The U.S. Food and Drug Administration (FDA) has released the final version of its long-anticipated guidance on patient-focused drug development (PFDD), marking a significant step in advancing the collection and use of patient experience data in clinical research. This guidance is the third in a four-part series that supports the requirements outlined in the 21st Century Cures Act, which calls for patient perspectives to be more effectively integrated into medical product development and regulatory decision making.
Strengthening Patient Experience Data in Drug Development
The latest final guidance provides a comprehensive framework for how sponsors should select, modify, and evaluate clinical outcome assessments (COAs). The FDA emphasizes that high-quality patient-focused measurements are essential for several reasons: they help ensure that clinical trials measure what truly matters to patients, improve clarity around what is being assessed, and strengthen the evaluation of a product’s safety, tolerability, and clinical benefit.
These COAs play a fundamental role in demonstrating the impact of a medical product on patient health. By refining the standards and expectations around COA development and implementation, the FDA aims to enhance the quality and consistency of evidence used to support product approvals.
Understanding the Four Types of Clinical Outcome Assessments
The guidance details four types of COAs that may be used to assess patient outcomes in clinical trials:
- Patient-reported outcomes (PROs)
- Observer-reported outcomes (ObsROs)
- Clinician-reported outcomes (ClinROs)
- Performance outcomes (PerfOs)
Importantly, the FDA discourages the use of proxy-reported outcomes when assessing a patient’s subjective experience. When self-reporting is not possible, the agency recommends using an observer-reported assessment that captures observable behaviors rather than relying on assumptions about how the patient feels.
A Roadmap for Selecting and Developing Fit-for-Purpose COAs
A key feature of the guidance is the inclusion of a “roadmap to patient-focused outcome measurement.” This roadmap provides sponsors with a structured pathway for identifying or developing COAs that are suitable and scientifically validated for their intended context of use.
Throughout this process, early and ongoing communication with the FDA is encouraged. Sponsors are advised to describe their COA-based endpoint strategy when engaging with the agency to ensure alignment and minimize regulatory uncertainty.
Notable Updates from the Draft Guidance
Several clarifications and enhancements have been incorporated into the final version, including:
- Clearer relationships between key terminology
- A restructured conceptual framework explaining how COA-based endpoints support regulatory decisions
- Additional emphasis on the role of qualitative data and direct patient input
- Updated scientific references and evidence expectations
- New considerations for how COAs should be administered within clinical trials
The FDA also introduced a new COA concept, the “meaningful aspect of health,” which focuses on attributes of health that are important to patients and can be directly improved by treatment.
At EMMA International
At EMMA International, we support organizations in developing patient-centered clinical programs that align with FDA expectations. Our experts help ensure that COA strategies, endpoint development, and patient experience data are incorporated seamlessly into regulatory and quality frameworks. From early engagement planning to documentation and submission readiness, we provide guidance that keeps your development program compliant and patient focused.
For more information on how EMMA International can assist, visit www.emmainternational.com or contact us at (248) 987-4497 or info@emmainternational.com.
Reference:
U.S. Food and Drug Administration. Patient-Focused Drug Development: Selecting, Developing, and Modifying Clinical Outcome Assessments. Final Guidance, November 2025.
Federal Register Notice. FDA Finalizes PFDD Guidance under 21st Century Cures Act, 2025.
