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Cost-Effectiveness Analyses (CEAs) alongside randomised controlled trials (RCTs) are increasingly often designed to collect resource use and preference-based health status data for the purpose of healthcare technology assessment. However,…

Applications · Statistics 2016-07-22 Andrea Gabrio , Alexina Mason , Gianluca Baio

Health economic evaluations based on patient-level data collected alongside clinical trials~(e.g. health related quality of life and resource use measures) are an important component of the process which informs resource allocation…

Applications · Statistics 2020-05-25 Andrea Gabrio , Rachael Hunter , Alexina J. Mason , Gianluca Baio

Trial-based economic evaluations are typically performed on cross-sectional variables, derived from the responses for only the completers in the study, using methods that ignore the complexities of utility and cost data (e.g. skewness and…

Methodology · Statistics 2018-05-21 Andrea Gabrio , Michael J. Daniels , Gianluca Baio

Randomized control trials (RCTs) are the gold standard for estimating causal effects, but often use samples that are non-representative of the actual population of interest. We propose a reweighting method for estimating population average…

Methodology · Statistics 2022-02-09 Kellie Ottoboni , Jason Poulos

In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the…

Methodology · Statistics 2025-11-06 Bingkai Wang , Fan Li , Rui Wang

Missing data is a systemic problem in practical scenarios that causes noise and bias when estimating treatment effects. This makes treatment effect estimation from data with missingness a particularly tricky endeavour. A key reason for this…

Machine Learning · Statistics 2023-02-27 Jeroen Berrevoets , Fergus Imrie , Trent Kyono , James Jordon , Mihaela van der Schaar

We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling…

Methodology · Statistics 2023-02-27 Imke Mayer , Julie Josse , Traumabase Group

Randomized controlled trials (RCTs) face inherent limitations, such as ethical or resource constraints, which lead to a limited number of study participants. To address these limitations, recent research endeavors have sought to incorporate…

Methodology · Statistics 2025-02-20 Peijin Wang , Hwanhee Hong , Kyungeun Jeon , Laine Elliott Thomas

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

Methodology · Statistics 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa

Randomized controlled trials (RCTs) are widely regarded as the gold standard for causal inference in biomedical research. For instance, when estimating the average treatment effect on the treated (ATT), a doubly robust estimation procedure…

Methodology · Statistics 2025-09-26 Chi-Shian Dai , Chao Ying , Yang Ning , Jiwei Zhao

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

Artificial Intelligence · Computer Science 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

The current literature regarding generation of complex, realistic synthetic tabular data, particularly for randomized controlled trials (RCTs), often ignores missing data. However, missing data are common in RCT data and often are not…

Other Statistics · Statistics 2025-12-02 Niki Z. Petrakos , Erica E. M. Moodie , Nicolas Savy

Missing data is pervasive in econometric applications, and rarely is it plausible that the data are missing (completely) at random. This paper proposes a methodology for studying the robustness of results drawn from incomplete datasets.…

Econometrics · Economics 2025-12-29 Daniel Ober-Reynolds

The analysis of randomized trials is often complicated by the occurrence of intercurrent events and missing values. Even though there are different strategies to address missing values it is still common to require missing values…

Methodology · Statistics 2025-11-11 A. Ruiz de Villa , Ll. Badiella

Noncompliance and missing data often occur in randomized trials, which complicate the inference of causal effects. When both noncompliance and missing data are present, previous papers proposed moment and maximum likelihood estimators for…

Methodology · Statistics 2014-09-04 Hua Chen , Peng Ding , Zhi Geng , Xiao-Hua Zhou

Current clinical decision support systems (DSS) are trained and validated on observational data from the target clinic. This is problematic for treatments validated in a randomized clinical trial (RCT), but not yet introduced in any clinic.…

In this paper we present tools for applied researchers that re-purpose off-the-shelf methods from the computer-science field of machine learning to create a "discovery engine" for data from randomized controlled trials (RCTs). The applied…

Machine Learning · Statistics 2019-05-13 Jens Ludwig , Sendhil Mullainathan , Jann Spiess

Public policy-makers use cost-effectiveness analyses (CEA) to decide which health and social care interventions to provide. Appropriate methods have not been developed for handling missing data in complex settings, such as for CEA that use…

Methodology · Statistics 2012-06-27 Karla Diaz-Ordaz , Michael G. Kenward , Richard Grieve

This tutorial aims to provide signal processing (SP) and machine learning (ML) practitioners with vital tools, in an accessible way, to answer the question: How to deal with missing data? There are many strategies to handle incomplete…

Signal Processing · Electrical Eng. & Systems 2026-01-06 Alexandre Hippert-Ferrer , Aude Sportisse , Amirhossein Javaheri , Mohammed Nabil El Korso , Daniel P. Palomar

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
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