On June 26, 2024, the U.S. Food and Drug Administration (FDA) released draft guidance addressing use of diversity action plans (Plans) in the conduct of clinical research entitled “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies Guidance for Industry” (Guidance). The Guidance is available for comment until September 26, 2024. If finalized the Guidance will replace previously issued FDA guidance on the use of such Plans.

In 2022, Congress passed the Food and Drug Omnibus Reform Act of 2022 (FDORA) as part of the Consolidated Appropriations Act. FDORA requires sponsors of clinical studies to submit Plans to the Secretary of the U.S. Department of Health and Human Services (Secretary). FDORA also requires the Secretary to publish guidance regarding the format and contents of Plans related to clinical study enrollment goals for specific demographic characteristics of study populations including age group, sex, and racial and ethnic demographic characteristics.

A Plan is required for a clinical investigation of a new drug for a phase 3 study and for other pivotal studies as appropriate. For medical devices, a Plan is required to be included in the Investigational Device Exemption application. Generally, per the Guidance, Plans are intended to increase enrollment of participants who are “members of historically underrepresented populations in clinical studies to help improve the strength and generalizability of the evidence for the intended use population.” Additionally, improved representativeness of different demographics in clinical studies can support “more equitable and timely access to medical discoveries and innovations,…improve our understanding of the disease and/or medical product under study and inform the safe and effective use of medical product for all patients.”

Although the Guidance focuses on ensuring adequate representativeness of study participants that reflect different age groups, sexes, and racial and ethnic demographic, the FDA also notes that it recognizes that health disparities and differential access to health care and clinical studies may occur based on other factors such as geographic location, gender identity, sexual orientation, socioeconomic status, physical and mental disabilities, pregnancy status, lactation status and co-morbidity, possibly indicating an expanded view by the FDA.

Under the new Guidance, a Plan must include the sponsor’s goal for clinical study enrollment broken out by specific demographics of sex, age, race and ethnicity. The submitted Plan by the sponsor should use appropriate available sources to obtain information about the prevalence of incidence of the disease and/or condition across an affected population. The estimated prevalence or incidence of the disease should inform enrollment goals. Additionally, the Plan should include background information such as an overview of the natural history of the disease and risk factors.

The sponsor must also, in the Plan, explain how it intends to meet these enrollment goals through recruiting and retention strategies. The FDA notes that these strategies may include implementing sustained community engagement, providing cultural competency and proficiency training for clinical investigation research staff to facility a trusting relationship, and improving access to clinical studies by limited clinical study exclusion criteria, electing study sites that facilitate enrollment such as study sites that have had previous success enrolling diverse populations and considering the accessibility needs of people with disabilities.

Finally, the FDA clarifies that if a clinical study is multi-national, the Plan must describe enrollment goals for all sites, not just the U.S. enrolled participants. However, the FDA recognizes that the lack of uniformity language for population descriptors across the globe may pose a challenge to achieving this goal.

Although compliance with the Guidance is not required until 180 days after the Guidance has been finalized, sponsors should review the Guidance to assess the proposed obligations for Plan composition, content and submission. Sponsors should begin to contemplate how they will comply with these requirements and consider whether they would like to provide any commentary based on the proposed guidance.

Finally, sponsors should be aware that the FDA’s focus on increased diversity in clinical trials will only increase with more time. The Guidance is only further evidence that the FDA has made this issue a priority and more regulations and recommendations will be released over time. Although only draft guidance, sponsors must be prepared to implement proposed changes if and when they may be finalized. Sponsors must also begin to consider how they will expand diversity in any clinical trials and quickly implement future guidance that likely will be announced over the next several years.