Today, the EveryLife Foundation is thrilled to learn of FDA’s establishment of the Rare Disease Innovation Hub, an intercenter collaboration to address common scientific, clinical, and policy issues related to rare disease product development, including relevant cross-disciplinary approaches related to product review, and promote consistency across offices and Centers.
While interest in rare disease therapy development has increased since the passage of the historic Orphan Drug Act of 1983, the regulatory systems we have in place struggle to meet the unique challenges and complexities inherent in rare diseases.
Time is the most precious commodity for our rare disease community. Each time a promising therapeutic target faces delays or demise due to the complexities in rare disease or strain on the existing regulatory infrastructure, investment wanes, scientific promise goes unfulfilled, and lives are lost.
In 2018, our community together conceived of a solution at the EveryLife Foundation Scientific Workshop and, since that time has been advocating for the formation of a rare disease intercenter within FDA. Alongside our broad coalition partners and Congressional rare disease leaders, we championed the STAT Act in 2021. In 2022, our Congressional champions advocated for the formation of an FDA Task Force focused on rare diseases. And this year, EveryLife Foundation and our advocates worked to lead appropriations requests aimed at the establishment of a rare disease intercenter.
These efforts have laid a strong foundation for the changes we seek. And have led us to this moment.
All along, this effort has been informed by Congressional, scientific, and policy leaders – and championed by thousands of rare disease community members. But most importantly, this effort has been informed by the experiences of our rare disease community. A community with unparalleled unmet need. A community whose research innovations offer much promise, yet who have watched countless therapeutic pipelines disappear due to regulatory inconsistencies, complexities, and hurdles.
We are grateful to FDA for recognizing the opportunity to enhance and streamline their existing processes. As a community, we are eager to support implementation and inform the strategic direction of the Rare Disease Innovation Hub. And we are optimistic that the Hub will address these regulatory challenges, centralizing and optimizing the extensive rare disease expertise across the FDA and organizing FDA rare disease resources, facilitating statisticians, regulatory scientists, and experts in clinical trial design for small populations – all working together to advance therapeutic discoveries alongside rare disease developers and patient communities.
Read the FDA’s Innovation Hub Announcement Here
Read the EveryLife Foundation’ Press Release Here
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About the EveryLife Foundation for Rare Diseases:
EveryLife Foundation for Rare Diseases is a nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation works to improve the lives of the millions of Americans suffering from rare diseases by advocating for policies that foster innovation and remove barriers to life-saving treatments. Founded in 2009 and headquartered in Washington, DC, the EveryLife Foundation for Rare Diseases has programs throughout the United States. To learn more, visit EveryLifeFoundation.org.
