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It has been argued for many years that models used to analyze data from crossover designs are not appropriate when simple carryover effects are assumed. Furthermore, a statistical model that could estimate complex carry-over effects in…

Methodology · Statistics 2025-08-22 N. A. Cruz , K. Mylona , O. O. Melo

While the HVTN 505 trial showed no overall efficacy of the tested vaccine to prevent HIV infection over placebo, previous studies, biological theories, and the finding that immune response markers strongly correlated with infection in…

Applications · Statistics 2018-11-12 Peter B. Gilbert , Bryan S. Blette , Bryan E. Shepherd , Michael G. Hudgens

Staggered treatment adoption arises in the evaluation of policy impact and implementation in many settings, including both randomized stepped-wedge trials and non-randomized quasi-experiments with panel data. In both settings, getting an…

Methodology · Statistics 2024-10-14 Lee Kennedy-Shaffer

This paper considers the identification of treatment effects on conditional transition probabilities. We show that even under random assignment only the instantaneous average treatment effect is point identified. Since treated and control…

Econometrics · Economics 2017-09-27 Johan Vikström , Geert Ridder , Martin Weidner

Principal stratification is a causal framework to analyze randomized experiments with a post-treatment variable between the treatment and endpoint variables. Because the principal strata defined by the potential outcomes of the…

Statistics Theory · Mathematics 2015-07-22 Zhichao Jiang , Peng Ding , Zhi Geng

Path-specific effects are a broad class of mediated effects from an exposure to an outcome via one or more causal pathways with respect to some subset of intermediate variables. The majority of the literature concerning estimation of…

Generalizing treatment effects from a randomized trial to a target population requires the assumption that potential outcome distributions are invariant across populations after conditioning on observed covariates. This assumption fails…

Methodology · Statistics 2026-04-16 Amir Asiaee , Samhita Pal , Cole Beck , Jared D. Huling

In sequential causal inference, one estimates the causal net effect of treatment in treatment sequence on an outcome after last treatment in the presence of time-dependent covariates between treatments, improves the estimation by the…

Methodology · Statistics 2014-11-18 Li Yin , Xiaoqin Wang

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…

Biomarker measurements can be relatively easy and quick to obtain and they are useful to investigate whether a compound works as intended on a mechanistic, pharmacological level. In some situations, it is realistic to assume that patients,…

Methodology · Statistics 2018-06-26 Björn Bornkamp , Georgina Bermann

This paper considers the problem of design-based inference for the average treatment effect in finely stratified experiments. Here, by "design-based'' we mean that the only source of uncertainty stems from the randomness in treatment…

Econometrics · Economics 2025-05-08 Yuehao Bai , Xun Huang , Joseph P. Romano , Azeem M. Shaikh , Max Tabord-Meehan

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

Semiparametric inference on average causal effects from observational data is based on assumptions yielding identification of the effects. In practice, several distinct identifying assumptions may be plausible; an analyst has to make a…

Methodology · Statistics 2025-10-07 Tetiana Gorbach , Xavier de Luna , Juha Karvanen , Ingeborg Waernbaum

Principal stratification (PS) is a commonly used approach for understanding the mechanisms through which a treatment affects an outcome. The goal of this work is to extend the PS framework to studies with continuous treatments, which…

Methodology · Statistics 2025-05-20 Joseph Antonelli , Minxuan Wu , Fabrizia Mealli , Brenden Beck , Alessandra Mattei

Randomized controlled trials are the gold standard for evaluating the efficacy of an intervention. However, there is often a trade-off between selecting the most scientifically relevant primary endpoint versus a less relevant, but more…

Methodology · Statistics 2026-01-12 Jack M. Wolf , Joseph S. Koopmeiners , David M. Vock

Inferring causal effects of continuous-valued treatments from observational data is a crucial task promising to better inform policy- and decision-makers. A critical assumption needed to identify these effects is that all confounding…

Principal stratification is essential for revealing causal mechanisms involving post-treatment intermediate variables, in real-world applications like surrogate marker evaluation. Principal stratification analysis with continuous…

Methodology · Statistics 2025-01-31 Yichi Zhang , Shu Yang

In most real-world systems units are interconnected and can be represented as networks consisting of nodes and edges. For instance, in social systems individuals can have social ties, family or financial relationships. In settings where…

Methodology · Statistics 2018-07-31 Laura Forastiere , Fabrizia Mealli , Albert Wu , Edoardo Airoldi

The case-crossover design (Maclure, 1991) is widely used in epidemiology and other fields to study causal effects of transient treatments on acute outcomes. However, its validity and causal interpretation have only been justified under…

Methodology · Statistics 2021-11-22 Zach Shahn , Miguel A. Hernan , James M. Robins

Estimating treatment effects for subgroups defined by post-treatment behavior (i.e., estimating causal effects in a principal stratification framework) can be technically challenging and heavily reliant on strong assumptions. We investigate…

Methodology · Statistics 2017-08-17 Luke Miratrix , Jane Furey , Avi Feller , Todd Grindal , Lindsay C. Page