非酒精性脂肪性肝炎新药研发临床终点评价

佟小非; 孙亚朦; 尤红

doi:10.3969/j.issn.1001-5256.2021.06.003

非酒精性脂肪性肝炎新药研发临床终点评价

DOI: 10.3969/j.issn.1001-5256.2021.06.003

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

利益冲突声明：所有作者均声明不存在利益冲突。

作者贡献声明：佟小非负责文献检索，文章框架设计，论文撰写；孙亚朦负责论文修订；尤红负责文章结构和关键内容审定，终审定稿。

详细信息

作者简介:
佟小非(1986—)，女，主要从事非酒精性脂肪性肝病的临床研究

通信作者:
尤红，youhong30@sina.com

中图分类号: R575.5
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- 被引次数: 0
出版历程
- 收稿日期: 2021-05-04
- 录用日期: 2021-05-04
- 出版日期: 2021-06-20

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

Liver Research Center, Beijing Friendship Hospital, Capital Medical University, National Clinical Research Center for Digestive Diseases, Beijing 100050, China

摘要

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。
- 非酒精性脂肪性肝病 /
- 药物发现 /
- 临床试验
Abstract: 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.
- Non-alcoholic Fatty Liver Disease /
- Drug Discovery /
- Clinical Trial

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参考文献(38)

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