Background
Pediatric anticancer drug development has numerous challenges. The
Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for
Children Act (BPCA) were passed to address the deficiency in pediatric
drug development in general. Until recently, the requirement for
pediatric evaluation of most oncology products developed for adult
cancers has been waived, because children typically do not have the same
type of cancers which occur commonly in adults or the indication or drug
had been granted orphan designation. PREA therefore had no impact.
Pediatric studies for labeling updates are largely done through BPCA by
a Written Request (WR), issued by FDA. Because pediatric and adult
populations do not share the same biology, natural history, or disease
progression, there are limited opportunities to extrapolate adult
efficacy and safety to the pediatric population. The requirements for
the pediatric studies have varied greatly over time.