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

项目摘要 随机对照试验(RCT)仍然是确定两者之间因果关系的金标准 药物和健康结果。然而，对于一些临床问题，RCT可能是不可行的，不道德的， 成本高昂，或者只能推广到非常有限的人群。在这些情况下，常规的观察性研究 收集的“真实世界”健康数据(RWD)对于补充来自随机对照试验的证据至关重要。RWD是 在心血管结果方面特别有用，这通常在医疗保健中被很好地捕捉到 数据库，但对RWD研究有效性的担忧减轻了它们的影响。这个项目的目的是 通过对以下大样本进行系统复制，对RWD研究的有效性进行经验性评估 心血管结局的随机试验，包括已有结果的已完成试验和 正在进行的尚未公布结果的试验，以确保有效性不受 试验结果的可用性。RWD研究旨在尽可能地与相应的RCT相匹配， 包括暴露、结果和纳入/排除标准，RWD研究使用了各种设计 和分析方法，以便提供指导，说明哪些问题可以在RWD和 用什么方法。作为该项目的一部分，将开发一种新的荟萃分析模型来模拟 RCT和RWD之间的治疗效果评估，结合RWD方法学方法的数据 以及各种临床问题。所有研究的所有治疗效果评估，包括随机对照试验和随机对照试验 结果，将在网上报告，正在进行的随机对照试验的结果将在可用时添加。这 该项目是第一个评估基于经验的RWD心血管结果研究的有效性的项目 在大样本临床问题上的表现，涉及广泛的流行病学和分析 方法：研究方法。研究结果将指导研究人员计划对心血管结果进行RWD研究并增加 对RWD研究回答对患者、提供者和其他人重要问题的能力的信心 利益相关者。

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.