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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…

Clustering and dependence are common in trials. For example, in some cluster randomized trials (CRTs), pre-existing clusters are enrolled, randomized, and serve as the basis of intervention delivery. Such CRTs are "fully clustered":…

Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyze them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level.…

统计方法学 · 统计学 2021-08-06 Fangzhou Su , Peng Ding

The ability to generalize experimental results from randomized control trials (RCTs) across locations is crucial for informing policy decisions in targeted regions. Such generalization is often hindered by the lack of identifiability due to…

计量经济学 · 经济学 2021-12-10 Xinkun Nie , Guido Imbens , Stefan Wager

Completely randomized experiments have been the gold standard for drawing causal inference because they can balance all potential confounding on average. However, they may suffer from unbalanced covariates for realized treatment…

统计理论 · 数学 2022-10-18 Yuhao Wang , Xinran Li

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…

统计方法学 · 统计学 2026-01-22 Xinyuan Chen , Fan Li

Combinatorial linear semi-bandits (CLS) are widely applicable frameworks of sequential decision-making, in which a learner chooses a subset of arms from a given set of arms associated with feature vectors. Existing algorithms work poorly…

机器学习 · 统计学 2019-09-11 Kei Takemura , Shinji Ito

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

The paper considers two different designs; a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome, and a twoarm superiority cluster RCT with a binary outcome. From a Bayesian perspective, for the analysis…

统计方法学 · 统计学 2025-11-11 Abdullah Aloufi , Kevin Wilson , Nina Wilson , Lisa Shaw , Christopher Price

In randomized experiments, treatment and control groups should be roughly the same--balanced--in their distributions of pretreatment variables. But how nearly so? Can descriptive comparisons meaningfully be paired with significance tests?…

统计方法学 · 统计学 2008-08-29 Ben B. Hansen , Jake Bowers

Randomization is a common technique used in clinical trials to eliminate potential bias and confounders in a patient population. Equal allocation to treatment groups is the standard due to its optimal efficiency in many cases. However, in…

应用统计 · 统计学 2020-04-09 Thevaa Chandereng , Xiaodan Wei , Rick Chappell

We consider the task of evaluating policies of algorithmic resource allocation through randomized controlled trials (RCTs). Such policies are tasked with optimizing the utilization of limited intervention resources, with the goal of…

人工智能 · 计算机科学 2023-02-07 Aditya Mate , Bryan Wilder , Aparna Taneja , Milind Tambe

This paper studies inference in cluster randomized trials where treatment status is determined according to a "matched pairs" design. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the level of the…

计量经济学 · 经济学 2025-08-14 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

Recently, there as been an increasing interest in the use of heavily restricted randomization designs which enforces balance on observed covariates in randomized controlled trials. However, when restrictions are strict, there is a risk that…

统计方法学 · 统计学 2021-10-15 Mattias Nordin , Mårten Schultzberg

Current practices for designing cluster-randomized trials (cRCTs) typically rely on closed-form formulas for power calculations. For cRCTs using covariate-constrained randomization, the utility of conventional calculations might be limited,…

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

统计方法学 · 统计学 2022-11-10 Wenqi Shi , Xi Lin

The randomization inference literature studying randomized controlled trials (RCTs) assumes that units' potential outcomes are deterministic. This assumption is unlikely to hold, as stochastic shocks may take place during the experiment. In…

计量经济学 · 经济学 2022-12-15 Antoine Deeb , Clément de Chaisemartin

External controls (ECs) from historical trials or real-world data have gained increasing attention as a way to augment hybrid and single-arm trials, especially when balanced randomization is infeasible. While most existing work has focused…

统计方法学 · 统计学 2025-12-15 Yujing Gao , Xiang Zhang , Shu Yang

Cluster randomized trials (CRTs) offer a practical alternative for addressing logistical challenges and ensuring feasibility in community health, education, and prevention studies, even though randomized controlled trials are considered the…

统计方法学 · 统计学 2025-10-30 Jooyeon Lee , M. S. , Evan Kwiatkowski , Ph. D

The intra-cluster correlation coefficient (ICC) plays an important role while designing the cluster randomized trials (CRTs). Often optimal CRTs are designed assuming that the magnitude of the ICC is constant across the clusters. However,…

统计计算 · 统计学 2019-02-21 Satya Prakash Singh , Pradeep Yadav