Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub

37 stakeholders provided public comment during the live meeting. Many naturally aligned with the emerging meeting theme of five Cs:

• Communication: Effective and transparent communication was highlighted as a foundation for addressing the complexities of rare disease drug development. 
• Community: Building an inclusive and engaged community was emphasized as a critical element for sustained progress in rare disease drug development initiatives. 
• Coordination: Enhanced coordination with external organizations and across FDA Centers and review divisions was seen as pivotal for reducing inefficiencies and aligning efforts in the rare disease drug development space. 
• Creativity: Innovative approaches to clinical trial design and drug development were viewed as essential to addressing the unique challenges of rare diseases.
• Cooperation: Collaboration between stakeholders was identified as a critical driver of progress in rare disease drug development.

The public comments reflected the community’s perspective on challenges and opportunities inherent in rare disease drug development and some outlined strategies to enhance regulatory processes, foster innovation, and deliver meaningful patient outcomes. In addition, some comments, as presented, would be outside the scope of FDA’s authority.

Almost without exception, the comments fell into high-level themes that broadly address approaches for external and within/cross Agency Engagement and for Innovation.