Ochsner Diabetes Clinical Research Unit, Department of Endocrinology, Frank Riddick Diabetes Institute, Ochsner Medical Center, New Orleans, LA, USA.
Diabetes Research Centre, Leicester General Hospital, University of Leicester, Leicester, UK.
Adv Ther. 2018 Nov;35(11):1763-1774. doi: 10.1007/s12325-018-0805-y. Epub 2018 Oct 24.
Real-world studies have become increasingly important in providing evidence of treatment effectiveness in clinical practice. While randomized clinical trials (RCTs) are the "gold standard" for evaluating the safety and efficacy of new therapeutic agents, necessarily strict inclusion and exclusion criteria mean that trial populations are often not representative of the patient populations encountered in clinical practice. Real-world studies may use information from electronic health and claims databases, which provide large datasets from diverse patient populations, and/or may be observational, collecting prospective or retrospective data over a long period of time. They can therefore provide information on the long-term safety, particularly pertaining to rare events, and effectiveness of drugs in large heterogeneous populations, as well as information on utilization patterns and health and economic outcomes. This review focuses on how evidence from real-world studies can be utilized to complement data from RCTs to gain a more complete picture of the advantages and disadvantages of medications as they are used in practice.Funding: Sanofi US, Inc.
真实世界研究在为临床实践中治疗效果提供证据方面变得越来越重要。虽然随机对照试验（RCT）是评估新治疗药物安全性和疗效的“金标准”，但必要的严格纳入和排除标准意味着试验人群通常不能代表临床实践中遇到的患者人群。真实世界研究可以使用来自电子健康和索赔数据库的信息，这些数据库提供了来自不同患者群体的大量数据集，并且/或者可以是观察性的，在很长一段时间内收集前瞻性或回顾性数据。因此，它们可以提供有关长期安全性（特别是罕见事件）和药物在大型异质人群中的有效性的信息，以及有关使用模式以及健康和经济结果的信息。本综述重点介绍如何利用真实世界研究的证据来补充 RCT 的数据，以更全面地了解药物在实践中的优缺点。
赛诺菲美国公司。
