Covariate imbalance and random allocation in clinical trials.

abstract：:In the recent two decades, data mining methods for signal detection have been developed for drug safety surveillance, using large post-market safety data. Several of these methods assume that the number of reports for each drug-adverse event combination is a Poisson random variable with mean proportional to the unknow...

journal_title：Statistics in medicine

authors： Hu N,Huang L,Tiwari RC

更新日期：2015-08-30 00:00:00

abstract：:Data monitoring committees (DMCs) have become an increasingly common component of randomized clinical trials in recent years. As experience has accumulated, and more individuals and organizations have become involved in such activities, a variety of approaches to the operation of such committees has inevitably arisen....

journal_title：Statistics in medicine

authors： Ellenberg SS

更新日期：2001-09-15 00:00:00

abstract：:The induced smoothing technique overcomes the difficulties caused by the non-smoothness in rank-based estimating functions for accelerated failure time models, but it is only natural when the estimating function has Gehan's weight. For a general weight, the induced smoothing method does not provide smooth estimating f...

journal_title：Statistics in medicine

authors： Chiou S,Kang S,Yan J

更新日期：2015-04-30 00:00:00

abstract：:In many chronic disease processes subjects are at risk of two or more types of events. We describe a bivariate mixed Poisson model in which a copula function is used to model the association between two gamma distributed random effects. The resulting model is a bivariate negative binomial process in which each type of...

journal_title：Statistics in medicine

authors： Cook RJ,Lawless JF,Lee KA

更新日期：2010-03-15 00:00:00

abstract：:We describe the usual statistical concepts and consequent appropriate simulations of a prospective study for the simple case of a single risk variable and an assumed logistic model. We examine the simulations of Lilienfeld and Pyne, and show that they are seriously flawed. Contrary to those authors' claims, the estima...

journal_title：Statistics in medicine

authors： Halperin M,Abbott RD,Blackwelder WC,Jacobowitz R,Lan KK,Verter JI,Wedel H

更新日期：1985-04-01 00:00:00

abstract：:In monitoring a clinical trial or other observational study with a survival endpoint, sometimes the numbers of patients entering and dying at each time point are presented, but the connections between them are kept confidential. Hence, the exact time to failure or censoring for each individual is missing. We refer to ...

journal_title：Statistics in medicine

authors： Wu Y,Shih WJ,Moore DF

更新日期：2007-08-30 00:00:00

abstract：:In this paper we examine the efficiency of a generalization of the traditional normal linear (LDA) or quadratic (QDA) discriminant analysis. This procedure (the generalized discriminant analysis, GDA) replaces each normal density used in the traditional classification rule by a Fourier series density estimator which '...

journal_title：Statistics in medicine

authors： Odom-Maryon T,Langholz B,Niland J,Azen S

更新日期：1991-03-01 00:00:00

abstract：:We describe rank-based approaches to assess principal stratification treatment effects in studies where the outcome of interest is only well-defined in a subgroup selected after randomization. Our methods are sensitivity analyses, in that estimands are identified by fixing a parameter and then we investigate the sensi...

journal_title：Statistics in medicine

authors： Lu X,Mehrotra DV,Shepherd BE

更新日期：2013-11-20 00:00:00

abstract：:In the pharmaceutical industry, some tests for quality assurance before, during and after the manufacture of a drug product involve a two-stage sampling technique. We propose statistical testing procedures in a two-stage sampling problem with large within-class sample sizes. We also derive a two-stage sampling plan by...

journal_title：Statistics in medicine

authors： Shao J,Chow SC

更新日期：1993-11-15 00:00:00

abstract：:In biomedical literature, numerous prediction models for clinical outcomes have been developed based either on clinical data or, more recently, on high-throughput molecular data (omics data). Prediction models based on both types of data, however, are less common, although some recent studies suggest that a suitable c...

journal_title：Statistics in medicine

authors： De Bin R,Sauerbrei W,Boulesteix AL

更新日期：2014-12-30 00:00:00

abstract：:The explanation of heterogeneity plays an important role in meta-analysis. The random effects meta-regression model allows the inclusion of trial-specific covariates which may explain a part of the heterogeneity. We examine the commonly used tests on the parameters in the random effects meta-regression with one covari...

journal_title：Statistics in medicine

authors： Knapp G,Hartung J

更新日期：2003-09-15 00:00:00

abstract：:Analyses to compare non-randomized groups are more and more common in both post hoc analyses of randomized clinical trials data and in analyses of long-term observational data. In such cases, it is quite likely that there are unknown or uncollected sources of heterogeneity in event rates. Research has shown that an un...

journal_title：Statistics in medicine

authors： Eberly LE

更新日期：2004-07-15 00:00:00

abstract：:Multi-type recurrent event data arise when two or more different kinds of events may occur repeatedly over a period of observation. The scientific objectives in such settings are often to describe features of the marginal processes and to study the association between the different types of events. Interval-censored m...

journal_title：Statistics in medicine

authors： Chen BE,Cook RJ,Lawless JF,Zhan M

更新日期：2005-03-15 00:00:00

abstract：:Several large-sample confidence intervals for the ratio of independent binomial proportions are compared in terms of exact coverage probability and width. A non-iterative approximate Bayesian interval is derived and its frequency properties are superior to all of the non-iterative confidence intervals considered. The ...

journal_title：Statistics in medicine

authors： Price RM,Bonett DG

更新日期：2008-11-20 00:00:00

abstract：:In many countries, the monitoring of child growth does not occur in a regular manner, and instead, we may have to rely on sporadic observations that are subject to substantial measurement error. In these countries, it can be difficult to identify patterns of poor growth, and faltering children may miss out on essentia...

journal_title：Statistics in medicine

authors： Anderson C,Xiao L,Checkley W

更新日期：2019-08-30 00:00:00

abstract：:Assessing the QT prolongation potential of a drug is typically done based on pivotal safety studies called thorough QT studies. Model-based estimation of the drug-induced QT prolongation at the estimated mean maximum drug concentration could increase efficiency over the currently used intersection-union test. However,...

journal_title：Statistics in medicine

authors： Dosne AG,Bergstrand M,Karlsson MO,Renard D,Heimann G

更新日期：2017-10-30 00:00:00

abstract：:Missing data arise in crossover trials, as they do in any form of clinical trial. Several papers have addressed the problems that missing data create, although almost all of these assume that the probability that a planned observation is missing does not depend on the value that would have been observed; that is, the ...

journal_title：Statistics in medicine

authors： Ho WK,Matthews JN,Henderson R,Farewell D,Rodgers LR

更新日期：2012-07-20 00:00:00

abstract：:In a variety of biomedical applications, particularly those involving screening for infectious diseases, testing individuals (e.g. blood/urine samples, etc.) in pools has become a standard method of data collection. This experimental design, known as group testing (or pooled testing), can provide a large reduction in ...

journal_title：Statistics in medicine

authors： Chen P,Tebbs JM,Bilder CR

更新日期：2009-10-15 00:00:00

abstract：:In many studies, interest lies in determining whether members of the study population will undergo a particular event of interest. Such scenarios are often termed 'mover-stayer' scenarios, and interest lies in modelling two sub-populations of 'movers' (those who have a propensity to undergo the event of interest) and ...

journal_title：Statistics in medicine

authors： O'Keeffe AG,Tom BD,Farewell VT

更新日期：2013-02-20 00:00:00

abstract：:Patient non-compliance and drop-out can bias analyses of clinical trial data. I describe a parametric model for treatment cross-over and drop-out and demonstrate how the concept of ignorability, originally defined for incomplete-data problems, can elucidate sources of bias in clinical trials. I discuss some implicatio...

journal_title：Statistics in medicine

authors： Heitjan DF

更新日期：1999-09-15 00:00:00

abstract：:A statistical definition of surrogate endpoints as well as validation criteria was first presented by Prentice. Freedman et al. supplemented these criteria with the so-called proportion explained. Buyse and Molenberghs pointed to inadequacies of these criteria and suggested a new definition of surrogacy based on (i) t...

journal_title：Statistics in medicine

authors： Molenberghs G,Geys H,Buyse M

更新日期：2001-10-30 00:00:00

abstract：:Testing the equality of 2 proportions for a control group versus a treatment group is a well-researched statistical problem. In some settings, there may be strong historical data that allow one to reliably expect that the control proportion is one, or nearly so. While one-sample tests or comparisons to historical cont...

journal_title：Statistics in medicine

authors： Gabriel EE,Nason M,Fay MP,Follmann DA

更新日期：2018-03-30 00:00:00

abstract：:In a cluster randomized cross-over trial, all participating clusters receive both intervention and control treatments consecutively, in separate time periods. Patients recruited by each cluster within the same time period receive the same intervention, and randomization determines order of treatment within a cluster. ...

journal_title：Statistics in medicine

authors： Turner RM,White IR,Croudace T,PIP Study Group.

更新日期：2007-01-30 00:00:00

abstract：:This article deals with determination of a sample size that guarantees the success of a trial. We follow a Bayesian approach and we say an experiment is successful if it yields a large posterior probability that an unknown parameter of interest (an unknown treatment effect or an effects-difference) is greater than a c...

journal_title：Statistics in medicine

authors： Brutti P,De Santis F,Gubbiotti S

更新日期：2008-06-15 00:00:00

abstract：:Most longitudinal growth curve models evaluate the evolution of each of the anthropometric measurements separately. When applied to a 'reference population', this exercise leads to univariate reference curves against which new individuals can be evaluated. However, growth should be evaluated in totality, that is, by e...

journal_title：Statistics in medicine

authors： Willemsen SP,Eilers PH,Steegers-Theunissen RP,Lesaffre E

更新日期：2015-04-15 00:00:00

abstract：:The two-period cross-over design with two sequences of drug administration is a standard experimental design when bioequivalence of one test formulation is to be assessed in comparison with a reference formulation. Previously, an approach based on Fieller's confidence interval has been presented for the assessment of ...

journal_title：Statistics in medicine

authors： Vuorinen J

更新日期：1997-10-15 00:00:00

abstract：:In many biomedical and epidemiological studies, data are often clustered due to longitudinal follow up or repeated sampling. While in some clustered data the cluster size is pre-determined, in others it may be correlated with the outcome of subunits, resulting in informative cluster size. When the cluster size is info...

journal_title：Statistics in medicine

authors： Chen Z,Zhang B,Albert PS

更新日期：2011-07-10 00:00:00

abstract：:When sample size is recalculated using unblinded interim data, use of the usual t-test at the end of a study may lead to an elevated type I error rate. This paper describes a numerical quadrature investigation to calculate the true probability of rejection as a function of the time of the recalculation, the magnitude ...

journal_title：Statistics in medicine

authors： Wittes J,Schabenberger O,Zucker D,Brittain E,Proschan M

更新日期：1999-12-30 00:00:00

abstract：:Meta-analysis to investigate the joint effect of multiple factors in the aetiology of a disease is of increasing importance in epidemiology. This task is often challenging in practice, because studies typically concentrate on studying the effect of only one exposure, sometimes may report the interaction between two ex...

journal_title：Statistics in medicine

authors： Salanti G,Higgins JP,White IR

更新日期：2006-12-30 00:00:00

abstract：:A good measure of field vaccine efficacy should evaluate the direct protective effect of vaccination on the person who receives the vaccine. The conventional estimator for vaccine efficacy depends on population level factors that are either unrelated or indirectly related to the direct biological action of the vaccine...

journal_title：Statistics in medicine

authors： Longini IM Jr,Halloran ME,Haber M,Chen RT

更新日期：1993-02-01 00:00:00