The FDA’s Rare Disease Innovation Hub (the Hub) has released its Strategic Agenda, which outlines the actions the Hub plans to undertake during its first year with extensive involvement from the rare disease community, while also addressing questions about the ultimate structure and programs of the Hub. The Strategic Agenda reflects extensive input from and partnership with the larger rare disease community, and identifies the following goals for the Hub:
- Goal 1 – Further Advance Regulatory Science of Rare Disease Therapies
- Goal 2 – Enhance and Strengthen Coordination and Alignment Between FDA’s Medical Product Centers, with Particular Focus on the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER)
- Goal 3 – Create a Centralized Point of Contact for External Partners
It is the hope and expectation of FDA leadership and the Hub that this Agenda will evolve as the Hub evolves. It is also the expectation that the rare disease community will remain heavily engaged in this evolution. Throughout the year, there will be multiple ways for the community to engage with and offer suggestions to the Hub, both for immediate use and for consideration for the Hub’s 2026 Strategic Agenda.
Fundamental to the mission of the U.S. Food and Drug Administration is to engage patients and caregivers – to understand their unique perspectives and experiences and keep these front of mind as we review medical products for rare disease patients.
An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare diseases and conditions are life threatening, and most do not have approved treatments.
FDA created the Rare Disease Innovation Hub (the Hub) to serve as a point of collaboration and connectivity between Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) with the goal of ultimately improving outcomes for patients. Although the Hub will work across rare diseases, it will particularly focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.
The Hub is co-led by the directors of Center for Biologics Evaluation and Research (CBER) and the FDA’s Center for Drug Evaluation and Research (CDER), in close collaboration with the FDA’s Center for Devices and Radiological Health, Oncology Center of Excellence, Office of Orphan Products Development, and Office of Combination Products.
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