Randomized Cardiovascular Trials Duplicated Using Prospective Longitudinal Insurance Claims: Applying Techniques of Epidemiology (RCT DUPLICATE)

使用前瞻性纵向保险索赔重复的随机心血管试验：应用流行病学技术（RCT DUPLICATE）

• 批准号: 10392863
• 负责人: Sebastian G. Schneeweiss
• 金额: $ 71.34万
• 依托单位: BRIGHAM AND WOMEN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-04-01 至 2024-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10392863
• 关键词: Cardiovascular system; Characteristics; Clinical; Data; Data Analyses; Databases; Decision Making; Drug Approval; Educational workshop; Ensure; Epidemiologic Methods; Epidemiology; Evaluation; Exclusion Criteria; Factor Analysis; Future; Gold; Health; Healthcare; Individual; Internet; Knowledge; Lead; Measures; Meta-Analysis; Methodology; Methods; Modeling; Observational Study; Outcome; Patients; Performance; Pharmaceutical Preparations; Population; Provider; Public Health; Publishing; Randomized; Randomized Controlled Trials; Reporting; Research; Research Design; Research Personnel; Review Literature; Sampling; Savings; Techniques; Variant; Work; World Health; analytical method; base; cost; design; health data; improved; innovation; insight; insurance claims; patient population; prospective; randomized trial; searchable database; success; treatment comparison; treatment effect; unethical

Project Abstract Randomized controlled trials (RCTs) remain the gold standard for establishing the causal relationship between medications and health outcomes. However, for some clinical questions RCTs may be infeasible, unethical, costly, or generalizable to only a very narrow population. In these cases, observational studies from routinely collected “real-world” health data (RWD) are crucial for supplementing the evidence from RCTs. RWD is particularly useful in the context of cardiovascular outcomes, which are typically well-captured in healthcare databases, but concern about the validity of RWD studies mitigate their impact. The purpose of this project is to empirically evaluate the validity of RWD studies by conducting a systematic replication of a large sample of randomized trials of cardiovascular outcomes, including both completed trials with results already available and ongoing trials where results are not yet released, in order to ensure that validity is not influenced by the availability of trial results. RWD studies are designed to match the corresponding RCT as closely as possible, including exposures, outcomes, and inclusion/exclusion criteria, and RWD studies utilize a variety of design and analysis approaches in order to provide guidance on which questions can be answered in RWD and with which methods. As part of this project, a new meta-analysis model will be developed to model the variation in treatment effect estimates between RCTs and RWD, combining data across RWD methodologic approaches and across clinical questions. All treatment effect estimates for all studies, including both RCT and RWD results, will be reported online, and the results of ongoing RCTs will be added as they become available. This project is the first to evaluate the validity of RWD studies of cardiovascular outcomes based on empirical performance in a large sample of clinical questions across a wide variety of epidemiologic and analytical methods. Findings will guide investigators planning RWD studies of cardiovascular outcomes and increase confidence in the ability of RWD studies to answer questions of importance to patients, providers, and other stakeholders.

项目摘要 随机对照试验（RCT）仍然是建立因果关系的黄金标准 药物和健康结果。但是，对于某些临床问题，RCT可能是不可行的，不道德的， 代价高昂，或者只能向非常狭窄的人口概括。在这些情况下，经常进行的观察性研究 收集的“现实世界”健康数据（RWD）对于补充RCT的证据至关重要。 RWD是 在心血管结局的背景下，特别有用 数据库，但关注RWD研究的有效性减轻其影响。这个项目的目的是 通过经验评估RWD研究的有效性，通过进行大量样本的系统复制 心血管结局的随机试验，包括两个已完成的试验，并具有可用的结果和 正在进行的结果尚未发布的试验，以确保有效性不受任何影响 试验结果的可用性。 RWD研究旨在使相应的RCT尽可能接近， 包括曝光，结果和包容/排除标准，RWD研究使用了各种设计 和分析方法，以提供指导，以便在RWD中回答哪些问题 哪种方法。作为该项目的一部分，将开发一个新的荟萃分析模型来建模 RCT和RWD之间的治疗效应估计，将RWD方法学方法的数据结合在一起 以及跨临床问题。所有研究（包括RCT和RWD）的所有治疗效应估计值 结果将在线报告，并且将在可用时添加正在进行的RCT的结果。这 项目是第一个评估基于经验的心血管结局RWD研究的有效性的项目 在各种流行病学和分析中的大量临床问题样本中的表现 方法。调查结果将指导研究人员计划心血管结局的RWD研究并增加 对RWD研究回答对患者，提供者和其他重要性问题的能力的信心 利益相关者。