The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) are both up for congressional re-authorization this year. On February 29, 2012, the Institute of Medicine released a study committee report examining these and other pieces of legislation intended to increase the number of safe and effective medical treatments available for children.
The study committee found that economic incentives to conduct pediatric studies, coupled with FDA authority to require pediatric studies when certain conditions are met, have been effective. However, mandating studies is not enough to guarantee success. Barriers include difficulties with enrollment, delaying pediatric trials until late in the drug development process, and the lack of information about the long-term safety of drugs that may be taken by pediatric patients for many years or even decades.
The overall conclusion of the report is that FDA’s efforts to improve the timeliness, quality and informational value of pediatric studies should continue.