Dive Brief:
- The Food and Drug Administration is seeking feedback on health equity for medical devices to inform a potential regulatory approach to the topic, the agency said Monday. The paper is open for comment until Oct. 4.
- In the discussion paper, the FDA shared its thinking on how sponsors can select study populations that adequately reflect the intended use for a particular medical device.
- The discussion paper is part of a broader focus on health equity, one of the Center for Devices and Radiological Health’s strategic priorities, that also includes advice on diversity action plans.
Dive Insight:
The FDA framed the discussion paper as a response to the “urgent public health need for innovative technologies that help to reduce barriers to achieving health equity.” Seeking to address that need, the CDRH has committed to developing “a framework for when a device should be evaluated in diverse populations to support marketing authorization” as part of its strategic focus on health equity.
Running clinical trials that generate results consistent with how a device will perform in the real world is a way to improve health equity. Acknowledging that generating clinical data “can be complex,” the agency said it has focused its discussion paper on “a few important considerations that may be relevant for FDA’s evaluation of clinical evidence.”
The paper outlines factors that may help sponsors and investigators develop study objectives. To inform early trial design, the FDA recommends asking how the burden of disease and how the prognosis of a disease varies across a device’s intended use population. Sponsors can also ask how a device may introduce, exacerbate or mitigate differences in outcomes across the study population.
Another section of the document describes considerations related to the FDA’s evaluation of safety and effectiveness. The FDA reviews marketing authorization filings to determine whether there is reasonable assurance the device will be safe and effective in the intended population. As such, the agency is looking at whether clinical data “are generalizable to, and representative of, the intended use population.”
The FDA said it “considers it important to understand a sponsor’s rationale regarding the relevance of the provided clinical data to the intended use population.” The rationale could cover the processes used to select and enroll study populations, the agency said, or help the FDA understand difficulties a sponsor encountered when trying to obtain data from certain populations.
In the final section of the paper, the FDA describes example scenarios intended to prompt feedback on its assessment of the benefits and risks of devices. The section features a table that shows how the FDA may respond depending on whether the evidence suggests there are differences in patient populations and health outcomes, as well as whether the sponsor included specific populations in the clinical study.
In some cases, clinical trial sponsors may choose to design studies with “enriched” populations, meaning they intentionally enroll people from groups where differences are expected. The table suggests sponsors that fail to enrich study populations to reflect the intended use may face difficulties at the FDA if the available information suggests differences in patient populations.
The FDA said the absence of enriched populations may “introduce uncertainty” on the applicability of the data to the intended use population.
