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.
On April 29, 2021 the FDA made two awards under this funding opportunity announcement (RFA-FD-21-004). 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 2021 awards are:
- Preparing a clinical outcomes assessment set for nephrotic syndrome [Prepare-NS] – This project will develop and establish a core set of COAs for nephrotic syndrome, with a primary focus on fluid overload. A diverse team of experts and stakeholders will be led by Debbie Gipson, M.D., M.S., of University of Michigan and John Devin Peipert, Ph.D., M.S., of Northwestern University to design studies necessary to identify a rigorous and patient-centered endpoint definition and core set of COAs. The team will work in close consultation with the project stakeholder engagement and clinician expert panels while also incorporating the perspectives of patients, clinicians, regulators, payers, and drug developers. The investigators expect their work to result in a core set of highly relevant COAs that will advance Patient-Focused drug development in nephrotic syndrome.
- Expanding the Observer-Reported Communication Ability (ORCA) Measure: Measuring the communication ability of individuals with rare, neurodevelopmental disorders (NDDs) – This project will expand on the existing ORCA measure, a measurement tool created by Duke University to assess caregiver observations of a child’s ability for expressive communication in nonverbal patients with Angelman Syndrome who experience complex communication needs. The project team, led by Christina Zigler, Ph.D., M.S.Ed., of Duke University, will evaluate changes in communication ability among a broad range of populations with neurodevelopmental disorders (NDDs). The investigators expect their work to result in an expanded use of the ORCA measure in the research field that can capture communication ability across a diverse range of NDDs and provide an endpoint framework for researchers to evaluate interventions using an outcome that is important to families of children and adolescents with NDDs.
Previously, the FDA made three awards under this grant program (RFA-FD-19-006).
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: FDA-2020-N-1727-0003 or go to 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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