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成人用药数据外推至儿科人群的技术要求及审评考虑
成人用药数据外推至儿科人群的技术要求及审评考虑
孙艳喆,耿 莹,赵德恒,杨志敏

国家食品药品监督管理总局药品审评中心,北京 100038
Guideline on extrapolation of data from adults to pediatric  population and relevant evaluation considerations
(Center for Drug Evaluation, China Food and Drug Administration, Beijing 100038, China)

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起始页:2154

摘要:[摘要] 儿童药短缺及其带来的儿科人群临床超说明书用药现象是困扰全球的严峻问题。鉴于儿科人群药物临床试验难以开展,将成人数据外推至儿科人群的方法,可以最大化利用现有数据、减少不必要的儿科试验,从而加速儿童药的研发,是保障儿童药临床需求、并用科学的方法保证其安全性、有效性的最有效措施之一。本文分析了我国的现状和近年来鼓励儿童药研发的政策背景,介绍了CFDA最新发布的《成人用药数据外推至儿科人群的技术指导原则》、中国数据外推的模式和几种常见的应用数据外推的情况;并从药品审评角度提出了建议优先开展外推的儿童药种类、及对其审评资料的考虑,以期促进和引导我国数据外推的开展。

关键词:[关键词] 数据外推; 儿童用药品; 儿科人群; 超说明书; 指导原则; 评价

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Abstract:[Abstract] Pediatric drug shortage and the phenomenon of off-label use caused by it are serious problems disturbing the whole world. Considering the difficulty of conducting clinical trials in pediatric population, extrapolation of data from adults to pediatric population is one of the most effective measures to meet clinical needs for pediatric drugs and ensure drugs' efficacy and safety in a scientific way simultaneously, since this approach could maximally use existing data and reduce unnecessary pediatric trials, thus accelerating pediatric drug development. We analyzed the current situation and the policy background about encouraging pediatric drug development in recent years. Latest guideline about extrapolation of date from adults to pediatric population in China published by CFDA, modes of extrapolation, and several circumstances using data extrapolation in China were introduced. Suggestions on the category of pediatric drugs to be extrapolated as priority, along with the considerations about data needed for evaluation of these drugs were discussed, hoping to improve and guide the development of data extrapolation in China.

Key words:[Key words] data extrapolation; pediatric drug; pediatric population; off-label; guideline; evaluation

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