On November 9, 2020, FDA issued final guidance on “Enhancing the Diversity of Clinical Trial Populations—Eligibility Criteria, Enrollment Practices, and Trail Designs,” which was first issued as draft guidance in June 2019. FDA intends for the guidance to provide sponsors with recommendations and approaches to help broaden eligibility criteria and improve clinical trial recruitment.

The guidance highlights the importance of broadening eligibility criteria during clinical trials and moving away from “commonly accepted” exclusions of certain populations, including older adults, children, patients with physical and/or mental disabilities, and those with malignancies or certain infections. FDA warns that unnecessary exclusions of such participants could lead to a failure to discover important drug safety information.

FDA recommends two approaches to help broaden eligibility criteria and increase diversity in enrollment. First, FDA encourages inclusive trial practices by enrolling participants across all clinically relevant populations. Second, FDA recommends that sponsors should consider various trial designs and methodological approaches to enroll participants of a broader population, for example, adaptive clinical trials.

In addition, FDA provides other recommendations for improving enrollment by making trial participation less burdensome for participants, adopting practices that enhance inclusiveness, and taking advantage of FDA’s expanded access regulations. The guidance concludes with discussing the current challenges faced for clinical trials involving drugs that treat rare diseases. FDA recommends additional approaches to broaden eligibility, such as engaging with patient advocacy groups on trial design and re-enrolling participants from early-phase trials into later-phase trials.