Title: A survey of using EHR as real-world evidence for discovering and validating new drug indications Show Chinese title

Title: 使用电子健康记录作为真实世界证据发现和验证新药适应症的调查

Abstract: Electronic Health Records (EHRs) have been increasingly used as real-world evidence (RWE) to support the discovery and validation of new drug indications. This paper surveys current approaches to EHR-based drug repurposing, covering data sources, processing methodologies, and representation techniques. It discusses study designs and statistical frameworks for evaluating drug efficacy. Key challenges in validation are discussed, with emphasis on the role of large language models (LLMs) and target trial emulation. By synthesizing recent developments and methodological advances, this work provides a foundational resource for researchers aiming to translate real-world data into actionable drug-repurposing evidence. Show Chinese abstract

Abstract: 电子健康记录（EHRs）已被越来越多地用作真实世界证据（RWE），以支持新药适应症的发现和验证。 本文综述了基于EHR的药物再利用的当前方法，涵盖了数据来源、处理方法以及表示技术。 它讨论了评估药物疗效的研究设计和统计框架。 讨论了验证中的关键挑战，重点介绍了大型语言模型（LLMs）和目标试验模拟的作用。 通过综合近期的发展和方法学进展，这项工作为研究人员提供了一个基础资源，旨在将真实世界的数据转化为可操作的药物再利用证据。