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New guidance eliminates requirement for identifiable, individual-level patient data in RWE for some medical device submissions

On Dec. 15, the Food and Drug Administration  removing a long-standing expectation that identifiable, individual-level patient data must always accompany real-world evidence (RWE) submitted in marketing applications for certain medical devices. The new guidance clarifies that the agency may accept RWE derived from real-world data sources even when sponsors of those devices are unable to provide patient-identifiable record-level datasets — for example, due to privacy, contractual, or governance constraints.

While this policy change only affects certain device submissions for now, FDA says it intends to consider similar updates to its guidance for drugs and biologics in the future.

Why did FDA change its policy on real-world evidence?

FDA explains they updated this guidance because prior RWE expectations for private, confidential information at the individual patient level created a "major barrier," making it impractical to leverage many large-scale data resources. The agency observed that meaningful evidence can sometimes be derived from large data sources and networks without submitting private individual information, citing examples such as the National Cancer Institute's Surveillance, Epidemiology, and End Results (SEER) Program, hospital databases, insurance claims, and electronic health record (EHR) networks. Agency reviewers will now evaluate the strength of submitted RWE on an application-by-application basis. 

What are the practical implications for sponsors and manufacturers?

According to FDA's announcement, "past agency expectations have meant that most RWE could not be considered in product applications." This shift is likely to be most relevant for organizations that have struggled to use RWE because they could not share identifiable, record-level data, even when the underlying data sources were large and potentially informative.

Nevertheless, the FDA's removal of a blanket expectation for identifiable data does not remove the need for defensible evidence. Sponsors and manufacturers will still be expected to enable clear, transparent documentation of data provenance and governance, privacy controls, endpoint definitions and validation, bias management, and reproducibility/auditability for their RWE.

Organizations considering leveraging RWE in their regulatory submissions can evaluate how this shift in FDA guidance may expand the feasible set of data sources available to support their strategies for demonstrating reliability, relevance, and fit-for-purpose to FDA reviewers.