2025 Volume 15 Issue 1 Pages 7-27
The development of pediatric drugs has long been recognized as a challenging endeavor internationally, primarily due to the small market size and the difficulties in conducting clinical trials. Additionally, there has been growing concern in Japan regarding the increasing drug loss of pediatric drugs in recent years. To address these challenges, the Ministry of Health, Labour and Welfare (MHLW) has been discussing on the direction of measures to promote such development, through a panel convened since 2023. Efforts have been made to enhance the regulatory environment, including the issuance of relevant notifications, to improve the current situation of pediatric drug development. The Clinical Evaluation Expert Committee of the Drug Evaluation Committee in Japan Pharmaceutical Manufacturers Association (JPMA) conducted an investigation on pediatric drugs approved from April 2018 to March 2023 in Japan aiming to help the planning and formulation of Pediatric Drug Development strategies and market authorization filings in Japan. As a result, the number of approved pediatric drugs showed an increasing trend during the investigation period. However, when compared to approval timing of the drug in Europe and the United States (US), approximately 30-40％ of approvals experienced a lag of over one year. The composition of clinical data-packages and approval lag between Japan and overseas was also compared. In clinical data-packages consisting of domestic clinical trials, 54.8％ were approved only in Japan, and 41.9％ had approval lag more than one year after approval in Europe or the US, indicating a polarization. In contrast, in clinical data-packages consisting of Multi Regional Clinical Trials (MRCTs), 69.6％ had an approval lag less than one year. Based on this investigation, it is considered effective to participate in MRCTs in order to obtain approval for pediatric drugs in Japan without delay from the Europe and the US. Furthermore, it is deemed important to maximize the use of existing data and construct flexible data-packages for approval when formulating development plans in Japan.
