Article Summary:
The FDA has issued a final guidance that removes the requirement for identifiable patient-level data in medical device submissions using real-world evidence (RWE). The guidance acknowledges that meaningful information can be derived from large de-identified databases, such as cancer and cystic fibrosis registries, without needing access to individual patient data.
This policy change enables the use of a wider range of real-world data sources, including cancer registries, hospital systems, insurance claims, and electronic health records. The FDA will now assess the strength of submitted RWE on an application-by-application basis, rather than requiring patient-level data in all cases.
The guidance also provides new examples of when the FDA will and will not accept investigational device exemptions (IDEs) that leverage real-world data (RWD) in submissions, as well as a demonstration of how RWE can be obtained for a device under emergency use authorization (EUA).
The final guidance complies with a Congressional mandate under the Food and Drug Omnibus Reform Act of 2022 (FDORA) and a commitment made in the Medical Device User Fee Amendments Performance Goals and Procedures for Fiscal Years 2023 through 2027 (MDUFA V) to clarify the use of RWE and RWD in regulatory decision-making. The FDA has reported that the integration of RWE into device approvals has been more extensive than for drugs and biologics, with over 250 premarket authorizations including RWE since 2016, compared to 35 drugs, biologics, or vaccines in the same timeframe.