The Role of Regulatory Agencies in Pediatric Cancer Drug Development

Abstract

AbstractAlthough most of the legislation providing authority to the United States (US) Food and Drug Administration (FDA) to regulate the investigation and marketing of new drugs arose as a result of catastrophic events primarily affecting children, focused inclusion of children in clinical research activities investigating new drugs emerged much later in US regulations after it was finally recognized that they were being detrimentally affected by their exclusion. Specific legislative initiatives to promote the assessment of effectiveness and safety of drugs in children originated in the United States as a system of incentives and mandates that were followed nearly a decade later in the European Union (EU) by a single initiative that required and incentivized manufacturers to study drugs in children when there was a likelihood of pediatric use. Over time, recognition of the need for specific pediatric provisions has also emerged in Canada, Japan, Australia, and other parts of the world. Recent pediatric legislative initiatives to address the paradigm shift brought about by the advent of precision oncology have dramatically altered the global regulatory environment for pediatric cancer drug development and are reviewed here. Leveraging these recent changes offers the prospect for accelerating the development of novel agents for pediatric patients with cancer.KeywordsUS Food and Drug Administration (FDA)European Medicines Agency (EMA)Best Pharmaceuticals for Children Act (BPCA)Pediatric Research Equity Act (PREA)Paediatric Regulation 1901/2006Paediatric Investigation Plan (PIP)Pediatric Study Plan (PSP)Common Commentary