FDA Issues Draft Guidance for Using RWD in Medical Device Regulation

On December 18, 2023, the United States Food and Drug Administration (FDA) published a draft guidance called “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices.” The document outlines proposed strategies for using real-world data (RWD) — also called real-world evidence (RWE) — to support regulating medical devices in the US.

According to the FDA website, a previous document was developed and finalized in 2017. While the 2017 document remains in effect until a final decision is made, the draft guidance provides updated recommendations based on scientific advancements and discoveries in recent years.

“RWD may provide an efficient means of generating the necessary 85 clinical evidence to support regulatory decisions. Information specific to the clinical performance 86 of a device can be generated through a number of methodological and operational approaches,” noted the FDA in the guidance.

However, the administration also emphasizes that while clinical trials are narrow in scope, with the ability to control and minimize bias and error, research conducted using RWD can be more challenging to manage. This conclusion highlights the importance of protocols to reduce the weaknesses of RWD.

The guidance is divided into four primary sections.

• Regulatory context for using RWE: This section highlights cases where using RWE to make regulatory decisions is appropriate. Beyond general considerations, it provides context for investigational device exemptions (IDE) and emergency use devices.
• Data relevance and reliability: Divided into two additional subsections, this category focuses on factors that impact data relevance, including availability, linkages, timeliness, and generalizability. The other subsection focuses on reliability, including data accrual, quality, and integrity.
• Methodologies: Another section of the draft guidance highlights principles for study designs using RWD and other study elements.
• Documentation: The final category provides guidelines on documenting the use of RWD to submit for FDA review. The documentation outline includes cover letters, fit-for-purpose assessments, protocols, reports, and additional information.

The FDA’s Center for Devices and Radiological Health and Center for Biologics Evaluation and Research has left the document open for commentary until February 20, 2024. At that point, the administration will evaluate the comments and make a final decision on the guidance.