More Efficient Compliance with European Medicines Agency and Food and Drug Administration Regulations for Pediatric Oncology Drug Development: Problems and Solutions.

The morbidity and mortality toll of pediatric cancer affects the public health of children worldwide, but despite the gains in the fight against cancer, more progress needs to take place against this disease, which is a leading cause of death and chronic disability in children. In response, leading regulatory authorities in the developed world have been ratcheting up their efforts to induce the private sector to expand their research and development focus during drug development for adult cancers to include children. In mid-May 2016, the Center for the Study of Drug Development at Tufts University held a roundtable workshop on pediatric oncology to explore how companies could maximize the efficiency of pediatric assessment of adult cancer indications while minimizing resource expenditures to comply with regulatory requirements under the European Medicines Agency and the U.S. Food and Drug Administration. Although worldwide a child is diagnosed with cancer every 3 minutes, pediatric cancer is a rare disease, and trial participants are hard to come by. Thus, the market hardly sustains research and development expenses, advances in pharmacogenomics are not reaching down the age scale, and even in the public sector, basic research funding for pediatric cancer pales in comparison to the amount spent on cancer overall. The goal of the roundtable was to acknowledge these problems, and more importantly, to raise the level of awareness of potential solutions, including: more efficient use of the data hierarchy of informative events in clinical trials; new innovative clinical trial platforms for rapid assessment of new drugs in children; new developments in formulation technology; and optimization of speed in pediatric drug development through a multi-stakeholder network collaboration separate from the adult development plan.