The U.S. Food and Drug Administration (FDA) has announced a significant update to its approach to real-world evidence (RWE), removing a major limitation that has long restricted its use in drug and medical device reviews. Under new guidance for certain medical device submissions, the FDA will now accept RWE without requiring the routine submission of identifiable, individual-level patient data. The agency has also indicated that similar updates may follow for drugs and biologics.
Although RWE has been recognized as a valuable tool in regulatory decision-making, previous FDA requirements meant that much of this data could not be practically used. Since 2016, only 35 drug, biologic, or vaccine applications have incorporated RWE, compared with more than 250 medical device authorizations. Even in the device space, however, the use of RWE has recently slowed due to data submission challenges.
Historically, the FDA required RWE to include detailed, patient-level information, making it difficult to leverage large databases that primarily offer aggregated or de-identified insights. This new policy reflects a shift in thinking, acknowledging that robust, meaningful conclusions can be drawn from high-quality real-world data without compromising patient privacy.
Going forward, FDA reviewers will evaluate the quality and relevance of RWE on a case-by-case basis, rather than applying a one-size-fits-all data requirement.
FDA Commissioner Marty Makary, M.D., M.P.H., emphasized that this change removes unnecessary obstacles and accelerates patient access to important therapies. He noted that the updated approach will enable the FDA to better utilize large, established data sources such as cancer and cystic fibrosis registries to understand how treatments perform in real-world settings.
This policy shift significantly expands the FDA’s ability to use de-identified datasets containing millions of patient records, including national disease registries, hospital databases, insurance claims, and electronic health record networks. These resources provide a broader and more diverse view of patient outcomes, complementing traditional clinical trials and strengthening regulatory decision-making.
Learn more: FDA Eliminates Major Barrier to Using Real-World Evidence in Drug and Device Application Reviews
