Statistical applications for in vitro diagnostic tests and other medical device clinical trials.

abstract：:For the assessment of biosimilarity of biosimilar products, the United States (US) Food and Drug Administration (FDA) proposed a stepwise approach for providing the totality-of-the-evidence of similarity between a proposed biosimilar product and a US-licensed (reference) product. The stepwise approach starts with the ...

journal_title：Journal of biopharmaceutical statistics

authors： Wang T,Chow SC

更新日期：2017-01-01 00:00:00

abstract：:The use of adaptive methods in clinical development has become very popular in recent years due to its flexibility in modifying trial procedures and/or statistical procedures of on-going clinical trials. Modifications to trial procedures are usually documented by protocol amendments. However, the actual patient popula...

journal_title：Journal of biopharmaceutical statistics

authors： Chow SC,Shao J

更新日期：2005-01-01 00:00:00

abstract：:The sequential parallel comparison design (SPCD) is a two-stage design recommended for trials with possibly high placebo response. A drug-placebo comparison in the first stage is followed in the second stage by placebo nonresponders being re-randomized between drug and placebo. We describe how SPCD can be used in tria...

journal_title：Journal of biopharmaceutical statistics

authors： Wang JJ,Ivanova A

更新日期：2014-01-01 00:00:00

abstract：:Nonparametric methods are presented for the analysis of the two-treatment, two-period crossover design with multivariate response. After forming within-subject sums and differences, the usual tests, including those for carry-over effects and direct treatment effects, can be constructed using a multivariate analysis of...

journal_title：Journal of biopharmaceutical statistics

authors： Johnson WD,Grender JM

更新日期：1993-03-01 00:00:00

abstract：:Dichotomizing a continuous biomarker is a common practice in medical research. Various methods exist in the literature for dichotomizing continuous biomarkers. The most widely adopted minimum p-value approach uses a sequence of test statistics for all possible dichotomizations of a continuous biomarker, and it chooses...

journal_title：Journal of biopharmaceutical statistics

authors： Su M,Fang L,Su Z

更新日期：2013-05-01 00:00:00

abstract：:The design of a three-arm trial including the experimental treatment, an active reference treatment, and a placebo is recommended as a useful approach to the assessment of noninferiority of the experimental treatment. The inclusion of the placebo arm enables the assessment of assay sensitivity and internal validation,...

journal_title：Journal of biopharmaceutical statistics

authors： Hida E,Tango T

更新日期：2013-01-01 00:00:00

abstract：:Bayesian decision procedures have already been proposed for and implemented in Phase I dose-escalation studies in healthy volunteers. The procedures have been based on pharmacokinetic responses reflecting the concentration of the drug in blood plasma and are conducted to learn about the dose-response relationship whil...

journal_title：Journal of biopharmaceutical statistics

authors： Whitehead J,Zhou Y,Hampson L,Ledent E,Pereira A

更新日期：2007-01-01 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 adverse events data of randomized clinical trials are often analyzed based on either crude incidence rates or exposure-adjusted incidence rates. These rates do not adequately account for an individual patient's profile of adverse events over the study period when an individual may remain in the trial after experie...

journal_title：Journal of biopharmaceutical statistics

authors： Siddiqui O

更新日期：2009-09-01 00:00:00

abstract：:The small n, Sequential, Multiple Assignment, Randomized Trial (snSMART) is a two-stage clinical trial design for rare diseases motivated by the comparison of three active treatments for isolated skin vasculitis in the ongoing clinical trial ARAMIS (a randomized multicenter study for isolated skin vasculitis, NCT09239...

journal_title：Journal of biopharmaceutical statistics

authors： Wei B,Braun TM,Tamura RN,Kidwell K

更新日期：2020-09-06 00:00:00

abstract：:In confidence interval estimation of the difference between two proportions with overdispersion due to positive correlations, the usual asymptotic normality-based method generally has lower coverage rates than desired, especially when sample size is moderate. Applying the concept of effective sample size to existing m...

journal_title：Journal of biopharmaceutical statistics

authors： Chen C,Li J,Zhou Z

更新日期：2004-05-01 00:00:00

abstract：:With the use of finite mixture models for the clustering of a data set, the crucial question of how many clusters there are in the data can be addressed by testing for the smallest number of components in the mixture model compatible with the data. We investigate the performance of a resampling approach to this latter...

journal_title：Journal of biopharmaceutical statistics

authors： McLachlan GJ,Rathnayake SI

更新日期：2011-11-01 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：:Using multistage adaptive group sequential test designs, the investigator may perform data-driven changes in the design during the course of the trial without inflation of the Type I error rate. This is possible, for example, through the use of the inverse normal method of combining the p-values from the separate stag...

journal_title：Journal of biopharmaceutical statistics

authors： Wassmer G

更新日期：2003-11-01 00:00:00

abstract：:Hypoglycemia is a major safety concern for diabetic patients. Hypoglycemic events can be modeled based on time to recurrent events or count data. In this article, we evaluated a gamma frailty model with variance estimated by the inverse of observed Fisher information matrix, a gamma frailty model with the sandwich var...

journal_title：Journal of biopharmaceutical statistics

authors： Ma C,Qu Y,Fu H

更新日期：2020-05-18 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：: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

abstract：:For approval of generic drugs, the U.S. Food and Drug Administration (FDA) requires the evidence of bioequivalence in average bioavailability be provided. This is based on the Fundmental Bioequivalence Assumption from FDA that if two drug products are shown to be bioequivalent, it is assumed that they are therapeutica...

journal_title：Journal of biopharmaceutical statistics

authors： Lu Y,Zhang ZZ,Chow SC

更新日期：2014-01-01 00:00:00

abstract：:To ensure that a drug product will meet standards for identity, strength and stability as specified in the United States Pharmacopedia and National Formulary (USP/NF), it needs to pass a number of tests such as the content uniformity test and dissolution test at various stages of the manufacturing process. The sponsor...

journal_title：Journal of biopharmaceutical statistics

authors： Wang H

更新日期：2007-01-01 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：: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：:Multiple comparisons are commonly seen in clinical trials and many other fields. An example, which is the focus of this paper, is the comparison of several test treatments (possibly different doses of a compound) with placebo (control). It is well known that steps must be taken to control the type I error rate when mu...

journal_title：Journal of biopharmaceutical statistics

authors： Chen M,Kianifard F

更新日期：1997-07-01 00:00:00

abstract：:There is often a need to determine parallelism or linearity between pairs of dose-response data sets for various biological applications. This article describes a technique based on a modification of the well-known extra-sum-of-squares principle of statistical regression. The standard extra-sum-of-squares method uses ...

journal_title：Journal of biopharmaceutical statistics

authors： Gottschalk PG,Dunn JR

更新日期：2005-01-01 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：:The United States Pharmacopeia (USP) content uniformity sampling acceptance plan consisting of a two-stage sampling plan with criteria on sample mean and number of out-of-range tablets is the standard for compendium. It is, however, often used mistakenly for lot quality assurance. In comparison to the Japan Phamacopei...

journal_title：Journal of biopharmaceutical statistics

authors： Tsong Y,Shen M

更新日期：2007-01-01 00:00:00

abstract：:Fisher's exact test and Pearson's chi-square with continuity correction are frequently employed in the analysis of epidemiological data involving a 2 x 2 contingency table. This paper reviews the concepts and controversies underlying these procedures and discusses their appropriateness and adequacies in analyzing such...

journal_title：Journal of biopharmaceutical statistics

authors： Sahai H,Khurshid A

更新日期：1995-03-01 00:00:00

abstract：:A model-based approach is developed to estimate the distribution of time from seroconversion to diagnosis with acquired immunodeficiency syndrome (AIDS) as a function of selected time-dependent covariates. The approach is applied to longitudinal data collected over 4 years of follow-up from 450 men seropositive for th...

journal_title：Journal of biopharmaceutical statistics

authors： Dunlop DD,Tamhane AC,Chmiel JS,Phair JP

更新日期：1994-07-01 00:00:00

abstract：:In cancer drug development, demonstrated efficacy in tumor xenograft models is an important step toward bringing a promising compound to human use. A key outcome variable is tumor volume measured over a period of time, while mice are treated with certain treatment regimens. A constrained parametric model has been prop...

journal_title：Journal of biopharmaceutical statistics

authors： Fang HB,Deng D,Zhang T,Tan M

更新日期：2014-01-01 00:00:00

abstract：:Group sequential methods to allow the possibility of early termination of a trial due to sufficiently convincing results are a standard in therapeutic clinical trials but have been little considered in bioequivalence trials. We investigate the statistical properties of one group sequential approach to bioequivalence t...

journal_title：Journal of biopharmaceutical statistics

authors： Hauck WW,Preston PE,Bois FY

更新日期：1997-03-01 00:00:00

abstract：:Predictive probability is particularly useful in aiding a decision-making process related to drug development. This is especially true for decisions occurring as the result of interim analyses of clinical trials. Examples of clinical trial applications of Bayesian predictive probability and the use of the beta-binomia...

journal_title：Journal of biopharmaceutical statistics

authors： Johns D,Andersen JS

更新日期：1999-03-01 00:00:00