The final guidance provides the agency’s current thinking regarding steps to broaden eligibility criteria in clinical trials through inclusive trial practices, trial designs, and methodological approaches.
The FDA has issued final guidance on designing and executing clinical trials of drugs and biologics that include people with different demographic characteristics and non-demographic characteristics.1
The final guidance, titled “Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs,” was first issued as a draft in 2019 and now provides the agency’s current thinking regarding steps to broaden eligibility criteria in clinical trials through inclusive trial practices, trial designs, and methodological approaches. The aim of the guidance is to provide recommendations on how sponsors can increase enrollment of underrepresented populations in their clinical trials.
“We have seen these health care disparities, for example, during our fight against COVID-19, as certain segments of the population (eg, older adults, pregnant women, children, and racial and ethnic minorities) are affected in different ways,” FDA commissioner Stephen M. Hahn, MD, wrote in a press release.2 “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.”
Specifically, the guidance offers recommendations on how product sponsors can increase clinical trial diversity by accounting for logistical and other participant-related factors that might limit participation. For instance, clinical trials which require frequent visits to specific sites may place an added burden on participants; thus, 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 or other digital health technology tools can replace site visits and provide investigators with real-time data.
Further, the guidance provides recommendations on broadening clinical trial eligibility criteria for clinical trials of investigational drugs intended to treat rare diseases and recommendations on enhancing enrollment and retention of participants with rare diseases. The guidance also suggests that sponsors should consider early engagement with patient advocacy groups and patients to gather suggestions for designing trials that participants would be willing to enroll in and support.
“Broadening eligibility criteria and adopting more-inclusive enrollment practices should improve the quality of studies by ensuring that the study population is more representative of the population that will use the drug if the drug is approved; by facilitating the discovery of important safety information about use of the investigational drug in patients who will take the drug after approval; and by increasing the ability to understand the therapy’s benefit-risk profile in later stages of drug development for the phase 3 population across the patient population likely to use the drug in clinical practice,” the authors of the guidance wrote.
Additionally, the updated guidance includes other high-level considerations regarding the 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 guidance as well.
“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,” Hahn wrote in the release. “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.”
1. FDA. Enhancing the Diversity of Clinical Trial Populations — Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry. FDA website. Published November 2020. Accessed November 10, 2020. https://www.fda.gov/media/127712/download
2. FDA Offers Guidance to Enhance Diversity in Clinical Trials, Encourage Inclusivity in Medical Product Development [news release]. Published November 9, 2020. Accessed November 10, 2020. https://www.fda.gov/news-events/press-announcements/fda-offers-guidance-enhance-diversity-clinical-trials-encourage-inclusivity-medical-product?utm_source=STAT+Newsletters&utm_campaign=0352b17b14-MR_COPY_01&utm_medium=email&utm_term=0_8cab1d7961-0352b17b14-149715325