Children in Clinical Trials: FDA’s Proposed Rules of Engagement

FDA issued draft guidance on ethical considerations and special safeguards for having children participate in clinical trials.

The new statement addresses how to protect children in clinical investigations for drugs, biological products, and medical devices. Some of the fundamental concepts that the new guidance included for consideration by industry, sponsors, and institutional review boards (IRBs) were:

• Scientific necessity of conducting a clinical investigation in children
• Risk categories for interventions or procedures that do not offer a prospect of direct benefit to the child
• How to evaluate whether an intervention or procedure offers a prospect of direct benefit to the child
• Assessment of risk for intervention of procedures with a prospect of direct benefit
• Component analysis of the risks of intervention or procedures
• Potential for review, under a regulatory revision of research that is not otherwise approvable by an IRB
• Parental or guardian permission and child assent

“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, MD, director of the FDA’s Office of Pediatric Therapeutics, in a press release.

FDA acknowledged that children had been excluded from trials in the past in the erroneous belief that this would protect them. This has led to many drugs, biological products, and medical devices going to market lacking pediatric-specific labeling — forcing these patients to rely on products thought to be the best available treatment option without the backing of a rigorous FDA review of safety and effectiveness.

In 2017, the American Academy of Pediatrics deemed off-label use of medical devices “often appropriate” in pediatric populations but urged the generation of more data for pediatric device labeling, especially when it comes to high-risk devices. As candidates for cardiac devices, for instance, children may encounter sizing difficulties and anatomic issues.

“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,” Green said.

The FDA’s draft guidance was developed by the agency’s Office of Pediatric Therapeutics with contributions from the Center for Drug Evaluation and Research, the Center of Biologics Evaluation and Research, and the Center for Devices and Radiological Health.

The FDA is accepting comments from the public on the draft for the next 90 days.