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

项目总结/摘要 传统的随机对照试验（RCT）是评估治疗效果的金标准。 然而，RCT可能存在数据缺失，可能无法捕获所有有效性和安全性结局， 对患者很重要，随访时间可能有限，其结果可能不总是可推广到 不同的患者群体在现实世界的设置。将RCT与真实世界数据（RWD）联系起来，例如保险 索赔和电子健康记录，提供了一个宝贵的机会，以加强传统的RCT， 解决这些缺点。在这个项目中，我们建议使用两种情况将RCT与参与者的RWD联系起来 研究：流感疫苗有效阻止心胸事件和失代偿性心力衰竭 （INVESTED）试验和达格列净评价改善保留性射血患者的生存期 心力衰竭分数（DELIVER）试验。本项目的总体目标是使用关联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。我们将使用关联RCT-RWD实现以下特定目标： 目标1。使用长期、经验证、基于RWD的结局指标增强RCT结果。 目标二。开发使用RWD替代或插补缺失RCT数据的方法。 目标3。将RCT结果推广至现实世界中更广泛的患者人群。 目的4：开发方法来识别RCT和基于RWD的研究之间不一致结果的来源。

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.