Regulatory and Scientific Support for Developing Innovative Medicines for Children with Cancer

European legislation (Regulation (EC) No 726/2004) determines that a new medicine, which „constitutes a significant therapeutic, scientific or technical innovation“, be centrally authorised for the European Union by the European Medicines Agency. For paediatric malignancies, scientific innovation may present as increased efficacy due to a novel mechanism of action, or as molecularly defined target population. Technical innovation may present as delivery system permitting better maintaining dose intensity. For developing medicines for children with cancer, the Agency has broad responsibilities subsequent to Regul…