Enhancing evidence generation by linking randomized clinical trials (RCTs) to real world data (RWD)

通过将随机临床试验 (RCT) 与真实世界数据 (RWD) 联系起来增强证据生成

• 批准号: 10185038
• 负责人: Mehdi Najafzadeh
• 金额: $ 152.39万
• 依托单位: BRIGHAM AND WOMEN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-01 至 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10185038
• 关键词: Enhancing; evidence; generation; linking; randomized

Project Summary/Abstract Traditional randomized controlled trials (RCTs) are the gold standard for assessing the efficacy of treatments. However, RCTs may have missing data, may not capture all effectiveness and safety outcomes that are important to patients, may have limited follow-up duration, and their results may not always generalizable to diverse patient populations in real-world settings. Linking RCTs to real-world data (RWD), such as insurance claims and electronic health record, provides a valuable opportunity to enhance traditional RCTs by addressing these shortcomings. In this project, we propose linking RCTs to participants’ RWD using two case studies: The INfluenza Vaccine to Effectively Stop Cardio Thoracic Events and Decompensated Heart Failure (INVESTED) trial and Dapagliflozin Evaluation to Improve the LIVEs of Patients With PReserved Ejection Fraction Heart Failure (DELIVER) trial. The overarching objective of this project is to use linked RCT-RWD to develop and validate methods that can enrich both randomized and non-randomized comparative effectiveness studies. We will use linked RCT-RWD to accomplish the following specific aims: Aim 1. To enhance RCT findings using long-term, validated, RWD-based outcome measures. Aim 2. To develop methods for using RWD to replace or impute missing RCT data. Aim 3. To generalize RCT results to broader patient populations in real-world setting. Aim 4: To develop methods to identify sources of discrepant results between RCTs and RWD-based studies.

项目概要/摘要 传统的随机对照试验（RCT）是评估治疗效果的黄金标准。 然而，随机对照试验可能缺少数据，可能无法捕获所有有效性和安全性结果。 对患者很重要，随访时间可能有限，而且他们的结果可能并不总能推广到 现实世界中不同的患者群体。将 RCT 与真实世界数据 (RWD) 连接起来，例如保险 索赔和电子健康记录，为增强传统随机对照试验提供了宝贵的机会 解决这些缺点。在这个项目中，我们建议使用两个案例将 RCT 与参与者的 RWD 联系起来 研究：流感疫苗可有效阻止心胸事件和失代偿性心力衰竭 （已投资）试验和达格列净评估可改善保留射血患者的 LIVE 部分心力衰竭（DELIVER）试验。该项目的总体目标是使用链接的 RCT-RWD 开发和验证可以丰富随机和非随机比较的方法 有效性研究。我们将使用链接的 RCT-RWD 来实现以下具体目标： 目标 1. 使用长期、经过验证、基于 RWD 的结果测量来增强 RCT 结果。 目标 2. 开发使用 RWD 替换或估算缺失的 RCT 数据的方法。 目标 3. 将 RCT 结果推广到现实世界中更广泛的患者群体。 目标 4：开发方法来识别 RCT 和基于 RWD 的研究之间差异结果的来源。