FDA Issues Ethical Guidance to Protect Children who Participate in Clinical Trials

The U.S. Food and Drug Administration issued a draft guidance to assist industry, sponsors and institutional review boards on considerations when enrolling children in clinical trials.

Historically, children were not included in clinical trials because of a misperception that excluding them from research was in fact protecting them. This resulted in many FDA-approved, licensed, cleared or authorized drugs, biological products, and medical devices lacking pediatric-specific labeling information. If the medical product was the best available treatment option for the child, doctors were left with no choice but to use a product that had not been reviewed by the FDA for safety and effectiveness in children. It became clear that children can be better protected by including them in clinical research. 

“Children need access to safe and effective medical products and health care professionals need data to make evidence-based decisions when treating children. However, children are a vulnerable population who can’t provide consent for themselves and are afforded additional safeguards when participating in a clinical investigation,” said Dionna Green, director of the FDA’s Office of Pediatric Therapeutics. “The best way to provide children with safe and effective treatment options is by including them in clinical research and providing these additional safeguards to protect them during clinical trials.”

The draft guidance describes the ethical framework for protecting children in clinical research, which includes risk and benefit considerations. The draft guidance outlines and explains fundamental concepts for the ethical framework that IRBs, sponsors and industry should consider when reviewing or conducting clinical trials involving children.

This includes the scientific necessity of conducting a clinical investigation in children, how to evaluate whether there is a prospect of direct benefit to the child, and how to assess risk among other considerations.

The agency is accepting comments on the draft guidance. Any comments should be submitted within 90 days to ensure that the agency considers them when finalizing the draft guidance. The FDA remains committed to protecting children in clinical trials and assuring the safety and effectiveness of medical products for children.