the relevant expertise of investigators and the appropriateness of the facilities to be used. The plans are then reviewed as part of the NIH peer review process for proposals. The 1998 NIH guidelines also describe the federal rules for protecting children in research.

Congress encouraged, but did not require, NIH to establish pediatric research priorities (U.S. Congress, 1995; NIH, 1998a). Although NIH does not appear to have developed an overall set of priorities, some individual institutes have identified priorities for certain clinical problems or services, including kidney disease (NIDDK, 2001), HIV/AIDS (NIH, 2003b), and emergency medical services (NIH, 2003b).


Recent years have seen a number of actions to encourage research involving children and help investigators cope with the many methodological, practical, and ethical challenges of pediatric studies. One apparent result is an increase in the number of children participating in research. Another is an increase in the number of priority drugs that have labeling information for at least some pediatric age groups.

More can be done. Chapter 8 includes a recommendation for strengthening educational programs to develop pediatric investigators who are prepared to design and conduct valid, ethical clinical research. In addition, to ensure the continuation of well-designed and well-conducted pediatric research that will improve children’s health, federal policymakers should sustain and extend other aspects of the critical financial and infrastructure for this research. They should likewise support important research that is often not attractive to commercial sponsors, including long-term studies and projects to improve outcome measures relevant to infants, children, and adolescents. Because most pediatric conditions are sufficiently uncommon that statistically sound, ethical research requires multiple study sites, the federal government should continue to establish and fund discipline-specific, age-relevant research groups or consortia with the expertise and administrative infrastructure to conduct multicenter studies.

Furthermore, as research continues to become more international, it is important for governments, investigators, industry, and international organizations to cooperate to support the conduct of ethical multinational pediatric studies, increase the pace of therapeutic development for rare pediatric conditions, and move toward greater consistency in the regulatory protections for child participants in research. The next chapter, which summarizes the regulatory framework for protecting human research participants in the United States, also briefly reviews international guidelines and efforts to develop consistent or uniform—that is, harmonize—regulations across countries.

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