This document provides guidance that is generally applicable to COAs, including patientreported outcome (PRO), observer-reported outcome (ObsRO), clinician-reported outcome (ClinRO), and performance-based outcome (PerfO) measures.
Appendices A, B, C, and D include additional considerations for each type of COA, respectively, and multiple illustrations of conceptual frameworks.
This guidance is intended to help sponsors use high quality measures of patients’ health in medical product development programs.
Ensuring high quality measurement is important for several reasons: measuring what matters to patients; being clear about what was measured; appropriately evaluating the effectiveness, tolerability, and safety of medical products.
Findings from high quality measures may help support regulatory decision-making in a variety of contexts. For example, findings based on a well-defined and reliable COA-based endpoint in an appropriately designed and conducted investigation may be used to support a claim in medical product labeling if the claim is consistent with the findings and the COA’s documented measurement capabilities