Osako Ryohei, Matsumaru Naoki, Tsukamoto Katsura
Global Regulatory Science, Gifu Pharmaceutical University, 1-25-4 Daigakunishi, Gifu, 501-1196, Japan.
Ther Innov Regul Sci. 2025 Jan;59(1):142-149. doi: 10.1007/s43441-024-00714-6. Epub 2024 Oct 23.
The lack of label information for the pediatric population has been a global issue, leading to the introduction of several countermeasures by major health authorities. Despite various efforts by Japanese health authorities, some drugs are approved only for adults in Japan, while the United States (US) label includes information on pediatric usage for the same drugs. This suggests a potential for pediatric-specific drug loss in Japan, where overall drug loss has recently become a major concern.
In this study, we compared the pediatric usage status between Japan and the US, focusing on the indications approved in both countries.
Of the 404 indications, 70 (17.3%) and 102 (25.2%) included pediatric usage in Japan and the US, respectively. The proportion of indications, including pediatric usage, was significantly higher in the US than in Japan (χ test, p < 0.001). Multivariate analysis of indications for pediatric usage in the US demonstrated that simultaneous development with adults (odds ratio (OR), 24.9; 95% confidence interval (CI), 6.79-91.1) and Japan-first development (OR, 31.5; 95% CI, 2.59-384) were significantly affecting the inclusion of pediatric usage in Japan.
Our results suggest that there was pediatric-specific drug loss in Japan compared to that in the US. The multivariate analysis demonstrated that US-first development and non-simultaneous development had a negative impact on the inclusion of pediatric usage in Japan; however, pediatric assessment request was not a significant factor. Further frameworks to promote pediatric drug development should be introduced in Japan to address pediatric-specific drug loss issues.
儿科人群缺乏药品标签信息一直是一个全球性问题，促使各大卫生当局采取了多项应对措施。尽管日本卫生当局做出了各种努力，但在日本，一些药物仅被批准用于成人，而美国的药品标签则包含了这些药物在儿科使用的信息。这表明在日本存在儿科专用药物流失的可能性，而整体药物流失最近已成为一个主要问题。
在本研究中，我们比较了日本和美国的儿科用药状况，重点关注两国都批准的适应症。
在404种适应症中，日本和美国分别有70种（17.3%）和102种（25.2%）包含儿科用药信息。美国包含儿科用药的适应症比例显著高于日本（χ检验，p < 0.001）。对美国儿科用药适应症的多变量分析表明，与成人同步研发（优势比（OR），24.9；95%置信区间（CI），6.79 - 91.1）和日本率先研发（OR，31.5；95% CI，2.59 - 384）对日本儿科用药信息的纳入有显著影响。
我们的结果表明，与美国相比，日本存在儿科专用药物流失的情况。多变量分析表明，美国率先研发和非同步研发对日本儿科用药信息的纳入有负面影响；然而，儿科评估要求并非一个重要因素。日本应引入进一步的框架来促进儿科药物研发，以解决儿科专用药物流失问题。
