FDA Rare Disease Innovation Hub Future Programming; Request for Comments

Dr. Richard Faris of PANTHERx Rare proposes a specific workshop topic for the RISE Fall Series focused on the role of specialty pharmacies in generating real-world evidence (RWE) for rare diseases. He argues that specialty pharmacies are uniquely positioned to provide longitudinal data on treatment effects, safety, and patient outcomes that can supplement clinical trials and support regulatory decision-making.

A law student studying food and drug regulation supports the FDA Rare Disease Innovation Hub's mission and suggests a specific focus for future workshops. They argue that the FDA should prioritize a workshop on real-world evidence (RWE) and natural history data to help navigate the challenges of conducting traditional clinical trials for small patient populations.