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Related papers: Estimands in Real-World Evidence Studies

200 papers

A randomized trial allows estimation of the causal effect of an intervention compared to a control in the overall population and in subpopulations defined by baseline characteristics. Often, however, clinical questions also arise regarding…

Ordinal outcomes are common in clinical settings where they often represent increasing levels of disease progression or different levels of functional impairment. Such outcomes can characterize differences in meaningful patient health…

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…

Estimating a causal query from observational data is an essential task in the analysis of biomolecular networks. Estimation takes as input a network topology, a query estimation method, and observational measurements on the network…

Contrasting marginal counterfactual survival curves across treatment arms is an effective and popular approach for inferring the causal effect of an intervention on a right-censored time-to-event outcome. A key challenge to drawing such…

Methodology · Statistics 2022-04-29 Andrew Ying , Yifan Cui , Eric J. Tchetgen Tchetgen

Propensity score weighting is an important tool for comparative effectiveness research.Besides the inverse probability of treatment weights (IPW), recent development has introduced a general class of balancing weights, corresponding to…

Methodology · Statistics 2022-09-05 Tianhui Zhou , Guangyu Tong , Fan Li , Laine E. Thomas , Fan Li

The health effects of environmental exposures have been studied for decades, typically using standard regression models to assess exposure-outcome associations found in observational non-experimental data. We propose and illustrate a…

Applications · Statistics 2017-09-20 Marie-Abele C. Bind , Donald B. Rubin

Statistical samples, in order to be representative, have to be drawn from a population in a random and unbiased way. Nevertheless, it is common practice in the field of model-based diagnosis to make estimations from (biased) best-first…

Artificial Intelligence · Computer Science 2022-08-05 Patrick Rodler , Fatima Elichanova

Clinical decision-making is a feedback system where risk estimates influence treatment, which in turn changes disease trajectories, and both shape clinicians' measurement practices. Static prediction often fails clinically: models trained…

Artificial Intelligence · Computer Science 2026-05-19 Pujun Feng , Xiaoyu Guo , Seyed Ehsan Saffari , Min Hun Lee , Siew-Kei Lam , Erik Cambria , Xibin Sun , Yangtao Zhou , Tong Yang , Xiaoyu Zhang , Tao Tan , Yue Sun , Bin Cui

A stepped wedge design is a unidirectional crossover design where clusters are randomized to distinct treatment sequences. While model-based analysis of stepped wedge designs is standard practice to evaluate treatment effects accounting for…

Methodology · Statistics 2024-09-13 Bingkai Wang , Xueqi Wang , Fan Li

Selection bias is a major obstacle toward valid causal inference in epidemiology. Over the past decade, several graphical rules based on causal diagrams have been proposed as the sufficient identification conditions for addressing selection…

Methodology · Statistics 2025-12-18 Yichi Zhang , Haidong Lu

Strong experimental papers in electrical and computer engineering and computer science (ECE/CS), especially in systems, networking, and applied machine learning, rest on more than a single impressive number. They rest on a chain of design,…

Methodology · Statistics 2026-05-04 Bhaskar Krishnamachari

Rehabilitation research focuses on determining the components of a treatment intervention, the mechanism of how these components lead to recovery and rehabilitation, and ultimately the optimal intervention strategies to maximize patients'…

We define causal estimands for experiments on single time series, extending the potential outcome framework to dealing with temporal data. Our approach allows the estimation of some of these estimands and exact randomization based p-values…

Methodology · Statistics 2020-02-17 Iavor Bojinov , Neil Shephard

In this review, we present econometric and statistical methods for analyzing randomized experiments. For basic experiments we stress randomization-based inference as opposed to sampling-based inference. In randomization-based inference,…

Methodology · Statistics 2017-10-26 Susan Athey , Guido Imbens

The weighted average treatment effect (WATE) is a causal measure for the comparison of interventions in a specific target population, which may be different from the population where data are sampled from. For instance, when the goal is to…

Methodology · Statistics 2018-04-17 Yebin Tao , Haoda Fu

Many Artificial Intelligence systems depend on the agent's updating its beliefs about the world on the basis of experience. Experiments constitute one type of experience, so scientific methodology offers a natural environment for examining…

Artificial Intelligence · Computer Science 2013-04-08 Harold P. Lehmann

Human decision-making heavily relies on active sensing, a well-documented cognitive behaviour for evidence gathering to accommodate ever-changing environments. However, its operational mechanism in the real world remains non-trivial.…

Artificial Intelligence · Computer Science 2026-01-09 Hongliang Lu , Yunmeng Liu , Junjie Yang

In studies of educational production functions or intergenerational mobility, it is common to transform the key variables into percentile ranks. Yet, it remains unclear what the regression coefficient estimates with ranks of the outcome or…

Econometrics · Economics 2024-06-11 Lihua Lei

For obtaining causal inferences that are objective, and therefore have the best chance of revealing scientific truths, carefully designed and executed randomized experiments are generally considered to be the gold standard. Observational…

Applications · Statistics 2008-11-12 Donald B. Rubin