This was an in-person meeting and attendance was restricted to panel members. However, the public was welcome to listen in to the full meeting via webinar/teleconference.

We encouraged comments or questions for the panel at [email protected].

Members of this Advisory Panel

A portion of this meeting was devoted to small group discussions of 3 key issues: (1) human subject issues specific to rare diseases, (2) the importance of and best practices for research prioritization, and (3) considerations related to the challenges with producing reliable evidence for rare diseases.

Meeting Materials

• Agenda
• PCORI's Guidance on Research in Rare Diseases
  • Related PCORI Blog post: New PCORI Guidelines for Research on Rare Diseases
• Breakout Sessions -- 10:30 a.m. - 12:15 p.m. (ET)
  • 1. Human Subjects: Background Material
  • 2. Research Prioritization: Background Material
  • 3. Challenges with Producing Reliable Evidence for Rare Diseases: Background Material
• Presentation Slides
• Meeting Summary

Teleconference Audio Recording - Main Session Part 1

Teleconference Audio Recording - Main Session Part 2

Teleconference Audio Recording - Breakout Session 1

Teleconference Audio Recording - Breakout Session 2

Teleconference Audio Recording - Breakout Session 3