Department of Biomedical Informatics, Columbia University, New York, New York, USA.
Institute of Human Nutrition, Columbia University, New York, New York, USA.
J Am Med Inform Assoc. 2021 Jan 15;28(1):144-154. doi: 10.1093/jamia/ocaa224.
Real-world data (RWD), defined as routinely collected healthcare data, can be a potential catalyst for addressing challenges faced in clinical trials. We performed a scoping review of database-specific RWD applications within clinical trial contexts, synthesizing prominent uses and themes.
Querying 3 biomedical literature databases, research articles using electronic health records, administrative claims databases, or clinical registries either within a clinical trial or in tandem with methodology related to clinical trials were included. Articles were required to use at least 1 US RWD source. All abstract screening, full-text screening, and data extraction was performed by 1 reviewer. Two reviewers independently verified all decisions.
Of 2020 screened articles, 89 qualified: 59 articles used electronic health records, 29 used administrative claims, and 26 used registries. Our synthesis was driven by the general life cycle of a clinical trial, culminating into 3 major themes: trial process tasks (51 articles); dissemination strategies (6); and generalizability assessments (34). Despite a diverse set of diseases studied, <10% of trials using RWD for trial process tasks evaluated medications or procedures (5/51). All articles highlighted data-related challenges, such as missing values.
Database-specific RWD have been occasionally leveraged for various clinical trial tasks. We observed underuse of RWD within conducted medication or procedure trials, though it is subject to the confounder of implicit report of RWD use.
Enhanced incorporation of RWD should be further explored for medication or procedure trials, including better understanding of how to handle related data quality issues to facilitate RWD use.
真实世界数据（RWD）定义为常规收集的医疗保健数据，可能是解决临床试验中面临的挑战的潜在催化剂。我们对临床试验背景下特定数据库的 RWD 应用进行了范围综述，综合了突出的用途和主题。
查询了 3 个生物医学文献数据库，研究文章使用电子健康记录、行政索赔数据库或临床登记处，无论是在临床试验内部还是与临床试验相关的方法学同时使用。文章必须至少使用 1 个美国 RWD 来源。所有的摘要筛选、全文筛选和数据提取均由 1 名评审员完成。两名评审员独立验证所有决策。
在 2020 篇筛选的文章中，有 89 篇符合条件：59 篇文章使用电子健康记录，29 篇文章使用行政索赔，26 篇文章使用登记处。我们的综合分析是由临床试验的一般生命周期驱动的，最终形成了 3 个主要主题：试验过程任务（51 篇文章）；传播策略（6 篇）；和普遍性评估（34 篇）。尽管研究了各种疾病，但只有不到 10%的使用 RWD 进行试验过程任务的试验评估了药物或程序（5/51）。所有文章都强调了数据相关的挑战，如缺失值。
特定于数据库的 RWD 偶尔被用于各种临床试验任务。我们观察到在进行的药物或程序试验中对 RWD 的使用不足，尽管这受到 RWD 使用的隐含报告的混杂因素的影响。
应进一步探索增强 RWD 的纳入，包括更好地理解如何处理相关的数据质量问题，以促进 RWD 的使用。
