利用补充真实世界数据研究获得更深层次的认识

Using Enriched Real-World Research for Deeper Insights

随着患者、医护人员、药品注册人员和政策决定者对现实世界中各种诊疗手段的安全性和有效性相关证据的迫切需求,我们需要新的研究设计来高效有效地对此类问题进行评估。与此同时,越来越多的二手数据可以用来提供此种相关证据,常用的二手数据包括电子医疗档案和医疗保险数据等。补充真实世界数据研究同时利用了现有二手数据和通过医生/患者收集的一手数据,并将两者进行结合。在现有数据的基础上,补充真实世界数据研究可以集中精力收集缺失的重要信息来满足研究目的。本文将对补充真实世界数据研究进行介绍,包括研究设计要素,研究的优点和局限性,实施要点以及应用实例等。

As patients,medical care providers,regulators and policymakers seek additional evidence about the safety and effectiveness of various treatments as used in real-world settings,new study designs are needed to efficiently and effectively address these challenging research questions. While challenges exist,secondary health data such as electronic medical records and health insurance claims are becoming increasingly available to support generation of key evidence. Enriched real-world research studies combine data collected directly from physicians and patients with existing data such as medical records and insurance claims. By utilizing readily available data,enriched studies can focus data collection efforts on critical study information that may not have been recorded anywhere and which are important for the study purposes. We discuss here important design components of enriched studies,including strengths and limitations,practical considerations,and case examples.