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The National Research Council panel on prevention and treatment of missing data in clinical trials recommends that primary analysis methods are carefully selected before appropriate sensitivity analysis methods can be chosen. In this paper,…

Applications · Statistics 2021-05-10 Abdul-Karim Iddrisu , Abukari Alhassan

In this paper, a methodology is proposed that enables to analyze the sensitivity of the outcome of a therapy to unavoidable high dispersion of the patient specific parameters on one hand and to the choice of the parameters that define the…

Systems and Control · Electrical Eng. & Systems 2022-05-17 Mazen Alamir

Objective: Researchers often use model-based multiple imputation to handle missing at random data to minimize bias while making the best use of all available data. However, there are sometimes constraints within the data that make…

Methodology · Statistics 2020-11-03 Chinchin Wang , Tyrel Stokes , Russell Steele , Niels Wedderkopp , Ian Shrier

This paper provides clear and practical guidance on the specification of imputation models when multiple imputation is used in conjunction with doubly robust estimation methods for causal inference. Through theoretical arguments and…

Methodology · Statistics 2025-12-19 Lucy D'Agostino McGowan

No unmeasured confounding is often assumed in estimating treatment effects in observational data when using approaches such as propensity scores and inverse probability weighting. However, in many such studies due to the limitation of the…

Applications · Statistics 2019-08-06 Rong Huang , Ronghui Xu , Parambir S. Dulai

Missing data is frequently encountered in many areas of statistics. Propensity score weighting is a popular method for handling missing data. The propensity score method employs a response propensity model, but correct specification of the…

Methodology · Statistics 2024-03-28 Hengfang Wang , Jae Kwang Kim , Jeongseop Han , Youngjo Lee

The positivity assumption is central in the identification of a causal effect, and especially the stochastic variant is an issue many applied researchers face, yet is rarely discussed, especially in conjunction with continuous treatments or…

Applications · Statistics 2026-02-03 Katharina Ring , Michael Schomaker

Estimating an individual treatment effect (ITE) is essential to personalized decision making. However, existing methods for estimating the ITE often rely on unconfoundedness, an assumption that is fundamentally untestable with observed…

Methodology · Statistics 2022-07-13 Mingzhang Yin , Claudia Shi , Yixin Wang , David M. Blei

Algorithms and technologies are essential tools that pervade all aspects of our daily lives. In the last decades, health care research benefited from new computer-based recruiting methods, the use of federated architectures for data…

Computers and Society · Computer Science 2023-01-26 Chiara Criscuolo , Tommaso Dolci , Mattia Salnitri

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…

Methodology · Statistics 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

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

Causal inference, especially in observational studies, relies on untestable assumptions about the true data-generating process. Sensitivity analysis helps us determine how robust our conclusions are when we alter these underlying…

Machine Learning · Computer Science 2026-05-11 Nikita Dhawan , Daniel Shen , Leonardo Cotta , Chris J. Maddison

Blinded sample size re-estimation and information monitoring based on blinded data has been suggested to mitigate risks due to planning uncertainties regarding nuisance parameters. Motivated by a randomized controlled trial in pediatric…

Applications · Statistics 2019-03-07 Tobias Mütze , Susanna Salem , Norbert Benda , Heinz Schmidli , Tim Friede

Studies investigating the neural bases of cognitive phenomena such as perception, attention and decision-making increasingly employ multialternative task designs. It is essential in such designs to distinguish the neural correlates of…

Neurons and Cognition · Quantitative Biology 2013-10-17 Devarajan Sridharan , Nicholas A. Steinmetz , Tirin Moore , Eric I. Knudsen

From scientific experiments to online A/B testing, the previously observed data often affects how future experiments are performed, which in turn affects which data will be collected. Such adaptivity introduces complex correlations between…

Machine Learning · Statistics 2018-01-03 Xinkun Nie , Xiaoying Tian , Jonathan Taylor , James Zou

Sensitivity analysis for unmeasured confounding under incremental propensity score interventions remains relatively underdeveloped. Incremental interventions define stochastic treatment regimes by multiplying the odds of treatment, offering…

Methodology · Statistics 2026-01-27 Shuying Shen , Valerio Bacak , Edward H. Kennedy

Trial engagement effects are effects of trial participation on the outcome that are not mediated by treatment assignment. Most work on extending (generalizing or transporting) causal inferences from a randomized trial to a target population…

Methodology · Statistics 2024-07-23 Lawson Ung , Tyler J. VanderWeele , Issa J. Dahabreh

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups…

Although randomized experiments are widely regarded as the gold standard for estimating causal effects, missing data of the pretreatment covariates makes it challenging to estimate the subgroup causal effects. When the missing data…

Statistics Theory · Mathematics 2014-01-08 Peng Ding , Zhi Geng

Recent FDA guidance on adaptive clinical trial designs defines bias as "a systematic tendency for the estimate of treatment effect to deviate from its true value", and states that it is desirable to obtain and report estimates of treatment…