The trials and tribulations of pediatric drug trials

Children are not little adults. This oft-repeated mantra implies that to study the safety and efficacy as well as the pharmacokinetics of drugs in children, it is not sufficient to simply extrapolate adult data. This is the position of the American Academy of Pediatrics. Moreover, the Food and Drug Administration (FDA) grants approval for drugs based on the populations in which they were studied. In partial seizures in older children, one can usually presume that drugs effective in adults with partial seizures will also be effective in children, although the safety and pharmacokinetics will be different as noted in a National Institute of Neurologic Disorders and Stroke consensus conference on the development of new antiepileptic drugs (AEDs) in children.¹ In neonates and infants, no such assumptions can be made.^1,2 Yet despite financial incentives to pharmaceutical manufacturers in the form of additional patent exclusivity period on a product, there are few drugs (and in particular, few AEDs) that have obtained a pediatric indication, particularly in infants and younger children. The study of oxcarbazepine in very young children by Piña-Garza et al.³ in this issue of Neurology is a welcome addition and illustrates many of the advantages as well as the difficulties of doing pediatric drug trials.

Why are so few good quality randomized clinical trials of newer AEDs being done in children? Historically, new AEDs were studied primarily in adult males. Recent FDA …