Request for Information (RFI): FDA Standard Core Clinical Outcome Assessments and Endpoints

Description

Purpose FDA is seeking information submissions from the public. Information is being solicited to inform planned work to promote development of publicly available standard core sets of Clinical Outcome Assessment (COA) measures for specific disease indications. A core set would reflect measures, tools, and endpoints that assess a minimum list of impacts that matter most to patients and are likely to demonstrate change relating to disease burden, treatment burden, and, if applicable, physical function. The information requested may be used to structure a program to facilitate the development of publicly available core set(s) of COA measures and endpoints. Under the envisioned program qualifying third parties would conduct a well-managed, transparent, and methodologically sound process following a development protocol that provides for: 1) Consistent application of appropriate methods for COA set development applying new FDA patient-focused drug development methodological guidance as appropriate; 2) Consideration and use of publicly available measures that have already undergone extensive vetting whenever possible; 3) Publication of interim work products at each milestone in the COA set development process to make them publicly available for all stakeholders to review, comment, and utilize; and 4) Engagement of key stakeholders from the patient disease advocacy community, FDA medical product reviewers, regulated medical product industry, government and academic researchers, health care providers, health technology assessors and health payers, and others, at key milestones via public meetings and workshops, to ensure that identified concepts, measures, tools, and endpoints reflect what is most important and relevant in stakeholder decision making. Background Since 2012 FDA has been conducting patient-focused meetings in different disease areas to hear directly from patients about the impact of disease on their daily life, and the burden of current treatments. Patient experiences are providing new insights for FDA reviewers and reinforce the importance of incorporating the patient’s experience to inform drug development. This includes not only the clinical context for FDA decisions, but more direct evidence regarding drug benefits and risks. This can be achieved when methodologically sound COA measures, tools and endpoints that are developed and deployed in clinical trials provide evidence for FDA decision making that can be incorporated in drug labeling to better inform decisions by doctors and patients at the point of care. However, there is currently very little coordination of development of these COAs across all the diseases that need them. The separate independent efforts by different patient advocacy groups and drug sponsors within and across disease areas are resulting in a diversity of measures and proprietary COA tools that create a continued “learning curve”, a higher level of investment by these groups, and fewer opportunities for streamlining than would be best for long-term sustainability. Repeated significant start-up costs and varying levels of experience among sponsors, advocacy groups, and the consultants they may engage, may lead to variable quality in the resulting COA related endpoints. Inconsistent quality in COAs both limits their value to decision makers and can potentially undercut confidence in these data. These challenges can discourage company investment in obtaining the patient’s perspective and experience to inform decision making despite the potential to improve development and delivery of treatments for patients. PLEASE CLICK ON LINK TO SEE ADDITIONAL INFORMATION

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