NCHR Comments on FDA’s Patient-Focused Drug Development Draft Guidance
July 5, 2023: NCHR supports the FDA's efforts to increase patient engagement in medical product development and emphasizes the need for diverse and representative samples, the importance of transparency in data analysis, and the use of COA-based endpoints in regulatory decision-making and drug labeling.
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July 5, 2023
The National Center for Health Research (NCHR) appreciates the opportunity to submit public comments to the Food and Drug Administration (FDA) regarding their draft guidance on Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments (COAs) Into Endpoints for Regulatory Decision-Making.
NCHR is a non-profit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We have worked closely with hundreds of patients to help them understand the role of the FDA and to train them in basic information about clinical trials, real world evidence, and the difference between statistically significant results and clinically meaningful results. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.
We support FDA’s efforts to increase patient engagement and input into medical product development and testing. We recognize how challenging it is to ensure that the information will reflect the views and experiences of a representative sample of patients. In our experience, the patients that are most likely to be recruited (usually by industry and industry-supported patient organizations) and engaged in these efforts with the FDA are those that are focused on speedy approvals, not evidence of safety or efficacy. Patient and consumer advocates who have been harmed by an unsafe medical product or the off-label use of a medical product want to partner with the FDA on patient-focused drug development but they tell us that they have been excluded from these efforts. That challenge must be met in order to develop treatments that have a more meaningful impact on patients’ lives – benefits that outweigh the risks that patients consider acceptable. Unfortunately, there was not a single mention of the need of a diverse and representative sample in the entirety of this guidance document. Although engaging participants that represent all patients was the focus of Guidance 1, diversity and representation should be key in the selection of all COA-based endpoints. We urge the FDA to include specific guidance on approaches to recruit and maintain study samples that evaluate efficacy that can be generalizable to the larger disease population.
COAs should be used as endpoints to assess safety and tolerability that take into account the different patient perspectives on these issues. The guidance mentions that additional considerations are needed when the COA is to inform risk, but these considerations are not specified, nor are there citations about where these considerations can be found. We urge that the guidance specifically state whether a COA to inform risk is intended to be in the scope of this guidance. We urge that it be included and that the FDA provide specific information about what these considerations are. It is important that sponsors have guidance on how to incorporate COAs into endpoints that cover the full range of patient experience, including safety and tolerability. This information would be invaluable as the FDA works to advance COAs that are meaningful and rigorous.
In addition to gathering patients’ feedback to inform the type of COA to collect, it is essential to ensure transparency in how the data is analyzed and utilized and the weight of COA-related endpoints in regulatory decision-making. We encourage explicitly describing the COA-based endpoints in the labeling of the drugs, to enhance understanding and trust among stakeholders. Further, it would be essential for the FDA to state how it weighs COA-based endpoints in the totality of all evidence collected during clinical trials.
[1] The Food and Drug Administration. (2020). Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-collecting-comprehensive-and-representative-input
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