Flexible design clinical trial methodology in regulatory applications.

abstract：:There are inconsistencies between the formulas for the variance of standardized mean difference (SMD) in the Cochrane Handbook for Systematic Reviews and the variance reported in other sources. Instead of the variance appropriate for the SMD of a crossover experiment, the Cochrane Handbook uses the variance appropriat...

journal_title：Statistics in medicine

authors： Kitchenham B,Madeyski L

更新日期：2020-11-30 00:00:00

abstract：:Within the rich literature on generalized linear models, substantial efforts have been devoted to models for categorical responses that are either completely ordered or completely unordered. Few studies have focused on the analysis of partially ordered outcomes, which arise in practically every area of study, includin...

journal_title：Statistics in medicine

authors： Zhang Q,Ip EH

更新日期：2012-01-13 00:00:00

abstract：:Recurrent event data are commonly encountered in health-related longitudinal studies. In this paper time-to-events models for recurrent event data are studied with non-informative and informative censorings. In statistical literature, the risk set methods have been confirmed to serve as an appropriate and efficient ap...

journal_title：Statistics in medicine

authors： Wang MC,Chiang CT

更新日期：2002-02-15 00:00:00

abstract：:We propose a two-step procedure to personalize drug dosage over time under the framework of a log-linear mixed-effect model. We model patients' heterogeneity using subject-specific random effects, which are treated as the realizations of an unspecified stochastic process. We extend the conditional quadratic inference ...

journal_title：Statistics in medicine

authors： Zhu X,Qu A

更新日期：2016-10-30 00:00:00

abstract：:Meta analysis is a collection of quantitative methods devoted to combine summary information from related but independent studies. Because research reports usually present only data reductions and summary statistics rather than detailed data, the reviewer must often resort to rather crude methods for constructing summ...

journal_title：Statistics in medicine

authors： Di Pietrantonj C

更新日期：2006-07-15 00:00:00

abstract：:Comparative studies of the accuracy of diagnostic procedures often use a paired design to gain in efficiency. Standard methods for analysing data from paired designs require complete observations. In many studies, however, one of the test results may be missing for some patients. In this paper, we propose a simple cor...

journal_title：Statistics in medicine

authors： Zhou XH,Gatsonis CA

更新日期：1996-08-15 00:00:00

abstract：:Multivariate outcomes measured longitudinally over time are common in medicine, public health, psychology and sociology. The typical (saturated) longitudinal multivariate regression model has a separate set of regression coefficients for each outcome. However, multivariate outcomes are often quite similar and many out...

journal_title：Statistics in medicine

authors： Jia J,Weiss RE

更新日期：2009-06-15 00:00:00

abstract：:We consider using observational data to estimate the effect of a treatment on disease recurrence, when the decision to initiate treatment is based on longitudinal factors associated with the risk of recurrence. The effect of salvage androgen deprivation therapy (SADT) on the risk of recurrence of prostate cancer is in...

journal_title：Statistics in medicine

authors： Kennedy EH,Taylor JM,Schaubel DE,Williams S

更新日期：2010-11-10 00:00:00

abstract：:Regression to the mean arises often in statistical applications where the units chosen for study relate to some observed characteristic in the extreme of its distribution. Gardner and Heady attribute the effect of regression to the mean to measurement errors. They assume the model Yi = U + ei, where U is a fixed withi...

journal_title：Statistics in medicine

authors： Johnson WD,George VT

更新日期：1991-08-01 00:00:00

abstract：:Many critical questions in medicine require the analysis of complex multivariate data, often from large data sets describing numerous variables for numerous subjects. In this paper, we describe CoPlot, a tool for visualizing multivariate data in medicine. CoPlot is an adaptation of multidimensional scaling (MDS) that ...

journal_title：Statistics in medicine

authors： Bravata DM,Shojania KG,Olkin I,Raveh A

更新日期：2008-05-30 00:00:00

abstract：:The use of classification and regression tree (CART) methodology is explored for the diagnosis of patients complaining of anterior chest pain. The results are compared with those previously obtained using correspondence analysis and independent Bayes classification. The technique is shown to be of potential value for ...

journal_title：Statistics in medicine

authors： Crichton NJ,Hinde JP,Marchini J

更新日期：1997-04-15 00:00:00

abstract：:The receiver operating characteristic (ROC) curve can be utilized to evaluate the performance of diagnostic tests. The area under the ROC curve (AUC) is a widely used summary index for comparing multiple ROC curves. Both parametric and nonparametric methods have been developed to estimate and compare the AUCs. However...

journal_title：Statistics in medicine

authors： Yao W,Li Z,Graubard BI

更新日期：2015-04-15 00:00:00

abstract：:The asymptotic Pearson's chi-squared test and Fisher's exact test have long been the most used for testing association in 2x2 tables. Unconditional tests preserve the significance level and generally are more powerful than Fisher's exact test for moderate to small samples, but previously were disadvantaged by being co...

journal_title：Statistics in medicine

authors： Lydersen S,Fagerland MW,Laake P

更新日期：2009-03-30 00:00:00

abstract：:Multilevel mixed effects survival models are used in the analysis of clustered survival data, such as repeated events, multicenter clinical trials, and individual participant data (IPD) meta-analyses, to investigate heterogeneity in baseline risk and covariate effects. In this paper, we extend parametric frailty model...

journal_title：Statistics in medicine

authors： Crowther MJ,Look MP,Riley RD

更新日期：2014-09-28 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：:Delay in the outcome variable is challenging for outcome-adaptive randomization, as it creates a lag between the number of subjects accrued and the information known at the time of the analysis. Motivated by a real-life pediatric ulcerative colitis trial, we consider a case where a short-term predictor is available fo...

journal_title：Statistics in medicine

authors： Kim MO,Liu C,Hu F,Lee JJ

更新日期：2014-10-15 00:00:00

abstract：:The mutation and natural selection phenomenon can and often does cause the failure of antimicrobial, herbicidal, pesticide and cancer treatments selection pressures. This phenomenon operates in a mathematically predictable behavior, which when understood leads to approaches to reduce and prevent the failure of the use...

journal_title：Statistics in medicine

authors： Kleinman A

更新日期：2014-12-20 00:00:00

abstract：:This paper discusses design considerations and the role of randomization-based inference in randomized community intervention trials. We stress that longitudinal follow-up of cohorts within communities often yields useful information on the effects of intervention on individuals, whereas cross-sectional surveys can us...

journal_title：Statistics in medicine

authors： Gail MH,Mark SD,Carroll RJ,Green SB,Pee D

更新日期：1996-06-15 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：:The present study investigates the performance of several statistical tests to detect publication bias in diagnostic meta-analysis by means of simulation. While bivariate models should be used to pool data from primary studies in diagnostic meta-analysis, univariate measures of diagnostic accuracy are preferable for t...

journal_title：Statistics in medicine

authors： Bürkner PC,Doebler P

更新日期：2014-08-15 00:00:00

abstract：:In many studies, a K degree of freedom large sample chi2 test is used to assess the effect of treatment on a multivariate response, such as an omnibus T2-like test of a difference between two treatment groups in any of K repeated measures. Alternately, a K df chi2 test may be used to test the equality of K+1 groups in...

journal_title：Statistics in medicine

authors： Lachin JM,Greenhouse SW,Bautista OM

更新日期：2003-11-15 00:00:00

abstract：:When designing a study to develop a new prediction model with binary or time-to-event outcomes, researchers should ensure their sample size is adequate in terms of the number of participants (n) and outcome events (E) relative to the number of predictor parameters (p) considered for inclusion. We propose that the mini...

journal_title：Statistics in medicine

authors： Riley RD,Snell KI,Ensor J,Burke DL,Harrell FE Jr,Moons KG,Collins GS

更新日期：2019-03-30 00:00:00

abstract：:We extend the shared frailty model of recurrent events and a dependent terminal event to allow for a nonparametric covariate function. We include a Gaussian random effect (frailty) in the intensity functions of both the recurrent and terminal events to capture correlation between the two processes. We employ the penal...

journal_title：Statistics in medicine

authors： Zhangsheng Y,Liu L

更新日期：2011-09-30 00:00:00

abstract：:The relationship between association and surrogacy has been the focus of much debate in the surrogate marker literature. Recently, the individual causal association (ICA) has been introduced as a metric of surrogacy in the causal inference framework, when both the surrogate and the true endpoint are normally distribut...

journal_title：Statistics in medicine

authors： Meyvisch P,Alonso A,Van der Elst W,Molenberghs G

更新日期：2020-11-20 00:00:00

abstract：:We consider evaluation and comparison of the diagnostic accuracy of biomarkers with continuous test outcomes, possibly correlated due to repeated measurements. We develop nonparametric group sequential testing procedures to evaluate and compare the area of biomarkers under their receiver operating characteristic curve...

journal_title：Statistics in medicine

authors： Liu A,Wu C,Schisterman EF

更新日期：2008-05-10 00:00:00

abstract：:This paper concerns the statistical analysis of certain binary data arising in molecular studies of cancer. In allelic-loss experiments, tumour cell genomes are analysed at informative molecular marker loci to identify deleted chromosomal regions. The resulting binary data are used to infer properties of putative supp...

journal_title：Statistics in medicine

authors： Newton MA,Gould MN,Reznikoff CA,Haag JD

更新日期：1998-07-15 00:00:00

abstract：:Multivariate finite mixture models have been applied to the identification of dietary patterns. These models are known to have many parameters, and consequently large samples are usually required. We present a special case of a multivariate mixture model that reduces the number of parameters to be estimated and seems ...

journal_title：Statistics in medicine

authors： Rita Gaio A,Costa JP,Santos AC,Ramos E,Lopes C

更新日期：2012-08-30 00:00:00

abstract：:A popular method for analysing repeated-measures data is generalized estimating equations (GEE). When response data are missing at random (MAR), two modifications of GEE use inverse-probability weighting and imputation. The weighted GEE (WGEE) method involves weighting observations by their inverse probability of bein...

journal_title：Statistics in medicine

authors： Seaman S,Copas A

更新日期：2009-03-15 00:00:00

abstract：:This paper reviews Bayesian strategies for monitoring clinical trial data. It focuses on a Bayesian stochastic curtailment method based on the predictive probability of observing a clinically significant outcome at the scheduled end of the study given the observed data. The proposed method is applied to derive efficac...

journal_title：Statistics in medicine

authors： Dmitrienko A,Wang MD

更新日期：2006-07-15 00:00:00

abstract：:Conditional power (CP) is the probability that the final study result will be statistically significant, given the data observed thus far and a specific assumption about the pattern of the data to be observed in the remainder of the study, such as assuming the original design effect, or the effect estimated from the c...

journal_title：Statistics in medicine

authors： Lachin JM

更新日期：2005-09-30 00:00:00