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The treatment effect in a specific subgroup is often of interest in randomized clinical trials. When the subgroup is characterized by the absence of certain post-randomization events, a naive analysis on the subset of patients without these…

应用统计 · 统计学 2018-09-12 Baldur P. Magnusson , Heinz Schmidli , Nicolas Rouyrre , Daniel O. Scharfstein

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying…

统计方法学 · 统计学 2024-07-18 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

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…

统计理论 · 数学 2007-06-13 Donald B. Rubin

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…

应用统计 · 统计学 2014-01-13 Alessandra Mattei , Fan Li , Fabrizia Mealli

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

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…

统计计算 · 统计学 2023-04-07 Bo Liu , Fan Li

Propensity score weighting approaches have been widely implemented in clinical research to estimate the effects of a treatment or exposure while mitigating the risk of confounding in the absence of random assignment. In practice, when…

统计方法学 · 统计学 2026-04-17 Emma K. Mackay , Amol A. Verma , Fahad Razak , Surain B. Roberts

Basic propensity score methodology is designed to balance multivariate pre-treatment covariates when comparing one active treatment with one control treatment. Practical settings often involve comparing more than two treatments, where more…

统计方法学 · 统计学 2021-11-09 Shasha Han , Donald Rubin

It has recently become popular to define treatment effects for subsets of the target population characterized by variables not observable at the time a treatment decision is made. Characterizing and estimating such treatment effects is…

统计理论 · 数学 2007-08-30 Marshall M. Joffe , Dylan Small , Chi-Yuan Hsu

Causal inference concerns not only the average effect of the treatment on the outcome but also the underlying mechanism through an intermediate variable of interest. Principal stratification characterizes such a mechanism by targeting…

统计方法学 · 统计学 2022-03-29 Zhichao Jiang , Shu Yang , Peng Ding

Although randomized controlled trials have long been regarded as the ``gold standard'' for evaluating treatment effects, there is no natural prevention from post-treatment events. For example, non-compliance makes the actual treatment…

统计方法学 · 统计学 2025-04-25 Qinqing Liu , Xiang Peng , Tao Zhang , Yuhao Deng

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

统计方法学 · 统计学 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

Interim analyses for group-sequential decision making are prevalent in clinical trials. Methodology is well established and has been routinely implemented over the last decades. Still, confusions and uncertainties on aspects of how to…

Motivated by a potential-outcomes perspective, the idea of principal stratification has been widely recognized for its relevance in settings susceptible to posttreatment selection bias such as randomized clinical trials where treatment…

应用统计 · 统计学 2011-11-08 Corwin M. Zigler , Thomas R. Belin

Clinical trials usually involve sequential patient entry. When designing a clinical trial, it is often desirable to include a provision for interim analyses of accumulating data with the potential for stopping the trial early. We review…

统计方法学 · 统计学 2023-03-13 Tianjian Zhou , Yuan Ji

In many causal studies, outcomes are censored by death, in the sense that they are neither observed nor defined for units who die. In such studies, the focus is usually on the stratum of always survivors up to a single fixed time s.…

统计方法学 · 统计学 2024-01-02 Giulio Grossi , Marco Mariani , Alessandra Mattei , Fabrizia Mealli

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

统计方法学 · 统计学 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

There is wide agreement on the importance of implementation data from randomized effectiveness studies in behavioral science; however, there are few methods available to incorporate these data into causal models, especially when they are…

统计方法学 · 统计学 2024-05-17 Sooyong Lee , Adam C Sales , Hyeon-Ah Kang , Tiffany A. Whittaker

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…

统计方法学 · 统计学 2017-08-17 Luke Miratrix , Jane Furey , Avi Feller , Todd Grindal , Lindsay C. Page

The principal stratification has become a popular tool to address a broad class of causal inference questions, particularly in dealing with non-compliance and truncation-by-death problems. The causal effects within principal strata which…

统计方法学 · 统计学 2022-06-20 Shanshan Luo , Wei Li , Wang Miao , Yangbo He