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  • Washington Highlights

    AAMC Comments on FDA Patient-Focused Drug Development Draft Guidance

    Daria Grayer, Director, Regulation and Policy
    Heather Pierce, Senior Director, Science Policy & Regulatory Counsel
    For Media Inquiries

    The AAMC submitted a Sept. 27 letter to the Food and Drug Administration (FDA) in response to the agency’s request for comments on the draft guidance, Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. This is the third of four methodological guidance documents in the patient-focused drug development (PFDD) guidance series, intended “to facilitate the advancement and use of systematic approaches to collect and use robust and meaningful patient and caregiver input that can better inform medical product development and regulatory decision making.”

    The AAMC has long supported the FDA’s patient engagement activities in recent comments on efforts to increase diversity in clinical trials through the establishment of a race and ethnicity diversity plan (refer to Washington Highlights, June 24), as well as the first PFDD draft guidance, Collecting Comprehensive and Representative Input (refer to Washington Highlights, Sept. 14, 2018), finalized in June 2020.

    The most recent draft guidance is intended to help sponsors select and develop clinical outcome assessments (COAs) to measure outcomes of importance to patients. The AAMC commended the agency’s interest in collecting patient experience data, emphasizing the need for patients to be “partners and co-developers” of the recommended road map for sponsor selection of COAs. Other key recommendations included:

    • Increase intra-agency and government-wide coordination of patient engagement activities utilizing the FDA’s existing Office of Patient Affairs as a “central entry point” for coordination (refer to Washington Highlights, June 16, 2017).
    • Ensure coordination with the Department of Health and Human Services’ broader diversity goals in furtherance of Executive Order 13985, Advancing Racial Equity and Support for Underserved Communities Through Federal Government, which the AAMC Center for Health Justice provided feedback on (refer to Washington Highlights, July 9, 2021).
    • Collaborate with federal efforts to advance equitable data collection, including the Office and Management and Budget’s current interest in reviewing and revising the statistical standards (Directive No. 15) for collecting race and ethnicity data.
    • Consider potential challenges associated with the identified outcome measures (e.g., “proxy reported outcomes” and “observer-reported outcomes”) and where appropriate, provide clarification or additional guidance.
    • Ensure continued evaluation of the draft guidance once finalized and develop related FAQs and resources to assist with the implementation of all PFDD guidance.