The FDA has published draft guidance outlining an ethical framework for involving children in clinical trials of drugs and devices, as well as trial design considerations.

The guidance offers what the agency deems to be fundamental ethical considerations that come into play when children are involved in trials. For instance, the guidance offers IRBs direction on approving trials that intend to enroll children and ways to assess whether an intervention/procedure offers a prospect of direct benefit to them. It also describes risk categories for interventions/procedures that don’t offer a prospect of direct benefit and methods to assess risks for those that do, in addition to other items.

The guidance also delves into trial design for both drugs and devices, covering general factors as well as specifics. Overall, trials that involve children “should be designed to maximize the amount of information gained and minimize the number of subjects involved,” the guidance advises.

“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,” Dionna Green, director of FDA’s Office of Pediatric Therapeutics, said. “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 guidance shares a number of points to consider when designing trials that involve pediatric patients:

• Age and physiological maturity of the child;
• Natural history of the condition;
• The current severity of the condition in the child;
• The presence of other complicating conditions;
• The safety/effectiveness of the drug or device in older participants or safety/effectiveness expected based on other clinical/nonclinical studies; and
• The likely duration of drug or device use and its effect on the growth and development of the child.

The guidance distinguishes between the differing challenges of drugs and medical devices, noting that devices come with different hurdles because of their varying applications and the range of technology they use.

For devices, the guidance advises sponsors to consider the available clinical data when putting a trial together and notes that trials for indications involving both adults and children may be able to be designed as a single pivotal trial that enrolls both groups. Doing this can reduce the burden of multiple trials and optimize sample sizes. The guidance also notes that in certain situations, expected benefit and safety can be derived without separate studies of each pediatric subgroup, although “every effort should be made to gather data that adequately address each targeted pediatric subgroup for the proposed indication.”

Read the full draft guidance here: https://bit.ly/3SaRVHB.