The Reagan-Udall Foundation for the FDA convened an invitation-only roundtable conversation with industry stakeholders and patient representatives to discuss challenges in rare disease drug development and seek ways to enhance the efficiency and agility of generating evidence for regulatory review of rare disease medicines. Our topline learnings summary report, capturing the insights from this roundtable, is now available.
Alnylam, argenx, BridgeBio, Jazz Pharmaceuticals, Lilly, and Stoke Therapeutics provided funding for this meeting.