Over the past few decades, FDA has promoted enrollment practices that would lead to clinical trials that better reflect the population most likely to use the drug if the drug is approved, primarily through broadening eligibility criteria. Despite these efforts, challenges to participation in clinical trials remain, and certain groups continue to be underrepresented in many clinical trials. This guidance recommends approaches that sponsors of clinical trials intended to support a new drug application3 or a biologics license application can take to increase enrollment of underrepresented populations in their clinical trials.
This guidance considers both demographic characteristics of study populations (e.g., sex, race, ethnicity, age, location of residency) and non-demographic characteristics of populations (e.g., patients with organ dysfunction, comorbid conditions, disabilities, those at the extremes of the weight range, and populations with diseases or conditions with low prevalence). Enrolling participants with a wide range of baseline characteristics may create a study population that more accurately reflects the patients likely to take the drug if it is approved and allow assessment of the impact of those characteristics on the safety and effectiveness of the study drug.