On October 23, 2025, FDA released its final guidance regarding Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. The guidance is the third guidance in a four-part series of FDA guidance focused on patient-focused drug development (PFDD) that describe how stakeholders, such as patients, caregivers, researchers, medical product developers and others can submit patient experience data and other relevant information that can be used for medical product development and regulatory decision making.
This guidance is intended to help sponsors use high-quality measures of patients’ health in medical product development. According to FDA, the guidance was informed by developments in research and applications of Clinical Outcome Assessments (COAs) to derive clinical trial endpoints. The guidance provides an overview of COAs in clinical trials, including the types of COAs, the role of COAs in evaluating the clinical benefit for a medical product, and determining whether a COA is fit-for-purpose.
The guidance also lays out a roadmap to patient-focused outcome measurement in clinical trials: understanding the disease or condition; conceptualizing clinical benefits and risk; selecting/developing the outcome measure and developing a conceptual frame to arrive at a fit-for-purpose COA. It then discusses developing the evidence to support the conclusion that a COA is appropriate in a particular context of use.
This latest guidance from FDA underscores that patient-focused drug development is an ongoing area of interest for the agency, industry, patient and clinical communities. The issuance of this final guidance also fulfills a PDUFA VI commitment and guidance requirements under the 21stCentury Cures Act. PFDD is likely to continue to be an area of focus in the upcoming PDUFA VIII reauthorization given the broad interest in advancing a patient-focused drug development paradigm.