Efficient two-stage sequential arrays of proof of concept studies for pharmaceutical portfolios.

abstract：:Semicompeting risks and interval censoring are frequent in medical studies, for instance when a disease may be diagnosed only at times of visit and disease onset is in competition with death. To evaluate the ability of markers to predict disease onset in this context, estimators of discrimination measures must account...

journal_title：Statistical methods in medical research

authors： Jacqmin-Gadda H,Blanche P,Chary E,Touraine C,Dartigues JF

更新日期：2016-12-01 00:00:00

abstract：:Understanding the limitation of solely relying on statistical significance, researchers have proposed methods to draw biomedical conclusions based on clinical significance. The minimal clinically important significance is one of the most fundamental concepts to study clinical significance. Based on an anchor question ...

journal_title：Statistical methods in medical research

authors： Zhou Z,Zhao J,Bisson LJ

更新日期：2020-03-01 00:00:00

abstract：:Monte Carlo evaluation of resampling-based tests is often conducted in statistical analysis. However, this procedure is generally computationally intensive. The pooling resampling-based method has been developed to reduce the computational burden but the validity of the method has not been studied before. In this arti...

journal_title：Statistical methods in medical research

authors： Fung WK,Yu K,Yang Y,Zhou JY

更新日期：2018-05-01 00:00:00

abstract：:Statistical methods for carrying out asymptotic inferences (tests or confidence intervals) relative to one or two independent binomial proportions are very frequent. However, inferences about a linear combination of K independent proportions L = Σβ(i)p(i) (in which the first two are special cases) have had very little...

journal_title：Statistical methods in medical research

authors： Martín Andrés A,Alvarez Hernández M,Herranz Tejedor I

更新日期：2011-08-01 00:00:00

abstract：:Censored data make survival analysis more complicated because exact event times are not observed. Statistical methodology developed to account for censored observations assumes that patients' withdrawal from a study is independent of the event of interest. However, in practice, some covariates might be associated to b...

journal_title：Statistical methods in medical research

authors： Willems S,Schat A,van Noorden MS,Fiocco M

更新日期：2018-02-01 00:00:00

abstract：:Nonlinear mixed-effects modeling is one of the most popular tools for analyzing repeated measurement data, particularly for applications in the biomedical fields. Multiple integration and nonlinear optimization are the two major challenges for likelihood-based methods in nonlinear mixed-effects modeling. To solve thes...

journal_title：Statistical methods in medical research

authors： Yuan M,Zhu Z,Yang Y,Zhao M,Sasser K,Hamadeh H,Pinheiro J,Xu XS

更新日期：2020-08-24 00:00:00

abstract：:Cancer mortality risk estimates are essential for planning resource allocation and designing and evaluating cancer prevention and management strategies. However, mortality figures generally become available after a few years, making necessary to develop reliable procedures to provide current and near future mortality ...

journal_title：Statistical methods in medical research

authors： Ugarte MD,Goicoa T,Etxeberria J,Militino AF

更新日期：2012-10-01 00:00:00

abstract：:Accelerated failure time model is a popular model to analyze censored time-to-event data. Analysis of this model without assuming any parametric distribution for the model error is challenging, and the model complexity is enhanced in the presence of large number of covariates. We developed a nonparametric Bayesian met...

journal_title：Statistical methods in medical research

authors： Zhang Z,Sinha S,Maiti T,Shipp E

更新日期：2018-04-01 00:00:00

abstract：:Pattern-mixture model (PMM)-based controlled imputations have become a popular tool to assess the sensitivity of primary analysis inference to different post-dropout assumptions or to estimate treatment effectiveness. The methodology is well established for continuous responses but less well established for binary res...

journal_title：Statistical methods in medical research

authors： Lu K

更新日期：2020-12-01 00:00:00

abstract：:This article is motivated by the need for discovering patterns of patients' health based on their daily settings of care to aid the health policy-makers to improve the effectiveness of distributing funding for health services. The hidden process of one's health status is assumed to be a continuous smooth function, cal...

journal_title：Statistical methods in medical research

authors： Wang S,Nie Y,Sutherland JM,Wang L

更新日期：2020-09-25 00:00:00

abstract：:Sample selection arises when the outcome of interest is partially observed in a study. Although sophisticated statistical methods in the parametric and non-parametric framework have been proposed to solve this problem, it is yet unclear how to deal with selectively missing covariate data using simple multiple imputati...

journal_title：Statistical methods in medical research

authors： Ogundimu EO,Collins GS

更新日期：2019-01-01 00:00:00

abstract：:The change-point model has drawn much attention over the past few decades. It can accommodate the jump process, which allows for changes of the effects before and after the change point. Intellectual disability is a long-term disability that impacts performance in cognitive aspects of life and usually has its onset pr...

journal_title：Statistical methods in medical research

authors： Cai B,Lawson AB,McDermott S,Aelion CM

更新日期：2016-04-01 00:00:00

abstract：:Many non-experimental studies use propensity-score methods to estimate causal effects by balancing treatment and control groups on a set of observed baseline covariates. Full matching on the propensity score has emerged as a particularly effective and flexible method for utilizing all available data, and creating well...

journal_title：Statistical methods in medical research

authors： Austin PC,Stuart EA

更新日期：2017-12-01 00:00:00

abstract：:Spatial resolution plays an important role in functional magnetic resonance imaging studies as the signal-to-noise ratio increases linearly with voxel volume. In scientific studies, where functional magnetic resonance imaging is widely used, the standard spatial resolution typically used is relatively low which ensure...

journal_title：Statistical methods in medical research

authors： Liu Z,Bartsch AJ,Berrocal VJ,Johnson TD

更新日期：2019-04-01 00:00:00

abstract：:For semi-continuous data which are a mixture of true zeros and continuously distributed positive values, the use of two-part mixed models provides a convenient modelling framework. However, deriving population-averaged (marginal) effects from such models is not always straightforward. Su et al. presented a model that ...

journal_title：Statistical methods in medical research

authors： Tom BD,Su L,Farewell VT

更新日期：2016-10-01 00:00:00

abstract：:This paper illustrates the use of multidimensional scaling methods (MDS) to examine space-time patterns in epidemic data. The paper begins by outlining the principles of MDS. The model is then formally specified and illustrated by application to two data sets. The first is partly a tutorial example. It uses monthly re...

journal_title：Statistical methods in medical research

authors： Cliff AD,Haggett P,Smallman-Raynor MR,Stroup DF,Williamson GD

更新日期：1995-06-01 00:00:00

abstract：:Repeated measures and multivariate outcomes are an increasingly common feature of trials. Their joint analysis by means of random effects and latent variable models is appealing but patterns of heterogeneity in outcome profile may not conform to standard multivariate normal assumptions. In addition, there is much inte...

journal_title：Statistical methods in medical research

authors： Pickles A,Croudace T

更新日期：2010-06-01 00:00:00

abstract：:This paper reviews models for incomplete continuous and categorical longitudinal data. In terms of Rubin's classification of missing value processes we are specifically concerned with the problem of nonrandom missingness. A distinction is drawn between the classes of selection and pattern-mixture models and, using sev...

journal_title：Statistical methods in medical research

authors： Kenward MG,Molenberghs G

更新日期：1999-03-01 00:00:00

abstract：:Aim To present a flexible model for repeated measures longitudinal growth data within individuals that allows trends over time to incorporate individual-specific random effects. These may reflect the timing of growth events and characterise within-individual variability which can be modelled as a function of age. Subj...

journal_title：Statistical methods in medical research

authors： Goldstein H,Leckie G,Charlton C,Tilling K,Browne WJ

更新日期：2018-11-01 00:00:00

abstract：:Mixed models are widely used for the analysis of one repeatedly measured outcome. If more than one outcome is present, a mixed model can be used for each one. These separate models can be tied together into a multivariate mixed model by specifying a joint distribution for their random effects. This strategy has been u...

journal_title：Statistical methods in medical research

authors： Fieuws S,Verbeke G,Molenberghs G

更新日期：2007-10-01 00:00:00

abstract：:We propose a cross-validated area under the receiving operator characteristic (ROC) curve (CV-AUC) criterion for tuning parameter selection for penalized methods in sparse, high-dimensional logistic regression models. We use this criterion in combination with the minimax concave penalty (MCP) method for variable selec...

journal_title：Statistical methods in medical research

authors： Jiang D,Huang J,Zhang Y

更新日期：2013-10-01 00:00:00

abstract：:Metagenomics enables the study of gene abundances in complex mixtures of microorganisms and has become a standard methodology for the analysis of the human microbiome. However, gene abundance data is inherently noisy and contains high levels of biological and technical variability as well as an excess of zeros due to ...

journal_title：Statistical methods in medical research

authors： Jonsson V,Österlund T,Nerman O,Kristiansson E

更新日期：2019-12-01 00:00:00

abstract：:In many experiments and especially in translational and preclinical research, sample sizes are (very) small. In addition, data designs are often high dimensional, i.e. more dependent than independent replications of the trial are observed. The present paper discusses the applicability of max t-test-type statistics (mu...

journal_title：Statistical methods in medical research

authors： Konietschke F,Schwab K,Pauly M

更新日期：2020-11-24 00:00:00

abstract：:The ability to evaluate effects of factors on outcomes is increasingly important for studies that control some but not all of the factors. Although important advances have been made in methods of analysis for such partially controlled studies, work on designs has been limited. To help understand why, we review the mai...

journal_title：Statistical methods in medical research

authors： Li F,Frangakis CE

更新日期：2005-08-01 00:00:00

abstract：:Regression models are frequently used to model the functional relationship between an interesting outcome parameter and one or more potentially relevant explanatory variables. Objectives can be to set up as a prognostic model, for example, or an estimation model for a certain parameter of interest. Determining half-li...

journal_title：Statistical methods in medical research

authors： Mayer B,Keller F,Syrovets T,Wittau M

更新日期：2016-10-01 00:00:00

abstract：:In biomedical research, various longitudinal markers measuring different quantities are often collected over time. For example, repeated measures of psychometric scores are very informative about the degradation process toward dementia. These trajectories are generally nonlinear with an acceleration of the decline a f...

journal_title：Statistical methods in medical research

authors： Segalas C,Helmer C,Jacqmin-Gadda H

更新日期：2020-09-01 00:00:00

abstract：:I comment here on a recent paper in this journal, on the fitting of truncated normal distributions by the EM algorithm. I show that the fitting of such distributions by direct numerical maximization of likelihood (rather than EM) is straightforward, contrary to an assertion made by the authors of that paper. ...

journal_title：Statistical methods in medical research

authors： MacDonald IL

更新日期：2018-12-01 00:00:00

abstract：:In recent years, disease mapping studies have become a routine application within geographical epidemiology and are typically analysed within a Bayesian hierarchical model formulation. A variety of model formulations for the latent level have been proposed but all come with inherent issues. In the classical BYM (Besag...

journal_title：Statistical methods in medical research

authors： Riebler A,Sørbye SH,Simpson D,Rue H

更新日期：2016-08-01 00:00:00

abstract：:Tracking a subject's risk factors or health status over time is an important objective in long-term epidemiological studies with repeated measurements. An important issue of time-trend tracking is to define appropriate statistical indices to quantitatively measure the tracking abilities of the targeted risk factors or...

journal_title：Statistical methods in medical research

authors： Wu CO,Tian X,Tian L,Reis JP,Zhao L,Allen NB,Bae S,Liu K

更新日期：2020-02-01 00:00:00

abstract：:This article aims to develop a probability-based model involving the use of direct likelihood formulation and generalised linear modelling (GLM) approaches useful in estimating important disease parameters from longitudinal or repeated measurement data. The current application is based on infection with respiratory sy...

journal_title：Statistical methods in medical research

authors： Mwambi H,Ramroop S,White Lj,Okiro E,Nokes Dj,Shkedy Z,Molenberghs G

更新日期：2011-10-01 00:00:00