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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

This research on data extraction methods applies recent advances in natural language processing to evidence synthesis based on medical texts. Texts of interest include abstracts of clinical trials in English and in multilingual contexts.…

Computation and Language · Computer Science 2020-01-31 Lena Schmidt , Julie Weeds , Julian P. T. Higgins

Abstruse learning algorithms and complex datasets increasingly characterize modern clinical decision support systems (CDSS). As a result, clinicians cannot easily or rapidly scrutinize the CDSS recommendation when facing a difficult…

Artificial Intelligence · Computer Science 2021-11-02 Bojian Hou , Hao Zhang , Gur Ladizhinsky , Gur Ladizhinsky , Stephen Yang , Volodymyr Kuleshov , Fei Wang , Qian Yang

Causal identification of treatment effects for infectious disease outcomes in interconnected populations is challenging because infection outcomes may be transmissible to others, and treatment given to one individual may affect others'…

Methodology · Statistics 2021-05-11 Xiaoxuan Cai , Eben Kenah , Forrest W. Crawford

The estimand framework is increasingly established to pose research questions in confirmatory clinical trials. In evidence synthesis, the uptake of estimands has been modest, and the PICO (Population, Intervention, Comparator, Outcome)…

Although reinforcement learning has found widespread use in dense reward settings, training autonomous agents with sparse rewards remains challenging. To address this difficulty, prior work has shown promising results when using not only…

Machine Learning · Computer Science 2022-10-25 Kai Yan , Alexander G. Schwing , Yu-Xiong Wang

Early identification of intensive care patients at risk of in-hospital mortality enables timely intervention and efficient resource allocation. Despite high predictive performance, existing machine learning approaches lack transparency and…

Machine Learning · Computer Science 2025-11-21 Alexander Bakumenko , Janine Hoelscher , Hudson Smith

Predicting the outcomes of prospective clinical trials remains a major challenge for large language models. Prior work has shown that both traditional correlational predictors, such as random forests and logistic regression, and strong…

Objective: Clinical trials are essential for advancing pharmaceutical interventions, but they face a bottleneck in selecting eligible participants. Although leveraging electronic health records (EHR) for recruitment has gained popularity,…

Computation and Language · Computer Science 2026-01-15 Mojdeh Rahmanian , Seyed Mostafa Fakhrahmad , Seyedeh Zahra Mousavi

Machine learning systems show significant promise for forecasting patient adverse events via risk scores. However, these risk scores implicitly encode assumptions about future interventions that the patient is likely to receive, based on…

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are…

Econometrics · Economics 2020-06-02 Charles F. Manski , Aleksey Tetenov

Motivation: Machine learning based prediction of compound-protein interactions (CPIs) is important for drug design, screening and repurposing studies and can improve the efficiency and cost-effectiveness of wet lab assays. Despite the…

Quantitative Methods · Quantitative Biology 2022-02-02 Adiba Yaseen , Imran Amin , Naeem Akhter , Asa Ben-Hur , Fayyaz Minhas

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

Many diagnostic errors occur because clinicians cannot easily access relevant information in patient Electronic Health Records (EHRs). In this work we propose a method to use LLMs to identify pieces of evidence in patient EHR data that…

Availability of diagnostic codes in Electronic Health Records (EHRs) is crucial for patient care as well as reimbursement purposes. However, entering them in the EHR is tedious, and some clinical codes may be overlooked. Given an…

Machine Learning · Computer Science 2023-05-10 Tsvetan R. Yordanov , Ameen Abu-Hanna , Anita CJ Ravelli , Iacopo Vagliano

In clinical trials, inferences on clinical outcomes are often made conditional on specific selective processes. For instance, only when a treatment demonstrates a significant effect on the primary outcome, further analysis is conducted to…

Methodology · Statistics 2025-04-15 Tianyu Pan , Vivek Charu , Ying Lu , Lu Tian

Traditional statistical inference in cluster randomized trials typically invokes the asymptotic theory that requires the number of clusters to approach infinity. In this article, we propose an alternative conformal causal inference…

Methodology · Statistics 2024-10-03 Bingkai Wang , Fan Li , Mengxin Yu

Systematic reviews in medicine play a critical role in evidence-based decision-making by aggregating findings from multiple studies. A central bottleneck in automating this process is extracting numeric evidence and determining study-level…

Artificial Intelligence · Computer Science 2026-01-26 Massimiliano Pronesti , Michela Lorandi , Paul Flanagan , Oisin Redmond , Anya Belz , Yufang Hou

Clinical language models have achieved strong performance on downstream tasks by pretraining on domain specific corpora such as discharge summaries and medical notes. However, most approaches treat the electronic health record as a static…

Computation and Language · Computer Science 2025-04-28 Tatsunori Tanaka , Fi Zheng , Kai Sato , Zhifeng Li , Yuanyun Zhang , Shi Li

In clinical research and clinical decision-making, it is important to know if a study changes or only supports the current standards of care for specific disease management. We define such a change as transformative and a support as…

Computation and Language · Computer Science 2021-12-28 Xuanyu Shi , Jian Du