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masthead--video alignfull" aria-label="Banner">  <div class="masthead__media"> <video autoplay muted loop poster="https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb.jpg"> <source src="https://www.ppd.com/wp-content/uploads/2022/08/1057544563-hd.mp4" type="video/mp4"> </video> <picture> <img src='https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb.jpg' width='1000' height='563' srcset='https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb.jpg 1000w, https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb-300x169.jpg 300w, https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb-768x432.jpg 768w, https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb-60x34.jpg 60w, https://www.ppd.com/wp-content/uploads/2022/09/kid-hugging-video-thumb-995x560.jpg 995w' alt='kid with rare disease hugging dad' class='object-pos-center' /> </picture> </div> <div class="masthead__overlay"> </div>  <div class="masthead__content-wrapper alignwide"> <div class="masthead__content"> <h1 class="masthead__title">Rare Disease CRO Services</h1> </div> </div> </section> <div class="breadcrumbs alignfull"><div class="breadcrumbs__inner"><div class="breadcrumbs__wrapper"><div class="breadcrumbs"><span><span><a href="https://www.ppd.com/therapeutic-expertise/">Therapeutic Expertise</a></span> <span class="breadcrumb-separator"></span> <span class="breadcrumb_last" aria-current="page">Rare Disease CRO Services</span></span></div></div><span class="breadcrumb-separator"></span><div class="breadcrumbs__dropdown"><span class="dropdown-trigger">Select a Topic</span><ul class="dropdown-menu"><li><a href="https://www.ppd.com/therapeutic-expertise/rare-disease/neuromuscular-disorders/">Neuromuscular Disorder Studies</a></li></ul></div></div></div> <div class="wp-block-emagine-columns alignfull" id="alleviating-burdens"><div id="em-layout-id_a87c9d-23" class="em-row-layout-inner em-margin-top--default em-margin-bottom--default em-tablet-margin-top--default em-tablet-margin-bottom--default em-mobile-margin-top--none em-mobile-margin-bottom--default em-layout-id_a87c9d-23"><div class="em-row-column-wrap em-has-2-columns em-gutter-default em-row-valign-top em-row-layout-left-golden em-tab-layout-left-golden em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--default em-padding-bottom--default em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--default em-mobile-padding-bottom--default alignwide"> <div class="wp-block-emagine-column emagine-column513 inner-column-1 rare-disease-intro"><div class="em-inside-inner-col em-padding-top--none em-padding-right--medium em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--medium em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--medium em-mobile-padding-right--none em-mobile-padding-bottom--medium em-mobile-padding-left--none"> <h2 class="wp-block-heading">Alleviating the burdens of rare diseases</h2> <p>There are no U.S. Food and Drug Administration (FDA)-approved treatments for 95% of rare disorders, creating an immeasurable physical and mental toll on patients and their families. Whether performing clinical trial therapeutic research for adult or pediatric rare diseases, the PPD™ clinical research business of Thermo Fisher Scientific recognizes the person behind the condition.</p> <p>Patients are at the heart of our rare disease contract research organization (CRO) services. By incorporating the experiences of trial participants, their caregivers and investigators, we enable a patient-centric approach for our clients’ transformational therapies and for positive change in the lives of people with rare diseases.</p> <p>Rare disease development is a priority for many drug developers, but they face persistent challenges when trying to advance treatments for these often-complicated disorders. These barriers to rare disease treatment and orphan drug development include:</p> <ul class="wp-block-list"> <li>A lack of consensus on endpoints</li> <li>Inexperienced, understaffed, or unavailable research sites</li> <li>A shortage of regulatory precedents</li> <li>Geographically dispersed patient populations</li> <li>Finding and retaining enough patients to participate in a rare disease clinical trial</li> </ul> <p>We enable clients to navigate these difficulties and overcome obstacles that arise in the clinical development, testing and regulatory approval of treatments for rare diseases.</p> <div style="height:1rem" aria-hidden="true" class="wp-block-spacer"></div> <div class="wp-block-emagine-button"><a class="wp-block-emagine-button__more has-purple-background-color block-btn block-btn--sm block-btn--primary" style="background-color:#633a97" href="#connectwithus" rel=""><span class="block-btn__hover" style="color:#fff;background-color:#4f2683;border-color:transparent">Connect with our rare disease experts </span></a></div> </div></div> <div class="wp-block-emagine-column emagine-column1722 inner-column-2"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><section id="cta-block-block_549ee64cf43d84de894e9f2deb848f03" class="cta-block aligndefault has-blue-background-color" > <div class="inner aligndefault"> <div class="cta-block__body"> <div class="cta-block__content"><p class="h4" style="color:#fff">Enabling Successful Rare Disease Clinical Trials</p> </div> </div> <div class="cta-block__button"> <a class="cta__button " href="/therapeutic-expertise/rare-disease/successful-rare-disease-trials-ebook/" target="">Download the e-book</a> </div> </div> </section> <div id="card-test" style="height:25px;"></div> <section id="anchor-link-menu-block_b004a76772a093db86b2a5903a7bd4bb" class="anchor-link-menu alignfull"> <div class="anchor-link-menu__title"> On this page </div> <div class="anchor-link-menu__links"> <ul> <li><a href="#committed-to-rare-disease-research">Committed to Rare Disease Research</a></li> <li><a href="#ppd-rare-disease-clinical-trial-services">Rare Disease Clinical Trial Services</a></li> <li><a href="#draw-on-global-rare-disease-study-experience">Draw on Global Rare Disease Study Experience</a></li> <li><a href="#rare-disease-trials">Rare Disease Trials</a></li> <li><a href="#recruit-rare-disease-clinical-trial-patients">Recruit Rare Disease Clinical Trial Patients</a></li> <li><a href="#make-the-right-decisions-earlier">Make the Right Decisions Earlier</a></li> <li><a href="#rare-disease-and-pediatrics-center-of-excellence">Rare Disease and Pediatrics Center Of Excellence</a></li> <li><a href="#rare-disease-expertise-across-therapeutic-areas">Rare Disease Expertise across Therapeutic Areas</a></li> <li><a href="#ppd-heroes-in-rare-disease">Our Heroes in Rare Disease</a></li> <li><a href="#related-resources">Related Resources</a></li> </ul> </div> </section> </div></div> </div></div></div> <div style="height:0rem" aria-hidden="true" id="committed-to-rare-disease-research" class="wp-block-spacer"></div> <section id="video-block-block_c1b48a39c4a30b0e420122d1523b2e1c" class="video-block alignfull video-block--no-title has-blue-l-background-color" style="background-image: url(https://www.ppd.com/wp-content/uploads/2019/11/hex_2.png);"> <div class="video-block__content alignwide"> <h3 class="h2" style="padding-top:6.25rem">Committed to rare disease research</h3> <p>Of all the rare diseases that have been identified globally, only 5% have an approved treatment or therapy. This number inspires us to drive research forward and to develop new rare disease therapies that change lives. Through our Rare Disease and Pediatric Center of Excellence, wide-ranging study experience and specialized rare disease trial framework, we deliver innovative solutions to overcome the challenges associated with orphan drug development.</p> </div> <div class="video-block__video"> <div class="aligndefault"> <div style="position: relative; display: block; max-width: 960px;"><div style="padding-top: 56.25%;"><iframe src=https://players.brightcove.net/2202959166001/CHiz2H2j_default/index.html?videoId=5739181607001 allowfullscreen="" allow="encrypted-media" style="position: absolute; top: 0px; right: 0px; bottom: 0px; left: 0px; width: 100%; height: 100%;border:0;" loading="lazy"></iframe></div></div></div> </div> </section> <div style="height:6.5rem" aria-hidden="true" class="wp-block-spacer"></div> <section id="ppd-rare-disease-clinical-trial-services" class="media-content alignfull has-bg-image dark-text thirds-layout align-content-center " style=""> <div class="alignwide align-bg"> <div class="media-content__image bg-image" style="background-image: url(https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-shutterstock_1633124830.jpg);"></div> <div class="media-content__content has-container right-content"> <div class="content-container"> <h2 class="media-content__title2">Rare disease clinical trial services</h2> <div class="media-content__desc"><p>Patients and the drug developers working to fight rare disorders face a number of challenges. The PPD clinical research business of Thermo Fisher Scientific designs and conducts rare disease studies that solve for those challenges. Our focus is to <a href="/blog/reducing-patient-burden-and-trial-complexity/">decrease patient burden</a> and help them and their caregivers navigate a complex health care system.</p> <p>Our patient services build on long-standing partnerships in the rare disease community to provide innovative solutions for increasing access to clinical trial participation and patient retention. These services include:</p> <ul> <li>Booking and coordinating patient transportation to and from study sites, providing convenience and travel expense verification for patients</li> <li>Offering flexible patient reimbursement options in more than 150 currencies</li> <li>Connecting patients to options for home health care, including remote staff partnerships</li> </ul> </div> </div> </div> </div> </section> <div class="wp-block-emagine-columns alignfull" id="draw-on-global-rare-disease-study-experience"><div id="em-layout-id_618d97-00" class="em-row-layout-inner em-margin-top--none em-margin-bottom--none em-tablet-margin-top--none em-tablet-margin-bottom--none em-mobile-margin-top--none em-mobile-margin-bottom--none em-row-has-bg em-layout-id_618d97-00"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--xlarge em-padding-bottom--none em-tablet-padding-top--xlarge em-tablet-padding-bottom--none em-mobile-padding-top--xlarge em-mobile-padding-bottom--none alignwide"> <div class="wp-block-emagine-column emagine-column1138 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"> <h2 class="wp-block-heading has-text-align-center">Draw on global rare disease study experience</h2> <p class="has-text-align-center">In the past five years, we’ve partnered with rare disease developers across <a href="/our-solutions/clinical/phase-ii-iv-clinical-trial-management/">all phases</a>, indications and therapeutic areas.</p> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull"><div id="em-layout-id_d3b15d-2a" class="em-row-layout-inner em-margin-top--none em-margin-bottom--none em-tablet-margin-top--none em-tablet-margin-bottom--none em-mobile-margin-top--none em-mobile-margin-bottom--none em-row-has-bg em-layout-id_d3b15d-2a"><div class="em-row-column-wrap em-has-4-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-two-grid em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--none em-padding-bottom--xlarge em-tablet-padding-top--none em-tablet-padding-bottom--xlarge em-mobile-padding-top--default em-mobile-padding-bottom--default alignwide"> <div class="wp-block-emagine-column emagine-column4240 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><div id="countup-block-block_3bff7e9f3b57b457a9985a30145fabb4" class="countup-block alignfull"> <div class="countup-block__wrapper purple"> <div class="countup-block__hexagon"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2020/01/icon-pediatrics.svg" alt=""> </div> <div class="countup-block__content"> <span class="countup-block__number" data-number="600">0</span> <span class="countup-block__before">+</span> <div class="countup-block__label"><p>Rare disease studies</p> </div> </div> </div> </div> </div></div> <div class="wp-block-emagine-column emagine-column9295 inner-column-2"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><div id="countup-block-block_e54e91f83b3bab49c1f2bbcf5cf8b779" class="countup-block alignfull"> <div class="countup-block__wrapper purple"> <div class="countup-block__hexagon"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2021/08/icon-gmp-building.svg" alt=""> </div> <div class="countup-block__content"> <span class="countup-block__number" data-number="17800">0</span> <div class="countup-block__label"><p>Sites</p> </div> </div> </div> </div> </div></div> <div class="wp-block-emagine-column emagine-column4929 inner-column-3"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><div id="countup-block-block_b55bb1d3875668fec2dd4acb60ae2033" class="countup-block alignfull"> <div class="countup-block__wrapper purple"> <div class="countup-block__hexagon"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2021/01/icon-north-America-60074e8e337f9.svg" alt=""> </div> <div class="countup-block__content"> <span class="countup-block__number" data-number="101">0</span> <div class="countup-block__label"><p>Countries</p> </div> </div> </div> </div> </div></div> <div class="wp-block-emagine-column emagine-column7603 inner-column-4"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><div id="countup-block-block_a0bb6c59c7e3a2ecaa75610f862a67db" class="countup-block alignfull"> <div class="countup-block__wrapper purple"> <div class="countup-block__hexagon"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2020/10/icon-group-people.svg" alt=""> </div> <div class="countup-block__content"> <span class="countup-block__number" data-number="106500">0</span> <div class="countup-block__label"><p>Global patients</p> </div> </div> </div> </div> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull" id="rare-disease-trials"><div id="em-layout-id_55f220-bc" class="em-row-layout-inner em-margin-top--default em-margin-bottom--default em-tablet-margin-top--default em-tablet-margin-bottom--default em-mobile-margin-top--default em-mobile-margin-bottom--default em-layout-id_55f220-bc"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--default em-padding-bottom--default em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--default em-mobile-padding-bottom--default aligndefault"> <div class="wp-block-emagine-column emagine-column3746 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"> <h2 class="wp-block-heading">Rare disease trials</h2> <p>To enable you to create and test your rare disease therapy, our team creates an unmatched, customer approach for deep understanding of the diagnostic journey, care landscape and patient experience.</p> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull commercial-break-cta"><div id="em-layout-id_838f8e-63" class="em-row-layout-inner em-margin-top--none em-margin-bottom--none em-tablet-margin-top--none em-tablet-margin-bottom--none em-mobile-margin-top--none em-mobile-margin-bottom--none em-layout-id_838f8e-63"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--default em-padding-bottom--default em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--default em-mobile-padding-bottom--default alignfull"> <div class="wp-block-emagine-column emagine-column3844 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><section id="cta-block-block_3440b4135f106b9b7904dd239ed15cd6" class="cta-block alignfull horizontal has-blue-background-color" > <div class="inner aligndefault"> <div class="cta-block__body"> <h2 class="cta-block__title">Understanding the patient journey webinar</h2> <div class="cta-block__content"><p>Gain insights into the challenges of accessing global patient populations, manufacturing, regulations and patient-centered protocol design in rare disease clinical trials.</p> </div> </div> <div class="cta-block__button"> <a class="cta__button " href="/therapeutic-expertise/rare-disease/rare-disease-trials-and-gene-therapy-webinar-on-demand/" target="">Watch the webinar on demand</a> </div> </div> </section> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull" id="recruit-rare-disease-clinical-trial-patients"><div id="em-layout-id_354e4a-20" class="em-row-layout-inner em-margin-top--default em-margin-bottom--small em-tablet-margin-top--default em-tablet-margin-bottom--default em-mobile-margin-top--default em-mobile-margin-bottom--default em-layout-id_354e4a-20"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--default em-padding-bottom--large em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--default em-mobile-padding-bottom--none aligndefault"> <div class="wp-block-emagine-column emagine-column5033 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"> <h2 class="wp-block-heading">Recruit rare disease clinical trial patients</h2> <p>Finding the right patient for your rare disease trial can be a challenge. We offer flexible, innovative protocols for reaching, recruiting and retaining the small, globally dispersed populations of patients with rare diseases. Patients also benefit from our decentralized trial services, which help them stay engaged while ensuring that clinical trial data and materials are handled appropriately and in a timely fashion.</p> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull reduce-media-content-padding"><div id="em-layout-id_e6f5de-a4" class="em-row-layout-inner em-margin-top--none em-margin-bottom--default em-tablet-margin-top--default em-tablet-margin-bottom--none em-mobile-margin-top--default em-mobile-margin-bottom--none em-layout-id_e6f5de-a4"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--none em-padding-bottom--default em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--none em-mobile-padding-bottom--none alignfull"> <div class="wp-block-emagine-column emagine-column672 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><section id="media-content-block_a0a20ead9952343bbeb4cdb893b2d603" class="media-content alignfull has-bg-image thirds-layout align-content-center " style=""> <div class="alignwide align-bg"> <div class="media-content__image bg-image" style="background-image: url(https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-shutterstock_1683359032.jpg);"></div> <div class="media-content__content has-container right-content"> <div class="content-container"> <div class="media-content__desc"><h3>Virtual rare disease trials</h3> <p>By offering virtual trials, the patient experience can be optimized with minimal delays and inconvenience by allowing patients to visit a local care hub or arranging for a nurse to make home visits. Participants are assigned a <a href="/blog/value-patient-concierge-services-clinical-trials/">patient concierge</a>, who can proactively help them with resources and solutions to reduce the burden for patients and their caregivers.</p> <p>You can employ our digital and decentralized trial technologies that help bring clinical research to the patient, meeting them where they are and simplifying participation through the use of:</p> <ul> <li>Mobile sites</li> <li>Wearables</li> <li>Telehealth</li> <li>E-consent</li> </ul> </div> <a href="/how-we-help/decentralized-clinical-trials/dct-therapeutic-expertise/rare-diseases-article-download/" class="media-content__link " target="" target="">Explore the latest insights on DCTs in rare disease</a> </div> </div> </div> </section> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull"><div id="em-layout-id_4c3432-0c" class="em-row-layout-inner em-margin-top--small em-margin-bottom--default em-tablet-margin-top--none em-tablet-margin-bottom--default em-mobile-margin-top--none em-mobile-margin-bottom--default em-layout-id_4c3432-0c"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--small em-padding-bottom--xlarge em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--none em-mobile-padding-bottom--default aligndefault"> <div class="wp-block-emagine-column emagine-column8893 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"> <h3 class="wp-block-heading">Patient-centered rare disease research</h3> <p>To drive your treatments forward, we leverage patient-centered partnerships that are key to our approach. We actively build strategic relationships with advocacy groups, academic institutions, health care providers and pharma industry groups to gain firsthand insight into the patients’ journeys. We also tap into our own network of employees who have volunteered to <a href="https://www.ppd.com/therapeutic-expertise/rare-disease/recognizing-rare-disease-day/">share their experiences</a> as both patients and caregivers.</p> </div></div> </div></div></div> <section id="make-the-right-decisions-earlier" class="media-content alignfull has-blue-l-background-color bg-color has-bg-image dark-text thirds-layout invert-mobile align-content-center " style=""> <div class="alignwide align-bg"> <div class="media-content__content has-container left-content"> <div class="content-container"> <h2 class="media-content__title2">Make the right decisions earlier</h2> <div class="media-content__desc"><p>The wrong decision can upend your study in rare disease research — especially for clients developing an orphan drug. That’s why we take a holistic approach from the outset, accelerating study startup by providing an upfront understanding of data, cost and necessary clinical expertise for your trial. The advance planning in our CRO services accounts for:</p> <ul> <li>Site costs</li> <li>Indication-specific experience and available staff</li> <li>Disease diagnosis and progression</li> <li>Clear endpoints</li> </ul> <p>Your project is assigned a dedicated, cross-functional team from the start, meaning you’ll have consistency as our experts design and deliver your clinical trial with rapid patient recruitment, retention and experience in mind.</p> <p>We also run efficient <a href="/blog/natural-history-studies-in-rare-diseases-and-genetic-biomarkers/">natural history studies</a> in parallel with therapeutic studies, which:</p> <ul> <li>Generate the real-world evidence required by the FDA and other regulatory bodies to better characterize patient populations and delineate target populations</li> <li>Focus on describing disease frequency, features and evolution by collecting real-world data from patients</li> <li>Are often performed early in the clinical development process to support and guide the design of rare disease clinical trials</li> </ul> </div> </div> </div> <div class="media-content__image bg-image" style="background-image: url(https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-shutterstock_1701457318.jpg);"></div> </div> </section> <section id="rare-disease-and-pediatrics-center-of-excellence" class="media-content alignfull has-blue-l-background-color bg-color has-bg-image dark-text thirds-layout align-content-center " style=""> <div class="alignwide align-bg"> <div class="media-content__image bg-image" style="background-image: url(https://www.ppd.com/wp-content/uploads/2020/02/iStock-1132295262.jpg);"></div> <div class="media-content__content has-container right-content"> <div class="content-container"> <h2 class="media-content__title2">Rare Disease and Pediatrics Center of Excellence</h2> <div class="media-content__desc"><p>The PPD™ Rare Disease and Pediatrics Center of Excellence (COE) focuses on shaping trial design, strategy and delivery for adult and pediatric rare indications. The COE has performed more than 240 pediatric studies and supported more than 545 total rare disease studies across all phases, indications and therapeutic areas. Our cross-functional rare disease experts deliver innovative, patient-centric solutions and train our study teams to skillfully address the complex challenges faced by these young patients and their caregivers.</p> <p>To decrease the participation burden for <a href="/therapeutic-expertise/pediatrics-research/">pediatric patients</a> and their loved ones, we aim to increase trial flexibility. We can design hybrid study approaches that include both in-clinic and at-home endpoints by leveraging novel trial design solutions, our bespoke digital platforms, and the widespread availability of decentralized clinical trial options.</p> </div> </div> </div> </div> </section> <div class="wp-block-emagine-columns alignfull" id="rare-disease-expertise-across-therapeutic-areas"><div id="em-layout-id_a91ffc-a8" class="em-row-layout-inner em-margin-top--default em-margin-bottom--default em-tablet-margin-top--default em-tablet-margin-bottom--default em-mobile-margin-top--default em-mobile-margin-bottom--default em-layout-id_a91ffc-a8"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--default em-padding-bottom--default em-tablet-padding-top--default em-tablet-padding-bottom--default em-mobile-padding-top--default em-mobile-padding-bottom--default aligndefault"> <div class="wp-block-emagine-column emagine-column2941 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"> <h2 class="wp-block-heading">Rare disease expertise across therapeutic areas</h2> <p>Rare disease trials span many indications. Explore our dedicated rare disease expertise in neurology, metabolic and neuromuscular clinical research development.</p> </div></div> </div></div></div> <section id="panel-slider-block_be9d652f2292bb7c8e431063f71b7f6b" class="panel-slider alignwide"> <div class="panel-slides"> <div class="panel-slide" data-bg="https://www.ppd.com/wp-content/uploads/2021/10/rare-neurological-header-shutterstock_1958796979-616979a6c93c5.jpg"> <div class="panel-slide__background" style="background-image: url(https://www.ppd.com/wp-content/uploads/2021/10/rare-neurological-header-shutterstock_1958796979-616979a6c93c5.jpg);"> </div> <a href="/therapeutic-expertise/neuroscience/rare-neurological-disorders/" target=""> <div class="panel-slide__content-wrap"> <div class="panel-slide__content"> <h4 class="panel-slide__title">Rare neurological disorders</h4> <div class="panel-slide__desc">From orphan drugs to gene therapies, we have vast experience designing and executing more than 100 studies for therapies treating rare neurological disorders including neuromuscular diseases, genetic disorders, cerebral cavernous malformations and lysosomal storage disorders.</div> </div> <span class="panel-slide__link" href="/therapeutic-expertise/neuroscience/rare-neurological-disorders/" target="">Learn more</span> </a> </div> </div> <div class="panel-slide" data-bg="https://www.ppd.com/wp-content/uploads/2021/10/Rare-metabolic-disease-header-shutterstock_1858356064.jpg"> <div class="panel-slide__background" style="background-image: url(https://www.ppd.com/wp-content/uploads/2021/10/Rare-metabolic-disease-header-shutterstock_1858356064.jpg);"> </div> <a href="/therapeutic-expertise/metabolic-endocrine/rare-metabolic-disease/" target=""> <div class="panel-slide__content-wrap"> <div class="panel-slide__content"> <h4 class="panel-slide__title">Rare metabolic disease</h4> <div class="panel-slide__desc">With extensive experience in studies for therapeutics treating rare metabolic diseases like Gaucher and glycogen storage diseases, we provide specialized and flexible development strategies that put the patient at the center.</div> </div> <span class="panel-slide__link" href="/therapeutic-expertise/metabolic-endocrine/rare-metabolic-disease/" target="">Learn more</span> </a> </div> </div> <div class="panel-slide" data-bg="https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-neuromuscular-1024x499-1.jpg"> <div class="panel-slide__background" style="background-image: url(https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-neuromuscular-1024x499-1.jpg);"> </div> <a href="/therapeutic-expertise/rare-disease/neuromuscular/" target=""> <div class="panel-slide__content-wrap"> <div class="panel-slide__content"> <h4 class="panel-slide__title">Rare neuromuscular</h4> <div class="panel-slide__desc">Our global team can help clients overcome even the most complex clinical development challenges in developing therapies to treat rare neuromuscular diseases, such as ALS.</div> </div> <span class="panel-slide__link" href="/therapeutic-expertise/rare-disease/neuromuscular/" target="">Learn more</span> </a> </div> </div> </div> </section> <div style="height:6rem" aria-hidden="true" id="ppd-heroes-in-rare-disease" class="wp-block-spacer hide-on-mobile"></div> <section id="video-block-block_b739e91d9eaa31ed5c0c565ad6bdfaea" class="video-block alignfull has-blue-l-background-color" style="background-image: url(https://www.ppd.com/wp-content/uploads/2019/11/hex_2.png);"> <div class="video-block__content alignwide"> <h2>Our heroes in rare disease</h2> <p>Our Heroes program is dedicated to sharing the stories of individuals facing significant health conditions like rare diseases. These patients have benefitted from the therapies that are made possible by clinical research.</p> </div> <div class="video-block__video"> <div class="aligndefault"> <div style="position: relative; display: block; max-width: 960px;"><div style="padding-top: 56.25%;"><iframe loading="lazy" src=https://players.brightcove.net/2202959166001/CHiz2H2j_default/index.html?videoId=6306786542112 allowfullscreen="" allow="encrypted-media" style="position: absolute; top: 0px; right: 0px; bottom: 0px; left: 0px; width: 100%; height: 100%;border:0;"></iframe></div></div></div> </div> </section> <div style="height:4rem" aria-hidden="true" id="related-resources" class="wp-block-spacer"></div> <h3 class="wp-block-heading has-text-align-center" style="text-transform:none">Related resources in rare disease</h3> <div class="wp-block-emagine-columns alignfull"><div id="em-layout-id_1aacdb-38" class="em-row-layout-inner em-margin-top--none em-margin-bottom--none em-tablet-margin-top--none em-tablet-margin-bottom--none em-mobile-margin-top--none em-mobile-margin-bottom--none em-layout-id_1aacdb-38"><div class="em-row-column-wrap em-has-3-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-equal em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--default em-padding-bottom--none em-tablet-padding-top--default em-tablet-padding-bottom--none em-mobile-padding-top--none em-mobile-padding-bottom--none alignwide"> <div class="wp-block-emagine-column emagine-column8023 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--medium em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><a href="https://www.ppd.com/wp-content/uploads/2024/08/PPD-Rare-Diseases-Brochure.pdf" target="" id="ppd-card-block_adfa87220f6232aa1cf06595c831adee" class="ppd-card alignfull "> <div class="ppd-card__image"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2022/12/RareDisease.png" alt="Woman with child"> </div> <div class="ppd-card__inner"> <div class="ppd-card__top"> <div class="ppd-card__title">Rare disease overview brochure</div> <p class="ppd-card__content">Download our rare disease brochure to learn about our specialized approach to rare disease studies. </p> </div> <div class="ppd-card__bottom"> <span class="ppd-card__link" >Learn more</span> </div> </div> </a> </div></div> <div class="wp-block-emagine-column emagine-column7239 inner-column-2"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--medium em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><a href="https://www.ppd.com/blog/the-personal-impact-of-rare-diseases/" target="_blank" id="ppd-card-block_4adef449cb66f0a9f6e2aec5d0b45332" class="ppd-card alignfull "> <div class="ppd-card__image"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-shutterstock_418460920-640x440.jpg" alt="child in hospital holding stuffed animal"> </div> <div class="ppd-card__inner"> <div class="ppd-card__top"> <div class="ppd-card__title">The personal impact of rare diseases</div> <p class="ppd-card__content">Read the personal stories of our employees living and working with rare diseases. </p> </div> <div class="ppd-card__bottom"> <span class="ppd-card__link" >Learn more</span> </div> </div> </a> </div></div> <div class="wp-block-emagine-column emagine-column7846 inner-column-3"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--medium em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><a href="https://www.ppd.com/wp-content/uploads/2019/11/Patient-Concierge-Services-Overview-PPD.pdf" target="_blank" id="ppd-card-block_38a2b1064e1978b2966f1551a67c91a9" class="ppd-card alignfull "> <div class="ppd-card__image"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2022/08/patient-concierge-service-640x440.jpg" alt="patient-concierge-service"> </div> <div class="ppd-card__inner"> <div class="ppd-card__top"> <div class="ppd-card__title">Patient Concierge Service</div> <p class="ppd-card__content">Learn more about the unprecedented level of support a patient concierge provides.</p> </div> <div class="ppd-card__bottom"> <span class="ppd-card__link" >Learn more</span> </div> </div> </a> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull"><div id="em-layout-id_a2d1b5-53" class="em-row-layout-inner em-margin-top--none em-margin-bottom--none em-tablet-margin-top--none em-tablet-margin-bottom--none em-mobile-margin-top--none em-mobile-margin-bottom--default em-layout-id_a2d1b5-53"><div class="em-row-column-wrap em-has-3-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-row em-m-colapse-left-to-right em-mobile-layout-row em-padding-top--none em-padding-bottom--default em-tablet-padding-top--none em-tablet-padding-bottom--none em-mobile-padding-top--none em-mobile-padding-bottom--default alignwide"> <div class="wp-block-emagine-column emagine-column4868 inner-column-1"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><a href="https://www.ppd.com/wp-content/uploads/2021/05/Rare-Dieases-of-the-Eye-PPD-White-Paper.pdf" target="_blank" id="ppd-card-block_3c0d7399c18e07643b203d78da9b9234" class="ppd-card alignfull "> <div class="ppd-card__image"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-eye-development-paper-640x440.jpg" alt="rare-diseases-eye-exam"> </div> <div class="ppd-card__inner"> <div class="ppd-card__top"> <div class="ppd-card__title">Concerns and success factors in developing therapies for rare diseases</div> <p class="ppd-card__content">This paper discusses key concerns and success factors in developing therapies for rare diseases of the eye.</p> </div> <div class="ppd-card__bottom"> <span class="ppd-card__link" >Read the white paper</span> </div> </div> </a> </div></div> <div class="wp-block-emagine-column emagine-column7583 inner-column-2"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><a href="https://www.ppd.com/natural-history-studies-in-rare-diseases-and-genetic-biomarkers/" target="" id="ppd-card-block_a364ac92b5c2598867d36fb6c76ef0e1" class="ppd-card alignfull "> <div class="ppd-card__image"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2022/08/rare-diseases-shutterstock_417491413-1-640x440.jpg" alt="rare-diseases-puzzle pieces"> </div> <div class="ppd-card__inner"> <div class="ppd-card__top"> <div class="ppd-card__title">Natural history studies in rare diseases</div> <p class="ppd-card__content">New product introductions in rare diseases and precision medicine have allowed improved outcomes for patients who would otherwise face grim prognoses.</p> </div> <div class="ppd-card__bottom"> <span class="ppd-card__link" >Read the blog post</span> </div> </div> </a> </div></div> <div class="wp-block-emagine-column emagine-column2551 inner-column-3"><div class="em-inside-inner-col em-padding-top--none em-padding-right--none em-padding-bottom--none em-padding-left--none em-tablet-padding-top--none em-tablet-padding-right--none em-tablet-padding-bottom--none em-tablet-padding-left--none em-mobile-padding-top--none em-mobile-padding-right--none em-mobile-padding-bottom--none em-mobile-padding-left--none"><a href="https://www.ppd.com/wp-content/uploads/2019/09/Risk-Based-Monitoring-Considerations-Rare-Diseases-Trials.pdf" target="_blank" id="ppd-card-block_4100490cc18c920792c9d8caacff8379" class="ppd-card alignfull "> <div class="ppd-card__image"> <img decoding="async" src="https://www.ppd.com/wp-content/uploads/2023/01/rare-disease-blog-shutterstock_362338955-760x440.jpg" alt="Pediatric doctor visiting young girl in hospital dad with dad smiling in the background"> </div> <div class="ppd-card__inner"> <div class="ppd-card__top"> <div class="ppd-card__title">Risk-based monitoring considerations in rare disease trials </div> <p class="ppd-card__content">Download this white paper to learn uniquely tailored monitoring strategies for rare diseases.</p> </div> <div class="ppd-card__bottom"> <span class="ppd-card__link" >Read the white paper</span> </div> </div> </a> </div></div> </div></div></div> <div class="wp-block-emagine-columns alignfull" id="connectwithus"><div id="em-layout-id_c439fd-5d" class="em-row-layout-inner em-margin-top--default em-margin-bottom--none em-tablet-margin-top--default em-tablet-margin-bottom--none em-mobile-margin-top--none em-mobile-margin-bottom--none em-row-has-bg em-layout-id_c439fd-5d"><div class="em-row-column-wrap em-has-1-columns em-gutter-default em-row-valign-top em-row-layout-equal em-tab-layout-inherit 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