The US Food and Drug Administration is recommending sponsors include a plan to increase diversity when proposing clinical trial designs for investigational products to agency reviewers. The move is part of the Biden administration’s focus on addressing racial and ethnic disparities in health care. On 13 April, the FDA published a draft guidance titled, Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials. In it the agency recommends sponsors submit a “Race and Ethnicity Diversity Plan” whenever submitting an application for an investigational new drug (IND), biologics license application (BLA) or an investigational device exemption (IDE). Regulators note they will consider the plan “an important part” of the product’s development program.
More on Guidance
FDA’s recommendations arrive at a time in which several bills focused at clinical trial diversity have been recently introduced in Congress. Although the proposed bills take a variety of approaches, at least one pending bill directs FDA to issue regulations requiring sponsors to submit diversity plans to the agency and mandate post approval surveillance for sponsors who fall short of diverse enrollment goals. In issuing this draft guidance, FDA has articulated a framework in the absence of legislative action or notice and comment rulemaking that aims to make clinical trial sponsors take more intentional steps towards achieving research diversity in their studies.
FDA cites health care access inequities, racism, and historical mistrust of the clinical research system as reasons for the absence of underrepresented racial and ethnic populations in clinical research. Notably, FDA also highlights that “increasing reliance” on small studies, intermediate endpoints, and other methods to expedite approval of medical products underscores the “importance of prospectively defining the approach to generating data for a broader and more diverse population early in the development program.”
The draft guidance outlines the content of five elements recommended for inclusion in the Plan. Specifically, the plan should describe available data on the pathophysiology of the disease or condition in underrepresented racial and ethnic populations. Additionally, it should discuss any variations across racial and ethnic populations in the applications or uses of available prevention, screening, or diagnostic strategies and treatments for the disease or condition.
Additionally, sponsors should describe the planned trials for the program. This section of the Plan would include outlines of the proposed studies and how the inclusion of underrepresented racial and ethnic populations would be addressed.
Sponsors should define enrollment goals for underrepresented racial and ethnic participants based on the epidemiology of the disease and a priori information that may impact outcomes across racial and ethnic groups. The authors note that in some cases greater than proportional enrollment of certain populations may be needed to study potential important differences. The Plan should further detail the specific plan of action and strategies to enroll and retain diverse participants throughout the trial, including site selection and access considerations, community engagement efforts, and plans to reduce access burdens due to the design and conduct of the study.
As the Plan is updated and the study proceeds, sponsors will also discuss the status of the enrollment goals with FDA. If the enrollment goals are not met, sponsors may be expected to discuss a plan and justification for collecting relevant data in the post-marketing setting.
The Plan should also outline the sponsor’s proposed approach to collecting data to explore the potential differences in the drug’s safety and effectiveness associated with racial and ethnic backgrounds throughout the product’s life cycle, and plans for pediatric studies that include racial and ethnically diverse populations as part of the medical product development program.