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Summer 2004 Vol. 4 No. 2

Table of Contents

©Comstock ImagesPutting Research to the Test

Improvements Needed in Clinical Studies Involving Children

Society wants kids to benefit from many of the dramatic medical advances that stem from scientific research. But herein lies the dilemma: No one wants to put children at risk from research participation -- even when their involvement may help improve children's health and health care overall.

It is possible to strike an appropriate balance, protecting those kids who take part in clinical research today while helping tomorrow's children reap more benefits from biomedical science, says a new report from the Institute of Medicine. For starters, an effective and adequately funded system for protecting all human research participants is a must. However, safeguarding child participants requires extra attention and resources because kids lack the intellectual and emotional maturity, and usually the legal right, to consent to experiments on their own behalf.

The federal government should play a major role in this balancing act. Existing federal rules to protect children from risky or unethical clinical research should be extended to cover all pediatric research in both the public and private sectors, the report says. Currently, the rules apply primarily to studies that are supported by the U.S. Department of Health and Human Services or regulated by the Food and Drug Administration, although many research institutions voluntarily apply them to all of their studies.

The government also should offer better guidance -- and in more accessible formats -- to clinical researchers and institutional review boards (IRBs) to help them interpret federal rules, which are more stringent for children than adults. IRBs -- the bodies responsible for approving human research -- should be more thorough and explicit in judging whether research involving children meets the highest ethical and scientific standards, the report adds.

In recent years, Congress, the National Institutes of Health, and FDA have expanded pediatric research, especially studies that test the safety and efficacy of drugs for infants, young children, and adolescents. In medical circles, these groups have been called "therapeutic orphans" because drug companies have been reluctant to conduct tests involving them after medications have been approved for adults. Physicians often have to choose not to prescribe particular medications for children, or must extrapolate drug doses based on data from studies with adults, the report notes.

But such practices may come with a high price. Physiologically, children are not just "little adults." Extrapolation may lead to children getting too much or too little of a drug, or suffering adverse effects not seen with adults. Plus, this type of calculation cannot generate a reliable knowledge base for future clinical care, said the committee that wrote the report.

In weighing possible harm from pediatric research protocols, reviewers of clinical studies involving children should compare potential risks with those commonly encountered by healthy, average children, the report says. Research should not expose participants to higher levels of risk simply because they are already imperiled by illness, unsafe neighborhoods, or other aspects of their daily lives.

Parents must be in the loop, too. Discussions with them and, when appropriate, children should allow sufficient time for questions and explanations of the research, the report emphasizes. In addition, IRBs should adopt explicit written policies that define ethical payment arrangements for children's participation in research.

The report points out numerous holes in the knowledge base regarding pediatric research and protection programs for research involving children. HHS should develop and carry out a plan for collecting and reporting data on pediatric research and its oversight, the report says. Also, IRBs that review protocols for pediatric clinical studies should include at least three members with expertise in pediatric research, research ethics, children's health care, and child development -- or the boards should consult with people who have this knowledge.   -- Vanee Vines

The Ethical Conduct of Clinical Research Involving Children. Committee on Clinical Research Involving Children, Board on Health Sciences Policy, Institute of Medicine (2004, 448 pp.; ISBN 0-309-09181-0, available from the National Academies Press, tel. 1-800-624-6242; $47.95 plus $4.50 shipping for single copies).

The committee was chaired by Richard E. Behrman, executive chair, Education Steering Committee, Federation of Pediatric Organizations, Palo Alto, Calif.; and clinical professor of pediatrics at both the University of California, San Francisco, and George Washington University, Washington, D.C. The study was sponsored by the National Institute of Child Health and Development.

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Copyright 2005 by the National Academy of Sciences