Clinical Perspective Paediatric clinical pharmacology: Reclaiming the therapeutic orphan

Abstract

The current lack of adequate paediatric drug labelling information has placed children at undue risk because physicians caring for these patients are forced to decide between withholding potentially useful therapies or using drugs outside of the labelling recommendations. The importance of specific drug labelling information for infants and children rests on the frequent observation of substantial age-related differences in drug action and disposition. Because of these observations, the American Academy of Pediatrics has repeatedly called for increased paediatric drug research to support drug labelling information. Recently, the United States Food and Drug Administration has proposed new guidelines to promote paediatric dosing information for drugs which may be used in children. Also, to improve paediatric drug labelling and encourage collaboration between established academic centres and the pharmaceutical industry, the National Institutes of Child Health and Human Development established the Pediatric Pharmacology Research Unit Network, consisting of clinical pharmacology programs with extensive experience in paediatric drug research.

This review will focus on those changes which it is hoped will lead to better paediatric drug labelling. Another objective of this review will be to address some of the ethical and methodologic concerns raised by paediatric drug research. This review will address those aspects of research which define the unique risks, benefits and characteristics of paediatric drug research as described in publications by the American Academy of Pediatrics Committee on Drugs and recent reviews in the pharmacologic literature of the ethical conduct of drug research in children. The consensus of these reviews clearly demonstrates that the potential benefits to be gained through paediatric drug research far outweigh the potential risks. Thus, a body of literature exists which demonstrates that paediatric drug research is methodologically feasible, ethically defensible and vital to the continued improvement of pharmacotherapy of infants and children.