Many prescription drugs approved for adult use lack pediatric labeling information, resulting in their off‐label use in children. Drug regulatory authorities have developed legal and regulatory frameworks to promote pediatric drug development. However, the current state of pediatric indication approval and quality of efficacy evidence, which forms the basis of regulatory approvals, is unknown. Here, we analyzed novel therapeutics approved in Japan, the United States, and the European Union during 2005–2014 to investigate the timing and frequency of pediatric indication approval, and characterized the design of pediatric studies supporting regulatory approval. We found that the United States and European Union experienced a higher frequency of supplemental indication approval in pediatrics based on better‐designed studies than Japan. The speed and efficiency of pediatric drug development will improve by coordinating pediatric studies on an international basis. The results also implied the necessity for a robust system of postmarketing monitoring of pediatric efficacy and safety.

中文翻译：

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提交监管机构的小儿药物批准的时间和临床证据：日本，美国和欧盟之间的国际比较。

许多批准用于成人的处方药缺乏儿科标签信息，导致其在儿童中的标签外使用。药品监管机构已经制定了法律和监管框架，以促进儿科药物的开发。然而，目前尚不知道小儿适应症批准的现状和疗效证据的质量，这是监管批准的基础。在这里，我们分析了2005-2014年在日本，美国和欧盟批准的新型疗法，以研究儿科适应症批准的时间和频率，并描述了支持监管批准的儿科研究设计。我们发现，基于更好设计的研究，与日本相比，美国和欧盟在儿科中获得辅助适应症批准的频率更高。通过在国际上协调儿科研究，将提高儿科药物开发的速度和效率。结果还暗示，有必要建立健全的售后监测儿科疗效和安全性的系统。