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Treatment policy estimands are frequently favored by regulators, as they assess the effect of treatment assignment regardless of post-randomization events. Despite best efforts, missing data due to study discontinuation cannot be fully…

Methodology · Statistics 2026-05-13 Ajmal Oodally , Craig Wang , Zheng Li , Tim Morris , Tobias Mütze , Arunava Chakravartty

Missing data is inevitable in longitudinal clinical trials. Conventionally, the missing at random assumption is assumed to handle missingness, which however is unverifiable empirically. Thus, sensitivity analysis is critically important to…

Methodology · Statistics 2022-03-18 Siyi Liu , Shu Yang , Yilong Zhang , Guanghan , Liu

Identifying causal treatment (or exposure) effects in observational studies requires the data to satisfy the unconfoundedness assumption which is not testable using the observed data. With sensitivity analysis, one can determine how the…

Methodology · Statistics 2023-01-31 Yang Ou , Lu Tang , Chung-Chou H. Chang

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

The ICH E9(R1) Addendum (International Council for Harmonization 2019) suggests treatment-policy as one of several strategies for addressing intercurrent events such as treatment withdrawal when defining an estimand. This strategy requires…

Methodology · Statistics 2023-08-28 Suzie Cro , James H Roger , James R Carpenter

We provide guidance on multiple imputation of missing at random treatments in observational studies. Specifically, analysts should account for both covariates and outcomes, i.e., not just use propensity scores, when imputing the missing…

Methodology · Statistics 2025-01-23 Joseph Feldman , Jerome P. Reiter

Omitted variable bias can affect treatment effect estimates obtained from observational data due to the lack of random assignment to treatment groups. Sensitivity analyses adjust these estimates to quantify the impact of potential omitted…

Methodology · Statistics 2010-11-10 Carrie A. Hosman , Ben B. Hansen , Paul W. Holland

We provide an approach to exploratory data analysis in matched observational studies with a single intervention and multiple endpoints. In such settings, the researcher would like to explore evidence for actual treatment effects among these…

Methodology · Statistics 2025-12-10 Mengqi Lin , Colin Fogarty

While observational data are routinely used to estimate causal effects of biomedical treatments, doing so requires special methods to adjust for observed confounding. These methods invariably rely on untestable statistical and causal…

Methodology · Statistics 2026-03-02 Arman Oganisian

Missing observations are common in cluster randomised trials. Approaches taken to handling such missing data include: complete case analysis, single-level multiple imputation that ignores the clustering, multiple imputation with a fixed…

Methodology · Statistics 2014-07-18 Karla Diaz-Ordaz , Michael G. Kenward , Manuel Gomes , Richard Grieve

One of the fundamental challenges in drawing causal inferences from observational studies is that the assumption of no unmeasured confounding is not testable from observed data. Therefore, assessing sensitivity to this assumption's…

Methodology · Statistics 2024-06-25 Md Abdul Basit , Mahbub A. H. M. Latif , Abdus S Wahed

A sensitivity analysis in an observational study assesses the robustness of significant findings to unmeasured confounding. While sensitivity analyses in matched observational studies have been well addressed when there is a single outcome…

Methodology · Statistics 2015-11-05 Colin B. Fogarty , Dylan S. Small

We present a framework for generating multiple imputations for continuous data when the missing data mechanism is unknown. Imputations are generated from more than one imputation model in order to incorporate uncertainty regarding the…

Applications · Statistics 2013-01-14 Juned Siddique , Ofer Harel , Catherine M. Crespi

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

Methodology · Statistics 2025-09-29 Lan Wen , Aaron L Sarvet

Multiple imputation is a popular method for handling missing data, with fully conditional specification (FCS) being one of the predominant imputation approaches for multivariable missingness. Unbiased estimation with standard…

In longitudinal studies, it is not uncommon to make multiple attempts to collect a measurement after baseline. Recording whether these attempts are successful provides useful information for the purposes of assessing missing data…

Methodology · Statistics 2023-05-10 Michael J. Daniels , Minji Lee , Wei Feng

We present a new procedure for conducting a sensitivity analysis in matched observational studies. For any candidate test statistic, the approach defines tilted modifications dependent upon the proposed strength of unmeasured confounding.…

Methodology · Statistics 2025-03-14 Colin B. Fogarty

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

Data collected in clinical trials are often composed of multiple types of variables. For example, laboratory measurements and vital signs are longitudinal data of continuous or categorical variables, adverse events may be recurrent events,…

Methodology · Statistics 2023-01-12 Tuo Wang , Rachel Zilinskas , Ying Li , Yongming Qu

Causal inference from observational data requires assumptions. These assumptions range from measuring confounders to identifying instruments. Traditionally, causal inference assumptions have focused on estimation of effects for a single…

Machine Learning · Statistics 2019-03-04 Rajesh Ranganath , Adler Perotte
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