FDA finalizes long-awaited guidance on using real-world evidence for medical device decisions

The FDA has finalized its guidance on the use of real-world evidence (RWE) to support regulatory decision-making for medical devices, replacing its 2017 framework and finalizing the 2023 draft guidance. The updated document provides clearer expectations around data relevance, reliability, and documentation, offering sponsors a more practical roadmap for integrating real-world data (RWD) into regulatory submissions.

As anticipated following earlier announcements this week, the US Food and Drug Administration (FDA) has published its long-awaited final guidance, Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices. The guidance, coming 2 years after the release of the draft version, expands on how RWD can be used to generate RWE in support of regulatory submissions and reflects both legislative direction under the Food and Drug Omnibus Reform Act of 2022 (FDORA) and the FDA’s broader efforts to modernize evidence generation across the medical device lifecycle.

The 44-page guidance provides greater clarity and structure around the FDA’s expectations for RWE, explaining how the agency evaluates whether RWD are fit-for-purpose and when their use may be appropriate to inform or support a particular regulatory decision. A central theme throughout is the need for sponsors to understand and clearly articulate the strengths and limitations of the underlying data, how these characteristics affect relevance and reliability in the context of the regulatory question being addressed, and how these assessments are supported by clear, well-documented evidence in regulatory submissions.

Importantly, the FDA emphasizes that these considerations apply regardless of the RWD source. Electronic health records, administrative claims, registries, device-generated data, and patient-generated data may all be suitable, provided they are shown to be relevant and reliable for the intended use. Consistent with other recent FDA guidance, the agency does not endorse any single data source over another, instead prioritizing methodological rigor, transparency, and appropriate study design.

The non-binding guidance outlines a broad range of regulatory purposes for which RWD may be used, including generating hypotheses to be tested in clinical studies, supporting marketing authorization as primary clinical evidence, providing additional safety or effectiveness information generated directly by the device, and supporting postmarket surveillance or labeling updates. To support these uses, the guidance includes examples on previously used and accepted methodologies.

As indicated by the FDA earlier this week, when it clarified its acceptance of de-identified RWD in regulatory reviews, the agency does not require that identifiable individual patient data from RWD sources always be included in a marketing application. This position is reflected in the final guidance, which addresses a longstanding practical challenge for sponsors: access to patient-level data. While the FDA reiterates its preference for sponsors to have access to the “first instance” of data used in analyses, it acknowledges that some RWD sources restrict access to participant-level data or provide only aggregate-level outputs. This is particularly relevant where privacy, consent, or data protection requirements, including those governing non-US data sources, limit the ability to share individual-level data across jurisdictions. In such cases, the agency does not discourage use of these data sources, provided sponsors clearly describe who controls the data, what level of access can be shared with the FDA, and how any limitations affect the interpretation and reliability of the resulting evidence.

Another important addition to the guidance for sponsors is the new section on documentation for FDA review, which effectively serves as a roadmap for what the agency expects to see when RWE is included in a regulatory submission. This includes recommendations for protocols, analysis plans, and a structured relevance and reliability assessment, as well as supporting documentation describing data access, traceability, and quality controls.

For device developers, the guidance warrants close scrutiny to understand how its recommendations may be applied in practice. In particular, it may encourage earlier integration of RWE planning into development programs, influencing study design and data strategy earlier in the product lifecycle.

The final guidance also raises questions about whether similar clarifications may follow for drugs and biologics. The FDA’s guidance Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products, finalized in August 2023, already outlines expectations for RWD and RWE in that context. However, the more detailed treatment of data access, documentation, and fit-for-purpose assessments in the device guidance may prompt consideration of whether future updates are needed to maintain consistency across FDA’s RWE frameworks.