Combating childhood cancer: Paediatric patients living with Neuroblastoma - Regulatory ramifications and roadblocks

Abstract

This paper relates to Neuroblastoma (NBL), a rare, solid cancer affecting children and aims to describe regulatory obligations to adhere to during development, marketing authorisation application (MAA) and post-authorisation stage. The focus is on European Union (EU) paediatric legislation, although essential US Food and Drug Administration (FDA) elements are briefly outlined. Practical regulatory aspects and reporting requirements, players in the therapeutic area as well as clinical management are described. The feasibility and limitations of future harmonised clinical development are pointed out.