Important New FDA Guidance on Postmarketing Approaches to Obtain Data on Under-Represented Populations in Clinical Trials

On August 10, 2023, the U.S. Food & Drug Administration (FDA) published new guidance on obtaining data on underrepresented populations in clinical trials after approval of a New Drug Application (NDA) or Biologic License Application (BLA).

Drug and Biologic sponsors are required to present information from premarket clinical trials on the safety and effectiveness of drugs in terms of gender, age, and racial subgroups. Clinical trials should include patient populations that are historically under-represented in clinical research (e.g., populations based on race, ethnicity, sex, or age). However, because sometimes sponsors are unable to enroll under-represented populations in premarket clinical trials, FDA is providing guidance on when it may be appropriate to collect such data in the postmarketing setting. 

Specifically, FDA guidance discusses the following: 

• Mechanisms by which FDA can require or request information on safety and effectiveness be collected in the postmarketing setting, 
• Design and statistical considerations for subpopulation analyses,
• Postmarketing approaches to obtain information on the benefit-risk profile in under-represented clinical trial populations.