FDA Guidance: Enhancing the Diversity of Clinical Trial Populations

To further promote and protect public health, it is important that people who are in clinical trials represent the populations most likely to use the potential medical product. In that spirit, today the U.S Food and Drug Administration issued final guidance with the agency’s recommendations on designing and executing clinical trials of drugs and biologics that include people with different demographic characteristics (e.g., sex, race, ethnicity, age, location of residency) and non-demographic characteristics (e.g., patients with organ dysfunction, comorbid conditions, and disabilities; those at weight range extremes; and populations with diseases or conditions with low prevalence).

The final guidance issued today, “Enhancing the Diversity of Clinical Trial Populations–Eligibility Criteria, Enrollment Practices, and Trial Designs,” which was first issued as a draft in 2019, provides the agency’s current thinking on steps to broaden eligibility criteria in clinical trials through inclusive trial practices, trial designs, and methodological approaches. The guidance aims to provide recommendations for how sponsors can increase enrollment of underrepresented populations in their clinical trials.

This 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. Sponsors are encouraged to think about reducing visit frequency, when appropriate, in addition to considering whether flexibility in visit windows is possible and whether electronic communications, such as phone, email, social media platforms, or other digital health technology tools can replace site visits and provide investigators with real-time data.

Additionally, this guidance provides recommendations on broadening clinical trial eligibility criteria for clinical trials 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 includes 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.

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