FDA Requires Diversity Plan for Clinical Trials to Increase Participants from Underrepresented Groups

The US Food and Drug Administration (FDA) recently announced a significant change in its requirements for late-stage clinical trials, which would mandate researchers and companies seeking approval to submit a plan for ensuring diversity among trial participants.

However, scientists have expressed concern that the FDA’s broad waiver authority, which allows the agency to waive the need for a diversity-action plan in certain circumstances, could undermine the legislation.  

According to Nature, before the new requirement takes effect, the FDA must finalize its draft guidance and offer the public an opportunity to comment, which could take over two years. Researchers are also concerned about how strictly the FDA will enforce the requirement and the agency’s options if a researcher fails to follow through on their action plan.  

The US National Academies of Sciences, Engineering, and Medicine stated in 2022 that white female participation had increased while minority male engagement had “essentially stopped.” This revelation resulted in the establishment of the diversity quota system.

According to a recent assessment, clinical research in the United States continues to under-represent and, in some cases, exclude entirely people over 65, pregnant women, and people with disabilities. An examination of clinical trials used to support FDA approval of new cancer drugs between 2012 and 2017 found that 79% sufficiently represented women, 27% adequately represented older persons, and just 11% effectively represented minority racial and ethnic groups.  

According to Marian Knight, a perinatal epidemiologist at the University of Oxford in the United Kingdom, the clinical-research findings may only apply to some because the makeup of the pool of participants does not match the group that may benefit from the treatment. Furthermore, she claims that it undermines trust in the medical community.  

Some pregnant women were hesitant to get vaccinated during the early stages of the COVID-19 pandemic, for example, since many of the studies that assessed the vaccines did not include pregnant women, she says. Knight, who co-authored a study published in The BMJ on February 6th suggesting that researchers include more pregnant individuals in clinical trials, feels that vaccine hesitancy may be to blame for the disproportionate occurrence of COVID-19-related mortality among pregnant people.  

To meet the diversity requirement, researchers and pharmaceutical firms must outline their demographic goals for the study group, including arguments for those goals and strategies for achieving them. Jennifer Miller, a bioethicist at Yale School of Medicine in New Haven, Connecticut, believes this strategy has the potential for cancer treatment. Businesses will be forced to submit enrollment plans to authorities for approval for the first time.  

While the legislation is a step in the right direction, researchers and companies must focus on the structural problems underlying the lack of diversity in clinical-trial populations to achieve their goals. These efforts could include relocating research sites to places that are more accessible for under-represented groups and building trust in local communities that may have been wary of exploitative medical research in the past.

While the legislation alone may not address long-standing disparities in clinical-trial enrollment, it could inspire other countries, such as the United Kingdom, to introduce similar requirements, encouraging researchers to prioritize diversity in their trials.