Best Pharmaceuticals for Children: How Far Have We Come?

In response to mounting concerns regarding the safety and efficacy of medications used in pediatric populations, the Best Pharmaceuticals for Children Act (BPCA) was enacted in 2002. The purpose of the BPCA was to promote clinical trials of pharmaceuticals in children that would generate safety and efficacy data. The ultimate goal was pediatric-use approval for more pharmaceuticals and expansion of current drug labeling. Additional legislation, in the form of the Pediatric Research Equity Act (PREA), passed in 2003. The BPCA and PREA were reauthorized in 2007 and made permanent in 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA). Since its inception, the BPCA has stimulated pediatrics-focused clinical pharmacology research and led to hundreds of drug labeling revisions. Despite these major strides, there remains a paucity of data regarding the safe and effective use of medication in pediatric populations. Here, we highlight the existing challenges in optimizing pediatric drug therapy and offer potential solutions.