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<p>The complexities inherent in EHR data create discrepancies between real-world evidence and RCTs, posing substantial challenges in determining whether a treatment is likely to have a beneficial impact compared to the standard of care in RCTs. The objective of this study is to enhance the prediction of efficacy direction for repurposed drugs tested in RCTs for heart failure (HF). To achieve this, we propose an efficacy direction prediction framework that integrates drug-target predictions with EHR-based Emulation Trials (ET) to derive surrogate endpoints for prediction using HF prognostic markers. Our validation of the proposed novel drug-target prediction model against the BETA benchmark demonstrates superior performance, surpassing existing baseline algorithms. Furthermore, an evaluation of our framework in identifying 17 repurposed drugs—derived from 266 phase 3 HF RCTs—using data from 59,000 patients at the Mayo Clinic highlights its remarkable predictive accuracy.</p>

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Advancing efficacy prediction for electronic health records based emulated trials in repurposing heart failure therapies

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年5月24日

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  • Nansu Zong,
  • Shaika Chowdhury,
  • Shibo Zhou,
  • Sivaraman Rajaganapathy,
  • Yue Yu,
  • Liewei Wang,
  • Qiying Dai,
  • Pengyang Li,
  • Xiaoke Liu,
  • Suzette J. Bielinski,
  • Jun Chen,
  • Yongbin Chen,
  • James R. Cerhan

摘要

The complexities inherent in EHR data create discrepancies between real-world evidence and RCTs, posing substantial challenges in determining whether a treatment is likely to have a beneficial impact compared to the standard of care in RCTs. The objective of this study is to enhance the prediction of efficacy direction for repurposed drugs tested in RCTs for heart failure (HF). To achieve this, we propose an efficacy direction prediction framework that integrates drug-target predictions with EHR-based Emulation Trials (ET) to derive surrogate endpoints for prediction using HF prognostic markers. Our validation of the proposed novel drug-target prediction model against the BETA benchmark demonstrates superior performance, surpassing existing baseline algorithms. Furthermore, an evaluation of our framework in identifying 17 repurposed drugs—derived from 266 phase 3 HF RCTs—using data from 59,000 patients at the Mayo Clinic highlights its remarkable predictive accuracy.