Patient Focused Drug Development Grant Program Awards Today, as part of Patient Focused Drug Development (PFDD) efforts, the U.S. Food and Drug Administration (FDA) announces two new grant awards 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-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. 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. 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.
Final additional information at: CDER Pilot Grant Program: Standard Core Clinical Outcome Assessments (COAs) and their Related Endpoints | 
No comments:
Post a Comment