still be prescribed for children based on physicians’ judgment about how data from studies with adults might be extrapolated to children. Because children differ physiologically from adults in myriad ways that can affect how drugs work in the body, extrapolation based on adult drug doses and children’s weight or age can be dangerous and lead to underdosing, overdosing, or specific adverse effects not evident in adults.

The U.S. Congress, the Food and Drug Administration (FDA), and the National Institutes of Health (NIH) have acted in recent years to expand research involving children. Notwithstanding the expected benefits of these efforts, some caution is appropriate. Unlike most adults, children usually lack the legal right and the intellectual and emotional maturity to consent to research participation on their own behalf. Their vulnerability demands special consideration from researchers and policymakers and additional protections beyond those provided to mentally competent adult participants in research.

In the United States, research that is supported, conducted, or regulated by the U.S. Department of Health and Human Services (DHHS) is now subject to a (mostly) common set of regulations to protect adult and child participants in research. Nonetheless, deficiencies in the conduct of human research—most of which are fairly minor but some of which result in deaths or serious injuries—continue to be revealed.

Concerns about the adequacy of the system for protecting child participants in research, combined with the public commitment to expanding clinical research involving children, provided the impetus for this Institute of Medicine (IOM) report, which was requested in the Best Pharmaceuticals for Children Act of 2002 (P.L. 107-109). The legislation charged the IOM with preparing a report that reviewed federal regulations, reports, and research and that made recommendations about desirable practices in clinical research involving children. Specifically designated topics were (1) the appropriateness of the regulations for children of various ages, (2) the interpretation of regulatory criteria for approving research, (3) the processes for securing parents’ and children’s agreement to a child’s participation in research, (4) the expectations and comprehension of children and parents about participating in research, (5) the appropriateness of payments related to the child’s participation in research, (6) compliance with and enforcement of federal regulations, and (7) the unique roles and responsibilities of institutional review boards (IRBs).

The report, prepared by a 14-member committee of the Institute of Medicine, focuses primarily on clinical research involving preventive, diagnostic, treatment, or similar interventions and direct interactions with children. It stresses three broad themes:



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