The National Institutes of Health and the Best Pharmaceuticals for Children Act

Abstract

The majority of drugs used to treat children are not labeled for use in children. The Best Pharmaceuticals for Children Act of 2002, re-authorized as the US FDA Amendments Act of 2007, directs the National Institutes of Health (NIH) to sponsor pediatric clinical trials of drugs lacking patent protection, if the FDA request for studies has been declined. The NIH is currently sponsoring 17 clinical studies. Challenges encountered include a paucity of investigators who are trained in pediatric clinical pharmacology; inadequate knowledge of the mechanisms of drug action in a growing child; and lack of pediatric formulations.