The last draft guidance on using clinical outcomes assessments (COA) as endpoints in patient-focused drug development (PFDD) should use terminology more consistently, align more closely to other PFDD guidance documents in the series, and provide more high-level examples of concepts outlined in the guidance, according to parties who commented on the US Food and Drug Administration (FDA) final draft guidance in their series.
The draft guidance describes a number of considerations for COA-based endpoints in PFDD, such as how to select an endpoint, recommendations for analyzing endpoint results, how to handle missing data, and other study design considerations (RELATED: FDA issues last guidance in patient-focused drug development series, Regulatory Focus 05 April 2023).
As with other documents in the PFDD series, stakeholders generally expressed support for FDA’s efforts to include patient voices in drug development. Some commenters of previous draft guidance documents in the series were unclear about whether the PFDD series was intended to replace the agency’s 2009 document, “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims,” and said they appreciated that FDA clarified this point for stakeholders in the draft guidance on COA-based endpoints in PFDD (RELATED: Stakeholders want more clarity on concepts introduced in third PFDD draft guidance, Regulatory Focus 03 October 2022)…
