非酒精性脂肪性肝炎新药研发临床终点评价  

Evaluation of clinical endpoints in new drug research and development for nonalcoholic steatohepatitis

作　　者：佟小非[1] 孙亚朦[1] 尤红[1] TONG Xiaofei;SUN Yameng;YOU Hong(Liver Research Center, Beijing Friendship Hospital, Capital Medical University, National Clinical Research Center for Digestive Diseases, Beijing 100050, China)

机构地区：[1]首都医科大学附属北京友谊医院肝病中心,国家消化系统疾病临床医学研究中心,北京100050

出　　处：《临床肝胆病杂志》2021年第6期1249-1253,共5页Journal of Clinical Hepatology

摘　　要：非酒精性脂肪性肝炎(NASH)日益成为肝硬化、肝细胞癌和肝脏相关死亡的常见原因,目前尚无已批准上市的NASH有效治疗药物。因此,迫切需要NASH新药以改善临床相关终点,减轻疾病负担。NASH的发病和疾病进展与代谢密切相关,且异质性较强,其临床结局的观察需要时间较长,这些特点也为NASH新药的研发带来了挑战。药物研发过程中,治疗终点的选择对于药物疗效的评价至关重要,是否能够反映临床结局、预测临床获益是终点选择的基本原则。总结和论述了目前NASH新药研发中不同阶段的治疗终点的选择。Nonalcoholic steatohepatitis(NASH)has gradually become a common cause of liver cirrhosis,hepatocellular carcinoma,and liver-related deaths,and currently no effective therapeutic drugs have been approved for the treatment of NASH.Therefore,there is an urgent need for effective new drugs to improve clinical endpoints and reduce disease burden.The development and progression of NASH are closely associated with metabolism and have strong heterogeneity,and it takes a long time to observe its clinical outcome.These characteristics bring challenges to the research and development of new drugs for NASH.In the process of drug research and development,the selection of treatment endpoints is crucial to the evaluation of drug efficacy,and the basic principle of endpoint selection is whether it can reflect clinical outcome and predict clinical benefit.This article summarizes and discusses the selection of treatment endpoints at different stages of the research and development of new drugs for NASH.

关 键 词：非酒精性脂肪性肝病 药物发现 临床试验 

分 类 号：R575.5[医药卫生—消化系统]