Strategies for Pediatric Drug Evaluation: A View from the Trenches

Abstract

Strategies for pediatric drug evaluation include nine essential areas. Ethical guidelines no longer restrain drug evaluations in children because Department of Health and Human Services (DHHS) and American Academy of Pediatrics (AAP) guidelines enable institutional review boards (IRBs) to protect children sufficiently while participating as research subjects. The opportunistic nature of study design across several age groups is accommodated by a team approach operating in comprehensive research facilities designed to meet the special needs of children. Technological innovations foster application of sensitive analytical methods to a disparate array of biologic fluids from children. Generally recognized as requiring more financial support, pediatric drug studies maximize data to obtain Food and Drug Administration (FDA) approved labeling. Manpower has increased because of recent events for career path definition. Increased levels of institutional cooperation are identified from industry, the FDA, academic medical centers, and the National Institutes of Health (NIH). Integration of cooperative efforts reveals numerous advantages to industry when pediatric pharmacology research unit (PPRU) resources are used for pediatric clinical trials. Medicolegal affairs recognize the low risk of pediatric drug evaluations. While the marketplace economics do not often support an investment in pediatric labeling, alternatives for cost recovery or FDA Orphan Product Drug (OPD) program assistance are likely. Coordination and application of all the above strategies are incorporated into logical and necessary steps for execution of pediatric clinical trials. This review presents a highly pragmatic approach (a view from the trenches) which attends to each strategy in order to expedite FDA approval of drug labeling for pediatric indications.