FDA’s Califf Calls for Greater Focus on Clinical Trial Diversity

Clinical trial diversity must be a more prominent priority among health research administrators, FDA Commissioner Robert Califf said Wednesday as the agency readies updated guidance on the topic.

The Food and Drug Administration is preparing a manuscript of the agency’s thinking on how to improve diversity in clinical trials, Califf said at the Milken Institute’s Future of Health summit.

Ensuring studies are representative of the population likely to benefit from a new drug or medical product has been a key priority during Califf’s tenure. Lawmakers and health policy advocates have also ramped up calls to include provisions mandating trial diversity in an end-of-year government spending package.

“It’s asking a lot of the clinical trials system to overcome a health system which is structurally configured to create these disparities in the first place,” Califf said.

“What I’m hearing from clinicians out there is they would love to do some of this stuff, but they’re not encouraged to do it by the administrators they’re working with,” he said. “It’s going to take progress in the health system if we’re going to get to where we need to be.”

Califf highlighted that National Institutes of Health-funded clinical trials “have made a lot of progress” in promoting clinical trial diversity, and that now is the time “to focus on solidifying and improving the gains we’ve made.” This will include addressing diversity across gender, race, ethnicity, and geographic location, he said.

Representation in clinical trials has improved over the years, though the FDA noted in its most recent Drug Trials Snapshot Summary Report that racial and ethnic representation in many of the trials for the 50 novel therapies approved by the agency in 2021 remained low.

For example, 1% of participants in trials on Biogen’s Alzheimer’s drug Aduhelm identified as Black even though Black people are about twice as likely to develop Alzheimer’s disease as White people.

The forthcoming guidance would mark the latest effort by the agency to address a longstanding challenge in the clinical trial enterprise. The FDA issued draft guidance in April to help drug companies on coming up with diversity plans early in the development process.

Yasmeen Long, director of the Milken Institute’s FasterCures, said in an email following Califf’s remarks that members of the health-care profession should be asking, “Why is it that administrators are not encouraging researchers to do something that will improve health outcomes for all people?”

“Perhaps if we as a system can determine the answers to that question, then maybe those answers will help us develop concrete and sustainable solutions to eliminate these barriers to increasing diversity in clinical trials for the long-term,” she said.

Legislative Action

Members of the Doctors for America’s FDA Task Force held another round of meetings with lawmakers this week, urging them to pass certain FDA improvements that were dropped from September’s reauthorization of industry user fees to the FDA.

Among them are clinical trial diversity measurements based on the DEPICT Act (H.R. 6584) from the House health subcommittee chair, Rep. Anna Eshoo (D-Calif.).

The provisions would allow the FDA to require drug and device companies to submit diversity action plans for their clinical trials.

The House passed these provisions as part of a package (H.R. 7667) guaranteeing FDA user fee funding, though this portion of the FDA’s budget ultimately moved forward in a stopgap funding measure without broader FDA policy changes.

Eshoo told Bloomberg Law Wednesday that clinical trial diversity is a top priority for her to get into an end-of-year omnibus spending bill.

“We’re working very hard on that, and the atmospherics change by a few degrees each day,” she said.

Reshma Ramachandran, chair of the FDA task force, said in an email Wednesday that “besides encouraging administrators and clinicians to enroll more diverse populations, FDA should take meaningful steps to require more representative populations within clinical trials.”

“I hope that in their manuscript FDA will outline more clearly how they can exert their authority as a regulator in ensuring that new drugs and devices approved by the agency are reflective of diverse populations—not just recommend that administrators encourage clinicians do so,” added Ramachandran, a Yale School of Medicine family physician and professor.