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Some patients benefit from a treatment while others may do so less or do not benefit at all. We have previously developed a two-stage network meta-regression prediction model that synthesized randomized trials and evaluates how treatment…

In medical treatment and elsewhere, it has become standard to base treatment intensity (dosage) on evidence in randomized trials. Yet it has been rare to study how outcomes vary with dosage. In trials to obtain drug approval, the norm has…

Econometrics · Economics 2023-05-30 Charles F. Manski

The doubly robust estimator, which models both the propensity score and outcomes, is a popular approach to estimate the average treatment effect in the potential outcome setting. The primary appeal of this estimator is its theoretical…

Methodology · Statistics 2024-09-11 Kaoru Babasaki , Shonosuke Sugasawa , Kosaku Takanashi , Kenichiro McAlinn

When studying treatment effects in multilevel studies, investigators commonly use (semi-)parametric estimators, which make strong parametric assumptions about the outcome, the treatment, and/or the correlation structure between study units…

Methodology · Statistics 2022-05-12 Chan Park , Hyunseung Kang

Shortcomings of randomized clinical trials are pronounced in urgent health crises, when rapid identification of effective treatments is critical. Leveraging short-term surrogates in real-world data (RWD) can guide policymakers evaluating…

Methodology · Statistics 2021-04-13 Larry Han , Xuan Wang , Tianxi Cai

Estimating the effect of a treatment on a given outcome, conditioned on a vector of covariates, is central in many applications. However, learning the impact of a treatment on a continuous temporal response, when the covariates suffer…

Machine Learning · Computer Science 2019-06-11 Guangyi Zhang , Reza Ashrafi , Anne Juuti , Kirsi Pietiläinen , Pekka Marttinen

This paper presents methods for meta-analysis of $2 \times 2$ tables, both with and without allowing heterogeneity in the treatment effects. Meta-analysis is common in medical research, but most existing methods are unsuited for $2 \times…

Methodology · Statistics 2026-02-23 Céline Cunen , Nils Lid Hjort

With the advancement in drug development, multiple treatments are available for a single disease. Patients can often benefit from taking multiple treatments simultaneously. For example, patients in Clinical Practice Research Datalink (CPRD)…

Applications · Statistics 2018-04-17 Muxuan Liang , Ye Ting , Haoda Fu

Regression models are essential for a wide range of real-world applications. However, in practice, target values are not always precisely known; instead, they may be represented as intervals of acceptable values. This challenge has led to…

Machine Learning · Computer Science 2025-12-08 Tung L Nguyen , Toby Dylan Hocking

Propensity score weighting is a common method for estimating treatment effects with survey data. The method is applied to minimize confounding using measured covariates that are often different between individuals in treatment and control.…

Methodology · Statistics 2026-02-06 Yukang Zeng , Fan Li , Guangyu Tong

Given n experiment subjects with potentially heterogeneous covariates and two possible treatments, namely active treatment and control, this paper addresses the fundamental question of determining the optimal accuracy in estimating the…

Machine Learning · Statistics 2024-11-13 Jiachun Li , David Simchi-Levi , Yunxiao Zhao

Adaptive designs are increasingly used in clinical trials and online experiments to improve participant outcomes by dynamically updating treatment allocation as data accumulate. In practice, experimenters often consider multiple candidate…

Methodology · Statistics 2026-04-08 Wenxin Zhang , Aaron Hudson , Maya Petersen , Mark van der Laan

In a poisoning attack, an adversary with control over a small fraction of the training data attempts to select that data in a way that induces a corrupted model that misbehaves in favor of the adversary. We consider poisoning attacks…

Machine Learning · Computer Science 2021-04-22 Fnu Suya , Saeed Mahloujifar , Anshuman Suri , David Evans , Yuan Tian

In applications of linear mixed-effects models, experimenters often desire uncertainty quantification for random quantities, like predicted treatment effects for unobserved individuals or groups. For example, consider an agricultural…

Methodology · Statistics 2022-10-19 Nicholas Syring , Fernando Miguez , Jarad Niemi

Instrumental variables (IV) regression is widely used to estimate causal treatment effects in settings where receipt of treatment is not fully random, but there exists an instrument that generates exogenous variation in treatment exposure.…

Econometrics · Economics 2021-08-10 Stephen Coussens , Jann Spiess

In clinical trials with recurrent events, such as repeated hospitalizations terminating with death, it is important to consider the patient events overall history for a thorough assessment of treatment effects. The occurrence of fewer…

Methodology · Statistics 2025-09-08 Alessandra Ragni , Torben Martinussen , Thomas Scheike

Causal mediation analysis examines causal pathways linking exposures to disease. The estimation of interventional effects, which are mediation estimands that overcome certain identifiability problems of natural effects, has been advanced…

Medication recommendation is one of the most critical health-related applications, which has attracted extensive research interest recently. Most existing works focus on a single hospital with abundant medical data. However, many small…

Information Retrieval · Computer Science 2024-12-31 Qidong Liu , Zhaopeng Qiu , Xiangyu Zhao , Xian Wu , Zijian Zhang , Tong Xu , Feng Tian

Weighting methods are popular tools for estimating causal effects; assessing their robustness under unobserved confounding is important in practice. In the following paper, we introduce a new set of sensitivity models called "variance-based…

Methodology · Statistics 2023-03-14 Melody Huang , Samuel D. Pimentel

When multiple measures are collected repeatedly over time, redundancy typically exists among responses. The envelope method was recently proposed to reduce the dimension of responses without loss of information in regression with…

Methodology · Statistics 2021-03-25 Yuyang Shi , Linquan Ma , Lan Liu
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