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Prescriptive process monitoring methods seek to optimize the performance of business processes by triggering interventions at runtime, thereby increasing the probability of positive case outcomes. These interventions are triggered according…

Artificial Intelligence · Computer Science 2025-05-20 Mahmoud Shoush , Marlon Dumas

A growing number of methods aim to assess the challenging question of treatment effect variation in observational studies. This special section of "Observational Studies" reports the results of a workshop conducted at the 2018 Atlantic…

Methodology · Statistics 2019-09-17 Carlos Carvalho , Avi Feller , Jared Murray , Spencer Woody , David Yeager

Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…

Applications · Statistics 2026-05-26 Yifei Xu , Hwiyoung Lee , Zhenyao Ye , Yezhi Pan , Jingsong Zhou , Yun Yang , Chixiang Chen , Shuo Chen

While randomised controlled trials (RCTs) are the gold standard for estimating causal treatment effects, their limited sample sizes and restrictive criteria make it difficult to extrapolate to a broader population. Observational data, while…

Methodology · Statistics 2025-09-09 Stephanie Riley , Ricardo Silva , Matthew Sperrin

Generalization methods offer a powerful solution to one of the key drawbacks of randomized controlled trials (RCTs): their limited representativeness. By enabling the transport of treatment effect estimates to target populations subject to…

Methodology · Statistics 2025-05-20 Ahmed Boughdiri , Clément Berenfeld , Julie Josse , Erwan Scornet

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

Methodology · Statistics 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

Despite their cost, randomized controlled trials (RCTs) are widely regarded as gold-standard evidence in disciplines ranging from social science to medicine. In recent decades, researchers have increasingly sought to reduce the resource…

Methodology · Statistics 2026-02-24 Guilherme Duarte

Combining observational and experimental data for causal inference can improve treatment effect estimation. However, many observational data sets cannot be released due to data privacy considerations, so one researcher may not have access…

Methodology · Statistics 2024-08-26 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

In randomized controlled trials (RCTs) that focus on time-to-event outcomes, intercurrent events can arise in two ways: as semi-competing events, which modify the hazard of the primary outcome events, or as competing events, which make the…

Methodology · Statistics 2026-05-26 Yuhao Deng , Shasha Han , Xiao-Hua Zhou

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

Methodology · Statistics 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

Observational studies are valuable tools for inferring causal effects in the absence of controlled experiments. However, these studies may be biased due to the presence of some relevant, unmeasured set of covariates. One approach to…

Methodology · Statistics 2026-02-17 William Bekerman , Abhinandan Dalal , Carlo del Ninno , Dylan S. Small

When evaluating the effectiveness of a drug, a Randomized Controlled Trial (RCT) is often considered the gold standard due to its perfect randomization. While RCT assures strong internal validity, its restricted external validity poses…

Applications · Statistics 2024-06-07 Kuan Jiang , Xin-xing Lai , Shu Yang , Ying Gao , Xiao-Hua Zhou

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…

A further understanding of cause and effect within observational data is critical across many domains, such as economics, health care, public policy, web mining, online advertising, and marketing campaigns. Although significant advances…

Machine Learning · Computer Science 2023-04-11 Zhixuan Chu , Sheng Li

Observational studies often benefit from an abundance of observational units. This can lead to studies that -- while challenged by issues of internal validity -- have inferences derived from sample sizes substantially larger than randomized…

Methodology · Statistics 2020-08-24 Rachael C. Aikens , Dylan Greaves , Michael Baiocchi

Randomized controlled trials (RCTs) provide strong internal validity compared with observational studies. However, selection bias threatens the external validity of randomized trials. Thus, RCT results may not apply to either broad public…

Methodology · Statistics 2017-04-26 Ziyue Chen , Eloise Kaizar

Intercurrent (post-treatment) events occur frequently in randomized trials, and investigators often express interest in treatment effects that suitably take account of these events. A naive conditioning on intercurrent events does not have…

Methodology · Statistics 2021-11-17 Mats J. Stensrud , Oliver Dukes

Causal effects are commonly defined as comparisons of the potential outcomes under treatment and control, but this definition is threatened by the possibility that the treatment or control condition is not well-defined, existing instead in…

Methodology · Statistics 2019-04-26 Raiden B. Hasegawa , Sameer K. Deshpande , Dylan S. Small , Paul R. Rosenbaum

A potential causal relationship between head injuries sustained by NFL players and later-life neurological decline may have broad implications for participants in youth and high school football programs. However, brain trauma risk at the…

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…