Clinical trials are used to evaluate the safety and effectiveness of medical products intended for the prevention, treatment, or diagnosis of a disease. However, if the study population is not diverse, the trial results may not be generalizable to other populations, and the safety and efficacy of the treatment may not be fully understood. Clinical trial diversity is a crucial issue because, without a diverse study population, drug and device sponsors may not have access to the data needed to ensure products are safe and effective for any patient, regardless of race, sex, or ethnicity, within the scope of the product’s indication and intended use. Integrating a more diverse population of patients into the clinical research ecosystem has the added benefit of promoting broader health equity goals as well.
The lack of representation of certain populations in clinical trials reflects, in part, a broader issue of healthcare disparities in the US. Access to health care often differs along sex, gender, race, and ethnicity generally, which affects patient access to and awareness of academic medical centers conducting clinical trials. For this reason, traditional clinical trial recruitment methods such as physician referrals may not reach diverse communities effectively. Additionally, mistrust of the clinical research system may stem from historical events that adversely impacted racial and ethnic minorities, such as the unethical Tuskegee experiments. This mistrust, coupled with a lack of representation, may fuel skepticism and reluctance to participate in clinical trials.
Congress and FDA have taken several steps to promote clinical trial diversity. In 2022, FDA proposed an ambitious framework to encourage sponsors to address diversity enrollment gaps in clinical trials. Additionally, with the passage of the Food and Drug Omnibus Reform Act of 2022, or FDORA, Congress strengthened FDA’s authority to require drug and device sponsors take action to promote diversity in clinical trials.
In particular, FDORA amended the Federal Food, Drug, and Cosmetic Act to require drug and device sponsors to submit diversity action plans articulating goals for increasing enrollment of subjects from historically underrepresented populations. FDORA requires diversity action plans to include the (1) sponsor’s enrollment goals, (2) rationale for the goals, and (3) an explanation of how the sponsor intends to meet the goals. However, FDORA also permits FDA to waive the diversity action plan requirement for an individual sponsor at the agency’s own discretion or upon request. The waiver may be granted depending on what is known or suspected about the prevalence or incidence of the disease for which the product is under investigation; if requiring a diversity action plan would be impracticable; or if a waiver is necessary to protect public health during an emergency.
FDORA requires FDA to update or draft guidance documents on diversity action plans related to the sponsor’s goals for enrollment by age group, sex, and racial and ethnic demographic characteristics of clinically relevant study populations, including the rationale for enrollment and explanation of how the diversity goals will be attained. FDA is also directed to hold public workshops with stakeholders to solicit input on strategies to improve the recruitment, enrollment, and retention of historically underrepresented populations in clinical studies.
Although FDORA is a significant step advancing clinical trial diversity, it remains to be seen what actions FDA will take against sponsors who fall short of their diversity enrollment goals. For example, if an application lacks sufficient data about certain racial and ethnic groups, would FDA deny the marketing application? Would the FDA restrict the product’s use in certain populations? What, if any, post-marketing requirements will be imposed on the product’s sponsor?
Despite the lack of clarity on enforcement, what is clear is FDA’s focus on this issue and its commitment to encouraging clinical trial inclusivity. FDA’s current guidance documents emphasize the importance of recruiting diverse populations, collecting and reporting data on participant demographics, and suggesting strategies to enhance participant representation. FDA also collaborates with industry and stakeholders on raising awareness diverse clinical trial enrollment.
The lack of diversity in clinical trials can negatively impact our understanding of the safety and efficacy of new treatments. Addressing the barriers that hinder diverse participation can enhance the generalizability of research findings, foster patient trust in the clinical research ecosystem, and improve healthcare outcomes for all.
Robert Church
Robert Church is a partner with Hogan Lovells.
Stephanie Agu
Stephanie Agu is an associate with Hogan Lovells.
