A survey of using EHR as real-world evidence for discovering and validating new drug indications
It addresses the problem of translating real-world data into actionable drug-repurposing evidence for researchers, but is incremental as a survey.
This paper surveys approaches for using Electronic Health Records as real-world evidence to discover and validate new drug indications, synthesizing recent developments to provide a foundational resource for researchers.
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.