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A growing number of oncology treatments, such as bevacizumab, are used across multiple indications. However, in health technology assessment (HTA), their clinical and cost-effectiveness are typically appraised within a single target…

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

Methodology · Statistics 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Ordinal outcomes are common in clinical settings where they often represent increasing levels of disease progression or different levels of functional impairment. Such outcomes can characterize differences in meaningful patient health…

Suppose one is interested in estimating causal effects in the presence of potentially unmeasured confounding with the aid of a valid instrumental variable. This paper investigates the problem of making inferences about the average treatment…

Methodology · Statistics 2020-12-15 BaoLuo Sun , Wang Miao

In causal inference, it is common to estimate the causal effect of a single treatment variable on an outcome. However, practitioners may also be interested in the effect of simultaneous interventions on multiple covariates of a fixed target…

Methodology · Statistics 2022-11-24 Jaime Roquero Gimenez , Dominik Rothenhäusler

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

A method is discussed that allows combining sets of differential or inclusive measurements. It is assumed that at least one measurement was obtained with simultaneously fitting a set of nuisance parameters, representing sources of…

Data Analysis, Statistics and Probability · Physics 2018-01-09 Jan Kieseler

Clinical trials are essential to drug development but time-consuming, costly, and prone to failure. Accurate trial outcome prediction based on historical trial data promises better trial investment decisions and more trial success. Existing…

Machine Learning · Computer Science 2023-04-12 Zifeng Wang , Cao Xiao , Jimeng Sun

Many lifestyle intervention trials depend on collecting self-reported outcomes, like dietary intake, to assess the intervention's effectiveness. Self-reported outcome measures are subject to measurement error, which could impact treatment…

Methodology · Statistics 2020-04-06 Benjamin Ackerman , Juned Siddique , Elizabeth A. Stuart

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

Methodology · Statistics 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for…

Methodology · Statistics 2023-06-27 Wenjie Hu , Xiaohua Zhou , Peng Wu

In the era of big data, secondary outcomes have become increasingly important alongside primary outcomes. These secondary outcomes, which can be derived from traditional endpoints in clinical trials, compound measures, or risk prediction…

Methodology · Statistics 2025-12-16 Daxuan Deng , Peisong Han , Shuo Chen , Ming Wang , Chixiang Chen

Many scientific questions in biomedical, environmental, and psychological research involve understanding the effects of multiple factors on outcomes. While factorial experiments are ideal for this purpose, randomized controlled treatment…

Methodology · Statistics 2025-12-03 Ruoqi Yu , Peng Ding

We investigate how to exploit structural similarities of an individual's potential outcomes (POs) under different treatments to obtain better estimates of conditional average treatment effects in finite samples. Especially when it is…

Machine Learning · Statistics 2021-10-26 Alicia Curth , Mihaela van der Schaar

Using multiple ion beam analysis measurements, or techniques, combined with self-consistent data processing, generally allows extracting more (or more accurate) information from the measurements than processing separately data from single…

Data Analysis, Statistics and Probability · Physics 2022-11-23 Tiago F. Silva , Cleber L. Rodrigues , Manfredo H. Tabacniks , Udo von Toussaint , Matej Mayer

Observational studies of treatment effects require adjustment for confounding variables. However, causal inference methods typically cannot deliver perfect adjustment on all measured baseline variables, and there is often ambiguity about…

Methodology · Statistics 2024-02-16 Lauren D. Liao , Yeyi Zhu , Amanda L. Ngo , Rana F. Chehab , Samuel D. Pimentel

As artificial intelligence and machine learning tools become more accessible, and scientists face new obstacles to data collection (e.g. rising costs, declining survey response rates), researchers increasingly use predictions from…

Methodology · Statistics 2024-02-06 Kentaro Hoffman , Stephen Salerno , Awan Afiaz , Jeffrey T. Leek , Tyler H. McCormick

Causal inference on multiple non-independent outcomes raises serious challenges, because multivariate techniques that properly account for the outcome's dependence structure need to be considered. We focus on the case of binary outcomes…

Methodology · Statistics 2018-05-11 Monia Lupparelli , Alessandra Mattei

Often both Aggregate Data (AD) studies and Individual Patient Data (IPD) studies are available for specific treatments. Combining these two sources of data could improve the overall meta-analytic estimates of treatment effects. Moreover,…

Methodology · Statistics 2021-11-15 Neha Agarwala , Junyong Park , Anindya Roy

Longitudinal passive sensing studies for health and behavior outcomes often have missing and incomplete data. Handling missing data effectively is thus a critical data processing and modeling step. Our formative interviews with researchers…