Paediatric clinical trials: Redressing the imbalance

Compared with the quantity and quality of information available for adults, the data guiding the dosing, efficacy and safety of medications for children have lagged substantially. This is ironic, because tragedies that primarily involved children spurred the passage of the major pieces of legislation that govern the development of drug products. Clinicians faced with the prospect of confining their practice to medications with adequate information have frequently resorted to prescribing drugs for unapproved uses ( different dose, frequency, age group, route, indication or formulation). Although a long time in coming, the past six years, particularly in the United States, have witnessed a new era in drug development for children - an era that is still in its infancy, but which is now showing signs of maturation. This paper will review some of the history and current progress in paediatric drug development, with an emphasis on the developments in the United States, and will examine some of the current controversies and prospects for future progress.