Duke Clinical Research Institute, Durham, North Carolina.
Vanderbilt University Medical Center, Nashville, Tennessee.
JAMA Cardiol. 2024 Sep 1;9(9):852-857. doi: 10.1001/jamacardio.2024.2019.
Pragmatic randomized clinical trials (RCTs) often use multiple data sources to examine clinical events, but the relative contribution of data sources to clinical end-point rates is understudied.
To assess the contribution of data sources (electronic health records [EHRs], public/private insurance claims, and/or participant-reported data) to clinical end points among ADAPTABLE participants who had available data.
DESIGN, SETTING, AND PARTICIPANTS: The ADAPTABLE study was an open-label, pragmatic RCT from April 2016 through June 2019 conducted in research networks within clinical practice. Participants had existing atherosclerotic cardiovascular disease and available data to analyze. The characteristics of patients by combinations of data source availability were compared to examine the contribution of each of the data sources to end-point ascertainment. Data for this prespecified analysis were examined from January 2022 to June 2023.
Randomized exposure to 81 mg or 325 mg of aspirin daily.
Number of events for the primary end point (composite of death, hospitalization for myocardial infarction, and hospitalization for stroke) that were contributed by EHR or claims data and then number of events contributed by each additional data source.
Of 15 006 participants randomized with at least 1 other source of data available beyond participant-reported data, there were 8756 (58.3%) with participant-reported and EHR data; 4291 (28.6%) with participant-reported, EHR, and claims data; 1412 (9.4%) with EHR-only data; 262 (1.7%) with participant-reported and claims data; 202 (1.3%) with EHR and claims data; and 83 (0.6%) with claims-only data. Participants with EHR-only data were younger (median age, 63.7 years; IQR, 55.8-71.4) compared with the other groups (range, 65.6-71.9 years). Among participants with both EHR and claims data, with or without participant-reported data (n = 4493), for each outcome, most events (92%-100%) were identified in the EHR or in claims data. For all clinical end points, participant-reported data contributed less than 10% of events not otherwise available from claims or EHR data.
In this analysis of a pragmatic RCT, claims and EHR data provided the most clinical end-point data when compared with participant-reported events. These findings provide a framework for collecting end points in pragmatic clinical trials. Further work is needed to understand the data source combinations that most effectively provide clinical end-point data in RCTs.
实用随机临床试验（RCT）通常使用多种数据源来检查临床事件，但数据来源对临床终点率的相对贡献仍研究不足。
评估 ADAPTABLE 参与者中数据来源（电子健康记录 [EHR]、公共/私人保险索赔和/或参与者报告的数据）对临床终点的贡献，这些参与者有可用数据。
设计、设置和参与者：ADAPTABLE 研究是一项 2016 年 4 月至 2019 年 6 月在临床实践中的研究网络中进行的开放性、实用 RCT。参与者患有现有的动脉粥样硬化性心血管疾病，并有可用的数据进行分析。通过数据来源可用性的组合比较患者的特征，以检查每个数据来源对终点确定的贡献。本预定分析的数据于 2022 年 1 月至 2023 年 6 月进行检查。
随机接受每天 81 毫克或 325 毫克的阿司匹林。
主要终点（死亡、心肌梗死住院和中风住院的复合）的事件数量，这些事件归因于 EHR 或索赔数据，然后归因于每个额外数据来源的事件数量。
在随机分配至少有 1 种其他数据源（除了参与者报告的数据）的 15006 名参与者中，有 8756 名（58.3%）参与者报告的数据和 EHR 数据；4291 名（28.6%）参与者报告的数据、EHR 和索赔数据；1412 名（9.4%）仅 EHR 数据；262 名（1.7%）参与者报告的数据和索赔数据；202 名（1.3%）EHR 和索赔数据；83 名（0.6%）仅索赔数据。仅 EHR 数据的参与者更年轻（中位数年龄，63.7 岁；IQR，55.8-71.4），与其他组（范围，65.6-71.9 岁）相比。在有 EHR 和索赔数据的参与者中，无论是否有参与者报告的数据（n=4493），对于每个结果，大多数事件（92%-100%）是在 EHR 或索赔数据中确定的。对于所有临床终点，参与者报告的数据仅占无法从索赔或 EHR 数据中获得的事件的不到 10%。
在这项实用 RCT 的分析中，与参与者报告的事件相比，索赔和 EHR 数据提供了最多的临床终点数据。这些发现为实用临床试验中终点的收集提供了框架。需要进一步研究来了解在 RCT 中最有效地提供临床终点数据的数据来源组合。
