用电子病历发现新药用途，回顾当前研究方法与挑战
A survey of using EHR as real-world evidence for discovering and validating new drug indications
- 系统梳理电子病历在药物重定位中的数据与建模方法
- 强调真实世界证据在药物疗效验证中的统计设计与评估框架
- 突出大语言模型和目标试验模拟在提升可信度中的作用
电子病历（EHR）正越来越多地作为真实世界证据（RWE），支持新药适应症的发现与验证。本文综述了基于EHR的药物重定位研究方法，涵盖数据来源、处理流程与表示技术，讨论了评估药物疗效的研究设计与统计框架。重点分析了验证过程中的关键挑战，并强调大型语言模型（LLMs）与目标试验模拟（target trial emulation）的重要作用。通过整合近期进展与方法创新，本工作为研究人员将真实世界数据转化为可行动的药物重定位证据提供了基础资源。
原文摘要 · Abstract (English)
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.
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