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In clinical trials the comparison of two different populations is a frequently addressed problem. Non-linear (parametric) regression models are commonly used to describe the relationship between covariates as the dose and a response…

Methodology · Statistics 2019-02-12 Kathrin Möllenhoff , Frank Bretz , Holger Dette

We consider two problems that are attracting increasing attention in clinical dose finding studies. First, we assess the similarity of two non-linear regression models for two non-overlapping subgroups of patients over a restricted…

Methodology · Statistics 2017-09-12 Frank Bretz , Kathrin Möllenhoff , Holger Dette , Wei Liu , Matthias Trampisch

The identification of similar patient pathways is a crucial task in healthcare analytics. A flexible tool to address this issue are parametric competing risks models, where transition intensities may be specified by a variety of parametric…

Methodology · Statistics 2024-01-10 Kathrin Möllenhoff , Nadine Binder , Holger Dette

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

Assessing whether two patient populations exhibit comparable event dynamics is essential for evaluating treatment equivalence, pooling data across cohorts, or comparing clinical pathways across hospitals or strategies. We introduce a…

Methodology · Statistics 2026-04-10 Zoe Kristin Lange , Maryam Farhadizadeh , Holger Dette , Nadine Binder

Identifying subgroups of patients with an enhanced response to a new treatment has become an area of increased interest in the last few years. When there is knowledge about possible subpopulations with an enhanced treatment effect before…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Martin Posch , Franz König

To claim similarity of multiple dose-response curves in interlaboratory studies in regulatory toxicology is a relevant issue during the assay validation process. Here we demonstrated the use of dose-by-laboratory interaction contrasts,…

Applications · Statistics 2020-09-28 Ludwig A. Hothorn , Dimitrios Spiliotopoulos

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

In this paper we develop a novel bootstrap test for the comparison of two multinomial distributions. The two distributions are called {\it equivalent} or {\it similar} if a norm of the difference between the class probabilities is smaller…

Statistics Theory · Mathematics 2023-05-16 Patrick Bastian , Holger Dette , Lukas Koletzko

This paper investigates the problem whether the difference between two parametric models $m_1,m_2$ describing the relation between a response variable and several covariates in two different groups is practically irrelevant, such that…

Methodology · Statistics 2016-06-09 Holger Dette , Kathrin Möllenhoff , Stanislav Volgushev , Frank Bretz

In this paper we compare two regression curves by measuring their difference by the area between the two curves, represented by their $L^1$-distance. We develop asymptotic confidence intervals for this measure and statistical tests to…

Statistics Theory · Mathematics 2023-02-03 Patrick Bastian , Holger Dette , Lukas Koletzko , Kathrin Möllenhoff

We consider the problem of testing for a dose-related effect based on a candidate set of (typically nonlinear) dose-response models using likelihood-ratio tests. For the considered models this reduces to assessing whether the slope…

Methodology · Statistics 2015-10-22 Georg Gutjahr , Björn Bornkamp

In recent years there have been a lot of interest to test for similarity between biological drug products, commonly known as biologics. Biologics are large and complex molecule drugs that are produced by living cells and hence these are…

Methodology · Statistics 2019-02-21 Lin Dong , Sujit K. Ghosh

Statistical methodology for the design and analysis of clinical Phase II dose response studies, with related software implementation, are well developed for the case of a normally distributed, homoscedastic response considered for a single…

Methodology · Statistics 2014-05-09 José Pinheiro , Björn Bornkamp , Ekkehard Glimm , Frank Bretz

Several approaches to testing the hypothesis that two histograms are drawn from the same distribution are investigated. We note that single-sample continuous distribution tests may be adapted to this two-sample grouped data situation. The…

Data Analysis, Statistics and Probability · Physics 2008-04-03 Frank C. Porter

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 common problem in clinical trials is to test whether the effect of an explanatory variable on a response of interest is similar between two groups, e.g. patient or treatment groups. In this regard, similarity is defined as equivalence up…

Methodology · Statistics 2024-01-12 Niklas Hagemann , Giampiero Marra , Frank Bretz , Kathrin Möllenhoff

The recent availability of routine medical data, especially in a university-clinical context, may enable the discovery of typical healthcare pathways, i.e., typical temporal sequences of clinical interventions or hospital readmissions.…

Methodology · Statistics 2021-09-22 Nadine Binder , Kathrin Möllenhoff , August Sigle , Holger Dette

In this paper we discuss how to evaluate the differences between fitted logistic regression models across sub-populations. Our motivating example is in studying computerized diagnosis for learning disabilities, where sub-populations based…

Methodology · Statistics 2023-03-24 Guy Ashiri-Prossner , Yuval Benjamini

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt
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