New drugs for children with cancer: What needs to change in Europe

Precision cancer medicine and immunotherapy are delivering very promising results for the treatment of adult cancers and more than 1000 new oncology compounds are currently developed par the Pharmaceutical industry. It is crucial that children and adolescents are not left apart since cancer remains the leading cause of death by disease beyond 1 year of age. The European Pediatric Medicine Regulation that was launched in 2007 clearly changed the landscape for evaluation of medicines in children, but it is far for having addressed yet adequately the urgent therapeutic needs in pediatric oncology. In 2017, there is an opportunity to change the regulation and its implementation. All stakeholders (pediatric oncologists, researchers, parents, regulators, industry) joined forces through the Accelerate platform and the Unite2Cure parents organization alliance to analyze, together, the limitations of the current regulation and to propose solutions: suppress waivers that are both scientifically and medically unjustified; set up an efficient prioritization system to choose the potentially best new oncology drugs to develop in children; break the 18 years dogma; install an efficient and adapted system of rewards and sanctions. Several initiatives are currently being developed in France and in Europe to mobilize members of the Parliament and Member-states in order to improve the regulatory environment and to better incentivize industrial investment in research and development of innovations for children and adolescents with cancer. It is crucial to act now in order to cure them more and better.