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We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling…

Methodology · Statistics 2023-02-27 Imke Mayer , Julie Josse , Traumabase Group

Mobile health systems in recent times, have notably improved the healthcare sector by empowering patients to actively participate in their health, and by facilitating access to healthcare professionals. Effective operation of these mobile…

Artificial Intelligence · Computer Science 2021-10-18 Chinazunwa Uwaoma , Gunjan. Mansingh

Missing data is a systemic problem in practical scenarios that causes noise and bias when estimating treatment effects. This makes treatment effect estimation from data with missingness a particularly tricky endeavour. A key reason for this…

Machine Learning · Statistics 2023-02-27 Jeroen Berrevoets , Fergus Imrie , Trent Kyono , James Jordon , Mihaela van der Schaar

The analysis of randomized trials is often complicated by the occurrence of intercurrent events and missing values. Even though there are different strategies to address missing values it is still common to require missing values…

Methodology · Statistics 2025-11-11 A. Ruiz de Villa , Ll. Badiella

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

Methodology · Statistics 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

Reinforcement learning (RL) plays a central role in improving the reasoning and alignment of large language models, yet its efficiency critically depends on how training data are selected. Existing online selection strategies predominantly…

Machine Learning · Computer Science 2026-03-03 Xinyu Zhou , Boyu Zhu , Haotian Zhang , Huiming Wang , Zhijiang Guo

The current literature regarding generation of complex, realistic synthetic tabular data, particularly for randomized controlled trials (RCTs), often ignores missing data. However, missing data are common in RCT data and often are not…

Other Statistics · Statistics 2025-12-02 Niki Z. Petrakos , Erica E. M. Moodie , Nicolas Savy

When analyzing data from randomized clinical trials, covariate adjustment can be used to account for chance imbalance in baseline covariates and to increase precision of the treatment effect estimate. A practical barrier to covariate…

Methodology · Statistics 2023-07-04 Chia-Rui Chang , Yue Song , Fan Li , Rui Wang

Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

Methodology · Statistics 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…

Machine Learning · Computer Science 2023-03-07 Zeshan Hussain , Michael Oberst , Ming-Chieh Shih , David Sontag

Data from observational studies (OSs) is widely available and readily obtainable yet frequently contains confounding biases. On the other hand, data derived from randomized controlled trials (RCTs) helps to reduce these biases; however, it…

Methodology · Statistics 2024-10-30 Dong Yao , Caizhi Tang , Qing Cui , Longfei Li

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

Researchers often run resource-intensive randomized controlled trials (RCTs) to estimate the causal effects of interventions on outcomes of interest. Yet these outcomes are often noisy, and estimated overall effects can be small or…

Econometrics · Economics 2023-12-21 Jann Spiess , Vasilis Syrgkanis , Victor Yaneng Wang

We consider estimating the conditional average treatment effect for everyone by eliminating confounding and selection bias. Unfortunately, randomized clinical trials (RCTs) eliminate confounding but impose strict exclusion criteria that…

Machine Learning · Statistics 2021-06-15 Eric V. Strobl , Thomas A. Lasko

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

With the uptake of targeted therapies, instead of the "one-fits-all" approach, modern randomized clinical trials (RCTs) often aim to develop treatments that target a subgroup of patients. Motivated by analyzing the Age-Related Eye Disease…

Applications · Statistics 2020-03-25 Yue Wei , Jason C. Hsu , Wei Chen , Emily Y. Chew , Ying Ding

Efficiently managing and utilizing large-scale medical imaging datasets with limited resources presents significant challenges. While coreset selection helps reduce computational costs, its effectiveness in medical data remains limited due…

Computer Vision and Pattern Recognition · Computer Science 2026-03-19 Yan Liang , Ziyuan Yang , Zhuxin Lei , Mengyu Sun , Yingyu Chen , Yi Zhang

Versatile medical image segmentation (VMIS) targets the segmentation of multiple classes, while obtaining full annotations for all classes is often impractical due to the time and labor required. Leveraging partially labeled datasets (PLDs)…

Computer Vision and Pattern Recognition · Computer Science 2025-09-08 Shengqian Zhu , Jiafei Wu , Xiaogang Xu , Chengrong Yu , Ying Song , Zhang Yi , Guangjun Li , Junjie Hu

Disruptions in clinical trials may be due to external events like pandemics, warfare, and natural disasters. Resulting complications may lead to unforeseen intercurrent events (events that occur after treatment initiation and affect the…

Applications · Statistics 2024-08-20 Rachael V. Phillips , Mark J. van der Laan

A main research goal in various studies is to use an observational data set and provide a new set of counterfactual guidelines that can yield causal improvements. Dynamic Treatment Regimes (DTRs) are widely studied to formalize this…

Machine Learning · Computer Science 2023-06-06 Soroush Saghafian