Abstract:
:Noninferiority multiregional clinical trials (MRCTs) have recently received increasing attention in drug development. While a major goal in an MRCT is to estimate the global treatment effect, it is also important to assess the consistency of treatment effects across multiple regions. In this paper, we propose an intuitive definition of consistency of noninferior treatment effects across regions under the random-effects modeling framework. Specifically, we quantify the consistency of treatment effects by the percentage of regions that meet a predefined treatment margin. This new approach enables us to achieve both goals in one modeling framework. We propose to use a signed likelihood ratio test for testing the global treatment effect and the consistency of noninferior treatment effects. In addition, we provide guidelines for the allocation rule to achieve optimal power for testing consistency among multiple regions. Extensive simulation studies are conducted to examine the performance of the proposed methodology. An application to a real data example is provided.
journal_name
J Biopharm Statjournal_title
Journal of biopharmaceutical statisticsauthors
Diao G,Zeng D,Ibrahim JG,Rong A,Lee O,Zhang K,Chen Qdoi
10.1080/10543406.2017.1293075subject
Has Abstractpub_date
2017-01-01 00:00:00pages
933-944issue
6eissn
1054-3406issn
1520-5711journal_volume
27pub_type
杂志文章abstract::In preclinical tumor xenograft experiments, the antitumor activity of the tested agents is often assessed by endpoints such as tumor doubling time, tumor growth delay (TGD), and log10 cell kill (LCK). In tumor xenograft literature, the values of these endpoints are presented without any statistical inference, which ig...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2010.481802
更新日期:2011-05-01 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::I review the designs available for Phase I dose-finding studies of chemotherapeutic agents in cancer patients. The designs are based on the assumption that both efficacy and toxicity increase with dose, and thus attempt to minimize the number of patients treated at low doses, and also to minimize the chance that patie...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543400600860295
更新日期:2006-01-01 00:00:00
abstract::Some statistical methods applied to in vitro diagnostic tests for the three primary indications (screening, diagnosis, and monitoring) are discussed. Various examples with practical statistical applications are presented, including test for k by k ordered categorical matched-pair data for screening of cervical cancer,...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409708835208
更新日期:1997-11-01 00:00:00
abstract::An ability to predict the metabolic fate of a drug is important to drug design. Programs for predicting drug metabolites are becoming available, as are databases that will facilitate the development of such programs. Objective analysis of the performance of these programs will require statistical methods. The developm...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543409108835004
更新日期:1991-01-01 00:00:00
abstract::An adaptive enrichment design is a randomized trial that allows enrollment criteria to be modified at interim analyses, based on a preset decision rule. When there is prior uncertainty regarding treatment effect heterogeneity, these trial designs can provide improved power for detecting treatment effects in subpopulat...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2018.1489401
更新日期:2018-01-01 00:00:00
abstract::Classification measures play essential roles in the assessment and construction of classifiers. Hence, determining how to prevent these measures from being affected by individual observations has become an important problem. In this paper, we propose several indexes based on the influence function and the concept of l...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2017.1377728
更新日期:2018-01-01 00:00:00
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
pub_type: 杂志文章
doi:10.1080/10543406.2011.550107
更新日期:2011-03-01 00:00:00
abstract::In this study, a Bayesian approach was suggested to estimate a change-point according to a covariate threshold when some patients never experienced the event of interest. Gibbs sampler algorithm with latent binary cure indicators was used to simplify the implementation of Markov chain Monte Carlo method. Then, the acc...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2019.1632877
更新日期:2020-03-01 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
pub_type: 杂志文章
doi:10.1080/10543409708835171
更新日期:1997-03-01 00:00:00
abstract::A framework is proposed for making quality predictions in situations for which only systematically inaccurate data are available. The predictions are based on the systematically inaccurate data, complete data from similar situations, and expert knowledge. The proposed predictive model is well suited to functional data...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2013.860153
更新日期:2014-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2014.979195
更新日期:2016-01-01 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::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
pub_type: 杂志文章
doi:10.1081/BIP-100101000
更新日期:1999-03-01 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
pub_type: 杂志文章
doi:10.1080/10543409508835115
更新日期:1995-11-01 00:00:00
abstract::In clinical trials, it is important to set up a design to reach a decision on effectiveness of a drug in treating a disease with the loss of the minimum number of patients. Group sequential designs are very beneficial on this point. However, the proportional hazards assumption must hold to work under a group sequentia...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2011.616975
更新日期:2013-03-11 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
pub_type: 杂志文章
doi:10.1081/BIP-120016231
更新日期:2002-11-01 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
pub_type: 杂志文章
doi:10.1080/10543400500508952
更新日期:2006-01-01 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
pub_type: 杂志文章
doi:10.1081/BIP-200056532
更新日期:2005-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2014.971166
更新日期:2015-01-01 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
pub_type: 杂志文章
doi:10.1080/10543400701697158
更新日期:2008-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2013.736810
更新日期:2013-01-01 00:00:00
abstract::In various pharmaceutical applications, repeated measurements are taken from each subject, and model parameters are estimated from the collected data. Examples include dose response modeling and PK/PD studies with serial blood sampling, among others. The quality of the information in an experiment is reflected in the ...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1081/bip-200040853
更新日期:2005-01-01 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
pub_type: 杂志文章
doi:
更新日期:2001-11-01 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::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
pub_type: 杂志文章
doi:10.1080/10543400701645165
更新日期:2007-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2014.1000546
更新日期:2015-01-01 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
pub_type: 杂志文章
doi:10.1080/10543400601177426
更新日期:2007-01-01 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
pub_type: 杂志文章
doi:10.1080/10543409708835187
更新日期:1997-05-01 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