Multivariate nonparametric analysis for the two-period crossover design with application in clinical trials.

abstract：:Laboratory data with a lower quantification limit (censored data) are sometimes analyzed by replacing non-quantifiable values with a single value equal to or less than the quantification limit, yielding possibly biased point estimates and variance estimates that are too small. Motivated by a three-period, three-treatm...

journal_title：Journal of biopharmaceutical statistics

authors： Karon JM,Wiegand RE,van de Wijgert JH,Kilmarx PH

更新日期：2015-01-01 00:00:00

abstract：:Investigating the prevalence of a disease is an important topic in medical studies. Such investigations are usually based on the classification results of a group of subjects according to whether they have the disease. To classify subjects, screening tests that are inexpensive and nonintrusive to the test subjects are...

journal_title：Journal of biopharmaceutical statistics

authors： Tang ML,Qiu SF,Poon WY,Tang NS

更新日期：2012-01-01 00:00:00

abstract：:The purpose of the research is to develop a statistical decision support algorithm for patients who may benefit from Adjuvant Cisplatin/Vinorelbine (ACT) and improve their survival rates. Genome-wide microarray data are used to identify feasible sets of genes and probe sets that constitute the gene signature. The data...

journal_title：Journal of biopharmaceutical statistics

authors： Moon H,Chao T,Ahn H

更新日期：2020-05-03 00:00:00

abstract：:We introduce the idea of a design to detect signals of efficacy in early phase clinical trials. Such a design features three possible decisions: to kill the compound; to continue with staged development; or to continue with accelerated development of the compound. We describe how such studies improve the trade-off bet...

journal_title：Journal of biopharmaceutical statistics

authors： Brown MJ,Chuang-Stein C,Kirby S

更新日期：2012-01-01 00:00:00

abstract：:The van Elteren test, as a type of stratified Wilcoxon-Mann-Whitney test for comparing two treatments accounting for stratum effects, has been used to replace the analysis of variance when the normality assumption was seriously violated. The sample size estimation methods for the van Elteren test have been proposed an...

journal_title：Journal of biopharmaceutical statistics

authors： Zhao YD,Qu Y,Rahardja D

更新日期：2006-01-01 00:00:00

abstract：:Some current approaches to modeling crossover trials in two treatments are critically reviewed from the perspective of the practical requirements of the drug developer. Particular attention is paid to the AB/BA design, and the inadequacies of the once popular two-stage procedure are discussed in detail. The use of bas...

journal_title：Journal of biopharmaceutical statistics

authors： Grieve A,Senn S

更新日期：1998-05-01 00:00:00

abstract：:ETRANK is a statistical software which uses nonparametric (randomization) technique to analyze incomplete repeated measures data, where the pattern of withdrawal is treatment related. This stand alone program is written in C, presently runs in MS-DOS, and a Windows version is in development. The program has been devel...

journal_title：Journal of biopharmaceutical statistics

authors： Entsuah R

更新日期：1996-11-01 00:00:00

abstract：:The concept of quality by design (QbD) as published in ICH-Q8 is currently one of the most recurrent topics in the pharmaceutical literature. This guideline recommends the use of information and prior knowledge gathered during pharmaceutical development studies to provide a scientific rationale for the manufacturing p...

journal_title：Journal of biopharmaceutical statistics

authors： Lebrun P,Giacoletti K,Scherder T,Rozet E,Boulanger B

更新日期：2015-01-01 00:00:00

abstract：:A method is proposed for the estimation of drug or toxicity potencies using in vitro data. A typical experiment in cancer research is presented where cells from a tumor-derived cell line were deposited as fixed volumes in 12-well cell culture plates. After waiting for 72 hours (for further growth), the wells were expo...

journal_title：Journal of biopharmaceutical statistics

authors： Le Chap T,Grambsch PM,Liu A

更新日期：2005-01-01 00:00:00

abstract：:In recent years, multi-regional clinical trials (MRCT) that conduct clinical trials simultaneously in Asian Pacific region, Europe, and the United States have become very popular for global pharmaceutical development. The main purpose of multi-regional clinical trials is to shorten the time for pharmaceutical developm...

journal_title：Journal of biopharmaceutical statistics

authors： Ying L,Song F,Chow SC,Zeng N,Zheng J,Li X,Henry D,Sethuraman V

更新日期：2018-01-01 00:00:00

abstract：:A useful testing strategy is proposed for a confirmatory phase III clinical trial. It consists of a combined test of superiority and test of equivalence, and it is easy to apply. By introducing the strategy, we can perform a post hoc analysis in a confirmatory experiment. Thus a more flexible decision will be possible...

journal_title：Journal of biopharmaceutical statistics

authors： Morikawa T,Yoshida M

更新日期：1995-11-01 00:00:00

abstract：:Large sample size imbalance is not uncommon in the biosimilar development. At the beginning of a product development, sample sizes of a biosimilar and a reference product may be limited. Thus, a sample size calculation may not be feasible. During the development stage, more batches of reference products may be added a...

journal_title：Journal of biopharmaceutical statistics

authors： Dong XC,Weng YT,Tsong Y

更新日期：2017-01-01 00:00:00

abstract：:A statistic, W, for measuring change from baseline is developed. Its distribution is found. Simulations using W and analysis of covariance (ANCOVA) are run and the results are compared. W is found to be less powerful than ANCOVA, yet is not seen to suffer some of the ill effects to which ANCOVA can fall prey, namely b...

journal_title：Journal of biopharmaceutical statistics

authors： Donahue RM

更新日期：1997-05-01 00:00:00

abstract：:Historical control trials (HCTs) are frequently conducted to compare an experimental treatment with a control treatment from a previous study, when they are applicable and favored over a randomized clinical trial (RCT) due to feasibility, ethics and cost concerns. Makuch and Simon developed a sample size formula for h...

journal_title：Journal of biopharmaceutical statistics

authors： Zhu H,Zhang S,Ahn C

更新日期：2016-01-01 00:00:00

abstract：:We studied the sensitivity of the number of unique design points and their placement, in Bayesian optimal designs for pharmacokinetic models, with respect to the magnitude of prior uncertainty. We used two and three-parameter pharmacokinetic models with fixed and mixed effects and two Bayesian optimal design criteria,...

journal_title：Journal of biopharmaceutical statistics

authors： Dokoumetzidis A,Aarons L

更新日期：2007-01-01 00:00:00

abstract：:Analyses of multicenter trials consider the estimated treatment effect differences of the individual centers and combine them into an estimate of the overall treatment effect. There has been much debate in the literature concerning the best way to combine these treatment effect differences. We emphasize that first of ...

journal_title：Journal of biopharmaceutical statistics

authors： Dragalin V,Fedorov V,Jones B,Rockhold F

更新日期：2001-11-01 00:00:00

abstract：:A new two-stage design is proposed that is suitable for early detection of the anticancer activity of experimental therapies in Phase II oncology trials. The endpoints of interest are response rate and early progression rate. The anticancer activity is defined by a positive signal in one endpoint and a non-negative si...

journal_title：Journal of biopharmaceutical statistics

authors： Sun LZ,Chen C,Patel K

更新日期：2009-01-01 00:00:00

abstract：:Recently, a design was proposed for the Simultaneous Global Drug Development Program (SGDDP) to assess the impact of ethnic factors on the effect of a new treatment for a targeted ethnic (TE) population. It used weighted Z tests to combine the information collected from the TE and non-TE (NTE) subgroups in the SGDDP b...

journal_title：Journal of biopharmaceutical statistics

authors： Liu K,Yuan Z,Chen G,Huang Q,Wenrich J

更新日期：2015-01-01 00:00:00

abstract：:The outcome selection of bioequivalence evaluations for abbreviated new drug applications results in bias towards unity of test-reference ratio estimates, and underestimation of intrasubject variability for the test drug product. In this study, the selection bias in the estimates of testreference ratio and intrasubjec...

journal_title：Journal of biopharmaceutical statistics

authors： Wang Y

更新日期：2000-08-01 00:00:00

abstract：:The problem of testing treatment difference in the occurrence of a study endpoint in a randomized parallel-group comparative clinical trial with repeated responses under the assumption that the responses follow a bivariate zero-inflated Poisson (ZIP) distribution is considered. Likelihood ratio test for homogeneity of...

journal_title：Journal of biopharmaceutical statistics

authors： Yuen HK,Chow SC,Tse SK

更新日期：2015-01-01 00:00:00

abstract：:Pressures for rapid drug development, especially for treatments that may affect public health significantly, drive a need to reconsider what is necessary to establish the "substantial evidence" of efficacy and safety required for regulatory approval. The concept of substantial evidence of effect can be stated fairly s...

journal_title：Journal of biopharmaceutical statistics

authors： Gould AL

更新日期：2002-02-01 00:00:00

abstract：:Rank-based test procedures in censored survival data differ considerably in their sensitivity to various alternatives to the hypothesis of equality of underlying distributions. Procedures based on the simultaneous use of multiple statistics provide an appealing approach to obtaining more global sensitivity while maint...

journal_title：Journal of biopharmaceutical statistics

authors： Yang P,Fleming TR

更新日期：2006-01-01 00:00:00

abstract：:This paper introduces exact permutation methods for use when there are independent clusters of data with arbitrary within-cluster correlation. To eliminate the problem of clustering, we randomly select a data point from each cluster and for this now independent data, and calculate our test statistic and the associated...

journal_title：Journal of biopharmaceutical statistics

authors： Follmann D,Fay M

更新日期：2010-07-01 00:00:00

abstract：:The issue of consistency and reliability arises frequently in applied clinical trials, and there is a considerable amount of literature on this topic. For a continuous random variable, we describe a process to assess whether the mean and variance are the same from one evaluation to the other and we derive the intracla...

journal_title：Journal of biopharmaceutical statistics

authors： Kao TC,Sparling Y,Rochon J

更新日期：2003-08-01 00:00:00

abstract：:For noninferiority testing with the maximum allowable noninferiority margin being prespecified, one can perform valid statistical testing at the same alpha level for multiple noninferiority hypotheses with margins being smaller than this maximum margin. This is easily comprehensible because only one confidence level i...

journal_title：Journal of biopharmaceutical statistics

authors： Hung HM,Wang SJ

更新日期：2004-05-01 00:00:00

abstract：:Receptor occupancy (RO) PET is a non-invasive way to determine drug on target. Given the complexity of procedures, long acquisition times, and high cost, ligand displacement imaging trials often have a limited size and produce sparse RO results over the time course of the blocking drug. To take the best advantage of t...

journal_title：Journal of biopharmaceutical statistics

authors： Vandenhende F,Renard D,Nie Y,Kumar A,Miller J,Tauscher J,Witcher J,Zhou Y,Wong DF

更新日期：2008-01-01 00:00:00

abstract：:The purpose of this paper is to describe, illustrate, and compare a number of different approaches to the analysis of repeated binary and categorical data. These approaches include empirical generalized least squares and generalized estimating equations, as well as traditional log-linear modeling methods. It is shown ...

journal_title：Journal of biopharmaceutical statistics

authors： Kenward MG,Jones B

更新日期：1992-01-01 00:00:00

abstract：:The Wilcoxon-Mann-Whitney (WMW) test is the most commonly used nonparametric method to compare two treatments when the underlying distribution of the outcome variable is not normally distributed. In the presence of stratum effects, the van Elteren (vE) test, a stratified WMW test, can be used to adjust for the stratum...

journal_title：Journal of biopharmaceutical statistics

authors： Qu Y,Zhao YD,Rahardja D

更新日期：2008-01-01 00:00:00

abstract：:A new three-arm parallel design was recently proposed to investigate the biosimilarity between a biological product and a reference product by using the relative distance. The purpose of this article is to extend their results to binary endpoints for three popular metrics: the risk difference, the log relative risk, a...

journal_title：Journal of biopharmaceutical statistics

authors： Shin W,Kang SH

更新日期：2016-01-01 00:00:00

abstract：:We evaluate properties of sample size re-estimation (SSR) designs similar to the promising zone design considered by Mehta and Pocock (2011). We evaluate these designs under the assumption of a true effect size of 1.1 down to 0.4 of the protocol-specified effect size by six measures: 1. The probability of a sample siz...

journal_title：Journal of biopharmaceutical statistics

authors： Gaffney M,Ware JH

更新日期：2017-01-01 00:00:00