Scientific considerations for assessing biosimilar products.

abstract：:Tree-based methods have become popular for analyzing complex data structures where the primary goal is risk stratification of patients. Ensemble techniques improve the accuracy in prediction and address the instability in a single tree by growing an ensemble of trees and aggregating. However, in the process, individua...

journal_title：Statistics in medicine

authors： Banerjee M,Ding Y,Noone AM

更新日期：2012-07-10 00:00:00

abstract：:This paper develops methods of analysis for active extension clinical trials. Under this design, patients are randomized to treatment or placebo for a period of time (period 1), and then all patients receive treatment for an additional period of time (period 2). We assume a continuous outcome is measured at baseline a...

journal_title：Statistics in medicine

authors： Hu Z,Follmann D

更新日期：2007-05-30 00:00:00

abstract：:In clinical research, we are often interested in assessing how a biomarker changes with time, and whether it could be used as a surrogate marker when evaluating the efficacy of a new drug. However, when the longitudinal marker is correlated with survival, linear mixed models for longitudinal data may be inappropriate....

journal_title：Statistics in medicine

authors： Deslandes E,Chevret S

更新日期：2007-12-30 00:00:00

abstract：:Most phase I dose-finding methods in oncology aim to find the maximum-tolerated dose from a set of prespecified doses. However, in practice, because of a lack of understanding of the true dose-toxicity relationship, it is likely that none of these prespecified doses are equal or reasonably close to the true maximum-to...

journal_title：Statistics in medicine

authors： Chu Y,Pan H,Yuan Y

更新日期：2016-09-10 00:00:00

abstract：:The case-control study is a simple and an useful method to characterize the effect of a gene, the effect of an exposure, as well as the interaction between the two. The control-free case-only study is yet an even simpler design, if interest is centered on gene-environment interaction only. It requires the sometimes pl...

journal_title：Statistics in medicine

authors： Lee WC,Wang LY,Cheng KF

更新日期：2010-10-30 00:00:00

abstract：:Quality of life (QOL) assessment is a key component of many clinical studies and frequently requires the use of single global summary measures that capture the overall balance of findings from a potentially wide-ranging assessment of QOL issues. We propose and evaluate an irregular multilevel latent variable model sui...

journal_title：Statistics in medicine

authors： Kifley A,Heller GZ,Beath KJ,Bulger D,Ma J,Gebski V

更新日期：2012-05-20 00:00:00

abstract：:In epidemiological studies we often want to learn about the direct effect of an exposure on an outcome, i.e. the effect that is not relayed by a specific intermediate variable. In the literature, there are two common definitions of direct effects; controlled and natural. When the intermediate variable and the outcome ...

journal_title：Statistics in medicine

authors： Sjölander A

更新日期：2009-02-15 00:00:00

abstract：:In randomised trials, continuous endpoints are often measured with some degree of error. This study explores the impact of ignoring measurement error and proposes methods to improve statistical inference in the presence of measurement error. Three main types of measurement error in continuous endpoints are considered:...

journal_title：Statistics in medicine

authors： Nab L,Groenwold RHH,Welsing PMJ,van Smeden M

更新日期：2019-11-30 00:00:00

abstract：:A problem occurring in chronic disease modeling is the estimation of transition probabilities of moving from one state of a categorical risk factor to another. Transitions could be obtained from a cohort study, but often such data may not be available. However, under the assumption that transitions remain stable over ...

journal_title：Statistics in medicine

authors： Kassteele Jv,Hoogenveen RT,Engelfriet PM,Baal PH,Boshuizen HC

更新日期：2012-03-15 00:00:00

abstract：:The Box-Cox power exponential (BCPE) distribution, developed in this paper, provides a model for a dependent variable Y exhibiting both skewness and kurtosis (leptokurtosis or platykurtosis). The distribution is defined by a power transformation Y(nu) having a shifted and scaled (truncated) standard power exponential ...

journal_title：Statistics in medicine

authors： Rigby RA,Stasinopoulos DM

更新日期：2004-10-15 00:00:00

abstract：:In observational studies, many continuous or categorical covariates may be related to an outcome. Various spline-based procedures or the multivariable fractional polynomial (MFP) procedure can be used to identify important variables and functional forms for continuous covariates. This is the main aim of an explanatory...

journal_title：Statistics in medicine

authors： Binder H,Sauerbrei W,Royston P

更新日期：2013-06-15 00:00:00

abstract：:We present a method to determine the number of subjects, k, and number of repeated measurements, n, that minimize the overall cost of conducting a reliability study, while providing acceptable power for tests of hypotheses concerning the reliability coefficient rho. Tables showing optimal choices of k and n under vari...

journal_title：Statistics in medicine

authors： Eliasziw M,Donner A

更新日期：1987-09-01 00:00:00

abstract：:Studies based on aggregated hospital outcome data have established that there is a relationship between nurse staffing and adverse events. However, this result could not be confirmed in Belgium where 96 per cent of the variability of nurse staffing levels over nursing units (belonging to different hospitals) is explai...

journal_title：Statistics in medicine

authors： Diya L,Lesaffre E,Van den Heede K,Sermeus W,Vleugels A

更新日期：2010-03-30 00:00:00

abstract：:Testing involving the intra-class kappa coefficient is commonly performed in order to assess agreement involving categorical ratings. A number of procedures have been proposed, which make use of the limiting null distribution as the sample size goes to infinity in order to compute the observed significance. As with ma...

journal_title：Statistics in medicine

authors： Wilding GE,Consiglio JD,Shan G

更新日期：2014-07-30 00:00:00

abstract：:This paper presents tables analogous to T-tables for use in the 2 x 2 multinomial trial, where the continuity corrected Z-statistic is used to make exact unconditional inference. This is the first solution of a discrete exact unconditional inference problem involving a multivariate nuisance parameter for which no anci...

journal_title：Statistics in medicine

authors： Shuster JJ

更新日期：1992-05-01 00:00:00

abstract：:We simulated multinomial AIDS incidence counts from 27 'representative' AIDS epidemics that spanned a period corresponding to previous applications of backcalculation (1 January 1977 to 1 July 1987) and assessed mean square error for several back-calculated estimators of HIV prevalence and short-term AIDS projections....

journal_title：Statistics in medicine

authors： Rosenberg PS,Gail MH,Pee D

更新日期：1991-08-01 00:00:00

abstract：:In genetic association studies, it is typically thought that genetic variants and environmental variables jointly will explain more of the inheritance of a phenotype than either of these two components separately. Traditional methods to identify gene-environment interactions typically consider only one measured enviro...

journal_title：Statistics in medicine

authors： Pashova H,LeBlanc M,Kooperberg C

更新日期：2013-01-30 00:00:00

abstract：:We consider bivariate survival times for heterogeneous populations, where heterogeneity induces deviations in an individual's risk of an event as well as associations between survival times. The heterogeneity is characterized by a bivariate frailty model. We measure the heterogeneity effects through deviations associa...

journal_title：Statistics in medicine

authors： Xue X,Ding Y

更新日期：1999-04-30 00:00:00

abstract：:In this paper, we define a modified version τ(b) of Kendall's tau to measure the association in a pair (X,Y) of random variables subject to fixed left censoring due to known lower detection limits. We provide a nonparametric estimator of τ(b) and investigate its asymptotic properties. We then assume an Archimedean cop...

journal_title：Statistics in medicine

authors： Romdhani H,Lakhal-Chaieb L

更新日期：2011-11-20 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：:The general linear mixed model provides a useful approach for analysing a wide variety of data structures which practising statisticians often encounter. Two such data structures which can be problematic to analyse are unbalanced repeated measures data and longitudinal data. Owing to recent advances in methods and sof...

journal_title：Statistics in medicine

authors： Cnaan A,Laird NM,Slasor P

更新日期：1997-10-30 00:00:00

abstract：:Group sequential testing procedures have been proposed as an approach to conserving resources in biomarker validation studies. Previously, we derived the asymptotic properties of the sequential empirical positive predictive value (PPV) and negative predictive value (NPV) curves, which summarize the predictive accuracy...

journal_title：Statistics in medicine

authors： Koopmeiners JS,Feng Z

更新日期：2016-04-15 00:00:00

abstract：:Optimal prevention and treatment strategies for a disease of multiple causes, such as pneumonia, must be informed by the population distribution of causes among cases, or cause-specific case fractions (CSCFs). CSCFs may further depend on additional explanatory variables. Existing methodological literature in disease e...

journal_title：Statistics in medicine

authors： Wu Z,Chen I

更新日期：2021-02-20 00:00:00

abstract：:This paper assesses the coverage probability of commonly used confidence intervals for the standardized mortality ratio (SMR) when death certificates are missing. It also proposes alternative confidence interval approaches with coverage probabilities close to .95. In epidemiology, the SMR is an important measure of ri...

journal_title：Statistics in medicine

authors： Timkova J,Kotik L,Tomasek L

更新日期：2017-11-30 00:00:00

abstract：:Statistical methods are available for performing a meta-analysis when the response variable of interest is the same in each study. Problems arise when studies exploring a common therapeutic question use different patient response types. This article presents statistical methods for combining studies which involve diff...

journal_title：Statistics in medicine

authors： Whitehead A,Jones NM

更新日期：1994-12-15 00:00:00

abstract：:A calibration line is used to define the relationship between a new clinical technique and a standard in vitro laboratory methodology. Discrimination intervals quantify the reliability of inverse estimates obtained from the calibration line. Applied to transcutaneous PCO2 monitoring, a new in vivo measurement, discrim...

journal_title：Statistics in medicine

authors： Kost GJ

更新日期：1986-07-01 00:00:00

abstract：:Huntington's disease (HD) is a neurodegenerative disorder with a dominant genetic mode of inheritance caused by an expansion of CAG repeats on chromosome 4. Typically, a longer sequence of CAG repeat length is associated with increased risk of experiencing earlier onset of HD. Previous studies of the association betwe...

journal_title：Statistics in medicine

authors： Ma Y,Wang Y

更新日期：2014-04-15 00:00:00

abstract：:Linear mixed effects (LME) models are increasingly used for analyses of biological and biomedical data. When the multivariate normal assumption is not adequate for an LME model, then a robust estimation approach is preferable to the maximum likelihood one. M-estimators were considered before for robust estimation of t...

journal_title：Statistics in medicine

authors： Chervoneva I,Vishnyakov M

更新日期：2011-06-30 00:00:00

abstract：:A method is proposed to infer the randomized treatment effect on survival after an adjustment for a post-randomization variable. The post-randomization variable is made independent of the treatment assignment and is considered a surrogate for baseline prognostic factors. The relationship between the post-randomization...

journal_title：Statistics in medicine

authors： Heller G

更新日期：2001-11-30 00:00:00

abstract：:Often multiple outcomes are of interest in each study identified by a systematic review, and in this situation a separate univariate meta-analysis is usually applied to synthesize the evidence for each outcome independently; an alternative approach is a single multivariate meta-analysis model that utilizes any correla...

journal_title：Statistics in medicine

authors： Riley RD,Abrams KR,Lambert PC,Sutton AJ,Thompson JR

更新日期：2007-01-15 00:00:00