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Clinical trials are crucial for drug development but are time consuming, expensive, and often burdensome on patients. More importantly, clinical trials face uncertain outcomes due to issues with efficacy, safety, or problems with patient…

Computers and Society · Computer Science 2022-03-15 Tianfan Fu , Kexin Huang , Cao Xiao , Lucas M. Glass , Jimeng Sun

Clinical trials constitute a critical yet exceptionally challenging and costly stage of drug development (\$2.6B per drug), where protocols are encoded as complex natural language documents, motivating the use of AI systems beyond manual…

Artificial Intelligence · Computer Science 2026-04-06 Sixue Xing , Kerui Wu , Xuanye Xia , Meng Jiang , Jintai Chen , Tianfan Fu

Clinical trials are a systematic endeavor to assess the safety and efficacy of new drugs or treatments. Conducting such trials typically demands significant financial investment and meticulous planning, highlighting the need for accurate…

Machine Learning · Computer Science 2025-11-03 Tien Huu Do , Antoine Masquelier , Nae Eoun Lee , Jonathan Crowther

In the era of precision medicine, more and more clinical trials are now driven or guided by biomarkers, which are patient characteristics objectively measured and evaluated as indicators of normal biological processes, pathogenic processes,…

Methodology · Statistics 2024-01-02 Hong Zhang , Jie Pu , Shibing Deng , Satrajit Roychoudhury , Haitao Chu , Douglas Robinson

Background and Objectives: Multidrug Resistance (MDR) is a critical global health issue, causing increased hospital stays, healthcare costs, and mortality. This study proposes an interpretable Machine Learning (ML) framework for MDR…

Background. Designing trials to reduce treatment duration is important in several therapeutic areas, including TB and antibiotics. We recently proposed a new randomised trial design to overcome some of the limitations of standard two-arm…

Clinical trials need to recruit a sufficient number of volunteer patients to demonstrate the statistical power of the treatment (e.g., a new drug) in curing a certain disease. Clinical trial recruitment has a significant impact on trial…

Machine Learning · Computer Science 2024-07-19 Ling Yue , Sixue Xing , Jintai Chen , Tianfan Fu

Clinical trials are essential to drug development but time-consuming, costly, and prone to failure. Accurate trial outcome prediction based on historical trial data promises better trial investment decisions and more trial success. Existing…

Machine Learning · Computer Science 2023-04-12 Zifeng Wang , Cao Xiao , Jimeng Sun

Predicting clinical outcome is remarkably important but challenging. Research efforts have been paid on seeking significant biomarkers associated with the therapy response or/and patient survival. However, these biomarkers are generally…

Machine Learning · Computer Science 2020-10-09 Jiancheng Yang , Jiajun Chen , Kaiming Kuang , Tiancheng Lin , Junjun He , Bingbing Ni

Electronic health records represent a holistic overview of patients' trajectories. Their increasing availability has fueled new hopes to leverage them and develop accurate risk prediction models for a wide range of diseases. Given the…

Clinical trials are critical for drug development. Constructing the appropriate eligibility criteria (i.e., the inclusion/exclusion criteria for patient recruitment) is essential for the trial's success. Proper design of clinical trial…

Computation and Language · Computer Science 2023-10-10 Zifeng Wang , Cao Xiao , Jimeng Sun

Clinical trials are essential for drug development but often suffer from expensive, inaccurate and insufficient patient recruitment. The core problem of patient-trial matching is to find qualified patients for a trial, where patient…

Artificial Intelligence · Computer Science 2021-04-13 Xingyao Zhang , Cao Xiao , Lucas M. Glass , Jimeng Sun

In clinical trials, an experimental treatment is sometimes added on to a standard of care or control therapy in multiple treatment phases (e.g., concomitant and maintenance phases) to improve patient outcomes. When the new regimen provides…

Methodology · Statistics 2020-11-19 Sudipta Bhattacharya , Jyotirmoy Dey

Clinical trial outcome prediction seeks to estimate the likelihood that a clinical trial will successfully reach its intended endpoint. This process predominantly involves the development of machine learning models that utilize a variety of…

Biomolecules · Quantitative Biology 2024-05-14 Chufan Gao , Tianfan Fu , Jimeng Sun

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

Methodology · Statistics 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

Many existing models for clinical trial outcome prediction are optimized using task-specific loss functions on trial phase-specific data. While this scheme may boost prediction for common diseases and drugs, it can hinder learning of…

Machine Learning · Computer Science 2025-05-27 Yiqing Zhang , Xiaozhong Liu , Fabricio Murai

Transformers-based models, such as BERT, have dramatically improved the performance for various natural language processing tasks. The clinical knowledge enriched model, namely ClinicalBERT, also achieved state-of-the-art results when…

Computation and Language · Computer Science 2022-04-18 Yikuan Li , Ramsey M. Wehbe , Faraz S. Ahmad , Hanyin Wang , Yuan Luo

This work presents the development and evaluation of an NLP-enabled probabilistic classifier designed to estimate the probability of technical and regulatory success (pTRS) for clinical trials in the field of neuroscience. While…

Machine Learning · Computer Science 2025-12-02 Michael R. Doane

Significant advancements have been made in recent years to optimize patient recruitment for clinical trials, however, improved methods for patient recruitment prediction are needed to support trial site selection and to estimate appropriate…

Machine Learning · Computer Science 2021-11-16 Jingshu Liu , Patricia J Allen , Luke Benz , Daniel Blickstein , Evon Okidi , Xiao Shi

Clinical trials are an indispensable part of the drug development process, bridging the gap between basic research and clinical application. During the development of new drugs, clinical trials are used not only to evaluate the safety and…

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