Simultaneous confidence interval methods for analytical similarity assessment.

abstract：:In many clinical trials for chronic conditions a run-in period is used prior to randomization. Often, only those participants who meet certain criteria during the run-in phase go on to get randomized. The others, along with the information that they might have provided, are excluded from the study. This exclusion of t...

journal_title：Journal of biopharmaceutical statistics

authors： Berger VW,Vali B

更新日期：2011-03-01 00:00:00

abstract：:The dose-response models for full agonists and for a particular type of partial agonist can be described by sigmoidal curves and bell-shaped curves, respectively. The methods currently used to evaluate the interaction of a full agonist and a partial agonist require a large number of experimental units and base their a...

journal_title：Journal of biopharmaceutical statistics

authors： Ghosh K,Shen ES,Arey BJ,López FJ

更新日期：1998-11-01 00:00:00

abstract：:Sample size calculation formulas for testing equality, noninferiority, superiority, and equivalence based on odds ratio were derived under both parallel and one-arm crossover designs. An example concerning the study of odds ratio between a test compound (treatment) and a standard therapy (control) for prevention of re...

journal_title：Journal of biopharmaceutical statistics

authors： Wang H,Chow SC,Li G

更新日期：2002-11-01 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：:In the early stages of traditional drug development, the frequency of dosing (e.g., QD, BID, etc.) is typically determined by the pharmacokinetic properties of a compound. After an appropriate dose frequency is chosen, the magnitude of dose is then evaluated via parallel-group dose-response trials. For some drugs, how...

journal_title：Journal of biopharmaceutical statistics

authors： Hafner KB,Ruberg SJ

更新日期：1996-07-01 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：:Bowker's test, a generalization of McNemar's test, performs well under the hypothesis of symmetry, but the estimator of variance used in the test is biased when the table is asymmetric and this calls into question the test's performance in non-null situations. We seek an alternative to Bowker's test in search of metho...

journal_title：Journal of biopharmaceutical statistics

authors： May WL,Johnson WD

更新日期：2001-02-01 00:00:00

abstract：:This article deals with seven special issues related to the assumptions, applicability, and practical use of formulas for calculating power or sample size, respectively, for comparative clinical trials with time-to-event endpoints, with particular focus on the well-known Freedman and Schoenfeld methods. All problems a...

journal_title：Journal of biopharmaceutical statistics

authors： Abel UR,Jensen K,Karapanagiotou-Schenkel I,Kieser M

更新日期：2015-01-01 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：:The aim of this article is to propose a multilevel combined model for repeated, hierarchical, and overdispersed time-to-event outcomes, extending the so-called combined model proposed by Molenberghs et al. (2010), and using three different estimation strategies: full likelihood, pseudo-likelihood, and Bayesian estimat...

journal_title：Journal of biopharmaceutical statistics

authors： Efendi A,Molenberghs G

更新日期：2013-01-01 00:00:00

abstract：:A common question in clinical studies is how to use historical data from earlier studies, leveraging relevant information into the design and analysis of a new study. Bayesian approaches are particularly well-suited to this task, with their natural ability to borrow strength across data sources. In this paper, we prop...

journal_title：Journal of biopharmaceutical statistics

authors： Li JX,Chen WC,Scott JA

更新日期：2016-01-01 00:00:00

abstract：:Testing for noninferiority and equivalence between an experimental therapy and a standard therapy in terms of the ratio of binomial proportions is considered. New tests based on the Fieller-Hinkley distribution of the ratio of random variables are proposed. Restricted maximum likelihood estimates of the null variances...

journal_title：Journal of biopharmaceutical statistics

authors： Koti KM

更新日期：2007-01-01 00:00:00

abstract：:We propose an adaptive two-stage dose-response design where a prespecified adaptation rule is used to add and/or drop treatment arms between the stages. We extend the multiple comparison procedures-modeling (MCP-Mod) approach into a two-stage design. In each stage, we use the same set of candidate dose-response models...

journal_title：Journal of biopharmaceutical statistics

authors： Franchetti Y,Anderson SJ,Sampson AR

更新日期：2013-01-01 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：:The aim of this work is to quantitatively assess the impact of structural model misspecifications on the estimates of mean and interindividual variability of clearance in the context of population approaches. This assessment is conducted from simulated datasets. Our results show that impact magnitude of model misspeci...

journal_title：Journal of biopharmaceutical statistics

authors： Merlé Y,Aouimer A,Tod M

更新日期：2004-02-01 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 1968 the Food and Drug Administration (FDA) established the Adverse Event Reporting System (AERS) database containing data on adverse events (AEs) reported by patients, health care providers, and other sources through a spontaneous reporting system. FDA uses AERS for monitoring the safety of the drugs on the market...

journal_title：Journal of biopharmaceutical statistics

authors： Huang L,Zalkikar J,Tiwari RC

更新日期：2013-01-01 00:00:00

abstract：:As more biologic products are going off patent protection, the development of follow-on biologics products has received much attention from both biotechnology industry and the regulatory agencies. Unlike small-molecule drug products, the development of biologic products is very different and variable via the manufactu...

journal_title：Journal of biopharmaceutical statistics

authors： Hsieh TC,Chow SC,Liu JP,Hsiao CF,Chi E

更新日期：2010-01-01 00:00:00

abstract：:Pharmaceutical product development culminates in confirmatory trials whose evidence for the product's efficacy and safety supports regulatory approval for marketing. Regulatory agencies in countries whose patients were not included in the confirmatory trials often require confirmation of efficacy and safety in their p...

journal_title：Journal of biopharmaceutical statistics

authors： Gould AL,Jin T,Zhang LX,Wang WW

更新日期：2012-09-01 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：:In recent years, development of biosimilar products has attracted considerable attention. Because of the structural complexity of biologics, it is difficult to demonstrate that a biosimilar product is identical to the reference product. Therefore, for the development of biosimilar products, we need to adopt an approac...

journal_title：Journal of biopharmaceutical statistics

authors： Nagasaki M,Ando Y

更新日期：2014-01-01 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：: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：: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：:An important application of confirmatory adaptive designs is the data-driven selection of treatment arms in multi-armed trials. A general methodology for adaptive designs is based on the combination testing principle. Using this principle, selection of treatment arms in multi-armed designs, recalculation of sample siz...

journal_title：Journal of biopharmaceutical statistics

authors： Wassmer G

更新日期：2011-07-01 00:00:00

abstract：:In the era of precision medicine, drugs are increasingly developed to target subgroups of patients with certain biomarkers. In large all-comer trials using a biomarker stratified design, the cost of treating and following patients for clinical outcomes may be prohibitive. With a fixed number of randomized patients, th...

journal_title：Journal of biopharmaceutical statistics

authors： Wang X,Zhou J,Wang T,George SL

更新日期：2018-01-01 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：: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：:Randomized controlled clinical trials often use a composite endpoint as a primary endpoint especially when treatment effects or frequency of individual components of the composite are likely to be small and combining them makes clinical sense for the disease under study. An advantage of the composite endpoint is that,...

journal_title：Journal of biopharmaceutical statistics

authors： Huque MF,Alosh M,Bhore R

更新日期：2011-07-01 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