A quantitative assessment of factors influencing the probability of postmarketing out-of-specification observations.

abstract：:A new two-stage design is proposed that is suitable for early detection of the anticancer activity of experimental therapies in Phase II oncology trials. The endpoints of interest are response rate and early progression rate. The anticancer activity is defined by a positive signal in one endpoint and a non-negative si...

journal_title：Journal of biopharmaceutical statistics

authors： Sun LZ,Chen C,Patel K

更新日期：2009-01-01 00:00:00

abstract：:The Youden index, a main summary index for the receiver operating characteristic (ROC) curve, is a comprehensive measurement for the effectiveness of a diagnostic test. For a continuous-scale diagnostic test, the optimal cut point for positive disease is the cut point leading to the maximization of the sum of sensitiv...

journal_title：Journal of biopharmaceutical statistics

authors： Zhou H,Qin G

更新日期：2012-01-01 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：:The ICH E14 guidance (ICH, 2005) recommend that a concurrent positive control should be included in a thorough QTc clinical trial to validate the study. The ICH E14 guidance (ICH, 2005) state that "The positive control should have an effect on the mean QTc interval of about 5 ms (i.e., an effect that is close to the Q...

journal_title：Journal of biopharmaceutical statistics

authors： Zhang J

更新日期：2008-01-01 00:00:00

abstract：:We address the issue of analysis of clinical data generated by the bridging study conducted in the new region to evaluate the similarity for extrapolation of the foreign clinical data. A bridging study is usually conducted in the new region only after the test product is approved for commercial marketing in the origin...

journal_title：Journal of biopharmaceutical statistics

authors： Liu JP,Hsiao CF,Hsueh H

更新日期：2002-08-01 00:00:00

abstract：:We evaluate properties of sample size re-estimation (SSR) designs similar to the promising zone design considered by Mehta and Pocock (2011). We evaluate these designs under the assumption of a true effect size of 1.1 down to 0.4 of the protocol-specified effect size by six measures: 1. The probability of a sample siz...

journal_title：Journal of biopharmaceutical statistics

authors： Gaffney M,Ware JH

更新日期：2017-01-01 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

authors： Huang L,Zalkikar J,Tiwari RC

更新日期：2013-01-01 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

authors： Kenward MG,Jones B

更新日期：1992-01-01 00:00:00

abstract：:In traditional dose-finding studies, dose-limiting toxicity (DLT) is determined within a fixed time observation window where DLT is often defined as a binary outcome. In the setting of oncology dose-finding trials, often patients in advanced stage of diseases are enrolled. Therefore, disease progression may occur with...

journal_title：Journal of biopharmaceutical statistics

authors： Biard L,Cheng B,Manji GA,Lee SM

更新日期：2020-09-15 00:00:00

abstract：:The study of drug combinations has become important in drug development due to its potential for efficacy at lower, less toxic doses and the need to move new therapies rapidly into clinical trials. The goal is to identify which combinations are additive, synergistic, or antagonistic. Although there exists statistical ...

journal_title：Journal of biopharmaceutical statistics

authors： Fang HB,Tian GL,Li W,Tan M

更新日期：2009-07-01 00:00:00

abstract：:According to ICH Q6A (1999), a specification is defined as a list of tests, references to analytical procedures, and appropriate acceptance criteria, which are numerical limits, ranges, or other criteria for the tests described. For drug products, specifications usually consist of test methods and acceptance criteria ...

journal_title：Journal of biopharmaceutical statistics

authors： Dong X,Tsong Y,Shen M

更新日期：2015-01-01 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

authors： Lao CS

更新日期：1997-11-01 00:00:00

abstract：:In randomized controlled trials, patients are recruited and randomly allocated to treatments. Patients are never randomly sampled from large population of patients on treatments under study. Therefore, it is important to consider the design and statistical analysis based on the randomization model. In this article, we...

journal_title：Journal of biopharmaceutical statistics

authors： Hasegawa T,Tango T

更新日期：2009-01-01 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：: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：:Due to the potential impact of ethnic factors on clinical outcomes, the global registration of a new treatment is challenging. China and Japan often require local trials in addition to a multiregional clinical trial (MRCT) to support the efficacy and safety claim of the treatment. The impact of ethnic factors on the t...

journal_title：Journal of biopharmaceutical statistics

authors： Huang Q,Chen G,Yuan Z,Lan KK

更新日期：2012-09-01 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：:In cancer drug development, demonstrated efficacy in tumor xenograft models is an important step toward bringing a promising compound to human use. A key outcome variable is tumor volume measured over a period of time, while mice are treated with certain treatment regimens. A constrained parametric model has been prop...

journal_title：Journal of biopharmaceutical statistics

authors： Fang HB,Deng D,Zhang T,Tan M

更新日期：2014-01-01 00:00:00

abstract：:Clinical trials with data-driven decision rules often pursue multiple clinical objectives such as the evaluation of several endpoints or several doses of an experimental treatment. These complex analysis strategies give rise to "multivariate" multiplicity problems with several components or sources of multiplicity. A ...

journal_title：Journal of biopharmaceutical statistics

authors： Kordzakhia G,Dmitrienko A,Ishida E

更新日期：2018-01-01 00:00:00

abstract：:We study the statistical efficiency for rising-dose designs in the context of first-in-human studies. Specifically, we identify a class of crossover designs that are appealing in terms of both subject safety and statistical efficiency and, for a three-period, two-panel design in such a class, we compare its A-efficien...

journal_title：Journal of biopharmaceutical statistics

authors： Yan Z,Hosmane B,Locke C

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

authors： Gagnon R,Leonov S

更新日期：2005-01-01 00:00:00

abstract：:There is no consensus on determination of sample size in phase II clinical trials. The use of Bayesian decision theory has been proposed by Stallard (1), among others. In this article, optimal three-stage designs are obtained using decision theory. These are compared with procedures proposed by Schoenfeld (2), Ensign ...

journal_title：Journal of biopharmaceutical statistics

authors： Stallard N

更新日期：1998-07-01 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

authors： Berger VW,Vali B

更新日期：2011-03-01 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

authors： Luo X,Li M,Shih WJ,Ouyang P

更新日期：2012-01-01 00:00:00

abstract：:Administration of biological therapeutics can generate undesirable immune responses that may induce anti-drug antibodies (ADAs). Immunogenicity can negatively affect patients, ranging from mild reactive effect to hypersensitivity reactions or even serious autoimmune diseases. Assessment of immunogenicity is critical a...

journal_title：Journal of biopharmaceutical statistics

authors： Zhang J,Yu B,Zhang L,Roskos L,Richman L,Yang H

更新日期：2015-01-01 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

authors： Dragalin V,Fedorov V,Jones B,Rockhold F

更新日期：2001-11-01 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：: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

authors： Ke BS,Chiang AJ,Chang YI

更新日期：2018-01-01 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：: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