Comment from Baltazar Ruiz Guerrero

I am a law student studying food and drug regulation and I appreciate the FDA Rare Disease Innovation Hub's commitment to advancing regulatory science for rare disease therapies through the RISE Workshop series. I would suggest that a future workshop focus on the use of real-world evidence (RWE) and natural history data in rare disease drug approvals. Because rare diseases affect small patient populations, traditional randomized controlled trials are often infeasible. Patients, advocacy organizations, and developers would benefit from greater FDA guidance on what types of RWE are sufficient to support accelerated approval or full approval decisions in these contexts. A workshop bringing together patient organizations, clinical researchers, biostatisticians, and FDA reviewers could help harmonize expectations and reduce uncertainty in the development process. This topic is cross-cutting and relevant across many rare disease categories, consistent with the Hub's stated mission. I respectfully urge the FDA to prioritize this issue in upcoming RISE sessions.