FDA Rare Disease Innovation Hub
FDA’s Rare Disease Innovation Hub (the Hub) serves as a point of collaboration and connectivity between FDA’s medical products centers – the Center for Biologics Evaluation and Research (CBER), the Center for Drug Evaluation and Research (CDER), and the Center for Devices and Radiological Health (CDRH) – 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 CBER, CDER and CDRH, in close collaboration with the FDA’s Oncology Center of Excellence and Office of Orphan Products Development.
The below video provides an overview of the Hub, its mission, structure, and Strategic Agenda.
On February 2, 2026, the FDA Rare Disease Innovation Hub released its Strategic Agenda, publicly communicating its goals and priorities for the upcoming year to the rare disease community. The Strategic Agenda reflects extensive input from and partnership with the larger rare disease community.
Upcoming meetings
- September 29, 2026: RISE to the Challenge: Statistical Considerations for Rare Disease Clinical Investigations
- October 16, 2026: Patient Voice in Rare Disease Medical Product Development
Connect with the Hub
FDA Rare Connections is a newsletter that connects partners in the rare disease and patient advocacy communities with the latest rare disease program updates from across FDA. RDInnovationHub@fda.hhs.gov
Subscribe to FDA Rare Connections
Resources
- CBER Rare Disease Program
- CDER’s Accelerating Rare disease Cures (ARC) Program
- FDA Voices: FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub
- FDA Voices: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
- Public Meeting: Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub
- FDA Rare Disease Educational Materials: A Landscape Assessment, Gap Analysis, and Recommendations Report (PDF 1.7 MB)