The Center for Pediatric Clinical Development
The Center for Pediatric Clinical Development

In the US, legislation such as the Better Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have helped to increase development of safe and effective pharmaceuticals for pediatrics. While these have been effective for many diseases, they have had virtually no impact on the development of more treatments for childhood cancer for two key reasons:

  • The requirement to study drugs in children that are being developed for adults has been based on indication, and if that is not relevant for children then trials have not been required. However, children’s cancers often originate in different parts of the body than for adults, despite having a similar molecular basis.
  • Despite being the leading cause of disease-related death in children in the USA, pediatric cancer is technically a rare disease and thus eligible for orphan designation and exempt from PREA obligations.

Staggering statistics for pediatric oncology in tandem with research in personalized medicine have given rise for an amendment to PREA which we anticipate will result in more drugs in the pediatric oncology pipeline. Incorporated as Title V of the FDA Reauthorization Act (FDARA), enacted August 18, 2017, the Research to Accelerate Cure and Equity (RACE) Act will dramatically impact the landscape of pediatric oncology research.

Precision or personalized medicine has for years transformed adult therapies by targeting cancer based on molecular basis rather than diagnosis. Pediatric therapies as such have lagged behind in part due to waivers enacted through PREA because it went by clinical indication (ex. Breast, lung, prostate) rather than molecular basis (ex. BRAF, FLT3, NTRK). The RACE for Children Act updates PREA and intends to ensure pediatric evaluations are conducted based on mechanism of action so that novel therapies are developed for pediatric oncology patients.

This legislation requires that starting August 18, 2020, applicants subject to PREA submit an initial pediatric study plan (iPSP) prior to the submission of an original NDA (New Drug Application) or BLA (Biologics Licensing Application) for an oncology therapy. Specifically, if the new NDA/BLA is intended for treatment of an adult cancer and directed at a molecular target the FDA determines to be substantially relevant to the growth or progression of a pediatric cancer, reports on the molecularly targeted pediatric cancer investigation must be submitted with the marketing application.

RACE will increase the number of pediatric oncology studies, yet the number of patients will not increase, necessitating a global effort and innovation to run pediatric trials. PRA has positioned itself at the forefront of this legislation.

To proactively address the inevitable challenges created by RACE for sponsors and the industry, the CPCD put together a cross-functional steering committee, RACE-R(evolution), with members from Therapeutic Expertise, Business Development, Marketing, Real World Solutions, Regulatory, Project Delivery.

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