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A Review of the Experience with Pediatric Written Requests Issued for Oncology Drug Products
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  • Xi Meng,
  • Alemayehu Akalu,
  • Gregory Reaman,
  • Lian Ma,
  • Weishi Yuan,
  • Jingjing Ye
Xi Meng
University of Massachusetts Amherst School of Public Health and Health Sciences

Corresponding Author:[email protected]

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Alemayehu Akalu
US Food and Drug Administration
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Gregory Reaman
Children's National Health System
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Lian Ma
US Food and Drug Administration
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Weishi Yuan
US Food and Drug Administration
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Jingjing Ye
BeiGene US
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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.
20 May 2020Submitted to Pediatric Blood & Cancer
20 May 2020Assigned to Editor
20 May 2020Submission Checks Completed