COA-Based Endpoints Covered in FDA Patient-Focused Drug Development Guidance
The FDA has published the fourth and final guidance in its patient-focused drug development series, offering stakeholders recommendations for incorporating clinical outcome assessments (COA) into trial endpoints.
Specifically, the lengthy 48-page draft guidance provides recommended approaches, technologies and standards for gathering and assessing COA data as well as advice on choosing, justifying and creating COA-based endpoints and determining clinically meaningful change in those endpoints.
The content focuses on endpoints built from fit-for-purpose COAs that are meant to demonstrate how patients feel, function or survive, whether directly (a patient-reported outcome instrument measuring the intensity of pain, for instance) or indirectly (such as clinician-reported outcome instruments that track disease activity or extent). For indirect COAs, the guidance notes the importance of understanding how COA-based endpoints correspond to changes that are meaningful and relevant to patients and contains a section on evaluating the meaningfulness of treatment benefit.
The bulk of the guidance discusses considerations to take into account when selecting, justifying and creating COA-based endpoints, common statistical analysis considerations and recommendations for supporting data, potential ways to gather them and how they can be applied to help with the interpretation of trial results.
The guidance also contains an “additional considerations” section that touches on masking, general strategies for mitigating practice effects, the use of assistive devices by participants, and considerations for trials using non-randomized designs, external controls or nonconcurrent controls, among other items. It also offers stakeholders guidance resources for formatting and submitting patient experienced data.
Comments on the guidance are due by July 5.
Read the full draft guidance here: https://bit.ly/3GisobA.
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