The United States Food and Drug Administration (FDA) released a draft guidance that recommended more diverse patient cohorts in clinical trials and is intended to improve the data the agency receives in support of premarket applications by broadly describing what sponsors should include in their Diversity Action Plans as mandated by Congress.
The draft guidance outlines what should be included in a sponsor’s Diversity Action Plan, which products require such plans, and how they should be submitted to the agency. It also explains how FDA reviewers may decide whether a sponsor can waive the Diversity Action Plan requirement.
The requirements for Diversity Action Plans apply to clinical studies for which enrollment starts after 180 days from the publication of the final guidance. Because sponsors engage in study planning and implementing study activities prior to ] when enrollment commences, FDA does not expect a Diversity Action Plan to be submitted for clinical studies where the following circumstances are present: (1) clinical studies of drugs with protocols submitted within 180 days following the publication of the final guidance where enrollment is scheduled to begin 180 days after publication of the final guidance; (2) clinical studies of devices received by FDA in Investigational Device Exemption (IDE) applications within 180 days after publication of the final guidance; or (3) clinical studies of devices that do not require an IDE application to be submitted to FDA that are approved by an institutional review board (IRB) or independent ethics committee (IEC) within 180 days after the date of publication of the final guidance.
Under the guidance, the Diversity Action Plan must include the sponsor’s rationale and patient enrollment goals based on age, ethnicity, sex, and race. While the FDA primarily focuses on Diversity Action Plans including different age groups, sexes, and racial and ethnic demographic characteristics, the agency also recognizes the broader issues regarding health disparities and differential access to health care and clinical studies that may occur based on other factors, including but not limited to: geographic location, gender identity, sexual orientation, socioeconomic status, physical and mental disabilities, pregnancy status, lactation status, and comorbidity, and encourages sponsors to consider such additional factors when developing Diversity Action Plans.
“In general, clinical study diversity helps ensure that clinical studies appropriately test the product in a representative sample of the product’s intended use population,” says the guidance. “Factors to consider when setting enrollment goals include demographic characteristics (e.g., race, ethnicity, sex, age group), clinical characteristics (e.g., presence of comorbidities, disease etiology), and other characteristics (e.g., access to standard preventive and diagnostic care, access to standard treatments of the clinically relevant population).”
The guidance also provides general recommendations for sponsors who may wish to publicly post key information regarding their Diversity Action Plans. The guidance, however, is not intended to address all issues related to the clinical development of medical products such as the design of clinical studies, clinical study endpoints, or the data necessary to support a marketing submission; sponsors should refer to the appropriate FDA guidance documents for FDA recommendations on these matters.
As is often the case with FDA guidance documents, it does not fully establish legally enforceable responsibilities but instead describes the Agency’s current thinking on a topic. In this case, however, an exception to the general rule applies and insofar as the guidance specifies the form and manner for submission of a Diversity Action Plan, it will have binding effect once the guidance is finalized.
The FDA is asking for comments on the draft guidance to be submitted by September 26, 2024. When the draft guidance is finalized, it will replace the 2022 guidance from the agency on diversity plans for clinical trials. The 2022 guidance was issued prior to the Food and Drug Omnibus Reform Act of 2022 (FDORA) becoming law, which requires the FDA update guidance relating to the format and content of Diversity Action Plans.