中国和美国应用真实世界证据支持药品审批的比较研究  

A comparative study of the application of real real-world evidence to support drug approval in China and US

作　　者：木尼热·莫合塔尔 王素[1,2] 陈玉文 MOHETAER Munire;WANG Su;CHEN Yu-wen(School of Business and Management,Shenyang Pharmaceutical University,Shenyang 110016,China;Institute of Pharmaceutical Regulatory Sciences,Shenyang Pharmaceutical University,Shenyang 110016,China)

机构地区：[1]沈阳药科大学工商管理学院,沈阳110016 [2]沈阳药科大学药品监管科学研究院,沈阳110016

出　　处：《中国新药杂志》2024年第11期1082-1088,共7页Chinese Journal of New Drugs

摘　　要：本文旨在为促进我国真实世界证据用于药品审批的应用和制度建立提供参考。采用文献研究法对我国和美国两国关于真实世界证据支持药品审批的技术指南进行梳理,并对真实世界证据在美国药品及生物制品审批中的成功案例与我国进口化学药品与中药新药审批中的应用情况进行了比较。与美国相比,我国在药品审批中使用真实世界证据的相关制度、批准比例、应用程度与真实世界数据质量方面仍然存在一定的差距,而两国基于真实世界证据批准药品的适应证通常是需求未得到满足的罕见疾病。为促进我国将真实世界证据用于药品审批,我国应该完善在药品审批中使用真实世界证据的制度,提高在药品审批中使用真实世界证据的比例,明确使用真实世界证据支持药品审批的核心目标,跟踪国际、梳理经验,提升数据质量和规范化程度。The purpose of this paper is to provide a reference for promoting the application of real-world evidence in drug approval in China and the system establishment.We adopt the literature research to sort out the technical guidelines on real-world evidence supporting drug approval in China and US,and compare the successful cases of its application in some drugs and biologics in the United States,and in some imported chemicals and new Chinese medicines in China.Compared with the US,gap still exists in China's system related to the use of real-world evidence in drug approvals,the percentage of approvals,the degree of application,and the quality of real-world data.The indications for drugs that are approved based on real-world evidence in the two countries are usually rare diseases with unmet needs.In order to promote the use of real-world evidence for drug approval in China,our country should improve the system facilitating the use of real-world evidence in drug approval,increase the proportion of real-world evidence used in drug approval,clarify the core objectives of using real-world evidence to support drug approval,follow up the international practice,comb useful experiences,and improve the quality and standardization of the data.

关 键 词：真实世界数据 真实世界研究 真实世界证据 药品审批 比较研究 

分 类 号：R95[医药卫生—药学]