FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub

<!DOCTYPE html> <html lang="en" dir="ltr" prefix="og: https://ogp.me/ns#"> <head> <meta charset="utf-8" /> <script async src="https://www.googletagmanager.com/gtag/js?id=G-273DTKB5QW"></script> <meta name="description" content="The Hub will act as the single point of engagement and connection with outside parties for drug and biological product development and represents a dynamic new model for cross-center collaboration at the agency." /> <meta name="dcterms.title" content="FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub" /> <meta name="dcterms.creator" content="Office of the Commissioner" /> <meta name="dcterms.description" content="The Hub will act as the single point of engagement and connection with outside parties for drug and biological product development and represents a dynamic new model for cross-center collaboration at the agency." /> <meta name="dcterms.publisher" content="FDA" /> <meta name="dcterms.type" content="Article" /> <meta name="dcterms.source" content="FDA" /> <meta property="og:site_name" content="U.S. Food and Drug Administration" /> <meta property="og:type" content="Article" /> <meta property="og:url" content="https://www.fda.gov/news-events/fda-voices/fda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub" /> <meta property="og:title" content="FDA Takes Steps Toward Establishing the Rare Disease Innovation Hub" /> <meta property="og:description" content="The Hub will act as the single point of engagement and connection with outside parties for drug and biological product development." /> <meta property="og:image" content="https://www.fda.gov/files/fda_rdih_1600x900_0.jpg" /> <meta property="og:updated_time" content="Fri, 11/01/2024 - 00:00" /> <meta property="article:publisher" content="FDA" /> <meta property="article:published_time" content="Fri, 11/01/2024 - 08:50" /> <meta property="article:modified_time" content="Fri, 11/01/2024 - 00:00" /> <meta name="google-site-verification" content="tWxlDhm4ANdksJZPj7TBmHgNoMqZCnecPp0Aa2vC9XA" /> <meta name="twitter:card" content="summary_large_image" /> <meta name="twitter:site" content="@US_FDA" /> <meta property="twitter:description" content="The Hub will act as the single point of engagement and connection with outside parties for drug and biological product development." /> <meta name="twitter:title" content="FDA Takes Steps Toward Establishing the Rare Disease Innovation Hub" /> <meta name="twitter:creator" content="@US_FDA" /> <meta name="twitter:image" content="https://www.fda.gov/files/fda_rdih_1600x900_0.jpg" /> <meta name="MobileOptimized" content="width" /> <meta name="HandheldFriendly" content="true" /> <meta name="viewport" content="width=device-width, initial-scale=1.0" /> <script type="application/ld+json">{ "@context": "https://schema.org", "@graph": [ { "@type": "Article", "headline": "FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub", "name": "FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub", "description": "The Hub will act as the single point of engagement and connection with outside parties for drug and biological product development and represents a dynamic new model for cross-center collaboration at the agency.", "image": { "@type": "ImageObject", "representativeOfPage": "True", "url": "https://www.fda.gov/files/fda_rdih_1600x900_0.jpg" }, "datePublished": "Fri, 11/01/2024 - 08:50", "dateModified": "Fri, 11/01/2024 - 00:00", "author": { "@type": "Organization", "name": "Office of the Commissioner" }, "publisher": { "@type": "Organization", "name": "FDA" } } ] }</script> <link rel="icon" href="/themes/custom/preview/favicon.ico" type="image/vnd.microsoft.icon" /> <link rel="alternate" hreflang="en" href="https://www.fda.gov/news-events/fda-voices/fda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub" /> <link rel="canonical" href="https://www.fda.gov/news-events/fda-voices/fda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub" /> <link rel="shortlink" href="https://www.fda.gov/node/423792" /> <script src="/files/google_tag/production/google_tag.script.js?smgeuw" defer></script> <title>FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub | FDA</title> <link rel="stylesheet" media="all" href="/files/css/css_4qVI3__kNnM159lbX85PiehwLfcQbjhfFGV05vyo-Yg.css?delta=0&language=en&theme=preview&include=eJxlj0FuAzEIRS_khG4q9TYWg7Fj1TYW0ETT0yeTqJMo3SD4SI9HluF4YZPOkJ_9MRSR0jjiwLZ6JYP3INhqzh0WNA5T-Vz5AqXJgu1gvrY6yr-YtE63sIi4ueKEKVPOrCEnjPTNqbpo5HHCQdx5uAESiaYqI7qUTcBOok6SOGxog3s9dkk_jR8c6bPVjfDYxc-Pr5cw5NqcFQin37Ah32SGOBvs3e5Nir8rl-cjCef99N-8yxx2zyuWXoVv" /> <link rel="stylesheet" media="all" href="/files/css/css_xLeUZALg1CD0YmiSIzZnqkwKfNF_g3TkAjkxBt_uccA.css?delta=1&language=en&theme=preview&include=eJxlj0FuAzEIRS_khG4q9TYWg7Fj1TYW0ETT0yeTqJMo3SD4SI9HluF4YZPOkJ_9MRSR0jjiwLZ6JYP3INhqzh0WNA5T-Vz5AqXJgu1gvrY6yr-YtE63sIi4ueKEKVPOrCEnjPTNqbpo5HHCQdx5uAESiaYqI7qUTcBOok6SOGxog3s9dkk_jR8c6bPVjfDYxc-Pr5cw5NqcFQin37Ah32SGOBvs3e5Nir8rl-cjCef99N-8yxx2zyuWXoVv" /> <script src="https://script.crazyegg.com/pages/scripts/0024/3700.js" async></script> <script src="https://dap.digitalgov.gov/Universal-Federated-Analytics-Min.js?agency=HHS&subagency=FDA&sdor=fda.gov&dclink=true" language="javascript" id="_fed_an_ua_tag"></script> <script>window.dataLayer = window.dataLayer || [];function gtag(){dataLayer.push(arguments)};gtag("js", new Date());gtag("set", "developer_id.dMDhkMT", true);gtag("config", "G-273DTKB5QW", {"groups":"default","page_placeholder":"PLACEHOLDER_page_location","allow_ad_personalization_signals":false});</script> </head> <body class="role-anonymous path-node page-node-type-article has-glyphicons"> <div id="quicklinks" class="sr-only"> <ul> <li><a href="#main-content" class="sr-only sr-only-focusable" tabindex="1">Skip to main content</a></li> <li><a href="#search-form" class="sr-only sr-only-focusable" tabindex="1">Skip to FDA Search</a></li> <li><a href="#section-nav" class="sr-only sr-only-focusable" tabindex="1">Skip to in this section menu</a></li> <li><a href="#footer-heading" class="sr-only sr-only-focusable" tabindex="1">Skip to footer links</a></li> </ul> </div> <noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-M95XGZW" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript> <div class="dialog-off-canvas-main-canvas" data-off-canvas-main-canvas> <div class="main-container container-fluid"> <div class="row"> <header class="lcds-header container-fluid" role="heading" aria-level="1"> <div class="row us-masthead"> <div class="usa-banner col-xs-12"> <img class="usa-banner__us-flag" src="/themes/custom/preview/assets/images/US_Flag.png" alt="U.S. flag" /> <span>An official website of the United States government</span> <a id="USMenuButton" class="collapsed" data-toggle="collapse" data-target="#USABannerMenu" aria-controls="USABannerMenu">Here鈥檚 how you know <span class="toggle-indicator"> </span></a> <div class="col-xs-12 collapse usa-banner__menu" id="USABannerMenu" aria-labelledby="USMenuButton"> <div id="gov-banner" class="row usa-banner-content usa-grid usa-accordion-content" aria-hidden="true"> <div class="col-xs-12 col-sm-6 col-md-3"> <img class="usa-banner-icon usa-media_block-img" src="/themes/custom/preview/assets/images/icon-dot-gov.svg" alt="Dot gov" style="width:3em;"> <div class="usa-media_block-body"> <p><strong>The .gov means it鈥檚 official.</strong><br>Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site.</p> </div> </div> <div class="col-xs-12 col-sm-6 col-md-3"> <img class="usa-banner-icon usa-media_block-img" src="/themes/custom/preview/assets/images/icon-https.svg" alt="SSL" style="width:3em;"> <div class="usa-media_block-body"> <p><strong>The site is secure.</strong><br> The <strong>https://</strong> ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.</p> </div> </div> </div> </div> </div> </div> <div class="alert alert-warning margin-left-neg-2 margin-right-neg-2 margin-top-3 margin-bottom-1"> <p>On Oct. 1, 2024, the FDA began implementing a <a href="/about-fda/fda-organization/fda-modernization-efforts-establishing-unified-human-foods-program-new-model-field-operations-and" title="FDA Modernization Efforts for Establishing a Unified Human Foods Program, New Model for Field Operations and More">reorganization</a> impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.</p> </div> <div class="row fda-masthead"> <div class="col-xs-4 col-md-8"> <a href="/" title="FDA Homepage"> <h1 class="fda-masthead__fda-logo">U.S. Food and Drug Administration</h1> </a> </div> <div class="col-xs-8 col-md-4"> <ul class='fda-masthead__item-list'> <li><a title="" id="btn-search" class="btn btn-default btn-sm fda-masthead__btn-search"> <span class="fa fa-search" aria-hidden="true"> </span> <span class="fda-masthead__btn-label">Search</span> </a></li> <li><a id="menu-btn" class="btn btn-default btn-sm fda-masthead__btn-menu collapsed" href="#primary-nav" data-toggle="collapse" aria-expanded="true"> <span class="fa fa-bars" aria-hidden="true"> </span> <span class='fda-masthead__btn-label'>Menu</span> </a></li> </ul> </div> <form class="fda-masthead__search sr-only" role="search" action="/search" method="GET" name="searchForm" id="search-form" accept-charset="UTF-8" > <div class="search-popover" id="search-popover"> <div class="input-group pull-right" id="search-group"> <label class="sr-only" for="search-query">Search FDA</label> <input class="form-control search-input" id="search-query" name="s" aria-autocomplete="list" aria-haspopup="true" title="Enter the terms you wish to search for." placeholder="Search FDA" type="text" /> <span class="input-group-btn" id="input-group-btn"> <button type="submit" class="btn btn-danger search-btn" id="search-btn" title="Search"><span class="fa fa-search" aria-hidden="true"><span class="sr-only">Submit search</span></span></button> </span> </div> </div> </form> </div> <nav id="primary-nav" class="lcds-primary-nav row collapse"> <div class="col-md-5 col-lg-4"> <section class="lcds-primary-nav__group lcds-primary-nav__group--bordered"> <h2 class="lcds-primary-nav__group-heading">Featured</h2> <ul class="lcds-primary-nav__list lcds-primary-nav__list--featured"> <li class="lcds-primary-nav__list-item"><a href="https://www.safetyreporting.hhs.gov/smarthub#/" title="Report a Problem with an FDA Regulated Product">Report a Product Problem</a></li> <li class="lcds-primary-nav__list-item"><a href="/about-fda/contact-fda">Contact FDA</a></li> <li class="lcds-primary-nav__list-item"><a href="/regulatory-information/search-fda-guidance-documents">FDA Guidance Documents</a></li> <li class="lcds-primary-nav__list-item"><a href="/safety/recalls-market-withdrawals-safety-alerts">Recalls, Market Withdrawals and Safety Alerts</a></li> <li class="lcds-primary-nav__list-item"><a href="/news-events/newsroom/press-announcements">Press Announcements</a></li> <li class="lcds-primary-nav__list-item"><a href="/inspections-compliance-enforcement-and-criminal-investigations/compliance-actions-and-activities/warning-letters">Warning Letters</a></li> <li class="lcds-primary-nav__list-item"><a href="/advisory-committees">Advisory Committees</a></li> <li class="lcds-primary-nav__list-item"><a href="/fda-en-espanol">En Espa&#241ol</a></li> </ul> </section> </div> <div class="col-md-7 col-lg-8"> <section class="lcds-primary-nav__group lcds-primary-nav__group--bordered"> <h2 class="lcds-primary-nav__group-heading">Products</h2> <ul class="lcds-primary-nav__list"> <li class="lcds-primary-nav__list-item"><a href="/food">Food</a></li> <li class="lcds-primary-nav__list-item"><a href="/drugs">Drugs</a></li> <li class="lcds-primary-nav__list-item"><a href="/medical-devices">Medical Devices</a></li> <li class="lcds-primary-nav__list-item"><a href="/radiation-emitting-products">Radiation-Emitting Products</a></li> <li class="lcds-primary-nav__list-item"><a href="/vaccines-blood-biologics">Vaccines, Blood, and Biologics</a></li> <li class="lcds-primary-nav__list-item"><a href="/animal-veterinary">Animal and Veterinary</a></li> <li class="lcds-primary-nav__list-item"><a href="/cosmetics">Cosmetics</a></li> <li class="lcds-primary-nav__list-item"><a href="/tobacco-products">Tobacco Products</a></li> </ul> </section> <section class="lcds-primary-nav__group lcds-primary-nav__group--bordered"> <h2 class="lcds-primary-nav__group-heading">Topics</h2> <ul class="lcds-primary-nav__list"> <li class="lcds-primary-nav__list-item"><a href="/about-fda">About FDA</a></li> <li class="lcds-primary-nav__list-item"><a href="/combination-products">Combination Products</a></li> <li class="lcds-primary-nav__list-item"><a href="/regulatory-information">Regulatory Information</a></li> <li class="lcds-primary-nav__list-item"><a href="/safety">Safety</a></li> <li class="lcds-primary-nav__list-item"><a href="/emergency-preparedness-and-response">Emergency Preparedness</a></li> <li class="lcds-primary-nav__list-item"><a href="/international-programs">International Programs</a></li> <li class="lcds-primary-nav__list-item"><a href="/news-events">News and Events</a></li> <li class="lcds-primary-nav__list-item"><a href="/training-and-continuing-education">Training and Continuing Education</a></li> <li class="lcds-primary-nav__list-item"><a href="/inspections-compliance-enforcement-and-criminal-investigations">Inspections and Compliance</a></li> <li class="lcds-primary-nav__list-item"><a href="/science-research">Science and Research</a></li> </ul> </section> <section class="lcds-primary-nav__group lcds-primary-nav__group--bordered"> <h2 class="lcds-primary-nav__group-heading">Information For</h2> <ul class="lcds-primary-nav__list"> <li class="lcds-primary-nav__list-item"><a href="/consumers">Consumers</a></li> <li class="lcds-primary-nav__list-item"><a href="/patients">Patients</a></li> <li class="lcds-primary-nav__list-item"><a href="/industry">Industry</a></li> <li class="lcds-primary-nav__list-item"><a href="/health-professionals">Health Professionals</a></li> <li class="lcds-primary-nav__list-item"><a href="/federal-state-local-tribal-and-territorial-officials">Federal, State and Local Officials</a></li> </ul> </section> </div> </nav> </header> <div class="col-xs-12"> <div data-drupal-messages-fallback class="hidden"></div> </div> <section id="block-entityviewcontent" data-block-plugin-id="entity_view:node" class="block block-ctools block-entity-viewnode clearfix"> <a class="lcds-button--expandable collapsed hidden-md hidden-lg" data-toggle="collapse" href="#section-nav" >In this section<span class="visible-sm-inline-block">: FDA Voices </span> </a> <nav id="section-nav" class="lcds-card lcds-section-nav lcds-section-nav hidden-md hidden-lg collapse"> <ul class="lcds-section-nav__list"> <li> <a href="/news-events/fda-newsroom/fda-voices" title="FDA Voices" class="lcds-section-nav__section-link lcds-section-nav__link lcds-section-nav__parent-link visible-xs-block visible-sm-block"> FDA Voices </a> <div class="views-element-container form-group"><div class="view view-in-this-section view-id-in_this_section view-display-id-block_5 js-view-dom-id-4b8342267bdb55c59e51abdf845922a532e8e3adfe1cd57e1c87649f292c0124"> </div> </div> <div class="views-element-container form-group"><div class="view view-in-this-section-sub view-id-in_this_section_sub view-display-id-block_2 js-view-dom-id-e686110dc52b7c59844593674d49e329b72cdf2ab34a8c444db9c9cd5f6c0ac1"> </div> </div> </li> </ul> </nav> </section> <section id="block-entityviewcontent-15" data-block-plugin-id="entity_view:node" class="block block-ctools block-entity-viewnode clearfix"> <ol class="lcds-breadcrumb visible-md visible-lg"> <li > <a href="/">Home</a> </li> <li > <a href="/news-events">News & Events</a> </li> <li > <a href="/news-events/fda-newsroom">FDA Newsroom</a> </li> <li > <a href="/news-events/fda-newsroom/fda-voices">FDA Voices</a> </li> <li > <a class="current-link">FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub</a> </li> </ol> <ol class="lcds-breadcrumb visible-sm visible-xs"> <li> <a href="/news-events/fda-newsroom/fda-voices" title="FDA Voices"> FDA Voices </a> </li> </ol> </section> <main> <article id="main-content" class="article main-content container-fluid" role="article"> <header class="row content-header" role="heading" aria-level="1"> <section id="block-entityviewcontent-2" data-block-plugin-id="entity_view:node" class="block block-ctools block-entity-viewnode clearfix"> <div class="col-sm-12 col-md-8 col-md-offset-2"> <h1 class="content-title text-center">FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub</h1> <div class="lcds-toolbar lcds-toolbar--social"> <a href="#subscribe" class="btn btn-sm btn-default header-button-subscribe">Subscribe to Email Updates</a> <ul class="lcds-share lcds-share--default"> <li class="lcds-share__item"> <a href="https://www.facebook.com/sharer/sharer.php?u=https://www.fda.gov%2Fnews-events%2Ffda-voices%2Ffda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub" class="lcds-share__btn lcds-share--default__btn-facebook js-share" id="fb-share" target="_blank"><span class="fa icon-facebook" aria-hidden="true"></span>Share</a> </li> <li class="lcds-share__item"> <a href="https://x.com/intent/tweet/?text=FDA%20Takes%20Exciting%20Steps%20Toward%20Establishing%20the%20Rare%20Disease%20Innovation%20Hub&url=https://www.fda.gov%2Fnews-events%2Ffda-voices%2Ffda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub" target="_blank" class="lcds-share__btn lcds-share--default__btn-x-twitter js-share" id="twitter-share"> <svg class="icon-svg-inline" aria-hidden="true"> <use href="/themes/custom/preview/assets/images/fa-icons.svg#icon-x-twitter"> </svg> Post </a> </li> <li class="lcds-share__item hidden-xs"> <a href="https://www.linkedin.com/shareArticle?mini=true&url=https://www.fda.gov%2Fnews-events%2Ffda-voices%2Ffda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub&title=FDA%20Takes%20Exciting%20Steps%20Toward%20Establishing%20the%20Rare%20Disease%20Innovation%20Hub&source=FDA" class="lcds-share__btn lcds-share--default__btn-linkedin js-share" id="linkedin-share" target="_blank"><span class="fa icon-linkedin" aria-hidden="true"></span>Linkedin</a> </li> <li class="lcds-share__item"> <a href="mailto:?subject=FDA%20Takes%20Exciting%20Steps%20Toward%20Establishing%20the%20Rare%20Disease%20Innovation%20Hub&body=https://www.fda.gov%2Fnews-events%2Ffda-voices%2Ffda-takes-exciting-steps-toward-establishing-rare-disease-innovation-hub" class="lcds-share__btn lcds-share--default__btn-mail"><span class="fa icon-envelope" aria-hidden="true"></span>Email</a> </li> <li class=" lcds-share__item hidden-xs"> <a href="javascript:window.print();" title="Print this page" class="lcds-share__btn lcds-share--default__btn-print"><span class="fa icon-print" aria-hidden="true"></span>Print</a> </li> </ul> <div class="form-group"> </div> </div> </div> </section> </header> <div class="col-md-8 col-md-push-2" role="main"> <figure> <div> <div class="field field--name-image field--type-image field--label-inline"> <div class="field--label">Image</div> <div class="field--item"> <picture> <source srcset="/files/styles/main_image_small/public/fda_rdih_1600x900_0.jpg?itok=EVm7xRfk 1x" media="all and (max-width: 320px)" type="image/jpeg" width="320" height="180"/> <source srcset="/files/styles/main_image_medium/public/fda_rdih_1600x900_0.jpg?itok=sobxpdUQ 1x" media="all and (min-width: 321px) and (max-width: 480px)" type="image/jpeg" width="500" height="281"/> <source srcset="/files/styles/main_image_medium/public/fda_rdih_1600x900_0.jpg?itok=sobxpdUQ 1x" media="all and (min-width: 481px) and (max-width: 767px)" type="image/jpeg" width="500" height="281"/> <source srcset="/files/styles/main_image_medium/public/fda_rdih_1600x900_0.jpg?itok=sobxpdUQ 1x" media="all and (min-width: 768px) and (max-width: 992px)" type="image/jpeg" width="500" height="281"/> <source srcset="/files/styles/main_image_1/public/fda_rdih_1600x900_0.jpg?itok=n79UjuKF 1x" media="all and (min-width: 993px) and (max-width: 1200px)" type="image/jpeg" width="1020" height="574"/> <source srcset="/files/styles/main_image_1/public/fda_rdih_1600x900_0.jpg?itok=n79UjuKF 1x" media="all and (min-width: 1201px)" type="image/jpeg" width="1020" height="574"/> <img loading="eager" src="/files/styles/main_image_medium/public/fda_rdih_1600x900_0.jpg?itok=sobxpdUQ" width="500" height="281" alt="Rare Disease Innovation Hub background with DNA strands and FDA monogram logo." class="img-responsive" /> </picture> </div> </div> </div> </figure> <p><em>By: Patrizia Cavazzoni, M.D., Director, Center for Drug Evaluation and Research and Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research</em></p> <p>This summer, the U.S. Food and Drug Administration <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="938722b1-90cc-42ea-bb93-08c57efe8d39" href="/news-events/fda-voices/fda-rare-disease-innovation-hub-enhance-and-advance-outcomes-patients" title="FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients">announced</a> plans to establish the <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="c9f56660-1ec7-4be1-82ca-d3dfb8ecb686" href="/industry/medical-products-rare-diseases-and-conditions/fda-rare-disease-innovation-hub" title="FDA Rare Disease Innovation Hub">Rare Disease Innovation Hub</a> (the Hub), and today, we are excited to share more about our progress. The Hub is an FDA cross-center program that will act as the single point of engagement and connection with outside parties for drug and biological product development and as a forum for the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) to collaborate on cross-cutting rare disease-related issues. Through the Hub, we plan to foster a community at the FDA for open dialogue and knowledge sharing to identify new approaches to drug and biologic development and overcome hurdles that have traditionally impeded progress for rare disease treatments. </p> <h2>Gathering Feedback from the Rare Disease Community </h2> <figure class="align-left"> <div alt="Patrizia Cavazzoni, M.D." data-embed-button="media_browser" data-entity-embed-display="media_image" data-entity-embed-display-settings="Array" data-entity-type="media" data-entity-uuid="80a6ceaf-9175-4d6c-b88b-8b154118a012" data-langcode="en" title="Patrizia Cavazzoni_900x900" class="embedded-entity"> <img loading="lazy" src="/files/Patrizia%20Cavazzoni_900x900.png" alt="Patrizia Cavazzoni, M.D." title="Patrizia Cavazzoni_900x900" class="img-responsive"> </div> <figcaption class="caption">Patrizia Cavazzoni, M.D.</figcaption> </figure> <p>Our first major step toward establishing the Hub was to gather feedback from our partners in the rare disease community. In collaboration with the Reagan-Udall Foundation for the FDA, we hosted a public meeting, <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="19c6c141-e0da-433f-89b9-26364d25a19e" href="/drugs/news-events-human-drugs/advancing-rare-disease-therapies-through-fda-rare-disease-innovation-hub-10162024" title="Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub - 10/16/2024">Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub</a>, on October 16, 2024. The meeting brought together rare disease patient advocates, academic researchers, regulated industry, and others to discuss how the Hub can best engage with members of the community and prioritize its work. There was also a public docket to capture feedback from those unable to speak during the public meeting. </p> <p>We want to thank our partners in the rare disease community for participating in the meeting and contributing to the public docket. We heard you and will use your feedback to help inform the Hub鈥檚 priorities and initiatives.  </p> <h2>New Director of Strategic Coalitions for the Hub</h2> <figure class="align-left"> <div alt="Dr. Peter Marks photograph" data-embed-button="media_browser" data-entity-embed-display="media_image" data-entity-embed-display-settings="Array" data-entity-type="media" data-entity-uuid="aa45005e-ad11-4de9-9543-01988fc5a79a" data-langcode="en" title="Peter Marks 19929" class="embedded-entity"> <img loading="lazy" src="/files/peter_marks_19929%20%281%29.jpg" alt="Dr. Peter Marks photograph" title="Peter Marks 19929" class="img-responsive"> </div> <figcaption class="caption">Peter Marks, M.D., Ph.D.</figcaption> </figure> <p>Another key step toward establishing the Hub was creating a new senior leadership position, the Director of Strategic Coalitions for the Hub. We are pleased to share that we have selected Amy Comstock Rick, J.D., to serve in this role. Ms. Rick will be accountable directly to both of us and will act as a single point of external engagement on behalf of the Hub on cross-cutting rare disease-related issues and will also drive the development of a comprehensive strategic agenda for the Hub. </p> <p>Prior to joining the agency Ms. Rick served as principal consultant at Leavitt Partners advising clients and coalitions on health policy issues, with a primary focus on FDA-related issues. Before that, she served as President and Chief Executive Officer of the Food & Drug Law Institute. Ms. Rick holds a juris doctor from University of Michigan Law School and a bachelor鈥檚 degree from Bard College. </p> <p>Ms. Rick brings to this new role more than 20 years of experience as a leader and trusted ally in advocating for policies throughout health care, biomedical research, and nonprofit communities, which includes extensive engagement with patient communities and government agencies on issues related to rare diseases. We are excited to have her join and begin creating the important partnerships needed to strengthen the Hub鈥檚 engagement throughout the rare disease community.</p> <h2>Organizational Structure of the Hub </h2> <p>The Hub represents a dynamic new model for cross-center collaboration at the agency. As the directors of CBER and CDER, we are jointly leading the Hub and will collaborate with other areas that touch rare diseases across the FDA, including with the Center for Devices and Radiological Health, the Oncology Center of Excellence, and the Office of Orphan Products Development. This includes establishing a Rare Disease Innovation Hub Steering Committee that will bring together these groups and the creation of a new CBER-CDER Rare Disease Policy and Portfolio Council. The council will bring together experts from the centers to consider scientific, regulatory science, and policy issues related to rare disease product development that are common to both centers, leveraging the <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="3b6eb6bc-08da-4d67-bf14-a9e60e5a6aa2" href="/about-fda/center-drug-evaluation-and-research-cder/accelerating-rare-disease-cures-arc-program" title="Accelerating Rare disease Cures (ARC) Program">CDER鈥檚 Accelerating Rare disease Cures (ARC) Program</a> and <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="8f6f1c3a-9819-4751-8101-86166d65ce09" href="/vaccines-blood-biologics/cber-rare-disease-program" title="CBER Rare Disease Program ">CBER鈥檚 Rare Disease Program</a>. </p> <p>The collaborative model of the Hub will build upon the knowledge and skills of staff within their respective centers, promote adoption of best practices, and avoid confusion over decision-making authorities within those organizations. Meetings between the FDA and sponsors relating to specific drug or biological products will remain the same, as will the way decisions are made about specific programs and how advisory committee meetings are structured and run. </p> <p>By working together, we believe that we can further facilitate the development, evaluation, and availability of safe and effective therapies. We are committed to ongoing and open communication and look forward to continuing this dialogue with the rare disease community. For more information, please visit <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="c9f56660-1ec7-4be1-82ca-d3dfb8ecb686" href="/industry/medical-products-rare-diseases-and-conditions/fda-rare-disease-innovation-hub" title="FDA Rare Disease Innovation Hub">FDA Rare Disease Innovation Hub</a>. </p> <p><u>Related Links:</u><br> <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="0c0b3c7c-95b7-4e19-846e-469bc1725945" href="/patients/rare-diseases-fda" title="Rare Diseases at FDA">Rare Diseases at FDA For Patients</a><br> <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="8f6f1c3a-9819-4751-8101-86166d65ce09" href="/vaccines-blood-biologics/cber-rare-disease-program" title="CBER Rare Disease Program ">CBER鈥檚 Rare Disease Program</a><br> <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="3b6eb6bc-08da-4d67-bf14-a9e60e5a6aa2" href="/about-fda/center-drug-evaluation-and-research-cder/accelerating-rare-disease-cures-arc-program" title="Accelerating Rare disease Cures (ARC) Program">CDER鈥檚 Accelerating Rare disease Cures (ARC) Program</a><br> <a data-entity-substitution="canonical" data-entity-type="node" data-entity-uuid="938722b1-90cc-42ea-bb93-08c57efe8d39" href="/news-events/fda-voices/fda-rare-disease-innovation-hub-enhance-and-advance-outcomes-patients" title="FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients">FDA Voices: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients</a></p>   </div> <aside class="col-md-2 col-md-push-2" role="complementary"> <section id="block-entityviewcontent-4" data-block-plugin-id="entity_view:node" class="block block-ctools block-entity-viewnode clearfix"> <div class="region region-"> <aside class="lcds-card lcds-card--border-top"> <ul role="menu" class="lcds-description-list"> <li role="menuitem" class="node-current-date lcds-description-list__item"> <div> <h2 class="lcds-description-list__item-heading">Content current as of:</h2> <p class="lcds-description-list__item-text"><time datetime="2024-11-01T00:00:00Z">11/01/2024</time> </p> </div> </li> <li role="menuitem" class="lcds-description-list__item"> <div> <h2 class="lcds-description-list__item-heading">Regulated Product(s)</h2> <ul role="menu" class="lcds-metadata-list"> <li role="menuitem">Biologics</li> <li role="menuitem">Drugs</li> <li role="menuitem">Medical Devices</li> </ul> </div> </li> </ul> </aside> </div> </section> </aside> <aside class="col-md-2 col-md-pull-10" role="complementary"> <section id="block-entityviewcontent-3" data-block-plugin-id="entity_view:node" class="block block-ctools block-entity-viewnode clearfix"> <div class="region region-"> <nav id="section-nav"class="lcds-card lcds-section-nav lcds-section-nav--side hidden-xs hidden-sm"> <ul class="lcds-section-nav__list lcds-section-nav--side__list"> <li> <a href="/news-events/fda-newsroom/fda-voices" class="lcds-section-nav__link lcds-section-nav--side__link lcds-section-nav__parent-link lcds-section-nav--side__parent-link"> FDA Voices </a> <div class="views-element-container form-group"><div class="view view-in-this-section view-id-in_this_section view-display-id-block_4 js-view-dom-id-076950bedf7bc0fd4cf70366b6b54398a4d207dae704235de04e5e040c4d4303"> </div> </div> <div class="views-element-container form-group"><div class="view view-in-this-section-sub view-id-in_this_section_sub view-display-id-block_1 js-view-dom-id-8bd9f4b6682fd027b8546c815593c28ca71c916d43bf5397e43d8c678efa440d"> </div> </div> </li> </ul> </nav> </div> </section> </aside> </article> </main> </div> </div> <div class="region region-subscribe"> <section id="block-entityviewcontent-5" data-block-plugin-id="entity_view:node" class="block block-ctools block-entity-viewnode clearfix"> <section class="lcds-subscribe" id="subscribe"> <div class="row"> <div class="col-sm-12 col-md-3 col-md-offset-3"> <h2>FDA Voices Email</h2> <p>Subscribe to receive FDA Voices email notifications.</p> </div> <div class="col-sm-12 col-md-3"> <form class="form-horizontal" id="GD-snippet-form" action="https://public.govdelivery.com/accounts/USFDA/subscribers/qualify" accept-charset="UTF-8" method="post"> <input name="utf8" type="hidden" value="✓"> <input type="hidden" name="authenticity_token" value="VJuyc1HyD63+xVCkN+K9qiH1bD9zSSJ+BfdR1d4LXjZ0BncrJ/u9dEw/e0U869OeCw6JxhyYXfnZCCGNF1oVkQ=="> <input type="hidden" name="topic_id" id="topic_id" value="USFDA_656"> <label class="sr-only" for="email">Enter your email address to subscribe:</label> <div class="input-group subscribe-group"> <input type="email" name="email" id="email" placeholder="Email Address" class="form-control"> <span class="input-group-btn"><input class="btn btn-primary" type="submit" name="commit" value="Subscribe"></span> </div> </form> </div> </div> </section> </section> </div> <footer class="lcds-footer container-fluid"> <div class="row lcds-footer__primary"> <h2 id="footer-heading" class="sr-only">Footer Links</h2> <nav class="text-center"> <div class="col-sm-4"> <ul class="nav"> <li><a href="/about-fda/about-website/fdagov-archive" >FDA Archive</a></li> <li><a href="/about-fda" >About FDA</a></li> <li><a href="/about-fda/about-website/internet-accessibility" >Accessibility</a></li> </ul> </div> <div class="col-sm-4"> <ul class="nav"> <li><a href="/about-fda/visitor-information" >Visitor Information</a> </li> <li><a href="/about-fda/about-website/website-policies" >Website Policies / Privacy</a></li> <li><a href="/about-fda/jobs-and-training-fda/no-fear-act" >No FEAR Act</a></li> <li><a href="https://www.hhs.gov/vulnerability-disclosure-policy/index.html" >Vulnerability Disclosure Policy</a></li> </ul> </div> <div class="col-sm-4"> <ul class="nav"> <li><a href="/regulatory-information/freedom-information" title="Freedom of Information Act">FOIA</a></li> <li><a href="https://www.hhs.gov/" title="Health and Human Services" target="_blank">HHS.gov</a></li> <li><a href="https://www.usa.gov/" target="_blank">USA.gov</a></li> </ul> </div> </nav> </div> <div class="row lcds-footer__secondary"> <div class="col-sm-12 col-md-6 col-lg-4 lcds-footer__social-links"> <a href="/about-fda/contact-fda" class="btn btn-default btn-md">Contact FDA</a> <a href="https://www.facebook.com/FDA" title="Follow FDA on Facebook" class="no-disclaimer"> <span class="fa fa-facebook fa-2x" aria-hidden="true"> <span class="sr-only">Follow FDA on Facebook</span> </span> </a> <a href="https://x.com/US_FDA" title="Follow FDA on X" class="no-disclaimer"> <svg class="icon-svg-inline fa-2x" aria-hidden="true"> <use href="/themes/custom/preview/assets/images/fa-icons.svg#icon-x-twitter"> </svg> <span class="sr-only">Follow FDA on X</span> </a> <a href="https://instagram.com/FDA" title="Follow FDA on Instagram" class="no-disclaimer"> <span class="fa fa-instagram fa-2x" aria-hidden="true"> <span class="sr-only">Follow FDA on Instagram</span> </span> </a> <br class='visible-xs-inline'/> <a href="https://www.linkedin.com/company/fda/" title="Follow FDA on LinkedIn" class="no-disclaimer"> <span class="fa fa-linkedin fa-2x" aria-hidden="true"> <span class="sr-only">Follow FDA on LinkedIn</span> </span> </a> <a href="https://youtube.com/@US_FDA" title="View FDA videos on YouTube" class="no-disclaimer"> <span class="fa fa-youtube fa-2x" aria-hidden="true"> <span class="sr-only">View FDA videos on YouTube</span> </span> </a> <a href="/about-fda/contact-fda/subscribe-podcasts-and-news-feeds" title="Subscribe to FDA RSS feeds"> <span class="fa fa-rss fa-2x" aria-hidden="true"> <span class="sr-only">Subscribe to FDA RSS feeds</span> </span> </a> </div> <a href="/" title="FDA Homepage"> <div class="visible-lg-block col-lg-4 text-center lcds-footer__logo"> <span class="sr-only">FDA Homepage</span> </div> </a> <div class="col-sm-12 col-md-6 col-lg-4 text-center lcds-footer__contact-number"> <span class="fa fa-phone" aria-hidden="true"> </span> <span class="sr-only">Contact Number</span> 1-888-INFO-FDA (1-888-463-6332) </div> </div>  </footer> <a href="" id="btn-top" class="btn btn-primary btn-top"><span class="sr-only">Back to </span>Top</a> </div> <script type="application/json" data-drupal-selector="drupal-settings-json">{"path":{"baseUrl":"\/","pathPrefix":"","currentPath":"node\/423792","currentPathIsAdmin":false,"isFront":false,"currentLanguage":"en"},"pluralDelimiter":"\u0003","suppressDeprecationErrors":true,"fda_ckeditor_enhancements":{"basePath":"modules\/custom\/fda_ckeditor_enhancements"},"google_analytics":{"account":"G-273DTKB5QW","trackOutbound":true,"trackMailto":true,"trackTel":true,"trackDownload":true,"trackDownloadExtensions":"7z|aac|arc|arj|asf|asx|avi|bin|csv|doc(x|m)?|dot(x|m)?|exe|flv|gif|gz|gzip|hqx|jar|jpe?g|js|mp(2|3|4|e?g)|mov(ie)?|msi|msp|pdf|phps|png|ppt(x|m)?|pot(x|m)?|pps(x|m)?|ppam|sld(x|m)?|thmx|qtm?|ra(m|r)?|sea|sit|tar|tgz|torrent|txt|wav|wma|wmv|wpd|xls(x|m|b)?|xlt(x|m)|xlam|xml|z|zip"},"bootstrap":{"forms_has_error_value_toggle":1,"modal_animation":1,"modal_backdrop":"true","modal_focus_input":1,"modal_keyboard":1,"modal_select_text":1,"modal_show":1,"modal_size":"","popover_enabled":1,"popover_animation":1,"popover_auto_close":1,"popover_container":"body","popover_content":"","popover_delay":"0","popover_html":0,"popover_placement":"right","popover_selector":"","popover_title":"","popover_trigger":"click"},"ajax":[],"user":{"uid":0,"permissionsHash":"3aef67b4ac7d861d8624bf1f7ab2bc9cfc56d3c25b7d709dc15466dfa996dfb0"}}</script> <script src="/files/js/js_jmx6639WuKcEs1DgFSmm8g__5ByGF4YtLnPWvZO-XrM.js?scope=footer&delta=0&language=en&theme=preview&include=eJxlj0FuAzEIRS_khG4q9TYWg7Fj1TYW0ETT0yeTqJMo3SD4SI9HluF4YZPOkJ_9MRSR0jjiwLZ6JYP3INhqzh0WNA5T-Vz5AqXJgu1gvrY6yr-YtE63sIi4ueKEKVPOrCEnjPTNqbpo5HHCQdx5uAESiaYqI7qUTcBOok6SOGxog3s9dkk_jR8c6bPVjfDYxc-Pr5cw5NqcFQin37Ah32SGOBvs3e5Nir8rl-cjCef99N-8yxx2zyuWXoVv"></script> <script type="text/javascript" src="/Dc2Go5/dCelR/Sewog/rk5-/5VYNNfD46DcX/DQdKdDY/XS/EsHSMaAwQB"></script></body> </html>

CINXE.COM

FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub | FDA