In a recent Journal of Clinical Oncology article, members from the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) expressed the need for trial sponsors to coordinate their submissions of initial pediatric study plans (iPSP) and pediatric investigation plans (PIP) with regulatory authorities in order to facilitate pediatric cancer drug development.

The EMA has required PIP before granting market authorization since the European Pediatric Regulation (EC 1901/2006) took effect in 2007, but the FDA requirement for iPSP is new this year.

As a result of the Research Acceleration for Cure and Equity (RACE) for Children Act, which is part of the FDA Reauthorization Act of 2017, sponsors submitting a new drug application or biologics licensing application to the FDA on or after August 18, 2020, must include an iPSP for a new cancer drug intended for adults and directed at a molecular target that the FDA determines to be “substantially” relevant to the growth or progression of a pediatric cancer.

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The desire among agencies for coordinated submissions stems from the reality that cancer drug development is an “international enterprise” and that the numerous compounds in development may outpace the number of children who can enroll in biomarker-directed clinical trials.

The authors acknowledged that while the review period timelines for iPSP and PIP differ between the FDA and EMA, there may be the opportunity for discussion between and review by both regulatory agencies. Without coordinated submissions, the review of iPSP and PIP by regulatory authorities may be staggered and not allow authorities the opportunity to discuss discordant views, which can ultimately lead to study delays.

“The different timelines and procedures of regulatory agencies authorized to affect regional pieces of legislation should not pose a barrier for developers to prospectively discuss and design global pediatric development plans,” the authors wrote.


Reaman G, Karres D, Ligas F, et al. Accelerating the global development of pediatric cancer drugs: A call to coordinate the submissions of pediatric investigation plans and pediatric study plans to the European Medicines Agency and US Food and Drug Administration. J Clin Oncol. Published online September 18, 2020. doi:10.1200/JCO.20.02152