成人用药数据外推至儿科人群的科学决策过程和法规依据研究进展分析  

Scientific decision process and regulation requirements based on the current guidelines of data extrapolation from adult to pediatric population

作　　者：张慧敏 刘霭明 朱文烽 余灵芝[4] 陈方亮 ZHANG Huimin;LIU Aiming;ZHU Wenfeng;YU Lingzhi;CHEN Fangliang(Enze Hospital of Enze Medical Center (Group),Taizhou 317000,Zhejiang,China;Shunde Hospital of Southern Medical University,Shunde 528300,Guangdong,China;Zhejiang Huahai Pharmaceutical Co.,Ltd,Linhai 317000,Zhejiang,China;Taizhou Hospital of Zhejiang Province,Taizhou 317000,Zhejiang,China)

机构地区：[1]恩泽医疗中心(集团)恩泽医院药剂科 [2]南方医科大学顺德医院药学部临床药学科 [3]浙江华海药业股份有限公司 [4]浙江省台州医院药剂科

出　　处：《中国临床药理学与治疗学》2019年第1期49-56,共8页Chinese Journal of Clinical Pharmacology and Therapeutics

基　　金：浙江省药学会资助课题基金(2017ZJYY29);台州市路桥区科技局基金(2018A23001)

摘　　要：目的:通过分析现行基于成人用药数据外推至儿科人群的科学决策过程和法规依据,及数据外推的优势和局限性,为儿科药物开发提供一些思路和指导。方法:检索美国药品食品管理局(USFDA)、欧洲医药管理局(EMA)、国家药品食品监督管理局(CFDA)、人用药品注册技术要求国际协调会(ICH)相关网站,以及万方、CNKI、Pubmed等相关数据库,比较不同国家的成人用药数据外推至儿科人群的相关法规指南。结果:现行USFDA、EMA、CFDA以及ICH儿科数据外推指南基本上一致,但各自又有所区别,需对现行指南进行完善;现行数据外推指南对某些关键性概念及算法描述不清。结论:采用数据外推,可以加快儿科用药研发,增加儿科临床用药数量,即将推出的ICH E11(R1)儿科临床研究补充指南,将提供统一的数据外推指南,更好地指导儿科药品的开发。AIM:To provide a general overview of the regulatory guidelines on the extrapolation from adult and other data to pediatric population,and to provide some practical advices for following these guidelines.METHODS:Literatures about the topic of"data extrapolation/and pediatric"from website of CFDA,EMA,USFDA,ICH and databases such as Wanfang,CNKI and Pubmed were searched,then the current guidelines on this topic were analyzed and compared,and the strategy for this topic was discussed.RESULTS:The current guidelines of EMA,U.S.FDA and ICH maintained consistent in critical principles.But the difference still required further improvement.The current data extrapolation guidelines laced maneuverability,and the description for some critical concepts and algorithm was not clear.CONCLUSION:Extrapolating efficacy from adult data or other data to the pediatric population can streamline pediatric drug development and help to increase the number of approvals drug for pediatric use,and the ICH E11(R1)that may replace the current guidelines will provide a better way for the pharmaceutical manufacturers to develop drug for pediatric use.

关 键 词：数据外推 超说明书 儿科用药 建模与模拟 

分 类 号：R985[医药卫生—药品] R952[医药卫生—药学]