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A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

A treatment may be appropriate for some group (the ``sick" group) on whom it has a positive effect, but it can also have a detrimental effect on subjects from another group (the ``healthy" group). In a non-targeted trial both sick and…

Machine Learning · Computer Science 2025-04-23 Georgios Mavroudeas , Malik Magdon-Ismail , Kristin P. Bennett , Jason Kuruzovich

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

Traditional click-through rate (CTR) prediction models convert the tabular data into one-hot vectors and leverage the collaborative relations among features for inferring the user's preference over items. This modeling paradigm discards…

Information Retrieval · Computer Science 2023-12-19 Xiangyang Li , Bo Chen , Lu Hou , Ruiming Tang

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range…

Methodology · Statistics 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

In many scientific experiments, the data annotating cost constraints the pace for testing novel hypotheses. Yet, modern machine learning pipelines offer a promising solution, provided their predictions yield correct conclusions. We focus on…

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

Throughout the COVID-19 pandemic, government policy and healthcare implementation responses have been guided by reported positivity rates and counts of positive cases in the community. The selection bias of these data calls into question…

Applications · Statistics 2021-04-12 Len Covello , Andrew Gelman , Yajuan Si , Siquan Wang

A fundamental difficulty of causal learning is that causal models can generally not be fully identified based on observational data only. Interventional data, that is, data originating from different experimental environments, improves…

Methodology · Statistics 2021-11-04 Juan L. Gamella , Christina Heinze-Deml

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

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

Following the global COVID-19 pandemic, the number of scientific papers studying the virus has grown massively, leading to increased interest in automated literate review. We present a clinical text mining system that improves on previous…

Computation and Language · Computer Science 2020-12-09 Veysel Kocaman , David Talby

Integrating multimodal Electronic Health Records (EHR) data, such as numerical time series and free-text clinical reports, has great potential in predicting clinical outcomes. However, prior work has primarily focused on capturing temporal…

Machine Learning · Computer Science 2025-11-10 Fuying Wang , Feng Wu , Yihan Tang , Lequan Yu

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

We reframe clinician overrides of clinical AI recommendations as implicit preference data - the same signal structure exploited by reinforcement learning from human feedback (RLHF), but richer: the annotator is a domain expert, the…

Machine Learning · Computer Science 2026-05-19 Prabhjot Singh , Abhishek Gupta , Chris Betz , Abe Flansburg , Brett Ives , Sudeep Lama , Jung Hoon Son

Clinical trials (CTs) often fail due to inadequate patient recruitment. This paper tackles the challenges of CT retrieval by presenting an approach that addresses the patient-to-trials paradigm. Our approach involves two key components in a…

Information Retrieval · Computer Science 2023-07-04 Wojciech Kusa , Óscar E. Mendoza , Petr Knoth , Gabriella Pasi , Allan Hanbury

Due to the characteristics of COVID-19, the epidemic develops rapidly and overwhelms health service systems worldwide. Many patients suffer from systemic life-threatening problems and need to be carefully monitored in ICUs. Thus the…

Machine Learning · Computer Science 2020-07-20 Liantao Ma , Xinyu Ma , Junyi Gao , Chaohe Zhang , Zhihao Yu , Xianfeng Jiao , Wenjie Ruan , Yasha Wang , Wen Tang , Jiangtao Wang

Methods to handle missing data have been extensively explored in the context of estimation and descriptive studies, with multiple imputation being the most widely used method in clinical research. However, in the context of clinical risk…

Methodology · Statistics 2024-11-25 Junhui Mi , Rahul D. Tendulkar , Sarah M. C. Sittenfeld , Sujata Patil , Emily C. Zabor

Concept bottleneck models (CBMs), which predict human-interpretable concepts (e.g., nucleus shapes in cell images) before predicting the final output (e.g., cell type), provide insights into the decision-making processes of the model.…

Computer Vision and Pattern Recognition · Computer Science 2024-07-10 Winnie Pang , Xueyi Ke , Satoshi Tsutsui , Bihan Wen