Previous FDA efforts to address the problem of inadequate pediatric testing and drug labeling information had been unsuccessful. In 1997, the Congress recognized the importance of learning more about how drugs work in children by including in FDAMA a financial incentive for pharmaceutical manufacturers and drug sponsors to conduct pediatric studies and submit the results to FDA. The drug is in a class or for an indication for which additional therapeutic or diagnostic options are needed for pediatric patients. Officials at NICHD, which has conducted many pediatric drug studies, said costs vary depending on the number of children participating and type of drug being studied. Studies Have Led to Better Labeling and Infrastructure

Research conducted under the pediatric exclusivity provision is providing new and useful information about whether and how drugs work in children. As of April 1, 2001, labels for 18 of the 28 drugs granted marketing extensions had been changed to incorporate findings from research conducted to obtain the extensions. Second, the law provides no incentive to conduct pediatric research on drugs for which patents and marketing exclusivity have expired. We found a difference of opinion on whether a marketing extension should be contingent on label changes.
What GAO Found

Children fall ill with many of the same diseases as adults and are often treated with the same drugs. However, only about 25 percent of drugs used today have been labeled for pediatric patients. The lack of pediatric testing and labeling can place children at risk of under- or overdosing, and the lack of age-appropriate formulations, such as liquids or chewable tablets, can result in improper administration of drugs. The pediatric exclusivity provision of the Food and Drug Administration Modernization Act of 1997 has successfully encouraged drug sponsors to generate needed information on how drugs work in children. A wide range of drugs are being studied in many therapeutic areas. The infrastructure for conducting pediatric trials has also been greatly strengthened, which should help to support continued progress. Although several drug labels have been changed to incorporate findings from research done under the pediatric exclusivity provision, label changes typically occur long after the Food and Drug Administration has granted the extension of market exclusivity. In addition, there continues to be little incentive to conduct pediatric research on off-patent drugs.