The RARE Foundation submitted comments in response to the FDA’s request for information on the impacts of Patient-Focused Drug Development (PFDD) meetings. Comments reflected input from the Foundation’s Community Congress coalition partners as well as previous work from the Foundation’s PFDD Compendium Series.
The fifth reauthorization of the Prescription Drug User Fee Act in late 2012 was foundational to today’s Patient-Focused Drug Development (PFDD) ecosystem. PDUFA V established a formalized engagement between regulators and patient community experts through FDA’s initiation of the first twenty Patient-Focused Drug Development Workshops, the success of which gave rise to the expanded Externally-Led PFDD workshop mechanism. These meetings represented a transformative shift in how the FDA engaged with patient communities, creating a structured process to systematically capture insights and data on disease progression, community subpopulations, lived experiences, treatment priorities, and benefit-risk perspectives of both patients and caregivers.
PFDD workshops and progress, thanks to expanded PFDD-related work in PDUFA VI and VII and the 21st Century Cures Act, have established a scientific and regulatory framework for systematically incorporating patient perspectives into drug development and review. Thanks to PFDD initiatives, patient-centered clinical development programs are becoming the norm rather than the exception, helping ensure that therapies for rare diseases are evaluated based on outcomes that matter most to patients and families.
Recently, the FDA issued a request for information to understand how patient input from FDA-led PFDD and EL-PFDD meetings has informed stakeholder activities, including research, product development, and patient care, outside of specific regulatory decisions. In addition to reflecting on the contributions of the existing PFDD meeting mechanisms, the RARE Foundation made several suggestions related to the ongoing evolution of PFDD efforts, especially timely as the reauthorization of PDUFA VIII takes shape in the coming year. Examples of the suggestions include:
- The implementation of Science-Focused Drug Development (SFDD) meetings that complement PFDD to proactively and systematically discuss drug development considerations in particular rare diseases or groups of rare diseases that share a specific drug development challenge (e.g. study design, study duration, intended population, endpoints).
- The creation of additional infrastructure to develop outcome measures and measures that support multiple conditions.
- To provide more information on how and when to engage with the FDA through PFDD convenings to help patient communities focus their efforts towards relevant and actionable progress for therapy development.
- The creation of “Type P Meetings”, a meeting mechanism for patient advocacy organizations to discuss PFDD follow-up actions, including ways for relevant data and/or expertise to be considered as part of the outcome review process.
- To direct appropriate resources to the Rare Disease Innovation Hub to help facilitate these additional meetings.
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Additionally, this week, the RARE Foundation submitted comments in response to Representative Jake Auchincloss’ request for feedback on a legislative discussion draft of the Next-Generation U.S. Clinical Development to Accelerate Cures Proposal. This draft focuses on modernization and innovation throughout the biomedical product development cycle. The Foundation’s comments focused on supporting Representative Auchincloss’ proposal to codify the Rare Disease Innovation Hub in law and supply the Hub with specified duties and funding. We are encouraged by this proposal and the recognition of the important mission of the Rare Disease Innovation Hub, a position that is in line with the community’s appropriations requests for FY27 funding.
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