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Detecting associations between microbial compositions and sample characteristics is one of the most important tasks in microbiome studies. Most of the existing methods apply univariate models to single microbial species separately, with…

The commonplace description of phase 1 clinical trials in oncology as "primarily concerned with safety" is belied by their near universal adoption of dose-escalation practices which are inherently unsafe. In contrast with dose titration,…

Methodology · Statistics 2020-04-28 David C. Norris

This paper explores the intrinsic connections between the Bayesian false discovery rate (FDR) control procedures and their counterpart of frequentist procedures. We attempt to offer a unified view of FDR control within and beyond the…

Methodology · Statistics 2018-03-15 Xiaoquan Wen

Treatment effects can be estimated from observational data as the difference in potential outcomes. In this paper, we address the challenge of estimating the potential outcome when treatment-dose levels can vary continuously over time.…

Machine Learning · Statistics 2017-11-07 Hossein Soleimani , Adarsh Subbaswamy , Suchi Saria

State-level policy evaluations commonly employ a difference-in-differences (DID) study design; yet within this framework, statistical model specification varies notably across studies. Motivated by applied state-level opioid policy…

Treatment effects vary across different patients and estimation of this variability is important for clinical decisions. The aim is to develop a model to estimate the benefit of alternative treatment options for individual patients. Hence,…

This paper addresses the problem of deciding whether the dose response relationships between subgroups and the full population in a multi-regional trial are similar to each other. Similarity is measured in terms of the maximal deviation…

Statistics Theory · Mathematics 2024-04-30 Holger Dette , Lukas Koletzko , Frank Bretz

This paper describes a heuristic Bayesian method for computing probability distributions from experimental data, based upon the multivariate normal form of the influence diagram. An example illustrates its use in medical technology…

Artificial Intelligence · Computer Science 2013-04-11 Ross D. Shachter , David M. Eddy , Vic Hasselblad , Robert Wolpert

In a Phase II dose-finding study with a placebo control, a new drug with several dose levels is compared with a placebo to test for the effectiveness of the new drug. The main focus of such studies often lies in the characterization of the…

Methodology · Statistics 2020-07-14 Saswati Saha , Werner Brannath

Surrogate endpoints play an important role in drug development when they can be used to measure treatment effect early compared to the final clinical outcome and to predict clinical benefit or harm. Such endpoints are assessed for their…

The opioid epidemic remains a major public health challenge in the United States, requiring a multi-pronged intervention approach to mitigate harms to communities. Given the heterogeneity of the epidemic across the country, it is crucial…

Applications · Statistics 2026-04-13 Abdulrahman A. Ahmed , M. Amin Rahimian , Qiushi Chen , Praveen Kumar

Experiments on decision making under uncertainty are known to display a classical pattern of risk aversion and risk seeking referred to as "fourfold pattern" (or "reflection effect") , but recent experiments varying the speed and order of…

Neurons and Cognition · Quantitative Biology 2024-01-17 Francesco Fumarola , Lukasz Kusmierz , Ronald B. Dekker

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

An accurately identified maximum tolerated dose (MTD) serves as the cornerstone of successful subsequent phases in oncology drug development. Bayesian logistic regression model (BLRM) is a popular and versatile model-based dose-finding…

Methodology · Statistics 2021-05-17 Hongtao Zhang , Alan Y Chiang , Jixian Wang

Evaluating treatment effect heterogeneity across patient subgroups is a fundamental aspect of clinical trial analysis. Yet, these analyses have inherent limitations due to small sample sizes and the substantial number of subgroups…

Methodology · Statistics 2026-03-24 Marcel Wolbers , Miriam Pedrera Gómez , Alex Ocampo , Isaac Gravestock

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

In this paper, a methodology is proposed that enables to analyze the sensitivity of the outcome of a therapy to unavoidable high dispersion of the patient specific parameters on one hand and to the choice of the parameters that define the…

Systems and Control · Electrical Eng. & Systems 2022-05-17 Mazen Alamir

Joint models have proven to be an effective approach for uncovering potentially hidden connections between various types of outcomes, mainly continuous, time-to-event, and binary. Typically, longitudinal continuous outcomes are…

A common problem in Phase II clinical trials is the comparison of dose response curves corresponding to different treatment groups. If the effect of the dose level is described by parametric regression models and the treatments differ in…

Statistics Theory · Mathematics 2016-03-16 Chrystel Feller , Kirsten Schorning , Holger Dette , Georgina Bermann , Björn Bornkamp

The effects of treatments may differ between persons with different characteristics. Addressing such treatment heterogeneity is crucial to investigate whether patients with specific characteristics are likely to benefit from a new…

Methodology · Statistics 2024-03-29 Xynthia Kavelaars , Joris Mulder , Maurits Kaptein