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Mixed outcome endpoints that combine multiple continuous and discrete components to form co-primary, multiple primary or composite endpoints are often employed as primary outcome measures in clinical trials. There are many advantages to…

Methodology · Statistics 2019-12-12 Martina McMenamin , Jessica K. Barrett , Anna Berglind , James M. S. Wason

Recently, phase II trials with multiple schedules (frequency of administrations) have become more popular, for instance in the development of treatments for atopic dermatitis. If the relationship of the dose and response is described by a…

Applications · Statistics 2020-11-17 Burak Kürsad Günhan , Paul Meyvisch , Tim Friede

We develop an empirical Bayes procedure for estimating the cell means in an unbalanced, two-way additive model with fixed effects. We employ a hierarchical model, which reflects exchangeability of the effects within treatment and within…

Methodology · Statistics 2016-05-30 Lawrence D. Brown , Gourab Mukherjee , Asaf Weinstein

Adaptive experiment designs can dramatically improve statistical efficiency in randomized trials, but they also complicate statistical inference. For example, it is now well known that the sample mean is biased in adaptive trials.…

Machine Learning · Statistics 2021-02-16 Vitor Hadad , David A. Hirshberg , Ruohan Zhan , Stefan Wager , Susan Athey

An individualized dose rule recommends a dose level within a continuous safe dose range based on patient level information such as physical conditions, genetic factors and medication histories. Traditionally, personalized dose finding…

Methodology · Statistics 2020-07-21 Liangyu Zhu , Wenbin Lu , Michael R. Kosorok , Rui Song

We consider a setting where an agent's uncertainty is represented by a set of probability measures, rather than a single measure. Measure-by-measure updating of such a set of measures upon acquiring new information is well-known to suffer…

Computer Science and Game Theory · Computer Science 2016-11-04 Joseph Y. Halpern , Samantha Leung

Model-Agnostic Meta-Learning (MAML), a popular gradient-based meta-learning framework, assumes that the contribution of each task or instance to the meta-learner is equal. Hence, it fails to address the domain shift between base and novel…

Machine Learning · Computer Science 2021-12-02 Krishnateja Killamsetty , Changbin Li , Chen Zhao , Rishabh Iyer , Feng Chen

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu

Automated model selection is often proposed to users to choose which machine learning model (or method) to apply to a given regression task. In this paper, we show that combining different regression models can yield better results than…

Machine Learning · Computer Science 2022-06-24 Patrick Echtenbruck , Martina Echtenbruck , Joost Batenburg , Thomas Bäck , Boris Naujoks , Michael Emmerich

When estimating the treatment effect in an observational study, we use a semiparametric locally efficient dimension reduction approach to assess both the treatment assignment mechanism and the average responses in both treated and…

Methodology · Statistics 2020-10-26 Trinetri Ghosh , Yanyuan Ma , Xavier de Luna

In observational studies with dichotomous outcome of a population, researchers need to present the effects of exposures and interaction between the exposures jointly in order to learn the relationship between the exposure effects and the…

Methodology · Statistics 2015-01-22 Xiaoqin Wang , Weimin Ye , Li Yin

Standard regression discontinuity design (RDD) models rely on the continuity of expected potential outcomes at the cutoff. The standard continuity assumption can be violated by strategic manipulation of the running variable, which is…

Econometrics · Economics 2025-07-18 Rahul Singh , Moses Stewart

A promising way to improve the sample efficiency of reinforcement learning is model-based methods, in which many explorations and evaluations can happen in the learned models to save real-world samples. However, when the learned model has a…

Machine Learning · Computer Science 2022-09-14 Haoxin Lin , Yihao Sun , Jiaji Zhang , Yang Yu

The target trial framework enables causal inference from longitudinal observational data by emulating randomized trials initiated at multiple time points. Precision is often improved by pooling information across trials, with standard…

Methodology · Statistics 2026-01-08 Edoardo Efrem Gervasoni , Liesbet De Bus , Stijn Vansteelandt , Oliver Dukes

While meta-analyzing retrospective cancer patient cohorts, an investigation of differences in the expressions of target oncogenes across cancer subtypes is of substantial interest because the results may uncover novel tumorigenesis…

Methodology · Statistics 2023-07-03 Subharup Guha , David C. Christiani , S. V. Subramanian , Yi Li

Most conventional risk analysis methods rely on a single best estimate of exposure per person which does not allow for adjustment for exposure-related uncertainty. Here, we propose a Bayesian model averaging method to properly quantify the…

Applications · Statistics 2020-04-07 Deukwoo Kwon , F. Owen Hoffman , Brian E. Moroz , Steven L. Simon

When estimating treatment effects with two-way fixed effects (2WFE) models, researchers often use matching as a pre-processing step when the parallel trends assumption is thought to hold conditionally on covariates. Specifically, in a first…

Econometrics · Economics 2026-02-17 Yihong Liu , Gonzalo Vazquez-Bare

The era of big data is coming, and evidence-based medicine is attracting increasing attention to improve decision making in medical practice via integrating evidence from well designed and conducted clinical research. Meta-analysis is a…

Methodology · Statistics 2016-10-05 Dehui Luo , Xiang Wan , Jiming Liu , Tiejun Tong

Deep learning has facilitated the automation of radiotherapy by predicting accurate dose distribution maps. However, existing methods fail to derive the desirable radiotherapy parameters that can be directly input into the treatment…

Computer Vision and Pattern Recognition · Computer Science 2024-03-01 Jiaqi Cui , Yuanyuan Xu , Jianghong Xiao , Yuchen Fei , Jiliu Zhou , Xingcheng Peng , Yan Wang

I study identification, estimation and inference for spillover effects in experiments where units' outcomes may depend on the treatment assignments of other units within a group. I show that the commonly-used reduced-form linear-in-means…

Econometrics · Economics 2022-01-21 Gonzalo Vazquez-Bare