Accès aux essais précoces de nouveaux médicaments anticancéreux pour les enfants et les adolescents en France

Cancer is the leading cause of disease-related death in children and adolescents.Providing access to innovative therapies is a key strategic goal in pediatric oncology hematologyin Europe.Objective. - To study participation of children and adolescents to early drug trials of newoncology drugs in France.Method. - Data from all phase 1, phase 1/2 and phase 2 trials open to accrual in the SFCEcenters from 2010 to 2013 have been collected and analyzed.Results. - Thirty-seven early drug trials recruited 607 patients (487 with a malignant solidtumors [80%]; 120 with a hematological malignancies [20%]). There were 15 phase 1, 5 phase1/2 and 17 phase 2 trials evaluating 33 new anticancer drugs (66% were targeted therapies) andone new formulation. Sixty-eight percent were international trials run within the ITCC Europeannetwork. Sixty-two percent were industry-sponsored trials. In 2013, there were 195 inclusionsin 30 trials, representing +77% as compared to 2010. Ninety percent of inclusions were in the 12centres of the SFCE Pharmacology Group. Inclusions in France accounted for 50% of all inclusionsin ITCC over a period of 10 years.Conclusion. - The field of development of innovative therapies for children and adolescentswith cancer is European and France is attractive and leader in Europe. A 3-fold increase in thenumber of trials and a nearly doubling of the number of yearly inclusions over 4 years clearlyillustrate a dynamic activity and a real momentum. However, this lags beyond the real medicalneeds and parents expectations for children and adolescents dying of cancer each year. TheNational Cancer Plan 3 goal is to more than double access to innovative therapies.