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We propose new, optimal methods for analyzing randomized trials, when it is suspected that treatment effects may differ in two predefined subpopulations. Such sub-populations could be defined by a biomarker or risk factor measured at…

Methodology · Statistics 2016-11-26 Michael Rosenblum , Han Liu , and En-Hsu Yen

Randomized trials balance all covariates on average and provide the gold standard for estimating treatment effects. Chance imbalances nevertheless exist more or less in realized treatment allocations and intrigue an important question: what…

Methodology · Statistics 2023-07-18 Anqi Zhao , Peng Ding

Two-phase sampling offers a cost-effective way to validate error-prone covariate measurements in biomedical databases. Inexpensive or easy-to-obtain information is collected for the entire study in Phase I. Then, a subset of patients…

Methodology · Statistics 2026-05-21 Sarah C. Lotspeich , Cole Manschot

We introduce a new sufficient statistic for the population parameter vector by allowing for the sampling design to first be selected at random amongst a set of candidate sampling designs. In contrast to the traditional approach in survey…

Statistics Theory · Mathematics 2014-11-11 Kyle Vincent , Christopher S. Henry

The aim of survey statistics is to produce estimates with a minimal bias and a corresponding acceptable variance given a specific budget, preferable with a minor response burden for the participants. In recent years, considerable efforts…

Methodology · Statistics 2026-04-02 Martin Hyllienmark , Gustaf Strandell

The issue of determining not only an adequate dose but also a dosing frequency of a drug arises frequently in Phase II clinical trials. This results in the comparison of models which have some parameters in common. Planning such studies…

Methodology · Statistics 2017-11-16 Kirsten Schorning , Maria Konstantinou

A dynamic treatment regime is a sequence of decision rules in which each decision rule recommends treatment based on features of patient medical history such as past treatments and outcomes. Existing methods for estimating optimal dynamic…

Methodology · Statistics 2015-05-22 Kristin A. Linn , Eric B. Laber , Leonard A. Stefanski

Under two-phase designs, the outcome and several covariates and confounders are measured in the first phase, and a new predictor of interest, which may be costly to collect, can be measured on a subsample in the second phase, without…

Methodology · Statistics 2023-05-12 Jingjing Zou , Lori B. Daniels , Karen Messer , Daniel Rabinowitz

Dynamic treatment regimens (DTRs), also known as treatment algorithms or adaptive interventions, play an increasingly important role in many health domains. DTRs are motivated to address the unique and changing needs of individuals by…

With increasing interest in adaptive clinical trial designs, challenges are present to drug supply chain management which may offset the benefit of adaptive designs. Thus, it is necessary to develop an optimization tool to facilitate the…

Applications · Statistics 2023-10-16 Jincheng Pang , Hong Yan , Zoe Hua

Platform trials are randomized clinical trials that allow simultaneous comparison of multiple interventions, usually against a common control. Arms to test experimental interventions may enter and leave the platform over time. This implies…

Methodology · Statistics 2024-04-12 Marta Bofill Roig , Ekkehard Glimm , Tobias Mielke , Martin Posch

The goal of a well-controlled study is to remove unwanted variation when estimating the causal effect of the intervention of interest. Experiments conducted in the basic sciences frequently achieve this goal using experimental controls,…

Methodology · Statistics 2021-04-22 Kristen B. Hunter , Kristen Koenig , Marie-Abèle Bind

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

Methodology · Statistics 2026-03-31 Zhe Chen , Xinran Li

Subclassification and matching are often used in empirical studies to adjust for observed covariates; however, they are largely restricted to relatively simple study designs with a binary treatment and less developed for designs with a…

Methodology · Statistics 2022-01-28 Bo Zhang , Emily J. Mackay , Mike Baiocchi

Discrete Choice Experiments (DCEs) investigate participants' preferences by observing their choice behavior in hypothetical scenarios and are widely used in the domain of healthcare. To reduce participants' cognitive burden, especially when…

Methodology · Statistics 2026-04-07 Yicheng Mao , Roselinde Kessels

A main challenge of data-driven sciences is how to make maximal use of the progressively expanding databases of experimental datasets in order to keep research cumulative. We introduce the idea of a modeling-based dataset retrieval engine…

Quantitative Methods · Quantitative Biology 2015-06-19 Ali Faisal , Jaakko Peltonen , Elisabeth Georgii , Johan Rung , Samuel Kaski

Some of the most used sampling mechanisms that implicitly leverage a social network depend on tuning parameters; for instance, Respondent-Driven Sampling (RDS) is specified by the number of seeds and maximum number of referrals. We are…

Methodology · Statistics 2019-12-06 Simón Lunagómez , Marios Papamichalis , Patrick J. Wolfe , Edoardo M. Airoldi

Randomized experiments are widely used to estimate causal effects across a variety of domains. However, classical causal inference approaches rely on critical independence assumptions that are violated by network interference, when the…

Methodology · Statistics 2022-10-18 Mayleen Cortez , Matthew Eichhorn , Christina Lee Yu

Randomized experiments are an excellent tool for estimating internally valid causal effects with the sample at hand, but their external validity is frequently debated. While classical results on the estimation of Population Average…

Methodology · Statistics 2023-01-13 Apoorva Lal , Wenjing Zheng , Simon Ejdemyr

The dramatic increase of observational data across industries provides unparalleled opportunities for data-driven decision making and management, including the manufacturing industry. In the context of production, data-driven approaches can…

Optimization and Control · Mathematics 2018-01-09 Najibesadat Sadati , Ratna Babu Chinnam , Milad Zafar Nezhad
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