Patient-focused drug development presents increasing regulatory and methodological challenges for sponsors seeking to generate evidence that is both scientifically robust and meaningful to patients.
With the FDA’s 2025 Patient-Focused Drug Development (PFDD) Guidance 3, expectations are rising for how clinical outcome assessments (COAs) are selected, justified, and implemented across development programs. The focus is shifting from traditional, clinician-driven endpoints to fit-for-purpose, patient-centered measures that directly capture how patients feel and function, while meeting evidentiary standards for regulatory decision-making.
Read our guide to explore more about:
- How FDA’s 2025 PFDD Guidance shapes COA selection and regulatory expectations
- How to determine whether a clinical outcome assessment is truly fit for purpose
- How to link patient priorities to meaningful health outcomes and context of use
- How to build robust qualitative and quantitative evidence to support labeling and approval