The U.S. Food and Drug Administration has released long-anticipated guidelines for improving diversity in clinical trials, and some biopharma industry experts are hailing its arrival as a much-needed impetus for drug developers to include volunteers in their studies who have been largely excluded from the process in the past.
The FDA’s guidance stems from the 2022 Food and Drug Omnibus Reform Act, which aims to improve the representation of Black, Latino, Asian, Indigenous and other underrepresented groups in clinical trials for new drugs and medical devices. It provides suggestions for manufacturers on how to format and carry out “diversity action plans” for clinical trial enrollment that include a range of age groups, ethnicities and sexes. Complying with the guidelines will be mandatory for companies seeking FDA approval of their products.
“Clinical trials were historically filled with middle aged white men, but that doesn’t work anymore,” said Rivka Szafranski, co-founder and CEO Clinicaltrials.co, which provides information on clinical trials for patients and caregivers, as well as tools for trial sponsors to connect with potential participants. “As the number of clinical trials grows, including diverse populations will be important for ensuring these treatments will be effective for everyone.”
Despite a longstanding push from patient advocates to improve diversity in clinical trials, recent data suggests there’s still plenty of work to be done. In research published in JAMA, for example, the FDA reported that between 2015 and 2019, participation in clinical trials by Asian, Indigenous and Hispanic or Latino populations was below the U.S. census level for those groups. Data collected by the National Cancer Institute between 2005 and 2020 found significant under-representation of older adults, women, Hispanic or Latino, Indigenous, and Asian or Pacific Islanders in cancer trials. Furthermore, people with disabilities, chronic illnesses, eye or ear disorders or chronic illnesses are routinely excluded from clinical trials, a 2022 review of 97 trials found.
The FDA’s new guidance specifies that each clinical trial include a diversity action plan that reflects the estimated prevalence of the disease for which the drug or device is being evaluated. Biopharma developers will need to describe their enrollment goals for specific demographic groups and provide a rationale for those goals that includes information about how common the disease is in each population. Trial sponsors will also need to explain their strategy for monitoring their enrollment goals and retaining trial participants.
The FDA suggests several enrollment and retention strategies in the guidance document. They include engaging with community and patient advocacy groups in target communities, lowering barriers to entry in clinical trials and reducing the burden on prospective participants by providing transportation, expense reimbursement and other services.
The FDA also suggests that clinical trial sponsors consider the accessibility needs of participants who have disabilities. “It can be challenging for patients, particularly in rural areas, to access clinical trials. This guidance will help developers improve that access,” Szafranski said.
The FDA will collect feedback on the draft guidance for 90 days. Any clinical trials that begin enrollment 180 days after the final document is published will be subject to the requirement that they include a diversity action plan. Companies will be able to request waivers from the Diversity Action Plan requirement — and the new guidance spells out the procedure for doing so — but the FDA states that waivers “will only be granted in rare instances.”
The trade group Pharmaceutical Research and Manufacturers of America (PhRMA) is reviewing the draft guidance and plans to submit comments to the FDA, said Andrew Powaleny, a spokesperson for the organization, in a statement.
He added that PhRMA voluntarily developed its own principles for addressing clinical trial diversity in 2022 to drive more equitable access to clinical trials, so they better reflect the patients intended to be served. He stated that the organization and its members are “committed to working with the FDA, patient advocacy organizations and other stakeholders across the clinical research ecosystem to encourage more diverse participation in clinical trials.”