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I propose a finite sample inference procedure that uses a likelihood function derived from the randomization process within an experiment to conduct inference on various quantities that capture heterogeneous intervention effects. One such…

Methodology · Statistics 2020-09-03 Amanda Kowalski

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

The number of recurrent events before a terminating event is often of interest. For instance, death terminates an individual's process of rehospitalizations and the number of rehospitalizations is an important indicator of economic cost. We…

Methodology · Statistics 2021-12-30 Willem van den Boom , Maria De Iorio , Marta Tallarita

Researchers are often challenged with assessing the impact of an intervention on an outcome of interest in situations where the intervention is non-randomised, the intervention is only applied to one or few units, the intervention is…

Two-sided marketplace platforms often run experiments to test the effect of an intervention before launching it platform-wide. A typical approach is to randomize individuals into the treatment group, which receives the intervention, and the…

Methodology · Statistics 2021-04-27 Hannah Li , Geng Zhao , Ramesh Johari , Gabriel Y. Weintraub

The efficacy of an intervention can be assessed by randomizing patients to different diagnostic tests instead of directly to an intervention and control. This principle is applied by allocating individuals to intervention if the test result…

Applications · Statistics 2022-01-11 Huw Llewelyn

Count outcomes in longitudinal studies are frequent in clinical and engineering studies. In frequentist and Bayesian statistical analysis, methods such as Mixed linear models allow the variability or correlation within individuals to be…

Methodology · Statistics 2024-07-15 Alejandra Estefanía Patiño Hoyos , Johnatan Cardona Jiménez

Clinical trials often evaluate multiple outcome variables to form a comprehensive picture of the effects of a new treatment. The resulting multidimensional insight contributes to clinically relevant and efficient decision-making about…

Methodology · Statistics 2023-08-14 X. M. Kavelaars , J. Mulder , M. C. Kaptein

When longitudinal outcomes are evaluated in mortal populations, their non-existence after death complicates the analysis and its causal interpretation. Where popular methods often merge longitudinal outcome and survival into one scale or…

We consider the problem of designing a prospective randomized trial in which the outcome data will be self-reported, and will involve sensitive topics. Our interest is in misreporting behavior, and how respondents' tendency to under- or…

Methodology · Statistics 2021-08-27 Evan T. R. Rosenman , Rina Friedberg , Mike Baiocchi

It is common that in multiarm randomized trials, the outcome of interest is "truncated by death," meaning that it is only observed or well defined conditioning on an intermediate outcome. In this case, in addition to pairwise contrasts, the…

Methodology · Statistics 2016-11-22 Linbo Wang , Thomas S. Richardson , Xiao-Hua Zhou

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable…

Methodology · Statistics 2025-09-01 Jiaqi Tong , Chao Cheng , Guangyu Tong , Michael O. Harhay , Fan Li

Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events (AEs)…

Methodology · Statistics 2024-10-08 Anthony Sisti , Andrew Zullo , Roee Gutman

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes…

Methodology · Statistics 2021-12-21 Shuxi Zeng , Fan Li , Liangyuan Hu , Fan Li

Causal inference is crucial for understanding the true impact of interventions, policies, or actions, enabling informed decision-making and providing insights into the underlying mechanisms that shape our world. In this paper, we establish…

Methodology · Statistics 2024-03-26 Jingyue Huang , Changbao Wu , Leilei Zeng

To investigate intervention effects on rare events, meta-analysis techniques are commonly applied in order to assess the accumulated evidence. When it comes to adverse effects in clinical trials, these are often most adequately handled…

Methodology · Statistics 2026-04-03 Christian Röver , Qiong Wu , Anja Loos , Tim Friede

Decision-makers often deploy the best-performing treatment from a randomized experiment, creating a winner's curse: selection favors treatments whose observed outcomes are high partly because of statistical noise, so the na\"ive estimate of…

Econometrics · Economics 2026-05-20 Ron Berman , Walter W. Zhang , Hangcheng Zhao

Over the last decade, the number of randomized trials of programs to reduce intimate partner violence (IPV) has grown precipitously. However, most trials continue to measure and code violence using standards originally designed for global…

Applications · Statistics 2022-09-28 Christopher Boyer , Sangeeta Chatterji , Jasper Cooper , Lori Heise

Generalized linear models, such as logistic regression, are widely used to model the association between a treatment and a binary outcome as a function of baseline covariates. However, the coefficients of a logistic regression model…

Methodology · Statistics 2022-01-04 Jiaqi Yin , Sonia Markes , Thomas S. Richardson , Linbo Wang

Causal inference is best understood using potential outcomes. This use is particularly important in more complex settings, that is, observational studies or randomized experiments with complications such as noncompliance. The topic of this…

Statistics Theory · Mathematics 2007-06-13 Donald B. Rubin
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