DEVELOPPEMENT DES NOUVEAUX AGENTS ANTICANCEREUX CHEZ L'ENFANT. METHODOLOGIE, DIFFICULTES ET STRATEGIE

Survival in children with cancer has improved dramatically over the last 20 years. Although cure is achieved in two-thirds of cases overall, the prognosis remains grim in some malignancies. New drugs are needed to improve cure rates and quality of life in children with cancer. New drugs for children are first subjeected to Phase I trials, of which the methodological, ethical, and legal aspects are discussed herein. In Europe, the main problem is obtaining new drugs from the pharmaceutical companies that design them. The conduct of preclinical studies evaluating the efficacy of new drugs on models of childhood malignancies and the implementation of regulations similar to the American Orphan Drug Act would facilitate the early clinical development of potentially effective anticancer agents for children.