A recent publication from Triangle CERSI’s Patient-focused Approaches to Rigorous Clinical Research (PARCR) team highlights an important advancement in how clinical outcome assessments (COAs) are validated in support of regulatory decision-making and patient-focused drug development.
Published in Value in Health, the article “Applying the Argument-Based Approach to Validation With Clinical Outcome Assessments: Strategies for Constructing a Rationale,” co-authored by by Kevin Weinfurt, R.J. Wirth, Mike Edwards, and Bryce Reeve, describes a structured approach to validating clinical outcome assessments (COAs) that is closely aligned with the FDA’s Patient-Focused Drug Development (PFDD) Guidance. The authors outline the argument-based approach to validation, which emphasizes clearly defining how and why a COA score can be interpreted as reflecting a meaningful aspect of patient health. Rather than relying on a checklist of validation activities, this approach focuses on building a transparent, evidence-based rationale and evaluating the assumptions underlying how a measure works in a specific context of use.
The authors present two practical strategies to guide this process: systematically reviewing each step in how an assessment is designed and implemented, and identifying potential threats to validity, including missing aspects of the patient experience or external factors that could influence results. The paper also provides adaptable starting frameworks across multiple COA types, including patient-reported, clinician-reported, observer-reported, and performance outcomes.
This work reflects Triangle CERSI’s broader mission to advance the scientific foundations of regulatory science by developing methods that improve the quality, interpretability, and patient relevance of clinical evidence. By strengthening confidence in how outcomes are measured, these approaches support more informed FDA decision-making and help ensure that clinical trial results better capture what matters most to patients.
