myocardial perfusion imaging (CMR-MPI) in individuals with coronary calcium scores could be added to identify those who may benefit from preventive revascularisation. This study aims to evaluate the health and economic impact of screening with CT-CACS (1) or CT-CACS+CMR-MPI combined (2) compared to no screening (usual care) in the Dutch general population. METHODS: A patient-level state transition model with 10 health states was developed to estimate the lifelong impact of screening for both screening strategies and usual care in a simulated asymptomatic population of 10,000 from a societal perspective. A lifetime time horizon and one-year cycles were used. CVE risk was estimated using Multi-Ethnic Study of Atherosclerosis risk prediction. Data from the EARLY SYNERGY trial and literature informed the model. Total costs and quality-adjusted life years (QALYs) were calculated for each strategy and used to calculate the incremental cost-utility ratio (ICUR). Face validity of the conceptual model was ensured through clinical experts. Uncertainty analysis will be performed. RESULTS: In total, 1632 CVEs ocurred in usual care, 1605 with the CT-CACS strategy, and 1557 in the CT-CACS + CMR-MPI strategy. No screening led to an average cost of €1301 per individual, CACS to €1513, and CACS + CMR to €1639. The average QALYs per individual were 13.77, 13.85, and 13.87 for no screening, CACS screening and CACS + CMR screening respectively. The basecase ICURs were €2670 when comparing CACS to no screening, and €5216 when comparing CMR + CACS to CACS screening. CONCLUSIONS: Both screening strategies seem to be likely cost-effective, but CMR+CACS gained most QALYs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor2023oudewolcherinkhta1posterv2133475-pdf.pdf?sfvrsn=6b554d26_0","title":"ISPOR2023_OudeWolcherink_HTA1_POSTERV2133475.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133475","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Five-Year Healthcare Resource Consumption and Direct Costs of Women with a New Diagnosis of Hr+/HER2- Breast Cancer Primary or Advanced: Analysis of a Large Italian Administrative Database","id":"60ce0bfd-cab2-4c5f-b9af-b5fde197298d","sessionCode":"PT8","topDisplay":"Dell'Anno I1, Ronconi G1, Dondi L1, Dondi L1, Calabria S2, Piccinni C1, Esposito I3, Addesi A3, Pedrini A1, Maggioni AP4, Martini N1 1Fondazione Ricerca e Salute (ReS), Rome, Italy, 2Fondazione Ricerca e Salute (ReS), Roma, Italy, 3Drugs & Health srl, Rome, Italy, 4ANMCO Research Heart Care Center Foundation, Florence, Italy","locationCode":"5B","description":"\r\n\t<div>OBJECTIVES: Assess the healthcare resource consumption and direct costs by the Italian National Health Service (INHS) for women newly diagnosed with HR+/HER2- breast cancer (BC), by absence/presence of lymphnode (LNM)/distant metastases (DM). METHODS: From Fondazione Ricerca e Salute’s database (administrative data of ~5 million inhabitants/year), adult women with a new HR+/HER2- BC diagnosis in 2015 (index date) were categorized by absence (“primary”)/presence of in-hospital diagnoses of LNM/DM at index date or within the subsequent 60 days. Five-year overall (OS), distant relapse-free (DRFS) and invasive disease-free (IDFS) survivals (Kaplan Meyer analyses), and direct INHS costs were calculated. Among women with LNM, conservative/demolitive surgery and lymphadenectomy were assessed within one-year follow-up. RESULTS: In 2015, of 2,603 women newly diagnosed with HR+/HER2- BC (incidence: 1.2x1,000 inhabitants), 2,019 had primary BC (mean age 62±14; 39% with ≥2 comorbidities), 420 LNM (60±14; 32%) and 164 DM (67±13; 46%). Within 1-year follow-up, 407/420 women with LNM underwent conservative/demolitive surgery, 170/420 also lymphadenectomy. In 5-year follow-up: OS and IDFS probabilities were 89% and 82% for women with primary BC, 88% and 79% with LNM, and 28% and 17% with DM, respectively (p<0.01); the DRFS probability of BC-women with LNM was 86%. The 1st follow-up year resulted the most expensive for the INHS: on average, each patient with primary BC, LNM and DM costed, respectively, € 9,190, € 11,421 and € 16,320 (hospitalizations, including the index one, accounted for 45.1%, 48.8% and 48.3%). The subsequent mean per capita annual expenditures diminished up to € 3,529, € 3,761 and € 7,166, respectively; hospitalization costs halved, while pharmaceuticals almost doubled, and outpatient specialist care slightly reduced for primary BC and LNM and increased for DM. CONCLUSIONS: This study shows that HR+/HER2- BC-women diagnosed at advanced stages greatly impact on the INHS’s resource consumptions and direct costs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dellannopt8poster131255-pdf.pdf?sfvrsn=1094d97e_0","title":"ISPOREurope23_Dell'anno_PT8_POSTER131255.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131255","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Burden of Illness of Vasomotor Symptoms Associated with Menopause","id":"b58f21a1-dc5e-4e8e-80ca-b6578781625a","sessionCode":"SA10","topDisplay":"Malacan J1, Bolling KR2, Haberland C3, Gaianu L4, Smith N5, Woods M6, Smith M7 1Bayer Consumer Carer AG, Basel, Switzerland, 2Bayer U.S. LLC, Whippany, NJ, USA, 3Bayer AG, Berlin, BE, Germany, 4Bayer Public Limited Company, Reading, UK, 5Lumanity, Cheshire, CHW, UK, 6Lumanity, Sheffield, NYK, UK, 7Lumanity, London, UK","locationCode":"7018","description":"\r\n\t<div>OBJECTIVES: Menopausal transition is characterized by progressive decrease of estrogen levels causing various symptoms. Vasomotor symptoms (VMS; known as hot flashes) are the most common and have a significant impact on affected women. This review synthesizes the humanistic burden, economic burden and current treatment landscape of VMS associated with menopause. METHODS: A targeted burden of illness review was conducted including searches of MEDLINE®, Embase® and the National Health Service Economic Evaluation Database (NHS EED) to identify studies assessing the burden of VMS associated with menopause. Supplementary grey literature searches provided further evidence. RESULTS: Of 6,614 records identified, 276 studies were included into this review. Menopause affects 1 billion women worldwide. Approximately 80% of menopausal women experience VMS, with around 46% overall experiencing moderate-to-severe symptoms, lasting for up to 10 years. 24% of women with VMS reported that symptoms have a substantial impact on their quality of life (QoL), increasing with greater severity (mild: 11%; moderate: 27%; severe: 52%). More than 50% of women with VMS reported that VMS impacted their sleep, subsequently affecting their ability to perform daily tasks and work productivity. Furthermore, a greater severity of VMS has been associated with a marked increase in work impairment (mild or moderate: 4–14%; severe: 25–26%). Currently there are limited treatment options for menopausal symptoms, including VMS, with approximately 36% of women reporting dissatisfaction with their level of VMS control. Hormone therapy may not be suitable for all women, with 53–68% of women who are eligible for hormone therapy being averse to using it. CONCLUSIONS: VMS associated with menopause can be severe and have a detrimental impact on QoL and work/productivity. Despite this, there are limited effective treatment options and a significant percentage of women are unable or unwilling to use current therapy, highlighting the considerable unmet need.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23malacansa10poster130844-pdf.pdf?sfvrsn=b203c1c7_0","title":"ISPOREurope23_Malacan_SA10_POSTER130844.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130844","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evaluating the Cost-Effectiveness of Next-Generation Sequencing as a Biomarker Testing Strategy in Oncology and Implications for Policy: A Literature Review","id":"6decf9ef-1ae2-453b-bcf2-b7d35cbd4e6f","sessionCode":"EE65","topDisplay":"Wilsdon T1, Mirza M2, Goerke L3 1Charles Rivers Associates, London, UK, 2Charles Rivers Associates, Obertraubling, BY, Germany, 3Charles Rivers Associates, Munich, Germany","locationCode":"2011","description":"\r\n\t<div>OBJECTIVES: As personalized medicine becomes increasingly ubiquitous in oncology, there is a growing need to increase access to biomarker testing. One approach is greater use of Next-Generation Sequencing (NGS). There is an ongoing debate regarding whether NGS is cost-effectiveness today. This research aims to assess the current evidence base on the cost-effectiveness of NGS as a biomarker testing strategy in oncology and develop policy recommendations. METHODS: A structured literature review focusing on the period from 2017-2022 was conducted. Eligibility criteria were developed based on the indication and type of cost-effectiveness analysis provided. RESULTS: A total of 29 studies were selected and analyzed. Cost-effectiveness outcomes of NGS varied depending on the methodology used, type of NGS considered, number of genes tested, indication, and patient population. Overall, targeted panel testing (2-200 genes) demonstrated cost-effectiveness compared to single-gene testing when 4+ genes are tested. Larger NGS panels (200+ genes) were deemed less cost-effective. NGS was also shown to be less cost-effective as a first-line biomarker testing strategy in patient populations with 1 to 2 highly dominant mutations. In addition, when compared with single-gene testing, NGS provided reductions in turnaround time, personnel time, repeat hospital visits, and lower hospital costs. Finally, NGS demonstrated significant patient benefit by identifying additional mutations and increasing the number of patients receiving targeted therapies. CONCLUSIONS: The existing literature supports the cost-effectiveness of NGS as a biomarker testing strategy in oncology under the right circumstances. Furthermore, as the costs of NGS inevitably decrease and the number of viable biomarkers increases, it will become more cost-effective. These findings underline the need to develop effective policies which support holistic assessment benefits, equitable reimbursement and access, as well as infrastructure expansion.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/cra-ispor-poster-2023-next-generation-sequencing-as-a-biomarker-testing-strategy-v4132080-pdf.pdf?sfvrsn=614feff0_0","title":"CRA-ISPOR-Poster 2023-Next-Generation-Sequencing-as-a-Biomarker-Testing-Strategy-V4132080.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132080","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Cancer Deaths on Years of Life and Productivity Losses in Japan in 2019","id":"bdc1249b-4515-47bb-a545-b7d7ed43ddaa","sessionCode":"EPH25","topDisplay":"Aguiar-Ibáñez R1, Weston G2, Hughes R2, Abe M3, Taniguchi K4, Ohno T4, Bencina G5 1Merck Canada Inc, Toronto, ON, Canada, 2Adelphi Values PROVE, Bollington, UK, 3MSD K.K., Tokyo, Japan, 4MSD K.K., Tokyo, Chiyoda-ku, Japan, 5MSD, Center for Observational and Real World Evidence (CORE), Zagreb, 01, Croatia","locationCode":"3025","description":"\r\n\t<div>OBJECTIVES: The number of cancer-related deaths has increased in Japan over time, and there were 376,425 deaths reported only in 2019. We aimed to estimate the burden of cancer-related deaths in Japan in terms of life years lost and productivity losses. METHODS: Following a human capital approach, we estimated the years of life lost due to premature mortality (YLL), years of productive life lost (YPLL) and the present value of future loss of productivity (PVFLP) associated with cancer deaths in Japan. The YPLL were estimated as the years before retirement age that the person would have lived had the person not died prematurely, while PVFLP valued the YPLL according to the average salary per year in Japan. Age and sex-specific cancer-related-mortality data were obtained from Cancer Statistics in Japan. A retirement age of 65 years was assumed for both men and women. Average wages were sourced from the Ministry of Health, Labor and Welfare in Japan. RESULTS: In 2019 a total of 2,203,077 years of life were lost due to cancer-related deaths (YLL), with 454,762 of these (21%) being YPLL. Lung, stomach and colorectal cancers had the highest number of associated deaths and YLL, while colorectal, breast and lung had the highest number of YPLL. The total lost productivity costs due to premature cancer-related mortality was estimated to be ¥ 1.32 trillions. The average cost of lost productivity per premature cancer death was ¥3.51 millions across all tumors. CONCLUSIONS: The number of cancer deaths in Japan in 2019 resulted in a substantial burden, as reflected by the high number of YLL, YPLL and PVFLP. There is a need to increase the efforts in cancer care, including prevention, early detection and optimal treatment, to reduce the number of cancer-related deaths and the impact that these have in YLL, YPLL and PVFLP.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/eph25-aguiar-ibanez---japan-cancer-death-productivity-losses-ispor-europe-2023131327-pdf.pdf?sfvrsn=9eaa6092_0","title":"EPH25 Aguiar-Ibanez - Japan cancer death productivity losses ISPOR Europe 2023131327.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131327","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Where Can International HTA Collaboration Add Value? the Experience of NICE","id":"778e6f30-cd79-429c-b184-b8b09ea70d59","sessionCode":"HTA60","topDisplay":"Garrett Z National Institute for Health and Care Excellence (NICE), Manchester, UK","locationCode":"5008","description":"\r\n\t<div>OBJECTIVES: Collaboration can enable Health Technology Assessment (HTA) organisations to work more efficiently making better use of their resources, sharing expertise and improving the quality of their work. The National Institute for Health and Care Excellence (NICE) 5-year strategy places increased collaboration as a key development in the changing landscape in which NICE operates. This presentation describes work to support HTA collaboration at NICE and the outcomes of the work. METHODS: To support international collaboration NICE has recently put in place mechanisms to record and coordinate its international activity and to support teams to engage in international collaboration. RESULTS: NICE has over 150 active international partnerships across its programmes in countries around the world. Established areas of collaboration are in science and methods development, scientific advice and evidence generation. HTA collaboration in the areas of horizon scanning, topic identification and assessment and evaluation are less established, but there is ongoing activity to explore the potential opportunities and challenges. Challenges to collaborate arise from the jurisdiction specific nature of HTA and the procedural and methodological restrictions that can affect how flexible the agency is in the timelines and approach that can be adopted. CONCLUSIONS: Collaboration in the areas of science and methods, scientific advice and evidence generation can be easier to implement than collaboration in assessment and evaluation. This is because it is often less time sensitive and there is more flexibility in the approach that can be adopted. To overcome the challenges in setting up collaboration there is a need to identify the value of collaboration early on in a project and to embed it into projects during the planning stages. Collaboration approaches needs to be flexible with a focus on matching the right collaborator to the right project.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023-poster130441-pdf.pdf?sfvrsn=f0afd0ee_0","title":"ISPOR 2023 Poster130441.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130441","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparative Efficacy of Non-Statin Lipid-Lowering Therapies in Patients With Hypercholesterolemia at Increased Cardiovascular Risk: An Updated Network Meta-Analysis","id":"ba9a10c9-073e-4364-b243-b91420a0e7b7","sessionCode":"CO1","topDisplay":"Burnett H1, Neupane B1, Pierre V1, Fahrbach K2, Cichewicz A2, Natani H3, Bhowmik D3, Reichelt A4, Buesch K4, Jindal R4 1Evidera, St-Laurent, QC, Canada, 2Evidera, Waltham, MA, USA, 3Novartis Healthcare Pvt. Ltd., Hyderabad, Telangana, India, 4Novartis Pharma AG, Basel, Switzerland","locationCode":"1002","description":"\r\n\t<div>OBJECTIVES: To update the published network meta-analysis (NMA) from Burnett et al. 2022 comparing the efficacy of non-statin lipid-lowering therapies inclisiran, evolocumab, alirocumab, bempedoic acid, and ezetimibe in patients with hypercholesterolemia and/or increased cardiovascular risk having elevated low-density lipoprotein cholesterol (LDL-C) despite taking maximally-tolerated dose statins. METHODS: A systematic review was conducted using OvidSP (MEDLINE and Embase), Cochrane (Wiley), Pubmed, and Web of Science databases to identify more recently published randomized controlled trials through January 2023. The primary outcome of interest was the percentage change in LDL-C from baseline to week 24 (or closest available time point). A random-effects Bayesian NMA was performed to estimate the mean differences (MD) and 95% credible intervals (CrI) for the included therapies. RESULTS: A total of 20 studies were included in the analysis (inclisiran: 4 studies, evolocumab: 5 studies, alirocumab: 8 studies, ezetimibe: 2 studies, bempedoic acid: 3 studies, bempedoic acid + ezetimibe: 1 study). Inclisiran, evolocumab, and alirocumab delivered superior efficacy over placebo, bempedoic acid, and ezetimibe in reducing LDL-C from baseline at week 24. In particular, inclisiran provided statistically significant benefit over bempedoic acid, ezetimibe, and bempedoic acid + ezetimibe (MD: -44.24% [95% CrI: -51.84, -36.70], MD: -35.66% [95% CrI: -43.10, -28.49], and MD: -20.79% [95% CrI: -33.69, -7.98], respectively). There was no significant difference in LDL-C reduction between inclisiran and PCSK9 inhibiting monoclonal antibodies. (vs. alirocumab, MD: -1.93% [95% CrI: -8.56, 4.20]; vs. evolocumab, MD: 2.00% [95% CrI: -4.58, 8.60]). CONCLUSIONS: This updated NMA reaffirms that inclisiran, alirocumab, and evolocumab are expected to provide similar clinically meaningful improvements in LDL-C in patients with hypercholesterolemia on maximally-tolerated statins who are at increased cardiovascular risk; while they delivered superior efficacy over placebo, bempedoic acid, and ezetimibe in reducing LDL-C.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bueschco1poster131382-pdf.pdf?sfvrsn=e04118a_0","title":"ISPOREurope23_Buesch_CO1_POSTER131382.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131382","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Volume Vs Value: Understanding the Pricing Dynamics of Multi-Indication Therapies in Weighted-Average List Price Markets","id":"402aa424-7ff6-4db2-80fb-b9be388539d0","sessionCode":"HPR36","topDisplay":"Gerhardt M, Malliou-Najjar K, Jansen C, Kassenaar S Inbeeo, London, LON, UK","locationCode":"4002","description":"\r\n\t<div>OBJECTIVES: Multi-indication therapies commonly experience list price decreases following indication expansion, due to the budget impact associated with the newly targeted patient population and country-specific pricing policies. This research examines multi-indication pricing dynamics in average list price markets (Germany and France). The aim is to understand differences in multi-indication pricing and reimbursement (P&R) and identify cases where a new indication delivered added clinical value outweighing the increased budget impact. METHODS: European Commission (EC) authorized therapies between 2012-2022 were screened for products with ≥ 3 indications. For these products, list prices in Germany and France were collected from Navlin to examine pricing dynamics. For products with a ≥ 20% price increase, P&R data was extracted from the Gemeinsamer Bundesausschuss (G-BA) and Haute Autorité de Santé (HAS) to identify price increase drivers. RESULTS: Of 87 authorized multi-indication therapies, 40 had ≥ 3 indications. Whilst 29 products experienced price decreases or price maintenance, 10 products in Germany and 1 in France experienced at least one price increase. Average annual fluctuation levels ranged from -4% to 0% in France and -11% to +2% in Germany. Three products had price increases ≥ 20% in Germany (dapagliflozin, dapagliflozin and metformin, ruxolitinib), compared to 1 in France (human normal immunoglobulin). G-BA evaluation analysis demonstrated that price increases in Germany were linked to re-assessments based on additional data submission, following a change of comparator therapy or indication expansion with a higher benefit rating than the initial indication. Reasons for the price increase in France could not be identified from publicly available data. CONCLUSIONS: In Germany, a higher benefit rating of a new indication can occasionally lead to price increases. These increases seem mainly driven by re-assessment based on new data. This suggests that the German system allows more flexibility in applying value-based pricing for multi-indication products compared to France. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/gerhardthpr36postermulti-indication-therapies130576-pdf.pdf?sfvrsn=58986afa_0","title":"Gerhardt_HPR36_Poster_Multi-indication therapies130576.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130576","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Applying a Cost-Based Pricing Model for Innovative Cancer Treatments Subject to Indication Expansion: A Case Study for Pembrolizumab and Daratumumab","id":"296ac2d7-1e11-46a9-a495-ba89a71540db","sessionCode":"HPR7","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3066","description":"\r\n\t<div>OBJECTIVES: Expanding the indication of already approved immuno-oncology drugs presents treatment opportunities for patients but also strains healthcare systems. Cost-based pricing models are discussed as a possibility for cost containment. This study focuses on two drugs, pembrolizumab (Keytruda) and daratumumab (Darzalex), to explore the potential effect of indication broadening on the estimated price when using the cost-based pricing (CBP) model proposed by Uyl-de Groot and Löwenberg (2018). METHODS: The model was used to calculate cumulative yearly prices, cumulative prices per indication, and non-cumulative indication-based prices using inputs such as research and development (R&D) costs, manufacturing costs, eligible patient population, and a profit margin. A deterministic stepwise analysis and scenario analysis were conducted to examine how sensitive the estimated price is to the different input assumptions. RESULTS: The yearly cumulative cost-based prices (CBPs) ranged from €52 to €885 for pembrolizumab per vial and €823 to €31,941 for daratumumab per vial. Prices were higher in initial years or indications due to smaller patient populations, decreased over time or after additional indications. Sensitivity analysis showed that the number of eligible patients had the most significant impact on the estimated price. In the scenario analysis the profit margin contributed most to a higher CBPs for both drugs. Lower estimates resulted from assumed lower R&D costs. CONCLUSIONS: The estimated CBPs are consistently lower than Dutch list prices for pembrolizumab (€2,861), mainly resulting from larger patient populations in registered indications. However, daratumumab's list prices fall within the range of modeled CBPs depending on the year or indication (€4,766). Both CBPs decrease over time or with additional indications. The number of eligible patients and initial R&D costs have the most significant influence on the CBPs. These findings contribute to the ongoing discussions on pharmaceutical pricing, especially concerning cancer drugs with expanding indications.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132730","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Humanistic Burden of Paroxysmal Nocturnal Hemoglobinuria from the Patient Perspective: Results from a Cross-Sectional Study","id":"abe9944d-0911-4680-adaf-bb6b44d02b62","sessionCode":"PCR42","topDisplay":"Panse J1, O'Neill CB2, Wiyani A3, Snellman J4, Mellor J5, Earl L5, Taylor Y5, Simons A5, Balp MM4 1University Hospital RWTH Aachen, Aachen, NW, Germany, 2Novartis Business Services, Dublin, County Dublin, Ireland, 3Novartis Pharmaceuticals UK Limited, London, UK, 4Novartis Pharma AG, Basel, BS, Switzerland, 5Adelphi Real World, Bollington, Cheshire, UK","locationCode":"6041","description":"\r\n\t<div>OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood condition, caused by complement-mediated hemolysis with persistent anemia and fatigue, which has a negative impact on patients. Currently approved treatments include complement 5 inhibitors (C5i). The study aim was to assess the humanistic burden of PNH from the patient perspective. METHODS: Data were drawn from the Adelphi PNH Disease Specific Program™, a cross-sectional survey of PNH-treating physicians and their patients from Canada, France, Germany, Italy, Japan, Spain, and the USA (collected Jan-July 2022). Patients completed a survey comprising demographics, PNH-related symptoms, FACIT-Fatigue (recall over 7 days, score ranging 0-52), Short Form Survey (SF-36) allowing the calculation of mental and physical component summary scores (MCS, PCS), EQ-5D-5L. Lower scores indicate worse outcomes for all instruments. EQ-5D utility scores were calculated using UK tariffs. Physicians reported patient’s current treatment, dates of diagnosis and treatment initiation. Descriptive analyses were conducted on data from patients treated with C5i. RESULTS: Overall, 143 PNH patients on C5i were analyzed; mean (SD) age 48.2 (14.8) years (median 47.0) and 52% were male. Mean (SD) disease duration was 2.5 (2.8) years and duration on C5i therapy was 1.8 (2.1) years. The majority of patients (60%) reported their most bothersome symptom as tiredness/lack of energy. The FACIT-Fatigue score was 36.3 (10.1), the MCS 44.0 (8.7), PCS 43.9 (9.7), EQ-5D-VAS of 72.1 (15.4) and utility of 0.77 (0.2). In the EQ-5D-5L, 30% of patients reported moderate-severe anxiety/depression and 22% reported moderate-severe problems doing their usual activities. CONCLUSIONS: Despite current treatment with C5i, many PNH patients continue to experience symptoms including tiredness/lack of energy, with low mental, physical status and utility scores reported. Better therapies capable of providing comprehensive control of PNH may address the remaining clinical and humanistic burden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23pansepcr42posterv2130006-pdf.pdf?sfvrsn=30a6e40c_0","title":"ISPOREurope23_Panse_PCR42_POSTERV2130006.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130006","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Assessment of Validated Algorithms for Identifying Multiple Sclerosis Cases, Subtypes, and Relapses in Real-World Databases: A Systematic Review","id":"95c571d8-dfe8-4914-99fa-bda184497d3a","sessionCode":"EPH21","topDisplay":"Albalawi O, Almadani OA Saudi Food and Drug Authority, Riyadh, 01, Saudi Arabia","locationCode":"3023","description":"\r\n\t<div>OBJECTIVES: No specific diagnosis codes exist for either multiple sclerosis (MS) subtypes or MS relapses. This study aimed to (1) identify all original validation studies used algorithms to identify MS in RWD, and (2) assess evidence for algorithm validity. METHODS: A systematic review was completed according to to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Four key bibliographic databases were searched. RESULTS: Of 5,056 identified articles, and 21 met inclusion criteria. Most included studies (43%) aimed to identify MS cases; others focused on MS subtypes (28%), relapses (15%), diagnosis and severity (9%), or disability status (5%). Only eight (38%) reported all four algorithm validity metrics: sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV). Specificity was most frequently used (76 %), followed by sensitivity (71%), PPV (67 %), and NPV (52 %). Manual validation was the gold standard (76.1%) for sixteen studies, all of which found at least one algorithm with high sensitivity (77–99%), while specificity ranged from 88 to 99.9%, PPV from 38 to 95%, and NPV from 70 to 100%. All the studies aimed at identifying MS cases reported sensitivity (44%–100%) and specificity (87–100%), but only five reported PPV and NPV (38–99% and 17–100%, respectively). All three studies that identified MS relapses used administrative health databases with manual validation as the gold standard, finding sensitivity to range from 70 to 100%, specificity from 87 to 100%, PPV from 64 to 100%, and NPV from 53 to 100%. The best preformed algorithm for MS relapse identification included diagnoses, medications, hospitalization duration, or outpatient visits (sensitivity 70%, specificity 100%, PPV 100%, and NPV 96%). CONCLUSIONS: Although there are no relapse-specific MS diagnosis codes, there are few validated algorithms to detect MS relapses, and none have been developed for other important RWD sources, such as electronic health records.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130037","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Costing for Health Economics Evaluation: The Case of the Brazilian Universal Health System","id":"ff25433d-004c-4ddc-b7db-be13c75de23c","sessionCode":"HTA57","topDisplay":"Beume TM1, Caetano R1, Raymundo C2 1Instituto de Medicina Social, Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brazil, 2Medical Science College, University of State of Rio de Janeiro, Rio de Janeiro, Brazil","locationCode":"5002","description":"\r\n\t<div>OBJECTIVES: Brazilian has a universal health system (SUS) and Health Technology Assessment (HTA) was institutionalized in SUS in 2011 through CONITEC, the Commission that advises the Ministry of Health (MOH) for health technology incorporation. HEE and Budget Impact Analysis (BIA) as part of HTA process are mandatory. The aim of this study was to analyze cost information in the HEE in the HTA reports in the Brazilian SUS. METHODS: A descriptive study using CONITEC HTA recommendations from 2012 to 2020 (n=848). Data extraction used Excel spreadsheet developed for this purpose. The questions for analysis considered open and closed answers, the latter based on ISPOR CHEERS 2022. All variables are categorical and are expressed as several cases and percentages. Statistical analysis was conducted using the R software (R Development Core Team, 2021), version 4.1.1. RESULTS: From 848 recommendations, 123 were excluded because they were clinical protocol. The final sample had n=725 recommendations, in which 71.6 % (n=519) considered only direct medical cost, indirect cost in 0.7%, intangible cost were not considered in any recommendation. In 97% (n=703) the costing method was not reported. In 97% (n=703), the perspective was SUS as the payer, society 0.5%, others 0.7%, not reported 0.8%. ICER was calculated in 39.6% of reports of full HEE (N=376). Source of the costs information were literature, expert opinion, government procurement sites and reimbursement. CONCLUSIONS: The representative consideration of direct medical expense (drugs) instead of cost, without mentioning costing methods, signalize inconsistencies in the use of cost information in the HEE. Implementation of a costing system connecting SUS healthcare settings, training professionals in cost management, and development of a cost panel in healthcare, are urgent measures to minimize the consequences of disturbances in the access to health technologies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23beumehta57poster130261-pdf.pdf?sfvrsn=cd297967_0","title":"ISPOREurope23_BEUME_HTA57POSTER130261.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130261","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Can Machine Learning Accurately Predict Payer Behavior?","id":"1ae9ca87-0e6c-4e9c-bcae-be8192c77949","sessionCode":"MSR17","topDisplay":"Anstee K, Muniandy D Global Pricing Innovations, London, London, UK","locationCode":"5059","description":"\r\n\t<div>OBJECTIVES: Machine learning can be applied to predict outcomes. However, within market access, there is limited application due to complexities of payer decision-making. This research aimed to train and test the accuracy of machine learning models based on regression algorithm in forecasting the price of an orphan product in England, France, and Germany. METHODS: A MCDA model with key attributes reflecting payer drivers for orphan products was developed. Value scores were derived using HTA documents and annual COT (list price) was calculated for all products assessed by each HTA and reimbursed since 2016. Pegcetacoplan, a recently assessed orphan product, was removed for testing. Datasets were analysed using Python libraries and run in JupyterLab. Model training included various algorithm prediction, Spearman correlation analysis, linear and polynomial regression models. Pegcetacoplan price was predicted and compared to actual price. RESULTS: Most variables were positively correlated to price in France and the UK; UK specific variables (CEA threshold and HST appraisal) provided the greatest correlation. All Germany variables were negatively correlated or had no correlation to price, reflecting evidence uncertainty. Linear regression models had low R2 (France=0.17; Germany=0.31; UK=0.41). Trained linear regression models predicted pegcetacoplan price within -24% (Germany), -49% (France) and -113% (UK) of actual price. Polynomial models in France and the UK were overfitting (R2=1.0) and unsuitable for predictions. Power 2 and 3 polynomial models in Germany improved accuracy (R2=0.39 and 0.63), but provided less accurate pegcetacoplan price predictions (-102% and -197%). CONCLUSIONS: Analyses suggested that inclusion of all reimbursed products can be used to train models with high predictability. However, due to the nuances of payer decision-making, applying these models to real scenarios have varying accuracy. Limitations included small sample, inclusion of high cost ATMPs and confidential discounting. Further research on relevant analogues and in other indications is required.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132740","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Pemigatinib for the Treatment of Adult Patients with Locally Advanced or Metastatic Cholangiocarcinoma with a FGFR2 Fusion or Rearrangement That Have Progressed After Systemic Therapy in Greece","id":"5617b6d0-c25c-4b4e-b364-bf0ac5891844","sessionCode":"EE95","topDisplay":"Tzanetakos C1, Psarra M1, Batsi M2, Patterson K3, Vostitsanou Z4, Gourzoulidis G5 1Health Through Evidence, Athens, Greece, 2Genesis pharma, Athens, Greece, 3Lumanity, London, UK, 4Genesis, Athens, Greece, 5Health Through Evidence, Athens, A1, Greece","locationCode":"1044","description":"\r\n\t<div>OBJECTIVES: To evaluate the cost-effectiveness of pemigatinib compared to oxaliplatin-L-folinic-acid and fluorouracil plus active symptom control (mFOLFOX+ASC) and ASC alone for the treatment of patients with advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) rearrangement or fusion who have progressed on at least one line of prior systemic therapy in Greece. METHODS: A partitioned survival model, with five health states, was locally adapted from a Greek payer perspective over a lifetime horizon. Clinical and safety data as well as utility values were extracted from the literature. A matching-adjusted indirect comparison of pemigatinib and mFOLFOX+ASC was performed. Reimbursable direct costs pertaining to drug acquisition, administration, monitoring, adverse events, and end of life were considered in the analysis. All cost inputs were indexed to 2023 euros. Primary outcomes were patients’ life years (LYs), quality-adjusted life years (QALYs), total costs and incremental cost-effectiveness ratios (ICERs) per QALY and LY gained. All future outcomes were discounted at 3.5% per annum. Sensitivity analyses were conducted. RESULTS: Total lifetime cost per patient with pemigatinib, mFOLFOX+ASC and ASC alone were estimated to be €85,534, €2,537 and €1,010 respectively. In terms of health outcomes, pemigatinib was associated with 1.78 and 1.84 increment in LYs compared with mFOLFOX+ASC and ASC alone. Furthermore, pemigatinib appeared to yield more QALYs gained versus mFOLFOX+ASC and ASC alone (1.66 vs 0.44 and 0.41, respectively), resulting in ICERs of €69,928 per QALY gained and €46,626 per LY gained versus mFOLFOX+ASC and €69,345 per QALY gained and €45,935 per LY gained compared to ASC alone. Sensitivity analyses confirmed the robustness of base-case results. CONCLUSIONS: Pemigatinib, a therapy covering the unmet medical need of patients with previously treated locally advanced or metastatic CCA with an FGFR2 fusion or rearrangement, was estimated to be cost-effective compared with mFOLFOX+ASC and ASC alone in Greece.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/pemigatinibposterispor2023final130882-pdf.pdf?sfvrsn=dfb9ddc4_0","title":"Pemigatinib_poster_ISPOR2023_Final130882.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130882","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"5 Years of Real-World Data on the Use of Cladribine Tablets for the Treatments of Multiple Sclerosis in Portugal","id":"06a17685-7ac0-4b91-94e9-bf19b25319ca","sessionCode":"CO12","topDisplay":"Ribeiro M1, Pinto D2 1Adecco, Lisboa, 11, Portugal, 2Adecco, Porto, Portugal","locationCode":"1012","description":"\r\n\t<div>OBJECTIVES: To present real-world data from a patient registry of multiple sclerosis (MS) patients being treated with cladribine tablets (CT) since its launch in Portugal (PT). METHODS: Anonymized data was retrieved from a patient support program database of MS patients treated with CT in PT. Data was checked for missing values and descriptive analysis was run for the available information using STATA18. Missing data was censured, with number of samples being mentioned. Information was available from a total of 33 hospitals. Cut-off date for the analysis was May 17th, 2023. RESULTS: Overall, 300 patients were included in the analysis, 75,33% were female (23,67% male; 1% unknown). At time of cut-off 24 patients had dropped-out of treatment. Average age of the population (n=277) was 43,54 ± 11,71 years, with a median of 42,50 years, a minimum of 20,6 and a maximum of 77,9 years. The average time on treatment (ToT) with CT for the whole population (n=300) was 2,11 ± 1,24 years, with a median of 1,99 years, minimum of 0,05 and maximum of 4,88 years. For the 24 patients that had dropped-out of treatment ToT was on average 1,87 ± 1,16 years, with a median of 2,00 years. At the time of analysis 22% of patients were undergoing year 1 of treatment, with 29% in year 2, 21% in year 3, 20% in year 4 and 8% in year 5. CONCLUSIONS: Data observed seems to show that ToT for cladribine tablets seems in line with what has been observed in randomized-controlled trials and in other real-world studies. A total of 8% of patients were still under treatment beyond end of year 4.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129079","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Rethinking Economic Evaluation in Amyotrophic Lateral Sclerosis Treatment: Embracing Alternative Model Conceptualizations","id":"b6c048af-9704-43aa-9295-bfa20fcda8c7","sessionCode":"EE51","topDisplay":"Sharma R1, Davison N2, Öhrman S3, Strican L3, Phatak H4 1Maple Health Group, LLC, Brooklyn, NY, USA, 2Maple Health Group, LLC, Manchester, UK, 3Amylyx Pharmaceuticals EMEA BV, Zurich, Switzerland, 4Amylx Pharmaceuticals, Cambridge, MA, USA","locationCode":"1091","description":"\r\n\t<div>OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disease affecting voluntary muscle control. While no cure exists for ALS, treatments may slow its progression, as measured by clinical scales and staging systems (e.g., Fine’til 9, MiToS). Thakore et al. (2020) developed a Markov model (MM) with health states based on Fine’til 9 stages to assess the cost-effectiveness of ALS treatments. This approach has been leveraged in subsequent health technology assessments (HTAs) in ALS. In 2022, Amylyx conducted an advisory board with leading health economists, representing six European countries. The advisors recommended a partitioned survival model (PSM) as it may better capture the different clinical benefits of treatment and better represents the ALS disease course. This study considers how to best conceptualize ALS and treatment effects in a cost-effectiveness model. METHODS: The PSM approach involved a single partition based on time-to-hospitalization. A targeted literature review (TLR) was conducted to assess data availability for cost-effectiveness analysis, and an early PSM was developed. RESULTS: In previous HTAs in ALS, Markov transition probabilities were based on published evidence, and treatments effects were applied using relative risks. Whereas, a PSM allows inclusion of cross-over adjusted survival from trial data in a technically robust manner, which is crucial to understanding life years (LYs) and therefore quality-adjusted life years (QALYs). A potential limitation of the PSM approach is that information on subsequent worsening may be lost by dichotimizing progression. However, the TLR findings suggested little differentiation in utility and healthcare resource use between later disease stages (as defined by MiToS 2-4), which could be pooled for post-progression inputs. CONCLUSIONS: Overall, time-to-hospitalization is important to people living with ALS and clinicians, and a PSM seems to be a more optimal approach to represent the ALS disease course and capture the value of novel ALS interventions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sharmaee51poster133342-pdf.pdf?sfvrsn=e1e76a37_0","title":"ISPOREurope23_Sharma_EE51_POSTER133342.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133342","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical Manifestations and Disease Burden of Primary Mitochondrial Myopathies (PMM): Results from a Patient Journey Analysis Show Substantial Healthcare Resource Utilization","id":"f942c644-b3a0-4b34-ac48-c15971439d85","sessionCode":"EE90","topDisplay":"Sirimanne M1, Kates J1, S SS2, Warner M3, Shah S4, Lovink A4, Xue Y4, Yonan C5 1Reneo Pharmaceuticals, Irvine, CA, USA, 2Trinity Life Sciences, Burlington, MA, USA, 3Commercial Rx, Inc., CORONA DEL MAR, CA, USA, 4Trinity Life Sciences, Waltham, MA, USA, 5Reneo Pharmaceuticals, Pittsburgh, PA, USA","locationCode":"2032","description":"\r\n\t<div>OBJECTIVES: PMM are a group of underdiagnosed rare genetic disorders characterized by a range of clinical presentations and multisystemic impact. Diagnosis and management of PMM can be challenging due to this heterogeneity of clinical manifestations. With no approved treatments for PMM, current practices focus on symptom management and do not address the underlying cause. A patient journey analysis was performed to quantify barriers faced by US patients in their odyssey from clinical manifestation to management. METHODS: With no PMM-specific ICD-10 diagnosis code, a stepwise approach was needed to identify patients with suspected PMM from Komodo closed-claims data between 2016-2021. This entailed using ICD-10 diagnosis codes specific to mitochondrial disorders (MD) (e.g., chronic progressive external ophthalmoplegia, Kearns-Sayre Syndrome, and Leigh Syndrome), limiting analyses to patients with ≥1 myopathy claim, and excluding those with a secondary MD ICD-10 code. RESULTS: Of 3.7K patients included for analysis, 97% experienced multi-organ manifestation, impacting an average of 6 organ systems (e.g., nervous, cardiac, musculoskeletal). In the 12 months prior to diagnosis, 73% of patients reported nervous system manifestations and ~70% reported skeletal/muscular manifestations, compared to ~57% and 56% in the 24-36 months prior to diagnosis, respectively. When analyzed by myopathy-related presentations (e.g., impaired gait/mobility, fatigue, myalgia), 36% of patients with suspected PMM had moderate-to-severe presentations, as indicated by inpatient admission or myopathy-related complications (e.g., rhabdomyolysis). Healthcare resource utilization was high, including increased specialist engagement, with most patients seeing a neurologist an average of ~6×/year. CONCLUSIONS: This patient analysis confirms that PMM encompass a broad spectrum of clinical manifestations and suggests that diagnosis often occurs after multisystem involvement and extensive engagement with the healthcare system. Moreover, it underscores the need for further healthcare provider education about potential manifestations and multiorgan dysfunctions indicative of PMM, along with information to help clinicians make earlier clinical and genetic confirmatory diagnoses for appropriate management.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23yonanee90poster130193-pdf.pdf?sfvrsn=1813ef09_0","title":"ISPOREurope23_Yonan_EE90_POSTER130193.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130193","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Accuracy of Automated Data Extraction for Systematic Literature Reviews","id":"4ea3cc9f-2470-412b-b4a3-c1fb1e502693","sessionCode":"MSR5","topDisplay":"Hanegraaf P1, Mosselman JJ2, de Jong R2, Boersma C3, van der Schans J4 1Pitts, Zeist, Netherlands, 2Pitts, Zeist, UT, Netherlands, 3University of Groningen, Department of Health Sciences, UMCG; Open University, Heerlen, Department of Management Sciences and Health-Ecore Ltd, Zeist, The Netherlands, Groningen, Netherlands, 4Health-Ecore, Groningen, GR, Netherlands","locationCode":"5043","description":"\r\n\t<div>OBJECTIVES: Machine learning automation tools have the potential to reduce the workload in systematic literature reviews (SLRs). However, since the number of openly accessible tools that can assist in data extraction is low and the feasibility of data extraction beyond PICO (participants, interventions, comparators, outcomes) extraction is almost completely absent, evaluation of real-world inter-reviewer agreement metrics standards in data extraction are missing. Our main objective is to assess the accuracy of an proprietary GPT-3 autoregressive language model for the automated data extraction and assisted data extraction in a SLR. METHODS: Data was used of a SLR of randomized controlled trials on the treatment for patients with Achilles tendinopathy. After including all eligible studies, data was extracted by two authors, on the duration of participation, start date and end date of enrolment of the trial, type of Achilles Tendinopathy, and the number of treatment arms, as a representation of different data types extracted in SLRs. The GPT-3 model predicted both the extracted value as well as the place of the extracted value based on an engineered prompt. No training of the GPT-3 model was used. The accuracy of both the value as well as place were calculated using the author team’s decision as a reference standard. RESULTS: The data of 19 included studies was used for the accuracy assessment. On average, GPT-3 API could predict the value of the different data extraction parameters with 67.98% (range: 47.37-84.21%) accuracy. The place of the extracted value was predicted correctly with an accuracy of 78.95%. CONCLUSIONS: This study gives a first overview of the accuracy of using GPT-3 data extraction in an SLR. Our results show that automation of data extraction could be a valuable and scalable tool for SLRs in primarily assisted data extraction.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023vanderschansmsr5posterv2131449-pdf.pdf?sfvrsn=b32f7266_0","title":"ISPOREurope2023_vanderSchans_MSR5_POSTER_V2131449.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131449","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Broadening the Scope: Analysis of Oncology Reimbursement Decisions Following the Cancer Drugs List Introduction in Singapore","id":"c86bd88d-1873-4ec7-b3a2-c25137f2fab1","sessionCode":"HTA13","topDisplay":"Tzaras D, Sondhi A Precision Advisors, London, UK","locationCode":"4037","description":"\r\n\t<div>OBJECTIVES: Since 2015, the Agency for Care and Effectiveness (ACE) has aimed to drive better decision-making in healthcare in Singapore based on clinical effectiveness, safety, and cost-effectiveness. From September 2022, Singapore’s Ministry of Health (MOH) introduced the Cancer Drug List (CDL) to mitigate government spending on cancer drug treatments by allowing the latter to negotiate better prices and extend subsidies for more cancer drugs. This research aims to identify the number of publications in oncology in Singapore and their respective subsidy decisions to assess the impact of CDL on oncology product access. METHODS: An analysis of reimbursement decisions from Singapore’s ACE (Agency for Care Effectiveness) was conducted, dating from 2017 to 2022 to identify the total number of publications and extract their respective subsidy decisions. Additionally, the number of CDL-included drugs was also extracted from the MOH website. RESULTS: Between 2017 and 2021, ACE’s oncology evaluations were limited to 7 publications including 15 subsidy decisions. These were targeting mainly biosimilar and select oncology products with 60% leading to Standard Drug List (SDL) inclusion, 7% in Medicines Assistance Fund (MAF) inclusion, and 33% in non-recommendation. A noticeable uptrend was noted in 2022 with ACE releasing 31 publications involving 264 subsidy decisions. Out of these decisions, 18.6% led to SDL inclusion, 42.4% to MAF inclusion, and 39% in non-recommendation. This uptrend coincides with the CDL introduction of September 2022, which includes 181 products as of April 2023. CONCLUSIONS: Our results demonstrate a significant rise in oncology publications in 2022 which coincides with the creation of CDL, coupled with a greater percentage of MAF inclusion. SDL inclusions, while lower in percentage, increased in absolute figures. Whilst evidence demonstrate a promising early start to the CDL, subsequent future evaluations will determine whether it has truly enabled broader access to oncology products in Singapore.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23macaulayhta13poster133553-pdf.pdf?sfvrsn=10d868d6_0","title":"ISPOREurope23_Macaulay_HTA13_POSTER133553.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133553","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization and Cost of Renal Replacement Therapy in Brazil: A Five-Year Patient-Level Analysis","id":"049eaf10-aea8-4c04-8c06-c2981dc72da4","sessionCode":"RWD28","topDisplay":"Lopes N1, Fogolin Rosal G2, Tzanno Branco Martins C3, Pereira RG4, Birck M4, Julian G5, Forestieiro F6, Oliveira I2 1Novartis Biociências, São Paulo, Brazil, 2Novartis Biociências, Rio de Janeiro, Brazil, 3Centro Integrado de Nefrologia, Home Dialysis Center, São Paulo, São Paulo, Brazil, 4IQVIA, São Paulo, São Paulo, Brazil, 5IQVIA, São Paulo, SP, Brazil, 6Novartis Biociências, São Paulo, São Paulo, Brazil","locationCode":"7002","description":"\r\n\t<div>OBJECTIVES: In this patient-level analysis we explored HCRU, and medical costs of patients submitted to kidney transplant at SUS (Unified Health System) who were simultaneously covered by SH (Supplementary Health System). METHODS: This is patient-level retrospective analysis conducted over a 5-year period, from Jan-2015 to Dec-2019, used publicly available administrative claims data from the SUS and SH. The cohort included patients ≥ 18 years, submitted to kidney transplant at SUS and to dialysis or other RRT-related procedure at SH, ≥ 6 months of follow-up prior and after transplantation. Costs were in Brazilian real (BRL) and converted to US dollar (USD); 1 USD = 4.0943 BRL, in mid-December 2019. RESULTS: Cohort included 290 patients; mean age was 50.9 years (SD 12.9). Of total patients, 81.3% used ambulatorial care in SH pre-transplant with median of 1.33 times (Per Patient Per Month – PPPM) and 26.5% after kidney-transplant (0.45 times PPPM). In SUS, most patients used ambulatorial services before (81.3%) and after (93.1%) kidney-transplant, accounting for 0.74 times PPPM and 0.95 times PPPM, respectively. Of total patients, 44.7% received hemodialysis at SH before kidney-transplant (2.22 times PPPM). More than 70% of patients received drugs at SUS. Before transplantation, median costs of ambulatorial services were USD 127 PPPM in SH and USD 267 PPPM in SUS. CONCLUSIONS: Insured patients undergoing kidney transplant in SUS impose a burden for both public and supplemental health sectors in Brazil. In SUS, besides the transplant surgery, patients used ambulatorial services before and after transplant, including dialysis, transplant follow-up, and drug acquisition. SH is mostly used for dialysis procedures before the transplant.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster2b---patient-level-analysis-nlopesrevio261023133015-pdf.pdf?sfvrsn=561d1856_0","title":"Poster2B - Patient-level analysis-NLopes_Rev_IO_26_10_23133015.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133015","diseases":[{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Hearing the Patient Voice in Japan – Utilisation of Patient-Reported Outcomes and Wearable Device Data for Patient-Centric Real-World Research","id":"61135088-e082-4854-adcd-c30799112b55","sessionCode":"RWD32","topDisplay":"Hamaguchi A1, Kondo D2, Mitani A3, Yates M1, Pulfer A4, Lambrelli D5 1Evidera, London, UK, 2JMDC Inc., Tokyo, Japan, 3DeSC Healthcare, Inc., Tokyo, Japan, 4Evidera, Bishops Stortford, UK, 5Evidera, Thessaloniki, Greece","locationCode":"7007","description":"\r\n\t<div>OBJECTIVES: There is growing emphasis on the importance of real-world evidence (RWE) and patient centricity across the product life cycle. Japan real-world data (RWD) sources offer a unique opportunity in combining patient reported outcomes (PROs) with medical data. However, these data have been underutilised to date, possibly owing to language barriers or lack of awareness amongst researchers. We aim to: <ul> <li>Describe RWD sources in Japan with available PRO data and data from wearable devices; and</li> <li>Discuss the impact on research options available to researchers.</li> </ul> METHODS: Publicly available information and the authors’ experience from working with data in Japanwere used to summarise available datasets in Japan with PRO and/or wearable data. RESULTS: JMDC and are two large commercially available claims databases in Japan that facilitate the collection of PROs and wearable data. JMDC covers 16 million people. PROs can be collected via the “PepUp” app and linked to claims data. The app also facilitates the collection of data from wearable devices (activity, heart rate, sleep), from more than 30,000 users. DeSC covers 11.2 million people. PRO data can be collected using their health promotion support service app, kencom. Regularly collected PROs include the work productivity and activity impairment questionnaire, EQ-5D-5L, and a sleep survey. Both PepUp and kencom facilitate the prospective collection of bespoke PROs from a population already familiar with the apps. This has the potential to allow a deeper understanding of the impact of diseases, and facilitate identification of distinct phenotypic subgroups not identifiable with claims data alone. CONCLUSIONS: Patient centric data collected via apps can be linked to claims data in Japan with minimal patient burden. Both the existing PRO data and future bespoke collections will expand research opportunities to reflect the patient voice.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeu23lambrellirwd32poster134049-pdf.pdf?sfvrsn=859fefce_0","title":"ISPOREU23_Lambrelli_RWD32_Poster134049.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/134049","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Study of HTA Timelines and Outcomes for MHRA-Approved NASs in the Post-Brexit UK via Reliance/Work-Sharing Routes","id":"80e394df-eab9-4a20-92d4-c31f54aad404","sessionCode":"HTA45","topDisplay":"Sola B1, Wang T2, McAuslane N2 1Centre for Innovation in Regulatory Science, London, UK, 2Centre for Innovation in Regulatory Science, London, London, UK","locationCode":"5024","description":"\r\n\t<div>OBJECTIVES: Post-Brexit, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) pursued different reliance/work-sharing routes. This study investigates new active substances (NASs) approved by MHRA through three pathways: European Commission Decision Reliance Procedure (ECDRP), Access Consortium (Access), and Project Orbis (Orbis), and assesses the impact of these routes on Health Technology Assessment (HTA) review times and outcomes by England's National Institute for Health and Care Excellence (NICE) and Scotland's Scottish Medicines Consortium (SMC). METHODS: Data was extracted from MHRA reports, NICE guidance, and SMC advice from NASs approved by MHRA between 01-Jan-2021 to 31-Dec-2022 through ECDRP, Access, or Orbis. Time to recommendation was calculated as FDA approval to HTA recommendation, and HTA review times as time from HTA submission to recommendation. HTA recommendations were classified as positive, positive with restrictions, and negative. RESULTS: We identified 47 HTA assessments (59.6% [28/47] from NICE, 40.4% [19/47] from SMC), involving 31 unique NASs in total. The ECDRP route constituted 66.0% of all HTA assessments, followed by Orbis (12.8%) and Access (8.5%). The route of 12.8% of assessments was unclear. The mean (SD) time to recommendation was 482 days (230) for ECDRP, 256 (79) for Access, and 516 (222) for Orbis products. No difference was observed for the time to recommendation (p=0.074) or HTA review time (p=0.6) between pathways. HTA review times from SMC were significantly faster compared to NICE (p<0.001). HTA recommendations were generally evenly distributed across all pathways, with ECDRP, Access and Orbis products receiving 48%, 50%, and 67% of positive recommendations, respectively. CONCLUSIONS: The current post-Brexit regulatory landscape in the UK predominantly relies on the ECDRP. The contrast between NICE and SMC review times can relate to their different review processes. HTA recommendations were generally distributed evenly across all pathways, suggesting the reliance route does not impact HTA outcomes.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporbshta45132781-pdf.pdf?sfvrsn=fa0b411_0","title":"ISPOR_BS_HTA45132781.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132781","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Short-Run and Long-Run Behavioral Effects of Vaccination Enforcement in Turkey","id":"973775f7-8a32-4db8-95d9-c340efe03a36","sessionCode":"EPH49","topDisplay":"Baser O1, Rodchenko K2, Yapar N3, Chen L2 1City University of New York, New York, NY, USA, 2Columbia Data Analytics, New York, NY, USA, 3Columbia Data Analytics, New York City, NY, USA","locationCode":"3048","description":"\r\n\t<div>OBJECTIVES: Despite vaccine availability, only 54% of Turkey’s population received at least one dose of COVID-19 vaccine (as of August 23, 2021). The study aims to evaluate attitudes toward COVID-19 vaccination mandates and the impact of policies on vaccine adoption and future vaccination behavior utilizing self-determination theory (STD) in Turkey. METHODS: An online survey was conducted via Sogolytics among adults in Turkey between August 23, 2021, and April 5, 2022. Our primary outcome measures included: (1) psychological need satisfaction and frustration, (2) vaccination behavior, (3) willingness to get vaccinated, (4) attitudes toward COVID-19 vaccine mandates, and (5) motivations to get vaccinated or to remain unvaccinated. We conducted statistical analysis to investigate relationships between psychological needs, willingness, and intention to get vaccinated. Further, we assessed the correlation between need frustration and motivation. RESULTS: We included 430 participants in our analysis. Of 22 unvaccinated individuals, 73% said they intended to get vaccinated while 27% did not. Furthermore, participants vaccinated with at least one dose of the vaccine were highly willing to receive the vaccine. However, unvaccinated participants were hesitant to do so. Autonomy satisfaction was the strongest predictor of people’s willingness to get vaccinated (β=0.64835; p<0.0001), and relatedness satisfaction was the strongest predictor of one’s intention to get vaccinated (OR=3.597; p=0.0312). Thus, the more people felt autonomy over their decision and the more they felt cared for and understood by authorities, the stronger their willingness and intentions to receive the vaccine. Additionally, the strongest positive correlation was found between autonomy frustration and external motivation. CONCLUSIONS: Need satisfaction promotes positive vaccination behavior across Turkey’s population; however, control measures like domestic vaccine passports, which cause need frustration, may adversely affect motivation and willingness to get vaccinated.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23yapareph49poster129649-pdf.pdf?sfvrsn=4cfb0aae_0","title":"ISPOREurope23_Yapar_EPH49_POSTER129649.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129649","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Technology Assessment (HTA) Timelines Analysis in the Post-COVID Era: Comparison of HTA Process Duration in the First Half (H1) of 2023 to H1 2022","id":"fc3a55c2-6490-4250-978e-c36bd04e7fad","sessionCode":"HTA73","topDisplay":"Zlateva J1, Lalova T1, Szawara P2, Wagner P3, van Engen A4 1IQVIA, Sofia, Bulgaria, 2IQVIA, Kraków, Poland, 3IQVIA, Frankfurt, HE, Germany, 4IQVIA, Amsterdam, NH, Netherlands","locationCode":"5027","description":"\r\n\t<div>OBJECTIVES: The aim of this research was to compare if there has been any change in the duration of the Health Technology Assessment (HTA) process in the post-COVID era by comparing the median days from Marketing Authorization (MA) to publication of the HTA report in H1 2023 vs H1 2022. METHODS: We conducted a search of the HTAs to identify single drug assessments in original indications and extensions of indications published in the first half (H1) of 2023 and H1 2022. NICE, G-BA, ZIN, NoMA, CADTH, AOTMiT and Medicinrådet provide information on the analysed timepoints (regulatory approval, HTA start, decision, and publication of the HTA reports dates) in both periods and were therefore included. For HTAs from H1 2023 and H1 2022, we compared the median numbers of days from MA to HTA start (period 1), from HTA start to the decision date (period 2) and from the decision date to the publication date (period 3). RESULTS: The analysis identified 76 HTA reports for H1 2022 and 85 HTA reports for H1 2023. The duration of the total HTA process increased by 68 days (from 232 days in H1 2022 to 353 days in H1 2023). Median time in period 1 increased by 120 days in H1 2023, while periods 2 and 3 decreased by 39 and 14 days, respectively. Timelines increased in the Netherlands, Norway and the UK, although a shortening has been observed in Canada. For HTAs with negative recommendations, the total duration of HTA process increased by 128 days, while for HTAs with positive recommendations it decreased by 52 days. CONCLUSIONS: In the post-COVID era, the median time from MA to HTA report publication has increased in most countries. This delay was noted in period 1 of the HTA process and mainly for HTAs with negative recommendation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133648","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health-Related Quality of Life and Its Drivers for Patients Living With Cystic Fibrosis in Five Key European Countries: A Systematic Review","id":"0853b22a-0d57-47ca-9a7c-c422c50d56ea","sessionCode":"PCR35","topDisplay":"Verma R1, Telukuntla V2, Ahuja A3 1Lumanity, Delhi, DL, India, 2Lumanity, Delhi, India, 3Lumanity, Chandigarh, CH, India","locationCode":"6037","description":"\r\n\t<div>OBJECTIVES: Cystic fibrosis (CF) is a rare, progressive disorder affecting the lungs, pancreas, and other organs, and has a significant burden on patients' functioning and quality of life (QoL). We aimed to identify comprehensive evidence of the impact of CF on the health-related quality of life (HRQoL) in five key European countries. METHODS: We systematically searched Embase® and MEDLINE® databases via Embase.com to identify English-language articles published from 2013–2023 reporting QoL data for CF in children and adults in the UK, France, Germany, Italy and Spain. RESULTS: Among the 1,365 records screened, 22 studies were included reporting data for the UK (14), Germany (4), France (1), Spain (1), Italy (1) and all five countries (1). EQ-5D and Cystic Fibrosis Questionnaire-Revised (CFQ-R) were the most widely used QoL scales in all the studies. Mean EQ-5D utility values for participants were: 0.667 (France), 0.783 (Germany), 0.820 (Italy), 0.870 (Spain) and 0.640 (UK). EQ-5D and CFQ-R data showed that the worse the pulmonary exacerbation, the poorer the HRQoL. In addition to lung function, transplant status, age, body mass index, or having CF-related diabetes, B. cepacia complex or an implantable vascular access device affected many HRQoL domains. CF transmembrane conductance regulator modulators have shown improvements in disease progression and QoL. In a study conducted in Germany, elexacaftor/tezacaftor/ivacaftor combination improved CFQ-R respiratory domain score by 27.9 (IQR 5.6 to 47.2; p < 0.001). CONCLUSIONS: Although several factors are associated with QoL in children and adults with CF, pulmonary exacerbations and disease duration have the largest impact on HRQoL. Appropriate treatment can help in improving QoL. Further research is required to investigate the impact of other associated factors.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/pcr35qol-cfv1-0133077-pdf.pdf?sfvrsn=ceb9d48d_0","title":"PCR35_QoL CF_V1.0133077.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133077","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patient-Centered Instrument Design: Problems and Solutions Based on Patient Feedback","id":"04d1e3e7-115a-48d0-ad18-c495fd4ebeea","sessionCode":"PCR51","topDisplay":"Poepsel T1, Israel R1, Hadjidemetriou C2, Nolde A3, McKown S1 1RWS Life Sciences, East Hartford, CT, USA, 2RWS Life Sciences, Croydon, LON, UK, 3RWS Life Sciences, Chicago, IL, USA","locationCode":"6007","description":"\r\n\t<div>OBJECTIVES: Patient-centered approaches to PRO development must account for differences in understanding and experience between patients and healthcare or COA-professionals that can drive comprehension and translatability problems. Cognitive debriefing feedback of PGIs reveals repeated and serious comprehension issues which threaten accurate assessment of instruments’ target constructs and their ability to pool data across languages. We synthesize four investigations of conceptually unclear and difficult-to-translate terminology routinely found in PGIs in order to highlight problems posed to patients and data validity, and provide guidelines for improving PGI development and translation. We show that these problems arise largely from deviations from patient-centered design approaches, and suggest practical, evidence-based solutions to improve the patient experience of completing PGIs: eliminating complex medical terminology or replacing it with patient-friendly, easily-translatable terms; and simplifying ordinal response sets to reduce conceptual clustering and improve translatability. METHODS: We performed qualitative analysis of cognitive debriefing results, and thematic analysis of spontaneous feedback, from 996 patients representing 30 languages and 35 countries (aged 5-92 years; education 2-20 years) to assess comprehension of 16 PGIs. We conducted quantitative analysis of a response-option rating task from 115 separate patients (aged 18-75 years, education 8-17 years) from 12 English-speaking locales. RESULTS: We identified terms that cause comprehension problems (e.g., disease activity, global) for a significant proportion of patients, languages, and countries in our samples (25%+), and further note differential comprehension based on patient language and culture. We additionally found that larger ordinal response sets (7 vs 5-item) are 3 times more likely to cause comprehension problems, and provide quantitative data showing how patients perceive the conceptual spacing of common PGI response options. CONCLUSIONS: Despite their brevity, PGIs are prone to many design, conceptual, and translation problems. Patient-centered approaches, which avoid needlessly complex phrasing and improve translatability, are essential for improving the patient experience and increasing data validity.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/rwsispor841x1189posterpatientinstrumentdesignv2133742-pdf.pdf?sfvrsn=1f219b_0","title":"RWS_ISPOR_841x1189_POSTER_PATIENTINSTRUMENTDESIGN_v2133742.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133742","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Endometrial Cancer Care in Colombia: A Cost-of-Illness Study Based on Administrative Claims Databases","id":"1a174056-890c-4806-a33b-c4a290d681bd","sessionCode":"EE58","topDisplay":"Buitrago G1, Torres GF1, Rodriguez E2, Gonzalez Rangel A3 1Universidad Nacional de Colombia, Bogotá, Colombia, 2GlaxoSmithKline, Bogotá, Colombia, 3GlaxoSmithKline, Bogota, CUN, Colombia","locationCode":"2003","description":"\r\n\t<div>OBJECTIVES: Endometrial cancer (EC) is associated with substantial healthcare costs on a global scale; however, cost data from low- and middle-income countries are limited. Here, we describe healthcare costs borne by the health system and incurred by patients with EC affiliated with the contributory regime in Colombia. METHODS: We retrospectively identified a cohort of incident patients with EC from patient-level data in national administrative databases. Included patients were required to have a first recorded EC diagnosis between 1 January 2014–31 December 2015 (index). Healthcare costs incurred by patients from index to either death or 31 December 2019 were assessed. We used Kaplan-Meier sample-average estimators with monthly intervals to adjust for differences in survival and duration of follow-up. All costs are annual and shown using survival-adjusted medians with interquartile ranges (IQR) and are reported in 2019 international dollars. To identify characteristics associated with higher care costs, we used generalized linear models, with a squared-root link function and a Poisson distribution. RESULTS: In total, 833 patients with EC (mean age [standard deviation] 59.4 [12.5] years) were identified. The median costs of healthcare services were $9,101 (IQR $8,920 to $9,229), ranging from $7,941 (IQR $7,281 to $8,396) during the first year to $304 (IQR $285 to $322) the fifth year following diagnosis. Most of the costs were related to outpatient (63.5%), and inpatient (33.2%), distributed respectively on consultations (7.2%, 0.3%), medications (33.4%, 7.5%), and procedures (59.5%, 92.2%). We found that an older age at diagnosis, a higher comorbidity burden, and a higher proportion of services paid under fee for services schemes were associated with increased healthcare costs. CONCLUSIONS: EC is associated with substantial healthcare costs in the population affiliated with the contributory regime in Colombia. Besides clinical and demographic characteristics, how providers are paid remains a strong predictor of healthcare costs in EC. Funding: GSK (study 218730) </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gonzalezee58poster130930-pdf.pdf?sfvrsn=7deec54e_0","title":"ISPOREurope23_Gonzalez_EE58_POSTER130930.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130930","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"anti-VEGFs Use in Neovascular Age-Related Macular Degeneration: An Analysis of the National Report on Medicines Use in Italy","id":"2bca2dd0-84d8-4d0b-8260-c4afaaba9e26","sessionCode":"EE105","topDisplay":"Ghetti G1, Porta C1, Fasci A2 1AdRes HEOR, Torino, TO, Italy, 2Roche Spa, Monza, MB, Italy","locationCode":"2041","description":"\r\n\t<div>OBJECTIVES: Neovascular age-related macular degeneration (nAMD) is the leading cause of vision loss in the elderly, negatively affecting patients’ quality of life. Anti–vascular endothelial growth factor therapies (anti-VEGFs) are the current standard of care for nAMD. However, real-world studies revealed suboptimal outcomes due to the frequent need for therapeutic interventions, burdening patients and healthcare systems. The objective of this study was to analyze the consumption of anti-VEGFs in Italy and present potential strategies to enhance treatment appropriateness. METHODS: National consumption data were used. Approved anti-VEGFs differ in their ability to provide extended treatment intervals: <7 (IVT<7), 7 (IVT7), and >7 (IVT>7) injections/year, according to Nota 98 prescription appropriateness report. Appropriateness was defined as the ratio of the observed annual mean injections number with Nota 98 report and Summary of Product Characteristics value. National treatment acquisition and administration costs were considered. Two scenarios were investigated: (1) maximizing appropriateness while maintaining fixed expenditure, (2) minimizing expenditure to achieve 80% appropriateness level. RESULTS: 27,401 eyes started an approved anti-VEGF therapy in Italy in 2021: 33.5% received IVT>7, 57.0% received IVT7, and 9.5% received IVT<7. The estimated appropriateness was 46.6%, with a total expenditure of €93.9 million for 100,851 injections. Greater use of IVT<7 products can increase appropriateness to 57.7%, resulting in a reduction of both injections (-2.1%) and expenditure (-6.7%). Achieving 80% appropriateness required higher injection capacity (+35.8%) and increased expenditure (+29.6%). Additionally, considering anti-VEGF with <6 injections/year (IVT<6), achieving 80% appropriateness required less investments (+22.9%) and only a modest increase of injections (+26.6%). CONCLUSIONS: Greater use of IVT<7 products can enhance appropriateness (+11.1%) in the fixed expenditure scenario. Achieving 80% appropriateness depends on the capacity of treatment centers and the financial feasibility for payers. The availability of IVT<6 products resulted in achieving 80% appropriateness with the lowest economic effort and a limited increase in injections.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23portaee105poster131809-pdf.pdf?sfvrsn=dca0181_0","title":"ISPOREurope23_Porta_EE105_POSTER131809.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131809","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Modeling the Impact of the Vaccine Candidate TAK-003 on the Incidence of Dengue in French Overseas Territories","id":"47d3d717-dbc4-482d-9b1a-c6deb52d30e7","sessionCode":"EPH47","topDisplay":"Nucit A1, Moreau R2, Massetti M3, Cedric S4, Kharitonova E5, Tytula A6, Zawieja J6, Leleu H7, Shen J8 1Takeda France SAS, Paris, 75, France, 2Public Health Expertise, Paris, 75, France, 3Public Health Expertise, PARIS, 75, France, 4Takeda France SAS, Paris, France, 5Putnam PHMR, Paris, France, 6Putnam PHMR, Krakow, Poland, 7Public Health Expertise, Paris, France, 8Takeda Pharmaceuticals International AG, Zürich, ZH, Switzerland","locationCode":"3047","description":"\r\n\t<div>OBJECTIVES: Dengue is a mosquito-borne viral infection causing a flu-like illness which can potentially be severe or even fatal. With over 2 million people living in French endemic overseas territories (OT), better response to epidemic outbreaks and imported cases to mainland France have become a rising concern for health authorities. This study aims to estimate the impact of vaccination with the candidate dengue vaccine TAK-003 in French overseas territories. METHODS: Modeling of the impact of TAK-003 was performed using a static model with dynamic component to account for the indirect effect of vaccination. The underlying Markov process included 16 health states capturing the cohorts’ dengue history (from dengue-naïve to history of four infections), natural cross-protection and presence of symptoms and hospitalization. The model was fitted to local age-specific incidence rate of symptomatic reported dengue (averaged over the period 2013-2021) for each OT. Probability of dengue infection was differentiated by age and infection order (first to fourth). The considered time horizon was 10 years. An interventionist strategy allowing respective vaccine coverage rates (VCR) of 80% and 60% for the routine vaccination of 6-year-old and catch-up of 7-18 aged children and an opportunistic strategy with 20% VCRs for both cases were compared to the absence of vaccination in terms of avoided symptomatic infections, hospitalizations, and severe cases. Funding: Takeda. RESULTS: Over 10 years, the vaccination of 309,687 (resp. 89,645) people through the interventionist (resp. opportunistic) strategy on all OT, allowed TAK-003 to reduce the number of symptomatic infections, hospitalizations, and severe cases by 18%, 22% and 25% respectively (resp. 5%, 5% and 7%) when compared to no vaccination. CONCLUSIONS: This study shows that the vaccination of 6-18 years aged children with TAK-003 in French endemic regions can lead to significant reduction in symptomatic infections, hospitalizations, and severe cases potentially also reducing imported cases in mainland France.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23nuciteph47poster133016-pdf.pdf?sfvrsn=71778792_0","title":"ISPOREurope23_Nucit_EPH47_POSTER133016.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133016","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Opportunities and Challenges in Linkage of Early Access Databases With the French National Claims Database: A RWE Effectiveness Study of AZD7442 for Pre-Exposure Prophylaxis Against COVID-19 in Immunocompromised Patients","id":"a26d175c-4b27-4500-8ae1-c7ef1f29e0f6","sessionCode":"RWD27","topDisplay":"Luong L1, Jannot AS2, Burdet C3, Majed L4, Fabry-Vendrand C4, Cerceau T5, Bouee S6, Dube S7, Taylor S7, Thabut G4, Artaud C4 1CIC Cochin Pasteur, Paris, France, France, 2Assistance Publique Hôpitaux de Paris-Centre (AP-HP), Georges Pompidou European Hospital, Paris, France, France, 3INSERM, Paris, France, 4Astra Zeneca, Courbevoie, France, 5CEMKA, BOURG LA REINE, France, 6CEMKA, Bourg-la-Reine, France, 7AstraZeneca, Cambridge, Cambridgeshire, UK","locationCode":"7001","description":"\r\n\t<div>OBJECTIVES: In 2021, the French Health Authority recommended new guidelines for use of Early Access Programs (EAPs) linked with the French national claims database (SNDS, for ‘Système National des données de santé’), which covers 99% of the French population. Few studies have been conducted so far. Here, we describe challenges and opportunities of this innovative approach, using the EAP for AZD7442, a combination of 2 neutralizing antibodies authorized in 2021 for the prevention of COVID-19 in immunocompromised individuals. METHODS: Two databases will be linked: <ul> <li>The EAP database which includes the identification of patients who were treated with AZD7442 and the description of patients.</li> <li>The SNDS claims database to be used to identify outcomes of interest (e.g., overall deaths and hospitalizations and due to COVID-19 infection)</li> </ul> RESULTS: Out of the 27,782 patients enrolled in the EAP, treatment administration forms were completed for 13,387 (47.8%). AZD7442 was mostly prescribed by onco-hematologists (24.2%), followed by nephrologists (19.9%), internal medicine specialists (15.6%), and rheumatologists (6.5%). The majority (96.7%) had received at least three vaccine doses at inclusion. Most common comorbidities were cancer (34.9%), chronic kidney disease (23.7%), hypertension (23.2%), cardiovascular disease (19.4% >2 CV factors), and diabetes (9.2%). A matched control cohort will be extracted from SNDS. The matching will be based on 2 variables: <ul> <li>A Propensity based on calendar time, immune category, and confounding clinical characteristics.</li> <li>Comorbidities for which AZD7442 was prescribed.</li> </ul> CONCLUSIONS: The study will assess the effectiveness of AZD7442 using an innovative, large observational comparative study combining EAP data and SNDS. The reuse and enrichment of the EAP database linked to SNDS will provide valuable insights for future recommendations on COVID-19 prophylaxis in immunocompromised patients and this method could set the path for new RWE effectiveness studies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023boueerwd27poster132608-pdf.pdf?sfvrsn=fb99ea39_0","title":"ISPOREurope2023_BOUEE_RWD27_POSTER132608.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132608","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Cost-Effectiveness Analysis of Atezolizumab as Adjuvant Treatment Following Complete Resection and Platinum-Based Chemotherapy in Adult Patients With Early-Stage Non‑Small Cell Lung Cancer With a High Risk of Recurrence","id":"e15a47b6-8a41-42f8-aef3-c8c2ea972db0","sessionCode":"EE78","topDisplay":"Silva Miguel L1, Pinheiro B1, Carvalho P2, Jovanoski N3, Belleli R3, Abogunrin S3, Alves P4, Araújo A5, Barata F6, Hespanhol V7, da Luz R8, Borges M9 1IQVIA Portugal, Salvo, Oeiras, 13, Portugal, 2IQVIA Portugal, Lisboa, Portugal, 3F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland, 4Centro Hospitalar Universitário Lisboa Norte, Lisboa, Portugal, 5Centro Hospitalar Universitário de Santo António, Porto, Portugal, 6Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal, 7Centro Hospitalar Universitário de S. João, Porto, Portugal, 8Centro Hospitalar e Universitário Lisboa Central, Lisboa, Portugal, 9Laboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal","locationCode":"2017","description":"\r\n\t<div>OBJECTIVES: To assess the cost-effectiveness of atezolizumab as adjuvant treatment, compared to best supportive care (BSC), following complete resection and platinum-based chemotherapy in adult patients with early-stage non-small cell lung cancer (NSCLC) at high risk of recurrence, whose tumors have programmed death-ligand 1 (PD-L1) expression ≥50%, excluding epidermal growth factor receptor-mutant or anaplastic lymphoma kinase-positive NSCLC. METHODS: A Markov model including disease-free survival (DFS), locoregional recurrence, metastatic recurrence (1st and 2nd lines), and death health states was used. The model uses data from IMpower010 (atezolizumab’s pivotal trial in this therapeutic indication) for DFS and from external sources for the remaining health states. As patient reported outcomes were not collected during IMpower010, utility scores are based on the literature and on IMpower150. The use of this trial, in which atezolizumab was evaluated in metastatic NSCLC patients, allowed estimating EQ-5D-3L scores with Portuguese tariffs for the respective health states and standardizing the scores available from the literature. Portuguese-specific disease management resource use was based on an experts panel and Portuguese diagnosis-related group microdata. The main sources for unit costs were national legislation and official drug cost databases. The analysis was conducted from the National Health Service perspective, assuming a lifetime horizon and a 4% discount rate. Deterministic and probabilistic sensitivity analyses show that results are robust. RESULTS: Atezolizumab increases average life expectancy by 2.25 discounted life years (LY) or 1.64 discounted quality adjusted life years (QALY), with an incremental cost of 11,732€ that is due to higher treatment costs in DFS. The estimated incremental cost-effectiveness ratios (ICER) are 5,212€ per LY and 7,139€ per QALY. Deterministic sensitivity analyses show that results are robust. CONCLUSIONS: Treatment with atezolizumab allows for an increase of LY and QALY compared to BSC, with an ICER that is usually considered cost-effective in the Portuguese setting.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23silvamiguelee78poster132925-pdf.pdf?sfvrsn=949478d3_0","title":"ISPOREurope23_SilvaMiguel_EE78_POSTER132925.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132925","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of Abrocitinib Versus Dupilumab for Treatment of Moderate-to-Severe Atopic Dermatitis in Chinese Adults","id":"dab7f789-b908-4ae8-971b-c94a9cd7b5bd","sessionCode":"EE108","topDisplay":"Jiang Y1, Zuo C2, Sun Y1, Qiu J3, Bi H1, Xuan J4 1Southern Medical University, Guangzhou, Guangdong, China, 2Shanghai Centennial Scientific Co. Ltd, Guangzhou, 44, China, 3Southern Medical University, Guangzhou, 44, China, 4Health Economics Institute, School of Pharmaceutical Sciences, Sun Yat-Sen University, Guangzhou, China","locationCode":"2040","description":"\r\n\t<div>OBJECTIVES: To evaluate cost-effectiveness of abrocitinib versus dupilumab for treating moderate-to-severe atopic dermatitis in Chinese adults from the societal perspective. METHODS: A decision tree combined with a Markov model was developed to estimate 5-year costs and quality-adjusted life-years (QALYs) for patients with moderate or severe atopic dermatitis. The Markov model was divided into three states: maintenance, best-supportive care, and death. Patients with Eczema Area and Severity Index (EASI) scores ≥75 or EASI scores ≥90 were defined as the responders and stayed in the maintenance state. The model assumed no increased mortality risk associated with atopic dermatitis compared to the general population. The transition probabilities were derived from the JADE and SOLO-CONTINUE clinical studies, which included the Asian population. Direct medical costs, indirect costs, and utility values were derived from the published literature or public databases. One-way sensitivity and probabilistic sensitivity analyses were also conducted to examine the impact of uncertainty of the parameters. RESULTS: For patients with moderate atopic dermatitis, compared to dupilumab, abrocitinib led to a gain of 0.14 QALYs at additional cost of ¥39,070, resulting in an ICER of ¥275,626 per QALY gained. For patients with severe atopic dermatitis, abrocitinib showed an incremental gain of 0.23 QALYs at additional cost of ¥30,367, resulting in an ICER of ¥131,308/QALY. Abrocitinib exhibited a higher number of response-years compared to dupilumab over five years (1.77 vs. 1.18). Sensitivity analyses indicated that the model outputs were most sensitive to the utility of EASI-90 state and drug prices. At a willingness-to-pay threshold of ¥257,094/QALY (3 times of GDP-per-capita in China), abrocitinib was more likely to be cost-effective for severe patients than moderate patients. CONCLUSIONS: Abrocitinib offers more durable clinical efficacy and quality of life gains for patients with moderate-to-severe atopic dermatitis compared to dupilumab in China and is considered cost-effective for severe patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ad-poster-231016130515-pdf.pdf?sfvrsn=8692c59_0","title":"AD poster-231016130515.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130515","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical and Economic Impact of Pre-Exposure Prophylaxis With Tixagevimab+Cilgavimab in 2022 in Switzerland to Protect Immunocompromised Individuals","id":"c0f147cf-2a76-4c80-9a5d-c969eae3be7b","sessionCode":"EE29","topDisplay":"Rivolo S1, Bungey G2, Morris W3, Nagy E4, Williams S5, Maillat L6, Jańska A7, Arnetorp S8 1Evidera, a part of Thermo Fisher Scientific, San Felice Segrate, MI, Italy, 2Evidera, a part of Thermo Fisher Scientific, London, London, UK, 3Evidera, a part of Thermo Fisher Scientific, Cambridge, Cambridgeshire, UK, 4Evidera, a part of Thermo Fisher Scientific, Liverpool, Merseyside, UK, 5AstraZeneca, Zurich, Zurich, Switzerland, 6AstraZeneca, Lausanne, Vaud, Switzerland, 7AstraZeneca, Cambridge, Cambridgeshire, UK, 8AstraZeneca, Gothenberg, Västergötland, Sweden","locationCode":"1079","description":"\r\n\t<div>OBJECTIVES: The risk of severe COVID-19 (hospitalisation or death) during the Omicron waves remains significant in immunocompromised individuals (ICIs), despite vaccine boosters. Passive immunisation through pre-exposure prophylaxis (PrEP) can protect ICIs. This analysis uses tixagevimab+cilgavimab, made available in May 2022 in Switzerland, as a case study to quantify the clinical and economic impact of PrEP and compares it to alternative scenarios (broader ICI coverage, earlier access or both). METHODS: A combined decision tree–Markov model evaluated health and economic outcomes of introducing tixagevimab+cilgavimab in Switzerland in May 2022 and compared it to different hypothetical scenarios. The “real-life” scenario considered 6-month protection of one tixagevimab+cilgavimab dose with 15% uptake among 10,000 PrEP-eligible ICIs (13% 6-month infection risk). Hypothetical scenarios: 1) Broader coverage (one dose, 100% uptake, 13% 1-year infection risk), 2) Earlier access (two doses, 15% uptake, 30% 1-year infection risk) and 3) Broader coverage and earlier access (two doses, 100% uptake, 30% 1-year infection risk). Model inputs were informed by Omicron-specific literature. Extensive sensitivity analyses were conducted. RESULTS: Real-life scenario: tixagevimab+cilgavimab prevented 147 symptomatic cases (saved Swiss Francs [CHF] 434,939), 13 hospitalisations (CHF 284,993), 119 bed days (CHF 186,412) and 28 post-acute COVID-19 cases (CHF 209,469). Earlier access or broader ICI coverage would have prevented 2.3 and 6.7 times more COVID-19–related outcomes and healthcare-related costs, respectively. Both combined would have prevented 15.5 times more COVID-19–related outcomes (2280 symptomatic cases, 210 hospitalisations, 1848 bed days and 442 post-acute COVID cases), with CHF 2,894,988–6,754,608 potential saving in healthcare-specific resources. CONCLUSIONS: Tixagevimab+cilgavimab significantly reduced the COVID-19 disease burden in ICIs in Switzerland upon introduction in May 2022. However, tixagevimab+cilgavimab impact could have been more substantial with more timely access and broader ICI coverage. This should be considered when designing future strategies to optimally protect the immunocompromised population. Funding: AstraZeneca</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023rivoloee29poster132809-pdf.pdf?sfvrsn=75ff388c_0","title":"ISPOREurope2023_Rivolo_EE29_POSTER132809.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023rivoloee29suppl132809-pdf.pdf?sfvrsn=3223340f_0","title":"ISPOREurope2023_Rivolo_EE29_SUPPL132809.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132809","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimating the Projected Impact of Weight Loss on Obesity Prevalence and Healthcare Costs in a UK Population","id":"55ac356e-f784-42c4-a545-c988627c5369","sessionCode":"EPH7","topDisplay":"Grasic K1, Holloway S2, Capucci S3 1Lane Clark & Peacock LLP, London, UK, 2Lane Clark & Peacock LLP, Winchester, HAM, UK, 3Novo Nordisk A/S, Søborg, Denmark","locationCode":"3009","description":"\r\n\t<div>OBJECTIVES: People living with obesity typically incur higher healthcare costs than those with healthy weight, and these costs increase with obesity severity. We estimated the impact of weight loss in a UK population on the prevalence of obesity classes and associated healthcare costs. METHODS: The analysis considered adults aged 20–<70 years, defined according to body mass index (BMI) as having obesity class I, II or III (30–<35 kg/m2, 35–<40 kg/m2 or ≥40 kg/m2, respectively). The estimated prevalence of each obesity class in the UK in 2033 was derived from the 2022 World Obesity Atlas, combined with 2021 Health Survey for England obesity estimates and 2019 data from the Discover electronic health record data set, which covers 2.8 million people living in North West London. The effects of 10% weight loss on the number of people in each obesity class and associated healthcare costs were estimated using the BMI distribution and costs observed in the 2019 Discover data, and scaled to the UK population. RESULTS: Weight loss of 10% was estimated to result in a 47% reduction in the number of people with obesity in 2033, equating to 8.1 million fewer people in the UK <70 years old living with obesity (4.8, 2.0 and 1.3 million in obesity classes I, II and III, respectively). The estimated mean per-person annual cost reduction was £350 for moving from obesity class III to II, £207 for moving from obesity class II to I and £171 for moving from obesity class I to overweight. This would result in an estimated £2.5 billion annual cost saving, mostly contributed by people moving from obesity class I to overweight. CONCLUSIONS: Our estimates of the obesity prevalence and cost reductions that could be achieved via weight loss highlight the potential impact of targeted weight management support.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/grasic-et-al-value-of-weight-lossispor-poster-227-oct-23for-upload129218-pdf.pdf?sfvrsn=c7205a2c_0","title":"Grasic et al._Value of weight loss_ISPOR poster 2_27-Oct-23_for upload129218.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129218","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Assessing COA Label Claims Based on Single-Arm Trials in Rare Diseases: A Review of US FDA Labels","id":"4f752ae2-d1a3-4316-95d4-c9af4c1913fc","sessionCode":"HPR12","topDisplay":"Thakker DN1, Dubucq H2, Bozzi S3, Arnould B4, Jasso-Mosqueda JG3 1Sanofi, BANGALORE, KA, India, 2Sanofi, Barcelona, Spain, 3Sanofi, Chilly-Mazarin, France, 4Sanofi, Lyon, France","locationCode":"3069","description":"\r\n\t<div>OBJECTIVES: According to the 2009 FDA Patient Reported Outcome (PRO) guidance, one of the study design requirements for PRO labelling claims is a randomized double-blind clinical trial. However, in the context of rare diseases (RD), registration trials are frequently conducted without comparator arm. The aim of this review is to assess experience of clinical outcomes assessment (COA)/PRO with regards to label claims, using single-arm studies in the RD area. METHODS: The US FDA labels considering single arm trials in the RD area were identified through the Prismaccess™ platform (IQVIA) from inception. For labels including COA, the outcome was further categorized as a clinician-reported outcome (ClinRO), PRO, or performance outcome (PerfO). For these labels, the 'Indications and Usage' section as well as the 'Section 14 Clinical Studies' of each label were examined. The assessment of these label sections determined whether a COA /PRO label claim was granted for these molecules. RESULTS: As of April 6th, 2023, a total of 92 FDA labels for molecules that had FDA submissions based on single-arm studies in the RD field were found. Only four out of 92 included COA, three were ClinRO and one was PerfO. None was a PRO. The US FDA did not grant any PRO label claims, however in exceptional cases, ClinRO or PerfO label claim were awarded to products that underwent submission based on single-arm trials in RD. CONCLUSIONS: In RD area where non-conventional study design is accepted by regulatory bodies, it would be important to increase the recognition of ClinRO, PerfO and PRO label claims in order to better inform prescribers and patients about clinical benefits expected from innovative treatment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23thakkerhpr12poster132681-pdf.pdf?sfvrsn=664e09d_0","title":"ISPOREurope23_Thakker_HPR12_POSTER132681.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132681","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Broadening the HTA of Medical AI: A Review of Policy Reports to Inform Decision Makers","id":"8dee113e-3647-4090-a418-c9bb9b6172fb","sessionCode":"HTA44","topDisplay":"Boverhof BJ1, Redekop K2, Visser JJ3, Uyl-De Groot C4, Rutten-van Mölken M5 1Erasmus University, Rotterdam, ZH, Netherlands, 2Erasmus University Rotterdam, Rotterdam, Zuid Holland, Netherlands, 3Erasmus Medical Centre, Rotterdam, Rotterdam, Netherlands, 4Erasmus University, Rotterdam, Netherlands, 5Erasmus University Rotterdam, Rotterdam, ZH, Netherlands","locationCode":"5003","description":"\r\n\t<div>OBJECTIVES: As current health technology assessment (HTA) frameworks do not provide specific guidance on the assessment of medical artificial intelligence (AI), this study aimed to propose a conceptual framework for a broad HTA of medical AI. METHODS: A targeted literature search of policy documents was conducted to distill the medical AI issues relevant for impact-assessment. Publications by national (UK, ) and supranational (European Union) governing bodies, non-profit organizations and academic institutions were obtained from their websites and through a Google search. Three exemplary cases were selected to illustrate the relevance of the extracted issues: (1) An application supporting radiologists in stroke-care (2) A natural language processing application for clinical data abstraction (3) An ICU-discharge decision-making application. RESULTS: A total of 31 publications were selected, from which a long-list of extracted issues was reduced to a short-list of 25 issues, based on an iterative deductive and inductive approach. The issues were grouped by four focus areas: (1) Performance & Evidence, (2) Human & Organizational, (3) Legal & Ethical and (4) Transparency & Usability. Although some issues were relevant to all three case studies (e.g., clinical effectiveness, cost-effectiveness), they also showed variability, with workflow issues being particularly important in stroke-care, generalizability in clinical data abstraction and explainability & fairness in ICU-discharge decision-making. CONCLUSIONS: The current methodology of HTA requires extension to make it suitable for a broad evaluation of medical AI technologies. The application of the 25-item assessment list that we propose should be tailored to the intervention being assessed, since the case studies showed that conceptualization of the issues differs across applications.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23boverhofhta44poster130773-pdf.pdf?sfvrsn=d78239e0_0","title":"ISPOREurope23_Boverhof_HTA44_POSTER130773.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130773","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Comparison of the UK and Australian Interim Value Sets for the Weight-Specific Adolescent Instrument for Economic Evaluation (WAItE)","id":"7e6662f0-5b0d-4ef9-8d9b-ca7127b5d246","sessionCode":"PT1","topDisplay":"Robinson T1, Hill S2, Chen G3, Oluboyede Y4 1Newcastle University, Newcastle, UK, 2Putnam PHMR Ltd., London, LON, UK, 3Monash University, Melbourne, VIC, Australia, 4Putnam PHMR Ltd., Newcastle Upon Tyne, NT, UK","locationCode":"1A","description":"\r\n\t<div>OBJECTIVES: The Weight-Specific Adolescent Instrument for Economic Evaluation (WAItE) is a weight-specific Health-Related Quality of Life (HRQoL) measure, containing seven dimensions with five severity levels. Two valuation studies (in the UK and Australia) have recently been conducted. This study aimed to compare the preferences from these valuation studies. METHODS: Discrete Choice Experiments (DCEs) were conducted with adults in the UK (n=1,005) and Australia (n=1,005). DCE data were analysed using a mixed logit model. The interim UK value set was anchored onto the Quality Adjusted Life Year (QALY) scale using an external Time Trade-Off (TTO) study and the visual analogue scale (VAS). The interim Australian value set was anchored onto the QALY scale using the VAS method and the ‘Comparisons with Death’ (CWD) approach. Relative Attribute Importance (RAI) scores were calculated for both samples. RESULTS: Inconsistencies were observed for certain dimensions in both the UK and Australian samples, and therefore some levels were combined to ensure monotonicity within dimensions. RAI scores showed the same dimension was valued as the least important in both samples, however, the most important dimensions differed. In the UK sample, the different anchoring methods generated similar value sets. Values for the ‘PITS state’ (the worst health state possible from the WAItE classification system) were 0.230 and 0.289 using the TTO and VAS anchoring methods respectively. Conversely, the interim value sets generated using each anchoring method in the Australian sample were quite different, as the values for the PITS state were quite different. The values for the PITS state were 0.429 and -0.030 when using the VAS and CWD anchoring methods respectively. CONCLUSIONS: The UK and Australian interim value sets for the WAItE display several similarities, yet the values for the PITS state are not comparable. The choice of anchoring approach influences the results in both samples.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/yemi-olubooyede2023a-comparison-of-the-uk-and-australian-interim-value-sets-for-the-weight-specific-adolescent-instrument-for-economic-evaluation131338-pdf.pdf?sfvrsn=de59d1d0_0","title":"Yemi Olubooyede_2023_A Comparison of the UK and Australian Interim Value Sets for the Weight-Specific Adolescent Instrument for Economic Evaluation131338.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131338","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Review to Assess How Cure Is Modelled and Subsequently Evaluated in Health Technology Submissions to the National Institute for Health and Care Excellence (NICE)","id":"a1aaffd0-c828-45cc-870a-cb921066e979","sessionCode":"SA15","topDisplay":"Wigfield P1, Sillem E2, Heeg B3 1Ingress-Health, Rotterdam, ZH, Netherlands, 2Cytel, The Hague, ZH, Netherlands, 3Cytel, Rotterdam, Netherlands","locationCode":"7024","description":"\r\n\t<div>OBJECTIVES: Extrapolating clinical trial data is crucial for estimating long-term costs and consequences of new interventions. However, assessing the direct impact of cure on survival based solely on clinical trial results, which often focus on progression-free survival rather than overall survival, can be challenging. This research examines the modelling of cure in technology submissions to NICE. METHODS: A systematic literature review was conducted, analysing published technology appraisal guidance submitted to NICE by May 2023, mentioning 'cure' in the submission dossier. The following extraction items were included: intervention, year, indication, model structure, approach and rationale for modelling cure, maturity of cure endpoint, payer feedback, and decision outcome. RESULTS: Among 83 appraisals mentioning 'cure,' 42 included cure in their health economic models. Various disease indications were assessed, with the majority focusing on leukaemia (10/42), lymphoma (8/42), and lung cancer (6/42). Model structures comprised partitioned survival models (22/42), state transition models (18/42), and hybrid models (2/42). Mixture and non-mixture cure models were employed in 13/42 submissions. General population mortality equivalence was assumed in 40/42 submissions, with or without standardized mortality ratio application, while 2/42 submissions incorporated cure by assuming absence of treatment waning. 29/42 submissions mentioned that the possibility of cure had been validated by clinical experts. In only 13/42 appraisals, both the ERG and Committee aligned with the company's approach to modelling cure. CONCLUSIONS: Heterogeneity exists in how cure is modelled in technology submissions, as well as the supporting evidence for these assumptions. The findings suggest that, in general, the possibility of cure is supported by expert opinion. However, the extent to which cure is rooted in the clinical rationale for the respective interventions rather than driven by health economic considerations remains unclear. There is a need for more detailed methods incorporating statistical, clinical, and empirical evidence in the modelling of cure within technology submissions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23wigfieldsa15poster132765-pdf.pdf?sfvrsn=883989d6_0","title":"ISPOREurope23_Wigfield_SA15_POSTER132765.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132765","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Investigating the Impact of Early Versus Late Diagnosis in Fabry Disease on Healthcare Resource Utilization and Associated Costs in Greece","id":"f1af2fb1-1cb6-440d-944c-cc02990578da","sessionCode":"HSD15","topDisplay":"Naoum P1, Anastasakis A2, Dounousi E3, Hill M4, Kakavaki E4, Kaliontzoglou A5, Karamanis A4, Karasavvidou D6, Margaros I4, Mavrommati C4, Paliouras C5, Petras D7, Roussou D4, Stylianou K8, Tsivgoulis G9, Athanasakis K10 1Institute for Health Economics, Athens, Athens, Greece, 2Onassis Cardiac Surgery Center, Athens, Attiki, Greece, 3University of Ioannina, Ioannina, Ipiros, Greece, 4Takeda Hellas SA, Athens, Attiki, Greece, 5General Hospital of Rhodes, Rhodes, Dodekanisa, Greece, 6General Hospital of Ptolemaida “Mpodosakeio”, Ptolemaida, Kozani, Greece, 7“Hippokration” General Hospital, Athens, Attiki, Greece, 8Heraklion University Hospital, Heraklion, Crete, Greece, 9National & Kapodistrian University of Athens, “Attikon” University Hospital, Athens, Attiki, Greece, 10University of West Attica, Athens, Attica, Greece","locationCode":"4025","description":"\r\n\t<div>OBJECTIVES: To assess the differences in healthcare resource utilization and associated costs between patients with early versus late clinical diagnosis of Fabry disease (FD). METHODS: Due to scarcity of relevant local clinical data, the expert panel methodology was employed. A structured questionnaire was developed, concerning patient characteristics and resource use (medication, physician visits, tests, hospitalization, dialysis). Cost data derived from local official sources and the international literature. RESULTS: The expert panel - consisting of 8 hospital physicians - indicated nephrology as the most utilized medical specialty (followed by cardiology and neurology). On average, classical FD patients made 2.64 visits annually in early and 7.56 visits in late diagnosis to nephrologists. The respective visits of non-classical FD were 1.69 and 2.63. Agalsidase alfa is the medication with the highest proportion of utilization in all patient groups. In a 5-year time frame, 26.88% of classical FD patients with early and 43.13% with late diagnosis are expected to be hospitalized, while 2.13% and 10.63%, respectively, are expected to require admission to Intensive Care Unit (ICU). For non-classical FD, these proportions are 17.75% in early and 30.63% in late, respectively, for hospitalization in general ward and 1.25% in early and 5.75% in late for ICU. Lastly, 17.00% of early and 29.00% of late diagnosed classical FD patients and 7.50% of early and 14.00% of late diagnosed non-classical FD patients are estimated to be on dialysis. The mean weighted annual cost of late diagnosis patients is higher by 7,404.78€ in classical (156,572.64€ in early vs 163,997.42€ in late) and 3,538.74€ in non-classical FD (152,980.07€ in early vs 156,518.81€ in late). The main cost contributor in all patient groups is medication (>88%), followed by dialysis. CONCLUSIONS: This study highlights the importance of early diagnosis in FD, since late diagnoses resulted in higher healthcare resource use and costs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23naoumhsd15poster132580-pdf.pdf?sfvrsn=9bd9b7a3_0","title":"ISPOREurope23_Naoum_HSD15_POSTER132580.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132580","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Prevalence and Predictive Factors of Severe Coronary Lesions in Algerian Patients Undergoing Coronary Angiography","id":"9a50f0bf-2644-4cc1-8e82-cc1480a3857b","sessionCode":"EPH5","topDisplay":"Boukheloua M1, Berrehal M2, Baali A1, Chelghoum S3, Mahi L4, Nibouche D1 1CHU Nafissa Hamoud, Alger, Algeria, 2CHU Saadna Mohamed Abdenour, Sétif, Algeria, 3CHU Mustapha Bacha, Alger, Algeria, 4Axelys Santé DZ, Algiers, Algeria","locationCode":"3006","description":"\r\n\t<div>OBJECTIVES: The severity of coronary lesions in patients with coronary artery disease (CAD) has important prognostic and therapeutic consequences. Clinical symptoms not always reflect the disease severity and this study aimed to evaluate coronary lesions in the Algerian population where these data are scarce. METHODS: We conducted a prospective cross-sectional study in consecutive patients with an age ≥20 years, who underwent clinically-indicated coronary angiography in our center. The objective of the study was to determine the prevalence of severe coronary lesions assessed with the Gensini score. The predictive factors of severity were evaluated in a multivariate analysis. RESULTS: A total of 507 patients were included (male, 79.7%; mean age, 58.8 years). Patients were most frequently referred for coronary angiography for ST-segment elevation myocardial infarction (STMI) (46.9%) and non-STMI (38.1%). The prevalence of severe coronary lesions was 69.6% (95% CI 65.5–73.5). In multivariate analysis, the independent predictive factors of severe coronary lesions were male sex (odds ratio [OR] 2.00; p=0.0141), diabetes (OR 1.92; p=0.0070), left ventricular dysfunction (OR 1.81; p=0.0059), age (OR 1.72; p=0.0297) and no lipid-lowering treatment (OR, 0.47; p=0.0388). CONCLUSIONS: Severe coronary lesions were present in two out three patients in this cohort of Algerian patients undergoing coronary angiography. Independent predictive factors of severe coronary lesions were male sex, diabetes, age, left ventricular dysfunction and no lipid-lowering treatment. It is important to identify these at-risk patients, as they should be explored at an early asymptomatic stage, before any cardiovascular event, and receive prompt treatment with angioplasty or surgery.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129169","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Micro-Costing Analysis for the Treatment of Atrial Fibrillation: An Economic Evaluation","id":"e0453a56-a8a4-4e8c-ba27-cc461503974c","sessionCode":"EE3","topDisplay":"Maglionico G1, Tacconi E1, Sgarito G2 1Medtronic Italia, Milan, MI, Italy, 2Ospedale Civico di Palermo, Palermo, Palermo, Italy","locationCode":"1055","description":"\r\n\t<div>OBJECTIVES: Catheter ablation procedures for Atrial Fibrillation (AF) can be evaluated regarding the possibility of reducing the length of hospital stay. Considering the cryoablation procedures, the hospitalization setting is still widespread in Italy, despite the numerous evidence supporting the day hospital management. This analysis aims to evaluate the variation in resource consumption for the two different hospital pathways. METHODS: The analysis was conducted in a Southern Italian hospital by using Activity-Based Costing method. Through clinical and administrative staff interviews, data on patient pathway costs and dedicated staff time (per minute) have been collected. An economic valorization allowed the definition of the total costs for each hospital setting. The center shared the unit costs of drugs, imaging, laboratory exams, consumables, medical procedures, and visits, while non-clinical staff and overhead were quantified through literature (discounted costs).The focus was on patient journey costs and not on devices, drugs, and consumables that were used in equal amounts in both care settings. RESULTS: The population treated had an age of 56 ± 9 years (25.9% were women); most of the patients (75%) suffered from paroxysmal AF, 25% from persistent AF. No peri-procedural events were observed in any patient. The cost difference between the two hospitalization settings (ordinary with two nights and daytime with one night) was found to be €493.23 for each patient. The main cost driver for the path in ordinary hospitalization was represented by the hospital stay, which showed to be 36% of the total. CONCLUSIONS: Wider adoption of day-hospital cryoablation AF procedures, following careful patient selection, would lead to patient management costs and resource consumption optimization. It would result in the reduction of the length of stay and freeing up of beds (allowing their reallocation where necessary), reduction of waiting lists, and acceleration of patient access to care.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23maglionicoee3poster130440-pdf.pdf?sfvrsn=caf3b0e7_0","title":"ISPOREurope23_Maglionico_EE3_POSTER130440.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130440","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Disease Burden and Efficacy of Current Treatment for Non-Neuronopathic MPS II Patients: A Systematic Literature Review","id":"59bb98d9-59a5-41aa-916c-cca59f6f35e6","sessionCode":"CO30","topDisplay":"Schmidt E1, Ocampo AD2, DeOcampo GA2, Engmann N3 1Certara Inc., Loerrach, Germany, 2Certara Inc., Manila, Philippines, 3Denali Therapeutics, South San Francisco, CA, USA","locationCode":"1026","description":"\r\n\t<div>OBJECTIVES: Mucopolysaccharidosis II (MPSII, Hunter syndrome) is an ultra-rare genetic disease (incidence rate: 0.38 to 1.09/100,000 live male births) with manifestations caused by mutations in the iduronate-2-sulfatase gene, leading to impaired enzymatic function and accumulation of glycosaminoglycans in multiple systems and organs. The non-neuronopathic phenotype (nnMPSII) is characterized by delayed recognition and absent or milder CNS manifestations. Although enzyme replacement therapy (ERT) for MPSII was approved in 2006, ERT is unable to access the CNS, and characterization of unmet need has predominantly focused on the severe population. A systematic literature review was conducted to assess clinical, humanistic and economic burden, mortality, and efficacy/safety of nnMPSII patients on current SOC treatment. METHODS: Medline, Embase, Cochrane Central/Reviews were searched from 2003 to February 2023, for peer-reviewed articles and conference abstracts (in English), supplemented by a search on conference websites (last 3 years), and study registries. While a broad search was conducted for any phenotype, only data for nnMPSII was included during the selection. RESULTS: Of 2590 records identified, following full-text review, 57 publications were included. Clinical burden was derived from 36 studies (10 investigating joint-related comorbidities, 10 cardiac, 10 hearing, 9 respiratory, 17 CNS) with heterogenous symptoms and varying study quality. Humanistic burden was assessed in a variety of domains using different instruments (patient-reported in 16, caregiver-reported in 4). No economic analysis and only limited resource utilization data (5 studies) could be identified. 25 studies investigated efficacy/safety, with urinary glycosaminoglycan, 6-Minute-Walking-Test, and pulmonary function being most frequently assessed endpoints. CONCLUSIONS: Review suggests that despite the lack of severe CNS involvement, attenuated patients are still profoundly affected. Disease burden appears to be underestimated as many studies have not separated results for nnMPSII and hence could not be included in our review. There are substantial data gaps and further research into the impact of this phenotype is warranted.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/schmidt-et-al-disease-burden-and-efficacy-of-current-treatment-for-nnmps-ii-patients-slrvfinal2132993-pdf.pdf?sfvrsn=1398f06f_0","title":"Schmidt et al. Disease burden and efficacy of current treatment for nnMPS II patients SLR_vFinal2132993.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132993","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health Care Resource Utilization Patterns in Finnish Multiple Myeloma Patients: A Population-Based Cohort Study","id":"5679429e-30f5-483a-b12d-cd3c6a8f42db","sessionCode":"RWD20","topDisplay":"Metsä R1, Kosunen M2, Ruotsalainen J3, Purmonen T3, Raittinen P3, Kallio A3 1Pfizer Oy, Helsinki, 18, Finland, 2Pfizer Oy, Helsinki, Uusimaa, Finland, 3Oriola Finland Oy, Espoo, Uusimaa, Finland","locationCode":"6071","description":"\r\n\t<div>OBJECTIVES: To estimate the health care resource utilization (HCRU) among Finnish Multiple Myeloma (MM) patients, and to recognize potential resource utilization patterns over time. METHODS: A population-based cohort study was conducted of Finnish patients newly diagnosed with multiple myeloma (ICD-10:C90.0) between 1.1.2015 and 31.12.2019 and followed from the first diagnosis until the end of the study period (31.12.2020). Primary care (PC) and specialty care (SC) HCRU data from The Finnish Institute for Health and Welfare registries was collected and analyzed for all healthcare events including visits, inpatient stays and other contacts. To recognize HCRU patterns, resource use per patient-year (py) was grouped by years (1-5) since diagnosis. Additionally, HCRU was examined one year and 6 months prior to death, and from the start of palliative care to death. RESULTS: In total, 1615 patients with 4468 py of follow-up were identified. During the study period patients had on average 95.5 healthcare events/py, of which 62.0% were PC events and 27.9% were MM specific. Majority of MM specific events (93.6%) were in SC. HCRU tended to be higher during the first year after diagnosis than during the following four years. 417 and 505 patients could be followed for 1 year and 6 months before death, respectively. In addition, 145 patients received palliative care before death. Patients had on average 199.2 and 179.6 healthcare events/py 6 months and 1 year before death, of which 75.5% and 76.1% were PC events and 17.1% and 18.4% were MM specific. Between the initiation of the palliative care and death, patients had on average 222.3 healthcare events/py with 86.0% being PC events and 11.1% MM specific. CONCLUSIONS: Multiple myeloma imposes a significant burden in primary care and specialty care in Finland. The total HCRU increases towards the end of patients’ life and focuses to primary care setting.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-europe-2023mm-hcru-finlandrwd20129585-pdf.pdf?sfvrsn=a1f9d1bf_0","title":"ISPOR Europe 2023_MM HCRU Finland_RWD20129585.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129585","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Vascular Access Devices: A Comparative Analysis of Complications and Their Cost Implications","id":"8ede99ba-02bb-42f6-b022-ce039602505a","sessionCode":"PCR45","topDisplay":"Bouguerra H1, Waltl F2, Dierick K3 1Terumo BCT, arcueil, France, 2Terumo BCT, Garching bei München, BY, Germany, 3Terumo BCT Europe NV, Zavemtem, Belgium","locationCode":"6044","description":"\r\n\t<div>OBJECTIVES: This study aimed to conduct a literature review to identify adverse events associated with peripheral vascular access and other commonly used access devices, and assess these events' economic burden and cost implications. METHODS: A literature review was conducted using PubMed to gather relevant studies and publications reporting on adverse events related to peripheral catheters (PVC), peripherally inserted central catheters (PICC), midline catheters (MC), and central venous catheters (CVC). The identified adverse events included deep vein thrombosis (DVT), bloodstream infection (such as CLABSI or sepsis), pneumothorax, phlebitis, occlusion, and pulmonary embolism. To assess the economic burden, the costs associated with each adverse event were also obtained. RESULTS: The findings revealed varying rates of adverse events across different types of vascular access. The rates of DVT ranged from 2.36% for peripheral catheters (PVC) to 10.91% for central venous catheters (CVC). Bloodstream infections ranged from 0.21% for PVC to 11.82% for CVC. Other adverse events such as pneumothorax, phlebitis, occlusion, and pulmonary embolism also exhibited varying rates across the different access devices. Furthermore, the economic burden associated with these adverse events was calculated by multiplying the rates by their respective costs. The total economic burden was highest for CVC, with a total cost of $3,074.79, followed by PICC ($1,302.07), Midline Catheter (MC) ($1,424.02), and PVC ($328.33). CONCLUSIONS: The study highlights the prevalence of adverse events associated with different vascular access types, with varying rates observed among these devices. The economic burden analysis underscores the substantial cost implications linked to these adverse events. PICCs and CVCs, and MCs exhibited higher cost implications compared to PVCs. Understanding these events’ rates and economic impact can help healthcare providers improve patient safety and the cost-effectiveness of vascular access procedures. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/vascular-access-devices--a-comparative-analysis-of-complications-and-their-cost-implicationsams133854-pdf.pdf?sfvrsn=ed399385_0","title":"VASCULAR ACCESS DEVICES- A COMPARATIVE ANALYSIS OF COMPLICATIONS AND THEIR COST IMPLICATIONS_AMS133854.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133854","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"3f994852-a0d4-4706-9665-e4d867006d9a","parentId":"00000000-0000-0000-0000-000000000000","title":"Injury & Trauma","urlName":"injury-trauma"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Application of Health Economics and Outcomes Research (HEOR) Perspectives in a Clinical Case Study of Niemann-Pick Disease, a Rare Disease","id":"bb43ed4a-487a-47a1-b31e-ce0e77d7682b","sessionCode":"CO28","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1027","description":"\r\n\t<div>OBJECTIVES: This case study aims to present a clinical scenario of a 1-year-old male patient with Niemann-Pick Disease (NPD) and explore the potential application of Health Economics and Outcomes Research (HEOR) perspectives to inform healthcare decisions. The objectives include assessing the economic impact, treatment outcomes, and overall value of interventions for Niemann-Pick Disease and highlighting the importance of considering the economic and clinical implications of healthcare decisions in the context of a rare disease. METHODS: The methods involve the collection and analysis of clinical data, including the patient's medical history, symptoms, and diagnostic findings. The study also describes the interventions utilized, such as clobazam, Levetiracetam, platelet transfusion, and intravenous fluids, to address the patient's symptoms. While no original research or formal study design was implemented, the case study draws upon clinical expertise, medical records, and relevant literature to inform the presented findings and observations. RESULTS: As no research was conducted in this case study, there are no specific findings or quantitative results to report. However, the study describes the successful management of seizure activity and symptomatic relief observed in the patient through the use of clobazam, Levetiracetam, and supportive care measures such as platelet transfusion and intravenous fluids. CONCLUSIONS: This case study highlights the potential benefits of integrating Health Economics and Outcomes Research (HEOR) perspectives into clinical decision-making for rare diseases like Niemann-Pick Disease. It underscores the importance of considering both economic and clinical implications in healthcare decisions and emphasizes the need for early diagnosis, prompt access to appropriate treatments, and optimized resource allocation. While no research was performed in this particular case, the study encourages further exploration of HEOR approaches by stakeholders, including academic entities, government-affiliated organizations, payers, providers, and industry, to improve patient outcomes and enhance resource efficiency in the management of rare diseases.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132004","diseases":[{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparison of Methods for Modelling the Cost-Effectiveness of Atezolizumab as an Adjuvant Treatment in Stage II-IIIA Non-Small Cell Lung Cancer","id":"35f9edd0-0fc7-4daa-aaf6-ce12ede4ec62","sessionCode":"HTA17","topDisplay":"Jovanoski N1, Yip CY2 1F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland, 2Roche Products Ltd., Welwyn garden city, Herts, UK","locationCode":"4052","description":"\r\n\t<div>OBJECTIVES: A cost-effectiveness model was previously built to assess the use of atezolizumab in the adjuvant treatment of stage II-IIIA non-small cell lung cancer (NSCLC) using time-invariant for the post disease-free survival (DFS) transition probabilities. The choice to build a more complex model using tunnel states to allow time-variant transitions or a simpler model using time-invariant transitions needs to be weighed between time, resource, and how robust the model would be for decision making. The potential issue with the simpler model is that the modelled overall survival (OS) may be biased. Here, we build a more complex model that allows for time-varying health state transition probabilities. The objective of this analysis is to better understand the impact of the two methodologies on the outputs. METHODS: The cost-effectiveness model (Yip et al. (2022)) used for the NICE submission was updated to allow the use of time-variant transition probabilities using tunnel states and assessing the appropriate models using the NICE DSU 14 Technical Support Document (i.e. rather than the previous approach of assuming exponential distribution). RESULTS: The revised model shows that using either time-invariant or time-variant post-DFS transition probabilities has little impact on the estimated incremental cost-effectiveness ratios (ICERs). Moreover, using time-invariant transition probabilities did not have a major impact on the modeled OS, which were within the 95% confidence interval of the observed Kaplan-Meier OS from IMpower010. CONCLUSIONS: The results show that the use of a simpler approach that restricts the post-DFS health state transitions to being time-invariant is suitable for decision making. This reflects the fact that the main driver of the results is the significant improvement in DFS provided by adjuvant atezolizumab treatment. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23jovanoskihta17poster129280-pdf.pdf?sfvrsn=f12960fd_0","title":"ISPOREurope23_Jovanoski_HTA17_POSTER129280.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129280","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Monitoring of the Long-Term Impact of the COVID-19 Pandemic on the Decision Process of the Italian Medicine Agency","id":"9b18df14-dc05-41d9-ac69-cf166f0eeccb","sessionCode":"HTA36","topDisplay":"Canali B1, Caimmi M1, Candelora L1, Ciarlo A1, Fiorentino F2, Mattu I1, Vassallo C1, Urbinati D1 1IQVIA Solutions, Milan, Italy, 2IQVIA Solutions, Milano, Italy","locationCode":"4070","description":"\r\n\t<div>OBJECTIVES: The aim of this study is to update previous research on time to market (TTM) in Italy and evaluate the Covid-19 pandemic impact on drugs’ TTM in an expanded timeframe (2015-2022 vs 2015-2021), to provide a more comprehensive assessment of the work of the Italian Medicines Agency committees ahead of the reform foreseen in the next months. METHODS: The analysis was updated to include all the new active substances, excluding vaccines, that received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) between January 2015 and December 2022 and that completed the Italian negotiation pathway. Firstly, drugs were clustered into the Pre-Covid, Partially-Covid and Fully-Covid groups depending on the timing of their negotiation process. Differences in TTM among the three periods were inspected visually and through descriptive statistics, and two-sample tests were performed to evaluate the potential role of co-variates influencing TTM. Then, a nearest-neighbor matching estimator analysis was implemented based on the identified relevant co-variates. RESULTS: In 2022, 24 new drugs met the inclusion criteria for the analysis, bringing our sample to 387 unique combinations of molecule and indication. Descriptive analyses showed a difference in TTM only for the Partially-Covid period (average TTM: 435.7 days), while no difference was observed between the Fully-Covid vs Pre-Covid periods (average TTM: 283.7 and 283.2 days). Inferential analysis confirmed the results from the previous study: the average treatment effect of the Partially-Covid period was +124.6 days (p=0.00) (vs. +108.0 days [p=0.00] in the 2015-2021 timeframe), with moderate variation resulting from sensitivity analyses (101.0-138.7 days; p=0.00). CONCLUSIONS: This study confirmed previous evidence on the impact of Covid-19 on drugs TTM in Italy: while TTM increased significantly during the pandemic, it decreased back to its pre-pandemic averages in the Fully-Covid period, highlighting the temporary nature of the shock.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23fiorentinohta36poster130012-pdf.pdf?sfvrsn=4d535a6c_0","title":"ISPOREurope23_Fiorentino_HTA36_POSTER130012.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130012","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evidence of Differences in the Calculation of Disability Adjusted Life Years (DALY) for COVID-19 Population: A Scoping Review","id":"8f491c2e-b2ed-435a-82ad-cf34a72407f3","sessionCode":"EPH35","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3036","description":"\r\n\t<div>OBJECTIVES: To map the data pertaining to differences in the calculation of Disability Adjusted Life Years (DALY) for COVID-19 population METHODS: A literature search was conducted from inception till April 2023 using databases (PubMed, Medline, Embase) and grey literature searches. Data pertaining to differences in the DALY calculation for COVID-19 was collected. A scoping review was carried out following PRISMA-ScR guidelines. RESULTS: The vast majority of countries, including Ireland, France, India, Germany, Korea, Netherlands, Malta, Australia, Colombia, Denmark, and the United States, have adopted an incident-based strategy, whereas only a small number of countries—Mexico, Iran, and Italy—have adopted a prevalence-based strategy. There are substantial differences in the formulas used to calculate DALY for COVID-19 population in terms of applying disability weight, Global Burden of Disease (GBD) considerations and using temporal discounting technique. Globally, there are differences in the availability of data, healthcare systems, demographics, patterns of disease burden, cultural and contextual factors, differences in policy and health priorities, updates and revisions by authorities, disease-specific factors, and socioeconomic factors based on geography. Despite these challenges, estimating DALY for COVID-19 population can shed light on how the pandemic has impacted global health and guide public health policies and initiatives. CONCLUSIONS: The calculation of DALY is a key instrument in shaping public health policies, resource allocation, monitoring health outcomes, and lobbying for evidence-based treatments. There is a need for an accurate estimation of disease burden and uniformity in consideration of variables and approaches used in DALY calculation for COVID-19 population.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131510","diseases":[{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"How Immunotherapies Have Altered the Future of Cancer Treatment: Patients Living Longer, Healthier Lives","id":"fb5fc3a0-4cd0-4b33-99b2-d0558d1a88c2","sessionCode":"CO23","topDisplay":"Borges S1, Shaw D2, Meunier A2, Farrington J2 1Putnam PHMR, Newcastle Upon Tyne, Tyne and wear, UK, 2Putnam PHMR Ltd., London, LON, UK","locationCode":"1018","description":"\r\n\t<div>OBJECTIVES: Since entering the market immunotherapies have led to a paradigm shift in mortality rates for patients with melanoma resulting in an overall improvement in survival and quality of life. This review maps the shift by comparing changes in patient outcomes alongside the frequency of positive HTA decisions for immunotherapy treatments in the UK and Australia between 2019 and 2023. METHODS: A targeted literature review was conducted using Embase, Clinicaltrials.gov and PubMed, for pivotal clinical trials, and the NICE and PBAC databases for technology appraisals (TA) for immunotherapies in melanoma including ipilimumab, nivolumab and pembrolizumab. RESULTS: Thirty-five clinical trials were first published, or reported updated results, for ipilimumab, nivolumab and pembrolizumab. Of these studies, 7 (20%) were reported for ipilimumab, 7 (20%) for nivolumab and 4 (11.5%) for pembrolizumab, as monotherapy regimens. Results for nivolumab in combination with ipilimumab were reported across 17 (48.6%) studies. Overall survival (OS) was the most commonly reported outcome (n=30), followed by progression-free survival (PFS) (n=13) and recurrence-free survival (RFS) (n=6). A search of NICE’s TA database returned 9 single TA guidance documents for immunotherapies in melanoma, all of which (100%) received a positive NICE recommendation. A review of PBAC’s database returned 23 guidance documents for immunotherapy in melanoma, of which 52.2% (n=12) achieved a positive PBAC recommendation between 2019 and 2023. CONCLUSIONS: Overall, it is evident that an increased availability of safe and effective immunotherapies has led to a shift in mortality rates for patients with melanoma when assessed for key outcomes. A growing trend in positive recommendations for immunotherapies in melanoma in the last 5-years is suggestive of an increased confidence in the clinical trial data and a clear upward trajectory in the benefit being demonstrated by pembrolizumab, nivolumab and ipilimumab.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/co23-how-immunotherapies-have-altered-the-future-of-cancer-treatmentsborges133178-pdf.pdf?sfvrsn=362b1a2d_0","title":"CO23 How Immunotherapies Have Altered the Future of Cancer Treatment_SBorges133178.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/co23-how-immunotherapies-have-altered-the-future-of-cancer-treatmentsupplementary-appendix-1sborges133178-pdf.pdf?sfvrsn=edc1dd51_0","title":"CO23 How Immunotherapies Have Altered the Future of Cancer Treatment_Supplementary Appendix 1_SBorges133178.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133178","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Formulating Bayesian Poly-Hazard Models for Informed and Clinically Interpretable Lifetime Survival Extrapolations in Advanced Non-Small Cell Lung Cancer (aNSCLC)","id":"c7326d91-ffec-4a58-93e4-d07861686908","sessionCode":"MSR13","topDisplay":"Sharpe D1, Yates G1, García-Fernández L2, Yuan Y3, Lee A4, Chaudhary MA5 1Parexel, London, LON, UK, 2Parexel, Madrid, CA, Spain, 3Bristol Myers Squibb, Plainsboro, NJ, USA, 4Bristol Myers Squibb, Uxbridge, LON, UK, 5Bristol Myers Squibb, Princeton, NJ, USA","locationCode":"5053","description":"\r\n\t<div>OBJECTIVES: Immunotherapies have the potential to yield durable response and may therefore lead to complex lifetime hazards, which motivates leveraging external data to generate more reliable survival extrapolations. Bayesian methods provide a holistic framework to incorporate external data into parametric survival models, but some formulations require additional inputs that may be difficult to choose a priori. Here, we investigate Bayesian poly-hazard models, which have attractive features of flexibility, straightforward clinical interpretation, and absence of mandatory user-specified auxiliary parameters. We apply the approach to overall survival (OS) data for nivolumab plus ipilimumab (NIVO+IPI) in first-line aNSCLC from CheckMate 227 Part 1, with 29.3 months of minimum follow-up. METHODS: We consider a Bayesian poly-hazard model with background and disease-specific components, for which the corresponding prior distributions are estimated from general population and relevant SEER registry data, respectively, using a fixed background hazard in the latter step. Model predictions are compared to 5-year observations from extended follow-up and to previously reported estimates from Bayesian multi-parameter evidence synthesis (B-MPES) and an uninformed standard parametric model (SPM). RESULTS: Short-term extrapolations from the Bayesian poly-hazard model agree with later observations (5-year predicted OS: 20.7% [95% CrI (credible interval): 17.7-23.8%] vs 22.5% [95% CI (confidence interval): 19.2-26.2%] Kaplan-Meier), even though the SEER data represents a highly pessimistic expectation for NIVO+IPI survival. Long-term extrapolations are conservative compared to the naïve SPM and are in close agreement with B-MPES under a pessimistic scenario (20-year OS: 1.8% [95% CrI: 1.2-2.6%] poly-hazard vs 5.5% [95% CI: 4.1-7.2%] SPM vs 1.4% [95% CrI: 1.1-1.7%] B-MPES). The poly-hazard model predicts that the background and disease-specific hazards become equal at approximately 15 years. CONCLUSIONS: Bayesian poly-hazard models provide a flexible approach with clinically interpretable structure to plausibly estimate lifetime conditional survival for patients with aNSCLC receiving NIVO+IPI. Moreover, their data-driven formulation helps to reduce subjectivity in survival extrapolations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23leemsr13posterv2130111-pdf.pdf?sfvrsn=2f263e81_0","title":"ISPOREurope23_Lee_MSR13_POSTERV2130111.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130111","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Decision Making and the Questions Raised When Real World Evidence From a Federated Data Network Conflicts With RCT Data: A Use Case on COVID-19 Treatments","id":"0c00d2a9-1a05-4142-a623-d136a717d223","sessionCode":"RWD24","topDisplay":"Read C1, Claire R2, Elvidge J3, Dietz J4, Debray T5, Mayer MA6, Ramirez Anguita JM6, Dawoud D4, Rappoport N7 1University Medical Centre Utrecht, Utrecht, Utrecht, Netherlands, 2National Institute for Health and Care Excellence, Nottingham, UK, 3National Institute for Health and Care Excellence, Manchester, UK, 4National Institute for Health and Care Excelllence, London, LON, UK, 5Smart Data Analysis and Statistics B.V., Utrecht, Netherlands, 6Hospital del Mar, Barcelona, Catalonia, Spain, 7Ben-Gurion University of the Negev, Beer-Sheva, Israel","locationCode":"6078","description":"\r\n\t<div>OBJECTIVES: The European Health Data and Evidence Network (EHDEN) is a federated network of European partners, with real-world data standardised to the Observational Medical Outcomes Partnership Common Data Model (OMOP CDM). We demonstrate how EHDEN can be used to generate real-world evidence (RWE) in the context of COVID-19, and highlight how RWE may generate questions on how to interpret it when it conflicts with randomized controlled trial (RCT) data. METHODS: Evidence on the effectiveness of remdesivir and tocilizumab in people aged 18 years or older hospitalized with COVID-19 was collected from: 1) RCT data collected from a systematic literature review 2) RWE from a historical cohort study run using Institut Municipal Assistència Sanitària Information System (IMASIS, Spain) data. RCT data was synthesized in a Bayesian network meta-analysis (NMA). RWD were analysed using propensity score matching, logistic regression and cox proportional hazards. As the RWE is currently from a single data partner, this was compared qualitatively with the RCT data. RESULTS: The RCT data (n = 15,246) did not show an effect on all-cause mortality for remdesivir vs tocilizumab (odds ratio: 1.10, 95% confidence interval 0.72-1.40). RWE evidence (n = 475) showed lower odds of mortality for people treated with remdesivir compared with those treated with tocilizumab (odds ratio: 0.38, 95% confidence interval 0.20-0.69, P value 0.002). CONCLUSIONS: Federated data networks like EHDEN can be used to generate RWE. However, with the data analysed thus far, the RWE results are not consistent with RCT data. This difference may be attributable to remdesivir and tocilizumab being given at different stages of the disease course in people hospitalized with severe COVID-19. How health technology assessment (HTA) and regulatory agencies make decisions when presented with conflicting RCT and RWE evidence warrants special consideration.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-europe-2023covid133562-pdf.pdf?sfvrsn=15040578_0","title":"ISPOR Europe 2023_COVID133562.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133562","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Systematic Review of Renal Denervation for Uncontrolled Hypertension: Meta-Analysis Results for Office and 24-Hour Ambulatory Blood Pressure","id":"c1cc841b-4a04-42a6-b111-d1f54e3ff29b","sessionCode":"MT1","topDisplay":"Sanderson A1, Hansell N1, Reddish K1, Moss J1, Schmieder RE2, Strachan L3, Walleser Autiero S4, Sharp A5, Marshall CV1 1York Health Economics Consortium, York, UK, 2Universitätsklinikum Erlangen, Erlangen, Germany, 3Medtronic, Macquarie Park, NSW, Australia, 4Medtronic International Trading Sarl, Tolochenaz, Switzerland, 5University Hospital of Wales, Cardiff, Wales, UK","locationCode":"5030","description":"\r\n\t<div>OBJECTIVES: Hypertension remains a significant clinical and public health challenge as a leading cause of morbidity and mortality worldwide. Renal denervation (RDN) is a procedure that aims to treat uncontrolled hypertension, including resistant hypertension. The objective of this systematic review (SR) and meta-analyses (MA) was to assess the efficacy of RDN compared with no RDN or sham control in patients with uncontrolled hypertension. METHODS: A SR, adhering to Cochrane and PRISMA guidance, was undertaken to identify randomised controlled trials (RCTs) of ultrasound or radiofrequency, catheter-based RDN. Eligible RCTs included adult patients with uncontrolled hypertension (office blood pressure ≥140/90mmHg, with/without any previous baseline antihypertensive medication). Key outcomes included office and 24-hour ambulatory blood pressure change from baseline. A feasibility assessment was conducted to assess the suitability of included trials for MA. Random effects (RE) MAs were conducted at the primary endpoint and last reported follow-up. The robustness of these results was assessed in a series of sensitivity and subgroup analyses. RESULTS: Searches conducted between November 2022 and May 2023 identified 6,298 records. Following screening, 25 RCTs were included. Four trials classified as “off-med” (trials in patients not receiving antihypertensive medication) and 12 trials as “on-med” (patients receiving the same regimen of antihypertensive medication) were suitable for MA. A RE MA for office systolic blood pressure (SBP) estimated a mean difference of -8.5 (95% CI: -13.4 to -3.5) at the primary endpoint and -7.2 (95% CI: -12.5 to -2.0) at last follow-up between RDN and the control group. For 24-hour ambulatory SBP, the mean difference was -3.7 (95% CI: -5.3 to -2.0) and -3.3 (95% CI: -5.0 to -1.6) at the primary endpoint and last follow-up respectively. CONCLUSIONS: There is robust evidence to suggest that RDN is effective in achieving clinically meaningful blood pressure reductions compared to no RDN or sham among patients with uncontrolled hypertension.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sandersonmt1poster132812-pdf.pdf?sfvrsn=2a577915_0","title":"ISPOREurope23_Sanderson_MT1_POSTER132812.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132812","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Application of Stated Preference Methods in Rare Disease: A Systematic Review","id":"854909c7-3a5e-4fb2-a074-d3908dcd2838","sessionCode":"PCR33","topDisplay":"Hall R1, Chua GN1, Erhabor P1, Lo SH2 1Acaster Lloyd Consulting Ltd, London, UK, 2Acaster Lloyd Consulting Ltd, London, London, UK","locationCode":"6029","description":"\r\n\t<div>OBJECTIVES: Medical advances have allowed research and innovation in interventions of rare diseases to grow exponentially in the past few decades. However, clinical evidence in rare disease is often associated with high levels of uncertainty due to small patient numbers and heterogeneity of conditions. Stated preference data can provide supporting evidence during the development, regulatory approval and marketing of rare disease interventions in situations where decision-making may be preference-sensitive, for example, when considerable clinical uncertainty exists or where multiple viable treatments are available. The objective of this review was to identify and describe stated preference methods used to understand stakeholder preferences in rare disease and any associated challenges. METHODS: A systematic search of six electronic databases combining terms relating to “stated preference” and “rare disease” was conducted in April 2023. Narrative synthesis was used to summarize findings based on data extraction conducted by two independent researchers covering disease area, intervention type, study population, design features, analysis and outcomes. The quality of studies was assessed using the PREFS checklist. RESULTS: In total, 42 studies were identified for inclusion. Studies used a range of methods including discrete choice experiments (DCEs) (55%), best-worst scaling (19%) and multi-criteria decision analysis (10%). Studies were conducted in over 20 rare diseases, most commonly hemophilia A (29%). Interventions included treatments (83%), diagnostic testing (10%), screening (5%) and preventative care (2%). Preferences were derived from patients (50%), caregivers (52%), medical experts (17%), and general population (19%) with sample sizes ranging between 5 and 2382. The robustness and interpretation of findings often appeared limited by small sample sizes, particularly in DCEs. CONCLUSIONS: DCEs remain the leading stated preference method in rare disease despite challenges in participant recruitment leading to potentially underpowered studies. Alternative methods requiring smaller sample sizes should be considered for addressing stated preference research questions in rare disease.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132384","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Bridging the Gap: How to Align HTA and Health Care Decision Making in Switzerland","id":"b46e8727-1ec4-4ae0-943a-d3923fafb85a","sessionCode":"HTA48","topDisplay":"Van Haasteren G Federal Office of Public Health, Bern, Bern, Switzerland","locationCode":"5026","description":"\r\n\t<div>OBJECTIVES: The Swiss Health Technology Assessment (HTA) program is an innovative program that connects research with policy making. In 2017, a HTA unit was established within the Federal Office of Public Health following a decision by the Federal Council in 2015 to intensify efforts in HTA. METHODS: The legal basis of the HTA program is Article 32 of the Federal Health Insurance Act, which specifies that health technologies (i.e., all preventive, diagnostic, and therapeutic interventions in health care) covered by the compulsory health insurance must be effective (E), appropriate (A), and economically efficient (E), alias the EAE criteria. New health technologies that do not meet the EAE criteria are not eligible for coverage. For health technologies that are already reimbursed, re-evaluation of the criteria can result in reimbursement arrest. The HTA program was developed to facilitate the evaluation of the EAE criteria in a systematic, reproducible and transparent manner. RESULTS: Discrepancies were found between the information reported in the HTA reports and the information requested by the policy makers. For example, 1) the policy makers often requested Swiss clinical expert opinions, despite the presence of higher-level quality evidence, 2) cost-effectiveness findings were requested by some, but not all policy makers and 3) the length and the language used in the HTA reports was typically considered too long and too scientific. Some modifications in HTA process were implemented to address the discrepancies, e.g., <ul> <li>engaging Swiss clinical experts early in the HTA process </li> <li>organizing round table discussions between assessment team, experts and/or policy makers</li> <li>developing survey questionnaires to collect direct Swiss patient data and</li> <li>introducing lay language summaries.</li> </ul> CONCLUSIONS: The Swiss HTA program is an innovative program that is still exploring and testing alternative steps to minimize the gap between HTA and healthcare decision making.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128807","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Targeted Influenza Vaccination in at Risk Populations – How Avoided Cases Translate into Clinical and Economic Benefits for Czech Republic, Hungary, and Romania","id":"cac81f57-e179-471e-b8c1-d3b7b7c3042e","sessionCode":"EPH48","topDisplay":"Fraisier B1, Bartelt-Hofer J2 1Sanofi, Lyon, France, 2Sanofi, Lyon, 69, France","locationCode":"3045","description":"\r\n\t<div>OBJECTIVES: The World Health Organization(WHO) has urged European countries to reach 75% influenza vaccination coverage rate(VCR) in vulnerable populations (older adults, patients with comorbidities[PwC], children aged 6-24 months, healthcare workers[HCW], and pregnant women[PW]). We assessed the additional clinical and economic benefits of reaching suggested VCR in three selected countries. METHODS: A decision analytic model, capturing 2021/2022 epidemiologic inputs (for each subgroup: eligible population, VCR, influenza attack rates, quadrivalent influenza vaccine efficacy) and 2022 costs expressed in euros (direct medical costs of influenza-related physician consultations and hospitalizations) was built. For selected countries (Czech Republic[CZ], Hungary[HU] and Romania[RO]), a one-year comparison of observed vs 75% VCR in terms of influenza cases avoided and costs informed the potential benefits of reaching WHO recommendation (expressed in thousands[K]). Local inputs and robust, published sources were prioritized. RESULTS: Country-average observed VCR were 22.4%, 20.27%, 1.5%, 19% and 1.83% for older adults, PwC, children, HCW, and PW. Older adults consistently displayed the highest clinical benefit of reaching 75% VCR (47.9K up to 86.5K additional averted influenza cases for CZ and RO, respectively), followed by PwC and children. Additional averted hospitalizations and physician visits ranged from 1.8K for CZ up to 3.4K for RO, and 15.7K for CZ up to 34.1K for HU, respectively. Averted influenza cases translate into additional savings of 1.268K € for HU, 1.866K € for CZ and 2.929K € for RO in costs; hospitalizations average 68% (49%[HU] to 81%[RO]) of those spared monetary resources. CONCLUSIONS: A punctual objective to increase VCR up to 75% in vulnerable populations can avert influenza cases and its related clinical and economic consequences, reducing the burden of disease for CZ, HU and RO. Older adults will mostly benefit from such a policy, future research focused on enhanced influenza vaccines for this population might accentuate the importance of these findings.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/euisporvcreasteuropeposter2023vf-1132818-pdf.pdf?sfvrsn=23053106_0","title":"EU_ISPOR_VCR_East_Europe_Poster_2023_VF (1)132818.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132818","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Literature Review of Health-Related Quality of Life, Costs, and Healthcare Resource Use in Primary Biliary Cholangitis","id":"941122b5-c58a-4d4d-a1cc-d3f1c5ed99f3","sessionCode":"EE91","topDisplay":"Pashley A1, Boing E2, Serafini P2, Worthington E3 1Costello Medical, Cambridge, CAM, UK, 2Ipsen, Cambridge, MA, USA, 3Costello Medical, Cambridge, UK","locationCode":"2024","description":"\r\n\t<div>OBJECTIVES: Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterised by progressive cholestasis and biliary fibrosis. PBC symptoms and complications can negatively impact patients’ health-related quality of life (HRQoL) and may lead to substantial healthcare costs and resource use (CRU). A systematic literature review (SLR) was conducted to identify HRQoL and CRU studies in PBC. METHODS: This SLR was performed in accordance with PRISMA, Cochrane Collaboration and UK NICE guidelines. Searches were conducted in November 2022 in MEDLINE, EMBASE and the Health Technology Assessment (HTA) Database. HTA/economic websites (n=19), congress proceedings (n=8) from 2021–2022 and bibliographies were hand searched. RESULTS: 2,604 records, identified from database (n=1,480) and supplementary (n=1,124) searches, were screened against pre-determined eligibility criteria. The large number of studies identified in HRQoL (n=63) and CRU (n=33) prompted prioritisation of 5 HRQoL studies reporting EQ-5D utility data and 9 CRU studies reporting data from Europe in the last 10 years. 2/5 HRQoL studies specifically investigated PBC. 4/5 studies reported a substantial negative impact of PBC on patients’ HRQoL; where reported, pruritus (2 studies), as well as fatigue, bone ache and memory and concentration problems (1 study) were key drivers of reduced HRQoL. EQ-5D scores were lower overall and following liver transplant versus age/sex-adjusted mean UK population scores (1 study). Among studies reporting CRU data, 5/9 reported ursodeoxycholic acid use in 79.3–90.6% of patients. Liver transplants, reported by 4/9 CRU studies, were associated with substantial costs where reported (2 studies in the UK and Italy), exceeding cirrhosis- and cancer-related costs. CONCLUSIONS: The HRQoL and CRU data captured in this SLR provide insight into the substantial impact of PBC on patients' lives as well as key inputs for cost-utility analyses, which can be used to elucidate potential benefit of investigational treatments for patients and healthcare systems.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23boingee91poster-slr-cru-and-hrqol133268-pdf.pdf?sfvrsn=a24d66bb_0","title":"ISPOREurope23_Boing_EE91_POSTER SLR CRU and HRQoL133268.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23boingee91supplementary-material-slr-cru-and-hrqol133268-pdf.pdf?sfvrsn=7616551e_0","title":"ISPOREurope23_Boing_EE91_Supplementary Material SLR CRU and HRQoL133268.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133268","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Impact of COVID-19 Preventive Restrictions on the Development of Diseases of Despair: A Multi-Group Time Series Analysis","id":"e581b66f-3b87-49e5-81b9-d47ceb44d586","sessionCode":"EPH17","topDisplay":"Wolde F, Hayes C, Gressler L, Martin B University of Arkansas for Medical Sciences (UAMS), Little Rock, AR, USA","locationCode":"3021","description":"\r\n\t<div>OBJECTIVES: Diseases of despair (DOD; overdoses, alcohol related disorders, drug use disorders and suicide) are rising in the US and are exacerbated by social isolation and unemployment. States varied the level of social restrictions in response to the COVID-19 pandemic providing a natural experiment to assess the impact of these policies on development of DOD. METHODS: Using US commercial claims, we employed a multi-group interrupted time series analysis to examine the rates of DOD between states that had high levels of COVID-19 related social restrictions compared to states with fewer. Subjects included enrollees eligible for medical benefits for at least one month between June 2017 through October 2021. Using the PEW criteria, states were classified into three groups (most, less, and least restrictive) based on COVID-19 policy restrictiveness. Monthly rates of the number of persons with one or more DOD diagnoses and DOD-related healthcare encounters were calculated. March 2020 was the interruption time point and pair-wise comparisons were made between the three restrictiveness groups. RESULTS: A total of 4,316,563 eligible enrollees were included: least-restrictive (n=1,533,628), less-restrictive (n=2,347,033), and most-restrictive (n=435,902). The mean monthly rates of individuals with one or more DOD diagnoses per 100,000 eligible enrollees over the entire series were: least-restrictive=365.70, less-restrictive=359.53, most-restrictive=297.71. No significant differences were detected in the level or slope changes for most-restrictive states relative to least-restrictive states on composite measures of DOD and nearly all individual DOD (p>0.05) examined except the rate of individuals (slope difference=1.47; 95%CI:0.40-2.53) and encounters (slope difference=3.19, 95%CI:1.03-5.34) with opioid use disorders recorded. CONCLUSIONS: Except for a slight increase in opioid use disorder diagnosis rates following COVID-19, imposing more pandemic related restrictions, such as stay at home orders and closure of non-essential businesses, do not appear to affect rates at which persons seek care for diseases of despair in the near term.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/filmonwoldethe-impact-of-state-level-covid-19-preventive-restrictions-on-diseases-of-despair-an-interrupted-time-series-analysis-of-national-claims-databcm128916-pdf.pdf?sfvrsn=ced3ef97_0","title":"Filmon_Wolde_The-Impact-of-State-Level-COVID-19-Preventive-Restrictions-on-Diseases-of-Despair An-Interrupted-Time-Series-Analysis-of-National-Claims-DataBCM128916.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128916","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health-Related Quality of Life in Children with Pneumococcal Disease: What Can We Learn from Cost-Effectiveness Analyses of Pneumococcal Vaccines?","id":"903dc0e6-08f1-41e3-b73a-d48564cb4a12","sessionCode":"PCR48","topDisplay":"Huang M1, Xie J2, Mohanty S1, Elbasha E3 1Merck Research Laboratories, Merck & Co, Inc., Rahway, NJ, USA, 2XL Source Inc, Los Angeles, CA, USA, 3Merck Research Laboratories, Merck & Co, Inc., West Point, PA, USA","locationCode":"6046","description":"\r\n\t<div>OBJECTIVES: This study aimed to synthesize the health-related quality of life (HRQoL) impact in children with pneumococcal disease (PD) from cost-effectiveness analyses (CEAs) of pediatric pneumococcal vaccines in the United States (US). METHODS: A targeted literature review of US CEA of pediatric pneumococcal vaccines was conducted in 2023. Full-text articles reporting quality-adjusted life year (QALY) were included. HRQoL-related inputs from the CEAs were extracted. The original studies from which these inputs were estimated were identified and reviewed. RESULTS: A total of 8 CEAs were included, wherein QALY decrements were used as model inputs to derive total QALYs. The input values of QALY decrements per PD episode in US children ranged from 0.007-0.76 for meningitis, 0.0016-0.21 for non-meningitis invasive PD, 0.006-0.59 for inpatient pneumonia, 0.0004-0.19 for outpatient pneumonia, and 0.0016-0.36 for acute otitis media and tube placement. Five CEAs cited previously published CEAs as references for these inputs, while the rest referenced utility studies or meta-analyses of utility studies. The inputs were sourced to 11 original utility studies, which were conducted in the US, UK, Canada, or Thailand and were published in year 1995-2014. Most of the original studies were based on parent surveys. Utility measures used included HUI2/3, EQ-5D, and EQ-VAS. Direct methods applied included standard gamble and time-trade-off. The CEAs used the original utility studies as a basis for estimating the QALY decrement inputs, incorporating certain assumptions such as the application of utility values from different conditions, age groups, or countries. CONCLUSIONS: PD is associated with considerably reduced HRQoL, which largely impacts the projected cost-effectiveness of pneumococcal vaccines. However, substantial variations in QALY decrement inputs were observed in the US pediatric CEAs. These inputs were sourced to a limited number of original studies, among which there was a high-level of heterogeneity regarding study methods, quality, and results.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23elbashapcr48poster131325-pdf.pdf?sfvrsn=cedb103b_0","title":"ISPOREurope23_Elbasha_PCR48_Poster131325.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131325","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Utility and Budget Impact Analyses of Cervical Cancer Screening Using Self-Collected Sample Kit for HPV DNA Testing in Thailand","id":"5f751da4-c90b-4153-bc60-d4bb03b0c5a1","sessionCode":"EE83","topDisplay":"Kositamongkol C1, Kanchanasurakit S2, Mepramoon E3, Talungchit P4, Chaopotong P4, Kengkla K5, Chaisathaphol T3, Saokaew S5, Phisalprapa P3 1Division of Ambulatory Medicine, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, 10, Thailand, 2Phrae Hospital, Muang Phrae, Phrae, Thailand, 3Division of Ambulatory Medicine, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok Noi, Bangkok, Thailand, 4Department of Obstetrics and Gynecology, Mahidol University, Bangkok Noi, Bangkok, Thailand, 5University of Phayao, Mueang Phayao, Phayao, Thailand","locationCode":"2009","description":"\r\n\t<div>OBJECTIVES: Cervical cancer is the third most common cancer in Thai women. The success of cervical cancer screening policy is restrained due to various factors that minimize the screening rate. Self-screening could reduce barriers to screening in Thai women. This study aimed to evaluate the cost-utility and budget impact of cervical cancer screening using a self-collected sample kit for HPV DNA testing in Thailand. METHODS: A decision tree coupled with Markov model was used to estimate lifetime costs and health benefits of listing self-screening policy in the national list of health benefit coverage for women aged 25-65 years. This analysis was performed from a societal perspective. We compared the costs and outcomes of three options including (1) additional self-screening, (2) clinician screening only, and (3) no screening. All costs were reported in 2022 USD. Sensitivity analyses were conducted to assess robustness of the model. The 10-year budget impacts of the additional policy were calculated. RESULTS: Both additional self-screening and clinician screening only policies were cost-saving, compared to no screening. When compared between the two screening policies, additional self-screening was a dominant strategy. The incremental cervical cancer prevention benefit of adding self-screening into the heath benefit coverage was observed at any additional rate of screening that was expected to be gained by using self-collected sample kit. The sensitivity analyses give the same favorable results of the screening policies. Average annual budget impact of additional self-screening policy was 20.6 million USD. This budget would potentially grant more than 10 million women to undergo cervical cancer screening. CONCLUSIONS: Cervical cancer screening policies were cost-saving. The advantage of screenings predominated their incremental costs. Policy-makers should consider this evidence in the policy optimization process.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/hpvcuaposterisporprint128185-pdf.pdf?sfvrsn=74ea468d_0","title":"HPV_CUA_poster_ISPOR_PRINT128185.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128185","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"bd923eb7-0eba-48be-86a0-7e4a636bf00a","parentId":"00000000-0000-0000-0000-000000000000","title":"Reproductive & Sexual Health","urlName":"Reproductive---Sexual-Health"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of Hospitalized Respiratory Syncytial Virus Infection Among Children in Spain, 2016–2019","id":"55e405dd-f690-4ad5-80c9-d4cae729bf68","sessionCode":"EE31","topDisplay":"Law A1, Sato R2, López A3, Seabroke S4, Ramirez Agudelo JL4, Mora L5, Sarabia L4, Meroc E4, Aponte-Torres Z4, Haeberer M3 1Pfizer Inc., New York, NY, USA, 2Pfizer Inc., Collegeville, PA, USA, 3Pfizer S.L.U., Madrid, Spain, 4P95 Epidemiology & Pharmacovigilance, Leuven, Belgium, 5P95 Epidemiology & Pharmacovigilance, London, LON, UK","locationCode":"1078","description":"\r\n\t<div>OBJECTIVES: Respiratory syncytial virus (RSV) is a major cause of respiratory infection among children. The economic burden of RSV has been mainly driven by hospitalization costs. This study aimed to quantify the costs of hospitalized episodes and estimate the total economic burden of RSV in children <18 years admitted to the Spanish National Healthcare System between 2016–2019. METHODS: A retrospective observational study was conducted using a national hospital discharge database, that reports >90% of admissions. Children aged <18 years hospitalized for RSV-specific ICD-10 codes were included. The mean cost per hospitalization episode (€, 2022) was reported by age group (0–5, 6–11, 12–23 months, 2–5 and 6–17 years), by risk category (low and high) and by prematurity among infants aged <1 year. Total annual hospitalization costs were calculated from population incidence rates previously reported in the same population and the mean cost per episode. RESULTS: A total of 45,799 children, including 262 pre-term, were hospitalized with RSV during the study period. The mean cost per hospitalization episode was higher for RSV cases aged 0–5 months with at least one risk factor (€4,768 high vs €2,852 low risk) while the mean cost ranged from €3,664–4,413 for high risk and €2,733–3,529 for low-risk children of other ages and from €4,332 to €4,594 in pre-term infants. Total annual estimated costs increased during the study period, from €35.4 in 2016 to 44.8 million in 2019, with infants aged <6 months accounting for 66% (2016) and 59% (2019) of all hospitalization costs. CONCLUSIONS: RSV infection has a substantial economic burden amongst children in Spain, especially among infants <6 months.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/eu-ispor-posterchildren-spain-rsv-cost-studyv4131832-pdf.pdf?sfvrsn=163ce052_0","title":"EU ISPOR Poster_children Spain RSV cost study_v4131832.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131832","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Haemophilia Severity on Healthcare Resource Usage: Results from a Multinational Real-World Survey","id":"86f1b922-7f8a-4835-a7dc-d4ce061eb0ec","sessionCode":"RWD21","topDisplay":"Thakkar S1, Wilcox L2, Merla V2, Kane A2, Alvir J2, Pemmaraju S2, Morton E3, Garratt-Wheeldon J4, Ball N3, Olsen S3, Golden K3 1Pfizer Inc, Acton, MA, USA, 2Pfizer Inc, New York, NY, USA, 3Adelphi Real World, Bollington, UK, 4Adelphi Real World, Bollington, Cheshire, UK","locationCode":"6077","description":"\r\n\t<div>OBJECTIVES: This study describes hospitalisations, surgeries, and consultations for patients with haemophilia A (HA) and B (HB) to better understand healthcare resource utilisation (HCRU) based on disease severity. METHODS: Data were drawn from the Adelphi Haemophilia Disease Specific Programme™, a real-world, cross-sectional survey of haematologists and their male haemophilia patients, conducted in France, Germany, Italy, Spain, the United Kingdom, and United States, February 2020-May 2021. Haematologists provided data on demographics, clinical characteristics, and hospitalisations. Analyses were descriptive. Patients were grouped as mild (>5% of normal clotting factor (CF) levels), moderate (1-5%), and severe (<1%) using baseline CF at survey. RESULTS: Haematologists completed records for 1116 HA patients (mild n=236, moderate n=426, severe n=454) and 276 HB patients (mild n=84, moderate n=102, severe n=90) who had received haemophilia treatment (mean age HA: 28.9, HB: 30.1 years; mean body mass index HA: 24.0, HB: 23.7). In the year prior to survey, 17.4% of severe HA and 23.2% of severe HB patients had at least one hospitalisation (8.6% mild HA, 10.8% mild HB, 15.2% moderate HA, 13.5% moderate HB). During this year, overall mean consultations per HA patient with healthcare professionals were 6.9 mild HA, 9.7 moderate HA, and 15.2 severe HA. For HB patients, mean consultations were 9.2 mild HB, 10.2 moderate HB, and 9.7 severe HB. Specifically, a high mean number of consultations for severe patients were with haemophilia nurses (severe HA=13.0, severe HB=6.5) and physiotherapists (severe HA=11.0, severe HB=9.5). The proportion of patients who had ever undergone joint surgery differed with severity (mild HA=8.1%, mild HB=1.2%, moderate HA=10.8%, moderate HB=10.8%, severe HA=18.5%, severe HB=18.9%). CONCLUSIONS: Despite receiving treatment, severe HA and HB patients had high hospitalisation and consultation rates, highlighting increased HCRU and potentially greater economic burden. Future therapies should better manage CF levels and reduce haemophilia severity to lower HCRU and economic burden.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023thakkarrwd21poster131685-pdf.pdf?sfvrsn=54d054a6_0","title":"ISPOREurope2023_Thakkar_RWD21_POSTER131685.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131685","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Current State of Health Economic Models in Hereditary Angioedema","id":"4dc26a74-00db-4129-acfc-d62d1f9154a0","sessionCode":"EE94","topDisplay":"Tachdjian R1, Lahue B2, Cribbs K2, Fang DIK2, Czado S3, Goga L3, Desai V3, Rautenberg T4, Schwander B5 1University of California, Los Angeles, Santa Monica, CA, USA, 2Alkemi LLC, Manchester Center, VT, USA, 3KalVista Pharmaceuticals, Inc., Cambridge, MA, USA, 4Griffith University, Brisbane, QLD, Australia, 5Agency for Health Economic Assessment & Dissemination (AHEAD), Bietigheim-Bissingen, BW, Germany","locationCode":"2026","description":"\r\n\t<div>OBJECTIVES: New therapies for Hereditary Angioedema (HAE), a rare, genetic disease, are currently in development. <span class=\"NormalTextRun SCXW125040806 BCX8\" data-ccp-charstyle=\"normaltextrun\" data-ccp-charstyle-defn=\"{"ObjectId":"3f436a9b-17e7-401c-af7b-5521575a786e|5","ClassId":1073872969,"Properties":[469775450,"normaltextrun",201340122,"1",134233614,"true",469778129,"normaltextrun",335572020,"1",469778324,"Default Paragraph Font"]}\">This comprehensive review sought to assess the design and analysis capabilities of currently available HAE economic models. METHODS: We conducted a systematic literature review (SLR) (PROSPERO <span class=\"NormalTextRun SCXW90636482 BCX8\" data-ccp-charstyle=\"normaltextrun\" data-ccp-charstyle-defn=\"{"ObjectId":"3f436a9b-17e7-401c-af7b-5521575a786e|5","ClassId":1073872969,"Properties":[469775450,"normaltextrun",201340122,"1",134233614,"true",469778129,"normaltextrun",335572020,"1",469778324,"Default Paragraph Font"]}\">42022351716) of comparative health economic models in HAE between January 1, 2007, and July 1, 2022. We included models described in manuscripts, conference proceedings, and health technology assessment (HTA) reports. We used Grading of Recommendations Assessment, Development and Evaluation (GRADE) methodology to evaluate study quality; we assessed quality of reporting using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) guidelines. We abstracted data on model design, characterization of attacks (location, severity, duration), pharmacotherapies, analyses, and outcomes reported. RESULTS: Twenty-two models met inclusion criteria. Overall, HTA submissions had higher GRADE and CHEERS scores versus publications. Fourteen models evaluated on-demand therapy only; four evaluated long-term prophylaxis only, and four evaluated both. Markov modeling methodology was the most common (n=7), followed by decision tree modeling (n=5). Five long-term prophylaxis models had time-horizons long enough to sufficiently capture all relevant costs and outcomes associated with this lifetime chronic condition. Only five models included all important aspects of disease burden. Fifteen models included health-related quality of life, however only two included the impact of route of treatment administration on quality of life. Only five models included all costs relevant to patients, payers, and society. CONCLUSIONS: In this first-known systematic review of HAE economic models, we found that few existing frameworks accounted for the holistic burden of HAE attacks from multiple perspectives. To enable a comprehensive analysis, future HAE models should include all factors relating to disease burden, health related quality of life, and costs relevant to patients, carers, payers, and society. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/he-haeeconomicmodelsreview-poster-ispor-eu2023-116uploadfinal101223129317-pdf.pdf?sfvrsn=404e0fe0_0","title":"HE_-_HAE_economic_models_review.Poster.ISPOR-EU_2023.116_Upload_Final_101223129317.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129317","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of Short-Acting Beta-2 Agonist Overuse Among Asthma Patients in Turkiye: A Cost Analysis with Respect to Updated Recommendations of the Global Initiative for Asthma","id":"f76089e6-c638-4ae7-abfe-d730f9943925","sessionCode":"EE102","topDisplay":"Yorgancıoğlu A1, Aksu K2, Cura C3, Yaman Y4, Dinc M4, Malhan S5 1Manisa Celal Bayar University Faculty of Medicine, Manisa, on behalf of the SABINA Türkiye Study Group, Turkey, 2University of Health Sciences Ankara Ataturk Sanatoryum Training and Research Hospital, Ankara, Turkey, 3AstraZeneca, Izmir, Turkey, 4AstraZeneca, Istanbul, Turkey, 5Guven Healthcare Group, Ankara, Turkey","locationCode":"2035","description":"\r\n\t<div>OBJECTIVES: To determine economic burden of short-acting β2-agonist (SABA) overuse in Türkiye from payer perspective with respect to updated GINA 2022 treatment recommendations. METHODS: A total of 3,034,879 asthma patients including 525,034 (17.3%) mild asthma patients (0.3 exacerbations/year) and 2,509,845 (82.7%) moderate-severe asthma patients (1.3 exacerbations/year) comprised the study population, via estimations extrapolated from the Türkiye arm of the global SABINA III study. Patients were categorized by their SABA prescriptions with over-prescription defined as ≥ 3 SABA canister prescriptions/year. The economic burden (costs related to the drug and exacerbations) was compared in subgroups of overall (≥0 canisters/year) vs. GINA-recommended (0-2 canisters/year) SABA use, as well as in subgroups of appropriate use (0-2 canisters/year) vs. overuse (≥ 3 canisters/year) with extrapolation of SABINA Türkiye data to Türkiye asthma population. Monetary results were converted by using CBRT March 2023 effective buying rate (20.27 TL/€). RESULTS: The cost burden of overall SABA use with respect to GINA-recommended SABA use in mild asthma and moderate-severe asthma patients was calculated to be €20.43 million and €427.65 million in terms of exacerbations, and to be €829,352 and €7.20 million in terms of drug costs, respectively. The total economic burden arising from not applying recommended SABA use was estimated to be €456.11 million. Considering appropriate use vs. SABA overuse subgroups, SABA overuse in mild and moderate-severe asthma patients was estimated to yield an additional cost of €16.38 million and €385.59 million, respectively in terms of exacerbations, and a total €11.30 million additional drug cost. The overall economic burden arising from SABA overuse was estimated to be €413.27 million. CONCLUSIONS: The estimated total economic burden arising from not applying GINA recommended SABA use indicates that SABA overuse yields considerably high costs for the Turkish Healthcare System, corresponding up to 26% of the total direct cost of asthma reported in our country.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023dincee102poster131030-pdf.pdf?sfvrsn=f8940cb0_0","title":"ISPOREurope2023_Dinc_EE102_POSTER131030.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131030","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Inter-Reviewer Reliability of Literature Screening and Data Extraction for Human and Machine-Assisted Systematic Reviews","id":"1c9c88bc-0041-46aa-94fa-d7376ad6ad11","sessionCode":"MSR27","topDisplay":"Hanegraaf P1, Dagne AW2, Mosselman JJ3, de Jong R3, Abogunrin S4, Queiros L5, Lane M6, Postma M7, Boersma C8, van der Schans J9 1Pitts, Zeist, Netherlands, 2Health-Ecore, Zeist, Utrecht, Netherlands, 3Pitts, Zeist, UT, Netherlands, 4Evidera, Basel, BS, Switzerland, 5F. Hoffmann-La Roche, Basel, Switzerland, 6F. Hoffmann-La Roche, Basel, BS, Switzerland, 7Health-Ecore, Zeist, UT, Netherlands, 8University of Groningen, Department of Health Sciences, UMCG; Open University, Heerlen, Department of Management Sciences and Health-Ecore Ltd, Zeist, The Netherlands, Groningen, Netherlands, 9Health-Ecore, Groningen, GR, Netherlands","locationCode":"5044","description":"\r\n\t<div>OBJECTIVES: Machine learning can be used for both fully automated or assisted screening and eligibility assessment, as well as to support data extraction efforts, and has shown promising potential over the recent years. <div>However, both an assessment and a reference standard of the inter-reviewer reliability (IRR) between human literature reviewers or between human and machine- assisted reviewers is missing. <div>Our objective is to assess the IRR reported in published systematic reviews and to determine the expected IRR by authors of SLRs for both human and machine-assisted reviews.</div> <div> METHODS: We performed a review of SLRs of randomized controlled trials. Data was extracted on IRR by means of Cohen’s kappa score of abstract/title screening, full text screening, and data extraction. For the second part of this study, we performed a survey of authors of SLRs on their expectations of machine learning automation and human performed IRR in SLRs.</div> </div> RESULTS: Most studies in our SLR did not report on the IRR. In total, 45 eligible articles were included. The average Cohen’s kappa score reported was 0.82 (SD= 0.11, n=12) for abstract screening, 0.77 (SD= 0.18, n=14) for full text screening, 0.86 (SD=0.07, n=15) for the whole screening process, and 0.88 (SD= 0.08, n=16) for data extraction. The survey (n=37) showed overlapping expected Cohen’s kappa values ranging between approximately 0.6-0.9 for either human or machine learning assisted SLRs. In general, authors expect a higher-than-average IRR for machine learning assisted SLR compared to human based SLR in both the screening and the data extraction. CONCLUSIONS: Human performed SLRs likely show a moderate agreement between reviewers, while authors expect machine learning assisted SLRs to perform better. A minimal strong agreement between reviewers of machine learning assisted SLRs is recommended to ensure overall acceptance of machine learning in SLRs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023vanderschansmsr27poster130908-pdf.pdf?sfvrsn=868849da_0","title":"ISPOREurope2023_vanderSchans_MSR27_POSTER130908.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130908","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Estimating EQ-5D Utilities From the Facial Vitiligo Area Scoring Index (F-VASI) in Vitiligo Patients Using a Mapping Algorithm Applied to 2 Pooled Randomized Controlled Trials","id":"522050e7-f330-4462-a62f-d75f2b745bce","sessionCode":"EE107","topDisplay":"Begum R1, Loh J2, Delattre C3, Martina R4, Loizidou E5, Khan I6, Crott R7 1Regulatory Scientific and Health Solutions, Birmingham, West Midlands, UK, 2Incyte Biosciences UK Ltd, Leatherhead, England, UK, 3Incyte Biosciences International, Morges, VD, Switzerland, 4Open University UK, Milton Keynes, Buckinghamshire, UK, 5University of Cyprus, Nicosia, Cyprus, 6University of Warwick, Coventry, West Midlands, UK, 7Catholic University of Louvain, Brussels, Brussels 1200, Belgium","locationCode":"2039","description":"\r\n\t<div>OBJECTIVES: EQ-5D utilities are the preferred measure of health-related quality of life (HRQoL) for economic evaluation. Mapping is used to predict utilities from the Vitiligo Area Score Index (VASI), using data from TRuE-V1 and TRuE-V2 trials in patients treated for nonsegmental vitiligo using 1.5% ruxolitinib cream twice daily. METHODS: A published mapping model from VASI assessed on hands and head/face was applied to independent TRuE-V data. VASI computed as a percentage change from baseline was categorized as achieving 0%–24%, 25%–49%, 50%–74%, and 75%–100% repigmentation. Adjustments to VASI categories to reflect repigmentation vs depigmentation were also implemented. Scores for patients with depigmentation were truncated to identify those having skin pigmentation loss ≤37.5% (category –37.5% to –1%). Mean depigmentation for patients across both arms was 37.68%. Two further categories (–25% to –1%; –50% to –1%) for sensitivity analyses were included. The mapping model used was: EQ-5D utility = 0.709 + (0.0119*VASI Categories) – (0.000214*VASI Categories2) + (0.00000118* VASI Categories3). The algorithm was applied to facial VASI (F-VASI) outcomes at Weeks 12, 24, and 52. RESULTS: The correlation between total body VASI (T-VASI) and F-VASI from pooled TRuE-V studies was 0.73 (P<0.001). At Week 24, mean (SE) utility was 0.8588 (0.0108) for 1.5% ruxolitinib cream vs 0.8150 (0.0193) for vehicle (P=0.050, –37.5% depigmentation). Mean utility increased with Vitiligo Noticeability Scale (VNS) scores 4 or 5 (0.893 for “a lot less noticeable” or “no longer noticeable”) vs VNS scores 1 to 3 (0.831 for “more noticeable,” “as noticeable,” or “slightly less noticeable”). The mapping model confirmed statistical association between VASI on head/face and VASI on hands (P<0.001). CONCLUSIONS: Mean utilities at Week 24 were higher for 1.5% ruxolitinib cream compared with vehicle. Mean utilities increased as VNS and other HRQoL outcomes improved for patients with repigmentation vs depigmentation using F-VASI.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23begumee107133879-pdf.pdf?sfvrsn=586afa1f_0","title":"ISPOREurope23_Begum_EE107133879.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133879","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Retrospective Review of Recent NICE Technology Appraisals From 2021 to 2023 to Investigate the Consideration and Acceptability of Cure Modelling Assumptions","id":"d2b81005-8057-47d9-9b07-d7b04baea1bd","sessionCode":"HTA21","topDisplay":"Alexopoulos T, McLean T, Herbert A Merck Serono Ltd, Feltham, UK, an affiliate of Merck KGaA, Darmstadt, Germany","locationCode":"4046","description":"\r\n\t<div>OBJECTIVES: Curative therapies have the potential to restore patient health and significantly improve survival. However, cure assumptions in HTA appraisals can be difficult to model and support with evidence. The aim of this research was to investigate how cure assumptions have been clinically justified, modelled, and considered in recent NICE technology appraisals across all therapy areas. METHODS: A targeted search was conducted of all NICE single technology appraisals published in the last two years which included cure assumptions. Appraisals were excluded if a cure assumption was not explicitly modelled. Data extracted included details of the cure assumption, clinical data supporting the cure assumption, cure modelling approach and HTA feedback on the cure assumption. RESULTS: A total of 11 appraisals were identified in the search. Although the search was not limited to oncology therapies, all 11 appraisals were in oncology. Most appraisals (55%, n=6) applied a cure assumption after patients were progression-free for 5 years, while 27% (n=3) of appraisals used 2 years. The remaining two appraisals applied cure assumptions at 3 years and 30 months. There were two separate approaches to cure modelling noted. In one approach, general population mortality rate was assumed after the cure timepoint, which was applied in the majority of appraisals (73%, n=8). The other approach was the development of a mixture-cure model which was applied in 27% (n=3) of appraisals. Finally, in all 11 appraisals both the ERG/EAG and NICE considered the cure assumption uncertain, with varying degrees of acceptability. CONCLUSIONS: All NICE appraisals in the last two years where cure assumptions have been applied were in oncology. Despite variability in data informing the assumption and modelling approach, NICE and the ERG/EAG typically considered the assumption to be highly uncertain. However, in most cases the uncertainty around the cure assumption was accepted if the medicine was cost-effective. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23alexopouloshta21poster133224-pdf.pdf?sfvrsn=19069a1b_0","title":"ISPOREurope23_Alexopoulos_HTA21_POSTER133224.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133224","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Ribociclib Plus a Non-Steroidal Aromatase Inhibitor for the Treatment of Pre- and Peri-Menopausal Women with HR+/HER2 Advanced Breast Cancer in Turkey","id":"09f09137-dfae-4525-956a-d7e63f3b4e89","sessionCode":"EE80","topDisplay":"Tatar M1, Caglar P2, Arbay D2, Akdemir AC2 1Polar Health Economics and Policy, Ankara, Turkey, 2Novartis, İstanbul, Turkey","locationCode":"1041","description":"\r\n\t<div>OBJECTIVES: The objective of the study is to evaluate the cost effectiveness (CE) of ribociclib plus a non-steroidal aromatase inhibitor (NSAI) and goserelin (ribociclib treatment hereafter) for pre- and peri-menopausal women with HR+/HER2- advanced breast cancer (ABC) who have not received prior hormonal therapy. METHODS: A Markov model with a 15-year time horizon and a 28-day cycle length was designed to evaluate the CE of ribociclib treatment in comparison to an NSAI, exemestane, paclitaxel and docetaxel treatments. The model was comprised of three stages as progression free survival, post-progression survival and death. The results of MONALEESA-7 trial were used for effectiveness data. An indirect comparison was made to compare the effectiveness of ribociclib treatment with exemestane, paclitaxel and docetaxel. Only direct costs were included in the model as per the requirements of the Social Security Institution (SSI). Costs were estimated based on expert views of resource use for treatment of adverse events, monitoring, disease progression terminal care and pre- and post- progression. Both costs and outcomes were discounted by 3%. ICER was calculated as the incremental cost per incremental life years (LY) as required by the SSI. RESULTS: The ribociclib treatment estimated to yield 8.29 LYs. The incremental LY for comparators were 2.17, 2.33, 3.68 and 2.18 for an NSAI, exemestane, paclitaxel and docetaxel respectively. The incremental cost per incremental LY was 246,572 TRY for an NSAI treatment, 224,669 TRY for exemestane, 148,733 TRY for paclitaxel and 201,210 TRY for docetaxel. The sensitivity analyses indicated that the CE results were robust. CONCLUSIONS: Turkey does not have an official threshold for decision-making. If the GDP per capita is taken into account (three times GDP per capita is 546,806TRY) ribociclib treatment for pre- and peri-menopausal women with HR+/HER2- ABC is a cost-effective option in Turkey.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129640","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"\"I'll Live Less, You'll Live Better, Let's Make It Count\": A Qualitative Exploration of Drivers &AMP; Barriers for Sacrificing Remaining Life Expectancy to Restore Relatives to Full Health in Time Trade Off Exercises","id":"b01f7d00-8c1a-4d29-81c7-d87b36fea168","sessionCode":"EE147","topDisplay":"Wratten S1, Batchelder L2, Praet A3, Krol M4 1IQVIA, London, London, UK, 2IQVIA, Swindon, WIL, UK, 3Erasmus University, Rotterdam, Rotterdam, Netherlands, 4IQVIA, Amsterdam, ZH, Netherlands","locationCode":"2076","description":"\r\n\t<div>OBJECTIVES: To understand factors impacting the time people are willing to sacrifice to restore a non-healthy family member to full health in a new Time Trade Off (TTO) method for estimating non-patient utilities. METHODS: Semi-structured interviews lasting 45 minutes were conducted with 10 participants based in the United Kingdom. Draft TTO exercises describing hypothetical family members (child/partner/parent/grandparent) in moderately and severely poor health states (HS) were presented to participants. Cognitive interviewing was used to confirm participants’ understanding of each exercise and to identify factors influencing how many years of their own remaining life expectancy they’d be willing to sacrifice to restore each relative’s health in each exercise. Thematic analysis was used to identify themes in factors impacting participant responses. RESULTS: Participants (N=10) were male (n=6) or female (n=4). Most did not currently live with a non-healthy family member (n=7). The largest sacrifices (average: 83% of remaining life expectancy) were to restore a child to full health from a severe HS, and the smallest sacrifices (average: 8% of remaining life expectancy) were to restore a grandparent to full health from a moderate HS. Factors impacting the sacrifices made were organized into drivers (n=17; factors increasing number of years sacrificed) and barriers (n=18; factors decreasing number of years sacrificed). Key drivers were altruism (including improving relatives’ quality of life; n=10), benefits for self (including avoiding providing care for a relative; n=7), and the mutual benefit of having quality remaining time together (n=9). The relative’s age, the quality of the relationship with the relative, and one’s own goals and ambitions were both drivers and barriers depending on the context. CONCLUSIONS: Many factors influence the sacrifices one might make to restore a relative’s health. The strong influence of altruism may need to be corrected for to use this TTO approach to elicit non-patient utilities.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/non-patient-utilities-qual-poster-ispor-eu-v1-0129089-pdf.pdf?sfvrsn=118c969e_0","title":"Non patient utilities qual poster ISPOR EU v1.0129089.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129089","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness Analysis of LUNGFLAGTM Risk Prediction Model in the Selection of Individuals for Lung Cancer Screening in Spain","id":"d73788cc-2e16-42b8-9c5f-d9382a55982b","sessionCode":"EE74","topDisplay":"Pajares V1, Soriano JB2, Seijo LM3, Trujillo JC1, Marzo M4, Olmedo-García ME5, Higuera O6, Arrabal N7, Flores A7, García JF7, Carcedo D8, Crespo M9, Heuser C10, Olghi N11, Choman E12, Gorospe L5 1Hospital de la Santa Creu i Sant Pau, Barcelona, Spain, 2Hospital Universitario La Princesa, Madrid, Spain, 3Clínica Universidad de Navarra and Ciberes, Madrid, Spain, 4Grupo de Investigación del Cáncer en Atención Primaria, Barcelona, Spain, 5Hospital Universitario Ramón y Cajal, Madrid, Spain, 6Hospital Universitario La Paz, Madrid, Spain, 7Roche Farma S.A., Madrid, Spain, 8Hygeia Consulting, Madrid, M, Spain, 9Hygeia Consulting, Madrid, Spain, 10Hoffman-la Roche, Neuenburg am Rhein, BW, Germany, 11Hoffman-la Roche, Basel, Switzerland, 12Medial Early-Sign, Hod Hasharon, Israel","locationCode":"2007","description":"\r\n\t<div>OBJECTIVES: LungFlagTM is an A.I. risk prediction model that has proved to be effective in the selection of individuals for non-small cell lung cancer (NSCLC) screening. The aim of this analysis was to assess the cost-effectiveness of implementing LungFlagTM for the selection of individuals at-risk for enrollment in NSCLC screening programs in Spain. METHODS: A combination of a decision-tree and a Markov model was adapted to the Spanish setting. A multidisciplinary group of experts validated all the parameters and assumptions of the model. A hypothetical cohort of 3.835.128 individuals who met 2013 USPSTF criteria in Spain was defined as the target population. Screening with LungFlagTM was compared to screening the whole population with no risk model and versus no-screening (current situation). Sensitivity and specificity values for LungFlagTM were obtained from Gould et al. (2021). Adherence to screening programs was assumed to be the same for both screening scenarios. A lifetime horizon was used, so a 3% discount rate was applied for both cost and effects. Only direct medical costs were considered (€2023). Sensitivity analyses were performed to assess uncertainty. RESULTS: Using LungFlagTM to identify subjects at risk would result in 232,120 low-dose computed topographies (LDCTs) performed, compared to 2,1M of LDCTs if the whole target population was screened. Therefore, significant cost savings were obtained over the lifetime horizon (€-7,04M). However, this would mean fewer quality-adjusted life-years (QALYs) gained (-97,612 QALYs) because of fewer people screened. The incremental cost-utility ratio obtained showed that the savings generated by LungFlagTM offset the loss of QALYs, making it a cost-effective strategy. Both screening scenarios (using LungFlagTM or screening the whole target population) were dominant compared to no-screening. CONCLUSIONS: Using LungFlagTM for the selection of individuals at-risk of NSCLC may be a cost-effective strategy compared to screening all individuals meeting USPSTF 2013 criteria or no-screening.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23heuseree74poster131110-pdf.pdf?sfvrsn=839cd52e_0","title":"ISPOREurope23_Heuser_EE74_POSTER131110.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131110","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Empagliflozin in Patients Affected By Chronic Kidney Disease in Italy","id":"8ff26718-d5b8-45a5-ab22-d95bd1b3c81f","sessionCode":"EE117","topDisplay":"Di Costanzo A1, Uster A2, Vassallo C1, Fiorentino F1 1IQVIA Solutions S.r.l. Italy, Milan, Italy, 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany","locationCode":"2052","description":"\r\n\t<div>OBJECTIVES: Chronic Kidney Disease (CKD) is a prevalent condition associated to a great clinical and economic burden. The phase III randomized controlled trial EMPA-KIDNEY found empagliflozin, a sodium glucose co-transporter 2 inhibitor, to be effective in reducing CKD progression and cardiovascular mortality in both diabetic and non-diabetic patients with CKD. The study aims to assess the cost-effectiveness of empagliflozin in patients affected by CKD in stages II-IV from the Italian National Health Service (NHS) perspective. METHODS: A Markov state microsimulation cost-effectiveness model was developed to estimate lifetime costs and QALY of adult patients with stages II-IV CKD treated either with SoC (Renin-Angiotensin-System inhibitors) or with empagliflozin+SoC. At model entrance patients are randomly assigned baseline characteristics and are distributed across mutually exclusive health states based on KDIGO (Kidney Disease Improving Global Outcomes) classification. In each yearly cycle, patients’ risk factors evolve and disease progresses, with patients incurring in chronic and acute complications, such as cardiovascular diseases and infections, while proceeding individually through health states until reaching end-stage kidney disease (ESKD) or dying. Initial distribution among health states and treatment efficacy were retrieved from EMPA-KIDNEY trial, utilities from published literature, while risk of clinical events and unit costs were informed from literature and Italian reports and were validated by Italian clinical experts. RESULTS: In Italy, empagliflozin+SoC resulted as a dominant strategy for patients with stages II-IV CKD compared to SoC, yielding lower costs and higher QALY, driven by the lower incidence of patients reaching ESKD on kidney replacement therapy in the empagliflozin+SoC arm. Moreover, adding empagliflozin to SoC improved patient’s life expectancy. CONCLUSIONS: Our findings suggest that, by being a dominant strategy, utilization of empagliflozin+SoC for the treatment of patients with stages II-IV CKD would contribute for the financial sustainability of the Italian NHS while improving patients’ wellbeing and health status.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23vassalloee117poster130900-pdf.pdf?sfvrsn=e48fcf35_0","title":"ISPOREurope23_Vassallo_EE117_POSTER130900.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130900","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of Type 2 Diabetes in Peru: A Cost-of-Illness Study Valuing Cost Differences Associated With the Level of Glycemic Control","id":"f5df86fc-9e9d-4083-b72e-da14aefaceb4","sessionCode":"EE18","topDisplay":"Seinfeld J1, Sobrevilla A1, Rosales ML1, Ibañez M1, Ruiz D1, Penny E1, Londono S2 1Videnza Consultores, Lima, Peru, 2Sanofi, Bogota, CUN, Colombia","locationCode":"1063","description":"\r\n\t<div>OBJECTIVES: Type 2 diabetes mellitus (T2DM) represents a public health problem with a great impact in middle-income countries such as Peru. This study aimed to estimate the national economic burden of this disease for the public sector (SIS), social insurance (Essalud), and private sector (HMOs). METHODS: Direct healthcare costs were estimated for a cohort of 443,683 adults between 45 and 75 years old diagnosed with T2DM in 2019 (5.9% prevalence registered in national databases). Disease progression over a 20-year period was modeled using the PROSIT tool and published sources, including acute and chronic (macrovascular and microvascular) complications associated with T2DM. Costs were estimated considering the current level of glycemic control, with 35.8% of the population under optimal control (HbA1C<7%); and under two additional hypothetic scenarios (100% HbA1C<7% and 100% HbA1C>7%). Sensitivity analysis was performed for diabetes prevalence considering values reported for the Latin American region by the International Diabetes Federation. RESULTS: The total national economic burden was estimated at USD$ 15,405,448,731; cost that would oscillate between USD$ 12,853,113,596 and USD$ 16,828,713,495, depending on the level of glycemic control. The average annual cost of treating a T2DM patient was USD$ 2,158, which would decrease to USD$ 1,797 if patients are controlled and increase to USD$ 2,360 if they are not. Costs for patients with complications and risk factors (dyslipidemia, hypertension and obesity) were around 6.5 times greater compared to those without them, being stroke the complication that contributed the most to the economic burden. Sensitivity analysis showed an increase of 67.6% in total costs with a 9.9% prevalence. CONCLUSIONS: T2DM places a heavy burden on the Peruvian healthcare budget and will represent a higher cost impact if poor glycemic control is maintained. Addressing this disease at early stages could alleviate the economic burden and improve the quality of life of individuals.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23londonoee18poster132107-pdf.pdf?sfvrsn=1af4fc75_0","title":"ISPOREurope23_Londono_EE18_POSTER132107.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132107","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Making MCDA 'Shiny' With an Adaptive Support Tool for Healthcare Decision-Making: An Application in Broad Molecular Testing With R Shiny","id":"f3918c13-ee2e-4cf7-99dc-da8445843a14","sessionCode":"HTA40","topDisplay":"Fernandez Coves A1, van Schaik L2, Ramaekers B1, Grimm S1, Joore M1, Retel V2 1Maastricht University Medical Centre+, Maastricht, LI, Netherlands, 2The Netherlands Cancer Institute, Amsterdam, Noord-Holland, Netherlands","locationCode":"4076","description":"\r\n\t<div>OBJECTIVES: Reimbursement decisions about innovative medical technologies are notoriously challenging, as they commonly have limited and rapidly evolving evidence, an uncertain position in the care pathway, and may impact criteria beyond clinical-, and cost-effectiveness. Multiple-criteria decision analysis (MCDA) helps evaluate these often conflicting criteria. However, its use in current appraisal processes is scarce and complex. We aimed to develop an adaptive decision tool that facilitates the use of MCDA in the decision-making process. We used the case of broad molecular testing in oncology to illustrate its use. METHODS: We developed a decision support tool with a web browser-based interface using R Shiny to allow users to easily perform statistical analyses based on their decision-making needs. The tool was developed to include a customizable framework so users can incorporate their data and analyses into the existing tool. An online tutorial is available to showcase the necessary steps. For our application in broad molecular testing, we followed the guidance of the Dutch HealthCare Institute. RESULTS: The tool allows the entry of MCDA data, uncertainty information, and relative weights for each criterium. It provides visual communication of the MCDA input and results from different perspectives, which can be stored and downloaded. Furthermore, it allows the decision-maker’s assessment of the criteria and ultimate appraisal of the technology. In the case of broad molecular testing, we developed a spiderweb graph to communicate the weighting of five determinants derived from the literature and interviews: feasibility, implications of diagnostics results, laboratories organization, patient journey, and scientific spillover, next to clinical- and cost-effectiveness. CONCLUSIONS: The R Shiny MCDA decision support tool facilitates systematic and transparent communication of information and preferences from different stakeholders; while fostering evidence-informed decision-making. The systematic approach for including additional determinants besides health benefits and costs is an innovative approach to decision-making in broad molecular testing.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor23posterhta40132198-pdf.pdf?sfvrsn=c6ca23e2_0","title":"ispor23_poster_HTA40132198.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132198","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Linking the Price of a Drug to Its Real-Life Performance: A Solution for Financing Innovative Therapies in France?","id":"a0f14c3b-ba7d-4aab-8c16-dadd63de6027","sessionCode":"HPR41","topDisplay":"Azzazene S1, Martin T2, Borget I3 1Paris-Saclay University, Paris, 75, France, 2Assistance Publique des Hôpitaux de Paris, Paris-Saclay, 75, France, 3Gustave Roussy Institute, Villejuif, France","locationCode":"3078","description":"\r\n\t<div>OBJECTIVES: This research explores the use of risk-sharing agreements as a pricing scheme for innovative therapies in France. There is conceptual interest in this type of agreement, which stands out as an exception in the landscape of market access for medicines and determination of therapeutic value. Although they represent an attempt to price disruptive innovations, they are characterised by difficulties in implementation, particularly regarding the use of real-life data. The objective is to investigate the applicability of these agreements for innovative drug pricing and to clarify the framework surrounding the use of clinical evidence. METHODS: A qualitative approach examined the barriers and opportunities associated with the subject. Semi-structured interviews were conducted using a thematic interview grid to gather data from the relevant stakeholders. The interviews were recorded and transcribed, then the data was analysed using interview coding. An open and then axial coding analysis was employed to group the responses into themes and categories to obtain an in-depth understanding of the respondents' perceptions. RESULTS: Semi-structured interviews were conducted with nine stakeholders (out of 13 contacted, response rate =62%): two patient association representatives, four pharmaceutical industry employees responsible for market access and pricing, four public stakeholders (French Ministry of Health and French Ministry of the Economy), two researchers in health economics and law, one pharmaceutical industry union representative. Risk-sharing agreements are seen by stakeholders as an opportunity for providing contributory funding for innovative drugs and as leverage for price negotiation. However, major structural limitations were highlighted concerning the use of real data, the lack of budgetary predictability and divergences over their very purpose. CONCLUSIONS: The study highlights the need for structural changes in the use of such agreements, including improved management of medico-administrative databases and clarification of their ambitions regarding innovative health products, to guarantee the economic sustainability of healthcare expenditure.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133891","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Performance of Progression-Based and Time-to-Death Utility Analysis Methods and Their Implications on Economic Models: A Targeted Review","id":"f18bda14-4f7f-4099-a601-db0db5e6e255","sessionCode":"MSR11","topDisplay":"Hopmans Galofré G1, Clayson M1, Tanova-Yotova N2, Genestier V3 1Amaris Consulting, Barcelona, Spain, 2Amaris Consulting, Sofia, Bulgaria, 3Amaris Consulting, Toronto, ON, Canada","locationCode":"5058","description":"\r\n\t<div>OBJECTIVES: Oncology models commonly use health state utility values (HSUVs) related to progression-free and post-progression states, though recent utility analysis method (UAM) studies have considered time-to-death (TTD) covariates to predict utility. Guidance is not available from health technology assessment agencies to choose the appropriate UAM. This study aimed to compare statistical outcomes of UAMs and their effects on economic model outcomes. METHODS: Targeted literature reviews of UAM studies and economic models in oncology assessing progression-based, TTD-based, and combined utilities were conducted. RESULTS: 16 UAM studies and six economic models were included. All UAM studies reported numerically larger decrements from baseline to the period closest to death than baseline to post-progression and six provided goodness-of-fit statistics. One study found TTD producing more accurate predictions than progression (lower root mean squared error (RMSE) and higher R2) using SF-6D and EORTC-8D and another study in the same indication found only TTD to significantly predict utility using EORTC-8D. Progression-based and TTD covariates were both statistically significant in a study using EQ-5D-5L and EORTC-QLU-C10D, though goodness-of-fit was better (via Akaike information criterion and Bayesian information criterion) for progression-based models using EQ-5D-5L and similar using EORTC-QLU-C10D. Three analyses assessed combined UAMs, and two using EQ-5D had the best goodness-of-fit when using the combined approach. The third reported lower RMSE and mean absolute error for progression-based covariates using SF-6D data but the opposite using EORTC-8D. One included simulation study reported that combined UAMs consistently overestimated quality-adjusted life-years (QALYs). Among all economic models, progression-based HSUVs caused higher incremental cost-effectiveness ratios per QALY (range: 3.06―10.43%; N=6) and lower incremental QALYs (range: 6.84-9.47%; N=2) compared to TTD HSUVs. CONCLUSIONS: Incorporating TTD in UAMs may improve utility predictions for advanced oncology indications, particularly when using EORTC-based utilities. The UAM choice should be carefully assessed since significant heterogeneity exists between indications and data sets.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/msr11performance-of-progression-based-and-time-to-death-utility-methods-and-their-implications-on-economic-models-a-targeted-review133274-pdf.pdf?sfvrsn=f9f03ae6_0","title":"MSR11_Performance of progression-based and time-to-death utility methods and their implications on economic models. A targeted review133274.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133274","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Health-Economic Analysis of Digestive Stents in France Based on the French Social Insurance Healthcare Database","id":"f0334f09-2aee-4bb9-ad4c-db8f49d2dc36","sessionCode":"EE129","topDisplay":"de Launay J1, Saidani N2, Mison F3, Bertucat A4, Lamarsalle L5, Panes A5, Spoljar T6 1Becton, Dickinson and Company, Paris, Île-de-France, France, 2Boston Scientific, Voisins le Bretonneux, Yvelines, France, 3COOK France, Paris, Île-de-France, France, 4Duomed France Endoscopie, Bagnolet, Île-de-France, France, 5HEVA, Lyon, Rhône, France, 6Heva, USA","locationCode":"2061","description":"\r\n\t<div>OBJECTIVES: Describe cost of stays and implantation of digestive stents (DS) and simulate the impact of potential inclusion of DS in DRGs. METHODS: An observational retrospective study was conducted using the French Hospital Discharge System (Programme de Médicalisation des Systèmes d’Information, PMSI). The study population consisted of patients who received a DS implantation in France in 2019 and 2020. The costs of stays were described for public and private hospitals. The potential inclusion of the costs of these implants into DRG tariffs was also simulated. RESULTS: The frequency of stents used in the main DRGs was <1% for digestive non-biliary localizations in public and private hospitals. DS were used in 2,314 stays identified in 2 main DRGs (1,160 and 1,154) in public hospitals among 295,922 and 209,499 total stays respectively. Total cost of these stays with DS were €977,832 and €1,042,174 respectively, for an average DS cost/stay of €3 and €5. Simulation of the inclusion of these stents in DRGs was not conclusive. Biliary localizations were more frequent (9.7% in public hospitals, 17.6% in private hospitals). DS were used in 16,592 stays identified in 2 main DRGs (11,094 and 5,498) in public hospitals among 119,804 and 65,830 total stays respectively. Total cost of these stays with DS were €7,436,929 and €4,624,381 respectively, for an average DS cost/stay of €62 and €70. Simulation of the inclusion of these stents in DRGs resulted in a modest windfall effect with a maximum of €550,000. Results were similar for private hospitals. CONCLUSIONS: This study highlights that the inclusion of low numbers of digestive stents into a high-use DRG, could result in under-reimbursement under circumstances of stent use. The inclusion in DRGs could potentially reduce the chances of treatment for patients concerned, particularly for biliary localizations that are mostly done by centers of excellence.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23spoljaree129poster132899-pdf.pdf?sfvrsn=587af7e2_0","title":"ISPOREurope23_Spoljar_EE129_POSTER132899.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132899","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Non-Alcoholic Fatty Liver Disease Awareness in the United States: National Health and Nutrition Examination Survey Data 1999-2020","id":"9d2cfe91-fe5f-4298-916f-dbc77f5e4f34","sessionCode":"EPH9","topDisplay":"Chhatwal J1, Aaron A2, Loomba R3, Haseeb M4, Tapper E5, Sonawane K6, Kanwal F7 1Harvard University, Boston, MA, USA, 2Massachusetts General Hospital, Boston, MA, USA, 3University of California at San Diego, San Diego, CA, USA, 4Beth Israel Deaconess Medical Center, Boston, MA, USA, 5University of Michigan, Ann Arbor, MI, USA, 6Medical University of South Carolina, Charelston, SC, USA, 7Baylor College of Medicine, Houston, TX, USA","locationCode":"3012","description":"\r\n\t<div>OBJECTIVES: Non-alcoholic fatty liver disease (NAFLD) is one of the leading risk factors for liver cancer and liver transplant. However, most patients become aware of their disease in late stages. Our objective was to estimate the prevalence and factors associated with the diagnosis of NAFLD among adults in the United States from 1999 to 2020. METHODS: We used National Health and Nutrition Examination Survey (NHANES) data from 1999-2020. A diagnosis of NAFLD was determined based on a United States Fatty Liver Index (USFLI) score of greater than 30. Individuals were excluded if they were under 18, pregnant, had hepatitis B/C, were missing variables utilized in the USFLI, or had evidence of heavy drinking. An individual was assumed to have NAFLD with advanced fibrosis if their FIB-4 score was greater than 2.67. A respondent’s awareness of their liver condition was established by their responses to the NHANES medical questionnaire. The associations between demographic factors and NAFLD awareness were tested using multivariate logistic regression. RESULTS: Overall, as many as 32.4% (31.3-33.5%) of the U.S. adult population may have NAFLD, while only 4.9% (4.1-5.8%) are presently aware. 2.9% (2.5-3.3%) of U.S. adults may have NAFLD with advanced fibrosis, and among these individuals, 13.4% (9.4-17.4%) were aware of their condition. Individuals who were aware of their NAFLD were more likely to be Hispanic, OR=1.49 (1.00-2.22); Obese III, OR=2.60 (1.18-5.74); have a place for routine care, OR=3.20 (1.47-6.96); or have fibrosis, OR=3.14 (1.93-5.11) than those who were not aware. However, compared to white respondents, those who identified as non-Hispanic black were significantly less likely to be aware of their NAFLD, OR=0.39 (0.20-0.73). CONCLUSIONS: Nearly one-third of U.S. adults may have NAFLD; however, only one in twenty are aware. Public health interventions are needed to increase NAFLD diagnosis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23chhatwaleph9poster133402-pdf.pdf?sfvrsn=809860ff_0","title":"ISPOREurope23_Chhatwal_EPH9_POSTER133402.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133402","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Impact of Dispensed Medication on Mean Age of Death: A Brazilian Public Health System Study","id":"8dd86660-a79c-4a22-aff0-dc353b76c688","sessionCode":"EPH50","topDisplay":"Bigoni A1, Belli K2, Leonel L3, Pungartnik P3, Pereira RG3, Gomes Moura IC3 1IQVIA, Limeira - SP, Brazil, 2IQVIA, Porto Alegre, Brazil, 3IQVIA, São Paulo, São Paulo, Brazil","locationCode":"3051","description":"\r\n\t<div>OBJECTIVES: Medications improve quality of life and longevity, however their population-level impact on the average age of death remains unclear. This study aims to assess the association between public health system-dispensed medication and the mean age of death in Brazil from 2011-2019. METHODS: This is a retrospective database study of anonymized microdata from the Brazilian Ministry of Health. Information on mean age of death was obtained from the Brazilian Mortality Information System, while hospitalization and dispensed medication details were extracted from the Inpatient and Outpatient Information System. Prais-Winsten regression were used to estimate Monthly Average Percentage Change (MAPC). We created a panel using month and disease categories as the panel units. Fixed-effect regression models were used to control for potential confounders. RESULTS: Dispensed medication increased for all disease groups, except for skin and subcutaneous tissue diseases (-15.7) and neoplasms (-14.3). Largest increases were seen in circulatory system diseases (217.4) and blood-related disorders (73.7). Unspecified diseases showed a MAPC of 190.3. Mean age of death increased for all significant disease groups, more notably in infectious and parasitic diseases (12.1) and the nervous system (9.7). A significant positive correlation was detected between dispensed medication and mean age of death (Coef 0.00529; StdErr 0.00127), even after adjusting for hospitalization-related factors and disease categories. CONCLUSIONS: Dispensed medication in Brazil increased from 2011-2019, possibly contributing to a modest rise in life expectancy. Improving access to medication in the public health system may increase the mean age of death, indicating improved health outcomes. Further study is essential to understand the private health care system's influence on the connection between dispensed medication and the mean age of death for specific disease groups.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterisporeph50iqviav2131788-pdf.pdf?sfvrsn=d836c8a7_0","title":"Poster_ISPOR_EPH50_IQVIA_v2131788.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131788","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The CONNACT Program for Knee Osteoarthritis Can Potentially Lower 2-Year Costs of Outpatient Treatment","id":"719ffd89-c898-4332-8545-dc4167b7ad04","sessionCode":"HSD6","topDisplay":"Lim CJ1, Tan B1, Pua YH2, Pereira M3 1Woodlands Health, Singapore, Singapore, 2Singapore General Hospital, Singapore, Singapore, 3National Healthcare Group, Singapore, 04, Singapore","locationCode":"4016","description":"\r\n\t<div>OBJECTIVES: The Collaborative Model of Care Between Orthopaedics and Allied Healthcare Professionals Trial (CONNACT) is a randomized controlled trial (RCT) that evaluated the effectiveness of an individualised, community-based, multidisciplinary programme, involving physical activity, dietary and psychological support, on functional outcomes of patients with knee osteoarthritis. This CONNACT Total Knee Replacement (TKR) study is an extension of the RCT to examine the programme’s impact on 2-year TKR rates, healthcare utilisation and associated cost, compared to standard-care. METHODS: We mined national-level databases for 2-year outcomes of study patients. Outcomes were: TKR rates, time-to-surgery, acute hospital (AH) length of stay (LOS), community hospital (CH) admissions and LOS, and associated costs of admissions if TKR-positive; Polyclinic, orthopaedic specialist outpatient clinic (SOC) and emergency department (ED) visits, and associated costs of such outpatient visits. Ordinal regressions were used to explore programme effects on TKR and healthcare utilisation, while generalized linear models were used to compare costs between-groups. A propensity score was generated based on age, gender, body mass index, pain and function, and controlled for during analyses. RESULTS: 90 patients were studied. Unilateral and bilateral TKR rates – CONNACT: 6.82%; 2.27% versus standard-care: 10.87%; 4.35%, respectively. CONNACT patients had approximately half the odds of having a TKR [Odds ratio=0.53 (95%CI {confidence interval}=00.14-2.06); p=0.361], however, the wide 95%CI indicated uncertainty of true effects. Non-statistically significant findings were observed for time-to-surgery, AH LOS, CH admissions and LOS, costs of admissions for TKR patients, and visits to polyclinic, orthopaedic SOC and ED. However, lower costs were observed in the CONNACT group for orthopedic SOC [Incident risk ratio (IRR)=0.45 (95%CI=0.21-0.97); p=0.0143] and total associated outpatient costs (polyclinic, orthopedic SOC and ED) [IRR=0.49 (95%CI=0.27-0.88); p=0.018], compared to standard-care. CONCLUSIONS: While the CONNACT programme did not appear to prevent replacement surgery, it may potentially lower 2-year costs of outpatient healthcare utilisation.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-posterfinal30oct134048-pdf.pdf?sfvrsn=37d57e7b_0","title":"ISPOR poster_final_30Oct134048.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/134048","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Utility Analysis of Once-Weekly Semaglutide and Dulaglutide for Patients With Type 2 Diabetes in Saudi Arabia","id":"d6be729b-405e-4a24-b1b6-dc9c46188840","sessionCode":"EE23","topDisplay":"Hassan M1, Barham L2 1Novo Nordisk, Riyadh, 01, Saudi Arabia, 2Learna Ltd in partnership with the University of South Wales, Royston, HRT, UK","locationCode":"1062","description":"\r\n\t<div>OBJECTIVES: The prevalence of type 2 diabetes (T2D) in Saudi Arabia is one of the top ten prevalences globally, which imposes a substantial economic burden on the healthcare system. Glucagon-like peptide-1 agonists (GLP-1) are one of the novel treatments for diabetes. The research aims to determine whether Semaglutide 1.0 mg is cost-effective compared with Dulaglutide 1.5 mg from a Saudi public payer perspective. METHODS: A literature review was conducted to acquire the input parameters based on the available literature that could populate the cost-utility model to inform the Saudi decision-makers better. A literature review was developed to inform the cost-utility analysis (CUA) with the structure of the Markov model in terms of health states that could describe the patient journey, baseline risks that could simulate the patients' movements among the different health states, and clinical outcomes for the comparators, health utilities, and cost data. The second phase was developing the CUA based on the available literature, including only direct medical costs for ten years time horizon. The Incremental Net Monetary Benefit (INMB) was estimated based on the Saudi cost-effectiveness threshold (CET) of 50,000 SAR – 75,000 SAR. Structural and parameter uncertainties were explored by developing sensitivity and scenario analyses. RESULTS: Semaglutide 1.0 mg was dominant in the base case analysis with incremental 0.11 QALY, - 3,175 SAR. INMB ranged from 8,671 SAR to 11,419 SAR on the lower and upper bounds of the CET range, respectively. Semaglutide 1.0 mg sustained the results in all scenarios. One-way sensitivity analysis illustrated that the key drivers of the cost-effectiveness findings are treatment acquisition cost, the proportion of patients achieving HbA1C%, and hazard ratios of developing cardiovascular complications. CONCLUSIONS: At the current prices, Semaglutide 1.0 mg is cost-effective compared with Dulaglutide 1.5 mg when the model considered macrovascular complications and chronic kidney disease (CKD).</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hassanee23poster133131-pdf.pdf?sfvrsn=5522339b_0","title":"ISPOREurope23_Hassan_EE23_POSTER133131.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133131","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization (HCRU), Quality of Life (QoL), and Employment Changes for Early, Middle and Late-Stage People with Amyotrophic Lateral Sclerosis (pALS) in Italy: Results from a Real-World Survey","id":"cda4c2df-8597-48d1-82d7-dcd038280fc2","sessionCode":"EE48","topDisplay":"Stenson K1, Guerrieri E2, Santoni L3, Mellor J4, Wright J4, Earl L4, Ball N4, Iqbal H4, Thomas O4, Barlow S4, Ticozzi N5 1Biogen, Weymouth, MA, USA, 2Biogen Italy, Milan, Italy, 3Biogen Italy, Milano, MI, Italy, 4Adelphi Real World, Bollington, UK, 5Neurology Unit, IRCCS Istituto Auxologico Italiano, Università degli Studi di Milano, Milan, Italy","locationCode":"1093","description":"\r\n\t<div>OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a rare, neurodegenerative disease, leading to progressive loss of muscle function, and ultimately death. We assessed HCRU and impacts on QoL and employment in Italian pALS and their caregivers (cALS). METHODS: Data were drawn from the Adelphi ALS Disease Specific Programme™, a cross-sectional survey of neurologists, pALS and cALS. Data collection in Italy took place between July–October 2020. Neurologists reported demographics of pALS, HCRU including consultations with healthcare professionals (HCPs), ALS-related hospitalizations, use of mobility, ventilatory or feeding interventions, and overall professional caregiver requirements. Neurologists designated whether patients were in the early, middle, or late stage of ALS based on their clinical judgement. These same pALS and cALS reported on employment changes and their QoL. RESULTS: 21 neurologists recorded data on 129 Italian pALS (50, 45 and 34 designated as early-, middle- and late-stage ALS, respectively). Self-reported data was provided by 22 pALS (10, 9, 3) and 12 cALS (2, 5, 5). Early, middle, and late-stage pALS had a mean 7.1, 10.7, 36.3 HCP consultations, and 0.3, 0.3, 0.6 ALS-related hospitalisations in the previous 12 months. pALS at different stages frequently required mobility aids (44%, 64%, 82%), respiratory aids (0%, 27%, 79%), feeding tubes (2%, 4%, 76%), and professional caregiver support (8%, 18%, 59%; 10.0, 15.6, 63.2 hours/week). Later stage pALS rarely remained in employment (36%, 22%, 0%), and cALS frequently had to change their own working arrangements (0%, 40%, 60%). EQ-5D-5L outcomes were captured for both pALS (0.76, 0.66, -0.05), and cALS (1.00, 0.83, 0.72) at the different stages. CONCLUSIONS: Later-stage ALS appeared to be associated with greater HCRU and impact on pALS and cALS than earlier stages. These findings highlight the potential value of early diagnosis in ALS to prevent disease progression into a more resource-intensive stage.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-euadelphi-als-italy-hcru-poster-v4-0-27-10-23final130570-pdf.pdf?sfvrsn=2ab1f7aa_0","title":"ISPOR EU_Adelphi ALS Italy HCRU Poster v4.0 27.10.23_final130570.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130570","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Burden of Chronic Lymphocytic Leukemia in the United States: A Review of Literature","id":"439f616c-ce3a-49ba-a6e7-ddc47c92d767","sessionCode":"EPH28","topDisplay":"Bisen R, Thakur L, Bisen V, Shivsingwale G SRS Pharmaceuticals Pvt. Ltd., Mumbai, MH, India","locationCode":"3029","description":"\r\n\t<div>OBJECTIVES: In the US, the incidence of chronic lymphocytic leukemia (CLL) has been increased due to improved survival, therefore a comprehensive understanding of CLL burden is needed for future disease management. A targeted literature review was conducted to identify the published evidence on the epidemiology, clinical, humanistic, and the economic burden associated with CLL in the US. METHODS: Literature search from recent years (2017-2022) was performed in EMBASE®, and MEDLINE® databases. Studies published in the English language reporting on epidemiology and burden associated with CLL patients were included. RESULTS: Thirty studies were identified from 2,363 citations, with majority reported economic burden (n=13), followed by epidemiology (n=12), clinical burden (n=8), and humanistic burden (n=1). Based on SEER data, the rate of new cases of CLL was 4.7 per 100,000 per year (from SEER 12), and 5-year relative survival was 87.9% (from SEER 17 2012-2018). Comorbidities had a larger impact on survival in patients aged <65 years compared to ≥65 years, while CLL and CLL-related complications (infections and second cancers) were the increased cause of death in CLL patients. Goyal et al. reported that higher baseline cancer-specific stress (measured by the Impact of Event Scale – Revised) associated with poorer 5-month psychological functioning but not physical in relapsed/refractory CLL patients. CLL imposed a high economic burden associated with inpatient, pharmaceutical, adverse events, and indirect costs. Oral targeted therapies are projected to increase the lifetime cost of CLL treatment from $147,000 (in 2011) to $604,000 (by 2025). CONCLUSIONS: CLL is one of the most frequent types of leukemia, typically occurs in elderly patients. This literature review summarized the available literature and found that increase in incidence and survival in CLL patients and CLL can impose a high economic burden on patients and payers; however, evidence gaps related to the humanistic burden remain.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23thakureph28poster132026-pdf.pdf?sfvrsn=11c208bd_0","title":"ISPOREurope23_Thakur_EPH28_POSTER132026.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132026","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Budget Impact Analysis with and without Performance-Based Managed Entry Agreement (PBMEA) of Different Treatments for Spinal Muscular Atrophy (SMA) in the Kingdom of Saudi Arabia","id":"53ff8222-35b6-48cf-ab40-dddf254b2018","sessionCode":"EE97","topDisplay":"Al Jedai A1, AL-Mudaiheem H1, AlSakran A2, Bashiri F3, Ghamdi F4, AlMuhaizae M1, AlSamman A5, Awad N6, Ojeil R6 1Ministry of Health, Riyadh, Saudi Arabia, 2King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia, 3King Saud University, Riyadh, Saudi Arabia, 4King Fahad Specialist Hospital, Dammam, Saudi Arabia, 5National Neuroscience Institute, Riyadh, Saudi Arabia, 6Carexso Dubai - United Arab Emirate, Dubai, United Arab Emirates","locationCode":"2030","description":"\r\n\t<div>OBJECTIVES: SMA is a genetic neuromuscular disease. Onasemnogene abeparvovec (OA; gene therapy), nusinersen (disease-modifying therapy), and risdiplam (oral drug) are the first-in-class treatment options for SMA. This study aimed to assess the budgetary impact of introducing these treatments as a single therapy, with or without a PBMEA, for SMA types 1, 2, and 3 from the perspective of the Ministry of Health (MOH) of the Kingdom of Saudi Arabia. METHODS: A budget impact model was developed using the available data on various model inputs (patients, treatment options, resources, and cost) collected through an explorative literature review followed by primary market research and validation by expert committees. The model was used to estimate the total cost (drug acquisition, administration, and disease management) for the best supportive care (BSC) with and without these treatments, over a 5-year period. Results were presented as the overall net budget impact of using BSC plus the treatment over BSC alone. RESULTS: For SMA type 1, the overall net budget impact of using OA, nusinersen, or risdiplam was Saudi Riyal (SAR) 39,590,134 (156%), SAR 56,918,293 (225%), or SAR 28,373,040 (112%), without a PBMEA, while, SAR 19,575,411 (77%), SAR 5,946,301 (84%), or SAR 20,392,379 (81%), with a PBMEA. Similarly, for SMA type 2 and 3, it was SAR 12,379,218 (254%; only for type 2 patients <2 years old), SAR 265,013,008 (283%), or SAR 160,408,800 (171%), without a PBMEA, while SAR 5,693,128 (117%), SAR 23,273,924 (76%), or SAR 5,618,215 (36%), with a PBMEA. CONCLUSIONS: This is the first analysis of first-in-class SMA treatments to estimate the budgetary impact with or without PBMEAs. Their introduction will have an impact on the MOH budget with drug acquisition cost being the primary contributor. However, this can be offset by an improvement in clinical management and PBMEAs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23ojeilee97poster129910-pdf.pdf?sfvrsn=2866b897_0","title":"ISPOREurope23_Ojeil_EE97_POSTER129910.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129910","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Introduction to the Duchenne Muscular Dystrophy Burden-of-Illness Study Expanded (US AND SPAIN)","id":"a2776b1d-1350-4f88-ac74-de20c0208c66","sessionCode":"EE92","topDisplay":"Castro D1, Evans J2, Jones C2, Willock R2, Wu Y2, Reuben E3, Hofmeister S4, Vinci I5, Hale J6, Selby V7, Schrader R8, Posner N9, Merla V9, Mahn M9, Beaverson K9, Alvir J9 1Neurology & Neuromuscular Care Center, Denton, TX, USA, 2HCD Economics, Knutsford, UK, 3Duchenne UK, LONDON, LON, UK, 4World Duchenne Organization and Duchenne Data Foundation, Veenendaal, Netherlands, 5Parent Project aps, Roma, Italy, 6Pathfinders Neuromuscular Alliance, Hailsham, UK, 7Great Ormond Street Hospital, London, UK, 8Parent Project Muscular Dystrophy, Washington, DC, USA, 9Pfizer Inc., New York, NY, USA","locationCode":"2025","description":"\r\n\t<div>OBJECTIVES: The Duchenne muscular dystrophy (DMD) burden-of-illness (BOI) expanded study aims to quantify the real-world burden of patients with DMD in the United States of America (USA) and Spain, building on a previous analysis conducted in the UK in 2020. The objectives of this study are to estimate the socioeconomic burden (total cost) and health-related quality of life (HRQoL) impact of DMD. The results will be stratified by severity, categorized by eight progressive disease stages. METHODS: Patients are prospectively recruited by neuromuscular physicians in specialist DMD centers. Eligibility criteria include: male, diagnosed with DMD (via muscle biopsy or genetic testing), at least 12 months before baseline. For patients with cognitive delay or below 16 years, caregivers are asked to complete the study on the patient’s behalf and provide informed consent. Demographic, clinical, and healthcare resource utilisation (HCRU) data are captured by the physician using an electronic case report form (eCRF). HCRU data are captured retrospectively over the last 12 months. HRQoL (EQ-5D, DMDQoL), patient-reported outcomes, and costs are captured by the Patient Public Involvement and Engagement form (PPIE) for patients and their caregivers. Costs are calculated by multiplying resource use by country-specific unit costs. RESULTS: Data collection is currently on-going. Interim data include 83 eCRFs (73 and 11 from the USA and Spain, respectively) with 43 (52%) patient and 35 (42%) caregiver matching PPIEs. The mean age of the sample (N = 83) is 12.1 years, with a mean age-of-diagnosis of 5 years. Patients are distributed over all eight disease stages with 41 (49%) currently receiving corticosteroid treatment. CONCLUSIONS: The DMD BOI expanded study seeks to highlight the unmet need of DMD patients based on a final target sample of 420 patients from the US and Spain. The resultant data may be utilized to support access to new emerging DMD therapies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/dmd-boi-ex-ispor-eu-23-final-v1-1130925-pdf.pdf?sfvrsn=daf34f7d_0","title":"DMD BOI Ex. ISPOR EU 23 Final v1.1130925.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130925","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cardiovascular Medicines on Pharmaceutical Market in Armenia","id":"83969fec-699e-4618-94aa-de3614591e0f","sessionCode":"HSD20","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Cardiovascular diseases are the leading cause of death in Armenia; and access to appropriate medicines is vital for patients. The objective of this work was to study the situation with availability and affordability of cardiovascular medicines on pharmaceutical market in Armenia. METHODS: Lists of medicines registered in Armenia (April, 2023), price-lists of 2 main wholesalers and 2 on-line pharmacies were analyzed. The number of registered generics for 12 tracer medicines was identified for 2023 and 2018. Affordability of 14 essential medicines (2 from each subgroup) was calculated using methodology adjusted from the method developed by World Health Organization (WHO) and Health Action International (cost of treatment is less than money that a worker with the official minimal salary receive for 1 day). RESULTS: Only 24 of cardiovascular medicines and therapeutic equivalents listed in the WHO Model List of Essential Medicines (WHO EML) of 2021 are authorized in Armenia. Some essential medicines / equivalents, such as glyceryl trinitrate, dopamine, isosorbide dinitrate, hydralazine and others, are not registered in the country. Furthermore, some dosage forms and strengths of medicines included in WHO EML are also not in the List of Medicines registered in Armenia. The number of registered generic products for 8 of 12 tracer medicines is decreased from 2018 to 2023. Some of studied essential cardiovascular medicines are affordable; for some medicines only part of generic products are affordable; some medicines are affordable only if prescribed at low doses and unaffordable at higher doses. CONCLUSIONS: Only part of essential cardiovascular medicines and their dosage forms are authorized in Armenia that restricts their import to the country. Low availability and unaffordability of some medicines can compromise access to treatment for patients with cardiovascular diseases. Policy recommendations aimed to increase access to cardiovascular medicines have been developed for submitting to decision-makers.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133916","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Meta-Regression Tool: Assessing the Relationship Between Risk Factors and Events Development","id":"edaa73b8-6d36-4114-83d1-df59bf753425","sessionCode":"SA2","topDisplay":"Li X1, Hoogenveen R2, Gouwens S2, Feenstra T1, Van Giessen A2 1University of Groningen, Groningen, Groningen, Netherlands, 2National Institute for Public Health and the Environment, Bilthoven, Utrecht, Netherlands","locationCode":"7011","description":"\r\n\t<div>OBJECTIVES: The risk of developing an event (e.g. diabetes) is highly dependent on risk factors (e.g. BMI). This relative risk (RR) may be nonlinear and will vary depending on covariates such as age. However, meta-regressions currently presented in literature lack transparency of how the dose-response analysis was conducted. For instance, literature typically presents RRs of diabetes stratified by BMI categories (e.g., normal and overweight, etc.), whereas modellers desire a continuous relation, possibly further influenced by other covariates. Therefore, we developed a meta-regression tool enabling these calculation steps. METHODS: The meta-regression tool was coded in R. A case study examining the effect of BMI on diabetes incidence was conducted. We collected RRs from literature, both per BMI unit and for BMI classes, specified by gender and age. We fitted separate log-linear dose-response curves on the RR values presented for these classes, resulting in parameter estimates of RR values per unit. Then, we fitted a random effects regression model on the log-transformed RR values, with the individual studies as random effects and gender, age, and age-squared as fixed effects. We validated the random effects regressions using the R-package Metafor with the parameters of the individual dose-response curves and age-values as input. RESULTS: We included 25 studies that reported the effect of BMI on diabetes incidence. The parameter estimates of RR values per unit, following a log-linear dose-response curve, depend quadratically on age, with a maximum of 1.2 at age 45. These are the same results as using Metafor. CONCLUSIONS: With validated results, this tool enables a flexible and transparent dose-response meta-regression, featuring the flexibility to include studies with stratified categories without requiring pre-calculations. This is especially important when the relationship between a risk factor and event might be non-linear, like risk factors that may also have a protective effect in certain circumstances.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-poster---meta-regression-tool132118-pdf.pdf?sfvrsn=c1d708d3_0","title":"ISPOR Poster - meta-regression tool132118.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132118","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of Adults Hospitalized with Respiratory Syncytial Virus Infection in Spain, 2016–2019","id":"509d6b86-b5ad-42bc-863a-dffd4a8f9abc","sessionCode":"EE36","topDisplay":"Sato R1, Law A2, Haeberer M3, Seabroke S4, Ramirez Agudelo JL4, Mora L5, Sarabia L4, Meroc E4, Aponte-Torres Z4, López A3 1Pfizer Inc., Collegeville, PA, USA, 2Pfizer Inc., New York, NY, USA, 3Pfizer S.L.U., Madrid, Spain, 4P95 Epidemiology & Pharmacovigilance, Leuven, Belgium, 5P95 Epidemiology & Pharmacovigilance, London, LON, UK","locationCode":"1080","description":"\r\n\t<div>OBJECTIVES: Respiratory syncytial virus (RSV) is increasingly a recognized cause of respiratory infection among adults. This study estimated costs of RSV hospitalization for adults ≥18 years in Spain and descriptively contrasted this to influenza hospitalization costs between 2016–2019. METHODS: A retrospective observational study was conducted using a national hospital discharge database that reports >90% of admissions. RSV and influenza hospitalizations were identified using ICD-10-specific codes. Mean hospitalization costs (€, 2022) were stratified by age and risk groups. Total annual RSV costs were estimated by combining RSV-reported and RSV-attributed hospitalization rates previously reported in the same population and the current hospitalization costs. RESULTS: From 2016–2019, a total of 11,662 adults were admitted to hospital with RSV. The mean hospital length of stay (LoS) ranged from 5-12 days with higher LoS in higher-risk groups, regardless of age. Among adults with risk factors (moderate/high-risk), 2–15% required intensive care unit admission. Mean costs per RSV hospitalization event ranged from €3,435–5,246 for 18–49 years, €3,364–5,112 for 50–59 years, €3,195–5,549 for 60–69 years, €2,942–5,226 for 70–79 years, and €2,928–3,921 for ≥80 years. Costs were higher in the moderate and high-risk groups in each age category. Influenza costs ranged from €2,271–6,145 depending on age and risk. RSV mean costs were higher (22-34%) than influenza in low-risk groups of all ages, and in high-risk groups aged 70-79 years (14%), lower (21%) in high-risk groups aged 50–59 years, and generally comparable (<10%) in the rest. Total annual hospitalization costs for RSV were estimated at M(million) €12.1 with reported incidence and M€194.3 with RSV-attributed incidence vs influenza M€83.2. CONCLUSIONS: In adults, RSV has a significant economic burden to the Spanish National Healthcare System. If corrected for under-ascertainment, RSV likely has an economic burden greater than influenza.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/eu-isporspain-adults-rsv-cost-studyv4131580-pdf.pdf?sfvrsn=a4e1e23f_0","title":"EU ISPOR_Spain adults RSV cost study_v4131580.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131580","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Method Matters: Cost-Minimization Versus Cost-Effectiveness Frameworks in Assessing Opioid Use Disorder Treatments","id":"ffafb5ff-50f3-43ba-b209-e0169dcf1dfd","sessionCode":"EE37","topDisplay":"Rittenhouse B1, Beaulieu E2 1Massachusetts College of Pharmacy & Health Sciences, Winchester, MA, USA, 2Merck & Co., Inc, Rahway, NJ, USA","locationCode":"1084","description":"\r\n\t<div>OBJECTIVES: Optimal opioid use disorder (OUD) treatment is an opioid crisis priority. Novel approaches may prove important. In the US, methadone has traditionally been offered only in clinics (MC); it is not approved for physician offices (MO). MO has been assessed economically only using cost-minimization analysis (CMA). CMA has, however, largely been dismissed as a method. A previously published CMA (showing MC as optimal) provided data to examine the cost-effectiveness of OUD treatments that include this alternative and to compare cost-effectiveness analysis (CEA) and CMA results. METHODS: Costs from the previous CMA of MO, MC, and office-based buprenorphine (BO) and reported (but ignored in the CMA) effectiveness were used in a provider-perspective CEA. We adopted the CMA’s initial provider perspective (analysis A) – and modifications to it. The first modification (B) included costs to the initial providers of “failed” patients up until their failure time (ignored in the original work). The, preferred, second modification (C) incorporated costs subsequent to failure (to another potential provider). These were not reported, so a range per month ($0-200) was used. Decision uncertainty was examined via probabilistic sensitivity analysis (PSA) for the CEA and the CMA, the latter not done in the original work. RESULTS: BO was always dominated. The MO vs. MC incremental cost-effectiveness ratio ranged from $2855 (A) to $5879 (B) per additional patient retained in six-month treatment ($5,284 for C)). CEA decision uncertainty was minimal versus BO but significant for MO and MC. PSA of the CMA (effectiveness not a parameter in the PSA) showed no uncertainty. CONCLUSIONS: Depending on willingness-to-pay for retention, MO may be optimal, in contrast to the previous CMA conclusion, favoring MC (accompanied by our added assessment of zero decision uncertainty). More research can reduce CEA decision uncertainty and inform decision making for the possible future use of MO.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-euope23rittenhouseee37poster-a0132435-pdf.pdf?sfvrsn=c876aab5_0","title":"ISPOR Euope23_Rittenhouse_EE37_POSTER A0132435.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132435","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Public Health and Economic Impact of Replacing the Sequence PCV13→PPV23 with PCV20 in the French Adult Pneumococcal Vaccination Program","id":"df080923-86ce-467e-a249-e0263bf1d4f8","sessionCode":"EE143","topDisplay":"Fiévez S1, Blanc E2, Bougeois M2, Sivignon M3, Supiot R4, Vietri J5, Perdrizet J5, Bellier L6 1Pfizer SAS France, Paris, 75, France, 2Pfizer SAS France, Paris, France, 3Putnam PHMR, Lyon, 69, France, 4Putnam PHMR, Paris, France, 5Pfizer, Inc., Collegeville, PA, USA, 6Putnam PHMR, Paris, 75, France","locationCode":"2066","description":"\r\n\t<div>OBJECTIVES: Pneumococcal infections, including invasive pneumococcal disease (IPD, including meningitis and bacteraemia) and community-acquired pneumonia (CAP), are associated with an important burden in adults. The objective of this study was to evaluate the cost-effectiveness of replacing the sequential administration of 13-valent pneumococcal conjugate vaccine (PCV13) followed by pneumococcal polysaccharide vaccine (PPV23) with a single dose of the 20-valent conjugate vaccine (PCV20) in the French vaccination programme. METHODS: A static model follows a cohort of adults at increased risk of pneumococcal disease, based on the current recommendations, vaccinated in year 1 of the simulation. Observed vaccination uptake was derived from a real-world study (COVARISQ, 2018). Higher uptake was assumed with PCV20 (+40-50 points depending on the age and risk groups) to reflect the impact of an improved prevention and simplified schedule. Epidemiological and cost data for hospitalised cases were sourced from the French National Claims Database (SNDS, 2019). Effectiveness of the vaccines were estimated based on clinical studies of PCV13 and PPV23 and the serotype coverage published by surveillance network. Protection was supposed to wane over time. Other inputs, including quality of life (QoL), were informed by the literature RESULTS: Over a lifetime horizon, the switch from PCV13 à PPV23 to PCV20 and the increased uptake considered, would prevent additional IPD (~4,000 cases) and CAP (~55,000 cases). It would therefore translate into a gain in QoL due to the prevention of premature deaths (~7,000 deaths) and of acute episodes. In total, more than 388M€ would be saved for the management of pneumococcal infections. A strategy proposing PCV20 alone was dominant for the adult population at increased risk for pneumococcal disease overall; ICERs for select age/risk groups ranged from dominant to 5,500€ /QALY. CONCLUSIONS: This study shows that the switch from PCV13→PPV23 to PCV20 is expected to be the dominant strategy from the French collective perspective.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23fievezee143poster132709-pdf.pdf?sfvrsn=3b421be3_0","title":"ISPOREurope23_FIEVEZ_EE143_POSTER132709.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132709","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Shaping the Value Equation in Digital Healthcare: A Holistic Perspective on Patient, System, and Provider Interactions","id":"88c55510-ce5f-4ee6-9aee-e0f806f9c259","sessionCode":"MSR24","topDisplay":"Mühlbacher A, Fischer AK, Jordan Y Hochschule Neubrandenburg, Neubrandenburg, MV, Germany","locationCode":"5069","description":"\r\n\t<div>OBJECTIVES: Decision-making within a patient-centered healthcare system places emphasis on the value of services or products. The definition of success is tailored to encompass multidimensional clinical and non-clinical measures and metrics. However, the value of digital innovations is frequently overlooked. Given the novel nature of digital interventions, new evaluation approaches are necessary to comprehensively capture the entire healthcare process from multiple perspectives in a holistic manner. METHODS: This study utilizes a triangulation approach, incorporating qualitative semi-structured interviews (N=14), literature research on acceptance theory (N=53), and reviews of studies evaluating digital applications (N=86). Through this process, value measures across multiple dimensions were identified. RESULTS: The concept was developed to address the diverse requirements of all stakeholders involved in decision-making. As a result, three levels have been identified: patient level, system level, and provider level. These levels encompass value dimensions, including structural outcome measures, attitudinal outcome measures, behavioral outcome measures, clinical outcome measures, and financial outcome measures. Within these dimensions, a comprehensive catalog of measures and metrics was created to establish a composite value index for digital applications. CONCLUSIONS: This value concept identifies holistic criteria that influence decision-making, allowing stakeholders to support pricing and reimbursement of digital healthcare interventions. This enables the establishment of a patient-centered healthcare system that prioritizes satisfying patients' health-related needs. Political decision-makers, third-party payers, and innovators can utilize this cohesive approach to define value in healthcare decision-making. By applying the value equation as a comprehensive evaluation framework, the aim is to enhance clinical and financial outcomes in digital care.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/231107ispor-europe-2023-value-equation132975-pdf.pdf?sfvrsn=e260d307_0","title":"231107_ISPOR europe 2023 Value Equation132975.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132975","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Loss of Productivity, Use of Healthcare Resources and Cost Associated with Insomnia","id":"0eba1219-815b-4091-ad46-e142835471a3","sessionCode":"EE39","topDisplay":"Bellido MC1, Egea C2, Romero O3, Mur de Víu C4, Espallardo O5, Comellas Serra M6, Aceituno Mata S7 1Hospital General Universitario de Castellón, Castellón, Castellón, Spain, 2Hospital Universitario de Araba, Vitoria-Gasteiz, Spain, Vitoria-Gasteiz, Árava, Spain, 3Hospital Universitario Vall d'Hebron, Barcelona, Cataluña, Spain, 4Servei Andorrà d'Atenció Sanitària, Andorra, Andorra, Andorra, 5Idorsia España, Madrid, Comunidad de Madrid, Spain, 6Outcomes'10, Castellon de la plana , CS, Spain, 7Outcomes'10, Castellón de la Plana, CS, Spain","locationCode":"1082","description":"\r\n\t<div>OBJECTIVES: Insomnia prevalence has increased globally, imposing a great burden on patients’ lives and triggering social and economic problems. We aim to assess productivity losses, use of healthcare resources and costs (direct and indirect) associated with chronic insomnia in Spain. METHODS: Cross-sectional observational study based on an online electronic questionnaire targeted at the general population who meet chronic insomnia criteria (have problems initiating/maintaining/conciliating sleep at least 3 times/week during the last 3 months, unrelated to another medical condition/circumstance). Sociodemographic, productivity and healthcare-resource use data were collected. A descriptive analysis was performed (STATA v.14). RESULTS: A total of 1,504 individuals responded to the questionnaire, of whom 700 (46.5%) meet chronic insomnia criteria (55.4% female; mean (SD) age 44.0 (13.4) years; 75.0% employed/self-employed). Only 4.9% had a diagnosis of chronic insomnia. During last year, 33.0% of workers (n=172) required medical leave; a mean (SD) of 7.7 (32.8) insomnia-related days off work; 12.3% had to change or quit their job/studies due to insomnia. Absenteeism, presenteeism, work impairment and disability due to insomnia according to Work Productivity and Activity Impairment Questionnaire reached 6.4%, 41.1%, 44.1% and 44.2%, respectively. In the past 12 months, 51.9% required public and 23.9% private healthcare services: primary care (40.0%; 13.9%), psychology (9.6%; 6.6%), nurse (5.6%; 2.3%), psychiatry (5.3%; 2.0%), emergency room (6.0%; 1.7%) and sleep specialist (4.3%: 2.3%). To treat insomnia 47.9% used non-pharmacological and 41.6% pharmacological treatment. The mean of the total annual cost of insomnia population that require healthcare resources use (n=363) reached €1719.51/patient [€501.50/patient direct (n=363; 8.54% with insomnia diagnosis), €686.91/patient out-of-pocket (n=335; 7.16% with insomnia diagnosis), €531.10/patient indirect cost (n=172; 9.30% with insomnia diagnosis). CONCLUSIONS: Insomnia is under-diagnosed, and leads to work productivity loss, mainly associated with presenteeism, and out-of-pocket costs. Proper diagnosis and treatment would contribute to reducing the economic impact of insomnia.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23aceitunoee39poster131983-pdf.pdf?sfvrsn=2d6d0c28_0","title":"ISPOREurope23_Aceituno_EE39_POSTER131983.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131983","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Overview of the Challenges in the HTA of Interventions Involving Healthcare Reorganisation","id":"9e7351f9-0ab0-4995-845d-e1a027392904","sessionCode":"HTA47","topDisplay":"Teljeur C1, Walsh K2, O'Donnell H3, Spillane S3, O'Neill M1, Harrington P3, Ryan M4 1Health Information and Quality Authority, Dublin, Ireland, 2Health Information and Quality Authority (HIQA) & School of Pharmacy, University College Cork, Cork, Ireland, 3Health Information and Quality Authority (HIQA), Dublin, Ireland, 4Health Information and Quality Authority & Department of Pharmacology & Therapeutics, Trinity College Dublin, Dublin, Ireland","locationCode":"5025","description":"\r\n\t<div>OBJECTIVES: Most health technology assessment (HTA) is focused on pharmacological treatments and devices, utilising widely accepted methods. Assessments of organisational changes are less common, often requiring different approaches and types of evidence. This study outlines the key challenges to the assessment of healthcare reorganisation interventions. METHODS: Based on over ten years’ experience of conducting HTAs of complex interventions and healthcare reorganisation, we identified consistent challenges faced for these types of analyses. To describe these challenges, we use illustrative examples from completed assessments proposing substantial healthcare reorganisation. RESULTS: Healthcare organisation is generally tailored to a particular jurisdiction and system, with little or no applicable data on the effects of reorganisation. Relevant literature is often limited, creating a reliance on information from stakeholders who may be difficult to identify and may not be impartial. Reorganisation can also impact many clinical and patient groups who can identify barriers to implementation based on experience or anecdotal evidence. Extensive stakeholder engagement is therefore vital to inform these HTAs. Organisational changes can create geographic disparities and inequities for service users that would not typically be considered in the assessment of a treatment or device, but should be addressed. Reorganisation can also shift costs to patients, so the chosen perspective is important. Finally, reorganisation can shift responsibilities and accountability within the system, so consideration needs to be given to potential governance issues. CONCLUSIONS: HTAs of healthcare reorganisation can be challenging to perform and require a mix of approaches that don’t neatly fit into the classic HTA methods. Flexibility is key to ensure the decision maker’s needs are adequately addressed for the safe reorganisation of services. It is critical to involve key stakeholders directly in the process because the available literature will often not give assessors the necessary understanding of the main issues.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/cteljeurhta47133859-pdf.pdf?sfvrsn=fbf1f4d3_0","title":"CTeljeur_HTA47133859.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133859","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Exploring the Pandemic's Impact: An Examination of Adolescent and Adult Vaccination Rates in Different Countries During COVID-19","id":"6a3bc429-300f-45c2-98a1-e1b074e14402","sessionCode":"EPH45","topDisplay":"Masseria C1, Guyenet A2, Mody L3, Roeder C2, Schliep V3 1AESARA Inc., NEW YORK, NY, USA, 2AESARA Europe GmbH, Zug, ZG, Switzerland, 3AESARA Inc., Chapel Hill, NC, USA","locationCode":"3046","description":"\r\n\t<div>OBJECTIVES: The COVID-19 pandemic highlighted the importance of national immunization strategies. During COVID-19, there was increased focus on the importance of vaccination and challenges of vaccine hesitancy. Vaccine hesitancy is complicated by vaccine fatigue, stemming from the multiple doses of COVID-19 vaccine required. What is less understood is the impact of this increased focus on vaccination, and its relation to vaccine hesitancy/fatigue on other immunizations. The objectives of this study were to assess trends of influenza, Tdap, Shingrix, and HPV vaccination rates before, during, and after the pandemic, and to identify relationships between vaccination fatigue/hesitancy for COVID-19 and trends in other immunizations. METHODS: We reviewed vaccination guidelines and available data for COVID-19, shingles, influenza, pertussis, and human papilloma virus (HPV) among 12+ age groups across the United States (US), England, Germany, Italy, France, Germany, and Australia. Based on data accessibility, we narrowed country list to US, England, Italy, Germany, and Australia, and then compared guideline-compliant country-level rates of vaccination from pre-pandemic (2018-2020) to during the pandemic and post-pandemic (2020-2023), and assessed for noticeable trends. RESULTS: For influenza, data show an increasing trend, particularly during the 2020/2021 season, particularly among the elderly, and then a decrease in the 2021/2022 season with uptake on par with the 2019/2020 season. For other age groups the picture is less delineated. Differences across countries remained noticeable. There was a lack of identifiable trends across Shingrix, Tdap, and HPV vaccination pre-pandemic and post-pandemic, due to variations in available data, and an overall lack of available data in key national immunization strategies. CONCLUSIONS: Our study demonstrated that opportunities remain to assess the pandemic’s impact on vaccine willingness. There is a clear lack of data to monitor vaccination trends for important immunization strategies like HPV and Shingrix. We encourage countries to collect and share this data.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23masseriaeph45poster133446-pdf.pdf?sfvrsn=5e650cb4_0","title":"ISPOREurope23_Masseria_EPH45_poster133446.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133446","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"5b196821-963c-48d3-9e67-cc2da312735d","parentId":"00000000-0000-0000-0000-000000000000","title":"Vaccines","urlName":"Vaccines"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Correlation Between the Costs and Clinical Benefits of National Price-Negotiated Anticancer Drugs for Specific Cancers in China","id":"41ea8c2a-019f-45a0-b5dc-e1fd1facd49b","sessionCode":"HPR21","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: New anticancer drugs have become synonymous with dramatically high costs, leading to an eminent apprehension of healthcare stakeholders. Whether the survival benefit is in proportion to the economic expenditure is ambiguous. Thus, our study aimed to assess the correlation between the price and value of innovative drugs in specific tumors, considering negotiation policy implementation or not. METHODS: We identified new drugs that were negotiated in National Reimbursement Drug List (NRDL) from 2016 to 2022 for lung cancer (LC) and breast cancer (BC). Therapeutic value was combined the improvement percentage of OS and PFS (ΔOS%, ΔPFS%) with the quantified gain of the American Society of Clinical Oncology Value Framework (ASCO-VF) and the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). We calculated monthly drug costs and employed Spearman’s correlation coefficient for analysis. The consistency test was executed by Cohen’s kappa statistics. RESULTS: Up to 2022, a total of 26 innovative price-negotiated drugs were collected. The median monthly costs were US$3865.33 out of NRDL and US$1376.28 within NRDL. The ΔOS% and ΔPFS% of these drugs were 36.10% (IQR 18.78%-48.22%) and 84.22% (IQR 48.49%-100.79%), respectively. The median ASCO-VF score was 31.65, as well as 9 drugs, scored the meaningful benefit of ESMO-MCBS. No association was found between clinical benefits with their costs before NRDL, whereas the value of drugs measured by two frameworks positively correlated with therapeutic expenditure after NRDL admissions. An agreement between the two frameworks was stable. CONCLUSIONS: The negotiation policy has diminished medication costs and seemed to exhibit a notable correlation among specific cancers. Comprehensive value assessments need to be performed in future to support our findings and promote the affordability and availability of effective anticancer drugs.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128224","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Using Sensor-Based Glucose Monitors Dominates Self-Monitoring of Blood Glucose for Adults with Type 1 Diabetes in the Danish Healthcare Setting","id":"bacb0ef3-d4db-4daa-aa2b-e230aed21e67","sessionCode":"EE13","topDisplay":"Sørensen A The Danish Health Technology Council, Aalborg, North Denmark, Denmark","locationCode":"1066","description":"\r\n\t<div>OBJECTIVES: As a part of the Danish Health Technology Council’s (DHTC) 2022 health technology assessment concerning glucose monitoring for the treatment of adults with type 1 diabetes an economic evaluation was conducted to evaluate the cost-effectiveness of sensor-based glucose monitors with alarm (‘sensors’) compared with self-monitoring of blood glucose (SMBG). METHODS: A cost-utility analysis was conducted using a Markov model including 17 health states to reflect the disease progression for a patient cohort with a starting age of 27 and a 6-year history of type 1 diabetes. A lifetime horizon, a one-year cycle-length, and a Danish healthcare sector perspective were applied. Costs were expressed in EUR 2022 (DKK7.46 per EUR), and effectiveness was expressed as quality-adjusted life-years (QALYs). Utility estimates and associated uncertainty were based on EQ-5D values found in the literature. Probabilities and associated uncertainties were likewise retrieved from the literature and confirmed by clinical experts. Costs were primarily based on Danish estimates of resource consumption and prices were provided by distributors of sensors included in the procurement contract with the Danish regions. Cost and outcomes were discounted at 3.5% per year. One-way sensitivity and probabilistic sensitivity analyses were performed to evaluate the robustness of the results. RESULTS: Over a lifetime horizon, the use of sensors resulted in lower costs (-EUR4,740) and higher effect (1.67QALYs) compared with SMBG. Based on the included uncertainty, there was a 77 % probability of sensors dominating SMBG. Main drivers of costs were the costs associated with sensors and the consumption pattern associated with SMBG. The result was sensitive to the costs of sensors, the association between HbA1c and diabetic late complications, and the time horizon. CONCLUSIONS: The analysis indicates that sensors dominate SMBG in the Danish healthcare setting. The analysis supported DHTC’s positive recommendation of the use of sensors for adults with type 1 diabetes in Denmark.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130814","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Wayfind-R: A Global, Real-World Precision Oncology Registry – Socioeconomic Features","id":"4db6eb52-fc46-4ce1-8834-e28a4b24aecf","sessionCode":"RWD12","topDisplay":"Hackshaw A1, Dienstmann R2, Blay JY3, Le Tourneau C4, Geissler J5, Ferro A6, Schirghuber E7, Skatkova O7 1UCL Cancer Institute, London, UK, 2Oncoclínicas Group, São Paulo, Brazil, 3Centre Léon Bérard and Université Claude Bernard, Lyon, France, 4Institut Curie, Paris, France, 5Patvocates GmbH, Riemerling, Germany, 6Roche Products Limited, Welwyn Garden City, UK, 7F. Hoffmann-La Roche Ltd, Basel, Switzerland","locationCode":"6070","description":"\r\n\t<div>OBJECTIVES: WAYFIND-R (NCT04529122), a global, prospective pan-cancer precision oncology registry of patients diagnosed with a malignant solid tumor profiled with next-generation sequencing (NGS), collects long-term, high-quality real-world data on those patients’ characteristics and outcomes, as well as socioeconomic factors. It aims to be a fit-for-purpose data source for researchers internationally. METHODS: Patient data are standardized and collected longitudinally into a centralized database. These include comorbidities, tumor characteristics, biomarker, NGS and molecular tumor board decision-making, cancer treatment and outcomes. Here we describe patients' sociodemographic and socioeconomic characteristics. RESULTS: As of 30 April 2023, 976 patients from 64 sites, mostly academic institutions/university hospitals across 24 countries (Europe: 527 patients; South America: 150; Middle East: 176; Asia: 112; Africa and North America: 11), had complete baseline and cancer diagnosis information. Median age was 64 years (range: 20–93) and 51% of patients were female. Race/ethnicity was available for 675 patients (White: 73%; Asian: 17%; Mixed or other [mostly Hispanic]: <10%; Black: <10%). Overall, 116 cancer types were included and 82% of patients had metastatic disease when enrolled. The most common cancer types were lung (n=289), colon (n=105), pancreas (n=90), breast (n=69) and ovary (n=34). Sixty-six per cent of all patients had their treatment reimbursed. Out-of-pocket payment varied across regions (Europe: <10%; South America: <10%; Middle East: 21%; Asia: 51%), as did educational level (tertiary education: 24%, 53%, 38% and 39%, respectively) and government insurance (84%, 19%, 55% and 79%, respectively). CONCLUSIONS: The WAYFIND-R database provides insights into some social determinants of health that may influence patient care pathways, from which further epidemiological and health economic studies are encouraged.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/hackshawwayfind-rispor-eu-2023v30oct2023poster130073-pdf.pdf?sfvrsn=caf0fd54_0","title":"Hackshaw_WAYFIND-R_ISPOR EU 2023_v30Oct2023_poster130073.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/hackshawwayfind-rispor-eu-2023v30oct2023supplement130073-pdf.pdf?sfvrsn=d08a5d8b_0","title":"Hackshaw_WAYFIND-R_ISPOR EU 2023_v30Oct2023_supplement130073.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130073","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Decade of Alzheimer's Disease Management in Germany: The Increasing Impact of Specialists","id":"4a519b34-9ab4-4151-9518-e2dc174b8908","sessionCode":"RWD119","topDisplay":"Wartmann H1, Effenberger T2, Volmer T2 1SmartStep Data Institute, Hamburg, HH, Germany, 2SmartStep Data Institute, Hamburg, Germany","locationCode":"6065","description":"\r\n\t<div>OBJECTIVES: Alzheimer's Disease (AD) is growing in prevalence in Germany, posing substantial social, economic, and healthcare challenges to patients, caregivers, and the health system. General practitioners (GPs), often being the first point of contact, play a critical role in the early detection and management of AD. However, the complexity of AD symptoms often necessitates specialized knowledge and resources, typically provided by specialists such as neurologists or psychiatrists. METHODS: Using German claims data from 6.1 million individuals (2011-2022), a retrospective study analyzed patient contacts and AD-relevant prescriptions, stratified by type of physician (GP vs. specialist). The main endpoint was the occurrence of first hospitalization with a claims code for care level, indicative of potential disease progression. This was assessed using Cox regression. In Germany, care level, indicating patients' daily care needs and dictating reimbursement rates, are part of claims data. RESULTS: Of 42,681 identified AD patients, initial diagnoses by specialists increased from 54.3% (2011-2015) to 59.6% (2016-2022), as did AD-specific prescriptions (60.3% to 66.95%). While 70.2% visited both GPs and specialists, 23.1% saw no specialists. Patients mainly visiting GPs had a 1.29 hazard ratio for hospitalization with the co-occurrence of a care level compared to patients mainly visiting with specialist. CONCLUSIONS: These preliminary results indicate an increasing role for specialists in AD diagnoses and treatments in Germany from 2011-2022 and emphasize their importance in disease management. Notably, AD patients primarily seeing GPs had a higher risk of hospitalization with the co-occurrence of a care level. Effective collaboration between GPs and specialists is essential for optimal AD care.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23wartmannrwd119poster131992-pdf.pdf?sfvrsn=c93785b0_0","title":"ISPOREurope23_Wartmann_RWD119_POSTER131992.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131992","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Benefit of Ataluren Treatment in Milestones Not Related to Ambulatory Function in Nonsense Duchenne Muscular Dystrophy Versus Standard of Care","id":"47ee48b3-0b0c-4462-bb72-e36822d5d3d6","sessionCode":"CO29","topDisplay":"Machado D1, Paula E2, Bretas IDL3, Freitas ALM3, dos Santos APC4 1UNICAMP, Campinas, SP, Brazil, 2PTC Farmacêutica do Brasil Ltda., São Paulo, SP, Brazil, 3PTC Farmacêutica do Brasil Ltda., São Paulo, Brazil, 4PTC Farmcêutica, São Paulo, São Paulo, Brazil","locationCode":"1029","description":"\r\n\t<div>OBJECTIVES: To present the most recent data on real-world ataluren benefits on upper limb and pulmonary function, compared to standard of care (SoC). METHODS: A search at EMBASE and Medline was conducted using the terms: [ataluren + pulmonary or respiratory function]; [ataluren + upper limb]. This search brought 3 relevant references (McDonald, 2022; Michael, 2021 and Tulinius, 2022). The same search was performed on the abstracts of the 27th Annual Congress of the World Muscle Society (WMS), October 2022. This search brought 1 additional relevant reference (McDonald, LSVP #26). RESULTS: McDonald, 2022, an ataluren phase 3 clinical trial, showed that ataluren delayed by 3 years the predicted FVC < 60% compared with matched controls. Michael, 2021, a Swedish experience on ataluren treatment in long term, showed that even after loss of ambulation, PUL values continued to be above the expected over time in the majority of ataluren treated patients. And Tulinius, 2022, compared data from STRIDE, an ongoing observational registry study, with those from the DMD natural history study (CINRG): patients treated with SoC only. It was observed that Ataluren delayed the age at predicted FVC < 60% by 1.8 year. The additional abstract from the WMS congress compared data from STRIDE, as well with those from the DMD natural history study and showed that patients on Ataluren preserved the hand-to-mouth function by 3.4 years. CONCLUSIONS: These data suggest ataluren + SoC slow disease progression in milestones other than ambulatory function, such as pulmonary and upper limb function.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23machadoc029poster133795-pdf.pdf?sfvrsn=fff5a9c6_0","title":"ISPOREurope23_Machado_C029_POSTER133795.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133795","diseases":[{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Comparing UK Value Sets and Mapping Functions for EQ-5D","id":"911b1c6d-45bb-43c8-8343-e42f6b550ae1","sessionCode":"PT2","topDisplay":"Maervoet J1, Poirrier JE2, Rudell K3, Bergemann R4 1Parexel International, Wavre, Belgium, 2Parexel International, Wavre, WBR, Belgium, 3Parexel International, LONDON, LON, UK, 4Parexel International, Loerrach, Germany","locationCode":"2A","description":"\r\n\t<div>OBJECTIVES: The EQ-5D is preferred by NICE to measure health-related quality of life. Versions with three (EQ-5D-3L) and five (EQ-5D-5L) response levels exist. Due to concerns about its methodology, quality and reliability, NICE does not recommend using the EQ-5D-5L value set for England published in 2018. To derive utility values from EQ-5D-5L responses, 5L data should rather be mapped onto the 3L value set. NICE previously recommended using the Van Hout crosswalk, but its 2022 Manual now states that the EEPRU mapping function developed by the Decision Support Unit (DSU) should be used. Our aim was to compare these different value sets and mapping methods. METHODS: For each of the 3125 possible health states in the 5L system, utility values were obtained using the 2018 5L value set for England, the Van Hout crosswalk, and the DSU mapping function. In addition, utilities for the 243 possible EQ-5D-3L health states were calculated using the 3L value set for the UK, allowing comparison between corresponding 3L and 5L states. Density histograms and plots comparing utility values in comparable states were generated to visualize differences. RESULTS: Utility values obtained with the 5L value set were generally higher than 3L value set/crosswalk estimates. Proportions of health states worse than death (utilities below zero) were 5.1% with the 5L value set, around 22% for the DSU mapping function (age and sex-dependent), 26.7% for the Van Hout crosswalk, and 34.6% for the 3L value set. Whilst the Van Hout crosswalk produces identical values for the 243 3L health states as the 3L value set, the DSU mapping function does not. Its utility values are slightly lower in the best health states, and generally higher in moderate and worst health states. CONCLUSIONS: Utility values obtained with different UK value sets and mapping functions vary, potentially leading to differences in health-economic outcomes.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23maervoetpt2poster132034-pdf.pdf?sfvrsn=a1acde35_0","title":"ISPOREurope23_Maervoet_PT2_POSTER132034.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132034","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Institutional and Geographical Variation in Discharge Destination Among Admission Avoidance Municipal Inpatient Acute Care Units in Norway","id":"4677b37e-ffd3-4cae-bb7f-e45012740872","sessionCode":"HSD4","topDisplay":"Yang F, Burrell LV, Sogstad MKR, Skinner MS Norwegian University of Science and Technology, Gjøvik, Norway","locationCode":"4014","description":"\r\n\t<div>OBJECTIVES: The Municipal Inpatient Acute Care (MIPAC) units were established as part of the Coordination Reform in 2012 in Norway. It was intended to reduce the burden for acute hospital admissions by providing timely inpatient acute health care services closer to patients’ home in the municipalities. However, patients were found transferred to hospital after admitted in MIPAC. The aim of the study was to assess MIPAC patients’ discharge destination and to examine facility-level and geographical variation in further upward hospital referral. METHODS: This study was based on registered data from the Norwegian directorate of Health in 2019. Multinomial logistic regression was performed to measure the institutional and geographic differences on the likelihood of patients being discharged to different destinations. RESULTS: In total, 201 MIPAC units consisting of 400 municipalities were studied. Around 15% of the total discharged patients were further transferred to hospital, 16% went to nursing home and 67% were discharged back home. MIPAC units in the intermunicipal cooperation were more likely to transfer patients to hospital comparing to discharge home(RRR 1.505 [1.380-1.638]). Larger scale of MIPAC units(have more than 2 beds) were also more likely to discharge patients to hospital (2-5 beds: RRR 1.326 [1.170-1.503]; more than 5 beds: RRR 1.217 [1.010-1.470]). Geographic health regions of the country were also significantly associated with discharge disposition. Comparing with South-east health region, MIPAC units in the west and north were more likely to further upward transfer, while the central health region MIPAC units were less likely. CONCLUSIONS: Our findings indicate the geographical and institutional variation exists in the MIPAC service delivery. It also highlights the need for further research to understand regional differences in MIPAC triage decision-making and quality improvement. <quillbot-extension-portal></quillbot-extension-portal></div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope2023yanghsd4poster133842-pdf.pdf?sfvrsn=b7a19306_0","title":"ISPOREurope2023_Yang_HSD4_POSTER133842.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133842","diseases":[{"id":"dc752772-538c-4933-b6a5-3b5b6d079673","parentId":"00000000-0000-0000-0000-000000000000","title":"Geriatrics","urlName":"Geriatrics"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Systematic Review of Bariatric Surgery and GLP-1 Agonist Pharmacotherapy on Weight Loss Outcomes and Type 2 Diabetes Control","id":"c99d6425-1db6-4393-bb4d-e507feb597ae","sessionCode":"CO3","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1005","description":"\r\n\t<div>OBJECTIVES: The study aims to conduct a systematic review to assess and describe the current literature on weight loss and Type 2 Diabetes Mellitus (T2DM) outcomes given bariatric surgery (BS) and/or GLP-1 agonist (GA) pharmacotherapy alone or in combination with other interventions. METHODS: We conducted a search using PubMed for relevant studies published between January 2017 and May 2023. Keywords included obesity, bariatric surgery, drug therapy, weight loss, diabetes mellitus, and generic GLP-1 drug names. Key inclusion criteria included clinical trials starting in or after 2013, obesity with or without T2DM as the disease of interest, human adults 18+, weight loss and/or T2DM HbA1c control or remission. RESULTS: Of the 1035 studies identified, 72 met the inclusion criteria. 3 studies included both BS and GA. 35 studies had BS without GA, with 6 months to 5 years of follow-up. 34 studies had GA without BS, with 5 weeks to 2 years of follow-up. 15 studies excluded T2DM. The follow-up time for BS refers to the post-surgery period, while all but one of GA were administered throughout the entire follow-up period. Both interventions demonstrated significant weight loss and HbA1c control. 8 BS trials included T2DM remission as an outcome, with significant results. No GA trials examined T2DM remission. One study found BS and GA combined had greater excess weight loss and diabetes control compared to BS alone after 6 months. CONCLUSIONS: Compared to BS, current studies using GA to treat obesity generally have shorter follow-up times due to their novelty. Adherence, which is not applicable to BS, is a consideration with GA. Studies examining weight loss maintenance and T2DM control following discontinuation of GA medications are sparse compared to BS, especially for longer periods of follow-up. GA could potentially enhance the weight loss and T2DM control in combination with BS, but more evidence is needed.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130676","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Transferable Exclusivity Vouchers: A Silver Bullet?","id":"f39f3872-a5d9-4582-9743-e6670de740b8","sessionCode":"HPR4","topDisplay":"Herman K1, Tchoukouaha T2 1Cogentia Healthcare Consulting, Cambridge, CAM, UK, 2Cogentia Healthcare Consulting, Cambridge, Cambridgeshire, UK","locationCode":"3061","description":"\r\n\t<div>OBJECTIVES: The European Union (EU) has proposed a new Regulation to replace Regulation 2004/726, introducing the concept of a transferable data exclusivity voucher (TEV) as an incentive for the development of priority antimicrobials. Antimicrobial resistance is a significant issue in the EU, leading to deaths and substantial healthcare costs, but pharmaceutical companies have been hesitant to invest in antimicrobial development due to limited market prospects. METHODS: The EU aims to encourage the development of priority antimicrobials by offering a transferable data exclusivity voucher. We seek to summarise the pros and cons of this approach and draw conclusions on whether it is likely to be successful. RESULTS: TEVs may well incentivise innovation in this field. However, they also may lead to higher drug prices. Furthermore, they do not guarantee access to antimicrobials, and they may draw focus away from preventative measures, harming ongoing antimicrobial stewardship outcomes. CONCLUSIONS: The transferable data exclusivity voucher is intended to incentivize the development of priority antimicrobials and address antimicrobial resistance. Its forthcoming implementation has generated mixed opinions. Critics argue that it may indirectly burden member states with higher drug prices and exacerbate the limited access to innovative drugs in some markets However, the research-based pharmaceutical industry in Europe has generally supported the concept. The voucher has the potential to generate significant revenues for sellers, and it could serve as an initial step toward combating antimicrobial resistance in the future.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hermanhpr4poster133479-pdf.pdf?sfvrsn=5f3604d2_0","title":"ISPOREurope23_HERMAN_HPR4_POSTER133479.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133479","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Utilisation and Expenditure on Blood Glucose Test Strips in Ireland—The Impact of Health Technology Management in Reducing and Containing Expenditure","id":"17b926ad-6ba8-4e50-bead-e67b9f054582","sessionCode":"HPR24","topDisplay":"Doran S1, Clarke S1, Smith A2, Barry M1, Gorry C1 1HSE Medicines Management Programme, Dublin, Dublin, Ireland, 2HSE Medicines Management Programme, Dublin 8, Ireland","locationCode":"3056","description":"\r\n\t<div>OBJECTIVES: The management of type I and type II diabetes mellitus (DM) is assisted by blood glucose monitoring utilising blood glucose test strips (BGTS). These technologies are associated with significant expenditure. This study aims to quantify the impact of two health technology management (HTM) strategies implemented by the Health Service Executive-Medicines Management Programme (HSE-MMP), to manage utilisation and reduce expenditure on BGTS in the Irish publically funded health system. METHODS: Two HTM strategies were implemented to control expenditure on BGTS. Firstly, based on literature review and in conjunction with the National Clinical Programme for Diabetes, a series of automated validations were introduced to the national reimbursement claims software to limit the monthly and annual quantities of BGTS reimbursed for patients, with a separate mechanism to support individuals with additional BGTS requirements. Secondly, a HSE-MMP ‘Preferred BGTS’ initiative was implemented, where following a review of the clinical and commercial aspects of the marketed BGTS, a preferred list of BGTS was identified. Savings gained through these two initiatives were analysed using data extracted from the HSE-Primary Care Reimbursement Services (HSE-PCRS) national pharmacy claims databases. Data was compiled and analysed in Microsoft Excel™. RESULTS: Total expenditure on BGTS on the community drug schemes in 2015 was €48.5 million. In April 2016, the automated validations were implemented based on patients’ pharmacological management. This resulted in an immediate reduction in expenditure to €41.7 million in 2017; ongoing expenditure remained at this level (€40.2 million in 2020). The ‘preferred BGTS’ list was published in February 2021. This initiative resulted in a reduction in expenditure to €27.9 million in 2022. CONCLUSIONS: The implementation of HTM strategies by the HSE-MMP in the area of medical devices has demonstrated reductions in expenditure on BGTS. Similar strategies could be successfully applied to optimise utilisation of continuous glucose monitoring systems.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23doranhpr24poster132796-pdf.pdf?sfvrsn=a9e7dadd_0","title":"ISPOREurope23_Doran_HPR24_POSTER132796.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132796","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Hybrid Closed Loops – A Cost-Effective Future for Type 1 Diabetes Standard of Care? An Update to the TA10845 Economic SLR","id":"774200b3-922a-429c-bca4-e70059d1bf00","sessionCode":"EE22","topDisplay":"Brown L1, Jindal S2, Smith M1 1Lumanity, London, UK, 2Lumanity, Gurugram, India","locationCode":"1059","description":"\r\n\t<div>OBJECTIVES: Hybrid closed loops (HCLs) are an up-and-coming management system for patients with Type 1 diabetes in the UK. An insulin pump and continuous glucose monitor are connected using a mathematical algorithm to automate more precise and timely deliveries of insulin, therefore substantially alleviating burden for these patients. In 2022, the EAG for the NICE TA10845 appraisal conducted an SLR to review the existing cost-effectiveness evidence surrounding HCLs. We aimed to update the SLR to assess whether newer studies continue to support evidence of cost-effectiveness. METHODS: Using the same methodology published in NICE TA10845, the economic SLR was updated. The databases searched included MEDLINE®, Embase® and HTA websites (search period April 2022–June 2023). Screening was conducted by two independent reviewers. Outcomes of interest were extracted, such as model structure, cost/utilities information, model results and ICERs. RESULTS: The results highlighted studies from countries including Wales, the US, Australia and Greece. The majority continued to show that HCL systems are a cost-effective option compared with current standard of care, with the limitation that this outcome is based on the use of different WTP thresholds. Unlike the original SLR, a study by Health Technology Wales found that HCLs were not a cost-effective treatment option. The models were shown to be sensitive to multiple variables, including the patient’s baseline HbA1c value, the definition of severe hypoglycaemic events, and treatment costs. These studies used a range of models, including the iQVIA CDM and Sheffield T1DM model. CONCLUSIONS: Evidence to date supports the cost-effectiveness of HCLs for Type 1 diabetics across a wide range of geographies, while showing an improvement in the quality of life for these patients. Taking this evidence into account, further uptake of this technology may prove to be a beneficial choice for both the payer and the patient.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23brownee22poster133257-pdf.pdf?sfvrsn=133168b7_0","title":"ISPOREurope23_Brown_EE22_POSTER133257.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133257","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Analysis of Long-Term Survivorship (LTS) Rates for Stage 2B/2C Melanoma Using Published Real-World Data (RWD) and Data From Randomized Controlled Trials (RCTS)","id":"9e2be171-6074-476b-a5a8-e72c3e6a5743","sessionCode":"MSR12","topDisplay":"Alagoz O1, Winge-Main A2, Srinivasan S3, Dyer M4, Wolf R5, Thybo SH5, May JR4, Moshyk A3, Kurt M3 1University of Wisconsin-Madison, Madison, WI, USA, 2Oslo University Hospital, Oslo, Not applicable, Norway, 3Bristol Myers Squibb, Lawrenceville, NJ, USA, 4Bristol Myers Squibb, Uxbridge, UK, 5Bristol Myers Squibb, Sorgenfri, Capital Region, Denmark","locationCode":"5050","description":"\r\n\t<div>OBJECTIVES: Despite the curability of Stage IIB/IIC melanoma with resection, recurrence rates of the disease remain high but heterogeneous due to plateaus in the recurrence free survival (RFS) examined by several RCTs and RWD sources. This study explored the LTS rates among Stage IIB/IIC melanoma patients via mixture cure models (MCMs) using published RCTs and RWD sources. METHODS: MCMs were employed separately to digitized RFS data from 3 real-world cohort studies (2 from the US [n1 = 90, n2 = 567], 1 from Norway [n=2317]), data pooled across 10 RCTs (n=3927) identified by a targeted literature review, and data pooled across the surgery alone arms of a restricted subset of these RCTs published after year 2000 (n = 1635). In the MCMs, patients were classified in two latent subgroups as cured (long-term survivors) and uncured. Cured subgroup was subject to only non-disease-related mortality whereas the uncured subgroup was subject to both recurrence and all-cause mortality. For each cohort, survival of the cured was estimated using corresponding baseline demographic information, and age- and sex-adjusted background mortality rates reported by WHO. Time-to-event outcomes for the uncured were modeled by standard parametric distributions. Model selection was guided by statistical and visual fit to the reported data. RESULTS: Lognormal MCMs provided the best fit for all data sets with relatively more conservative LTS-rates than other candidate models. The estimated LTS rates (95% CI) were 0.34 (0.14-0.61) and 0.31 (0.21-0.44) for the US RWD sources; 0.56 (0.52-0.60) for the Norwegian RWD; and 0.46 (0.43-0.50) and 0.52 (0.48-0.56) for the pooled RCT data and its restricted subset, respectively. Estimated LTS rates varied <3% between the top-2 fitting MCMs across all data sets. CONCLUSIONS: The variability in estimated LTS-rates across different cohorts and treatment settings highlights a high unmet need for novel systemic therapies in postoperative treatment of Stage IIB/IIC melanoma.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/mcmmelanomaispor-eufinalmksg132865-pdf.pdf?sfvrsn=f9100c73_0","title":"MCM_melanoma_ISPOR-EU_final_MK_SG132865.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132865","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Evolution of Disease-Specific Quality of Life with Esketamine Nasal Spray Versus Quetiapine Extended Release in Treatment Resistant Depression: Results from the ESCAPE-TRD Phase IIIb Trial","id":"2570bda0-1cee-4904-b4a2-e7424a9cb4bd","sessionCode":"CO8","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"","description":"\r\n\t<div>OBJECTIVES: In ESCAPE-TRD, esketamine nasal spray (ESK-NS) significantly increased the probability of achieving remission at Week 8, and of being relapse‑free through Week 32 after remission at Week 8, versus quetiapine extended release (QTP-XR), in patients with treatment resistant depression (TRD). Here, we assess changes in health-related quality of life (HRQoL), as measured by the QoL in Depression Scale (QLDS). METHODS: ESCAPE‑TRD (NCT04338321) was a randomized phase IIIb trial comparing ESK-NS and QTP-XR, both alongside an ongoing selective serotonin reuptake inhibitor/serotonin norepinephrine reuptake inhibitor, in patients with TRD. Change from baseline (CfB) in QLDS was analyzed using mixed model for repeated measures. Time to clinically meaningful improvement on the initiated treatment (defined as CfB ≤–8) was analyzed using Kaplan‑Meier and Cox regression estimating hazard ratios (HR) and 95% confidence intervals (CIs). Analyses included all randomized patients, using observed data without imputation from on‑treatment visits and retrieved dropouts after treatment discontinuation (incorporating potential benefit of subsequent treatments). P values reported are not adjusted for multiple testing. RESULTS: Patients were randomized to ESK-NS (N=336) or QTP-XR (340). At Week 8, a greater CfB in QLDS was observed for ESK-NS (–11.293) versus QTP-XR (–8.337), with a –2.956 difference (95% CI: –4.346, –1.565; p<0.001). At Week 32, CfB was still greater for ESK-NS (–14.743) relative to QTP-XR (–12.435), corresponding to a −2.308 difference (95% CI: –3.827, –0.789; p=0.003). Most patients reported a clinically meaningful improvement (ESK-NS: 81.5%, QTP‑XR: 66.8%), with a shorter time to meaningful improvement with ESK-NS versus QTP‑XR (median 1.018 versus 1.906 months; HR: 1.417 [95% CI: 1.187, 1.691; p<0.001]). CONCLUSIONS: ESK-NS patients experienced significantly greater improvements in HRQoL versus QTP‑XR, plus had greater likelihood and speed of meaningful improvement over 32 weeks as measured by the QLDS. These results support the superior short- and long‑term clinical efficacy previously demonstrated for ESK-NS in ESCAPE-TRD.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129393","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Healthcare Resource Utilization (HCRU) and Associated Costs Among Patients with Acute Myeloid Leukemia (AML) Treated with Oral Azacitidine as Maintenance and Those Eligible but Not Treated Using a US Claims Database","id":"e320707b-f6d7-4543-9b01-e817fbd7631f","sessionCode":"EE86","topDisplay":"Borate U1, Seiter K2, Potluri R3, Mazumder D3, Heydendael W4, Chevli M4, Prebet T4, Strocchia M4, Vasconcelos A4, Sieluk J5 1Ohio State University, Columbus, OH, USA, 2New York Medical College, Valhalla, NY, USA, 3Putnam Associates, New York, NY, USA, 4Bristol Myers Squibb, Princeton, NJ, USA, 5Bristol Myers Squibb, Lawrenceville, NJ, USA","locationCode":"2016","description":"\r\n\t<div>OBJECTIVES: Patients with AML in remission following intensive chemotherapy who are not candidates for hematopoietic stem cell transplantation are eligible for oral azacitidine (Oral-AZA) as maintenance treatment. While the QUAZAR AML-001 trial (NCT01757535) demonstrated that Oral-AZA was associated with longer survival outcomes than placebo in this patient population, HCRU and costs associated with Oral-AZA treatment versus no maintenance treatment have not been studied in the real world. Therefore, we assessed HCRU and associated costs of these patient groups in the USA. METHODS: Using the IQVIA PharMetrics® Plus claims database, we identified newly diagnosed adult patients with AML who had a maintenance eligibility date for Oral-AZA (MaintDate) on or after September 1, 2020, and were treated with Oral-AZA or did not receive maintenance treatment (NoMaint). Patients were 1:3 propensity score matched on demographic characteristics. We assessed HCRU and costs on a per-patient per-month (PPPM) basis from MaintDate to end of follow-up using generalized linear models. RESULTS: 43 Oral-AZA and 129 NoMaint patients were identified (mean follow-up was 9.1 and 3.4 months, respectively). The Oral-AZA cohort had less HCRU than the NoMaint cohort in various categories (0.23 vs 0.61 hospitalizations PPPM, 2.47 vs 4.40 non-office outpatient visits PPPM, 2.84 vs 4.53 laboratory tests PPPM; all P<0.01). These differences translated into lower all-cause costs for the Oral-AZA cohort (US dollars [USD] 25,786 PPPM vs USD 38,530, P<0.001), including USD 9222 in maintenance drug costs for Oral-AZA versus none for NoMaint. CONCLUSIONS: The cost burden (PPPM) of HCRU of patients with AML treated with Oral-AZA is significantly lower than those who did not receive a maintenance treatment. This economic benefit is consistent with the clinical benefits observed in the QUAZAR AML-001 trial among Oral-AZA users compared with patients receiving no maintenance treatment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23borateee86poster128312-pdf.pdf?sfvrsn=fc08778a_0","title":"ISPOREurope23_Borate_EE86_POSTER128312.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128312","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"The Impact of Moderate to Severe Chronic Hand Eczema and the Effect of Improved Clinical Symptoms on Work Productivity Loss: Data from the DELTA 1 and DELTA 2 Phase 3 Trials","id":"e0cc5b3b-157c-4327-add2-e859a032ab60","sessionCode":"PCR41","topDisplay":"Bauer A1, Halling C2, Korn S2, Nyholm N2 1University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany, 2LEO Pharma A/S, Ballerup, Denmark","locationCode":"6040","description":"\r\n\t<div>OBJECTIVES: Chronic hand eczema (CHE), a heterogenous fluctuating inflammatory skin disease of the hands and wrists, can have a substantial impact on patients’ daily lives and physical functioning. The impact of CHE on work productivity was assessed in patients enrolled in two clinical trials. METHODS: DELTA 1 and 2 (NCT04871711 and NCT04872101) were phase 3 trials of identical design in which adults with moderate to severe CHE were randomized (2:1) to twice-daily delgocitinib cream 20 mg/g or cream vehicle for 16 weeks. Data were pooled from both trials and work productivity loss (i.e., absenteeism and presentism) was assessed using the Work Productivity and Activity Impairment (WPAI):CHE questionnaire. The association between treatment response as measured by relative change in Hand Eczema Severity Index (HECSI) and work productivity loss at 16 weeks was assessed. RESULTS: Across the two trials, 960 patients were randomized to delgocitinib cream or cream vehicle. A total of 772 patients (80.5%) reported being employed full-time (64.8%) or part-time (15.7%). An environmental trigger for CHE was reported by 485 patients (51.5%); of these, 268 (55.3%) considered CHE occupationally relevant. Onset/worsening of CHE symptoms during work was reported by 352 patients (36.7%). Among employed patients, 144 (18.7%) reported days home from work due to CHE during the last year (<7 days, 49.3%; 7–21 days, 20.1% and >21 days, 30.6%). Overall, least squares mean change from baseline in work productivity loss score was −17.7%-points after 16 weeks of delgocitinib cream. When analyzed by clinical response to delgocitinib cream, greater HECSI response was associated with a greater decrease in work productivity loss score from baseline to week 16 (HECSI<50, −5.2%-points; HECSI-50-<75, −16.2%-points; HECSI-75-<90, −24.8%-points; HECSI-90-<100, −27.4%-points). CONCLUSIONS: CHE is associated with impaired work productivity in adults with moderate to severe CHE. Clinical improvements in CHE were associated with reduced work productivity loss.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23bauerpcr41poster129237-pdf.pdf?sfvrsn=73a16271_0","title":"ISPOREurope23_Bauer_PCR41_POSTER129237.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129237","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Differences in Prices of Certain Drugs (Used for Rare Diseases, Diabetes, Asthma, and Oncology) in Central and Eastern European Countries","id":"ab5dcaba-843e-4ca5-b976-e85d691d5bcf","sessionCode":"HPR20","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"3074","description":"\r\n\t<div>OBJECTIVES: This study aims to compare drug prices from 4 therapeutic areas: rare diseases, diabetes, asthma, and oncology across selected Central and Eastern European (CEE) countries. The drugs chosen for comparison are Spinraza (for rare diseases), Forxiga (for diabetes), Fasenra (for asthma), and Opdivo (for oncology). The project also showed whether the countries have similar access to modern drug technologies. METHODS: A desk review of reimbursement documents in 10 CEE countries (members of the European Union) was conducted (Bulgaria, Croatia, the Czech Republic, Estonia, Greece, Hungary, Latvia, Poland, Slovakia, and Slovenia). The prices for the selected drugs were obtained from publicly available sources, such as the websites of the Ministry of Health. RESULTS: The chosen drugs are reimbursed in most of the selected CEE countries. Fasenra and Forxiga are the cheapest in Hungary and Opdivo – in Hungary and Slovenia. Forxiga’s price is almost twice as high in the Czech Republic as in Hungary and Latvia more than twice as high. Latvia has the highest price of Fasenra. Opdivo is available in smaller sizes (4 ml,10 ml) in most selected countries and for these packages, the price is the highest in Poland. Spinraza's price is the highest in Latvia and the lowest in Croatia and Slovenia. CONCLUSIONS: In most selected European countries, the drugs covered by the study are reimbursed, and their prices, with a few exceptions, are at a quite similar level.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133579","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Single-Use Vs Reusable Endoscopy Reprocessing: An Efficiency Survey of Nurses and Technicians","id":"0cad0df2-2429-43da-8e1a-e957186cd3d2","sessionCode":"OP3","topDisplay":"Hoffman D, Haislip I, Dehlholm-Lambertsen E, Cool C Ambu USA, Columbia, MD, USA","locationCode":"5076","description":"\r\n\t<div>OBJECTIVES: Numerous studies have highlighted the potential time savings single-use endoscopes (SUEs) may afford facilities when compared to reusable endoscopes (REs) due to their ability to eliminate post-procedure cleaning. This time savings may not only allow for more procedures to be performed, but free up the resources and time of individuals responsible for cleaning both the procedure rooms and REs. The purpose of this survey was to evaluate the impact SUEs could have on the individuals responsible for procedure room turnover and RE reprocessing. METHODS: Clinical training specialists across the United States distributed surveys to nurses, reprocessing technicians, and others involved in endoscope reprocessing. Data collection took place from September 2022 to February 2023. Proportions were calculated using each question’s applicable respondents. RESULTS: 52 participants participated in the survey. 100% believed using SUEs instead of REs could save time and allow them to spend more time on imperative tasks, with 66% saving 4+ hrs/week and 17% saving 15+ hrs/week. When considering time savings, 70% believed they would be able to reallocate 4+ hrs/week and 13% believed they could reallocate 15+ hrs/week. Additionally, 11% of individuals felt that SUEs could reduce 6+ hrs/week of after-hours and/or weekend time. Finally, 89% of reprocessing technicians felt that eliminating the reprocessing of just one type of scope would reduce the pressure to keep up with the demands of cleaning/reprocessing other types of scopes and equipment. CONCLUSIONS: SUEs may not only save employees time by eliminating endoscope reprocessing but allow them to spend time doing more imperative tasks and reduce job pressures. All respondents felt that utilizing only SUEs could save time and over 17% felt they could save 15+ hrs/week. If widely adopted, facilities may not only see a reduction in costs, but an increase in time savings, employee morale, and elimination of reprocessing related risks.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"bb8fd992-de86-4d1a-8bec-f6e2c928db96","parentId":"00000000-0000-0000-0000-000000000000","title":"Organizational Practices","urlName":"organizational-practices"}],"categoryIds":["bb8fd992-de86-4d1a-8bec-f6e2c928db96"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23dehlholmlambertsenop3poster129964-pdf.pdf?sfvrsn=47eab861_0","title":"ISPOREurope23_DehlholmLambertsen_OP3_POSTER129964.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129964","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Patients’ Satisfaction from Primary Healthcare Services in 1ST Local Health Unit (LHU) of N. Philadelphia-N. Chalkidona Municipality Greece","id":"d377cb5a-c2d4-41ca-b85e-e964cb729167","sessionCode":"HSD25","topDisplay":"Kalagia P1, Theodorou P2, Hatzikou M2 11st Tomy Neas Philadelfias - Neas Kalchidonas, Athens, Greece, 2Hellenic Open University, Patras, Greece","locationCode":"4033","description":"\r\n\t<div>OBJECTIVES: To assess patients' satisfaction from primary health services of 1st TOMY of Nea Philadelfia - Nea Chalkidona. METHODS: A cross-sectional study was performed from April 2022 to May 2022. A questionnaire for patient satisfaction from primary healthcare services was used Goula et al, 2019*, followed by demographic questions. The sample constituted by 118 patients who visited the 1st Local Health Unit (LHU) of Nea Philadelphia-Nea Chalkidona in Attica. RESULTS: The overall satisfaction level of the participants was very high (Μean 4.91+0.3), similarly, for the nursing staff (Μean 4.91+0,3), medical care (Μean 4.96+0,2), the facilities (Μean 4.78+0.4), and the administrative services (Μean 4.80+0.4). Additionally, there was a statistical significant difference for participants who waited longer, since they were less satisfied with nursing care (p=0.003), and facilities (p=0.002), while participants with more visits were more satisfied with nursing (p=0.036), and medical care (p=0.024). Finally, participants with chronic illnesses were less satisfied from administrative services (Mean 4.80 +0.4). The most frequent reasons for visiting the health center was the medicine prescription (32.4%), referral testing (28.8%) and examination (28%). Regarding the waiting time between the scheduled appointment and the visit, the majority (66.4%) waited less than 15 minutes, 13.8% waited 15-30 minutes while 12.9% had no waiting time. CONCLUSIONS: Primary Care Services are not very well established in Greece due to direct patients' access to secondary/hospital services and the degree of patients' satisfaction is important for the decision making process. * A. Goula et.al (2019) Development and validation of a patient satisfaction questionnaire for use in primary health care. Archives of Hellenic Medicine, 36(1):88–95</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23hatzikouhsd25poster132457-pdf.pdf?sfvrsn=9e6e2071_0","title":"ISPOREUROPE23_HATZIKOU_HSD25_POSTER132457.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132457","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Development of a Comprehensive List of Immunosuppressive Therapies to Enable a Multi-Data Source Global Real-World Effectiveness (RWE) Program of Immunocompromised Patients","id":"b2dba401-008c-438f-8d61-e9a3d9141b50","sessionCode":"RWD29","topDisplay":"Kamauu AW1, Parker CG2, Shields A3, Glaser L4, DuVall S5, Haidar G6, Lynch J5, Kamauu AG2, Taylor S7, Talarico C8 1Navidence LLC, Bountiful, UT, USA, 2Navidence LLC, Salt Lake City, UT, USA, 3Navidence LLC, Chapel Hill, NC, USA, 4Medical Affairs, Vaccines & Immune Therapies, BioPharmaceuticals Medical, AstraZeneca, Wilmington, DE, USA, 5VA Informatics and Computing Infrastructure, VA Salt Lake City Health Care System, Salt Lake City, UT, USA, 6UPMC and University of Pittsburgh, Pittsburgh, PA, USA, 7AstraZeneca, Cambridge, Cambridgeshire, UK, 8Epidemiology, Vaccines & Immune Therapies, BioPharmaceuticals Medical, AstraZeneca, Gaithersburg, MD, USA","locationCode":"","description":"\r\n\t<div>OBJECTIVES: Patients who are immunocompromised (IC) form a heterogeneous population in which evaluation of effectiveness and safety of medicines and vaccines is challenging — partly due to a lack of concordance on which medications are considered immunosuppressive. In 2021, AZD7442 (two monoclonal antibodies, tixagevimab/cilgavimab) received emergency use authorization to prevent COVID-19 in IC patients, including those treated with immunosuppressive therapies. To enable AZD7442 effectiveness studies conducted globally across IC patients, multiple sources and clinical input were used to define a comprehensive list of immunosuppressive therapies. METHODS: A rigorous process was used to develop the comprehensive list. <ol> <li>Immunosuppressive medications collected from 5 source lists: <ul> <li>NLM Value Set Authority Center</li> <li>Master Protocol, COVID-19 Natural History, Sentinel Initiative 2020</li> <li>Two health systems within the AZD7442 RWE program: US Department of Veterans Affairs and University of Pittsburgh Medical Center</li> <li>Medication classes from SUPERNOVA (AZD3152 trial); all medications per class in use worldwide were included</li> </ul> </li> <li>Medications on ≥3 source lists preliminarily accepted for comprehensive list</li> <li>Medications on 1-2 source lists further evaluated: <ul> <li>Use at health systems or in SUPERNOVA deemed relevant based on clinical judgment</li> <li>Others received additional review, including determining approved indications and mechanisms of action</li> </ul> </li> <li>Medical review of the comprehensive list by authors, with adjudication of select medications by SUPERNOVA investigators</li> <li>RxNorm (ingredient-level) and anatomical therapeutic chemical (ATC)-5 codes determined for each medication on comprehensive list</li> </ol> RESULTS: The five source lists each contained 71-110 medications, with the comprehensive list including 157 distinct medications across 21 classes. In the comprehensive list, 143 medications had RxNorm codes and 148 had ATC-5 codes; 4 medications without codes were only approved in countries not using those coding systems. CONCLUSIONS: A comprehensive list of immunosuppressive medications (with associated medication codes) was defined that can be used globally to increase consistency across studies of IC patients. Supported by AstraZeneca.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129704","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Safety and Effectiveness of Sodium Oxybate in the Management of Narcolepsy: A Systematic Literature Review","id":"78e091a7-12b7-48e9-b21f-ea8de36e66f7","sessionCode":"RWD10","topDisplay":"Kandregula N1, Choletti V1, Kalakota C1, Vullengala N1, Bhavanasai S1, Pesara NS1, Udhayasri R1, Gomes A1, Dang A2, Dang D1, Vallish BN1 1MarksMan Healthcare Communications, Hyderabad, Telangana, India, 2MarksMan Healthcare Communications, Hyderabad, AP, India","locationCode":"6063","description":"\r\n\t<div>OBJECTIVES: To examine the clinical (safety, effectiveness) and humanistic (QoL and other PROs) outcomes associated with sodium oxybate (SXB) in the management of narcolepsy in real-world settings. METHODS: We searched PubMed for relevant records on 10th November 2022, using appropriate search strategies. Publication details, demographics, intervention, comparator, effectiveness, safety and humanistic outcomes were extracted from eligible records. RESULTS: Of the 425 records screened, 21 publications from 17 studies containing 2,228 unique participants (females: 1,418, males: 810; adults (10 studies): 2,047; children/ adolescents (7 studies): 181) were included in analysis. Mean age ranged from 34.3 to 44.1 years in adults, 11.0 to 15.3 years in children. In 11 studies, SXB was administered as per recommended dosage. Of the 17 included studies, effectiveness, safety, and humanistic outcomes were evaluated by 17, 5, and 2 studies respectively. SXB treatment was associated with a reduction in Epworth Sleepiness Score, total sleep time, proportion of REM sleep, wakefulness after sleep onset, awakenings, and cataplexy. SXB increased sleep efficiency, duration of slow wave sleep (night and total), duration of daytime wakefulness, and sleep latency. SXB also improved humanistic outcomes including sleep quality, daytime functioning scores, SF-36 scores (physical component, mental component, and all 8 subscales) and patient-reported outcomes (presenteeism, overall work and activity impairment, and risk of motor vehicle accidents). No new safety signals were noted. There was a lack of uniformity in the outcomes assessed by included studies. CONCLUSIONS: SXB is safe and effective in narcolepsy patients treated in real-world settings. Future real-world studies need to evaluate standard outcomes consistently to enable better analysis of data on effectiveness and safety.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/real-world-safety-and-effectiveness-of-sodium-oxybate-in-the-management-of-narcolepsy131389-pdf.pdf?sfvrsn=e0f7c245_0","title":"Real-world Safety and Effectiveness of Sodium Oxybate in the Management of Narcolepsy131389.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131389","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Muscular Dystrophy-Related Hospitalizations and Outpatient Procedures in the Brazilian Public Healthcare System: A Real World Data Analysis","id":"4f8b9f95-aff4-473a-a1b7-eb47ceb6456c","sessionCode":"EPH20","topDisplay":"Lara L1, Marcolino M1, Schneider N1, Casiraghi Y1, Etges AP2, Polanczyk CA1 1Universidade Federal do Rio Grande do Sul (UFRGS), Porto Alegre, RS, Brazil, 2National Institute of Science and Technology for Health Technology Assessment (INCT-IATS), Porto Alegre, RS, Brazil","locationCode":"3022","description":"\r\n\t<div>OBJECTIVES: Muscular dystrophy (MD) patients are at risk of several complications, including respiratory failure as a main cause of death. Our aim is to describe the profile and outcomes of MD-related hospitalizations and outpatient procedures in the Brazilian Public Healthcare System (SUS). METHODS: We analyzed open data of hospital admissions and outpatient procedures, between 2018 and 2022, of patients with muscular dystrophy (ICD-10: G71.0) registered in the Public Information Systems. RESULTS: Between 2018 and 2022 were registered 3,034 MD-related hospitalizations. Most of them were male patients (57%), with a mean age of 27 (± 22) years. The overall in-hospital mortality rate for the five years was 4.8%. The primary procedure codes listed were muscular dystrophy treatment (66.2%), followed by treatment for neuromuscular diseases’ complications (13%). Additionally, pneumonia and respiratory failure were the most common secondary diagnosis reported, with a mortality of 21.9% and 22%, respectively. Average payment by hospitalization was BRL 3,544.5 (± BRL 6,876.3). In the same period, were registered 371,641 outpatient procedures (28% performed in healthcare facilities located in cities other than patient’s residence). Most of them for male patients (64%). Most procedures (63%) were physiotherapy-related (respiratory or physical rehabilitation) and home-based non-invasive mechanical ventilation (24.6%). Average annual cumulative payment by outpatient procedures was BRL 11,948,533 (± BRL 613,172). CONCLUSIONS: Data from the last five years shows that most MD-related procedures performed in the SUS were outpatient care. The majority of hospital procedures were due to MD treatment, while physiotherapy comprises most outpatient procedures. The primary comorbidities observed were respiratory system-related. These results are important to characterize the disease hospitalization and outpatient profile, and are consistent with the natural history of muscular dystrophies.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23marcolinoeph20poster133931-pdf.pdf?sfvrsn=9559d985_0","title":"ISPOREurope23_Marcolino_EPH20_POSTER133931.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133931","diseases":[{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Framework Concepts for Economic Evaluation of New Antibiotics","id":"d5aeec3f-e6d0-43eb-8854-eb576815d1f4","sessionCode":"EE30","topDisplay":"Yang W, Dong H Zhejiang University, Hangzhou, Zhejiang, China","locationCode":"1081","description":"\r\n\t<div>OBJECTIVES: Currently, the value of new antibiotics is underestimated since the special “externalities” are not considered in conventional economic evaluation. This conceptual paper aims to provide a practical framework with transmission value, namely the value of successful treatment in preventing or reducing the spread of infection.\nMETHODS: Framework: ICER (Incremental Cost-Effectiveness Analysis)=(∆C-St)/(∆V+Vt ).\nRESULTS: Under the hospital setting, we assume that there is just one bacterial infection and that the particular pathogen is resistant to the existing antibiotic. The incidence of infections will decrease as a result of prevented transmission once the new antibiotic has been administered to resistant inpatients for a while. The integers Nbegin and Nend represent the number of inpatients who are infected when admission and all infected inpatients after the given period, respectively. Na is the total number of avoided transmission cases between utilizing new and exiting antibiotics (1).\nNa=∆(Nend - Nbegin) (1)\nDifferences in Cost, Hospital LOS and Utility Value are between inpatients utilizing new and existing antibiotics (2), (3). ∆V is measured in quality-adjusted life years (QALYs) (3).\nIncremental Direct Costs (∆C)=Nbegin × ∆Cost (2) \nIncremental Direct Benefits (∆V)=Nbegin × ∆Hospital LOS × ∆Utility Value (3)\nInpatients could only be treated with existing antibiotics before the new antibiotic being available (4). We assume that the Hospital LOS for healthy individuals is 0. Differences in Hospital LOS and Average Utility Value are between healthy individuals and inpatients utilizing existing antibiotic (4),(5).\nTransmission Savings (St)=Na × Cost of Existing Antibiotic (4)\nTransmission Benefits (Vt)=Na × ∆Hospital LOS × ∆Average Utility Value (5)\nCONCLUSIONS: More scenarios, such as the population setting where Na is available from the dynamic disease transmission model, could be covered by the framework. To do so would better understand the direct and transmission value of new antibiotics and inform clinical and reimbursement decisions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23yangee30poster129733-pdf.pdf?sfvrsn=41ab3fce_0","title":"ISPOREurope23_Yang_EE30_POSTER129733.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129733","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Survey of Situation and Severity of Public Problems Affecting Health in an Industrial District in Thailand","id":"971c353d-297a-4e76-b573-eb6f19c769c6","sessionCode":"EPH52","topDisplay":"Rutjanathamrong P1, Siripattanakulkajorn W2, Phorntrakoonpakdee P2, Sooksriwong C3, Phodha T4 1Faculty of Pharmacy, Thammasat University, Khlong Luang District, 13, Thailand, 2Klong Luang Hospital, Khlong Luang District, Pathum Thani, Thailand, 3Faculty of Pharmacy, Thammasat University, Khlong Luang District, Pathum Thani, Thailand, 4Faculty of Pharmacy, Thammasat University, Klong Luang, 13, Thailand","locationCode":"3053","description":"\r\n\t<div>OBJECTIVES: Khlong Luang District, Pathum Thani Province, Thailand, could be recognized as a national distribution center of several kinds of goods especially cosmetics and medicines. If the factories in this area did not pass the standardize for industrial, it would be impact to the other regions across Thailand. This study is to survey the public problems affecting people health in Khlong Luang District and to investigate the contexts those related to the burden and severity of the public problems. METHODS: This study is a mixed method. The retrospective data from the hospital database was used to analyze the burden of the public problems. Focus group interview among the community leaders with an interactive multi-voting process were performed to gather the context relevant to the severity of the public problems. Health outcomes including incidence rate, length of stay (LOS), and cost of illness of inpatient admission affected by the adverse event of using illegal medicines sole by grocery stores. RESULTS: The survey in 2019 reported 48.5% of grocery stores (11 out of 24) sole NSAIDs. The hospital database shows gastrointestinal (GI) bleed was the first leading cause of hospitalizations. The incidence rate of GI bleed, average LOS, and average cost of inpatient admission from the year 2020 to 2022 were 51.55 per 1000 population, 8 days per patient, and 538.47$USD, 80.80 per 1000 population, 4.1 days per patient, and 225.66$USD and 45.45 per 1000 population, 4.5 days per patient, and 464.55$USD, respectively. This finding was consistency with the results from the qualitative study that people in Khlong Luang District are most likely to buy medicines especially NSAIDs from grocery store instead of pharmacy store. CONCLUSIONS: Linkage among stakeholders’ databases including community hospitals, community leaders, and private sectors is needed for implementation of the master plan for tackling those public problems.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23rutjanathamrongeph52poster130258-pdf.pdf?sfvrsn=a3c854f_0","title":"ISPOREurope23_Rutjanathamrong_EPH52_POSTER130258.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130258","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Evaluation of Mailuo Shutong Pill Vs Aescuven Forte Tablets Concomitated with Routine Treatment Separately for Deep Vein Thrombosis Patients in China","id":"b7bee69f-966f-4f1e-8075-eb94e4c4e2a3","sessionCode":"EE126","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"2057","description":"\r\n\t<div>OBJECTIVES: Deep vein thrombosis (DVT) is the third most common cardiovascular disorder in the world, especially in China. The addition of traditional Chinese medicine could improve clinical outcomes and save costs in a big way. The aim of this study is to conduct an economic evaluation between the Mailuo Shutong Pill + routine treatment (MSPC) vs Aescuven forte tablets + routine treatment (AFTC). METHODS: A Markov model was built to compare the cost and effectiveness of MSPC and AFTC for 5 years from both payer and society perspectives. The population in this analysis was a cohort with a DVT diagnosis, receiving MSPC and AFTC separately. The clinical outcome and cost (direct and indirect cost) are calculated based on real-world data from the Tianjin big data platform from January 2015 to December 2021. The utilities were extracted from the literature. Both costs and outcomes were discounted at 3.5%. The incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained was calculated. Probabilistic sensitivity analysis (PSA) was carried out to deal with uncertainty. RESULTS: The base analysis results demonstrated that, from a payer perspective, the two groups showed very close QALYs. AFTC was 0.0110 more QALY, compared to the MSPC group. However, AFTC requires ￥11,721,582.99 above MSPC’s payer cost to obtain that 0.0110 more QALY. Similarly, for the analysis from the society perspective, AFTC needs ￥12,891,333.85 more than MSPC just to achieve 0.0110 more QALY. CONCLUSIONS: This is the first study which used large scale real-world data, to assess the economic value between two traditional Chinese medicines. This study demonstrates that MSPC is a cost-effective alternative to AFTC for the treatment of DVT patients from both the payer and the society perspective.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130034","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of SLE in Nordic Countries - a Systematic Literature Review (SLR)","id":"66057c5a-b7d8-4d8e-b4aa-ee227dff1872","sessionCode":"EE112","topDisplay":"Sharma M1, Ansari S1, Takyar J2 1Parexel International India Pvt LTD, Mohali, PB, India, 2Parexel International, Panchkula, India","locationCode":"2045","description":"\r\n\t<div>OBJECTIVES: SLE has a varied geographic distribution with an incidence of 0.3 to 5.1 per 100 000 per year in Europe. Limited studies report the economic burden associated with SLE in Nordic countries. We undertook a SLR to capture up to date economic burden and resource utilization data for SLE in Nordic region. METHODS: Embase®, Medline® and Cochrane, were searched for studies reporting economic burden of SLE. Studies inclusion was limited by geography (Nordic countries: Denmark, Norway, Sweden, Finland, and Iceland), publication timeframe (January 2013 to June 2023) and language (English language only). RESULTS: Eight studies out of 153 publications met the inclusion criteria. Among these eight studies, five studies reported data for Sweden, two for Denmark and one for Finland. No study reported data for Norway and Iceland. In Sweden, the total cost for SLE was estimated to be 1.177 billion SEK ($188 million= 129.5 million €) with indirect costs being the major contributor (70% of these costs). Activity and duration of disease, aging and presence of comorbidities were associated with higher cost burden. In Denmark, mean annual hospitalization rate was 25%. Complications to SLE and its treatment caused more hospitalizations. The hospitalizations were greater with flares as compared to long quiescent phase. The Finnish nationwide register data on special reimbursements for medication costs reported higher use of DMARDs for treatment of SLE. CONCLUSIONS: The economic burden is prominent among SLE population. Sparse data is available on financial burden of SLE in Nordic region. Further studies are required to assess the direct and indirect cost associated with disease.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23sharmaee112poster132511-pdf.pdf?sfvrsn=83c024e8_0","title":"ISPOREurope23_Sharma_EE112_POSTER132511.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132511","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Time to HTA Assessment Completion By HTA Agency Archetype: Five-Year Review for 20 European Countries","id":"f244ac4b-39eb-426d-acff-ee4329f4f618","sessionCode":"HTA52","topDisplay":"Izmirlieva MA GlobalData, London, London, UK","locationCode":"5012","description":"\r\n\t<div>OBJECTIVES: Countries using health technology assessment (HTA) as part of their pricing and reimbursement review process differ in the way their HTA agency is organized. HTA agencies can be broadly split into four groups: advisory arm-length, advisory integrated, regulatory (i.e., issuing binding decisions) arms-length and regulatory integrated. We examine the length of HTA review by agency archetype to examine if there is a correlation between HTA agency archetype and the length of time between marketing authorization and the publication of an HTA assessment. METHODS: Twenty national-level European HTA agencies were categorized under the four archetypes. All final HTA decisions in the 20 European countries issued between 1 January 2018 and 31 December 2022, based on POLI data, were examined and the average time, in days, between marketing authorization and first HTA decision, as well as between marketing authorization and first positive HTA decision were calculated for each of the four agency archetypes. RESULTS: The longest time by far between marketing authorization and first HTA decision was for the regulatory integrated archetype (1,238 days), followed by advisory integrated agencies (782.5 days), while countries with advisory arms-length and regulatory arms-length agencies had a similar time to first HTA of 675.8 days and 674.8 days, respectively. The ranking was maintained if the time to first positive HTA decision is examines instead, with 1,244 days for regulatory integrated agencies, 813 days for advisory arms-length, 748.9 days for regulatory arms-length and 737.7 days for advisory arms-length agencies. CONCLUSIONS: Countries with arms-length agencies of both archetypes were found to produce HTA reviews faster, and were also more likely to issue the first positive HTA decision faster compared to integrated agencies. The long review time for the regulatory integrated archetype should be treated with caution as there was a single national-level HTA agency in this group.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23izmirlievahta52poster133302-pdf.pdf?sfvrsn=8f64ce6d_0","title":"ISPOREurope23_Izmirlieva_HTA52_POSTER133302.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133302","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Investigation of Value for Money of Oncology Drugs in Canada","id":"6e490347-7561-4d44-9663-eeb9b36970db","sessionCode":"EE53","topDisplay":"Jackson A1, Adamo RG2, Villeneuve PJA2 1University of Ottawa, Chatham, ON, Canada, 2University of Ottawa, Ottawa, ON, Canada","locationCode":"2008","description":"\r\n\t<div>OBJECTIVES: Cancer care cost is increasing to unsustainable levels due, largely in part, to rising drug prices. Therefore, it is key to better assess the value of oncology drugs to help guide future drug funding recommendations. We aim to examine the value-for-money of oncology drugs reviewed by the pan Canadian Oncology Drug Review (pCODR). METHODS: We systematically reviewed the oncology drugs for which pCODR made reimbursement recommendations from 2011 to 2019. Data were extracted from pCODR’s funding recommendation reports by two independent reviewers, and discrepancies were resolved by a third reviewer. Data was collected on drug characteristics, cost, clinical benefit, and incremental cost utility ratio (ICUR). RESULTS: Of the 142 submissions reviewed, 10 (7.0%) received a pCODR recommendation to fund the drug, 29 (20.4%) to not fund the drug, and the remaining 103 (72.5%) received a recommendation for funding on a conditional basis, most of which (96.1%) were due to poor cost-effectiveness. Most drugs (89.4%) were submitted for palliative intent, and the most common cancer type indicated was hematological malignancies (21.8%). Only 14 (9.8%) drugs demonstrated improved toxicity over a comparator while 77 (54.2%) exhibited worse toxicity. Regarding health-related quality of life, 46 (32.4%) drugs demonstrated an improvement over relevant comparators, while 57 (40.1%) were unchanged or worsened. The average ICUR of the submitted drugs was $233,273.90 (range -$16,690 to $2,120,433) and the average change in quality adjusted life-years (QALY) was 0.621 (range 0 to 4.74). The average ICUR increased over time from $194,014.50 in the first half of the study to $260,969.60 in the second half. CONCLUSIONS: The results of this study indicate that the increase in cost of oncology drugs has outpaced the increase in clinical benefit. Provinces should make more judicious choices when making reimbursement decisions, considering not only the drug price, but also the value for money. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23jacksonee53poster128562-pdf.pdf?sfvrsn=c8922f53_0","title":"ISPOREurope23_Jackson_EE53_POSTER128562.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128562","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical Outcomes Associated with Sodium Zirconium Cyclosilicate for Treating Hyperkalaemia in Patients Receiving Haemodialysis","id":"2e6cf09c-00ae-4b74-9942-ef4a7d3ef32f","sessionCode":"CO35","topDisplay":"Ferraro PM1, Booth D2, Silvanto J2, McEwan P2, Rao N3 1Dipartimento Universitario di Medicina e Chirurgia Traslazionale, Università Cattolica del Sacro Cuore, Rome, Italy, 2Health Economics and Outcomes Research Ltd, Cardiff, UK, 3AstraZeneca, Cambridge, CAM, UK","locationCode":"1034","description":"\r\n\t<div>OBJECTIVES: The Dialysis Outcomes and Practice Patterns Study (DOPPS) demonstrated that a high proportion of Italian patients on maintenance haemodialysis have elevated peak serum potassium (sK+), which is associated with increased risk of adverse outcomes. In the DIALIZE (NCT03303521) trial, sodium zirconium cyclosilicate (SZC) significantly increased the proportion of patients with end stage kidney disease who maintained predialysis sK+ 4.0–5.0 mmol/L versus placebo. This study assessed the projected impact of SZC versus placebo on clinical outcomes including kidney transplants in individuals on haemodialysis in Italy. METHODS: A semi-Markov state transition model was used, comprising seven health states: haemodialysis with and without cardiovascular disease (CVD), kidney transplant with and without CVD, CV hospitalisation, CV and non-CV death. Non-CV hospitalisation was modelled as a transient event. Population characteristics inputs and sK+-dependent risks of hospitalisation and death were informed by DOPPS-Italy data, while transplant rates were informed by an Italian registry study. Treatment efficacy was derived by sampling sK+ trajectories from DIALIZE SZC and placebo arms to predict peak sK+ across a four-month period. Outcomes were predicted over a lifetime horizon. RESULTS: SZC treatment was estimated to reduce overall hospitalisations by 30.2 per 1,000 patients over the modelled time horizon compared with placebo. CV deaths were also reduced compared with placebo, by 55.1 CV deaths per 1,000 patients. The incremental increase in life years of 0.289 associated with SZC treatment compared with placebo led to 6.9 more kidney transplants per 1,000 patients over the time horizon. CONCLUSIONS: Modelling using trial data from DIALIZE combined with real-world evidence from DOPPS-Italy indicate that management of hyperkalaemia with SZC in patients on haemodialysis may be associated with fewer hospitalisations and CV deaths. Additionally, treatment with SZC may allow more haemodialysis patients to survive to benefit from kidney transplantation and the anticipated associated improvements in quality of life.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23raoco35poster129919-pdf.pdf?sfvrsn=dc2dbce0_0","title":"ISPOREurope23_Rao_CO35_POSTER129919.pdf"},{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23raoco35handout129919-pdf.pdf?sfvrsn=35c0ac88_0","title":"ISPOREurope23_Rao_CO35_HANDOUT129919.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129919","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"7f059074-623e-4c96-8812-8affdc603f10","parentId":"00000000-0000-0000-0000-000000000000","title":"Urinary/Kidney Disorders","urlName":"Urinary-Kidney-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Excess Healthcare Expenditure in Adults Treated for Solid Cancer in Childhood: A Cohort Study in France","id":"4ffe10a8-f311-4af3-9f1b-efa6326940fa","sessionCode":"EE56","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1096","description":"\r\n\t<div>BACKGROUND: Due to late effects, childhood cancer survivors (CCS) are more likely to have multiple chronic conditions than the general population. However, little is known about the economic burden of care of CCS in the long term. OBJECTIVES: To estimate excess healthcare expenditure for long-term CCS in France compared to the general population and to investigate the associated factors. METHODS: We included 5353 5-year solid CCS diagnosed before the age of 21 years before 2000 from the French CCS cohort and obtained a random reference sample from the general population for each CCS, matched on age, gender and region of residence. We used the French national health data system to estimate annual healthcare expenditure between 2011 and 2018 for CCS and the reference sample and computed the excess as the net difference between CCS expenditure and the median expenditure of the reference sample. We used repeated-measures linear models to estimate associations between excess healthcare expenditure and CCS characteristics. RESULTS: Annual mean (95% CI) excess healthcare expenditure was €3,920 (3539; 4301), mainly for hospitalization (39.6%) and pharmacy expenses (17%). Higher excess was significantly associated with having been treated before the 1990s and having survived a central nervous system tumor, whereas lower excess was associated with CCS who had not received treatment with radiotherapy. CONCLUSIONS: Of the variables that influence excess healthcare expenditure, a lever for action is the type of treatment administered. Future research should focus on addressing the long-term cost-effectiveness of new approaches, especially those related to radiotherapy. <quillbot-extension-portal></quillbot-extension-portal> <quillbot-extension-portal></quillbot-extension-portal></div>\r\n\r\n","withdrawn":true,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128214","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"ac8e5c06-82a1-4ece-9b58-c1a36a72a607","parentId":"00000000-0000-0000-0000-000000000000","title":"Pediatrics","urlName":"Pediatrics"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Overview of Recent Systematic Literature Reviews on Glucagon-like Peptide-1 Receptor Agonists for Weight Loss in Adults with Obesity","id":"3a68b327-e55c-4ab6-a279-efb52fbc586f","sessionCode":"CO4","topDisplay":"Smoyer KE1, Moubarak L2, Singh S2, Kandola S3 1Envision Pharma Group, Philadelphia, PA, USA, 2Envision Pharma Group, London, LON, UK, 3Envision Pharma Group, London, UK","locationCode":"1004","description":"\r\n\t<div>OBJECTIVES: The global prevalence of obesity has drastically increased over the past three decades, increasing the risk of obesity-related morbidity and premature mortality. Lifestyle/behavioral interventions for weight loss have had limited success. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are emerging as highly effective anti-obesity medications (AOMs). This research aims to identify key trends in systematic literature reviews (SLRs) on GLP-1 RAs in adults with obesity. METHODS: Embase, Medline, and Cochrane databases were searched for SLRs published between 1/Jan/2018 and 16/May/2023 using a NICE-published search strategy for obesity, along with intervention and SLR terms. SLRs of adults with obesity treated with GLP-1 RAs were included. SLRs exclusively on other AOMs, herbal treatments, or non-pharmacological interventions were excluded. A single reviewer screened references, with a 10% check by a second reviewer. RESULTS: After removing duplicates, 1317 publications were identified and screened, with 106 retained for assessment of full text. A total of 47 relevant SLRs met inclusion criteria. The number of SLRs more than quadrupled from 2018 to 2022, and as of 2023, seven additional SLRs had been published. The majority included meta-analysis (80.85%). The most commonly assessed GLP-1 RA was liraglutide (85%), followed by semaglutide (43%), exenatide (34%), and dulaglutide (13%). Lixisenatide (9%), efpeglenatide (6%), albiglutide (2%), and tirzepatide (2%), were also identified. The most common subpopulations were those without diabetes (21.3%), diabetes (10.6%), polycystic ovary syndrome (8.5%), and schizophrenia/psychosis (6.4%). Overall, the SLRs found GLP-1 RAs to be effective AOMs. CONCLUSIONS: Recent SLRs reflect the growing number of GLP-1 RAs, which uniquely target both peripheral and brain mechanisms involved in weight regulation. GLP-1 RAs show promise as efficacious and safe pharmacological treatment for obesity; however, the long-term benefits are not yet known. Subsequent SLRs are needed to synthesize evidence on newer AOMs, including dual receptor agonists such as tirzepatide, once data are published.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kandolac04poster130014-pdf.pdf?sfvrsn=3fbf9156_0","title":"ISPOREurope23_Kandola_C04_POSTER130014.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130014","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of a Clinical Care Pathway for the Screening of Nonalcoholic Fatty Liver Disease in Patients with Type 2 Diabetes Mellitus","id":"ed450c42-1733-417d-b2b2-eff1f8caac5d","sessionCode":"HTA2","topDisplay":"Xiao J1, Haseeb M2, Kanwal F3, Kim S4, Ayer T5, Ajmera V4, Huang DQ4, Tincopa M4, Loomba R4, Chhatwal J6 1Georgia Institute of Technology, Wilmington, MA, USA, 2Beth Israel Deaconess Medical Center, Boston, MA, USA, 3Baylor College of Medicine, Houston, TX, USA, 4University of California at San Diego, San Diego, CA, USA, 5Georgia Institute of Technology, Atlanta, GA, USA, 6Massachusetts General Hospital, Boston, MA, USA","locationCode":"4043","description":"\r\n\t<div>OBJECTIVES: The American Gastroenterological Association (AGA) recently published a Clinical Care Pathway for the management of nonalcoholic fatty liver disease (NAFLD) with the aim of facilitating efficient, value-based care. Development of the Pathway was based on the expertise of a multidisciplinary task force. However, it has thus far not been evaluated in terms of cost-effectiveness. Our objective was to assess the cost-effectiveness of the Pathway for managing NAFLD in “high risk” patients, specifically, those with type 2 diabetes mellitus (T2DM). <div> METHODS: We developed a decision-analytic model to evaluate the performance of the Pathway on economic and health-related outcomes. The model simulated the natural history of NAFLD and imposed a multi-step testing and treatment pathway. We used data from a cohort of 501 T2DM patients to inform risk stratification and distribution of non-invasive test scores. We simulated the life course of 50-year-old T2DM patients under usual care versus the Clinical Care Pathway. Model outputs were used to perform cost-effectiveness analysis from the healthcare payer perspective. </div> <div> RESULTS: Compared with usual care, the Clinical Care Pathway detected 34,305 additional cases of NAFLD with significant fibrosis and prevented 2,048 cases of decompensated cirrhosis, 943 cases of hepatocellular carcinoma, 3,403 liver-related deaths, and 946 non-liver-related deaths per 100,000 patients. The Pathway increased quality-adjusted life years (QALYs) by 0.28 and total costs by $14,375 per patient lifetime. With an incremental cost-effectiveness ratio of $50,777 per additional QALY, the Pathway is cost-effective. Furthermore, cost-effectiveness was not sensitive to the value of key input parameters. </div> <div> CONCLUSIONS: The Clinical Care Pathway is cost-effective for the management of NAFLD in patients with T2DM. Widespread adoption of the Pathway in clinical practice could improve NAFLD-related outcomes.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23xiaohta2poster129540-pdf.pdf?sfvrsn=331e7d21_0","title":"ISPOREurope23_Xiao_HTA2_POSTER129540.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129540","diseases":[{"id":"fa5c47a6-1e67-420d-baf4-019c13fc2b50","parentId":"00000000-0000-0000-0000-000000000000","title":"Gastrointestinal Disorders","urlName":"Gastrointestinal-Disorders"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"How Will Recent Pricing Regulations Impact the Global Pricing Landscape?","id":"6f490239-6ad1-4996-9112-f010a6b327a3","sessionCode":"HPR18","topDisplay":"Rémy C1, Lubojemska O2, Chawla A3, Damsgaard MF4 1Parexel International, Milan, Italy, 2PAREXEL International, London, London, UK, 3Parexel, Fremont, CA, USA, 4Parexel International, Copenhagen, Denmark","locationCode":"3073","description":"\r\n\t<div>OBJECTIVES: In a challenging global economic context, pressure on drug prices has never been higher. This research aims to identify current trends in the global pricing landscape by comparing recent regulations in the US, Germany, and France. METHODS: Using a thematic analysis approach, we investigated drug pricing measures in the Inflation Reduction Act passed in the US (2022), the Financial Stabilization Act of the German Statutory Health Insurance System passed in Germany (2022), the French law on the health insurance budget (2023), and the latest agreement between the pharmaceutical industry and Economic Committee for Health Products in France. We used a set of pre-specified criteria including overall regulation type, drug types concerned and price control methods. RESULTS: Through new legislations, two new price control measures were introduced in the US, six in France, and five in Germany. Price control measures included price rebates (US, n=2; Germany, n=2; France, n=1), payback (France, n=1), price freeze (Germany, n=1), outcomes-based agreements (France, n=1), international reference pricing (France, n=1), reduction of free pricing period (Germany, n=1), stricter orphan drugs evaluation (Germany, n=1), price stability measure (France, n=1), and negotiation termination procedure (France, n=1). All measures applied to drugs that are covered by national health insurance but have trickledown effect on private payers. New rules targeted mainly high-cost, innovative and orphan drugs (Us, n=1; France, n=3; Germany, n=1), combination therapies (Germany, n=1), and single-source branded drugs that lost exclusivity (US, n=1). Most measures had a direct impact on launch price (US, n=1/2; France, n=5/6; Germany, n=3/5). CONCLUSIONS: This research shows that the use of drug price control mechanisms is increasing globally, and aligned between the US and Europe. Nevertheless, Europe has an additional price control tool in the form of health technology assessment, and the discrepancy between list price in the US and Europe remains high. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/hpr18isporhow-will-recent-pricing-regulations-impact-the-global-pricing-landscapehwv01132140-pdf.pdf?sfvrsn=8f9234e_0","title":"HPR18_ISPOR_How will recent pricing regulations impact the global pricing landscape_HWv01132140.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132140","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Value Criteria for Outcomes Based Managed Entry Agreement of CAR-T in Relapsed or Refractory Multiple Myeloma (RRMM) through MCDA","id":"7e3c1edd-445b-4e23-95de-f06766e34a04","sessionCode":"HPR9","topDisplay":"Poveda JL1, Mateos MV2, Trillo JL3, Gomez P4, Escudero V5, Oriol A6, Arribas A7, Pons A7, Ariznavarreta Martin A8, Lizán L9 1University Hospital La Fe, Valencia, Valencia, Spain, 2University Hospital of Salamanca/IBSAL/CIC/CIBERONC, Zamora, ZA, Spain, 3Department of Health of Valencia Clínico-Malvarrosa, Valencia, Valencia, Spain, 4Former National Transplant Organisation (ONT), Madrid, Madrid, Spain, 5Pharmacy Service, Gregorio Marañon University Hospital, Madrid, Madrid, Spain, 6Institut Català d’Oncologia and Institut Josep Carreras, Hospital Germans Trias i Pujol, Badalona, Barcelona, Spain, 7Janssen Pharmaceutical Companies of Johnson & Johnson, Madrid, Madrid, Spain, 8Outcomes'10, Castellon, CS, Spain, 9Outcomes'10, Castellón de la Plana, Castellón de la Plana, Spain","locationCode":"3063","description":"\r\n\t<div>OBJECTIVES: The aim of this study is to establish, agree and weigh the most appropriate criteria for the design and monitoring of a outcomes-based managed entry agreement (MEA) for a CAR-T therapy in patients with multiple myeloma who have progressed after a proteosome inhibitor (PI), immunomodulator (IMiD) and anti-CD-38 antibodies, from the point of view of healthcare professionals and decision-makers. METHODS: Multi-criteria Decision Analysis (MCDA) technique was used based on the EVIDEM framework. All the study was assisted by a multidisciplinary scientific committee (SC). The study was divided in two phases: a) selection and structuring criteria through a literature review and consensus with the SC and b) weighting of criteria and sub-criteria by a multidisciplinary group (n=21), composed of different profiles (9 hospital pharmacists, 8 physicians and 4 evaluators/decision-makers). The participants of the multidisciplinary group were selected based on their experience in RRMM or in outcomes-based MEAs or CAR-T therapies and/or MCDA. RESULTS: As a result of the literature review, 12 criteria were identified, of which 5 were selected for the weighting of these (efficacy, safety, Patient Reported Outcomes [PRO], cost of treatment and other direct medical costs of treatment) and their corresponding sub-criteria. The highest score for the design and monitoring of an outcomes-based MEA for CAR-Ts in RRMM was achieved by two efficacy sub-criteria: overall survival (OS) (4.21 ± 0.53) and progression-free survival (PFS) (4.05 ± 0.53). CONCLUSIONS: Based on reflective MCDA methodology and SC’s experience, OS and PFS are the most relevant value criteria for the design and monitoring of an outcomes-based MEA for CAR-T therapies in MMRR. These findings provide valuable insights for developing effective and evidence-based MEAs in the context of CAR-T therapy for RRMM patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130834","diseases":[{"id":"3f8630a8-7f8e-421f-8d3d-0d647b124c8d","parentId":"00000000-0000-0000-0000-000000000000","title":"Genetic, Regenerative & Curative Therapies","urlName":"genetic-regenerative-curative-therapies"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"An Economic Model Comparing Fever Management to Fever Prevention With Advanced Targeted Temperature Management (ATTM) in Out-of-Hospital Cardiac Arrest (OHCA) Patients","id":"3b1c68b6-982d-4d64-9112-f0b47b9da55c","sessionCode":"EE12","topDisplay":"Kelly T, Ramachandran R BD Urology and Critical Care, Atlanta, GA, USA","locationCode":"1054","description":"\r\n\t<div>OBJECTIVES: A large, randomized, multi-national trial of temperature management in OHCA patients found that 46% of patients assigned to the targeted normothermic arm became febrile and required ATTM to manage fever. Fever has been associated with 3.2 additional ICU-days in neurocritical care patients, however, the incremental length of stay (LOS) associated with fever in OHCA patients is unclear. A study in general surgery patients found the time-to-treatment without a fever practice guideline (FPG) to be 51.57 hours and 11.23 hours when an FPG was employed. It is hypothesized that fever management adds a minimum of 11.23 hours to the ICU LOS in febrile OHCA patients. An economic model was developed to compare a Fever Prevention strategy to a Fever Management strategy. METHODS: Two 100-OHCA-patient cohorts were modeled. In the Fever Prevention cohort, ATTM was applied proactively to all patients to prevent fever. In the Fever Management cohort, only patients who developed fever were treated. A 46% fever incidence rate, a 11.23-hour incremental ICU LOS for febrile patients, and a $4,772 cost per ICU-day were employed for the base case analysis. RESULTS: Total cost for the Fever Management cohort in the base case analysis was $176,313 or $1,763 per patient. The Fever Prevention cohort incurred $160,000 in total costs or $1,600 per patient. CONCLUSIONS: This model, subject to the assumptions for fever incidence and impact of fever management on ICU LOS, suggests that proactive application of ATTM for fever prevention in OHCA patients may save between $163 and $578 per patient in the base case analysis.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee12kelly---economic-model-of-fever-prevention-in-ohca---ispor-2023133803-pdf.pdf?sfvrsn=55b6d794_0","title":"EE12_Kelly - Economic Model of Fever Prevention in OHCA - ISPOR 2023133803.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133803","diseases":[{"id":"3597f8d1-60cd-4a60-b852-9d842ea4edf9","parentId":"00000000-0000-0000-0000-000000000000","title":"Cardiovascular Disorders (including MI, Stroke, Circulatory)","urlName":"cardiovascular-disorders-including-mi-stroke-circulatory"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Neoadjuvant Pembrolizumab Plus Chemotherapy Followed By Adjuvant Pembrolizumab for High-Risk Early-Stage Triple-Negative Breast Cancer (ETNBC) in France","id":"2fcb54fd-b2a6-4f01-a442-f0f01d93f222","sessionCode":"EE62","topDisplay":"Cagnan L1, Haiderali A2, Bensimon L3, Tehard B4, Carita M5, Khandelwal A6, Gandhi J7, Huang M8 1MSD France, Puteaux Paris, 76, France, 2Merck & Co., Inc., Rahway, NJ, USA, 3MSD France, Puteaux, 92, France, 4Vyoo Agency, Paris, 75, France, 5Vyoo agency, Paris, France, 6CHEORS, North Wales, PA, USA, 7Complete HEOR Solutions (CHEORS), North Wales, PA, USA, 8Merck Research Laboratories, Merck & Co, Inc., Rahway, NJ, USA","locationCode":"1099","description":"\r\n\t<div>OBJECTIVES: To evaluate the cost-effectiveness of pembrolizumab in combination with chemotherapy as neoadjuvant treatment and continued as a single-agent adjuvant treatment after surgery vs. neoadjuvant chemotherapy for patients with high-risk eTNBC, from the French healthcare system perspective. METHODS: A four-state transition model (event-free (EF), locoregional recurrence (LR), distant metastasis (DM), and death) was developed to estimate costs, effectiveness, and incremental cost-effectiveness ratio (ICER) of pembrolizumab + Carboplatin-Paclitaxel-Anthracycline-Cyclophosphamide (CPAC) vs CPAC and PAC. Transition probabilities and quality of life data were derived from KEYNOTE-522 phase III trial (cut-off 23rd March 2021) for EF & LR states, and from KEYNOTE-355 phase III trial (cut-off June 15th 2021) for DM state, and were extrapolated over a 40-year time horizon based on parametric functions. Patients who remained EF for longer than 8 years were assumed cured. EQ-5D data were converted to French population-based utilities using the French value set. Direct medical costs were considered, based on public sources. Costs and health outcomes were discounted. ICER was calculated as cost per quality-adjusted life year (QALY) gained and per life year gained (LYG). Sensitivity analyses and scenarios analyses were performed to assess the robustness of the results. RESULTS: According to the efficiency frontier, PAC is strictly dominated. Over a 40-year time horizon, the model projects that pembrolizumab + CPAC is associated with discounted 1.86 LYG and 1.32 additional QALY vs CPAC and an incremental cost of 64,475€. ICERs are 34,746€/LYG and 49,007€/QALY. There was an 80% probability for pembrolizumab + CPAC being cost-effective at a willingness-to-pay threshold of 69,000€/QALY. The ICER remains consistent when the transition probabilities exclusively rely on data from the KEYNOTE-522 trial. CONCLUSIONS: Pembrolizumab + CPAC improves life expectancy and appears cost-effective versus chemotherapy alone for neoadjuvant and adjuvant treatment of eTNBC in France, assuming a willingness-to-pay at 69,000€/QALY.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23cagnanee62poster130548-pdf.pdf?sfvrsn=d1b90b5a_0","title":"ISPOREurope23_Cagnan_EE62_POSTER130548.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130548","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Application of CPRD Data in NICE Submissions: Model Inputs for Functional Health States","id":"195217e6-5d50-49cc-9ae7-f174b741cf80","sessionCode":"SA17","topDisplay":"Marks T1, Artignan A2, Judge D3, Griffiths A2 1Costello Medical, London, LON, UK, 2Costello Medical, Cambridge, UK, 3Roche Products Ltd., Welwyn Garden City, UK","locationCode":"7021","description":"\r\n\t<div>OBJECTIVES: To understand the suitability of Clinical Practice Research Datalink (CPRD) data to inform inputs for functional model health states in National Institute for Health and Care Excellence (NICE) submissions. METHODS: Non-oncology NICE technology appraisals published since 2018 were reviewed to identify case studies where the suitability of CPRD data to inform health states based on functional outcomes (i.e. ability to perform daily activities) was explicitly discussed. A feasibility assessment in spinal muscular atrophy (SMA) is also presented. RESULTS: Three case studies were selected from 387 appraisals reviewed. Cannabidiol in tuberous sclerosis complex: suggested use of CPRD data to explore uncertainty was declined by the company due to lack of stratification by seizure-free days to match health states. Avalglucosidase alfa in Pompe disease: the model assumed CPRD resource use data would not differ between health states defined by progression to wheelchair and respiratory support, instead applying one-off and maintenance costs for these. Empagliflozin in chronic heart failure: suggested use of CPRD data for clinical events was rejected by the company as inappropriate to inform health states based on a patient-reported outcome instrument. Differential costs were populated from CPRD data for adverse events only. Feasibility assessment in SMA: following clinical consultation, development of an algorithm to stratify CPRD data into health states based on motor function was found to be infeasible, due to a lack of distinct resource use items associated with specific health states. CONCLUSIONS: While CPRD is generally considered to be an appropriate data source in NICE appraisals, it is not well suited to inform health states based on functional outcomes, which are often selected for rare disease models. To overcome the limitations of CPRD, manufacturers may have to assume no difference between health states, integrate other sources such as expert elicitation or exclude this potentially valuable data source altogether.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23griffithssa17poster132063-pdf.pdf?sfvrsn=faf3b953_0","title":"ISPOREurope23_Griffiths_SA17_POSTER132063.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132063","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Are General Population Utilities Appropriate to Use as Baselines for Age-Adjustment of Utility Values?","id":"7ce7c5fe-f20e-4539-835e-f18e234b5e0a","sessionCode":"EE148","topDisplay":"Chang-Douglass S1, Bungey G2 1Evidera, London, LON, UK, 2Evidera, London, UK","locationCode":"2075","description":"\r\n\t<div>OBJECTIVES: The updated NICE methods manual (PMG36) formally recommends adjusting utility values for age in economic models using general population utility as a baseline. However, NICE Decision Support Unit (DSU) guidance indicates that utility estimates without a condition may be most appropriate to use as a baseline when adjusting utility values for age. Outside of analyses performed on Health Survey for England (HSE) data for cardiovascular disease (CVD) patients (Ara 2010), very few studies have explored the difference between general population utility and utility for people without a condition. Furthermore, it is unclear whether similarities hold between general population utility and utility among people without a condition when fitting an adjusted limited dependent variable mixture model (ALDVMM), which may be a more appropriate and flexible model for EQ-5D data. We sought to test if general population utility was comparable to alternatively utility baselines for three specific patient groups available from HSE EQ-5D-3L datasets. METHODS: OLS and ALDVMM regression models were fitted individually for patient groups without specific conditions (diabetes [n=70,951], CVD [n=46,701], hypertension [n=58,129]) and compared against corresponding general population utility models using available EQ-5D-3L HSE datasets (2003-2014, total N=89,998). RESULTS: OLS and ALDVMM models for patients without each condition were very similar to corresponding general population models. While some differences in EQ-5D-3L utility were observed among the older age groups (>90 years old) in the female cohort in some model comparisons, this may be due to relatively limited sample sizes being available at older age groups. CONCLUSIONS: Among the three groups assessed, general population utility appeared to be a reasonable proxy for utility estimates without the conditions, indicating that use of general population utility as a baseline when adjusting utility values for age in economic models may be appropriate. However, further validation studies are required to test this assumption across other medical conditions.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ee148-chang-douglass-et-alispor-eu-2023-final-posterv1-027oct2023131560-pdf.pdf?sfvrsn=428bb932_0","title":"EE148 Chang-Douglass et al_ISPOR EU 2023 Final Poster_v1.0_27Oct2023131560.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131560","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Use of Clinical Outcome Assessments in Health Technology Appraisals for Rare Disease Indications: A Review of UK, US, and Canadian Submissions","id":"6bf44656-f012-4e20-a355-f1f0c7488682","sessionCode":"HTA27","topDisplay":"Jones E1, Hyman J2, Weijers L3, Johnson N4 1Lumanity, Sheffield, UK, 2Lumanity, Boston, MA, USA, 3Lumanity, Utrecht, Netherlands, 4Lumanity, Long Beach, CA, USA","locationCode":"4061","description":"\r\n\t<div>OBJECTIVES: This study aimed to review use of patient engagement and the use of clinical outcome assessment (COA) in health technology appraisals (HTA) when submitting internationally in a rare-disease indication. METHODS: Reviews were conducted in June 2023; four reviewers independently screened HTAs. The following HTA bodies were included in this search: National Institute for Health and Care Excellence (NICE) and The Institute for Clinical and Economic Review (ICER); and Canadian Agency for Drugs and Technologies in Health (CADTH). Appraisals were included if they were: conducted in the last 5 years (June 2018-June 2023), were complete and were within a rare-disease indication as defined as a life threatening or serious condition affecting ≤ 1 in 50,000 people in the country which the HTA body represents (the very rare disease criteria set by NICE). RESULTS: A total of 27 HTAs, specifically those reviewed under the highly specialized technology assessment, met the criteria. Of the 27 reviewed, 19 considered generic COAs as a key driver in their assessments, while 10 HTAs included disease-specific COAs. All HTAs utilized health utility methods, typically with the EQ-5D or using vignette studies. All HTAs also included patient insight or patient engagement research as part of the review. Only 2 HTAs included patient preference research (e.g., discrete choice experiments, conjoint analysis). Caregiver burden was considered in some of the NICE HTAs. CONCLUSIONS: Several recent HTAs in rare diseases have used COAs and patient engagement research as key drivers for cost-effectiveness models (CEMs) and to document disease burden. Caregiver burden is also of increasing interest and has been utilized in rare disease HTAs more often over the past 5 years. While generic COAs are used most frequently, disease-specific COAs should be considered in order to ensure relevant concepts are reported by patients and utilized appropriately for CEMs.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133974","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Squaring the Circle? Leveraging Early Access / Compassionate Use Pathways to Provide Market-Specific Real-World Evidence Before Launch","id":"4b1a3daa-4a9b-4957-a936-f33bd79b0b70","sessionCode":"HPR37","topDisplay":"Tzaras D Precision Advisors, London, UK","locationCode":"3058","description":"\r\n\t<div>OBJECTIVES: Real-World Evidence (RWE) is an increasing focus of discussion to address limitations in clinical trial data. However, generation of RWE for a new therapy can only typically be generated after the product has launched. Nevertheless, payers want to see product-specific RWE at launch to inform reimbursement decision-making. Early access / Compassionate Use programs (EAPs) offer the opportunity for patients to access therapies prior to marketing authorisation. This then offers the potential to generate RWE for a product in that market prior to its launch. This research evaluates the EAPs available in major European countries to inform a discussion on whether these can be leveraged for RWE collection pre-launch. METHODS: Publicly-available information was screened for information on EAPs in France, Germany, Italy, Spain, the UK, and the Netherlands using their respective websites and key information extracted. RESULTS: All 6 countries offer EAPs, many offer more than one (range: 1[Germany,Spain]–5[Italy]). Inclusion criteria is broadly aligned for most markets, in being for therapies in clinical development for severe/life-threatening diseases with no other treatment options available. Typically, applications can be made by either physicians or manufacturers although for some (e.g. Spain) it is physician-initiation only. Reimbursement/charging incentives vary between countries and within available country programs. In 2/6 (Germany, UK) manufacturers must provide their treatments at zero cost. Another 2/6 (Spain, Netherlands) offer reimbursement only in select cases. Only 2/6 (Italy and France) offer full-reimbursement (although in Italy this is only in select programs). CONCLUSIONS: All major European markets offer EAPs, potentially offering an avenue to collect product-specific RWE outcome data prior to its launch for reimbursement stakeholders. However, there are additional challenges/barriers to this approach, including lack of reimbursement incentives, challenges in collecting outcomes data, and only severe patients being eligible who may experience worse clinical outcomes than the label population.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23macaulayhpr37poster133542-pdf.pdf?sfvrsn=24cba4a6_0","title":"ISPOREurope23_Macaulay_HPR37_POSTER133542.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133542","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Unmet Needs in Rare Diseases: Insights on Definitions and Methodological Challenges From a Grey Literature Review","id":"08910f8e-3d1c-407e-bac0-f462b139c62c","sessionCode":"HPR13","topDisplay":"Broekmans J1, Vanneste A2, Claessens Z2, Cleemput I3, Maertens de Noordhout C3, Janssens R4, Barbier L2, Huys I4 1Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Tessenderlo, VLI, Belgium, 2Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, VBR, Belgium, 3Belgian Health Care Knowledge Centre (KCE), Brussels, Belgium, 4Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium","locationCode":"3068","description":"\r\n\t<div>OBJECTIVES: This study, part of the Belgian Healthcare Knowledge Centre (KCE) NEED-project, aims to gain insights into (1) definitions used for rare diseases by healthcare stakeholders (regulatory authorities, patient organisations and pharmaceutical industry associations) across Europe, and (2) methodological challenges for identifying and assessing unmet health-related patient and societal needs in rare diseases. Results aim to inform the KCE-NEED Framework for identifying unmet needs in rare diseases and contribute to the Belgian Presidency of the Council of the European Union in 2024. METHODS: A grey literature review was conducted, including a systematic screening of 127 relevant stakeholders' websites and a dedicated search in grey literature databases (Google, Google Scholar, ProQuest). Forward and backward snowballing techniques were employed. Data extraction followed a standardized Excel sheet. RESULTS: Most websites (54%) did not provide a concrete definition of rare diseases, focusing instead on specific aspects or issues without explicitly defining the term. While heterogeneity was found in the particular wording used, most of the definitions were based on the Regulation (EU) on Orphan Medicinal Products. Variations in the availability of rare disease definitions were observed across websites: patient organizations consistently mentioned a definition (80%), whereas industry websites (33%) and regulatory authorities (42%) included a specific definition less frequently. Additionally, this study identified several methodological challenges regarding unmet health-related needs in rare diseases. CONCLUSIONS: This study reveals that many industry and regulatory websites lack clear definitions of rare diseases. When a definition is provided, it generally relies on the Regulation on Orphan Medicinal Products. This study provides insights into rare disease definitions and lays the foundation to address remaining needs in those areas, pointing out methodological research challenges. </div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23broekmanshpr13poster133554-pdf.pdf?sfvrsn=1dde02cb_0","title":"ISPOREurope23_Broekmans_HPR13_POSTER133554.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133554","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Overall Survival of Patients with Lung Cancer Newly Diagnosed in the Period of the COVID-19 Pandemic Compared to Patients with an Incident Diagnosis Before the Pandemic: A Retrospective Analysis of German Claims Data","id":"80e17a04-6681-44dd-a166-f4731ce0d7d0","sessionCode":"PT11","topDisplay":"Krieger J1, Mueller S2, Hahn P3, Fuchs A4 1Cytel, Berlin, BE, Germany, 2Cytel Inc., Berlin, Berlin, Germany, 3Institut für Pharmakoökonomie und Arzneimittellogistik (IPAM), Wismar, Germany, 4AOK PLUS, Dresden, Saxony, Germany","locationCode":"2B","description":"\r\n\t<div>OBJECTIVES: This study aimed to compare the overall survival (OS) of patients with incident lung cancer (LC) diagnosed before and during the COVID-19 pandemic in Germany. METHODS: Using a retrospective, anonymized German claims database, cases with an incident LC diagnosis (=index) were identified during 01/07/2018-30/06/2021. Cases were included if they had at least two confirmed outpatient diagnoses or one inpatient diagnosis of LC (ICD-10-GM code: C34). A case was considered incident if there was a diagnosis-free period of 12 months before above date. Patients were divided into two groups based on their index year (2018/2019 versus 2020/2021) and followed for 12 months or until death (if earlier). OS after incident diagnosis was calculated using Kaplan-Meier estimation and compared between the two groups within a Cox regression model considering main patient characteristics (including metastasis status at index). RESULTS: The analysis included 4,210 incident LC patients, with 2,323 (55.2%) diagnosed in 2018/2019 (mean age: 69.2 years; male: 66.5%; Elixhauser comorbidity score [ECI]: 9.3) and 1,887 (44.8%) in 2020/2021 (mean age: 69.1 years; male: 66.6%; ECI: 8.9). In 2020/2021, a higher percentage of patients were metastasized at diagnosis (38.8% versus 2018/2019: 33.1%; p<0.001). Significantly more patients diagnosed in 2020/2021 died during the 12-month follow-up (50.2% versus 2018/2019: 46.2%; p=0.011). The median OS for patients diagnosed in 2020/2021 was 11.9 months, whereas the median was not reached for patients diagnosed in 2018/2019. Cox regression confirmed the tendency of shorter OS in patients with incident diagnosis in 2020/2021 but without statistical significance (HR: 1.05; p=0.318). CONCLUSIONS: This analysis showed that, generally, fewer cases were diagnosed during the pandemic. However, those that were detected were more likely in an advanced stage, which was reflected in worse OS outcomes. Further research is needed to investigate the pandemic's underlying factors and impact on real-world outcomes in LC patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-2023lung-ca-covid13oct2023poster-final130857-pdf.pdf?sfvrsn=fb569b9c_0","title":"ISPOR EU 2023_LUNG CA COVID_13Oct2023_Poster final130857.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130857","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Companion Diagnostics’ Launch and Market Access Landscape: Who Are the Trend Setters and Late Adopters?","id":"b7619eb6-b7c5-4c06-b99e-f512e4097a2e","sessionCode":"MT6","topDisplay":"Cesarec S1, Genane C2, Norwood-Knutsson C1 1GlobalData, London, LON, UK, 2GlobalData, Le Blanc-Mesnil, France","locationCode":"5032","description":"\r\n\t<div>OBJECTIVES: The companion diagnostics (CDx) market is growing exponentially, driven by increased incidences of cancer and disease awareness. Yet the pricing & reimbursement (P&R), market access and regulatory landscape is unclear and difficult to navigate. It is therefore essential for pharma and medical companies to be informed on market-specific best practices ahead of launch. Our study aims to highlight recent policy developments in most promising or challenging markets. METHODS: Over 40 key domains, including the level of transparency, which greatly contribute to development of launch strategies for innovative therapies alongside CDx, were investigated globally, such as P&R dossiers, funding and coding systems, testing and laboratory frameworks. A screening model was developed with different weighting categories, ranking countries based on key market access factors. RESULTS: Of 15 markets analyzed, only 20% have straightforward CDx P&R policies. Besides the US and France, Israel and Australia ranked amongst top markets for CDx launch, with encouraging P&R submission processes, ahead of other markets such as Germany and Japan. Whilst Germany has well-developed testing and reporting frameworks, it is held back by complex regulations and processes in comparison. Conversely, Canada, China, and Switzerland face the most challenges, with poorly regulated policies and infrastructures. Canada is challenging mostly due to its highly decentralized processes. Although Switzerland’s P&R processes are not amongst the most complex, factors such as high regulatory challenges and lack of transparency contribute to a harsher CDx launch environment. CONCLUSIONS: CDx P&R and regulatory landscapes are not as well-defined compared to pharmaceuticals, creating significant knowledge gaps and unparallel market access settings. Nonetheless, several markets show increasing efforts to improve access conditions. Israel, at the forefront, fosters an overall favorable environment with above-average policies, while Switzerland appears at the opposite end. With exponential market growth and continuously evolving policies, the CDx space requires monitoring to drive market access success.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"dda9555e-19ab-4405-936c-34532e5825c6","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Technologies","urlName":"medical-technologies"}],"categoryIds":["dda9555e-19ab-4405-936c-34532e5825c6"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/sara-cesarec-cdx-landscape-ispor-2023-poster133096-pdf.pdf?sfvrsn=36531a87_0","title":"Sara Cesarec CDx landscape ISPOR 2023 poster133096.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133096","diseases":[{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Change in Healthcare Resource Use and Associated Costs of Patients with Metastatic Lung Cancer between 2013 and 2019: An Observational Study from the French National Claims Database","id":"ded23985-ac59-4031-8327-f56452f8b354","sessionCode":"EE64","topDisplay":"Chouaid C1, Marchal C2, Apert M3, Bensimon L3, Guimard V3, Née M2, Belhassen M2, de Pouvourville G4, Blay JY5 1Service de Pneumologie, Pneumology, Intercommunal Hospital, Créteil, France, 2PELyon, Lyon, France, 3MSD France, Puteaux, 92, France, 4ESSEC Business School, Cergy Pontoise, France, 5Centre Léon Bérard and Université Claude Bernard, Lyon, France","locationCode":"1095","description":"\r\n\t<div>OBJECTIVES: Treatment landscape in metastatic lung cancer is quickly evolving, including targeted therapies and immunotherapies. Both treatments have shown improved survival but are also associated with increased costs. This study aimed to describe the evolution of costs associated with metastatic lung cancer in France. METHODS: From the French national claims database (SNDS), a dynamic cohort of patients identified between 2013 and 2019 with metastatic lung cancer and a marker for the presence of metastases (ICD-10 code or at least one reimbursement for Bevacizumab or Pemetrexed) was constituted. Healthcare resource use was described each year through the percentage of patients with at least one record for each expenditure item. The trend in total mean monthly cost over the study period was studied using Joinpoint software. RESULTS: Between 2013 and 2019, 116 686 patients with a metastatic lung cancer were identified (67.1% of men, median age of 65 years). The percentage of patients with at least one overnight hospitalization decreased from 85.2% in 2013 to 67.6% in 2019 while the use of day hospitalizations remained stable (about 40%). Between 2013 and 2019, the percentage of patients with outpatient care increased: medical visits (from 82.6% to 88.0%), lab tests (from 74.1% to 83.5%), medical procedures (from 72.0% to 83.2%). The total mean monthly cost per patient decreased (from 5,683€ to 4,653€), by 2.85% per year (95%CI: -4.13 to -1.56, p<0.0001). An increase in drugs acquisition costs (from 1,015€ to 1,365€) and a decrease in overnight hospitalizations costs (from 3,110€ to 1,990€) were observed. CONCLUSIONS: This study highlights that the increase in drugs acquisition costs has been offset by a decrease in hospitalization costs, resulting in a decrease in global management costs of patients with metastatic lung cancer in France between 2013 and 2019. These findings may be the result of French health policy and/or improved disease management.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/poster-elo-isporv1-0130463-pdf.pdf?sfvrsn=4c3243dd_0","title":"Poster ELO ISPOR_v1.0130463.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130463","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Post-Discharge Mortality in Patients with Mental Illness Hospitalized Compulsorily in Accordance with the Provisions of Article 29 of the Japanese Mental Health and Welfare Law","id":"a58226f2-186f-4f30-8c38-f65825ee9d60","sessionCode":"CO9","topDisplay":"Inagaki A1, Seto H2, Shimada T3, Otsuka T4, Iwanaga H5, Nakanishi K6, Nakamura H7, Watanabe J8, Kizaki E9, Tomita M9, Yokoshima T10, Okuno E11, Kishi Y12, Ota J13, Yoshizumi A14 1Aoyama Gakuin University, Shibuya, 13, Japan, 2Fukuoka Prefectural Psychiatric Center Dazaifu Hospital, Dazaifu, Fukuoka, Japan, 3Tochigi Prefectural Mental Health and Welfare Center, Utsunomiya, Tochigi, Japan, 4Tohoku University Graduate School of Medicine, Sendai, Miyagi, Japan, 5National Hospital Organization Hizen Psychiatric Medical Center, Yoshinogari, Saga, Japan, 6National Institute of Mental Health, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan, 7Nagasaki Medical Center of Psychiatry, Ohmura, Nagasaki, Japan, 8Inokashira Hospital, Mitaka, Tokyo, Japan, 9Oizumi Hospital, Nerima, Tokyo, Japan, 10Numazu Chuo Hospital, Numazu, Shizuoka, Japan, 11Okinawa Chuo Hospital, Okinawa, Okinawa, Japan, 12Okayama Psychiatric Medical Center, Okayama, Okayama, Japan, 13Mental Health and Welfare Center of Okayama City, Okayama, Okayama, Japan, 14Yahata Kosei Hospital, Kitakyusyu, Fukuoka, Japan","locationCode":"1009","description":"\r\n\t<div>OBJECTIVES: The objective of this study is to examine the post-discharge mortality risk in patients with severe mental illness who were hospitalized compulsorily in accordance with the provisions of Article 29 of the Japanese Mental Health and Welfare Law. METHODS: Since May 15, 2016, a prospective cohort study has been conducted on Article 29 patients admitted to eleven mental hospitals. In this report, the post-discharge three-year mortality risk in Article 29 patients was compared with that of the Japanese general population. RESULTS: The study sample consisted of 146 males and 90 females, with an average age of 41.7 years. The most frequent diagnoses were schizophrenia spectrum disorders (F2; n=114), followed by mood disorders (F3: n=52) and “other mental disorders” (n=70). The deaths of 11 patients were confirmed over a mean follow-up period of 766.8 days. Of these 11 deceased patients, five committed suicide, one died in an accident, and two died of natural causes. No information regarding the cause of death was available for the remaining three patients. The three-year mortality rate, estimated by the Kaplan-Meier method, was 2.8%, while the estimate for the expected three-year mortality rate was 0.772%. Therefore, the standardized mortality risk (SMR) was estimated at 8.04 (95%CI: 4.41-14.3). Subgroup analysis indicated that mortality risks of female patients (SMR: 15.4, 95%CI: 5.70-40.6), those with “other mental disorders” (24.1, 11.8-46.9), those with relatively high social functioning (defined as the Personal and Social Performance Global Score>70) at discharge (13.4, 10.4-25.4) and those who had been hospitalized compulsorily solely due to the risk of self-harm (95.8, 31.0-244) were much higher, respectively. CONCLUSIONS: The findings suggest that being female, having mental disorders other than F2/F3, being hospitalized solely due to the risk of self-harm, and having high social functioning are risk factors for death after discharge from Article 29 admission.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor20231113-7128326-pdf.pdf?sfvrsn=2b7ba9fe_0","title":"ISPOR20231113-7128326.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/128326","diseases":[{"id":"0dfdbf68-3434-4368-ac15-13526254832f","parentId":"00000000-0000-0000-0000-000000000000","title":"Mental Health (including addition)","urlName":"mental-health-including-addition"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Solidifying Usability Testing Guidelines for Ecoas From the Patient Perspective","id":"ef2b30c8-24d7-43ae-90f7-f6b698b45be4","sessionCode":"PCR22","topDisplay":"Hadjidemetriou C1, Poepsel T2 1RWS Life Sciences, Croydon, LON, UK, 2RWS Life Sciences, East Hartford, CT, USA","locationCode":"6006","description":"\r\n\t<div>OBJECTIVES: This paper focuses on the user perspective in usability testing (UT) for eCOAs and provides recommendations and guidelines to ensure that the patient perspective and experience becomes the focal point of the testing. We argue that UT needs to be performed consistently on new eCOA instruments. We emphasize that the interaction between a COA, an eCOA platform’s user interface, and its intended users is complex and requires consistent testing tailored to relevant target populations to improve the usability of the platform and COAs it administers, and provide a positive user experience. METHODS: We present qualitative results from a UT study conducted with 14 cancer patients, split into two groups of 7, each group using a different device (smartphone and tablet). The patients had different conditions due to their cancer diagnosis such as difficulty gripping, reading and concentrating, and tactile challenges. The UT session required participants to complete different tasks using a pre-designed interview script administered by a trained interviewer. RESULTS: Participants completed various activities allowing the interviewer to observe any challenges faced, and answered general and population-specific questions about usability of the platforms. The participants were also encouraged to talk through challenges encountered, and provide their impressions of the eCOA and device. The qualitative analysis of the results informed the development of guidelines and recommendations based on the patients’ experience. CONCLUSIONS: In UT, we determine a participant’s ease of responding a questionnaire electronically and their interaction with the eCOA platform’s user interface. When an eCOA has been designed specifically for electronic administration, UT serves to: assess operational efficiency of the eCOA system; identify design issues causing patient dissatisfaction and hindering data collection; explore software and working improvements; and understand and evaluate user interaction. Full realization of these UT objectives requires a testing process focused on and adapted for specific patient populations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/rwsisporposterusabilitytestingv2134062-pdf.pdf?sfvrsn=c5cf2130_0","title":"RWS_ISPOR__POSTER_USABILITYTESTING_v2134062.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/134062","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Arbitral Awards on Reimbursement Prices Since 2019 in Germany: What We Can Learn?","id":"b811d82b-0c9a-4f3e-ab28-f6e1eba4dca3","sessionCode":"HPR38","topDisplay":"Kiesel L1, Meyer-Acs M2, Esser M1 1co.value, Berlin, Germany, 2co.value, Berlin, BE, Germany","locationCode":"4006","description":"\r\n\t<div>OBJECTIVES: If price negotiations between pharmaceutical companies and the statutory health insurance funds in Germany fail, both parties usually end up before the AMNOG arbitration board. Disagreement usually exists about the reimbursement price. Although each arbitral award is a case-by-case decision, lessons can be derived from each decision. Thus, we aimed to identify recurring decision focal points concerning the reimbursement price which can utilize by companies for future successful negotiations in Germany. METHODS: In our analysis of arbitral awards on reimbursement prices, we focused on identifying recurring influencing factors that were used by the arbitration board to set the price. Furthermore, we evaluated the impact of each factor concerning the final reimbursement price. We only considered decisions made since 2019 under the chairmanship of Prof. Stefan Huster. RESULTS: Since 2019, 80.0% (16/20) arbitral awards have been issued on the reimbursement price: 35.0% (7/20) concerned drugs with additional benefit, 30% (6/20) referred to drugs without additional benefit, and 15.0% (3/20) related to drugs received more than one benefit assessment with various results. Only for drugs with considerable or major additional benefit, we were able to derive decision focal points on recurring influencing factors and their effect on the reimbursement price. We observed that the arbitration board weighted the monetization of the additional benefit at 50–90% in setting the reimbursement price, followed by the European prices, which were weighted at 5–30%, and costs of comparable drugs, which were weighted at 0–35%. CONCLUSIONS: Currently, reimbursement prices are strongly limited by the legislator. However, for drugs with considerable or major additional benefit, the recurring influencing factors should be used in a targeted argumentative manner to increase the possibility for a higher reimbursement price. Thereby, the focus should be especially on the monetization of the additional benefit.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"b3576717-528d-445f-95f1-dcd93dce9460","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Policy & Regulatory","urlName":"health-policy-regulatory"}],"categoryIds":["b3576717-528d-445f-95f1-dcd93dce9460"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kieselhpr38poster132469-pdf.pdf?sfvrsn=19408f6c_0","title":"ISPOREurope23_Kiesel_HPR38_POSTER132469.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132469","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Burden of Patients with Paroxysmal Nocturnal Hemoglobinuria in China","id":"200bce11-fe2f-4ec5-af9d-f74c22bac0ec","sessionCode":"EE98","topDisplay":"Dou L1, Li S2, Che S3 1Shandong University, Jinan, Shandong, China, 2Shandong University, Jinan, China, 3PNH China, Shenzhen, Guangdong, China","locationCode":"2027","description":"\r\n\t<div>OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH and a series of complications have brought heavy economic burden on patients and their families, however, there are few studies on the economic burden of PNH patients in China. This study aimed to evaluate the economic burden of patients with PNH in China. METHODS: The data was conducted by Shandong University in collaboration with the PNH China from May to June 2023, and patients with PNH who were clinically diagnosed for one year or more were included in the survey. Direct medical expenses, direct non-medical expenses and indirect costs were investigated. Descriptive analyses was conducted to analyze the annual average of various costs. RESULTS: A valid sample size of 303 PNH patients was analyzed. The annual direct medical cost per capita was ¥45,779, while the direct non-medical cost per capita was ¥13,628, and the indirect cost per capita was ¥46,738. There was 43.7% of patients were jobless or unemployed, and 93.5% of them were unemployed due to having PNH. The average annual direct medical cost of patients with comorbidities was ¥58,942, which was more than twice that of patients without comorbidities. The average annual out-of-pocket comorbidities related medical costs for patients with combined thrombosis and kidney injury were ¥33,000 and ¥15,900, respectively. 57.8% of patients spent more than 40% of their total annual household income on direct medical care, which means catastrophic household health expenditures were incurred. CONCLUSIONS: The economic burden of disease for Chinese PNH patients is high, especially in terms of hospitalization costs, transportation costs, and indirect costs. The existence of comorbidities is the main reason for the high economic burden of PNH patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23liee98poster131770-pdf.pdf?sfvrsn=965b20ef_0","title":"ISPOREurope23_Li_EE98_POSTER131770.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131770","diseases":[{"id":"38bbb968-9a07-4d26-983e-234f3e629d4f","parentId":"00000000-0000-0000-0000-000000000000","title":"Rare & Orphan Diseases","urlName":"rare-orphan-diseases"},{"id":"33800909-66da-424f-8548-418f4333d28f","parentId":"00000000-0000-0000-0000-000000000000","title":"Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)","urlName":"systemic-disorders-conditions-anesthesia-auto-immune-disorders-n-e-c--hematological-disorders-non-oncologic-pain"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost Utility Analysis of the Flash Glucose Monitoring System in the Management of People With Type 2 Diabetes Mellitus on Basal Insulin Therapy—An Italian Healthcare System Perspective","id":"12d33b2d-0064-4dc2-a06f-f7ddf0e4a517","sessionCode":"EE19","topDisplay":"Mennini FS1, Del Prato S2, Giorgino F3, Poon Y4, Szafranski K5 1Economic Evaluation and HTA (EEHTA CEIS), Department of Economics and Finance, Faculty of Economics, University of Rome “Tor Vergata”, ROMA, RM, Italy, 2Sant’Anna School of Advanced Studies, Pisa, Pisa, Italy, 3University of Bari Aldo Moro, Bari, BA, Italy, 4Abbott, Alameda, CA, USA, 5EVERSANA, Burlington, ON, Canada","locationCode":"1067","description":"\r\n\t<div>OBJECTIVES: Optimal management of glucose is important to people living with Type 2 diabetes mellitus (T2DM) to help reduce disease burden and costly healthcare complications. Glucose monitoring remains an essential component of patient self-management to help reach and maintain targeted glucose and glycated haemoglobin (HbA1c) levels. The FreeStyle Libre system (FSL) was designed to reduce burden of glucose monitoring and is currently reimbursed in Italy for people with T2DM on multiple daily insulin injections, except in Sicily where it is also reimbursed for people on basal insulin only. This analysis was performed to assess cost utility of FSL compared to self-monitoring of blood glucose (SMBG) for people with T2DM on basal insulin, from an Italian healthcare system perspective. METHODS: A patient-level microsimulation model was run using Microsoft Excel for 10,000 patients over a lifetime horizon with 3.0% cost- and 0% utility discounting. Patient characteristics (mean age, 68.1 years; mean HbA1c, 8.7%) were based on Italian population data, randomized controlled trials, and real-world evidence. Annual costs (€ 2023) comprised FSL and SMBG acquisition, complications, and acute diabetic events (ADEs; severe hypoglycaemia and diabetic ketoacidosis). The effect of FSL was modelled as a persistent 0.8% reduction in HbA1c relative to SMBG. Disutilities were applied annually for complications and per event for ADEs. Outcomes were assessed as quality-adjusted life years (QALYs). RESULTS: Total costs were €4,602 higher with FSL than with SMBG (€62,085 vs €57,483). FSL was associated with increase of 0.69 QALYs, generating an incremental cost-effectiveness ratio (ICER) of €6,641/QALY. Scenario analyses ICERs ranged from €2,433/QALY to €25,315/QALY. In probabilistic analysis, FSL was 93% likely to be cost-effective at a willingness-to-pay threshold of €10,000/QALY, and 100% likely at thresholds >€15,000/QALY. CONCLUSIONS: With respect to the Italian healthcare system, FSL is cost effective compared to SMBG for T2DM on basal insulin therapy.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-eu-poster-finaluploaded132206-pdf.pdf?sfvrsn=34cb18c8_0","title":"ISPOR EU Poster Final_uploaded132206.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132206","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Clinical Outcomes of Patients with Relapsed/Refractory Large B-Cell Lymphoma Receiving Second-Line Therapy in England: A Multicenter, Retrospective, Real-World Study","id":"2d626abf-33ca-4df3-a027-f7f2c7084074","sessionCode":"CO15","topDisplay":"Fox CP1, Toron F2, Tyas E3, Cooper M3, Richards J3, Williams P4 1University of Nottingham, Nottingham, Nottinghamshire, UK, 2Bristol Myers Squibb, LONDON, LON, UK, 3Lumanity, Sheffield, DBY, UK, 4Bristol Myers Squibb, Uxbridge, Buckinghamshire, UK","locationCode":"1021","description":"\r\n\t<div>OBJECTIVES: High-dose chemotherapy and autologous stem-cell transplant (HDT-ASCT) is standard second-line consolidation therapy in relapsed or refractory large B-cell lymphoma (R/R LBCL). However, a large proportion of patients are considered ineligible for this treatment. This study examines the impact of ASCT-eligibility on survival outcomes in R/R LBCL. METHODS: This retrospective, observational study included patients with R/R LBCL treated at one of six English referral centers. Patients who met inclusion criteria were stratified by ASCT-eligibility. The ASCT-eligible group comprised both those who had received ASCT, and those who were eligible for but did not receive ASCT. Eligibility was assessed based on second-line regimen received, and predefined physiological parameters validated by clinical experts. The Kaplan-Meier method was used to estimate survival probability. Hazard ratios (HRs) were estimated using a univariate Cox proportional hazards model with ASCT-eligible as reference. RESULTS: In total, 299 patients met eligibility criteria, of whom 223 were ASCT-eligible, 49 ineligible, and 27 unclassified. ASCT-eligible patients were younger than ineligible patients (mean age 56.5 vs. 74.7 years), with lower ECOG scores (60.1% vs. 42.9% with ECOG 0-1). Of the ASCT-eligible group, 98 patients (40.4%) underwent ASCT. Median overall survival was 18.71 months [95% CI: 13.15–33.44] and 6.94 months [95% CI: 4.77–9.11] for ASCT-eligible and ineligible patients respectively, with an HR of 2.39 [95% CI: 1.70–3.37] for death. Median progression-free survival was 6.67 months [95% CI: 4.67–8.71] and 4.27 months [95% CI: 2.40–6.35] respectively, with an HR of 1.54 [95% CI: 1.10–2.15] for progression. Median event-free survival was 6.08 months [95% CI: 3.95–7.99] and 4.27 months [95% CI: 2.40–6.35] respectively, with a HR of 1.46 [95% CI: 1.05–2.04] for an event. CONCLUSIONS: Over half of ASCT-eligible patients did not receive ASCT. Nevertheless, ASCT-eligible patients have significantly better survival outcomes than ineligible patients, highlighting an unmet need in the ASCT-ineligible population.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23foxco13poster129816-pdf.pdf?sfvrsn=aca21966_0","title":"ISPOREurope23_Fox_CO13_POSTER129816.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129816","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Real-World Persistence on Cladribine Tablets of Patients with RMS over 4 Years Based on Claims Data","id":"c8167a63-41e7-4405-ad9e-f8458105640d","sessionCode":"RWD11","topDisplay":"Giesl N1, Stahn D2, Osowski U3 1Merck Healthcare Germany GmbH, Weiterstadt, Germany, an affiliate of Merck KGaA, Weiterstadt, Germany, 2Barmer, Wuppertal, Germany, 3Merck Healthcare Germany GmbH, Weiterstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany","locationCode":"6064","description":"\r\n\t<div>OBJECTIVES: Cladribine has been approved in the EU for the treatment of patients with highly active relapsing multiple sclerosis in 2017. Cladribine tablets are administered during two treatment weeks in year 1 and 2, no additional treatment is required in year 3 and 4. This retrospective analysis aims to assess persistence and switch rates of patients receiving cladribine tablets using real-world claims data. METHODS: The analyses are based on German statutory health insurance records from BARMER, representing 8.9 million insured persons. Adult patients with an MS diagnosis (ICD-10 G35.x) and an initial prescription of cladribine from September 2017 - June 2021 were included. Patients with an exclusive coding PPMS (ICD-10 G35.2), non-active SPMS (ICD-10 G35.30) or non-specific MS (ICD-10 G35.9) were excluded. Prior therapy was recorded within the 12 months before initial prescription of cladribine tablets. RESULTS: 358 patients prescribed cladribine tablets were identified. 57.7% of these received treatment prior to initiating cladribine tablets. The overall persistence rate was 95.5%. 16 patients switched therapy, 4 of those patients switched in their first treatment year, 3/7/2 patients in years 2/3/4 respectively. CONCLUSIONS: More than 95% of incident cladribine patients continued their therapy as directed beyond year 2. Under cladribine, there was a low overall switch rate of 4.5%. In the therapy-free years 3 and 4, only 2.5% of patients required further active therapy for RMS.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"48c6b73a-07ce-409d-95f0-b8ade1a832c8","parentId":"00000000-0000-0000-0000-000000000000","title":"Real-World Data & Information Systems","urlName":"real-world-data-information-systems"}],"categoryIds":["48c6b73a-07ce-409d-95f0-b8ade1a832c8"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23gieslrwd11poster131544-pdf.pdf?sfvrsn=6e4251e8_0","title":"ISPOREurope23_Giesl_RWD11_POSTER131544.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131544","diseases":[{"id":"8e45721c-577e-4445-ba28-7da762aa0e0f","parentId":"00000000-0000-0000-0000-000000000000","title":"Drugs","urlName":"Drugs"},{"id":"e7063f12-1b6c-43a7-bc6e-23a7adf361a4","parentId":"00000000-0000-0000-0000-000000000000","title":"Neurological Disorders","urlName":"Neurological-Disorders"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Building a COA Strategy in Allergic Fungal Rhinosinusitis: Patient Experiences and Clinical Outcome Assessments from the Literature","id":"c21803af-80f1-41ea-b4ce-f8a01a171839","sessionCode":"PCR36","topDisplay":"Klooster B1, Chatterton K2, Ibrahim N1, Bernstein MC1, Shields A1, Allen V3 1Adelphi Values, Boston, MA, USA, 2Adelphi Values, Grafton, MA, USA, 3Sanofi, Bridgewater, NJ, USA","locationCode":"6035","description":"\r\n\t<div>OBJECTIVES: Allergic fungal rhinosinusitis (AFRS), a subtype of chronic rhinosinusitis with nasal polyps (CRSwNP), is a noninvasive fungal form of sinus inflammation. This work documents (1) key disease-related symptoms and impacts that individuals living with AFRS experience, (2) outcomes that are important to consider when developing new treatments, and (3) clinical outcome assessments (COAs) available to measure those outcomes. METHODS: A literature (n=6) and trial/product label (n=5) review was conducted to identify symptoms and impacts associated with AFRS among adult and adolescent patients as well as COAs used to assess these concepts in clinical trials. AFRS concepts identified in the literature were mapped to concepts assessed by selected COAs to determine conceptual coverage of each COA. RESULTS: A total of 18 AFRS-related symptoms and 7 impacts were identified in the 6 articles available for review. Nasal discharge (n=6) and obstruction (n=5) were the most frequently reported symptoms followed by headache (n=4), altered smell (n=3) and post-nasal drip (n=3). The most frequently reported impact of AFRS was having to adopt new behaviors followed by impacts on finances, relationships, sleep, social functioning, and work. A total of six COAs (e.g., Rhinosinusitis Disability Index [RSDI]) were evaluated for conceptual coverage of the concepts identified in the literature. CONCLUSIONS: Individuals with AFRS experience a range of symptoms with nasal discharge and obstruction, headache, altered smell, and post-nasal drip principal among them. Given the significant and varied life-limiting impacts these symptoms exert on patients, results from this research specify several symptom and impact outcomes that, if improved, would reflect benefit for patients living with AFRS. Several COAs emerged as potentially appropriate for use in AFRS trials to support clinical endpoints, however, results also suggest that these assessments individually may not comprehensively target for measurement what patients with AFRS are experiencing particularly among adolescent patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23kloosterpcr-36poster130205-pdf.pdf?sfvrsn=ae2327fc_0","title":"ISPOREurope23_Klooster_PCR 36_POSTER130205.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130205","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"},{"id":"4516d297-cd6a-4f78-8eaa-3ebf6f65ff78","parentId":"00000000-0000-0000-0000-000000000000","title":"Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)","urlName":"respiratory-related-disorders-allergy-asthma-smoking-other-respiratory"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Scientific Communication Plans in Health Economics and Real-World Evidence","id":"2b24f31e-ec27-4332-a924-f9938ee9bb99","sessionCode":"OP4","topDisplay":"Belli K1, Muller Bernz I2, Pereira RG1, Ribeiro A1, Gennaro C1 1IQVIA Solutions, São Paulo, SP, Brazil, 2IQVIA Solutions, São Paulo, Brazil","locationCode":"5078","description":"\r\n\t<div>OBJECTIVES: To review and report reasons to include a scientific communication plans (SCP) in projects of health economics and real-world evidence globally. METHODS: We conducted a narrative literature review to map reasons presented in literature for support implementations of SCP for research projects. We discussed the main reasons founded to support future adoptions of SCP in health economics and outcomes research. RESULTS: The use of SCP is an outline to guide effective communication for various audiences. Some projects are including a roadmap with milestones for sharing their methodologies, results and future steps for different audiences like other scientists, patients, policymakers, funding agencies. The main reasons to encourage an organization of scientific communication plans covered in this review were: collaboration, resource allocation, transparency, reproducibility and dissemination. A SCP allows researchers around the world to identify potential synergies and collaborate effectively in real-world studies, for example. It also permits institutions, stakeholders, policymakers and funding agencies to save and allocate resources more strategically. A SCP allows the scientific community to evaluate details from each study increasing the transparency and reproducibility. A structured SCP can contribute to dissemination of science, contributing to collective understanding of science. It also facilitates future research and inspires new ideas. CONCLUSIONS: The use of SCP has a main objective to expand the reach of science across different areas around the globe, as in health economics and real-world evidence. Including a strategy for SCP inside projects can promote collaboration and improve resource allocation. Also allows transparency, reproducibility and dissemination of science in different areas.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"bb8fd992-de86-4d1a-8bec-f6e2c928db96","parentId":"00000000-0000-0000-0000-000000000000","title":"Organizational Practices","urlName":"organizational-practices"}],"categoryIds":["bb8fd992-de86-4d1a-8bec-f6e2c928db96"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/posterisporop4iqvia130048-pdf.pdf?sfvrsn=58cf16ec_0","title":"Poster_ISPOR_OP4_IQVIA130048.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130048","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"\"I've Got a Text!\" Feedback from Prospective Participants on the Use of SMS Surveys in Real-World Studies","id":"c34d5c52-26f7-4777-bf85-fa6e9800cf30","sessionCode":"SA14","topDisplay":"Lawrence J1, Amini F2, Penduka T3, Llewellyn S1 1Vitaccess, Oxford, OXF, UK, 2Vitaccess, Edinburgh, UK, 3Vitaccess, Oxford, UK","locationCode":"","description":"\r\n\t<div>OBJECTIVES: SMS notifications can be a useful component of digital patient-reported real-world studies to encourage engagement and a more complete dataset, particularly considering that 98% of mobile phone users have been found to open their SMS messages. Less commonly, studies have implemented SMS surveys, such that participants’ responses to a single-question or short SMS survey could trigger the release of a follow-up survey on the study app – in this way, participants who are eligible for the follow-up survey can be identified efficiently and the completeness of the dataset may be improved. The objective of this project was to gain insights into patient preferences on the use of SMS surveys and notifications in longitudinal patient-reported digital real-world studies. METHODS: Participants completed a web-based survey consisting of demographic and multiple option questions eliciting their preferences regarding the use of SMS surveys and notifications in longitudinal digital real-world studies. RESULTS: Twenty-four participants (mean age: 46; range: 27-75) completed the survey, 71% of whom reported living with a chronic health condition. Overall, 71% of participants reported being very likely or somewhat likely to opt-in to receiving SMS surveys asking one question about their, or a dependent’s, health condition. The majority of participants (79%) reported that they were very likely or somewhat likely to subsequently respond to the SMS survey question. If the question triggered a follow-up survey, 63% of participants reported being very likely or somewhat likely to complete it. Once per week and once every few weeks (both 38%) were the most commonly reported frequencies participants would prefer to receive an SMS survey. Half of participants reported an overall positive opinion regarding SMS notifications. CONCLUSIONS: The use of SMS surveys and notifications in longitudinal digital real-world studies was well-accepted by participants and should be considered in future studies to improve engagement and dataset completeness.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d6301e91-40d4-4f22-8c55-9247675547c3","parentId":"00000000-0000-0000-0000-000000000000","title":"Study Approaches","urlName":"study-approaches"}],"categoryIds":["d6301e91-40d4-4f22-8c55-9247675547c3"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/lawrence-et-al-2023isporeu-postersa14smssurveys131823-pdf.pdf?sfvrsn=ebeeeaf3_0","title":"Lawrence et al. (2023)_ISPOR_EU Poster_SA14_SMS_Surveys131823.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131823","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Access to Precision Medicine in Mexico: Differences of Therapy Patterns for ALK+ and EGFR+ Non-Small Cell Lung Cancer (NSCLC) Patients","id":"938ea39a-5d75-4541-851a-fae564153984","sessionCode":"HSD9","topDisplay":"Sanchez L1, Jaimes D2, García-Appendini IC3, Osorio M3, Huerta JL2, Herrera SE2, Nateras V2, Olguin F2, Julian G4 1Pfizer Mexico, MEXICO CITY, MEX, Mexico, 2Pfizer Mexico, Mexico City, Mexico, 3IQVIA, Mexico City, Mexico, 4Pfizer, Sao Paulo, Sao Paulo, Brazil","locationCode":"4022","description":"\r\n\t<div>OBJECTIVES: Lung cancer has emerged as a major health concern in Mexico. Rarely detected in its early stages, 98% of patients are diagnosed with advanced disease, 35% presenting an epidermal growth factor receptor mutation (EGFR+) and 7.6% presenting anaplastic lymphoma kinase rearrangements (ALK+). For patients with these punctual alterations, targeted therapies are recommended in Clinical Practice Guidelines as first-line treatment options; however, Mexico’s scenario may differ due to the fragmented healthcare system, affordability and other barriers that limit patients’ access to treatment. Despite this, there is a lack of studies regarding real-world treatment patterns in Mexico. This real-world study aimed to characterize the therapy patterns for ALK+ and EGFR+ NSCLC patients in both private and public healthcare sectors in Mexico. METHODS: Using the IQVIA-Oncology Dynamics database, which collects cross-sectional anonymized patient-level data, we conducted a descriptive analysis using clinical and treatment data of NSCLC patients treated between 07/2021 and 07/2022 in Mexico. RESULTS: Data from 330 patients were analyzed, with 120 patients (36%) EGFR+ and 31 (9%) ALK+. 110 EGRF+ patients were in 1st line. From these, 33 (30%) were treated in the private sector, all of them with targeted therapy, and 77 (70%) were treated in the public sector with only 3 (4%) of them receiving chemotherapy. For the 30 ALK+ patients in 1st line, 14 (47%) were treated in private sector, all of them with targeted therapy, and 16 (53%) in public sector, with 8 (50%) of them receiving chemotherapy. CONCLUSIONS: Targeted therapies are the standard of care in the private sector whereas the public sector has a high use of targeted therapies for EGFR mutations but a very low use for ALK rearrangements. This inequality in access to innovative therapies for ALK+ patients in the public sector results in a higher out-of-pocket expense for these patients.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"14023d0b-776e-43fa-8552-0cd263b29305","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Service Delivery & Process of Care","urlName":"health-service-delivery-process-of-care"}],"categoryIds":["14023d0b-776e-43fa-8552-0cd263b29305"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-2023nsclcvfin133882-pdf.pdf?sfvrsn=d20b2a3e_0","title":"ISPOR 2023_NSCLC_vfin133882.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133882","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Insulin Analogs in Type 1 Diabetes Treatment: An Incorporation Process Study in Brazilian Unified Health System (SUS)","id":"3adf50f1-eb27-4c89-b9b1-fb8cf144606f","sessionCode":"EPH8","topDisplay":"Costa L1, Caetano R2, Oliveira I1 1Instituto de Medicina Social, Universidade do Estado do Rio de Janeiro, Rio de Janeiro, RJ, Brazil, 2Instituto de Medicina Social, Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brazil","locationCode":"3010","description":"\r\n\t<div>OBJECTIVES: Despite insulin analogs registration being dated since 1997 in Brazil, the availability in Brazilian Unified Health System (SUS) came twenty years later, in 2017. One way to understand this delay in both submission and incorporation is analyzing SUS’ Health Technology Assessment Committee (CONITEC) technical recommendation reports. In this context, this study aims to scan and examine insulin analogs for type 1 diabetes mellitus (T1DM) submissions to CONITEC and its respective reports. METHODS: This exploratory, descriptive, and retrospective research collected demands and recommendation reports of insulin analogs from CONITEC’s website amid 2011-2022. Data related to evaluation process, evidence on safety, efficacy, cost-effectiveness, and budgetary impact available in the reports and arguments set out in the report to justify CONITEC’s recommendations were defined as variables. RESULTS: Eight requests for evaluation of insulin analogues were identified until 2022, but four demands were not analyzed due to different indications for DM1. Four reports were considered, one of which had a non-incorporation decision, two had an incorporation decision and one had a maintenance incorporation recommendation. In relation to efficacy and safety, detemir was evaluated as clinically superior to NPH, reducing HbA1c and number of hypoglycemia episodes significantly. All reports have in common the high budgetary impact of insulin analogs as the main obstacle to incorporation, ranging from 2,7 million to more than 3,7 billion BRL in five years. The incorporation decision for long-acting analogs with cost conditional equal or lower than NPH made in 2019 was revisited in 2022, own to trading session failure in subsequent years after the incorporation. CONCLUSIONS: According to the findings, main difference between analogs and NPH is still related to adherence increase and post-prandial glycemic control. Either way, it is important to follow up actively CONITEC’s decisions, to maintain SUS sustainability.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"01f1d3ca-3d48-408e-a5d0-c99dbbf48b70","parentId":"00000000-0000-0000-0000-000000000000","title":"Epidemiology & Public Health","urlName":"epidemiology-public-health"}],"categoryIds":["01f1d3ca-3d48-408e-a5d0-c99dbbf48b70"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/luizarevione133129-pdf.pdf?sfvrsn=a10bb323_0","title":"Luiza_Rev_ione133129.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133129","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Potential Budget Impact of Negative Pressure Wound Therapy (NPWT) Versus Conventional Wound Treatment (CWT) in Diabetic Foot Ulcers (DFU), Surgical Abdominal Wounds with Healing Impairment (SAWHI), and Trauma Wounds for Mexico","id":"7d1b8a60-6351-4943-9923-fbbcc51ffaee","sessionCode":"EE133","topDisplay":"Campos D1, Vega Hernandez D2, Perez V3, Palka-Santini M4 13M Health Care, Pavas, Costa Rica, 23M Health Care, Ciudad de México, MEX, Mexico, 33M Argentina S.A.C.I.F.I.A., Escobar, B, Argentina, 43M Deutschland GmbH, Neuss, NW, Germany","locationCode":"2063","description":"\r\n\t<div>OBJECTIVES: To estimate the potential budget impact of adopting negative pressure wound therapy (NPWT) vs conventional wound treatment (CWT) for diabetic foot ulcers (DFU), surgical abdominal wounds with healing impairment (SAWHI), and traumatic wounds in Mexico. METHODS: A health economic model calculated the potential budget impact using costs attributed to length of therapy (LoT) and length of stay (LoS) from the perspective of public health care in Mexico, only patients receiving in-hospital care were assessed. The model considered average LoT (NPWT vs CWT) of 14.82 versus 44.57 days (DFU), 17.45 versus 32.76 days (trauma), and 22.8 versus 30.6 days (SAWHl). The average LoS (NPWT vs CWT) was 15.86 versus 29.0 days (DFU), 13.55 versus 20.67 days (trauma), and 13.9 versus 11.8 days (SAWHI), from published literature. The overall wound closure rate for SAWHI patients of 47.8% for NPWT compared to 27.6% for CWT. Local material costs were applied. All calculations were performed in local currency and converted to US dollars. RESULTS: Total cost reduction for 100 patients with DFUs and traumatic wounds using NPWT was $885,512 (44.9%) and $458,778 (32.6%), respectively. In SAWHI patients there was no estimated cost reduction with NPWT, rather an extra cost of -$160,178 (-19.8%). However, when overall wound closure rates are considered, total cost reduction per SAWHI patient with wound closure using NPWT was $160,088 (30.8%). CONCLUSIONS: NPWT use in DFU and trauma is likely to be cost saving for hospital budgets for Mexico. For patients with SAWHI, additional investment is likely to be required, and should be balanced to the benefits obtained. A Cost-Effectiveness analysis is recommended to position the incremental cost in the perspective of the willingness to pay for the improved health outcome.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/2023-ispor-europepotential-budget-impact-of-npwt-vs-cwt-in-dfu-mexico-17oct2023129388-pdf.pdf?sfvrsn=aaa66671_0","title":"2023 ISPOR Europe_Potential budget impact of NPWT vs CWT in DFU MEXICO (17oct2023)129388.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129388","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"3f994852-a0d4-4706-9665-e4d867006d9a","parentId":"00000000-0000-0000-0000-000000000000","title":"Injury & Trauma","urlName":"injury-trauma"},{"id":"bde7079b-7db2-4006-9dbf-54d654e04fc7","parentId":"00000000-0000-0000-0000-000000000000","title":"Surgery","urlName":"Surgery"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Working Together to Produce Better HTA Evaluations. the Elaboration of HTA Guideline for ORT and PRT - a Proposal for the Brazilian Public Health System.","id":"8da1cf53-3df4-4d18-99c5-fbee2016c435","sessionCode":"HTA38","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"4075","description":"\r\n\t<div>HTA studies focused on equipment have particularities. The evaluation of orthoses (ORT) and prostheses (PRT) are even more challenging and tend to present more difficulties when compared to technologies such as medicines. The need for HTA guidelines for the evaluation of non-implantable ORT and PRT has led to collaborative work between an academic research institute (NUTES) — a pioneer in the development of ORT and PRT manufactured by 3D printer— in association with local and regional centers of non-implantable ORT and PRT (beloging to the Brazilian Public health system, the SUS). OBJECTIVES: The main objectives are: a) to evaluate and disseminate the necessary elements to carry out adequate evaluations of non-implantable ORT and PRT; and, b) produce a guideline on HTA evaluation for non-implantable ORT and PRT. METHODS: On-site visits to ORT and PRT centers throughout Brazil were carried out to collect data on the production chain, dispensing, adaptation and evaluation of the patient's clinical results. At the same time, we are conducting systematic literature reviews on two topics: a) elements that must be taken into account in the manufacture, dispensing and adaptation of non-implantable ORT and PRT; and, b) HTA guidelines and/or HTA examples focused on ORT and PRT. RESULTS: Seven out of a total of 12 centers were visited. A scoping review was carried out on body sizing methods for manufacturing non-implantable ORT and PRT. We are now conducting a systematic review of the HTA guidelines and HTA assessment of ORT and PRT— to be completed shortly. CONCLUSIONS: Only the sum of different actors, such as government agencies, academia, health centers, working together can enable the expansion and knowledge of complex areas such as evaluation in HTA of TRO and PRT. However, even with this effort, the challenges are enormous, the gaps are significant, and the work to be done remains enormous.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130320","diseases":[{"id":"3f994852-a0d4-4706-9665-e4d867006d9a","parentId":"00000000-0000-0000-0000-000000000000","title":"Injury & Trauma","urlName":"injury-trauma"},{"id":"9f4dd7f6-8042-4f9a-bc9c-675cf1739ab4","parentId":"00000000-0000-0000-0000-000000000000","title":"Medical Devices","urlName":"Medical-Devices"},{"id":"e0294922-dcb4-4a59-8433-fea4d71449cc","parentId":"00000000-0000-0000-0000-000000000000","title":"Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal)","urlName":"musculoskeletal-disorders-arthritis-bone-disorders-osteoporosis-other-musculoskeletal"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Unleashing the Potential of Artificial Intelligence in Genomic Biomarker Testing for Precision Oncology: A Scoping Review","id":"7e56c2f5-80f8-4af4-8386-fc589fd3dd72","sessionCode":"MSR14","topDisplay":"Buch F1, Madhukumar M2, Nallamothu B3, Chhaya V3, Khambholja K4, Patel D5 1Genpro research pvt ltd, Thiruvananthapuram, Kerala, India, 2Genpro research pvt ltd, Baroda, Gujarat, India, 3Genpro Research Pvt. Ltd., Vadodara, GJ, India, 4Genpro Research Inc, Waltham, MA, USA, 5Genpro research Inc., Vadodara, GJ, India","locationCode":"5055","description":"\r\n\t<div>OBJECTIVES: The research aims to develop strategies to overcome the limitations of manual testing through the implementation of AI technologies in genomic biomarker testing, aiming to enhance the precision and efficiency of oncology diagnostics in the field of precision oncology. METHODS: The method involved literature search through artificial intelligence tool MaiA on PubMed database using search strategy Population, Concept, Context (PCC) criteria. Eligible study designs included clinical trials, observational studies, reviews, systematic literature reviews, and meta-analyses from January 1,2013 till present, excluding treatment and imaging methods. Data charting involved extracting key information such as cancer type, AI tools used, genomic marker type, key findings, and future directions from the selected studies. RESULTS: Among the initial pool of 391 screened articles, 40 full-text articles were reviewed for data charting. The application of AI in analyzing genetic data has demonstrated its potential to enhance diagnostic precision. Our findings reveal the utilization of various AI algorithms to integrate diverse data sources, to generate comprehensive patient profiles and personalized treatment strategies included support vector machines (SVM) for distinguishing melanoma from soft tissue sarcoma, convolutional neural networks (CNN) and Cox regression models in breast cancer detection, and genetic algorithms in bladder, breast, ovarian, liver, colorectal, and leukemia cancers. Challenges included the need for standardized data formats, robust validation methods, and regulatory considerations. Furthermore, ethical and privacy concerns surrounding the use of AI in healthcare necessitate careful attention. AI also aids in analyzing coding variants and establishing genotype-phenotype relationships, further improving genomic testing precision. CONCLUSIONS: The potential of AI in genomic biomarker testing for precision oncology is promising, as it can enhance precision, effectiveness, and personalized treatment options. The advancements in AI are transforming oncology, introducing novel tools for cancer detection, personalized treatment, patient care management, and various other aspects of the field.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"9a8a8cd2-8374-445e-898f-1c074569c9bd","parentId":"00000000-0000-0000-0000-000000000000","title":"Methodological & Statistical Research","urlName":"methodological-statistical-research"}],"categoryIds":["9a8a8cd2-8374-445e-898f-1c074569c9bd"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ai-in-genomic-biomarker-testing-in-precision-oncology133322-pdf.pdf?sfvrsn=5943e350_0","title":"AI in genomic biomarker testing in precision oncology133322.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133322","diseases":[{"id":"4c3286be-524e-4892-8f01-1b369a24d82e","parentId":"00000000-0000-0000-0000-000000000000","title":"Oncology","urlName":"Oncology"},{"id":"7caf4c95-ccab-4b02-bf10-d6baabcc6f2b","parentId":"00000000-0000-0000-0000-000000000000","title":"Personalized & Precision Medicine","urlName":"personalized-precision-medicine"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Economic Evaluation of Medical Devices By the French National Authority (2014-2022): Where Do We Stand?","id":"44ceef6d-4fe0-4d68-8f25-fe5b309b215a","sessionCode":"HTA65","topDisplay":"Midy F1, Sambuc C2, Tehard B1, Chevalier J1, Roze S3 1Vyoo Agency, Paris, 75, France, 2VYOO Agency, Paris, 75, France, 3Vyoo Agency, VILLEURBANNE, 69, France","locationCode":"5005","description":"\r\n\t<div>OBJECTIVES: To describe the cost-effectiveness evaluation of the medical device by the French Health Authority (HAS) issued between 2014 and 2022 and discuss the issues. METHODS: Based on cost-effectiveness analysis, HAS opinions concerning medical devices have been extracted from the Vyoo Agency’s database. The reservations issued by the HAS have been analyzed. RESULTS: Out of the 195 opinions, only 16 were for a medical device, covering 19 indications, mainly in the cardiology area (15/19) and 10 medical devices. The remaining cost-effectiveness opinions concerned drugs (173) and vaccines (6). Evaluations were invalidated due to a major reservation in 38.2% (68/178) of drugs’ indications and 47.4% (9/19) of medical devices’ indications. The uncertainty was targeted as a hurdle for interpreting the RDCR in 12.9% (23/178) of drugs’ indications and 10.5% (2/19) of medical devices’ indications.in 12.9% (23/178) of drugs’ indications and 10.5% (2/19) of medical devices’ indications. All in all, at least one RDCR was interpretable in 52.8% (94/178) of drugs’ indications and 47.4% (9/19) of medical devices’ indications. For medical devices, more than one out of four dossiers got a major reservation concerning the utility assessment (5/16 dossiers concerned). This is the first source of total reservations (20 reservations, 13/16 dossiers), followed by the efficacy and costs assessments (17 reservations each, 10/16 dossiers) and the simulated population (17 reservations, 10/16 dossiers) targeting primarily the analysis of the transposability to the French population. As a comparison, the efficacy assessment was the first source of reservations in the drugs’ opinions (312 reservations, 146/173 dossiers). CONCLUSIONS: No methodological specificities were identified for the economic evaluation of medical devices compared to drugs, except for the evaluation of incremental versions. The principal hurdle to tackle was the utility score assessment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23midyhta-65poster129475-pdf.pdf?sfvrsn=2b0ecfb0_0","title":"ISPOREurope23_Midy_HTA 65_POSTER129475.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/129475","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"A Preliminary Comparison between Analytical Hierarchy Process and Discrete Choice Experiment in Health State Preferences in Hong Kong SAR, China","id":"6b9bd684-0e3c-45f2-9d42-fe84aecf36a5","sessionCode":"PCR49","topDisplay":"Ng CCW, Cheung AWL, Wong E The Chinese University of Hong Kong, Hong Kong, 91, China","locationCode":"6053","description":"\r\n\t<div>OBJECTIVES: The discrete choice experiment(DCE) is utilized in the EQ-5D valuation protocol, which required participants to elicit preferences between hypothetical health states. However, complains were often received as many respondents find the health states presented in the trade-off task unrealistic and difficult to compare. Besides the conventional DCE, an innovative approach Analytical Hierarchy Process(AHP) may allow the respondents to rate the EQ-5D dimensions and levels directly. While comparisons between the AHP and DCE are common in decision-making research, such comparison has not been conducted in EQ-5D health state preferences to our knowledge. The project targeted to conduct a head-on comparison between DCE and AHP in EQ-5D-5L health state preference elicitation. METHODS: A cross-sectional survey was collected in person or via online Zoom meeting in Hong Kong SAR, and participants who aged ≥18 and with Chinese literacy were eligible for the study. Each respondent answered 15 AHP and 14 DCE tasks before collecting their demographics. RESULTS: Between July 2022 and March 2023, 368 cases were recruited. Concerning the ease of understanding the trade-off tasks, 51.4% of participants (n=189) preferred AHP to DCE and 37.0% (n=136) reported vice versa. The relative weighting of EQ-5D dimensions derived from AHP topped at 0.285 by Self-care, followed by Mobility(0.226), Usual Activities(0.187), Pain/Discomfort(0.175) and Anxiety/Depression(0.126). An identical order was reported by participants’ direct ranking exercise. Expanding the comparison to EQ-5D-5L, the Pearson Correlation coefficient and Spearman Rank Correlation coefficient between the utility levels derived from AHP and DCE were 0.816 and 0.880 respectively, hinting a strong agreement and monotonic relationship between the two health state preference elicitation methods. CONCLUSIONS: AHP had demonstrated its ability to report comparable findings to both patient-reported ranking and DCE-derived outcome. As participants often find AHP easier to understand, AHP could be an alternative with lower cognitive burden and applicable to cognitive-impaired community in preference elicitations.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"d72a145a-f216-45d6-a2d2-af65d912c83d","parentId":"00000000-0000-0000-0000-000000000000","title":"Patient-Centered Research","urlName":"patient-centered-research"}],"categoryIds":["d72a145a-f216-45d6-a2d2-af65d912c83d"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/ispor-poster-europe-2023ahp-dce-comparison130391-pdf.pdf?sfvrsn=e62087b1_0","title":"ISPOR Poster Europe 2023_AHP DCE Comparison130391.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/130391","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Literature Review of Fasting Glucose Management's Value for Diabetic Retinopathy","id":"7a0ed8f8-24dd-4c24-b29c-fedc5cece805","sessionCode":"CO5","topDisplay":"ABSTRACT WITHDRAWN","locationCode":"1006","description":"\r\n\t<div>OBJECTIVES: Previously, it was believed that Diabetic retinopathy (DR) was associated with HbA1c levels. In recent years, increasing evidence suggests that fasting plasma glucose（FPG） variability is a key factor influencing DR.The aim is to perform a literature review to elucidate the impact of fasting plasma glucose and its variability on the occurrence and progression of DR. METHODS: A systematic search and review of the PubMed literature database was conducted to evaluate the relationship between FPG and its variability with DR in patients with type 2 diabetes mellitus (T2DM). RESULTS: Multiple studies assessed the correlation between FPG variability and DR. It was found that the cumulative incidence of DR in T2DM patients without DR after a 5.2-year follow-up was 36.2%. A 33-year follow-up study demonstrated that the risk of retinopathy significantly increased when the average FPG during the initial 2 years of follow-up ranged from 6.9 to 7.4 mmol/L, and the risk escalated sharply when it ranged from 7.4 to 8.7 mmol/L. FPG standard deviation and coefficient of variation were independent risk factors for DR, as revealed by multivariate analysis. Another 8-year follow-up study found that both the standard deviation and coefficient of variation of FPG were associated with the development of proliferative diabetic retinopathy (PDR) (standard deviation: HR = 1.011, 95% confidence interval 1.003-1.018; coefficient of variation: HR = 6.858, 95% confidence interval 2.317-20.304), independent of HbA1c. CONCLUSIONS: In patients with T2DM, higher levels of standard deviation and variability of FPG are positively correlated with DR. Therefore long-term stability of fasting plasma glucose is crucial for reducing the occurrence and progression of DR.</div>\r\n\r\n","withdrawn":true,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/literature-review-of-fasting-glucose-managements-value-for-diabetic-retinopathy132448-pdf.pdf?sfvrsn=865d904b_0","title":"Literature Review of Fasting Glucose Management's Value for Diabetic Retinopathy132448.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/132448","diseases":[{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Effectiveness of Tocilizumab in Refractory Graves' Orbitopathy in a Real-World Setting","id":"49dad593-1cda-4011-bb0d-ff179fe2467b","sessionCode":"CO148","topDisplay":"Sandoval-Fernandez-del-Castillo S, Valera-Rubio M, Moya-Martín ML, Carrión-Madroñal IM, Garrido-Hermosilla AM, Méndez-Muros M Virgen Macarena University Hospital, Sevilla, Spain","locationCode":"1022","description":"\r\n\t<div>OBJECTIVES: To assess the effectiveness of the antiinterleukin-6 receptor monoclonal antibody tocilizumab in patients with moderate-to-severe Graves' orbitopathy(GO) in clinical practice. METHODS: A retrospective observational study of a series of patients with moderate-severe GO on subcutaneous tocilizumab treatment 162mg once a week was performed. Medical records of patients who received tocilizumab between September 2015 and May 2023 were reviewed. Effectiveness was assessed by CAS(Clinical Activity Score) reduction before and after treatment. A reduction of CAS ≥2 points together with obtaining inactivity(CAS < 3) was considered a favourable response. Measurements of proptosis and best-corrected visual acuity(BCVA) were also used to evaluate improvement in GO. RESULTS: 22 patients were treated with tocilizumab during the study period, 20 women and 2 men, with a mean age of 54 years. 8(36,4%) patients were active smokers, 3(13,6%) ex-smokers and 11(50%) non-smokers. Patients received tocilizumab for a mean of 44,6 weeks (range 4-83 weeks). Of 22 patients, 4(18.2%) are currently on treatment, 4(18,2%) dropped out(one patient due to adverse event) and 14(63,6%) completed treatment until remission. Among those who finished treatment, the median duration was 54,6 weeks(39-83). The mean initial CAS was 4,6/7 and the final CAS was 1,7/10. The CAS score was reduced by a mean of 2,8 points. 21 patients achieved favourable response:2/4 who continued, 3/4 patients who dropped out and 13/14 who finished. Furthermore, 40,5% of treated eyes achieved CAS ≤1(none of the smokers reached it). The mean initial BCVA was 0,76 and the final BCVA was 0,89. Initial exophthalmos mean size was 22,66mm and it was 21,65mm after treatment. CONCLUSIONS: The efficacy of tocilizumab is excellent in terms of rapid onset of action and ability to reduce severity in GO. Our results are similar to the outcomes of other published studies so it represents a great alternative in moderate-severe patients even in short courses of treatment.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"87546aa9-e592-49a6-ba7f-a9e9daa90133","parentId":"00000000-0000-0000-0000-000000000000","title":"Clinical Outcomes","urlName":"clinical-outcomes"}],"categoryIds":["87546aa9-e592-49a6-ba7f-a9e9daa90133"],"relatedPdfs":[],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133420","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & Biosimilars","urlName":"biologics-biosimilars"},{"id":"8da22847-bf2c-4e2d-9e3e-8f42192004ef","parentId":"00000000-0000-0000-0000-000000000000","title":"Diabetes/Endocrine/Metabolic Disorders (including obesity)","urlName":"diabetes-endocrine-metabolic-disorders-including-obesity"},{"id":"e29b1bc3-a98e-49c1-8bc2-2975bb5fac5e","parentId":"00000000-0000-0000-0000-000000000000","title":"Sensory System Disorders (Ear, Eye, Dental, Skin)","urlName":"sensory-system-disorders-ear-eye-dental-skin"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Social and Economic Burden of Hepatitis C in Russia","id":"fa9fdcc3-70e5-4f91-9db4-ff3ca1edc4eb","sessionCode":"EE35","topDisplay":"Avxentyev NA1, Avxentyeva M2, Makarov A3, Makarova Y4 1Financial Research Institute and Russian Presidential Academy of National Economy and Public Administration, Moscow, Russia. Pharmaceutical Analytics Middle East, Ras al Khaimah, United Arab Emirates, 2Financial Research Institute and Sechenov First Moscow State Medical University (Sechenov University), Moscow, Moscow, Russian Federation, 3Pharmaceutical Analytics Middle East, Ras al Khaimah, Ras al Khaimah, United Arab Emirates, 4Financial Research Institute, Moscow, MOW, Russia","locationCode":"1074","description":"\r\n\t<div>OBJECTIVES: In Russia, approximately 700,000 people have confirmed active hepatitis C virus (HCV) and are registered by the healthcare facilities. However, the real prevalence of HCV, including hidden, may be up to 2.3 million. The objective of this study is to evaluate the social and economic burden of HCV. METHODS: The social burden was assessed by examining excess mortality (including working-age adults) and decrease in life expectancy at birth. These indicators were estimated under a hypothetical scenario of no HCV and compared with the actual values in 2020. The economic burden included costs associated with HCV screening, diagnosis, and treatment. Additionally, direct non-medical costs such as disability benefits, and indirect costs, including the loss of GDP due to premature mortality and disability, were considered. All indicators were estimated for 2020 without considering future costs, and human capital theory was employed to estimate the indirect costs. RESULTS: In 2020, HCV resulted in 16.9 thousand excess deaths that reduced life expectancy at birth by 0.18 years. The economic burden of HCV was US$ 816.3 million. Mainly it was attributed to indirect economic costs of production losses - US$ 601.9 million (74% of the overall burden). Direct medical costs were US$ 179.4 million or 22% of the overall burden, including direct-acting antiviral drugs (DAAs) procurements (US$ 103.7 million), screening (US$ 44.7 million), and medical treatment of cirrhosis and liver cancer (US$ 29.5 million). Disability benefits (US$ 35.0 million) were the least component of the overall burden. The limited use of DAAs explains the high proportion of indirect costs: in 2020, only a small percentage of patients in Russia, 0.9% of those registered, received these medications. CONCLUSIONS: The social and economic burden of HCV in Russia is considerable and requires implementing new effective control measures.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/hcv-burdenisporposter-2023-11-081133753-pdf.pdf?sfvrsn=47940ded_0","title":"HCV burden_ISPOR_poster 2023 11 08_1133753.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133753","diseases":[{"id":"51dd65bf-b41e-4681-b7ab-844cd26e5fe6","parentId":"00000000-0000-0000-0000-000000000000","title":"Infectious Disease (non-vaccine)","urlName":"Infectious-Disease--non-vaccine-"},{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Proportionally Quicker or Easier? International Comparison of NICE’s Proportionate Approach to Technology Appraisals (PATT) With Global Peers","id":"c4774b19-9788-417b-9b34-ff8043c3ef87","sessionCode":"HTA53","topDisplay":"Kelly KR1, Thompson R1, Leach N2 1Red Thread Market Access, Oxford, UK, 2Red Thread Market Access, Oxford, OXF, UK","locationCode":"5014","description":"\r\n\t<div>OBJECTIVES: In 2022, the National Institute for Health and Care Excellence (NICE) introduced its proportionate approach to technology appraisals (PATT) to increase capacity and allow more rapid guidance development. We sought to evaluate initial scheme success by comparing PATT assessments with other reimbursement or regulatory agencies. METHODS: Drugs that had undergone PATT were identified via the NICE website. Appraisal timings and reimbursement restrictions were compared with Haute Autorité de santé, Institute for Quality and Efficiency in Health Care (IQWiG), Federal Joint Committee (G-BA), Food and Drug Administration (FDA), Pharmaceutical Benefits Advisory Committee (PBAC) and Canada’s Drug and Health Technology Agency (CADTH). Appraisal timings were estimated from published documentation. RESULTS: As of June 2023, five drugs had undergone PATT, and 31 published HTA or regulatory assessments were available (n=7 for somatrogon, nintedanib and eptinezumab, n=6 for vutrisiran and n=4 for nivolumab). For three drugs, NICE had neither the shortest appraisal time, nor the longest; appraisal lengths typically fell towards the shorter end of the range vs. peers: somatrogon (23 weeks [range: 12-37 weeks]), vutrisiran (18 weeks [range: 12-60 weeks]) and eptinezumab (31 weeks [range: 12-57 weeks]). PATT took 31 weeks for nivolumab, slower than the other two assessing agencies: FDA (11 weeks) and CADTH (26 weeks). Nintedanib assessment was also slower than international peers (35 weeks [range: 13-26 weeks]). NICE completed its assessment of somatrogon ahead of the FDA, of eptinezumab ahead of G-BA, vutrisiran ahead of G-BA and CADTH and nivolumab ahead of PBAC and CADTH. Nintedanib had already been approved by all other agencies prior to NICE assessment. CONCLUSIONS: While only five drugs have been assessed under PATT, early analysis shows a positive impact on appraisal length compared with NICE’s standard route. However, while review times have been shortened, calendar approval dates are mixed compared with international peers.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73","parentId":"00000000-0000-0000-0000-000000000000","title":"Health Technology Assessment","urlName":"health-technology-assessment"}],"categoryIds":["6f5ef72f-8f2d-4f1e-8746-b96cc7c07b73"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23leachhta53poster133449-pdf.pdf?sfvrsn=8ccb97d5_0","title":"ISPOREurope23_Leach_HTA53_POSTER133449.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/133449","diseases":[{"id":"e8a428f2-769e-4a1a-9cd7-c8bf0bdf664d","parentId":"00000000-0000-0000-0000-000000000000","title":"No Additional Disease & Conditions/Specialized Treatment Areas","urlName":"no-additional-disease-conditions-specialized-treatment-areas"}],"viewingMethods":[],"shouldShowPdfs":true},{"title":"Cost-Effectiveness of Second-Line Treatment for Advanced Endometrial Carcinoma in Taiwan: Lenvatinib Plus Pembrolizumab Versus Doxorubicin","id":"1fb2891c-9457-4322-a8fe-ff94939a369d","sessionCode":"EE68","topDisplay":"Chiang ST1, Chueh CH2, Ho PK3, Wen YW4, Shiu MN3, Liu JH5, Li WH5, Tsai YW3 1National Yang Ming Chiao Tung University, Taipei City, Taiwan, 2National Yang Ming Chiao Tung University, Taipei city, Taiwan, 3National Yang Ming Chiao Tung University, Taipei, Taiwan, 4Chang Gung University, Taoyuan, Taiwan, 5Cheng Hsin General Hospital, Taipei, Taiwan","locationCode":"1046","description":"\r\n\t<div>OBJECTIVES: This study evaluates the cost-effectiveness of the lenvatinib and pembrolizumab (LP) regimen as a second-line treatment for advanced or recurrent endometrial carcinoma (EC) under the context of Taiwan's National Health Insurance (NHI), as currently, the NHI has not covered LP for this indication, despite its recommendation by the European Society for Medical Oncology. Additionally, the study aims to propose a reimbursement price that addresses efficiency concerns associated with this treatment option. METHODS: A partitioned survival model with three health states and a 20-year time horizon analyzed a hypothetical advanced EC patient population using KEYNOTE-775 trial efficacy results. A piecewise method was employed to extrapolate the overall and progression-free survival curves. The pricing of LP was based on the NHI fee schedule for other cancers, while the direct medical costs were estimated from NHI claim data. Health state utilities and disutilities related to adverse events were obtained from the literature. The willingness-to-pay (WTP) threshold was set at three times the GDP per capita in 2022 (NT$2,925,582). Quality-adjusted life-years (QALYs) and costs were discounted at 3%. Scenario analyses included a 10% price reduction for LP and an extended time horizon. Deterministic and probabilistic sensitivity analyses were performed to assess the uncertainty. RESULTS: The LP regimen provided an incremental gain of 0.98 QALYs at incremental costs of NT$2,949,305, resulting in an incremental cost-effectiveness ratio of NT$3,009,494, slightly exceeding the WTP and with an acceptable possibility of 48.4%. The scenario analysis considering a 10% price reduction for LP showed a 67% probability of the regimen being cost-effective. Additionally, extending the time horizon to 40 years resulted in a 53% probability of LP being cost-effective. CONCLUSIONS: Although the LP regimen was not cost-effective at the current price schedule, both the 10% LP price reduction and time horizon extension scenario analyses produced acceptable cost-effectiveness results.</div>\r\n\r\n","withdrawn":false,"categories":[{"id":"63ee603c-f5ab-4644-b76c-0d8299764d84","parentId":"00000000-0000-0000-0000-000000000000","title":"Economic Evaluation","urlName":"economic-evaluation"}],"categoryIds":["63ee603c-f5ab-4644-b76c-0d8299764d84"],"relatedPdfs":[{"url":"https://www.ispor.org/docs/default-source/euro2023/isporeurope23chiangee68poster131986-pdf.pdf?sfvrsn=8c3f7c58_0","title":"ISPOREurope23_Chiang_EE68_POSTER131986.pdf"}],"detailUrl":"https://www.ispor.org/heor-resources/presentations-database/presentation/euro2023-3784/131986","diseases":[{"id":"bd738b2c-6ed3-4dcb-a254-49809b6311af","parentId":"00000000-0000-0000-0000-000000000000","title":"Biologics & 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