Virtual Clinical Trials Drive Success Prediction in Heart Failure Drug Development

Researchers at Mayo Clinic have pioneered an innovative approach to assess the potential effectiveness of existing drugs for treating heart failure by leveraging virtual clinical trials. This method combines sophisticated computer models with real-world patient data, streamlining the drug discovery process by reducing costs, time, and the risk of failure in traditional trials. The team developed an AI-enabled framework that emulates clinical trials by creating comparison groups from electronic health records (EHRs) of nearly 60,000 heart failure patients, allowing for the prediction of a drug's efficacy without conducting actual randomized controlled trials.

The approach incorporates drug-target modeling, which uses artificial intelligence to analyze chemical structures alongside biological data such as protein sequences and genes. This enhances the accuracy of predicting how drugs might perform in real-world settings. The researchers validated their model by retrospectively analyzing 17 drugs that had previously undergone Phase 3 trials in heart failure, successfully predicting the direction of outcomes in 7 beneficial and 10 non-beneficial cases.

This predictive capability enables scientists to identify promising repurposed drugs more efficiently, prioritizing candidates for further testing and potentially accelerating the availability of new treatments. As Dr. Nansu Zong explains, while the current model can determine whether a drug is likely to succeed, the next steps involve quantifying the level of efficacy. The technology has the potential to revolutionize clinical research by making it faster, more affordable, and more accessible, especially through a broader initiative led by Mayo Clinic under Dr. Cui Tao. This initiative explores trial emulation, simulation, and synthetic trials using AI, facilitating a new era of translational science.

Ultimately, these advancements could significantly impact how new therapies are developed and deployed, promising faster access for patients and decreased healthcare costs. Source: https://medicalxpress.com/news/2025-09-virtual-clinical-trials-success-heart.html