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The propensity score is a common tool for estimating the causal effect of a binary treatment in observational data. In this setting, matching, subclassification, imputation, or inverse probability weighting on the propensity score can…

Methodology · Statistics 2018-01-03 Michael J Lopez , Roee Gutman

The proximal causal inference framework enables the identification and estimation of causal effects in the presence of unmeasured confounding by leveraging two disjoint sets of observed strong proxies: negative control treatments and…

Methodology · Statistics 2025-12-16 Antonio Olivas-Martinez , Peter B. Gilbert , Andrea Rotnitzky

Principal stratification analysis evaluates how causal effects of a treatment on a primary outcome vary across strata of units defined by their treatment effect on some intermediate quantity. This endeavor is substantially challenged when…

Methodology · Statistics 2024-03-21 Chanmin Kim , Corwin Zigler

Effective therapy decisions require models that predict the individual response to treatment. This is challenging since the progression of disease and response to treatment vary substantially across patients. Here, we propose to learn a…

Computer Vision and Pattern Recognition · Computer Science 2025-09-19 Ivana Janíčková , Yen Y. Tan , Thomas H. Helbich , Konstantin Miloserdov , Zsuzsanna Bago-Horvath , Ulrike Heber , Georg Langs

Paired cluster-randomized experiments (pCRTs) are common across many disciplines because there is often natural clustering of individuals, and paired randomization can help balance baseline covariates to improve experimental precision.…

Methodology · Statistics 2024-07-03 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

When making treatment selection decisions, it is essential to include a causal effect estimation analysis to compare potential outcomes under different treatments or controls, assisting in optimal selection. However, merely estimating…

Machine Learning · Statistics 2024-10-08 Sherly Alfonso-Sánchez , Kristina P. Sendova , Cristián Bravo

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

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

We study estimation and inference for heterogeneous principal causal effects with binary treatments and binary intermediate variables. Principal causal effects are subgroup effects within strata defined by potential values of an…

Methodology · Statistics 2026-03-11 Rui Zhang , Charles R. Doss , Jared D. Huling

Post-treatment confounding is a common problem in causal inference, including special cases of noncompliance, truncation by death, surrogate endpoint, etc. Principal stratification (Frangakis and Rubin 2002) is a general framework for…

Computation · Statistics 2023-04-07 Bo Liu , Fan Li

It is common in medical studies that the outcome of interest is truncated by death, meaning that a subject has died before the outcome could be measured. In this case, restricted analysis among survivors may be subject to selection bias.…

Methodology · Statistics 2018-04-25 Linbo Wang , Xiao-Hua Zhou , Thomas S. Richardson

The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent guidelines and initiatives of regulatory agencies. Among the factors contributing to modernized drug development and…

In clinical trials involving both mortality and morbidity, an active treatment can influence the observed risk of the first non-fatal event either directly, through its effect on the underlying non-fatal event process, or indirectly,…

Methodology · Statistics 2025-11-19 Jiren Sun , Thomas D. Cook

The sufficient cause framework has been used for decades to improve our understanding of both basic and more complex causal concepts in epidemiology, such as mediation and interaction. Here, we make use of this framework to provide a…

Methodology · Statistics 2026-04-08 Bronner P. Gonçalves , Eiji Yamamoto , Etsuji Suzuki

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

Many interventions are both beneficial to initiate and harmful to stop. Traditionally, to determine whether to deploy that intervention in a time-limited way depends on if, on average, the increase in the benefits of starting it outweigh…

The causal effect of a randomized job training program, the JOBS II study, on trainees' depression is evaluated. Principal stratification is used to deal with noncompliance to the assigned treatment. Due to the latent nature of the…

Applications · Statistics 2014-01-13 Alessandra Mattei , Fan Li , Fabrizia Mealli

Breast cancer patients may experience relapse or death after surgery during the follow-up period, leading to dependent censoring of relapse. This phenomenon, known as semi-competing risk, imposes challenges in analyzing treatment effects on…

Methodology · Statistics 2024-07-03 Tonghui Yu , Mengjiao Peng , Yifan Cui , Elynn Chen , Chixiang Chen

In clinical trials, it is often of interest to understand the principal causal effect (PCE), the average treatment effect for a principal stratum (a subset of patients defined by the potential outcomes of one or more post-baseline…

Methodology · Statistics 2022-10-06 Yongming Qu , Ilya Lipkovich , Stephen J. Ruberg

Intercurrent events, common in clinical trials and observational studies, affect the existence or interpretation of final outcomes. Principal stratification addresses this challenge by defining local average treatment effect estimands…

Methodology · Statistics 2025-09-22 Jiaqi Tong , Brennan Kahan , Michael O. Harhay , Fan Li