FDA Offers Guidance to Enhance Diversity in Clinical Trials

The U.S. Food and Drug Administration has issued final guidance to ensure that people who are in clinical trials represent the populations most likely to use the potential medical product.

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In an effort to address issues related to disparities in health care, the U.S. Food and Drug Adminstration (FDA) has issued its final guidance on enhancing diveristy in clinical trials. In a statement issued on November 9, 2020, Stephen M. Hahn, MD, FDA commissioner of food and drugs, noted that one important step that researchers and medical product sponsors can take to confront health care disparities is to make sure that clinical trials for medical products are more inclusive of multiple populations.

The statement reads, in part:

We have seen these health care 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. 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.

To further promote and protect public health, it is important 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.

Clinical trials, and the people who volunteer to participate in them, are essential to help develop safe and effective medical products to fight diseases and illnesses. The FDA remains committed to increasing enrollment of diverse populations in medical product development and will continue to engage with federal partners, medical product manufacturers, medical professionals, and health advocates to encourage this important goal. 

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