Challenges in the international clinical trials for pediatric patients with cancer

Abstract

To improve outcomes in childhood cancer, it is important to evaluate the safety and efficacy of treatments through clinical trials. The Japan Children’s Cancer Group has been conducting late-phase clinical trials to establish standards of care. However, compared to Europe and the United States, which have requirements for pediatric drug development, Japan lacks an adequate framework for early-phase clinical trials and pediatric drug development, leading to the delay of drug approval. Participation in international clinical trials could be an option to avoid this delay, although there are some challenges. This article shows the results of the retrospective survey about the implementation of academic international clinical trials in childhood cancer patients and describes the preparation for participation in the Interfant-21 trial. In addition, by referencing clinical research support systems in Europe and the United States, frameworks to facilitate participation in international trials have been discussed.