Comment from Ainsley Peterson

I appreciate the opportunity to comment on the future programming of the FDA Rare Disease Innovation Hub. The Hub reflects an important shift in how the FDA approaches rare disease regulation. Given the structural challenges of rare disease drug development, this type of centralized initiative is both necessary and timely. As a law student interested in drug law, I strongly support the continued investment in addressing longstanding challenges in rare disease drug development through collaborative models. From a regulatory perspective, one of the most significant opportunities for the Hub is to help clarify how existing statutory and regulatory authorities can be applied more flexibly in the rare disease context. Many of the frameworks governing approval were not designed with small patient populations in mind. Future programming, especially within the RISE Workshop series, should focus on how FDA can interpret and apply these standards in a way that complies with scientific standards but is still realistic for the rare disease space. Through the RISE Workshop series, the Hub should continue to develop guidance around the use of innovative trial designs and evidence gleaned from these trials. Flexibility and adaptability within trials is necessary in rare disease settings. However, there is still uncertainty around what the agency will consider sufficient to meet approval standards. More transparency would be helpful in reducing regulatory risk and encouraging investment in rare disease drug development and research. The RISE Workshop series can add value by working to improve coordination across FDA centers. Rare disease products frequently raise issues involving drugs, biologics, and devices, which lead to inconsistent expectations as a result. Thus, future programming should more directly address how cross-center alignment will be operationalized in practice when it comes to rare disease innovation. Furthermore, the role of patients in this process also deserves more concrete attention. While patient-focused drug development has been a priority for the agency, the RISE Workshop series provides an opportunity to incorporate patient input more directly. Learning from patients with rare diseases will allow for the industry to address scientific and regulatory challenges more effectively. Finally, the FDA should consider how greater transparency and accountability can be built into the programming. Publishing clear takeaways, identifying next steps, and linking workshop discussions to future policy development would help ensure that workshop engagement translates into meaningful regulatory action. Overall, the Rare Disease Innovation Hub has the potential to play a key role in modernizing how FDA regulates rare disease innovation through the implementation of the RISE Workshop series. Continued focus on regulatory clarity, scientific flexibility, patient challenges, and cross-center coordination will be critical to its success. Thank you for the opportunity to comment.