A Review of the Experience with Pediatric Written Requests Issued for Oncology Drug Products [post]

Xi Meng, Alemayehu Akalu, Gregory Reaman, Lian Ma, Weishi Yuan, Jingjing Ye
2020 unpublished
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
more » ... 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. Abstract: 285 The number of Tables: 5 The number of Figures: 1 Short running title: Review of Pediatric Written Requests for Oncology Abstract: 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. 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 Of these WRs, 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. We have presented the current status of the WRs 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.
doi:10.22541/au.159007766.61484520 fatcat:oakude2adbgavhn5qhvpjf7lgy