Accelerate RARE: PDUFA VIII Working Group

2:00 - 5:00 pm EST on Monday, September 8, 2025

(by invitation only)

Objective:

Develop stakeholder consensus on regulatory priorities for rare disease–specific enhancements to PDUFA VII.

Prepare a briefing document and a roadmap to meaningfully accelerate rare disease clinical development.

2:00 – 2:45 PM | Welcome, Introductions, Where We’ve Been
Annie Kennedy, Chief of Policy, Advocacy & Patient Engagement, EveryLife Foundation for Rare Diseases

Five “themes” as a lens through which to consider opportunities for regulatory evolution

  • Evidence & Endpoints for Small Populations

  • FDA-Sponsor Interactions

  • RWE, Digital Health Technologies, & Decentralization

  • Patient Experience Data – clinical trials, regulatory review, Ph IV, labeling

  • FDA Resources, Accountability & Transparency

2:45 – 3:30 PM | Breakout Discussions

3:30 – 3:45 PM | Break

3:45 – 4:45 PM | Putting It All Together

4:45 – 5:00 PM | Where We’re Headed

Discussion Agenda