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Dynamic treatment regimes in oncology and other disease areas often can be characterized by an alternating sequence of treatments or other actions and transition times between disease states. The sequence of transition states may vary…

Applications · Statistics 2015-11-17 Yanxun Xu , Peter Mueller , Abdus S. Wahed , Peter F. Thall

One of the most significant barriers to medication treatment is patients' non-adherence to a prescribed medication regimen. The extent of the impact of poor adherence on resulting health measures is often unknown, and typical analyses…

Applications · Statistics 2018-12-04 Luis F. Campos , Mark E. Glickman , Kristen B. Hunter

In oncology the efficacy of novel therapeutics often differs across patient subgroups, and these variations are difficult to predict during the initial phases of the drug development process. The relation between the power of randomized…

Methodology · Statistics 2025-06-05 Boyu Ren , Federico Ferrari , Sandra Fortini , Steffen Ventz , Lorenzo Trippa

A dynamic treatment regimen (DTR) is a pre-specified sequence of decision rules which maps baseline or time-varying measurements on an individual to a recommended intervention or set of interventions. Sequential multiple assignment…

Methodology · Statistics 2019-10-23 Brook Luers , Min Qian , Inbal Nahum-Shani , Connie Kasari , Daniel Almirall

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

Methodology · Statistics 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

Clinical trials often aim to compare a new drug with a reference treatment in terms of efficacy and/or toxicity depending on covariates such as, for example, the dose level of the drug. Equivalence of these treatments can be claimed if the…

Methodology · Statistics 2019-10-22 Holger Dette , Kathrin Möllenhoff , Frank Bretz

Difference-in-differences (DiD) is one of the most popular approaches for empirical research in economics, political science, and beyond. Identification in these models is based on the conditional parallel trends assumption: In the absence…

Econometrics · Economics 2025-10-13 Philipp Bach , Sven Klaassen , Jannis Kueck , Mara Mattes , Martin Spindler

This note introduces a doubly robust (DR) estimator for regression discontinuity (RD) designs. RD designs provide a quasi-experimental framework for estimating treatment effects, where treatment assignment depends on whether a running…

Econometrics · Economics 2025-01-28 Masahiro Kato

What advantage do \emph{sequential} procedures provide over batch algorithms for testing properties of unknown distributions? Focusing on the problem of testing whether two distributions $\mathcal{D}_1$ and $\mathcal{D}_2$ on $\{1,\dots,…

Data Structures and Algorithms · Computer Science 2022-05-13 Omar Fawzi , Nicolas Flammarion , Aurélien Garivier , Aadil Oufkir

We are interested in the distribution of treatment effects for an experiment where units are randomized to a treatment but outcomes are measured for pairs of units. For example, we might measure risk sharing links between households…

Econometrics · Economics 2022-10-11 Eric Auerbach , Yong Cai

Identifying informative predictors in a high dimensional regression model is a critical step for association analysis and predictive modeling. Signal detection in the high dimensional setting often fails due to the limited sample size. One…

Methodology · Statistics 2020-04-08 Molei Liu , Yin Xia , Kelly Cho , Tianxi Cai

We develop an unsupervised probabilistic model for heterogeneous Electronic Health Record (EHR) data. Utilizing a mixture model formulation, our approach directly models sequences of arbitrary length, such as medications and laboratory…

Machine Learning · Computer Science 2022-09-02 Alan D. Kaplan , John D. Greene , Vincent X. Liu , Priyadip Ray

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

We propose a model for multiclass classification of time series to make a prediction as early and as accurate as possible. The matrix sequential probability ratio test (MSPRT) is known to be asymptotically optimal for this setting, but…

Machine Learning · Computer Science 2021-06-01 Taiki Miyagawa , Akinori F. Ebihara

We propose a sequential design method aiming at the estimation of an extreme quantile based on a sample of dichotomic data corresponding to peaks over a given threshold. This study is motivated by an industrial challenge in material…

Methodology · Statistics 2020-04-06 Michel Broniatowski , Emilie Miranda

Sequential multiple assignment randomized trials mimic the actual treatment processes experienced by physicians and patients in clinical settings and inform the comparative effectiveness of dynamic treatment regimes. In such trials,…

Methodology · Statistics 2025-04-07 Zi Wang , Yu Cheng , Abdus S. Wahed

In the last decade, machine learning techniques have gained popularity for estimating causal effects. One machine learning approach that can be used for estimating an average treatment effect is Double/debiased machine learning (DML)…

Econometrics · Economics 2025-01-17 Daniele Ballinari , Nora Bearth

Pooled testing is a common strategy for public health disease screening under limited testing resources, allowing multiple biological samples to be tested together with the resources of a single test, at the cost of reduced individual…

Computer Science and Game Theory · Computer Science 2026-02-02 Nicholas Lopez , Francisco Marmolejo-Cossío , Jose Roberto Tello Ayala , David C. Parkes

Weighted estimators are commonly used for estimating exposure effects in observational settings to establish causal relations. These estimators have a long history of development when the exposure of interest is binary and where the weights…

Mixed effects models are widely used to describe heterogeneity in a population. A crucial issue when adjusting such a model to data consists in identifying fixed and random effects. From a statistical point of view, it remains to test the…

Methodology · Statistics 2017-12-25 Charlotte Baey , Paul-Henry Cournède , Estelle Kuhn
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