Still orphans

Approximately 40 years ago, in the wake of thalidomide-induced phocomelia, Dr. Harry Shirkey1 labeled children as therapeutic orphans, raising awareness of the paucity of medication safety and efficacy data in children. In the interim, there have been some gains for children as the result of workshops focusing on pediatric drug development,2,3 programs such as the Pediatric Pharmacology Research Unit Network, and laws such as the Best Pharmaceuticals for Children Act (BPCA) of 2002 that grants an additional 6 months of marketing exclusivity for new medications in which pediatric studies are performed and provides for mechanisms that fund the study of off-patent medications used in children. Since the legislation’s implementation, >100 products have been granted exclusivity, including gabapentin, lamotrigine (LTG), oxcarbazepine, and levetiracetam. Of note, approximately a third of the products studied required relabeling because of important differences in dosage, safety, or efficacy in children.4,5 Despite advances, the picture has only minimally improved for children <2 years of age. Although they represent a significant proportion of patients with new onset epilepsy, none of the antiepileptic medications approved since 1993, including those granted …