FDA seeks to clarify use of real-world evidence to support regulatory decision-making for medical devices with new draft guidance

The US Food and Drug Administration (FDA) has released a draft guidance detailing its criteria for evaluating the quality of real-world data (RWD) used in regulatory decisions for medical devices. The guidance also offers broader recommendations for sponsors who intend to leverage real-world evidence (RWE) in their regulatory submissions for medical devices.

Continuing efforts to advance the use of RWE in regulatory decision-making, the FDA has today, December 18, 2023, issued a new draft guidance focused on the role of RWE in the context of medical devices. The guidance, called ‘Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices,’ comes hot on the heels of the final guidance issued by the agency confirming their stance on the role of RWD and RWE to support regulatory decision-making for drugs and biological products.

The draft guidance supersedes the previous FDA guidance issued in August 2017. By issuing the new draft guidance, the FDA satisfies the requirements of the Food and Drug Omnibus Reform Act of 2022 (FDORA) as well as meeting the commitments set out in the fifth reauthorization of the Medical Device User Fee Amendments (MDUFA V). As noted in the federal register, electronic or written comments on the draft guidance should be submitted to the FDA by February 20, 2024.

---

You may also be interested in:

Chasing the holy grail of using real-world evidence in regulatory decision-making for medical devices: an interview with Paul Coplan, Johnson & Johnson

 

 

---

The draft guidance proposes expanded recommendations to be followed by industry and other stakeholders in assessing if and how RWD can be determined fit-for-purpose for a particular regulatory decision relating to medical devices. The guidance covers topics specific to RWE generation that can be used to provide clinical evidence across the medical device product life cycle. As noted, the aim of the final guidance will be to complement and be used with other device-specific and good clinical practice guidance documents.

One of the notable changes in the guidance is the section on the expanded considerations for the use of RWE, which details the context in which RWD can be used:

“In general, FDA considers the use of RWD to be fit-for-purpose to support generation of clinical evidence for regulatory decision-making for medical devices when we conclude that the RWD used to generate the RWE are relevant to and reliable for informing or supporting a particular regulatory decision. It is important to understand the strengths and limitations of the underlying RWD and how these qualities impact their relevance and reliability.”

The guidance provides details of the types of data sources that can be used to generate RWD, including registries, electronic health records, data from digital health technologies and implantable devices and medical device data repositories. The instances when RWD can also be used regulatory submissions is expanded and now includes scenarios such as generating evidence to support the clinical validity of a biomarker or clinical outcome assessment, providing primary clinical evidence to support marketing authorization and as a mechanism for re-training artificial intelligence/machine learning-enabled medical devices.

The FDA also expands their criteria for assessing the relevance and reliability of the RWD sources, which they note depends on the particular regulatory decision being sought. They outline several key factors that need to be considered, including verifying the relevance of the RWD by considering the availability, timeliness, and generalizability of the data. Whether the RWD is reliable depends on the accrual, quality, and integrity of the data source.

The draft guidance features additional explanation on the suggested approaches for gathering and examining RWD, with the FDA acknowledging, “just as traditional clinical studies should be carefully designed, studies using RWD also should undergo careful assessment before embarking on the study or during the analysis to assure that the data are fit-for-purpose.” Several strategies for appropriate study design and data analysis are provided to ensure the study question can be appropriately addressed whilst minimizing any bias and uncertainty.

To assist industry in determining whether to use RWE, the guidance also documents several examples from actual uses of RWD explaining how the FDA assessed RWE in the context of medical device regulation.