Keywords: Oncology, pediatric cancer, platform trial
The Best Pharmaceuticals for Children Act (BPCA) in 2002 and Pediatric Research Equity Act (PREA) were established in 2003. Since then, there have been a steady increase in the number of pediatric clinical development programs initiated, completed and had labeling modifications. Recently, addendum to ICH E11: Clinical Investigation of Medicinal Products in the Pediatric Population final version was adopted in 2017. The addendum provides additional clarification (e.g., extrapolation) and current regulatory perspectives on topics in pediatric drug development. The EMA and ACCELERATE hosted several workshops on oncology pediatric strategies since early 2017. Moreover, the FDA Reauthorization Act (FDARA) enacted on August 18, 2017 requires evaluation of new molecularly targeted drugs and biologics “intended for the treatment of adult cancers and directed at a molecular target substantially relevant to the growth or progression of a pediatric cancer” while eliminating orphan exemption for pediatric studies for cancer drugs directed at relevant molecular targets. All these new regulations put urgency on the stakeholders in developing oncology drug treatments to strategizing pediatric programs early and efficiently. National Cancer Institute (NCI) Children’s Oncology Group (COG) has initiated a nationwide cancer treatment trial – pediatric MATCH. It is a precision medicine investigation in which pediatric cancer patients of 1-21 years of age with solid tumors that are not responding to treatment are assigned to an experimental treatment based on the genetic changes found in their tumors rather than on their type of cancer or cancer site. Treatments being tested in this study are investigational pediatric drugs donated by pharmaceutical companies that have partnered with NCI. This presentation will discuss the challenges we face when attempting to adopt the same trial concept in registrational pediatric trials mainly deigned to fulfill regulatory requirements.