FDA finalizes guidance on submitting RWD/RWE in application cover letters

Regulatory NewsRegulatory News | 13 September 2022 |  By 

To help better track submissions that include real-world data (RWD) and real-world evidence (RWE), the US Food and Drug Administration (FDA) is asking drug and biologics sponsors to indicate the inclusion of such data in their premarket application cover letters.
On 8 September, FDA published a final guidance titled, Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drug and Biological Products. The draft version of the guidance was published in 2019 and was meant to help clarify the agency’s thinking on how sponsors should summarize their RWD/RWE data in product applications.
The guidance is the result of the agency’s mandate under the 21st Century Cures Act to develop a RWE program to evaluate the use of RWE in regulatory decision-making.
“The availability of RWD and evolving analytic techniques to generate RWE have created interest within the research and medical communities in the use of RWD/RWE to enhance clinical research and support regulatory decision-making,” FDA noted. “By enhancing FDA’s understanding of the scope and use of RWD and RWE submitted to support regulatory decisions, internally tracking these submissions as described in this guidance can inform FDA’s RWE program.”
In the guidance, FDA notes that it plans to track a wide array of study designs that aggregate RWD/RWE for product submission, including randomized clinical trials that use RWD to capture clinical outcomes related to safety or effectiveness and single-arm trials that use RWD in an external control arm. The agency also plans to track observational studies, such as observational cohort and case-control studies, that generate RWE intended to help support an efficacy supplement; and clinical trials or observational studies that use RWD or RWE to fulfill a postmarketing requirement (PMR) or postmarketing commitment (PMC).
When submitting RWD/RWE as part of a premarket application, FDA said that sponsors should include information in their cover letter including the purposes of using RWD/RWE, details on the study designs using RWD to generate RWE and RWD sources that were used to generate RWE.
The agency also said that sponsors should not identify submissions in their cover letter that include RWD/RWE if the data collected is not meant to be used for product labeling. For example, submissions that include RWD only intended to generate a hypothesis or for clinical trial planning, studies that use RWD for exploratory modeling or simulations, or those that leverage RWD to validate an endpoint, should not be included.


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