IOM finds improvements needed in laws governing pediatric drug studies

March 8, 2012

Two laws promoting pediatric drug studies have provided clinicians with a wealth of data on drug efficacy and safety in children, but a new report from the Institute of Medicine suggests that several improvements are needed before they are reauthorized this year. Read how the findings could help Congress strengthen the scope of the current laws to continue critical advances in drug studies that benefit the nation?s children.

Two laws promoting pediatric drug studies have provided clinicians with a wealth of data on drug efficacy and safety in children, but a new report from the Institute of Medicine (IOM) suggests that several improvements are needed before they are reauthorized this year.

A committee from the IOM reviewed progress in pediatric drug studies since passage in the late 1990s of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) and found more than 400 drug labeling changes for pediatric use. Its report, however, calls for more study of drug use in neonates, for earlier completion of pediatric studies, and for eliminating delays in the completion of studies mandated by the US Food and Drug Administration (FDA).

Before BPCA and PREA, most medicines used in children had been tested only in adults, and many drugs were prescribed for children “off label.” BPCA and PREA provide incentives and requirements for drug companies to conduct pediatric studies. The IOM report-required by Congress as part of reauthorizing the laws that expire October 1-suggests that FDA require long-term follow-up studies after products reach the market.

The report notes that although drug makers have stepped up efforts to conduct research in children, it can still take a long time for needed information to be generated. It suggests that Congress specify that drug makers submit plans for pediatric studies at the end of phase 2 adult trials and that the FDA be given more flexibility to impose sanctions for delayed studies.

Many drugs used with neonates are older medications not covered under BPCA and PREA provisions. To promote studies of newer and more widely used drugs that have not been adequately evaluated in these youngest patients, Congress could provide additional resources for short- and long-term neonatal drug studies through the existing BPCA program at the National Institutes of Health, the report says.

The American Academy of Pediatrics supports many of the IOM’s findings, including the need to plan pediatric studies earlier and promote these studies in younger age groups.

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