Nearly 100 medicines for sale in the United States have received pediatric labeling since the late 1990s based on new clinical studies to determine appropriate dosages, safety, efficacy, and formulations for children, according to an analysis recently completed by the Tufts Center for the Study of Drug Development.

The new labeling information was developed between 1998 and 2005 in response to requests for pediatric clinical studies by the U.S. Food and Drug Administration (FDA).

"The increase in pediatric labeling signals a major advance in pediatric medicine," said Christopher Milne, assistant director of Tufts CSDD and author of the study. "This will improve the overall health of children, who comprise 25% of the U.S. population."

Tufts CSDD undertook the analysis in advance of the October 2007 sunset of the U.S. law granting pediatric exclusivity. Under this law, in return for conducting pediatric studies, drug sponsors are granted a six-month marketing exclusivity period during which the FDA may not approve another company's abbreviated new drug application submitted for the same drug. A similar program is being considered in the European Union.

Before the law went into effect, about 70% of drugs used in children in this country had been dispensed without adequate pediatric dosing information, according to Tufts CSDD. Limited return on investment, difficulty enrolling and studying pediatric patients, and exposure to potential product liability previously discouraged conducting pediatric studies.

"The new incentives are working," Milne said, noting that drug developers initiated pediatric studies in response to 95% of FDA requests between 1998 and 2001. He said the FDA had anticipated an 80% response rate.

Despite advances in pediatric labeling, difficulties remain for conducting pediatric clinical trials, said Milne. They include the occurrence of adverse drug events idiosyncratic to children, long-term effects of chronic use drugs on growth and development, and unanticipated differences in adult and juvenile forms of common diseases, such as depression.

"The good news is that none of these problems would have surfaced as quickly or as publicly without the pediatric exclusivity program," he said.

The Tufts CSDD analysis, summarized in the July/August Tufts CSDD Impact Report, also found that:

* Fewer FDA requests for pediatric studies and declining interest in incentives led to a drop in pediatric studies in 2000-04.

* Pediatric study costs can vary by 20-fold due to length, methodology, regulatory requirements, testing protocols, geographic region, and therapeutic area studied.

* Pediatric study costs are expected to rise through this decade as regulatory requirements expand, patients become more difficult to recruit, and trained investigators become scarcer.

About the Tufts Center for the Study of Drug Development

The Tufts Center for the Study of Drug Development (csdd.tufts.edu) at Tufts University provides strategic information to help drug developers, regulators, and policy makers improve the quality and efficiency of pharmaceutical development, review, and utilization. Tufts CSDD, based in Boston, conducts a wide range of in-depth analyses on pharmaceutical issues and hosts symposia, workshops, and public forums on related topics, and publishes the Tufts CSDD Impact Report, a bi-monthly newsletter providing analysis and insight into critical drug development issues.

csdd.tufts.edu