Children in Clinical Trials: Proposed FDA Rules of Engagement


The FDA has released draft guidelines on ethical considerations and special safeguards for involving children in clinical trials.

The new statement addresses how to protect children in clinical investigations of drugs, biologicals and medical devices. Some of the fundamental concepts that the new guidelines included for consideration by industry, sponsors and Institutional Review Boards (IRBs) were:

  • Scientific need to conduct a clinical investigation in children
  • Risk categories for interventions or procedures that do not offer the prospect of direct benefit to the child
  • How to assess whether an intervention or procedure offers the prospect of direct benefit to the child
  • Evaluation of the risk of intervention of the procedures with a perspective of direct benefit
  • Component analysis of the risks of intervention or procedures
  • Potential for review, as part of a regulatory review of research that is not otherwise approvable by an IRB
  • Parental or guardian authorization and child’s consent

“Children need access to safe and effective medical products and healthcare professionals need data to make evidence-based decisions when treating children. However, children are a vulnerable population that cannot give consent on their own and enjoy 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.

The FDA has acknowledged that children have been excluded from trials in the past in the mistaken belief that it would protect them. This has led to many drugs, biologics and medical devices being marketed without pediatric-specific labeling – forcing these patients to rely on products seen as the best treatment option available without the support of a rigorous review of safety and effectiveness by the FDA. .

In 2017, the American Academy of Pediatrics found that off-label use of medical devices was “often appropriate” in pediatric populations, but called for the generation of more data for the labeling of pediatric devices, especially when These are high risk devices. As candidates for cardiac devices, for example, children may encounter sizing difficulties and anatomical issues.

“The best way to provide children with safe and effective treatment options is to include them in clinical research and provide those extra safeguards to protect them during clinical trials,” Green said.

The draft FDA guidelines were developed by the agency’s Office of Pediatric Therapeutics with input 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 public comments on the draft for the next 90 days.

  • James Lopilato is an editor for Medpage Today. It covers a variety of topics explored in current medical science research.


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