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Pragmatic trials increasingly define outcomes using real-world data such as electronic health records, where assessments are collected during routine care rather than at fixed timepoints. Consequently, these uncontrolled assessments may be…

This paper considers conducting inference about the effect of a treatment (or exposure) on an outcome of interest. In the ideal setting where treatment is assigned randomly, under certain assumptions the treatment effect is identifiable…

Methodology · Statistics 2015-03-06 Amy Richardson , Michael G. Hudgens , Peter B. Gilbert , Jason P. Fine

In clinical trials with recurrent events, such as repeated hospitalizations terminating with death, it is important to consider the patient events overall history for a thorough assessment of treatment effects. The occurrence of fewer…

Methodology · Statistics 2025-09-08 Alessandra Ragni , Torben Martinussen , Thomas Scheike

Causal mediation analysis provides techniques for defining and estimating effects that may be endowed with mechanistic interpretations. With many scientific investigations seeking to address mechanistic questions, causal direct and indirect…

No unmeasured confounding is often assumed in estimating treatment effects in observational data when using approaches such as propensity scores and inverse probability weighting. However, in many such studies due to the limitation of the…

Applications · Statistics 2019-08-06 Rong Huang , Ronghui Xu , Parambir S. Dulai

Difference-in-differences (DID) approaches are widely used for estimating causal effects with observational data before and after an intervention. DID traditionally estimates the average treatment effect among the treated after making a…

Methodology · Statistics 2025-06-24 Julia C. Thome , Andrew J. Spieker , Peter F. Rebeiro , Chun Li , Tong Li , Bryan E. Shepherd

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

Methodology · Statistics 2025-09-29 Lan Wen , Aaron L Sarvet

A typical random effects meta-analysis of odds-ratios assumes binomially distributed numbers of events in a treatment and control group and requires the proportion of deaths to be extracted from published papers. This data is often not…

Methodology · Statistics 2014-10-13 Shemra Rizzo , Robert E. Weiss

In randomized experiments, interactions between units might generate a treatment diffusion process. This is common when the treatment of interest is an actual object or product that can be shared among peers (e.g., flyers, booklets,…

Methodology · Statistics 2021-09-17 Costanza Tortú , Irene Crimaldi , Fabrizia Mealli , Laura Forastiere

The use of instrumental variables for estimating the effect of an exposure on an outcome is popular in econometrics, and increasingly so in epidemiology. This increasing popularity may be attributed to the natural occurrence of instrumental…

Methodology · Statistics 2016-08-03 T. Martinussen , S. Vansteelandt , E. J. Tchetgen Tchetgen , D. M. Zucker

We consider time to treatment initiation. This can commonly occur in preventive medicine, such as disease screening and vaccination; it can also occur with non-fatal health conditions such as HIV infection without the onset of AIDS. While…

Methodology · Statistics 2026-05-29 Zhichen Zhao , Andrew Ying , Ronghui Xu

In longitudinal studies where units are embedded in space or a social network, interference may arise, meaning that a unit's outcome can depend on treatment histories of others. The presence of interference poses significant challenges for…

Methodology · Statistics 2025-08-26 Ye Wang , Michael Jetsupphasuk

Longitudinal modified treatment policies (LMTP) have been recently developed as a novel method to define and estimate causal parameters that depend on the natural value of treatment. LMTPs represent an important advancement in causal…

Methodology · Statistics 2025-02-06 Iván Díaz , Katherine L Hoffman , Nima S. Hejazi

Researchers are often interested in treatment effects on outcomes that are only defined conditional on a post-treatment event status. For example, in a study of the effect of different cancer treatments on quality of life at end of…

Two problems that arise in making causal inferences for non-mortality outcomes such as bronchopulmonary dysplasia (BPD) are unmeasured confounding and censoring by death, i.e., the outcome is only observed when subjects survive. In…

Methodology · Statistics 2018-02-20 Kwonsang Lee , Scott A. Lorch , Dylan S. Small

Post-randomization events, also known as intercurrent events, such as treatment noncompliance and censoring due to a terminal event, are common in clinical trials. Principal stratification is a framework for causal inference in the presence…

Methodology · Statistics 2023-01-19 Bo Liu , Lisa Wruck , Fan Li

In causal analysis, understanding the causal mechanisms through which an intervention or treatment affects an outcome is often of central interest. We propose a test to evaluate (i) whether the causal effect of a treatment that is randomly…

Econometrics · Economics 2026-03-05 Martin Huber , Kevin Kloiber , Lukáš Lafférs

Defining and identifying causal intervention effects for transmissible infectious disease outcomes is challenging because a treatment -- such as a vaccine -- given to one individual may affect the infection outcomes of others.…

Applications · Statistics 2019-12-11 Xiaoxuan Cai , Wen Wei Loh , Forrest W. Crawford

When comparing time-varying treatments in a non-randomised setting, one must often correct for time-dependent confounders that influence treatment choice over time and that are themselves influenced by treatment. We present a new two step…

Methodology · Statistics 2016-10-06 J. M. Gran , R. Hoff , K. Røysland , B. Ledergerber , J. Young , O. O. Aalen

We review standard mediation assumptions as they apply to identifying antibody effects in a randomized vaccine trial and propose new study designs to allow identification of an estimand that was previously unidentifiable. For these…

Methodology · Statistics 2024-06-11 Michael P. Fay , Dean A. Follmann