FDA announces grant awards for projects supporting the use of real-world data to generate real-world evidence in regulatory decision-making

The US Food and Drug Administration (FDA) has announced the award of four new U01 grants in 2023 for select projects addressing aspects of the use of real-world data (RWD) to generate real-world evidence (RWE) in regulatory decision-making. These awards are the latest step in the agency’s endeavors to encourage innovative approaches to further support the use of RWE while ensuring that scientific evidence supporting marketing approvals meet FDA’s evidentiary standards.

The FDA is committed to an ongoing extensive drive to realize the full potential of RWD and RWE in the development and regulation of therapeutic products — key landmarks are displayed in The Evidence Base’s Infographic: Milestones in the FDA’s Real-World Evidence activities. One component of this drive is an award program for grants to fund select research projects to advance the use of RWE, and this has contributed to a series of ongoing and completed RWD/RWE Demonstration Projects.

Under the award program, four U01 grants were awarded in 2020, and the FDA has announced that a further four new projects have been selected in their 2023 funding opportunity. The titles of the new research projects are:

• Methods to improve efficiency and robustness of clinical trials using information from real-world data with hidden bias — Project leads: Xiaofei Wang, Ph.D., at Duke University and Shu Yang, Ph.D., at North Carolina State University
• Generating reproducible real-world evidence with multi-source data to capture unstructured clinical endpoints for chronic diseases — Project leads: Tianxi Cai, Sc.D., and Florence Bourgeois, M.D, M.P.H., at the Harvard-MIT Center for Regulatory Science and Harvard Medical School
• Real-world data to generate real-world evidence in regulatory decision-making — Project lead: Peter O’Dwyer, MD at the ECOG-ACRIN Medical Research Foundation
• Development of novel methods to enable robust comparison of real-world progression free survival (rwPFS) and clinical trial PFS in multiple myeloma — Project leads: Khaled Sarsour, Ph.D., Ashita Batavia, M.D., M.S., and Jennifer Hayden, M.S. at Janssen Research & Development, LLC

The FDA grant awards webpage features a precis of the newly awarded projects and those awarded in 2020.

See also our recent coverage of an FDA article on aligning real-world terminology.