FDA Guidance Aims to Promote Clinical Trial Diversity

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“Throughout my career as a cancer doctor and medical researcher, I have been concerned about and have sought to address issues related to healthcare disparities,” U.S. Food and Drug Commissioner Stephen M. Hahn said today (November 9) as he announced a new agency guidance, “Enhancing the Diversity of Clinical Trial Populations–Eligibility Criteria, Enrollment Practices, and Trial Designs,” which was first issued as a draft in 2019.

The guidance provides the agency’s current thinking on steps to broaden eligibility criteria in clinical trials through inclusive trial practices, trial designs, and methodological approaches. It aims to provide recommendations for how sponsors can increase enrollment of underrepresented populations in their clinical trials.

“One important step that researchers and medical product sponsors can take to confront healthcare disparities is to make sure that clinical trials for medical products are more inclusive of multiple populations,” Hahn said.

“We have seen these healthcare disparities, for example, during our fight against COVID-19, as certain segments of the population (e.g., older adults, pregnant women, children, and racial and ethnic minorities) are affected in different ways,” Hahn said. “This difference in impact illustrates why we must encourage developers of any medical product such as treatments or vaccines for COVID-19—as well as medical products more broadly—to endeavor to include diverse populations to understand their risks or benefits across all groups,” he added.

The guidance offers recommendations on how product sponsors can improve clinical trial diversity by accounting for logistical and other participant-related factors that could limit participation. For example, clinical trials requiring frequent visits to specific sites may place an added burden on participants. When appropriate, sponsors are encouraged to consider reducing the frequency of patient visits; adding flexibility in visit windows; and using electronic communications such as phone, e-mail, social media platforms, or other digital health technology tools as replacements for site visits and as sources of real-time data for investigators.

Additionally, the guidance provides recommendations on broadening clinical trial eligibility criteria for studies of investigational drugs intended to treat rare diseases and recommendations on improving enrollment and retention of participants with rare diseases. The guidance notes that sponsors should consider early engagement with patient advocacy groups and patients to elicit suggestions for designing trials that participants would be willing to enroll in and support.

The guidance also addresses other high-level considerations about inclusion of other important groups, including, but not limited to, women (including pregnant women), racial and ethnic minorities, children, and older adults, and provides references to more specific guidances.

Edited by Michael Causey