Analysis of Pediatric Drug Approval Lag in Japan

Drugs should be made available to all patients in a timely manner regardless of whether they are adults or children. In contrast to the United States (US) and the European Union (EU), no laws mandating pediatric drug development have been established in Japan. The objective of this study was to examine the current status and characteristics of pediatric drug development in Japan using information on the approval lag for pediatric indications between Japan and the EU.

Drugs approved for pediatric indications between January 2007 and December 2018 in Japan were studied. The approval lag for pediatric indications between Japan and the EU was calculated and analyzed by approval time and the Anatomical Therapeutic Chemical (ATC) classification. Factors potentially affecting the approval lag, such as orphan drug status, company nationality, and clinical data package, were examined.

The median approval lag for 105 drugs with pediatric indications in both Japan and the EU was 1017 days (Japan was behind). The lag for ATC category B improved significantly after 2011, and for category L after 2015; the medians were less than half a year. The lag for drugs developed globally via multi-regional clinical trials was significantly shorter compared with drugs developed regionally.

Global clinical trials are the most effective means of shortening approval lag time in pediatric drug development. Global development is making rapid progress for many adult diseases, thereby creating an environment for proactive participation in global clinical trials even for pediatric drugs. For further improvement, more active drug development for pediatric indication is recommended in tandem with the US and the EU.