Nouveaux médicaments anticancéreux pour les enfants et les adolescents : où en sommes-nous en Europe ?

Cancer is the first cause of death by disease in children and adolescents. Innovative treatments are urgently needed to increase cure as well as quality of cure of survivors. Since 2007, the European Pediatric Medicine Regulation incites pharmaceutical companies to develop their drugs in children when they meet a pediatric need. They get a reward when they complete a pediatric investigation plan (PIP). They get a waiver if their drug does not meet a pediatric need. Five years later, the landscape of pediatric oncology drug development has started to change but we are still far from the goal: waivers are issued for drugs which are developed for adult malignancies that do not occur in children while they have a mechanism of action which is relevant for a pediatric malignancy; difficult to run PIP are requested for diseases occurring extremely rarely in children such as thyroid cancer or metastatic melanoma. Changes are needed in the implementation of the Pediatric Regulation. PIP should be based on the drug mechanism of action rather than the adult cancer indication. Cooperation with all stakeholders from pediatric oncology community, pharmaceutical industry, regulatory agencies, parents and patients associations as well as national and European agencies should be facilitated to run a strategy for new oncology drug development. New types of partnerships should be developed to meet the needs of children and adolescents with cancer.