乙型肝炎创新药物临床试验设计的方法学考量  

Methodological considerations in the design of clinical trial for innovative hepatitis B drugs

作　　者：孔媛媛[1] 刘晓清[2] 尤红[3] 贾继东[3] 胡蓓[4] 周贤忠 姚晨[6] Kong Yuanyuan;Liu Xiaoqing;You Hong;Jia Jidong;Hu Pei;Chow Sheinchung;Yao Chen(Department of Clinical Epidemiology and EBM Unit,Beijing Friendship Hospital,Capital Medical University,Beijing 100050,China;Infectious Diseases Division,Peking Union Medical College Hospital,Beijing 100010,China;Liver Research Center,Beijing Friendship Hospital,Capital Medical University/Beijing Key Laboratory of Translational Medicine on Liver Cirrhosis/National Clinical Research Center for Digestive Diseases,Beijing 100050,China;Clinical Pharmacology Research Center,Peking Union Medical College Hospital,Beijing 100032,China;Department of Biostatistics&Bioinformatics,Duke University School of Medicine,Durham,North Carolina 27710,USA;Department of Biostitics,Peking University Frist Hospital,Peking University,Beijing,China)

机构地区：[1]首都医科大学附属北京友谊医院临床流行病学与循证医学研究室,100050 [2]北京协和医院感染内科,100010 [3]首都医科大学附属北京友谊医院肝病中心肝硬化转化医学北京市重点实验室/国家消化系统疾病临床医学研究中心,100050 [4]北京协和医院临床药理中心,100010 [5]杜克大学医学院生物统计与生物信息中心,达拉姆,美国北卡罗来纳州27710 [6]北京大学第一医院医学统计室,100034

出　　处：《中华肝脏病杂志》2020年第8期658-661,共4页Chinese Journal of Hepatology

基　　金：国家科技重大专项(2018ZX09201016);北京市科委项目(Z191100007619037);北京市医院管理中心科研项目(XXX0104)。

摘　　要：近年来以慢性乙型肝炎临床治愈为目标的新药研发进入活跃期。在针对乙型肝炎的新药临床试验中,采用设计新理念、新方法、新技术来评价创新药物的疗效,有望缩短候选新药的临床研究过程、降低新药开发成本。但是,无缝适应性设计、主方案设计等新型设计在乙型肝炎创新药物临床试验中的实际应用尚少。现将聚焦乙型肝炎创新药物II/III期临床试验设计的方法学考量,重点介绍临床试验新型设计,以期为乙型肝炎创新药物的研发提供方法学参考。The research and development of innovative drugs targeting the clinical cure of chronic hepatitis B has become active in recent years.In the clinical trials of new drugs for hepatitis B,the use of new design concepts,new methods,and new technologies to evaluate the efficacy of innovative drugs is expected to shorten the clinical research process of candidate new drugs and reduce the cost of new drug development.However,the new designs such as seamless adaptive design and master plan design have few practical applications in clinical trials of innovative hepatitis B drugs.This article will focus on the methodological factors in the design of phase II/III clinical trials of innovative hepatitis B drugs,and introduce the key points of the new clinical trial design in order to provide methodological references for the development of innovative hepatitis B drugs.

关 键 词：肝炎病毒 乙型 功能性治愈 方法学 

分 类 号：R969.4[医药卫生—药理学] R512.62[医药卫生—药学]