The Ethical Conduct of Clinical Research Involving Children

In recent decades, advances in biomedical research have, each year, helped to save or lengthen the lives of tens of thousands of children around the world, prevent or reduce illness or disability in many more, and improve the quality of life for countless others. Beyond the infants, children, and adolescents directly affected, the benefits of research extend to the families, friends, and communities who love and care for them. Since the 1950s, for example, researchers have created vaccines against polio, measles, mumps, and a number of other childhood infections that have dramatically cut deaths, disability, and discomfort from these diseases. Children and their families have also benefited from research demonstrating the harm or ineffectiveness of what were once standard therapies, for instance, high-dose oxygen for premature infants. Despite these advances, pediatricians and others have argued that infants, children, and adolescents have not shared equally with adults in advances in biomedicine. In particular, many drugs with potential pediatric uses have not been tested in studies that include children. These drugs may 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.