Paediatric Drug Development in Japan: Current Status and Future Challenges.

IF 2 4区 医学 Q4 MEDICAL INFORMATICS
Rieko Inagaki, Mamoru Narukawa
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Abstract

Introduction: Until around 2000, the number of medicinal products labelled for paediatric use was limited worldwide. Regulatory measures to promote paediatric drug development in the US and Europe and the establishment of an international guideline (ICH-E11) have led to an increase in the number of paediatric labels. In Japan, efforts have been made to promote the development of paediatric drugs. This study was aimed to examine whether these supportive efforts are successful in Japan.

Methods: This study examined the number of new drugs approved for paediatric indications in Japan from 2006 to 2023, as well as the clinical data package, that is, characteristics of the approved paediatric drugs and paediatric clinical trials, and the percentage of extrapolation of adult data, in the most recent 9-year period.

Results: The number of paediatric drug approvals showed an increasing trend between 2006 and 2023 with some fluctuations. The proportion of drugs indicated for paediatric patients to the total number of approved drugs was about 30% until 2022, but increased to 48% in 2023. During the period from 2015 to 2023, simultaneous development in adults and children accounted for 59% (159/269) of paediatric development, but the complete extrapolation of adult data to paediatric populations has not been widely utilized (11.2%, 30/269).

Conclusions: The number of paediatric drug approvals has shown an upward trend, suggesting that measures to promote the development of paediatric drugs may have been exerting a favourable effect in Japan. However, there is still a limited number of drugs that have additional indications for paediatric use. Appropriate development strategies, such as the extrapolation of adult data to paediatric populations, should be considered if scientifically justified.

日本的儿科药物开发:现状与未来挑战》。
导言:直到 2000 年左右,全世界贴有儿科用药标签的药品数量还很有限。美国和欧洲采取了促进儿科药物开发的监管措施,并制定了国际指南(ICH-E11),从而使儿科标签的数量有所增加。日本也在努力促进儿科药物的开发。本研究旨在探讨这些支持性努力在日本是否成功:本研究调查了 2006 年至 2023 年日本批准的儿科适应症新药数量,以及最近 9 年的临床数据包,即批准的儿科药物和儿科临床试验的特点,以及外推成人数据的百分比:2006年至2023年期间,儿科药物批准数量呈上升趋势,但也有波动。到 2022 年,儿科患者用药占批准药物总数的比例约为 30%,但到 2023 年增至 48%。在2015年至2023年期间,成人和儿童同时开发的药物占儿科开发药物的59%(159/269),但将成人数据完全外推至儿科人群的做法并未得到广泛应用(11.2%,30/269):儿科药物的批准数量呈上升趋势,这表明促进儿科药物开发的措施可能在日本产生了有利影响。然而,拥有更多儿科适应症的药物数量仍然有限。如果科学合理,应考虑适当的开发战略,如将成人数据外推至儿科人群。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Therapeutic innovation & regulatory science
Therapeutic innovation & regulatory science MEDICAL INFORMATICS-PHARMACOLOGY & PHARMACY
CiteScore
3.40
自引率
13.30%
发文量
127
期刊介绍: Therapeutic Innovation & Regulatory Science (TIRS) is the official scientific journal of DIA that strives to advance medical product discovery, development, regulation, and use through the publication of peer-reviewed original and review articles, commentaries, and letters to the editor across the spectrum of converting biomedical science into practical solutions to advance human health. The focus areas of the journal are as follows: Biostatistics Clinical Trials Product Development and Innovation Global Perspectives Policy Regulatory Science Product Safety Special Populations
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