G. Vassal et al., DEVELOPMENT OF NEW ANTICANCER AGENTS IN C HILDREN - METHODOLOGY, DIFFICULTIES, AND STRATEGIES, Annales de pediatrie, 41(8), 1994, pp. 477-484
Survival in children with cancer has improved dramatically over the la
st 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. Ne
w drugs for children are first subjected to Phase I trials, of which t
he methodological, ethical, and legal aspects are discussed herein. In
Europe, the main problem is obtaining new drugs from the pharmaceutic
al companies that design them. The conduct of preclinical studies eval
uating the efficacy of new drugs on models of childhood malignancies a
nd the implementation of regulations similar to the American Orphan Dr
ug Act would facilitate the early clinical development of potentially
effective anticancer agents for children.