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In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and efficacious that maximises some optimality criterion based on safety and efficacy. This is further complicated when…

Applications · Statistics 2022-03-31 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

Nested error regression models are useful tools for analysis of grouped data, especially in the case of small area estimation. This paper suggests a nested error regression model using uncertain random effects in which the random effect in…

Methodology · Statistics 2017-02-28 Shonosuke Sugasawa , Tatsuya Kubokawa

Assessing the pre-operative risk of lymph node metastases in endometrial cancer patients is a complex and challenging task. In principle, machine learning and deep learning models are flexible and expressive enough to capture the dynamics…

Artificial Intelligence · Computer Science 2023-05-18 Alessio Zanga , Alice Bernasconi , Peter J. F. Lucas , Hanny Pijnenborg , Casper Reijnen , Marco Scutari , Fabio Stella

In many biological applications, the primary objective of study is to quantify the magnitude of treatment effect between two groups. Cohens'd or strictly standardized mean difference (SSMD) can be used to measure effect size however, it is…

Applications · Statistics 2020-11-18 Seongyong Park , Shujaat Khan , Muhammad Moinuddin , Ubaid M. Al-Saggaf

The tail of a bivariate distribution function in the domain of attraction of a bivariate extreme-value distribution may be approximated by the one of its extreme-value attractor. The extreme-value attractor has margins that belong to a…

Statistics Theory · Mathematics 2012-05-14 Simon Guillotte , Francois Perron , Johan Segers

We study treatment-effect estimation using panel data. The treatment may be non-binary, non-absorbing, and the outcome may be affected by treatment lags. We make a parallel-trends assumption, and propose event-study estimators of the effect…

Econometrics · Economics 2026-05-13 Clément de Chaisemartin , Xavier D'Haultfœuille

Staggered treatment adoption arises in the evaluation of policy impact and implementation in many settings, including both randomized stepped-wedge trials and non-randomized quasi-experiments with panel data. In both settings, getting an…

Methodology · Statistics 2024-10-14 Lee Kennedy-Shaffer

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

We consider the estimation of average treatment effects in observational studies and propose a new framework of robust causal inference with unobserved confounders. Our approach is based on distributionally robust optimization and proceeds…

Methodology · Statistics 2023-02-06 Dimitris Bertsimas , Kosuke Imai , Michael Lingzhi Li

We consider the problem of estimating parameters of stochastic differential equations (SDEs) with discrete-time observations that are either completely or partially observed. The transition density between two observations is generally…

Methodology · Statistics 2015-09-09 Libo Sun , Chihoon Lee , Jennifer A. Hoeting

Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the…

Methodology · Statistics 2024-06-11 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

The Mann-Kendall test for trend has gained a lot of attention in a range of disciplines, especially in the environmental sciences. One of the drawbacks of the Mann-Kendall test when applied to real data is that no distinction can be made…

Methodology · Statistics 2023-05-24 Stavros Nikolakopoulos , Eric Cator , Mart P. Janssen

In a meta-analysis, it is important to specify a model that adequately describes the effect-size distribution of the underlying population of studies. The conventional normal fixed-effect and normal random-effects models assume a normal…

Methodology · Statistics 2013-10-21 George Karabatsos , Elizabeth Talbott , Stephen G. Walker

We introduce bipol, a new metric with explainability, for estimating social bias in text data. Harmful bias is prevalent in many online sources of data that are used for training machine learning (ML) models. In a step to address this…

Computation and Language · Computer Science 2023-09-19 Lama Alkhaled , Tosin Adewumi , Sana Sabah Sabry

Big longitudinal observational medical data potentially hold a wealth of information and have been recognised as potential sources for gaining new drug safety knowledge. Unfortunately there are many complexities and underlying issues when…

Artificial Intelligence · Computer Science 2016-07-22 Jenna Marie Reps , Jonathan M. Garibaldi , Uwe Aickelin , Jack E. Gibson , Richard B. Hubbard

In mobile health, tailoring interventions for real-time delivery is of paramount importance. Micro-randomized trials have emerged as the "gold-standard" methodology for developing such interventions. Analyzing data from these trials…

Methodology · Statistics 2024-07-31 Xueqing Liu , Tianchen Qian , Lauren Bell , Bibhas Chakraborty

The conventional nonparametric tests in survival analysis, such as the log-rank test, assess the null hypothesis that the hazards are equal at all times. However, hazards are hard to interpret causally, and other null hypotheses are more…

Methodology · Statistics 2019-01-29 Mats Julius Stensrud , Kjetil Røysland , Pål Christie Ryalen

Nonlinear mixed effects models have received a great deal of attention in the statistical literature in recent years because of their flexibility in handling longitudinal studies, including human immunodeficiency virus viral dynamics,…

Methodology · Statistics 2021-09-28 Fernanda L. Schumacher , Dipak K. Dey , Victor H. Lachos

Detection of adversarial examples has been a hot topic in the last years due to its importance for safely deploying machine learning algorithms in critical applications. However, the detection methods are generally validated by assuming a…

Computer Vision and Pattern Recognition · Computer Science 2022-07-01 Federica Granese , Marine Picot , Marco Romanelli , Francisco Messina , Pablo Piantanida

We present two methods for bounding the probabilities of benefit and harm under unmeasured confounding. The first method computes the (upper or lower) bound of either probability as a function of the observed data distribution and two…

Methodology · Statistics 2023-08-08 Jose M. Peña