Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Center for Observational and Real-world Evidence, Merck & Co., Inc, Kenilworth, New Jersey, USA.
Pharmacoepidemiol Drug Saf. 2021 Jun;30(6):685-693. doi: 10.1002/pds.5222. Epub 2021 Mar 18.
There is increasing interest in utilizing real-world data (RWD) to produce real-world evidence (RWE) on the benefits and risks of medical products that could support regulatory approval decisions. The field of pharmacoepidemiology has a long history of focusing on data and evidence that would now be termed "real-world," including evidence from healthcare claims, registries, and electronic health records. However, several emerging trends over the past decade are converging to support the use of these and other RWD sources for approval decisions, and there are several recent examples and ongoing research that demonstrate how RWE may be used to support regulatory approval of new or expanded indications. The goal of this article is to review the current landscape and future directions of the use of RWE in this context. This manuscript is endorsed by the International Society for Pharmacoepidemiology (ISPE).
人们越来越关注利用真实世界数据（RWD）生成关于医疗产品效益和风险的真实世界证据（RWE），以支持监管批准决策。药物流行病学领域长期以来一直专注于现在被称为“真实世界”的数据和证据，包括来自医疗保健索赔、登记处和电子健康记录的数据。然而，过去十年中的几个新兴趋势正在汇聚，以支持使用这些和其他 RWD 来源进行批准决策，并且有一些最近的例子和正在进行的研究表明如何使用 RWE 来支持新的或扩大的适应证的监管批准。本文的目的是回顾在这种情况下使用 RWE 的现状和未来方向。本文由国际药物流行病学学会（ISPE）认可。
