Statistical considerations in setting product specifications.

abstract：:Bayesian statistical methodology has been used for more than 10 years in medical device premarket submissions to the U.S. Food and Drug Administration (FDA). A complete list of the publicly available information associated with these FDA applications is presented. In addition to the increasing number of Bayesian metho...

journal_title：Journal of biopharmaceutical statistics

authors： Campbell G

更新日期：2011-09-01 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

authors： Millen BA,Dmitrienko A,Song G

更新日期：2014-01-01 00:00:00

abstract：:A thorough QT trial is typically designed to test for two set of hypotheses. The primary set of hypotheses is for demonstrating that the test treatment will not prolong QT interval. The second set of hypotheses is to demonstrate the assay sensitivity of the positive control treatment in the study population. The conve...

journal_title：Journal of biopharmaceutical statistics

authors： Tsong Y

更新日期：2013-01-01 00:00:00

abstract：:A method is proposed for the estimation of drug or toxicity potencies using in vitro data. A typical experiment in cancer research is presented where cells from a tumor-derived cell line were deposited as fixed volumes in 12-well cell culture plates. After waiting for 72 hours (for further growth), the wells were expo...

journal_title：Journal of biopharmaceutical statistics

authors： Le Chap T,Grambsch PM,Liu A

更新日期：2005-01-01 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

authors： Tango T

更新日期：2017-01-01 00:00:00

abstract：:To confirm results obtained from local evaluation at investigational centers, many oncology studies utilize blinded independent central review (BICR) to make assessments of the primary endpoint, progression-free survival (PFS). The comparison of data often leads to large discordances between these observations, castin...

journal_title：Journal of biopharmaceutical statistics

authors： Mannino FV,Amit O,Lahiri S

更新日期：2013-01-01 00:00:00

abstract：:In this paper I consider a problem of identifying all effective and superior drug combinations. I formulate this problem in terms of a family of hypotheses and propose a two-stage method to solve it. The first stage uses individual p-values obtained via the Min tests, whereas Holm's approach is employed in the second ...

journal_title：Journal of biopharmaceutical statistics

authors： Soulakova JN

更新日期：2009-01-01 00:00:00

abstract：:In this article, we show how to estimate a transition period for the evolvement of drug resistance to antiretroviral (ARV) drug or other related treatments in the framework of developing a Bayesian method for jointly analyzing time-to-event and longitudinal data. For HIV/AIDS longitudinal data, developmental trajector...

journal_title：Journal of biopharmaceutical statistics

authors： Dagne GA

更新日期：2018-01-01 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：: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：:A mathematical model is described for selecting combinations of chemotherapeutic drugs in order to maximize antitumor dose intensity subject to constraints on toxicity to normal organ systems. Determination of which drugs to combine and in what proportions to combine them offers combinatorially huge numbers of possibi...

journal_title：Journal of biopharmaceutical statistics

authors： Simon R,Korn EL

更新日期：1991-01-01 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：: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：:An attractive application of expression technologies is to predict drug efficacy or safety using expression data of biomarkers. To evaluate the performance of various classification methods for building predictive models, we applied these methods on six expression datasets. These datasets were from studies using micro...

journal_title：Journal of biopharmaceutical statistics

authors： Man MZ,Dyson G,Johnson K,Liao B

更新日期：2004-11-01 00:00:00

abstract：:Four multidose animal carcinogenicity assay trend test procedures based on the chi-square, Hoel-Walburg, log-rank, and Peto procedures are compared under conditions of competing risks. A total of 42 different risk populations are simulated in which a simulated animal can contract one or more of five different tumor ty...

journal_title：Journal of biopharmaceutical statistics

authors： Graves TS

更新日期：1994-11-01 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

authors： Morikawa T,Yoshida M

更新日期：1995-11-01 00:00:00

abstract：:Assessment of quality of life (QOL) in clinical trials becomes a challenging task from the viewpoint of clinical biostatistics. The responses of the items for measuring QOL indices usually vary widely from patient to patient and from time to time. Measurement errors might be present in the responses of the items, and ...

journal_title：Journal of biopharmaceutical statistics

authors： Siddiqui O,Ali MW

更新日期：1999-11-01 00:00:00

abstract：:Sample size adjustment at an interim analysis can mitigate the risk of failing to meet the study objective due to lower-than-expected treatment effect. Without modification to the conventional statistical methods, the type I error rate will be inflated, primarily caused by increasing sample size when the interim obser...

journal_title：Journal of biopharmaceutical statistics

authors： Chen YHJ,Yuan SS,Li X

更新日期：2018-01-01 00:00:00

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

authors： Wu J

更新日期：2011-05-01 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

authors： Lindsey JK,Wang J,Byrom WD,Jones B

更新日期：1999-08-01 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

authors： Djira GD,Hothorn LA,Tsong Y

更新日期：2008-01-01 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：: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：:In recent years, multi-regional clinical trials (MRCT) that conduct clinical trials simultaneously in Asian Pacific region, Europe, and the United States have become very popular for global pharmaceutical development. The main purpose of multi-regional clinical trials is to shorten the time for pharmaceutical developm...

journal_title：Journal of biopharmaceutical statistics

authors： Ying L,Song F,Chow SC,Zeng N,Zheng J,Li X,Henry D,Sethuraman V

更新日期：2018-01-01 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：: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：: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 intui...

journal_title：Journal of biopharmaceutical statistics

authors： Diao G,Zeng D,Ibrahim JG,Rong A,Lee O,Zhang K,Chen Q

更新日期：2017-01-01 00:00:00

abstract：:The gold standard for evaluating treatment efficacy of a medical product is a placebo-controlled trial. However, when the use of placebo is considered to be unethical or impractical, a viable alternative for evaluating treatment efficacy is through a noninferiority (NI) study where a test treatment is compared to an a...

journal_title：Journal of biopharmaceutical statistics

authors： Gamalo-Siebers M,Gao A,Lakshminarayanan M,Liu G,Natanegara F,Railkar R,Schmidli H,Song G

更新日期：2016-01-01 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

authors： May WL,Johnson WD

更新日期：1996-05-01 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

authors： Kenward MG,Jones B

更新日期：1992-01-01 00:00:00