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Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

Methodology · Statistics 2026-03-31 Zhe Chen , Xinran Li

When estimating causal effects, it is important to assess external validity, i.e., determine how useful a given study is to inform a practical question for a specific target population. One challenge is that the covariate distribution in…

Methodology · Statistics 2025-01-03 Zhenghao Zeng , Edward H. Kennedy , Lisa M. Bodnar , Ashley I. Naimi

The accuracy of probability distributions inferred using machine-learning algorithms heavily depends on data availability and quality. In practical applications it is therefore fundamental to investigate the robustness of a statistical…

Machine Learning · Statistics 2018-10-01 Christiane Goergen , Manuele Leonelli

Estimation of local average treatment effects in randomized trials typically requires an assumption known as the exclusion restriction in cases where we are unwilling to rule out unmeasured confounding. Under this assumption, any benefit…

Methodology · Statistics 2020-08-17 Andrew J. Spieker , Robert A. Greevy , Lyndsay A. Nelson , Lindsay S. Mayberry

The estimation of the potential impact fraction (including the population attributable fraction) with continuous exposure data frequently relies on strong distributional assumptions. However, these assumptions are often violated if the…

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

Methodology · Statistics 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

Randomized trials are widely considered as the gold standard for evaluating the effects of decision policies. Trial data is, however, drawn from a population which may differ from the intended target population and this raises a problem of…

Methodology · Statistics 2024-10-30 Sofia Ek , Dave Zachariah

Comparative effectiveness evidence from randomized trials may not be directly generalizable to a target population of substantive interest when, as in most cases, trial participants are not randomly sampled from the target population.…

Methodology · Statistics 2022-04-12 Fan Li , Ashley L. Buchanan , Stephen R. Cole

Many modern causal questions ask how treatments affect complex outcomes that are measured using wearable devices and sensors. Current analysis approaches require summarizing these data into scalar statistics (e.g., the mean), but these…

Machine Learning · Computer Science 2024-03-22 Srikar Katta , Harsh Parikh , Cynthia Rudin , Alexander Volfovsky

The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard…

Methodology · Statistics 2026-01-08 Edoardo Efrem Gervasoni , Liesbet De Bus , Stijn Vansteelandt , Oliver Dukes

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

In the context of having an instrumental variable, the standard practice in causal inference begins by targeting an effect of interest and proceeds by formulating assumptions enabling its identification. We turn this around by adhering to…

Statistics Theory · Mathematics 2026-05-25 Carlos García Meixide , Mark J. van der Laan

Causal mediation analysis is a useful tool for epidemiological research, but it has been criticized for relying on a "cross-world" independence assumption that is empirically difficult to verify and problematic to justify based on…

Methodology · Statistics 2021-09-02 Ryan M. Andrews , Vanessa Didelez

In the absence of a randomized experiment, a key assumption for drawing causal inference about treatment effects is the ignorable treatment assignment. Violations of the ignorability assumption may lead to biased treatment effect estimates.…

Methodology · Statistics 2021-08-17 Liangyuan Hu , Jungang Zou , Chenyang Gu , Jiayi Ji , Michael Lopez , Minal Kale

To generalize inferences from a randomized trial to the target population of all trial-eligible individuals, investigators can use nested trial designs, where the randomized individuals are nested within a cohort of trial-eligible…

Sensitivity analysis informs causal inference by assessing the sensitivity of conclusions to departures from assumptions. The consistency assumption states that there are no hidden versions of treatment and that the outcome arising…

Methodology · Statistics 2025-12-29 Brian Knaeble , Qinyun Lin , Erich Kummerfeld , Kenneth A. Frank

Causal inference relies on the untestable assumption of no unmeasured confounding. Sensitivity analysis can be used to quantify the impact of unmeasured confounding on causal estimates. Among sensitivity analysis methods proposed in the…

Methodology · Statistics 2026-03-12 Yushu Zou , Liangyuan Hu , Amanda Ricciuto , Mark Deneau , Kuan Liu

The generalized g-formula can be used to estimate the probability of survival under a sustained treatment strategy. When treatment strategies are deterministic, estimators derived from the so-called efficient influence function (EIF) for…

Methodology · Statistics 2022-02-10 Lan Wen , Julia Marcus , Jessica Young

Causal inference from observational data can be viewed as a missing data problem arising from a hypothetical population-scale randomized trial matched to the observational study. This links a target trial protocol with a corresponding…

Methodology · Statistics 2022-07-27 Andrew Yiu , Edwin Fong , Stephen Walker , Chris Holmes

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