As part of our Patient Focused Drug Development (PFDD) efforts, the U.S. Food and Drug Administration (FDA) developed a pilot grant program to support the development of publicly available core set(s) of Clinical Outcome Assessments (COAs) and their related endpoints for specific disease indications (RFA-FD-19-006). On September 11, 2019 the FDA made three awards under this grant program. These awards will provide avenues to advance the use of patient input as an important part of drug development that can foster innovation and the availability of safe and effective drugs.
Patient input can help inform the therapeutic context for regulatory review. Patient input also can inform the selection of clinical outcomes, ensure the appropriateness of instruments used to collect trial data, and help ensure that investigations of the effect of treatments are assessing outcomes that are meaningful to patients. If methodologically-sound data collection tools are developed and used within clinical trials of an investigational therapy, patient input can provide a direct source of evidence regarding the benefits and risks of a drug.
Migraine Clinical Outcome Assessment System (MiCOAS) – This project will develop and standardize a core set of endpoints and related COAs for use across migraine clinical trials. The team of investigators, led by R.J. Wirth, Ph.D., at Vector Psychometric Group and Richard Lipton M.D., at Albert Einstein College of Medicine, will conduct research to further inform COA development and ensure the patient experience is captured and that endpoints address symptoms that are most important to patients suffering from migraines. The investigators expect that their work will advance the development and approval of migraine treatments.