Age-Appropriate Pediatric Formulation Development

By Jim Huang, PhD, Founder & CEO, Ascendia Pharmaceuticals

It has been recognized that the requirements of drug formulation for children are different from those of adults. The lack of appropriate pediatric formulations is the main obstacle for use of many drugs in children. Historically, children have been treated with off-label used medications by means of extemporaneous compounding, which, however, is not ideal due to potential safety/efficacy concerns. As a result of the US Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA) of 2007 and European Union medicinal products for pediatric use in 2006, more and more companies are encouraged to develop and generate clinical data for the pediatric population in order to gain accelerated drug approval and extended market exclusivity. The development of pediatric formulations can be challenging due to specific requirements of the patient population. A pediatric formulation should consider the following factors: difference in physiological and pharmacokinetic of patient populations, dosage form selection, route of administration, dose accuracy, dose flexibility, drug and excipient tolerability (safety and toxicity), patient compliance (palatability/swallowability), stability, and drug accessibility.