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.