Abstract:
:We provide a set of formulas that allow the combination of separately performed analyses of population pharmacokinetic (PK) studies, without any further computational effort. More specifically, given the point estimates and uncertainties of two population PK analyses, the formulas provide the point estimates and uncertainties of the combined analysis, including the mean population values, the between-subject variability, and the residual variability. To derive the formulas we considered distributional assumptions applicable for the conjugate priors of the Bayesian problem of "unknown mean and variance." In order to demonstrate the approach, the formulas were applied to an example involving the results of fitting two real experimental datasets. The formulas presented offer an easy-to-use method of combining different analyses particularly applicable to a combination of literature information.
journal_name
J Biopharm Statjournal_title
Journal of biopharmaceutical statisticsauthors
Dokoumetzidis A,Aarons Ldoi
10.1080/10543400802071360subject
Has Abstractpub_date
2008-01-01 00:00:00pages
662-76issue
4eissn
1054-3406issn
1520-5711pii
794802525journal_volume
18pub_type
杂志文章abstract::Clinical trials often use a binary "fold increase" endpoint defined according to the ratio of interval-censored measurement at end-of-study to that at baseline. We propose a simple yet principled analytic approach based on the linear mixed-effects model for interval-censored data for the analysis of such paired measur...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2014.919936
更新日期:2015-01-01 00:00:00
abstract::Parameter estimation following an adaptive design or group sequential design has been extremely challenging due to potential random high from its face value estimate. In this paper, we introduce a new framework to model clinical trial data flow based on a marked point process (MPP). The MPP model allows us to use meth...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2012.676534
更新日期:2012-01-01 00:00:00
abstract::In literature, there are a few unified approaches to test proof of concept and estimate a target dose, including the multiple comparison procedure using modeling approach, and the permutation approach proposed by Klingenberg. We discuss and compare the operating characteristics of these unified approaches and further ...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2017.1293081
更新日期:2017-01-01 00:00:00
abstract::Gene expression profiling has played an important role in cancer risk classification and has shown promising results. Since gene expression profiling often involves determination of a set of top rank genes for analysis, it is important to evaluate how modeling performance varies with the number of selected top ranked ...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400802277967
更新日期:2008-01-01 00:00:00
abstract::In a clinical dose finding study with active control a new drug with several dose levels is compared with an active comparator drug. The main focus of such studies often lies on the estimation of a target dose that leads to the same efficacy as the control. This article investigates the finite sample properties of the...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2014.920343
更新日期:2015-01-01 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
pub_type: 杂志文章,评审
doi:10.1080/10543409208835036
更新日期:1992-01-01 00:00:00
abstract::This paper addresses the problem of analyzing multivariate response with the variates having different distributions. We use the generalized estimating equations proposed by Prentice and Zhao (1) to estimate mean and covariance parameters. Wald statistics are used to test hypotheses about the parameters. Data from a t...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409608835129
更新日期:1996-05-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2019.1684310
更新日期:2020-05-03 00:00:00
abstract::Frequentist design for two-arm randomized Phase II clinical trials with outcomes from the exponential dispersion family was proposed previously, where the total sample sizes are minimized under multiple constraints on the standard errors of the estimated group means and their difference. This design was generalized fr...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2017.1402779
更新日期:2018-01-01 00:00:00
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
pub_type: 杂志文章
doi:10.1080/10543406.2016.1265539
更新日期:2017-01-01 00:00:00
abstract::Multipopulation tailoring trials provide a trial design option that supports the realization of tailored therapeutics or personalized medicine. Several recent publications have focused on statistical and clinical considerations that arise in these trials that are designed to study the overall treatment effect in a pop...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543406.2013.856025
更新日期:2014-01-01 00:00:00
abstract::After a group sequential test, the naive confidence interval (CI) is usually biased in the sense that it does not cover the true parameter at the correct nominal level. Furthermore, when the stopping time is taken into account, the actual conditional confidence coverage probability can be much less accurate. In this a...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400500406595
更新日期:2006-01-01 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
pub_type: 杂志文章,评审
doi:10.1080/10543409608835156
更新日期:1996-11-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2014.941985
更新日期:2014-01-01 00:00:00
abstract::Multiple testing problems in regulatory applications are often more challenging than the problems of handling a set of mathematical symbols representing multiple null hypotheses under testing. In the union-intersection setting, it is important to define a family of null hypotheses relevant to the clinical questions at...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400802541693
更新日期:2009-01-01 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
pub_type: 杂志文章
doi:10.1080/10543409308835045
更新日期:1993-03-01 00:00:00
abstract::All too often in clinical trials the assessment of quality of life is seen as a bolt-on study. Consequently insufficient consideration is often given to its design, collection, analysis and presentation, and its impact on the trial results and on clinical practice is minimal. In many trials quality of life is a key en...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1081/BIP-120028506
更新日期:2004-02-01 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
pub_type: 杂志文章,评审
doi:10.1080/10543406.2012.701579
更新日期:2012-09-01 00:00:00
abstract::We assess the QT effect using an exposure-response model in a "thorough QT/QTc study" with a four-period crossover design in which the treatments are placebo, positive control, higher dose of investigational drug, and therapeutic dose of investigational drug. In the study, QTc interval values and the drug concentratio...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400903582026
更新日期:2010-05-01 00:00:00
abstract::Tango (Biostatistics 2016) proposed a new repeated measures design called the S:T repeated measures design, combined with generalized linear mixed-effects models and sample size calculations for a test of the average treatment effect that depend not only on the number of subjects but on the number of repeated measures...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2017.1293083
更新日期:2017-01-01 00:00:00
abstract::When performing a pivotal clinical trial, it may be of interest to assess the probability of success (PoS) of that trial. Initially evaluated when the trial is designed, PoS can be updated as the trial progresses and new information about the drug effect becomes available. Such information can be external to the trial...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2014.972508
更新日期:2016-01-01 00:00:00
abstract::In this article, a parametric sequential test is proposed under the Weibull model. The proposed test is asymptotically normal with an independent increment structure. The sample size for a fixed sample test is derived for the purpose of group sequential trial design. In addition, a multi-stage group sequential procedu...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2014.971165
更新日期:2015-01-01 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
pub_type: 杂志文章
doi:10.1080/10543401003618884
更新日期:2010-07-01 00:00:00
abstract::Stability testing is a procedure frequently used in the pharmaceutical industry to estimate the shelf life of a drug. Hereby, a standard problem of interest is whether or not to pool a given number of batches to assign a single shelf life for the combined batches. In this paper, we propose two modified methods for the...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400802287230
更新日期:2008-01-01 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
pub_type: 杂志文章
doi:10.1081/BIP-120024196
更新日期:2003-11-01 00:00:00
abstract::We develop a Bayesian approach for estimating vaccine efficacy for susceptibility (VEs) and infectiousness (VEI) using outbreak size household data. Our method allows for heterogeneity in transmission probabilities due to factors that are related to individuals' characteristics, such as age, in addition to vaccination...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400600719467
更新日期:2006-01-01 00:00:00
abstract::In analytical similarity assessment of a biosimilar product, key quality attributes of the test and reference products need to be shown statistically similar. When there were multiple references, similarity among the reference products is also required. We proposed a simultaneous confidence approach based on the fiduc...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2019.1657142
更新日期:2019-01-01 00:00:00
abstract::One of the most critical decision points in clinical development is Go/No-Go decision-making after a proof-of-concept study. Traditional decision-making relies on a formal hypothesis testing with control of type I and type II error rates, which is limited by assessing the strength of efficacy evidence in a small isola...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2018.1489400
更新日期:2019-01-01 00:00:00
abstract::Pharmacokinetic studies of drug and metabolite concentrations in the blood are usually conducted as crossover trials, especially in phases I and II. A longitudinal series of measurements is collected on each subject within each period. However, much of the dependence among such observations, within and between periods...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1081/BIP-100101186
更新日期:1999-08-01 00:00:00
abstract::The Log-odds ratio for 2 × 2 contingency tables is often approximated by a normal distribution with an approximated variance. Hwang and Biswas (2008) illustrated that the standard expression for the variance should be modified in the presence of correlation. They also provided an adjustment to this variance expression...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2010.494268
更新日期:2011-01-01 00:00:00