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In the presence of competing events, many investigators are interested in a direct treatment effect on the event of interest that does not capture treatment effects on competing events. Classical survival analysis methods that treat…

Methodology · Statistics 2025-10-29 Takuya Kawahara , Sean McGrath , Jessica G Young

In both the fields of computer science and medicine there is very strong interest in developing personalized treatment policies for patients who have variable responses to treatments. In particular, I aim to find an optimal personalized…

Machine Learning · Computer Science 2014-07-01 Yousuf M. Soliman

The restricted mean survival time is a clinically easy-to-interpret measure that does not require any assumption of proportional hazards. We focus on two ways to directly model the survival time and adjust the covariates. One is to…

Methodology · Statistics 2022-11-03 Keisuke Hanada , Junji Moriya , Masahiro Kojima

Censored survival data are common in clinical trials, but small control groups can pose challenges, particularly in rare diseases or where balanced randomization is impractical. Recent approaches leverage external controls from historical…

Methodology · Statistics 2025-05-16 Chenyin Gao , Shu Yang , Mingyang Shan , Wenyu Wendy Ye , Ilya Lipkovich , Douglas Faries

Consider the problem of estimating average treatment effects when a large number of covariates are used to adjust for possible confounding through outcome regression and propensity score models. The conventional approach of model building…

Statistics Theory · Mathematics 2018-01-31 Zhiqiang Tan

We propose a reinforcement learning method for estimating an optimal dynamic treatment regime for survival outcomes with dependent censoring. The estimator allows the failure time to be conditionally independent of censoring and dependent…

Methodology · Statistics 2022-05-13 Hunyong Cho , Shannon T. Holloway , David J. Couper , Michael R. Kosorok

With the increasing adoption of electronic health records, there is an increasing interest in developing individualized treatment rules, which recommend treatments according to patients' characteristics, from large observational data.…

Methodology · Statistics 2021-05-05 Muxuan Liang , Young-Geun Choi , Yang Ning , Maureen A Smith , Ying-Qi Zhao

Propensity scores are commonly used to reduce the confounding bias in non-randomized observational studies for estimating the average treatment effect. An important assumption underlying this approach is that all confounders that are…

Methodology · Statistics 2022-08-02 Youfei Yu , Jiacong Du , Min Zhang , Zhenke Wu , Andrew M. Ryan , Bhramar Mukherjee

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

Imputing missing potential outcomes using an estimated regression function is a natural idea for estimating causal effects. In the literature, estimators that combine imputation and regression adjustments are believed to be comparable to…

Statistics Theory · Mathematics 2023-01-20 Zhexiao Lin , Fang Han

We propose a semiparametric data fusion framework for efficient inference on survival probabilities by integrating right-censored and current status data. Existing data fusion methods focus largely on fusing right-censored data only, while…

Methodology · Statistics 2025-09-15 Xiudi Li , Sijia Li

Real-world clinical decision making is a complex process that involves balancing the risks and benefits of treatments. Quality-adjusted lifetime is a composite outcome that combines patient quantity and quality of life, making it an…

Statistics Theory · Mathematics 2024-12-09 Hao Sun , Ashkan Ertefaie , Luke Duttweiler , Brent A. Johnson

Using offline observational data for policy evaluation and learning allows decision-makers to evaluate and learn a policy that connects characteristics and interventions. Most existing literature has focused on either discrete treatment…

Artificial Intelligence · Computer Science 2025-01-22 Cheuk Hang Leung , Yiyan Huang , Yijun Li , Qi Wu

When to initiate treatment on patients is an important problem in many medical studies such as AIDS and cancer. In this article, we formulate the treatment initiation time problem for time-to-event data and propose an optimal individualized…

Methodology · Statistics 2021-09-30 Xin Chen , Rui Song , Jiajia Zhang , Swann Arp Adams , Liuquan Sun , Wenbin Lu

Individual treatment effect (ITE) estimation is to evaluate the causal effects of treatment strategies on some important outcomes, which is a crucial problem in healthcare. Most existing ITE estimation methods are designed for centralized…

Machine Learning · Computer Science 2025-03-10 Changchang Yin , Hong-You Chen , Wei-Lun Chao , Ping Zhang

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

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

In clinical trials, multiple outcomes of different priorities commonly occur as the patient's response may not be adequately characterized by a single outcome. Win statistics are appealing summary measures for between-group difference at…

Methodology · Statistics 2025-06-04 Ying Cui , Bo Huang , Gaohong Dong , Ryuji Uozumi , Lu Tian

We introduce a statistical procedure that integrates survival data from multiple biomedical studies, to improve the accuracy of predictions of survival or other events, based on individual clinical and genomic profiles, compared to models…

Applications · Statistics 2020-07-20 Steffen Ventz , Rahul Mazumder , Lorenzo Trippa

Observational studies have recently received significant attention from the machine learning community due to the increasingly available non-experimental observational data and the limitations of the experimental studies, such as…

Machine Learning · Computer Science 2023-01-26 Vinod Kumar Chauhan , Soheila Molaei , Marzia Hoque Tania , Anshul Thakur , Tingting Zhu , David A. Clifton
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