对儿科药物开发的非临床安全性评价的考虑  

Non-clinical safety considerations for the development of pediatric drugs

作　　者：黄芳华[1] 王庆利[1] Jim Ridings 王英 张云 

机构地区：[1]国家食品药品监督管理总局药品审评中心,北京100038 [2]Glaxo Smith Kline(GSK),Toxicology and Biometabolism [3]葛兰素史克(上海)医药研发有限公司药物安全评价部,上海201203

出　　处：《中国新药杂志》2016年第18期2052-2058,共7页Chinese Journal of New Drugs

摘　　要：儿科药物研发是目前药物研发的一个热点,其中非临床安全性评价是儿科药物研发的一个必要和独特的部分。基于化合物类型/适应证(包括"只是儿科/先是儿科"用药)和儿科人群相关的特性,需要及时地制定一个适当的儿科药物非临床研发策略和计划,来支持儿科临床试验和新药上市申请。儿童用药的安全问题是独特的,且与器官系统的发育密切相关,需要根据一个透彻的"缺口"分析,制定一个总的非临床儿科药物研发计划。本文介绍了国外药品监管机构对儿科药物研发的非临床安全性评价指导原则、儿科药物研发过程中的科学咨询、儿科临床研发计划,以及对幼龄动物非临床安全性评价的策略和要求,包括药物安全性在成年和儿科患者中的差异及原因、何种情况下要求进行幼龄动物试验及其时间安排等,以期为国内研发者提供参考。Currently,pediatric drug development is a hot topic in China. Non-clinical safety evaluation is an essential and unique part of pediatric drug-development process. An appropriate non-clinical pediatric strategy and plan needs to be developed timely based on the compound class/indication（ including ＂pediatric-only/pediatric-first＂） and many pediatric population related features to support clinical trials and regulatory filing without any delays or gaps. Pediatric safety issues are unique and related to organ system development,and an overall nonclinical pediatric plan needs to be developed based on a thorough ＂gap ＂ analysis. In this paper,we briefly discussed the non-clinical safety evaluation guidelines for pediatric drug development issued by Drug Administration（ DA） outside of China,scientific consultation with DA during the pediatric drug development,Pediatric Investigation Plan（ PIP）/Pediatric Study Plan（ PSP）,strategy and requirement for conducting juvenile animal（ JA） toxicology studies,including the difference and reason of drug safety profile between adult and pediatric patients,and timing to conduct JA toxicology studies. We hope this article can serve a reference for the pharmaceutical RD professionals in China.

关 键 词：儿科药物研发 非临床安全性评价 非临床儿科药物研发计划 “缺口”分析 幼龄动物毒理学试验 

分 类 号：R95[医药卫生—药学] R99