FDA loosens restrictions on using patient-level RWD in medical device submissions

The US Food and Drug Administration (FDA) clarified in a final guidance dated 17 December that it will accept real-world evidence (RWE) without requiring identifiable patient-level data in medical device submissions. Moving forward, FDA reviewers will consider the strength of submitted RWE on an application-by-application basis.

This guidance finalizes a draft version issued in December 2023 (RELATED: FDA proposes updated medtech RWE guidance, Regulatory Focus, 19 December 2023). When finalized, the guidance will replace FDA’s former finalized guidance from 2017 (RELATED: FDA Finalizes Guidance on Using Real World Evidence for Medical Device Regulatory Decisions, Regulatory Focus, 30 August 2017).

“We're removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster,” FDA Commissioner Marty Makary said on 15 December, in advance of the final guidance. “This common-sense reform will unlock access to vast databases like cancer and cystic fibrosis registries that contain critical insights about how treatments work in the real world."

Historically, FDA has required that any application with RWE elements include private, confidential information at the individual patient level, a practice that “makes it impractical to use most large databases with valuable macro-level data,” according to an FDA statement.

The move acknowledges the position of sponsors and data scientists that meaningful information can be derived from certain big data sources that do not catalog individual-level personal information. FDA reviewers will now assess the strength of submitted RWE on an application-by-application basis.

“When the RWD source is not owned by the sponsor, where participant-level data is accessible, the sponsor should attempt to obtain participant-level data for each participant,” according to the final guidance.

The agency added that it “recognizes that some data sources either provide only aggregate-level outputs or will not allow sponsors to access the participant-level data. FDA does not discourage use of these data sources, and a sponsor’s inability to obtain participant-level data does not preclude FDA’s evaluation. Sponsors should describe how lack of participant level data does or does not affect the RWE.”

This policy change enables the use of de-identified databases containing millions of patient records, including cancer registries, such as the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program, as well as databases from hospital systems, insurance claims, and electronic health records. These resources have expanded significantly but were previously restricted under earlier policies.

Similar language was included in the 2023 guidance in acknowledging that it is not always possible to obtain patient-level data. Yet the prior guidance stopped short of endorsing the use of these large de-identified databases.

The earlier guidance stated that “when the RWD source is not owned by the sponsor, the sponsor should attempt to obtain participant-level data for each participant. If not available, the sponsor should define the entity(ies) which do have access/permission for data entry, quality assurance, storage, aggregation or other linkage, and assessment of traceability from data entry to dataset, as applicable. Sponsors should consider the level of access which could be shared with FDA and the potential for third parties to provide participant-level data directly to FDA. The availability of data should be described in the regulatory submission for FDA review.”

Some of the other changes from the 2023 guidance:

In the background section of the guidance, new examples were added to list some of the potential benefits of using RWD, including gathering information from a wider range of clinical experiences than what is typically seen in traditional clinical studies. Additionally, RWD can provide new insights into a device's performance, facilitate a timelier response to postmarket requirements and commitments, and decrease the time it takes to bring a product to market.

There are new examples illustrating when the FDA will and will not accept investigational device exemptions (IDEs) that leverage real-world data (RWD) in submissions.

There is a new example in the section on RWE use, which demonstrates 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), which directs the FDA to revise existing guidance on considerations for the use of RWD and RWE to support regulatory decision-making. In addition, it also satisfies 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 agency announced that the integration of RWE into device approvals has been more extensive than for drugs and biologics. Since 2016, there have been over 250 premarket authorizations that included RWE. In the same time frame, 35 drugs, biologics, or vaccines have utilized RWE in their applications.

Federal Register notice; Final guidance; FDA announcement

FDA loosens restrictions on using patient-level RWD in medical device submissions

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