FDA proposes updated medtech RWE guidance

The US Food and Drug Administration (FDA) has updated it’s guidance on how real-world data (RWD) can be used to provide real-world evidence (RWE) for regulatory needs. It includes new examples of where RWD is collected, how the data is determined to be fit-for-purpose, and when it can be used to regulate certain diagnostic tests.

The draft guidance is an update to the agency’s 2017 final guidance on how sponsors can use RWD to provide RWE in premarket applications. While the original guidance only addresses situations where RWE can be used for Humanitarian Device Exemption, Premarket Approval Application (PMA), and De Novo submissions, the draft guidance also addresses determinations under the Clinical Laboratory Improvement Amendments (CLIA).

The draft guidance is the result of a Congressional mandate under the 2022 Food and Drug Omnibus Reform Act of (FDORA) that directed FDA to update its policy on using RWE for regulatory decisions. It would also fulfill FDA’s requirement under the Medical Device User Fee Amendment (MDUFA V) deal to update the 2017 guidance and provide more clarity on RWD and RWE. FDA notes that until the guidance is finalized, its 2017 guidance remains in effect.

“This draft guidance is intended to provide expanded and updated recommendations to industry and FDA staff for conducting an assessment of relevance and reliability to demonstrate that RWD may be fit-for-purpose to generate clinical evidence for regulatory decision-making,” the guidance states. “This includes recommendations to provide clarity on least burdensome general expectations related to demonstrating that RWD is fit-for-purpose for premarket regulatory purposes.”

Specifically, the agency provided additional details in the draft guidance on its thinking behind the appropriate methodologies that can be used to collect RWD and how to analyze that to generate RWE. Furthermore, it provided new examples that sponsors can use to understand which methodologies are appropriate for their situation.

“This draft guidance also provides additional clarity regarding the use of clinical data collected from the use of a device authorized under an Emergency Use Authorization (EUA) and to describe the type of information that could be applicable to support a determination under the Clinical Laboratory Improvement Amendments (CLIA) (e.g., Waiver by Application),” said the agency. “FDA recognizes and anticipates that the agency and industry may need up to 60 days to perform activities to operationalize the recommendations within the final guidance.”

Similarly, FDA said it anticipates that applications that are pending at the time the guidance is finalized may also need 60 days to come up to speed. Therefore, it said pending applications and those that are submitted within 60 days of the guidance being finalized won’t need to conform to the guidance. Sponsors of such applications, however, are welcome to provide the data to FDA if they can.

Ultimately, FDA noted that many of the considerations and best practices to generate RWE are not all that dissimilar to generating data from traditional clinical studies based on good clinical practice (GCP).

“Additionally, as with all clinical evidence FDA evaluates, FDA’s assessment of RWE evaluated in support of a particular regulatory decision will be included as part of the totality of information available to FDA,” said the agency. “Further, as with all types of clinical data, FDA recognizes there may be uncertainty of the benefits and risks of a device that remain after completion of a study using RWD.”

“Some of these aspects are similar to those also present in more traditional forms of clinical data, but some are unique to RWD,” the agency added.

Stakeholders can comment on the draft guidance through 20 February, 2024, on www.regulations.gov under docket no. FDA-2023-D-4395.

Draft guidance

FDA proposes updated medtech RWE guidance

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