Fostering More Access To Medical Devices: FDA's Efforts To Advance Health Equity

Key Takeaways

• The U.S. Food and Drug Administration (FDA) recently published a Discussion Paper which outlines a potential framework for future guidance and regulations regarding the clinical evaluation of medical devices in diverse populations.
• The Discussion Paper mentions preliminary factors that sponsors and investigators should consider in study design as well as factors the agency will weigh when assessing a product's safety and effectiveness for a given indication.
• The agency is soliciting feedback on this framework and has provided example scenarios and questions to encourage constructive public commentary.

On August 5, 2024, FDA's Center for Devices and Radiological Health (CDRH) published a discussion paper entitled, "Discussion Paper: Health Equity For Medical Devices" (Discussion Paper), to aid in its efforts to advance health equity by working to ensure that all patients have timely access to safe, effective, and high-quality medical devices and safe radiation-emitting products.

As part of CDRH's 2022 to 2025 Strategic Priority to Advance Health Equity, CDRH has committed to:

• Facilitate the availability of and access to medical technologies for all populations;
• Empower people to make informed decisions regarding their healthcare;
• Support innovation of novel and existing technologies that address health inequities; and
• Reduce barriers to increase participation by diverse populations in evidence generation.

Participation levels from minority and other underrepresented groups in clinical trials has historically been low—largely due to a mistrust of medical research (spurred on by prominent public health mistreatments like The U.S. Public Health Service (USPHS) Untreated Syphilis Study at Tuskegee¹ or the "Mississippi appendectomy"²) or just missed educational opportunities. However, over the years, FDA has taken steps to help diversify clinical trials by issuing guidance on clinical trial diversity action plans (DAPs), which are required for certain clinical studies pursuant to section 3601 of the Food and Drug Omnibus Reform Act of 2022 (FDORA)³. Our previous alert on DAPs may be found here.

It is important to note that CDRH's recently published Discussion Paper is not intended to propose or implement a specific regulatory policy. Instead, the Discussion Paper is intended to stimulate discussion to inform further policy development in accordance with good guidance practices⁴ and includes a number of questions for public input and feedback. Ultimately, CDRH's aim is to develop a cohesive policy on when a device should be evaluated in diverse populations to support marketing authorization, so that the clinical data intended to support a marketing submission are representative of, and generalizable to, the intended use population identified for the device.

As it relates to the generation of clinical data to support medical device authorizations, the Discussion Paper focuses on a few important considerations that may be relevant for FDA's evaluation of clinical evidence. First, it discusses factors that may help sponsors and investigators during the design and development of study objectives and a clinical analysis plan. Second, the Discussion Paper describes considerations related to the intended use of a device that may be related to the device's safety, effectiveness, benefits, and risks. Lastly, the Discussion Paper includes generalized examples to further stimulate public feedback regarding FDA's assessment of a device's benefits and risks in light of the factors and considerations described elsewhere in the paper.

This alert provides a general summary of the Discussion Paper on each of these points along with important next steps.

I. Clinical Study Plan Development
The Discussion Paper describes three considerations (discussed briefly below) that may help inform design of a clinical study that adequately reflects the intended use population for a particular medical device.

1. Disease burden or condition – This consideration focuses on how the burden of the disease or condition under study varies across the U.S. intended use population.
2. Physiology, anatomy, and pathophysiology – This consideration focuses on how the etiology, progression, and prognosis of a disease or condition under study vary across the U.S. intended population based on physiology, anatomy, and pathophysiology.
3. Technology – This consideration focuses on how the device might introduce, exacerbate, or mitigate the potential for different outcomes across the study population.

According to the Discussion Paper, FDA believes that an evaluation of this relevant information will help identify sources of potential variability in expected outcomes or device performance within the intended use population. Further, as sponsors consider various mechanisms to generate relevant safety and effectiveness information, they may choose to use this information to inform the study design, not only assessing inclusion and exclusion criteria, formulating necessary study procedures, or identifying specific data to collect, but also considering approaches (e.g., in the study monitoring plan) that may help ensure recruitment, enrollment, and retention of important patient populations into a clinical study.

Discussion Questions (Clinical Study Plan Development)
The Discussion Paper asks for input on how a sponsor or investigator can incorporate the above considerations into the early study design process to help ensure that the study is representative of, and generalizable to, the intended use population identified for the subject device. Specifically, FDA is interested in learning whether the potential overlap in the three considerations is advantageous or not and whether they should be considered in isolation; whether there are other factors that should be considered when identifying a target population; how to prioritize and operationalize information when identifying patient factors; the challenges sponsors have faced in obtaining, analyzing, reviewing, and using relevant information; and other circumstances that may prompt a sponsor to design a clinical study enrolling an enriched population.

1. Clinical study population – This consideration focuses on the population included in the clinical data provided to support a premarket decision and the rationale given by the sponsor for the population.
2. Differences between populations – This consideration focuses on whether the data suggest that there are differences in outcomes between populations.

FDA is seeking input on how these two additional considerations may be incorporated into its evaluation of clinical data provided in marketing submissions or otherwise integrated into FDA's existing regulatory framework.

III. Example Scenarios
The Discussion Paper includes a table that explores the potential relationship between information on whether or not a clinical study adequately reflects the intended use population and potential considerations for FDA. To facilitate public input, FDA developed several examples with hypothetical data to further explore this relationship. FDA hopes that the table along with illustrative examples will allow the public to better understand these areas of consideration and provide feedback on the concepts discussed in the Discussion Paper. The Agency requests public comment and feedback on the table and example scenarios.

Comments on the Discussion paper are due by October 4, 2024. Should you have any questions about the Discussion Paper or need assistance in preparing comments, please feel to reach out to members of our FDA team. FDA is expected to use feedback submitted to the docket to help inform future policy development.

Footnotes

1 See https://www.cdc.gov/tuskegee/index.html

2 See "The Sweat and Blood of Fannie Lou Hamer," https://www.neh.gov/article/sweat-and-blood-fannie-lou-hamer.

3 See Consolidated Appropriations Act (December 2022) (P.L. 117-328).

4 See, e.g., 21 C.F.R. § 10.115.

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