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In meta-analysis, the random-effects models are standard tools to address between-study heterogeneity in evidence synthesis analyses. For the random-effects distribution models, the normal distribution model has been adopted in most…

Applications · Statistics 2021-07-28 Hisashi Noma , Kengo Nagashima , Shogo Kato , Satoshi Teramukai , Toshi A. Furukawa

Clinical randomized controlled trials (RCTs) collect hundreds of measurements spanning various metric types (e.g., laboratory tests, cognitive/motor assessments, etc.) across 100s-1000s of subjects to evaluate the effect of a treatment, but…

Machine Learning · Computer Science 2024-06-25 Sayeri Lala , Niraj K. Jha

Suppose we are interested in the effect of a treatment in a clinical trial. The efficiency of inference may be limited due to small sample size. However, external control data are often available from historical studies. Motivated by an…

Methodology · Statistics 2021-12-10 Xinyu Li , Wang Miao , Fang Lu , Xiao-Hua Zhou

With more and better clinical data being captured outside of clinical studies and greater data sharing of clinical studies, external controls may become a more attractive alternative to randomized clinical trials. Both industry and…

Randomised Controlled Trials (RCTs) are the gold standard for estimating treatment effects across many fields of science. Technology companies have adopted A/B-testing methods as a modern RCT counterpart, where end-users are randomly…

Social and Information Networks · Computer Science 2024-09-20 Olivier Jeunen

The performance of machine learning models under distribution shift has been the focus of the community in recent years. Most of current methods have been proposed to improve the robustness to distribution shift from the algorithmic…

Computer Vision and Pattern Recognition · Computer Science 2022-05-26 Ziquan Liu , Yi Xu , Yuanhong Xu , Qi Qian , Hao Li , Rong Jin , Xiangyang Ji , Antoni B. Chan

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

Switchback experiments--alternating treatment and control over time--are widely used when unit-level randomization is infeasible, outcomes are aggregated, or user interference is unavoidable. In practice, experimentation must support fast…

Methodology · Statistics 2026-02-27 Jizhou Liu , Liang Zhong

Research in machine learning is making progress in fixing its own reproducibility crisis. Reinforcement learning (RL), in particular, faces its own set of unique challenges. Comparison of point estimates, and plots that show successful…

Machine Learning · Computer Science 2024-02-07 Ted Fujimoto , Joshua Suetterlein , Samrat Chatterjee , Auroop Ganguly

We argue that randomized controlled trials (RCTs) are special even among settings where average treatment effects are identified by a nonparametric unconfoundedness assumption. This claim follows from two results of Robins and Ritov (1997):…

Methodology · Statistics 2021-09-28 P. M. Aronow , James M. Robins , Theo Saarinen , Fredrik Sävje , Jasjeet Sekhon

Randomized controlled trials (RCTs) are the gold standard for assessing drug safety and efficacy. However, RCTs have some drawbacks which have led to the use of single-arm studies to make certain internal drug development and regulatory…

When choosing estimands and estimators in randomized clinical trials, caution is warranted as intercurrent events, such as - due to patients who switch treatment after disease progression, are often extreme. Statistical analyses may then…

Applications · Statistics 2023-03-13 Hege Michiels , An Vandebosch , Stijn Vansteelandt

Despite their cost, randomized controlled trials (RCTs) are widely regarded as gold-standard evidence in disciplines ranging from social science to medicine. In recent decades, researchers have increasingly sought to reduce the resource…

Methodology · Statistics 2026-02-24 Guilherme Duarte

Ensuring generalization to unseen environments remains a challenge. Domain shift can lead to substantially degraded performance unless shifts are well-exercised within the available training environments. We introduce a simple robust…

Machine Learning · Computer Science 2021-10-20 Yilun Xu , Tommi Jaakkola

Conformal prediction is a popular, modern technique for providing valid predictive inference for arbitrary machine learning models. Its validity relies on the assumptions of exchangeability of the data, and symmetry of the given model…

Methodology · Statistics 2023-03-20 Rina Foygel Barber , Emmanuel J. Candes , Aaditya Ramdas , Ryan J. Tibshirani

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

Artificial Intelligence · Computer Science 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Randomized controlled trials (RCTs) have long been the gold standard for causal inference across various fields, including business analysis, economic studies, sociology, clinical research, and network learning. The primary advantage of…

Econometrics · Economics 2024-08-23 Carol Liu

Randomized controlled trials (RCTs) often include subgroup analyses to assess whether treatment effects vary across pre-specified patient populations. However, these analyses frequently suffer from small sample sizes which limit the power…

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…

Randomized controlled trials (RCTs) have been the cornerstone of clinical evidence; however, their cost, duration, and restrictive eligibility criteria limit power and external validity. Studies using real-world data (RWD), historically…

Methodology · Statistics 2025-11-26 Shu Yang , Margaret Gamalo , Haoda Fu