Improving predictability to promote drug development for pediatric cancers in Japan: From the perspective of pharmaceutical companies

Abstract

Pediatric cancers are the leading cause of pediatric death and a group of rare diseases comprising more than 50 distinct entities, with more than 2,000 new cases diagnosed annually. In Japan, the number of patients has been declining annually owing to the rapidly declining birth rate. Because pediatric cancers differ substantially from adult cancers in tumor biology, drugs developed for adult oncology are not necessarily applicable to pediatric indications. These characteristics place pediatric cancer drug development at a disadvantage in pharmaceutical companies’ investment decision-making, contributing to the stagnation of domestic development activities.

This article reviews the current status of pediatric cancer drug development in Japan and internationally; the structural challenges faced by pharmaceutical companies, incentive systems centered in the United States, and recent trends in the entry of emerging biopharmaceutical companies into the field. We discussed the importance of securing “predictability” as a key factor for promoting drug development in Japan examined policy issues, such as post-marketing surveillance, use of patient registries, the framework for investigator-initiated clinical trials, and government-led ecosystem building, and propose directions for future policy development.