A Review of the Experience with Pediatric Written Requests Issued for
Oncology Drug Products
Background Pediatric anticancer drug development has numerous
challenges. 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 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 information to pediatric.
The requirements for the pediatric studies can vary greatly. Procedure
In this study, we searched WRs that were issued by the FDA since 2001.
We found 42 requests for pediatrics in oncology drugs and biologics.
Results Studies included in 25 of the WRs have concluded, 18 have been
given exclusivity, and 4 drugs have been approved for use in pediatric
populations. The current status of the WRs are presented from
regulatory, study design, dosing, formulation, analysis plan, evidential
standard of efficacy and safety. Conclusions This would serve the
purposes to study what has been requested over the years and what have
been completed in response to the requirements. We consider this to be
the anchor of pediatric cancer development for current stage and can
potentially provide insight on how pediatric cancer drug development
would change for the future years.