To inform the understanding of how PFDD and related guidances can be applied to therapeutic development for rare diseases, the RARE Foundation joined with the National Health Council, BIO, and PhRMA to convene a collaborative effort to assess the existing patient-focused medical product development guidances through the lens of rare disease medical product development.
A series of four virtual workshops were conducted between June and October 2021 to consider the application of these guidances across four lifecycle stages of medical product development. The project steering committee members enlisted subject matter experts to help address the following questions:
- What stage-critical considerations and decisions can benefit most from patient experience? What are likely to be positive effects of gaining patient experience at this stage of development?
- For what stage-critical considerations and decisions is FDA guidance clear and appropriate for application in rare diseases?
- For what stage-critical considerations and decisions is FDA guidance challenging to achieve in the application to rare diseases? Why?
- What stage-critical considerations and decisions would benefit from additional guidance from FDA? Is it important that this additional guidance be specific to rare disease, or might it have broader utility?
- Are there citable examples (in the public domain) where regulatory decisions seem to support or depart from PFDD-related guidance?
The summaries of these Roundtables informed the creation of the “Guide to Patient Involvement in Rare Disease Therapy Development,” released in January 2022.
