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 member of the public supports the proposed workshops for bringing together laypeople and scientists to address the challenges of rare diseases. The commenter suggests that the workshops should specifically focus on equity issues for underrepresented groups and emphasizes the need for ongoing initiatives to improve care for these patients.