Optimal two-stage randomized multinomial designs for Phase II oncology trials.

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：:The world of medical devices while highly diverse is extremely innovative, and this facilitates the adoption of innovative statistical techniques. Statisticians in the Center for Devices and Radiological Health (CDRH) at the Food and Drug Administration (FDA) have provided leadership in implementing statistical innova...

journal_title：Journal of biopharmaceutical statistics

authors： Campbell G,Yue LQ

更新日期：2016-01-01 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 potency of a batch of drug product needs to meet a release limits at the time of release so that the potency at the end of shelf life remains above the lower registration limit (LRL). This article discusses two methods which determine the release limits such that the chance to fail LRL at the end of shelf life of ...

journal_title：Journal of biopharmaceutical statistics

authors： Wei GC

更新日期：1998-03-01 00:00:00

abstract：:When marketed lots of pharmaceutical products produce a number of out-of-specification (OOS) samples, it can be very disruptive. In some cases such lots must be recalled, at substantial expense. We present a model of the probability of OOS samples and show quantitatively how this probability depends on the underlying ...

journal_title：Journal of biopharmaceutical statistics

authors： Fairweather WR

更新日期：2003-08-01 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：:We present a new computational method for identifying regulated pathway components in transcript profiling (TP) experiments by evaluating transcriptional activity in the context of known biological pathways. We construct a graph representing thousands of protein functional relationships by integrating knowledge from p...

journal_title：Journal of biopharmaceutical statistics

authors： Pradines J,Rudolph-Owen L,Hunter J,Leroy P,Cary M,Coopersmith R,Dancik V,Eltsefon Y,Farutin V,Leroy C,Rees J,Rose D,Rowley S,Ruttenberg A,Wieghardt P,Sander C,Reich C

更新日期：2004-08-01 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

authors： Zheng J,Yin D,Yuan M,Chow SC

更新日期：2019-01-01 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：:The proportion ratio (PR) of a positive response between an experimental treatment and a standard treatment (or placebo) is often used to measure the relative treatment efficacy in a randomized clinical trial (RCT). For ethical reasons, it is almost inevitable to encounter some patients not complying with their assign...

journal_title：Journal of biopharmaceutical statistics

authors： Lui KJ,Chang KC

更新日期：2012-01-01 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

authors： Demirhan H,Demirhan YP,Bacanli S

更新日期：2013-03-11 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：: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：:During preclinical drug development, the immune system is specifically evaluated after prolonged treatment with drug candidates, because the immune system may be an important target system. The response of antibodies against a T-cell-dependent antigen is recommenced by the FDA and EMEA for the evaluation of immunosupp...

journal_title：Journal of biopharmaceutical statistics

authors： Shkedy Z,Straetemans R,Molenberghs G,Desmidt M,Vinken P,Goeminne N,Coussement W,Van Den Poel B,Bijnens L

更新日期：2005-01-01 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

authors： Chen DT,Schell MJ,Chen JJ,Fulp WJ,Eschrich S,Yeatman T

更新日期：2008-01-01 00:00:00

abstract：:Patient-reported outcomes are important for assessing the effectiveness of treatments in many disease areas. For this reason, many new instruments that capture patient-reported outcomes have been developed over the past several decades. With the development of each new instrument, there is the ensuing question of what...

journal_title：Journal of biopharmaceutical statistics

authors： Kirby S,Chuang-Stein C,Morris M

更新日期：2010-09-01 00:00:00

abstract：:In method comparison and reliability studies, it is often important to assess agreement between multiple measurements made by different methods, devices, laboratories, observers, or instruments. For continuous data, the concordance correlation coefficient (CCC) is a popular index for assessing agreement between multip...

journal_title：Journal of biopharmaceutical statistics

authors： Barnhart HX,Lokhnygina Y,Kosinski AS,Haber M

更新日期：2007-01-01 00:00:00

abstract：:The Food and Drug Administration of the United States issued a draft guidance on adaptive design clinical trials in February 2010. This draft guidance has attracted a lot of attention because of the increasing interest in adaptive trials by the pharmaceutical industry in recent years. In this paper, we report on highl...

journal_title：Journal of biopharmaceutical statistics

authors： Chuang-Stein C,Beltangady M

更新日期：2010-11-01 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

authors： Jiang W,Wick JA,He J,Mahnken JD,Mayo MS

更新日期：2018-01-01 00:00:00

abstract：:We would like to estimate the parameters of a dose-response function with the greatest precision as possible. For a two-parameter model, this is equivalent to minimizing the area of the confidence ellipsoid, i.e., a D-optimal design. Previous work on this particular model has included minimal designs. These designs ar...

journal_title：Journal of biopharmaceutical statistics

authors： Lupinacci PJ,Raghavarao D

更新日期：2000-02-01 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：:Sample size calculation based on normal approximations is often associated with the loss of statistical power for a single-arm trial with a time-to-event endpoint. Recently, Wu (2015) derived the exact variance for the one-sample log-rank test under the alternative and showed that a single-arm one-stage study based on...

journal_title：Journal of biopharmaceutical statistics

authors： Shan G

更新日期：2020-09-02 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：:Ratio of means (ROM) and difference of means (DOM) are often used in a superiority, noninferiority (NI), or average bioequivalence (ABE) test to evaluate whether the test mean is superior, NI, or equivalent to the reference (placebo or active control) mean. The literature provides recommendations regarding how to choo...

journal_title：Journal of biopharmaceutical statistics

authors： Sun W,Grosser S,Tsong Y

更新日期：2017-01-01 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：: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：: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：: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 uncer...

journal_title：Journal of biopharmaceutical statistics

authors： Dokoumetzidis A,Aarons L

更新日期：2008-01-01 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：: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