Background
There are numerous challenges facing the field of pediatric oncology and hematology drug development. Two legislative initiatives, Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA), has been put forth to address the deficiency in pediatric drug development in general. Until recently, the requirement for pediatric evaluation of most oncology products for adult cancers was waived, because children typically do not have adult-type cancers (e.g. lung cancer) or because the indication or drug had been granted orphan designation. PREA therefore had no impact on pediatric anticancer drug development. Pediatric studies for labeling updates are largely done through BPCA by the fulfillment of a Written Request (WR), issued by the FDA. Because pediatric populations and adult populations do not share the same biology, natural history, or disease progression, there are limited opportunities to extrapolate adult information to pediatric. Therefore, the requirements for the pediatric studies can vary greatly according to the disease indications.