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Knowing the effect of an intervention is critical for human decision-making, but current approaches for causal effect estimation rely on manual data collection and structuring, regardless of the causal assumptions. This increases both the…

Machine Learning · Computer Science 2024-10-29 Nikita Dhawan , Leonardo Cotta , Karen Ullrich , Rahul G. Krishnan , Chris J. Maddison

We develop methodology for valid inference after variable selection in logistic regression when the responses are partially observed, that is, when one observes a set of error-prone testing outcomes instead of the true values of the…

Methodology · Statistics 2025-04-17 Qinyan Shen , Karl Gregory , Xianzheng Huang

A new method is proposed to perform joint analysis of longitudinal and cross-sectional growth data. Clustering is first performed to group similar subjects in cross-sectional data to form a pseudo longitudinal data set, then the pseudo…

Methodology · Statistics 2025-09-24 Long Chen , Ji Chen , Yingchun Zhou

In linear models, omitting a covariate that is orthogonal to covariates in the model does not result in biased coefficient estimation. This in general does not hold for longitudinal data, where additional assumptions are needed to get…

Statistics Theory · Mathematics 2023-05-30 Zhuowei Sun , Hongyuan Cao , Li Chen , Jason P. Fine

Covariate adjustment is an important tool in the analysis of randomized clinical trials and observational studies. It can be used to increase efficiency and thus power, and to reduce possible bias. While most statistical tests in randomized…

Methodology · Statistics 2011-08-03 Xiaoru Wu , Zhiliang Ying

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

Computation · Statistics 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

In confirmatory clinical trials, survival outcomes are frequently studied and interim analyses for efficacy and/or futility are often desirable. Methods such as the log rank test and Cox regression model are commonly used to compare…

Methodology · Statistics 2024-03-27 Peter Zhang , Brent Logan , Michael Martens

Standard methods for estimating average causal effects require complete observations of the exposure and confounders. In observational studies, however, missing data are ubiquitous. Motivated by a study on the effect of prescription opioids…

Methodology · Statistics 2025-06-30 Lan Wen , Glen McGee

It is important to estimate the local average treatment effect (LATE) when compliance with a treatment assignment is incomplete. The previously proposed methods for LATE estimation required all relevant variables to be jointly observed in a…

Machine Learning · Statistics 2022-03-22 Kazuhiko Shinoda , Takahiro Hoshino

Estimating the long-term effects of treatments is of interest in many fields. A common challenge in estimating such treatment effects is that long-term outcomes are unobserved in the time frame needed to make policy decisions. One approach…

Methodology · Statistics 2024-08-23 Susan Athey , Raj Chetty , Guido Imbens , Hyunseung Kang

While analysing time-to-event data, it is possible that a certain fraction of subjects will never experience the event of interest and they are said to be cured. When this feature of survival models is taken into account, the models are…

Methodology · Statistics 2020-01-27 Khandoker Akib Mohammad , Yuichi Hirose , Budhi Surya , Yuan Yao

Causal mediation analysis of observational data is an important tool for investigating the potential causal effects of medications on disease-related risk factors, and on time-to-death (or disease progression) through these risk factors.…

We study estimation of and inference for the average causal effect of treating every member of a population, as opposed to none, using an experiment that treats only some. Considering settings where spillovers can occur between any pair of…

Econometrics · Economics 2026-01-29 Stefan Faridani , Paul Niehaus

It is often of interest in observational studies to measure the causal effect of a treatment on time-to-event outcomes. In a medical setting, observational studies commonly involve patients who initiate medication therapy and others who do…

Methodology · Statistics 2019-10-07 Reagan Mozer , Mark E. Glickman

Semi-parametric methods are often used for the estimation of intervention effects on correlated outcomes in cluster-randomized trials (CRTs). When outcomes are missing at random (MAR), Inverse Probability Weighted (IPW) methods…

Methodology · Statistics 2016-01-27 Melanie Prague , Rui Wang , Alisa Stephens , Eric Tchetgen Tchetgen , Victor DeGruttola

Analysis of longitudinal Electronic Health Record (EHR) data is an important goal for precision medicine. Difficulty in applying Machine Learning (ML) methods, either predictive or unsupervised, stems in part from the heterogeneity and…

Quantitative Methods · Quantitative Biology 2022-04-18 Alan D. Kaplan , Uttara Tipnis , Jean C. Beckham , Nathan A. Kimbrel , David W. Oslin , Benjamin H. McMahon

Uncertainty quantification of causal effects is crucial for safety-critical applications such as personalized medicine. A powerful approach for this is conformal prediction, which has several practical benefits due to model-agnostic…

Machine Learning · Computer Science 2026-02-04 Maresa Schröder , Dennis Frauen , Jonas Schweisthal , Konstantin Heß , Valentyn Melnychuk , Stefan Feuerriegel

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Medical advances have increased cancer survival rates and the possibility of finding a cure. Hence, it is crucial to evaluate the impact of treatments both in terms of cure and prolongation of survival. To achieve this, we may use a Cox…

Methodology · Statistics 2024-12-31 Marta Cipriani , Marta Fiocco , Marco Alfò , Maria Quelhas , Eni Musta