真实世界研究的方法学进展

The Progress in Methodology of Real World Study

近年来,如何利用真实世界数据辅助临床试验评价药物的有效性和安全性,已经成为全球相关监管机构、制药工业界和学术界共同关注且具挑战性的热点问题。本文简要介绍了国际主要监管机构发布的指南,并结合实例讨论该领域的难点和关注点。从统计学和流行病学角度,介绍了国内外监管机构、工业界和学术界成立的各项真实世界数据研究工作组及其在方法学研究的最新进展,以及真实世界数据/证据使用现况和存在的难点,为真实世界数据工作者提供启发和讨论的基础,以期更加科学、客观地使用合适的真实世界数据。

In recent years,how to use real world data to support clinical trials to evaluate the effectiveness and safety of drugs has become a hot topic,and challenging issue of global regulatory agencies,the pharmaceutical industry and academia.This paper briefly introduced the guidelines issued by major international regulatory agencies,and discussed the difficulties and concerns in this field with examples.From the perspective of statistics and epidemiology,this paper introduced various real world data research working groups formed by regulatory agencies,industry and academia at home and abroad,and their latest progress in methodology research,as well as the current situation and difficulties of real world data/evidence use.This aims to provide insights and discussion basis for real world data workers,so as to be more scientific and practical objective use of appropriate real world data.