Differences in the physiology of adults and children can produce significant differences in the way they respond to drugs and vaccines. Without adequate data on pediatric safety and efficacy of many products, physicians until recently had to make decisions on their use in pediatric patients based upon labeling that only addressed use in adults.

To correct this situation, the US Food and Drug Administration (FDA) has created regulations and Congress has passed a series of laws to ensure that marketed drugs will be evaluated for safety and efficacy in infants, children and adolescents. Initially, voluntary processes and incentives were implemented, but these did not lead to enough products being studied in pediatric patients. From 1998–2003, FDA developed additional processes that made it mandatory to address the use of a product in pediatric patients prior to approval for use in adults, in many cases requiring that studies be done in pediatric patients. The most recent law, the Food and Drug Administration Amendments Act of 2007, extended these incentives and requirements, introduced increased transparency to the process, and improved the review process for studies in pediatric patients.

Regulatory Focus 14:16-21, 2009.