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Design-based simulations - procedures that hold realized outcomes fixed and generate variation by resampling treatment assignment or shocks - are widely used in both methodological and applied work to assess inference procedures. This paper…

Econometrics · Economics 2026-03-13 Bruno Ferman

Randomized controlled trials are not only the golden standard in medicine and vaccine trials but have spread to many other disciplines like behavioral economics, making it an important interdisciplinary tool for scientists. When designing…

Methodology · Statistics 2021-11-30 Tassilo Schwarz

Matched case-control studies are commonly employed in epidemiological research for their convenience and efficiency. Analysis of secondary outcomes can yield valuable insights into biological pathways and help identify genetic variants of…

Methodology · Statistics 2026-02-24 Shanshan Liu , Guoqing Diao

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…

Methodology · Statistics 2025-10-28 Xi Fang , Zhiqiang Cao , Fan Li

When designing experimental studies with human participants, experimenters must decide how many trials each participant will complete, as well as how many participants to test. Most discussion of statistical power (the ability of a study…

Neurons and Cognition · Quantitative Biology 2021-08-30 Daniel H. Baker , Greta Vilidaite , Freya A. Lygo , Anika K. Smith , Tessa R. Flack , Andre D. Gouws , Timothy J. Andrews

The goal of this article is to investigate how human participants allocate their limited time to decisions with different properties. We report the results of two behavioral experiments. In each trial of the experiments, the participant…

Neurons and Cognition · Quantitative Biology 2016-07-20 Arash Khodadadi , Pegah Fakhari , Jerome R. Busemeyer

This paper studies the evaluation of methods for targeting the allocation of limited resources to a high-risk subpopulation. We consider a randomized controlled trial to measure the difference in efficiency between two targeting methods and…

Applications · Statistics 2018-04-04 Eric Potash

Experiments on online marketplaces and social networks suffer from interference, where the outcome of a unit is impacted by the treatment status of other units. We propose a framework for modeling interference using a ubiquitous deployment…

Methodology · Statistics 2023-08-21 Ariel Boyarsky , Hongseok Namkoong , Jean Pouget-Abadie

Clinical trials face mounting challenges: fragmented patient populations, slow enrollment, and unsustainable costs, particularly for late phase trials in oncology and rare diseases. While external control arms built from real-world data…

Machine Learning · Computer Science 2025-11-21 Perrine Chassat , Van Tuan Nguyen , Lucas Ducrot , Emilie Lanoy , Agathe Guilloux

A computer code can simulate a system's propagation of variation from random inputs to output measures of quality. Our aim here is to estimate a critical output tail probability or quantile without a large Monte Carlo experiment. Instead,…

Machine Learning · Statistics 2019-08-16 Hao Chen , William J. Welch

Estimating the effects of interventions in networks is complicated when the units are interacting, such that the outcomes for one unit may depend on the treatment assignment and behavior of many or all other units (i.e., there is…

Methodology · Statistics 2014-08-15 Dean Eckles , Brian Karrer , Johan Ugander

The empirical risk minimization approach to data-driven decision making requires access to training data drawn under the same conditions as those that will be faced when the decision rule is deployed. However, in a number of settings, we…

Methodology · Statistics 2025-09-17 Roshni Sahoo , Lihua Lei , Stefan Wager

Context: Software engineering has a problem in that when we empirically evaluate competing prediction systems we obtain conflicting results. Objective: To reduce the inconsistency amongst validation study results and provide a more formal…

Software Engineering · Computer Science 2021-01-15 Martin Shepperd , Stephen G. MacDonell

The recent explosion in the capabilities of large language models has led to a wave of interest in how best to prompt a model to perform a given task. While it may be tempting to simply choose a prompt based on average performance on a…

Machine Learning · Computer Science 2024-03-29 Thomas P. Zollo , Todd Morrill , Zhun Deng , Jake C. Snell , Toniann Pitassi , Richard Zemel

Clinical trials traditionally employ blinding as a design mechanism to reduce the influence of placebo effects. In practice, however, it can be difficult or impossible to blind study participants and unblinded trials are common in medical…

Applications · Statistics 2016-06-22 Elias Chaibub Neto

Time-to-event endpoints are central to evaluate treatment efficacy across many disease areas. Many trial protocols include interim analyses within group-sequential designs that control type I error via spending functions or boundary…

Methodology · Statistics 2026-01-19 Edoardo Ratti , Federico L. Perlino , Stefania Galimberti , Maria G. Valsecchi

Randomized network coding (RNC) greatly reduces the complexity of implementing network coding in large-scale, heterogeneous networks. This paper examines two tradeoffs in applying RNC: The first studies how the performance of RNC varies…

Information Theory · Computer Science 2009-02-18 Yingda Chen , Shalinee Kishore

The primary goal of randomized trials is to compare the effects of different interventions on some outcome of interest. In addition to the treatment assignment and outcome, data on baseline covariates, such as demographic characteristics or…

Applications · Statistics 2014-01-09 Alisa J. Stephens , Eric J. Tchetgen Tchetgen , Victor De Gruttola

Although recent model-free reinforcement learning algorithms have been shown to be capable of mastering complicated decision-making tasks, the sample complexity of these methods has remained a hurdle to utilizing them in many real-world…

Machine Learning · Computer Science 2020-04-21 Saeed Moazami , Peggy Doerschuk

Randomization tests are a popular method for testing causal effects in clinical trials with finite-sample validity. In the presence of heterogeneous treatment effects, it is often of interest to select a subgroup that benefits from the…

Methodology · Statistics 2025-04-29 Zijun Gao