Newborns and drug studies: The NICHD/FDA newborn drug development initiative

Abstract

Background:Progress has been made in research on the effects of drug therapy on pediatric patients, but neonates are still an understudied population. Those most likely to receive drug therapy (eg, preterm infants) are least likely to be studied. Objectives:The purposes of this article are to summarize an initiative developed jointly by the National Institute of Child Health and Human Development (NICHD) and the US Food and Drug Administration (FDA) and to introduce a series of articles developed as a result of this initiative. Methods:Information for this article was gathered from the proceedings of a workshop cosponsored by the NICHD and the FDA that took place March 29 and 30, 2004, in Rockville, Maryland. Results:Dosing based on use in adults and older children has resulted in adverse events among newborn infants, and may have long-term effects. Moreover, formulations appropriate for use in neonates are often unavailable, and compensatory efforts such as mixing crushed tablets into formula may interfere with accurate dose delivery. Under the Best Pharmaceuticals for Children Act of 2002, government agencies work with experts in pediatrics and pediatric research to develop and prioritize a list of off-patent drugs for which pediatric studies are urgently needed. Four such listings were published in the Federal Register from January 2003 through January 2005. The NICHD and FDA have also initiated the Newborn Drug Development Initiative (NDDI), a multiphase program to determine gaps in knowledge concerning neonatal pharmacology and clinical trial design and to explore novel study designs for use in newborns, with the ultimate goal of increasing our knowledge about the safety and efficacy of drugs used to treat newborns. Conclusions:Most drugs used to treat newborns still lack appropriate dosing, efficacy, and safety studies in this vulnerable population. The NICHD and FDA developed the NDDI as an ongoing process to identify and suggest strategies for addressing obstacles to conducting drug trials in the newborn.