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.
In 2019, 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.
FDA is in the process of reviewing applications (RFA-FD-21-004) for the next cycle of grants for the Standard Core COA Grant Program.
FDA is particularly interested in applications focusing on the following areas for the development of standard core set(s) under this Funding Opportunity Announcement (FOA):
- COA(s) and associated endpoint(s) to assess volume or fluid overload (hypervolemia) and related impacts, specifically for use in nephrotic syndrome trials
- COA(s) and associated endpoint(s) to assess age-appropriate domains of pediatric daily function for use in clinical trials of pediatric rare diseases.
- COA(s) and associated endpoint(s) that evaluate 1) the mechanics of swallowing needed for safe eating and drinking, and 2) motor production of speech to assess the ability to develop or retain quality oral communication from infancy to adulthood.
- COA(s) and associated endpoint(s) that are clinically meaningful and directly capture how a patient feels, functions, or survives to assess treatment response in Systemic sclerosis.
On September 11, 2019 the FDA made three awards under this grant program (RFA-FD-19-006). 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.
The 2019 awards are:
- 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 RJ Wirth, Ph.D., M.A., at Vector Psychometric Group and Richard Lipton M.D., at Albert Einstein College of Medicine, will conduct patient-centered qualitative 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. Additional information on this project, and milestone documents can be found on the project website.
- Clinical Outcome Assessments for Acute Pain Therapeutics in Infants and Young Children (COA APTIC) – This project, led by Kanecia Zimmerman M.D., M.P.H., and Bryce Reeve Ph.D., M.A., at Duke University, will identify COAs and endpoints for use when developing acute pain therapeutics in infants and young children, primarily those 0-2 years. The team will engage with patients, caregivers and clinicians to identify meaningful outcomes for acute pain clinical trials and will further research potential clinician-, observer-, and patient-reported COAs in a prospective trial among infants and young children. The investigators expect that their work will facilitate the development of treatments that provide safe and effective pain management in infants and young children. Additional information on this project, and milestone documents can be found on the project website.
- Northwestern University Clinical Outcome Assessment Team (NUCOAT) – This project will develop and validate COAs with applicability across a range of chronic conditions that assess physical function using patient-reported and performance outcomes. The team of investigators, led by David Cella Ph.D., M.A., at Northwestern University, will research a full range of physical function severity. The investigators expect their work to result in core physical function outcome sets that measure the full range of physical function severity with potential generalizability across conditions. Additional information on this project, and milestone documents can be found on the project website.
SUBMIT A COMMENT TO THE DOCKET
We encourage stakeholders to submit written comments on the grantee milestone documents and public meeting content to the docket: https://www.regulations.gov/document?D=FDA-2020-N-1727-0003 or go to https://www.regulations.gov/ and search for: FDA-2020-N-1727. This docket will remain open throughout the remainder of the grant program.
For more information on these awards please query the NIH RePORTER database.
COMPLETED August 28, 2020: Public Meeting on CDER Standard Core Sets Clinical Outcome Assessments and Endpoints Grant Program
- The U.S Food and Drug Administration (FDA) is hosting a public meeting to provide an opportunity for grantees funded as part of the FDA Standard Core Clinical Outcome Assessments (COAs) and Endpoints Pilot Grant Program to share their development plans for the standard core COA sets and to receive feedback from stakeholders. FDA will provide an introduction and discuss plans for the pilot grant program including future public meetings.
COMPLETED December 5, 2019: Public Meeting on CDER Standard Core Clinical Outcome Assessments (COAs) and Endpoints Pilot Grant Program
- The FDA hosted a public meeting which provided an opportunity for grantees funded as part of the FDA Standard Core COAs and Endpoints Pilot Grant Program to share their development plans for the standard core COA sets and to receive feedback from stakeholders. FDA provided an introduction and discussed plans for the pilot grant program including future public meetings.
For any inquiries related to the grant program, please email CDER_StandardCoreCOAs@fda.hhs.gov.
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