Rare Disease Framework Initiative Public Meeting

About the meeting 

On October 28, 2026, the U.S. Food and Drug Administration, in partnership with the Reagan-Udall Foundation for the FDA, convened a hybrid public meeting to seek input related to the FDA’s Rare Disease Framework Initiative to reimagine the agency’s approach to rare disease drug development. The intent of this new initiative is to develop a fit-for-purpose Framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type, and the evidence that realistically can be generated in small patient populations. 

Opportunity for comment 

The FDA has opened a public docket for comments related to the Rare Disease Framework Initiative.

Comments can be submitted electronically through Regulations.gov or as written/paper submissions and are due December 11, 2026. Commenters should reference Docket No. FDA-2026-N-12214. To submit a written comment, click here.

Topics for input

Feedback is welcome related to the Framework Initiative, and in particular, the following topic areas: 

• Fit-for-purpose review of rare disease therapies based on attainable evidence, particular disease state, and population
• FDA consistency and predictability of drug development and review regulatory requirements
• FDA engagements with external parties – including patients, disease experts, and researchers – to inform review decision-making
• Considerations for chemistry, manufacturing, and controls (CMC) specific to rare disease therapies

This meeting is funded by the U.S. Food and Drug Administration, Center for Drug Evaluation and Research, under FDA contract 75F40126C00214.