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There is a movement in design of experiments away from the classic randomization put forward by Fisher, Cochran and others to one based on optimization. In fixed-sample trials comparing two groups, measurements of subjects are known in…

统计方法学 · 统计学 2018-10-22 Adam Kapelner , Abba M. Krieger , Uri Shalit , David Azriel

Randomized experiments are the gold standard for estimating the causal effects of an intervention. In the simplest setting, each experimental unit is randomly assigned to receive treatment or control, and then the outcomes in each treatment…

统计方法学 · 统计学 2020-06-05 Guillaume Basse , Yi Ding , Panos Toulis

The regression discontinuity (RD) design is a quasi-experimental design that estimates the causal effects of a treatment by exploiting naturally occurring treatment rules. It can be applied in any context where a particular treatment or…

统计方法学 · 统计学 2014-03-10 Sara Geneletti , Aidan G. O'Keeffe , Linda D. Sharples , Sylvia Richardson , Gianluca Baio

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…

应用统计 · 统计学 2026-05-29 Zach Shahn , Mats Stensrud

Technological advancements in the field of mobile devices and wearable sensors have helped overcome obstacles in the delivery of care, making it possible to deliver behavioral treatments anytime and anywhere. Increasingly the delivery of…

应用统计 · 统计学 2017-11-13 Walter Dempsey , Peng Liao , Santosh Kumar , Susan A. Murphy

Adaptive approaches, allowing for more flexible trial design, have been proposed for individually randomized trials to save time or reduce sample size. However, adaptive designs for cluster-randomized trials in which groups of participants…

统计方法学 · 统计学 2022-01-10 Junwei Shen , Shirin Golchi , Erica E. M. Moodie , David Benrimoh

Motivated by customer loyalty plans and scholarship programs, we study tie-breaker designs which are hybrids of randomized controlled trials (RCTs) and regression discontinuity designs (RDDs). We quantify the statistical efficiency of a…

统计方法学 · 统计学 2020-08-03 Art B. Owen , Hal Varian

Loss of power and clear description of treatment differences are key issues in designing and analyzing a clinical trial where non-proportional hazard is a possibility. A log-rank test may be very inefficient and interpretation of the hazard…

应用统计 · 统计学 2021-01-13 Satrajit Roychoudhury , Keaven M Anderson , Jiabu Ye , Pralay Mukhopadhyay

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

统计方法学 · 统计学 2021-08-31 Elja Arjas , Dario Gasbarra

This article studies the benefits of using spatially randomized experimental designs which partition the experimental area into distinct, non-overlapping units with treatments assigned randomly. Such designs offer improved policy evaluation…

统计理论 · 数学 2025-11-18 Ying Yang , Chengchun Shi , Fang Yao , Shouyang Wang , Hongtu Zhu

Though platform trials have been touted for their flexibility and streamlined use of trial resources, their statistical efficiency is not well understood. We fill this gap by establishing their greater efficiency for comparing the relative…

统计方法学 · 统计学 2022-01-28 Tzu-Jung Huang , Alex Luedtke , the AMP Investigators Group

Understanding causality should be a core requirement of any attempt to build real impact through AI. Due to the inherent unobservability of counterfactuals, large randomised trials (RCTs) are the standard for causal inference. But large…

Prior probabilities of clinical hypotheses are not systematically used for clinical trial design yet, due to a concern that poor priors may lead to poor decisions. To address this concern, a conservative approach to Bayesian trial design is…

统计方法学 · 统计学 2023-09-21 Fabio Rigat

Background: Well-designed phase II trials must have acceptable error rates relative to a pre-specified success criterion, usually a statistically significant p-value. Such standard designs may not always suffice from a clinical perspective…

应用统计 · 统计学 2020-02-10 Satrajit Roychoudhury , Nicolas Scheuer , Beat Neuenschwander

Suppose an online platform wants to compare a treatment and control policy, e.g., two different matching algorithms in a ridesharing system, or two different inventory management algorithms in an online retail site. Standard randomized…

统计方法学 · 统计学 2022-12-27 Peter Glynn , Ramesh Johari , Mohammad Rasouli

Background: trials to identify the minimal effective treatment duration are needed in different therapeutic areas, including bacterial infections, TB and Hepatitis--C. However, standard non-inferiority designs have several limitations,…

Public service programs often allocate limited resources under uncertainty about their benefits, creating a need for randomization to support credible evaluation. In practice, however, applicants commonly enter waitlists where resources are…

机器学习 · 计算机科学 2026-05-26 JungHo Lee , Johnna Sundberg , Pim Welle , Bryan Wilder

Typically, a randomized experiment is designed to test a hypothesis about the average treatment effect and sometimes hypotheses about treatment effect variation. The results of such a study may then be used to inform policy and practice for…

统计方法学 · 统计学 2026-05-01 Elizabeth Tipton , Michalis Mamakos

This paper analyzes difference-in-differences designs with a continuous treatment. We show that treatment-on-the-treated-type parameters are identified under a parallel trends assumption analogous to the binary treatment case. However,…

计量经济学 · 经济学 2026-01-05 Brantly Callaway , Andrew Goodman-Bacon , Pedro H. C. Sant'Anna

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
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