There are persistent gaps in the representativeness of clinical trials for new drugs across disease areas, highlighted in recent pivotal aducanumab clinical trials for Alzheimer’s disease—where just 0.6% of study participants identified as Black. In pivotal trials for new oncology drugs from 2008 to 2018, the percentage of Black participants only increased from 2.9% to 3.6% (JAMA Network).
A recent National Academies of Sciences, Engineering, and Medicine report outlines the potentially serious health, economic, equity, and scientific consequences of underrepresentation in clinical trials. However, existing policies for ensuring inclusive participation are limited. After decades of mostly voluntary initiatives from the Food and Drug Administration (FDA), pivotal trials for new drugs continue to lack participants from historically marginalized racial and ethnic groups, older adults, and those who are pregnant or lactating.
New U.S. legislation passed in 2022 marks the first major policy change for clinical research equity in nearly 30 years. It requires study sponsors to submit a diversity action plan for phase 3 or pivotal studies of new drugs, including steps to achieve goals for study enrollment according to demographic group.
However, Thomas J. Hwang, MD, of the Division of Urological Surgery at Brigham and Women’s Hospital, and Otis W. Brawley, MD, of Johns Hopkins University, suggest this will not adequately resolve the underlying causes of underrepresentation in clinical trials. In the New England Journal of Medicine, they propose that a combination of federal incentives and regulations is necessary to advance clinical trial equity meaningfully.
Support Trial Sites Serving Underrepresented Populations
The FDA could provide direct grants to trial sites that serve and are most likely to enroll participants from underrepresented populations. To increase enrollment capacity and site infrastructure, this support could lower financial barriers to trial participation and ensure the facility’s participation in clinical research does not create undue burdens on staff. Additionally, the diversity action plans could require those trial enrollment criteria not to limit participation from underrepresented populations over the entire development life cycle.
Financial Incentives for Drug Manufacturers
Congress could provide financial incentives to drug manufacturers contingent on achieving representative trial enrollment for pivotal studies of their products. These incentives could include tax credits, as other incentives, such as extended market exclusivity or expedited FDA regulatory review, have several challenges.
Encourage Early Representative Enrollment
The FDA could encourage early enrollment of target populations and work to ensure representative enrollment in postapproval and real-world population studies is underway by the time a drug is approved. Relevant advisory committees could be informed about enrollment disparities and any attempted remediation in a post-marketing trial.