Draft guidance focuses on improving the diversity of patient populations in clinical trials.

According to a new draft guidance from the U.S. Food and Drug Administration (FDA) on increasing patient diversity in clinical trials, trials sponsors have historically excluded some groups of patients from clinical studies without “strong clinical or scientific justification.”

Some patients with certain concomitant illnesses are excluded due to concerns regarding susceptibility to adverse effects, as are those that have other diseases or are taking other medications that would prevent the establishment of clear results for the investigational drug. However, by excluding these types of patients, clinical studies are often limited to a narrow population that does not represent the wider patient population, which varies with respect to weight, age, gender, race, or other illness and organ dysfunction, who may eventually receive the drug. 

To address this issue, the new FDA guidance provides recommendations for increasing diversity of clinical trial participants through enrichment and inclusive trial practices and design. One example provided involves conducting substudies with higher-risk populations at sites with expertise in working with these types of patients. Another is the provision of expanded access, which is generally only granted to treat, monitor or diagnose a patient’s illness under a compassionate-use rationale.

For rare diseases trials, the guidance provides a separate set of recommendations. According to the agency, “Because rare disease often affect small, geographically dispersed patient populations with disease-related travel limitations, special efforts may be necessary to enroll and retain these participants to ensure that a broad spectrum of the patient population is represented.”

Public comments will be collected through August 6, 2019.