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 Regulation (EC) 1901/2006 („Paediatric Regulation“) since 26 January 2007. For all new and many authorised medicines, a paediatric investigation plan (PIP) has to be submitted early to the Agency's Paediatric Committee, to ensure that paediatric data with high ethical and scientific quality are generated where they are required to address unmet paediatric needs. Indeed, from PIPs for 51 anti-cancer active substances submitted until December 2009, 40 concerned innovative or targeted medicines, including 4 cell-based medicines.
The Agency scientifically supports developing innovative medicines for children. For example, scientific advice is free of charge for paediatric medicines, including innovative ones, or qualifying biomarkers. Such advice is recommendable to companies and to academic groups, to increase the chances of success at approval time. An Innovation Task Force at the Agency offers multidisciplinary meetings to discuss developing a medicine.
Overall, the Agency ensures consistent approaches and sharing of information between its scientific committees.