Federal laws that motivate or require drug and biologic developers to conduct pediatric studies have yielded beneficial information to guide the use of medications in children, according to a new report by the Institute of Medicine(IOM). Still, studies involving children continue to be limited, especially in certain areas such as medication use in newborns and long-term safety and effectiveness in children.
The report identifies ways Congress and the FDA could further improve the utility of clinical information obtained from pediatric studies, including expanding innovative strategies to research drugs and biologics in children, using the FDA’s authority to require long-term pediatric studies of possible safety risks and giving the FDA flexibility to impose sanctions for unreasonably delayed studies.
Conducting research with children is inherently more difficult than with adults. Pediatric patients also offer drug companies a much smaller market and potential economic return. Clinicians often treat children with drugs that have been approved for use in adults but have not been studied with children, even though the drugs may have different risk/benefit profiles for pediatric patients.
Recognizing a shortage in knowledge of how medications affect children, Congress has sought to increase pediatric studies under two laws, the Best Pharmaceuticals for Children Act (BPCA), which offers companies economic incentives to study medications in children, and the Pediatric Research Equity Act (PREA), which requires such studies in specific situations. Both laws are due for reauthorization this year. As specified by Congress, the FDA asked the IOM to review certain aspects of studies that have been conducted under the laws. Read More