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Inferring treatment effects on a survival time outcome based on data from an observational study is challenging due to the presence of censoring and possible confounding. An additional challenge occurs when a unit's treatment affects the…

Methodology · Statistics 2025-08-13 Chanhwa Lee , Donglin Zeng , Michael Emch , John D. Clemens , Michael G. Hudgens

In randomized trials, the per-protocol effect, that is, the effect of being assigned a treatment strategy and receiving treatment according to the assigned strategy, is sometimes thought to reflect the effect of the treatment strategy…

Methodology · Statistics 2025-09-25 Issa J. Dahabreh , Lawson Ung , Miguel A. Hernán , Yu-Han Chiu

Estimands can help to clarify the research questions being addressed in randomised trials. Because the choice of estimand can affect how relevant trial results are to patients and other stakeholders, such as clinicians or policymakers, it…

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

Many policy evaluations occur in settings where treatment is randomized at the cluster level, and there is treatment noncompliance within each cluster. For example, villages might be assigned to treatment and control, but residents in each…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

We investigate large-sample properties of treatment effect estimators under unknown interference in randomized experiments. The inferential target is a generalization of the average treatment effect estimand that marginalizes over potential…

Statistics Theory · Mathematics 2019-10-25 Fredrik Sävje , Peter M. Aronow , Michael G. Hudgens

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

Methodology · Statistics 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Randomized saturation designs are two-stage experiments: they first randomly assign treatment probabilities over the clusters and then randomly assign the treatment to the units within the clusters. The existing literature on randomized…

Methodology · Statistics 2026-05-29 Sizhu Lu , Lei Shi , Peng Ding

Treatment effect estimation is a fundamental problem in causal inference. We focus on designing efficient randomized controlled trials, to accurately estimate the effect of some treatment on a population of $n$ individuals. In particular,…

Machine Learning · Computer Science 2022-10-14 Raghavendra Addanki , David Arbour , Tung Mai , Cameron Musco , Anup Rao

We present new results on average causal effects in settings with unmeasured exposure-outcome confounding. Our results are motivated by a class of estimands, e.g., frequently of interest in medicine and public health, that are currently not…

Methodology · Statistics 2023-12-25 Lan Wen , Aaron L. Sarvet , Mats J. Stensrud

Randomized trials are often conducted with separate randomizations across multiple sites such as schools, voting districts, or hospitals. These sites can differ in important ways, including the site's implementation, local conditions, and…

Methodology · Statistics 2018-03-19 Lo-Hua Yuan , Avi Feller , Luke W. Miratrix

Vaccination is an effective way to prevent and control the occurrence and epidemic of infectious diseases. However, many factors influence whether the residents decide to get vaccinated or not, such as the efficacy and side effects while…

Physics and Society · Physics 2022-03-09 Yingyue Ke , Jin Zhou

Randomized controlled trials (RCTs) are increasingly prevalent in education research, and are often regarded as a gold standard of causal inference. Two main virtues of randomized experiments are that they (1) do not suffer from…

Combating the SARS-CoV2 pandemic will require the fast development of effective preventive vaccines. Regulatory agencies may open accelerated approval pathways for vaccines if an immunological marker can be established as a mediator of a…

Methodology · Statistics 2021-03-05 David Benkeser , Iván Díaz , Jialu Ran

Identification of standard mediated effects such as the natural indirect effect relies on heavy causal assumptions. By circumventing such assumptions, so-called randomized interventional indirect effects have gained popularity in the…

Methodology · Statistics 2023-10-03 Caleb H. Miles

This paper investigates the case of interference, when a unit's treatment also affects other units' outcome. When interference is at work, policy evaluation mostly relies on the use of randomized experiments under cluster interference and…

Methodology · Statistics 2023-06-13 Laura Forastiere , Davide Del Prete , Valerio Leone Sciabolazza

We examine study designs for extending (generalizing or transporting) causal inferences from a randomized trial to a target population. Specifically, we consider nested trial designs, where randomized individuals are nested within a sample…

The success of a vaccination program is crucially dependent on its adoption by a critical fraction of the population, as the resulting herd immunity prevents future outbreaks of an epidemic. However, the effectiveness of a campaign can…

Physics and Society · Physics 2019-07-02 Anupama Sharma , Shakti N. Menon , V. Sasidevan , Sitabhra Sinha

Survey data are often collected under multistage sampling designs where units are binned to clusters that are sampled in a first stage. The unit-indexed population variables of interest are typically dependent within cluster. We propose a…

Methodology · Statistics 2021-08-26 Luis G. Leon-Novelo , Terrance D. Savitsky

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