Pediatric Drug Development: Focus on US Regulatory Issues

The need for pediatric drug development arises from the facts that drug information about adults cannot routinely be extrapolated to children, and clinical trials in children cannot routinely be based upon adult trials for the same product. Drugs have often been dispensed on the basis of extrapolation of adult doses, anecdotal practitioner experience and sub-optimal studies. In reality, most drugs prescribed for children do not have sufficient pharmacokinetic, pharmacodynamic, efficacy or safety data to support their use. Almost half of the medications prescribed for children are used off label. Off-label use peaks in the newborn population, where sick, immature patients often receive multiple drugs, 60%-90% of which are unlabeled.