美国儿童药品上市审批情况分析及其对我国儿童用药保障工作的启示  

Analysis of the Marketing Approval of Pediatric Drugs in the United States and Its Enlightenment to Pediatric Drug Use Guarantee in China

作　　者：王雅君 韩容[1] 岳志华[3] 许馨文 赵志刚[1] WANG Yajun;HAN Rong;YUE Zhihua;XU Xinwen;ZHAO Zhigang(Dept.of Pharmacy,Beijing Tiantan Hospital,Capital Medical University,Beijing 100070,China;College of Pharmacy,Capital Medical University,Beijing 100069,China;Dept.of Chemical Drug,Chinese Pharmacopoeia Commission,Beijing 100061,China;Dept.of Secretariat,Safe Medication for Children Branch of China Medical News and Information Association,Beijing 101200,China)

机构地区：[1]首都医科大学附属北京天坛医院药学部,北京100070 [2]首都医科大学药学院,北京100069 [3]国家药典委员会化药处,北京100061 [4]中国医药新闻信息协会儿童安全用药分会秘书处,北京101200

出　　处：《中国药房》2021年第2期133-138,共6页China Pharmacy

基　　金：国家科技重大专项项目(No.2018ZX-09721003)。

摘　　要：目的:为完善我国儿童药品品种、保障儿童用药安全提供借鉴。方法:总结美国在儿童药品上市审批方面的政策法规历史沿革及相关政策实施效果;结合我国儿童药品发展现状,提出保障我国儿童用药可及性和安全性的相关建议。结果与结论:美国自1994年起就出台了一系列的政策法规以鼓励发展儿童药品,目前其儿童药品上市审批主要依据《最佳儿童药品法》(BPCA)和《儿科研究平等法》(PREA)两部法律。1998-2019年,美国药品说明书儿童用药信息的补充数量总体呈现波动性增长;截至2020年4月,已经有854种药品进行了儿科说明书的修改,其中792种药品进行了上市后儿科临床研究,其儿科说明书信息不全等问题得到了很大改善。目前,我国对儿童药品制定的政策主要包括鼓励研发创新、优先审评审批,以及加强研发技术指导等,虽取得了一定的成绩,但仍存在相关政策法规不完善、开展儿童药品临床试验困难较大等问题。建议我国借鉴美国儿童药品相关政策法规、儿科研究及儿童药品目录,建立和完善适合我国国情的儿童药品制度和目录,提高儿童药品的安全性、有效性和可及性。OBJECTIVE:To provide suggestions for improving the variety of pediatric drugs and ensuring the safety of pediatric drug use in China.METHODS:The historical evolution of laws and regulations on the marketing approval of pediatric drugs in the United States and the implementation results of relevant policies were summarized.Combined with the current situation of the development of pediatric drugs in China,some suggestions were put forward to ensure the accessibility and safety of pediatric drug use in China.RESULTS&CONCLUSIONS:Since 1994,the United States had issued a series of laws and regulations to encourage the development of pediatric drugs.At present,the marketing approval of pediatric drugs were mainly based on the two laws of Best Pediatric Drug Act(BPCA)and Pediatric Research Equality Act(PREA).From 1998 to 2019,the amount of supplements of pediatric drug information in drug instructions of the United States showed a fluctuating growth.As of April 2020,854 kinds of drugs had been modified in pediatric instructions,792 of which had been carried out post marketing pediatric clinical research,and the problem of incomplete pediatric instructions had also been greatly improved.At present,China’s policies on pediatric drugs mainly included encouraging R&D innovation,giving priority to review and approval,and strengthening R&D technical guidance.Although certain achievements had been made,there were still some problems,such as imperfect policies and regulations,and great difficulties carrying out pediatric drug clinical trials.It is suggested that our country should draw lessons from the American regulations on pediatric drugs,pediatric research and the catalogue of pediatric drugs,and establish a system and catalogue of pediatric drug use suitable for China’s national conditions,so as to improve the effectiveness,safety and accessibilityof pediatric drugs.

关 键 词：儿童药品 上市审批 政策法规 美国 启示 

分 类 号：R95[医药卫生—药学] R985[医药卫生—药品]