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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 pivotal for developing new medical treatments but typically carry risks such as patient mortality and enrollment failure that waste immense efforts spanning over a decade. Applying artificial intelligence (AI) to predict…

Large Language Models (LLMs) and multi-agent systems have shown impressive capabilities in natural language tasks but face challenges in clinical trial applications, primarily due to limited access to external knowledge. Recognizing the…

Computation and Language · Computer Science 2024-07-23 Ling Yue , Sixue Xing , Jintai Chen , Tianfan Fu

Clinical trials are critical for advancing medical treatments but remain prohibitively expensive and time-consuming. Accurate prediction of clinical trial outcomes can significantly reduce research and development costs and accelerate drug…

Machine Learning · Computer Science 2025-06-06 Fengze Liu , Haoyu Wang , Joonhyuk Cho , Dan Roth , Andrew W. Lo

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

Methodology · Statistics 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

Developing AI models that are useful in clinical practice, requires efficient collaboration between clinicians and AI developers. This poses a practical challenge: clinicians must repeatedly communicate and refine their requirements with AI…

Computer Vision and Pattern Recognition · Computer Science 2026-05-28 Zihao Zhao , Frederik Hauke , Juliana De Castilhos , Mathis Bode , Jakob Nikolas Kather , Sven Nebelung , Daniel Truhn

The clinical trial process, a critical phase in drug development, is essential for developing new treatments. The primary goal of interventional clinical trials is to evaluate the safety and efficacy of drug-based treatments for specific…

Machine Learning · Computer Science 2024-07-19 Ling Yue , Jonathan Li , Sixue Xing , Md Zabirul Islam , Bolun Xia , Tianfan Fu , Jintai Chen

The deployment of AI models in clinical practice faces a critical challenge: models achieving expert-level performance on benchmarks can fail catastrophically when confronted with real-world variations in medical imaging. Minor shifts in…

Artificial Intelligence · Computer Science 2025-07-09 Lukas Kuhn , Florian Buettner

Artificial intelligence (AI) holds great promise for supporting clinical trials, from patient recruitment and endpoint assessment to treatment response prediction. However, deploying AI without safeguards poses significant risks,…

Machine Learning · Computer Science 2025-10-09 Yao Chen , David Ohlssen , Aimee Readie , Gregory Ligozio , Ruvie Martin , Thibaud Coroller

As a future trend of healthcare, personalized medicine tailors medical treatments to individual patients. It requires to identify a subset of patients with the best response to treatment. The subset can be defined by a biomarker (e.g.…

Methodology · Statistics 2021-08-16 Yitao Lu , Julie Zhou , Li Xing , Xuekui Zhang

This report embarks on a mission to revolutionize clinical trial protocol development through the integration of advanced AI technologies. With a focus on leveraging the capabilities of generative AI, specifically GPT-4, this initiative…

Computational Engineering, Finance, and Science · Computer Science 2024-08-06 Morteza Maleki , SeyedAli Ghahari

Background The cost of drug discovery and development is substantial, with clinical trial outcomes playing a critical role in regulatory approval and patient care. However, access to large-scale, high-quality clinical trial outcome data…

Artificial Intelligence · Computer Science 2025-03-07 Chufan Gao , Jathurshan Pradeepkumar , Trisha Das , Shivashankar Thati , Jimeng Sun

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

Computation · Statistics 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

Clinical trials are vital for evaluation of safety and efficacy of new treatments. However, clinical trials are resource-intensive, time-consuming and expensive to conduct, where errors in trial design, reduced efficacy, and safety events…

Artificial Intelligence · Computer Science 2025-05-19 Trisha Das , Afrah Shafquat , Beigi Mandis , Jacob Aptekar , Jimeng Sun

Clinical trials are conducted to test the effectiveness and safety of potential drugs in humans for regulatory approval. Machine learning (ML) has recently emerged as a new tool to assist in clinical trials. Despite this progress, there…

Artificial Intelligence · Computer Science 2023-10-06 Zifeng Wang , Brandon Theodorou , Tianfan Fu , Cao Xiao , Jimeng Sun

The integration of artificial intelligence [AI] into clinical trials has revolutionized the process of drug development and personalized medicine. Among these advancements, deep learning and predictive modelling have emerged as…

Machine Learning · Computer Science 2024-12-11 Sydney Anuyah , Mallika K Singh , Hope Nyavor

A clinical trial is an essential step in drug development, which is often costly and time-consuming. In silico trials are clinical trials conducted digitally through simulation and modeling as an alternative to traditional clinical trials.…

Quantitative Methods · Quantitative Biology 2022-09-20 Zifeng Wang , Chufan Gao , Lucas M. Glass , Jimeng Sun

A profound gap persists between artificial intelligence (AI) and clinical practice in medicine, primarily due to the lack of rigorous and cost-effective evaluation methodologies. State-of-the-art and state-of-the-practice AI model…

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

Methodology · Statistics 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Most clinical AI systems operate as prediction engines -- producing labels or risk scores -- yet real clinical reasoning is a time-bounded, sequential control problem under uncertainty. Clinicians interleave information gathering with…

Artificial Intelligence · Computer Science 2026-01-21 Dipayan Sengupta , Saumya Panda
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