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Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…

Methodology · Statistics 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

Efficiently allocating treatments with a budget constraint constitutes an important challenge across various domains. In marketing, for example, the use of promotions to target potential customers and boost conversions is limited by the…

Machine Learning · Computer Science 2024-05-06 Toon Vanderschueren , Wouter Verbeke , Felipe Moraes , Hugo Manuel Proença

We propose a dynamic allocation procedure that increases power and efficiency when measuring an average treatment effect in sequential randomized trials. Subjects arrive iteratively and are either randomized or paired via a matching…

Methodology · Statistics 2013-05-23 Adam Kapelner , Abba Krieger

Randomized experiments (a.k.a. A/B tests) are a powerful tool for estimating treatment effects, to inform decisions making in business, healthcare and other applications. In many problems, the treatment has a lasting effect that evolves…

Machine Learning · Computer Science 2022-10-17 Ziyang Tang , Yiheng Duan , Stephanie Zhang , Lihong Li

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

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…

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

Variable-ratio matching is a flexible alternative to conventional $1$-to-$k$ matching for designing observational studies that emulate a target randomized controlled trial (RCT). To achieve fine balance -- that is, matching treated and…

Methodology · Statistics 2025-10-07 Qian Meng , Zhe Chen , Bo Zhang

Clinical trials usually target average treatment effects, but treatment decisions are made for individuals. This tension motivates a common criticism of evidence-based medicine: a treatment that is beneficial on average may be inappropriate…

Applications · Statistics 2026-05-29 Zach Shahn , Mats Stensrud

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Methodology · Statistics 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

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

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical…

Methodology · Statistics 2015-06-30 Stanislav Minsker , Ying-Qi Zhao , Guang Cheng

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

Applications · Statistics 2019-02-04 Timothy NeCamp , Josh Gardner , Christopher Brooks

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

This paper proposes an adaptive randomization procedure for two-stage randomized controlled trials. The method uses data from a first-wave experiment in order to determine how to stratify in a second wave of the experiment, where the…

Econometrics · Economics 2022-07-06 Max Tabord-Meehan

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

We consider the optimal experimental design problem of allocating subjects to treatment or control when subjects participate in multiple, separate controlled experiments within a short time-frame and subject covariate information is…

Methodology · Statistics 2024-12-16 William Fisher , Qiong Zhang , Lulu Kang , Xinwei Deng

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

Methodology · Statistics 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

Controlled experiments are widely used in many applications to investigate the causal relationship between input factors and experimental outcomes. A completely randomized design is usually used to randomly assign treatment levels to…

Methodology · Statistics 2026-05-12 Yiou Li , Lulu Kang , Xiao Huang

Subgroup selection in clinical trials is essential for identifying patient groups that react differently to a treatment, thereby enabling personalised medicine. In particular, subgroup selection can identify patient groups that respond…

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