Drug and device companies should prepare now for new legal requirements aimed at improving clinical trial diversity—even as they wait for more action from the FDA.
The 2023 omnibus spending bill enacted last month (Public Law 117-328) requires diversity action plans for the clinical trials used by the Food and Drug Administration to decide whether drugs are safe and effective.
The idea is to encourage investigators to develop a strategy for reaching a broad study population on the front end, instead of either failing to do so altogether, or acting later and increasing an already lengthy and costly clinical trial process.
“What’s happening is with this plan is we’re all putting more focus and intentionality to how we do better with the sites to help them achieve the goals we need,” Rachael Fones, strategic adviser for diversity in clinical trials for the clinical research organization giant IQVIA, said.
The law, which incorporates the DEPICT Act, marks an important step in addressing decades of testing drugs primarily on White men. That practice left knowledge gaps on how drugs would work on a large swath of the population. Black Americans account for about 13% of the US population and 22% of yearly cancer cases, but the median enrollment of Black Americans in clinical trials was about 4.5%, according to a 2020 study.
The law is both good policy and good science, bill sponsor Rep. Anna G. Eshoo (D-Calif.), said in an interview. “This is the first time that there is a statutory requirement for diversity in clinical trials. And FDA has made efforts to encourage this since the 1980s. But it’s always been through voluntary guidance. This changes it.”
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“A draft guidance gets a lot of attention. A draft guidance that now has a statute saying it needs to be finalized and enforced will get a higher level of attention,” Susan C. Winckler, CEO of the Reagan-Udall Foundation, said at an American Enterprise Institute webinar on accurate race and ethnic representation in pivotal clinical trials.
“I expect us to hear more from companies about providing additional comment to the agency about what they’d like to see changed in the final [guidance],” Winckler said. “But the idea that the company says, ‘Here’s who I want to reach and here’s my plan to reach it’ is generally a pretty good structure.”
The law allows for exemptions to the diversity plan requirement if trial sponsors can justify a waiver. Starting in 2024, the FDA will have to publish annually an aggregate report of these action plans, along with the reasons any trials fell short of those goals.
There are some differences between the law and draft guidance that the agency will need to iron out.
“Congress was clearly trying to pick up on what FDA had done and codify what FDA had done in the April guidance,” Rachel L. Sher, a partner at Manatt Health, said. “But with that, FDA is going to have to go back and flesh out more of what’s in the guidance.”
Those details include when and how the FDA will grant waivers, how sponsors submit modifications to the action plan, and how they’ll meet the reporting requirements laid out by Congress.
FDA’s recommendations also focused on on race and ethnicity, but Congress also calls for demographic information such as age, sex, geographic location, and socioeconomic status.
“FDA is going to have to elaborate on what that looks like in the guidance and how a plan would go about addressing those things,” Sher said.
An FDA spokesperson said the agency is reviewing the law and the agency’s authority.
“Clinical trials, and the people who volunteer to participate in them, are critical for evaluating whether a medical product is safe and effective to prevent or treat diseases,” the spokesperson said. “The agency will continue to engage with sponsors, manufacturers, clinical research organizations, academia, medical professionals, health advocates, patients, and other stakeholders to achieve this important goal.”
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But clinical trial sponsors shouldn’t wait to take action.
“The fact that the guidance is coming from FDA, and the fact that it is now law, are not the only reasons why clinical trial sponsors should be thinking about diversity in clinical trials,” Heather H. Pierce, senior director for science policy and regulatory counsel for the Association of American Medical Colleges. “The same motivations that put this in play should already be driving the development and thinking about designing clinical trials with an understanding of their relevance, usefulness and application at the end of the trial.”
Diversity plans need to be integrated into clinical research processes as a standard operating procedure, said Yasmeen Long, a director at FasterCures who heads up their health equity programs.
“Perhaps there needs to be training on including a diversity action plan as a part of your research in order for it to be funded and executed,” Long said. Then, “the incentive is not only making trials more diverse, but the incentive is your research won’t be approved unless it includes this in your protocol and SOPs.”
Institutions need to tackle clinical trial diversity internally, with staff from clinical operations, ethics, and the compliance office, said Sarah Thompson Schick, an FDA regulatory attorney who is of counsel at Reed Smith LLP.
“The guidance is a great jump off point. The legislation is a great jump off point. But where do we go next? Thinking about those governmental partnerships, and then also those inter-institutional partnerships to enhance diversity and developing those types of programs to get us there,” Schick said.
Meanwhile, finding enough research volunteers is already a challenge. More than 80% of clinical trials fail to enroll enough patients on time.
Donna M. O’Brien, a national adviser at Manatt Health, underscored the importance of choosing research sites in a way that optimizes enrollment.
“If you want diverse populations, you have to go to the community, and those organizations aren’t set up to be to be research institutions,” O’Brien said.
Interactions between providers and patients in community settings will be the key to finding the right patients, “and if those settings aren’t well-equipped to even offer clinical trials, it’s going to be very challenging to make an impact.”