On the establishment of equivalence acceptance criterion in analytical similarity assessment.

abstract：:Rubinstein et al. presented a procedure for determining the required duration of accrual for a clinical trial comparing the survival distributions of two treatments using a classical hypothesis testing formulation. Here their testing procedure is modified in two ways. First, the asymptotic variances used in computatio...

journal_title：Journal of biopharmaceutical statistics

authors： Bristol DR

更新日期：1993-09-01 00:00:00

abstract：:Nonparametric versions of normal theory step-down multiple-test procedures for inferring minimum effective dose (see Tamhane et al. (1)) were developed and studied by Monte Carlo simulation. Two types of step-down testing procedures were examined. For both procedures, pairwise, linear, or Helmert contrasts of mean ran...

journal_title：Journal of biopharmaceutical statistics

authors： Sidik K,Morris RW

更新日期：1999-05-01 00:00:00

abstract：:Dichotomizing a continuous biomarker is a common practice in medical research. Various methods exist in the literature for dichotomizing continuous biomarkers. The most widely adopted minimum p-value approach uses a sequence of test statistics for all possible dichotomizations of a continuous biomarker, and it chooses...

journal_title：Journal of biopharmaceutical statistics

authors： Su M,Fang L,Su Z

更新日期：2013-05-01 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：: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

authors： Rufibach K,Jordan P,Abt M

更新日期：2016-01-01 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：: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：:We studied the sensitivity of the number of unique design points and their placement, in Bayesian optimal designs for pharmacokinetic models, with respect to the magnitude of prior uncertainty. We used two and three-parameter pharmacokinetic models with fixed and mixed effects and two Bayesian optimal design criteria,...

journal_title：Journal of biopharmaceutical statistics

authors： Dokoumetzidis A,Aarons L

更新日期：2007-01-01 00:00:00

abstract：:As more biologic products are going off patent protection, the development of follow-on biologics products has received much attention from both biotechnology industry and the regulatory agencies. Unlike small-molecule drug products, the development of biologic products is very different and variable via the manufactu...

journal_title：Journal of biopharmaceutical statistics

authors： Hsieh TC,Chow SC,Liu JP,Hsiao CF,Chi E

更新日期：2010-01-01 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

authors： Zheng J,Yin D,Yuan M,Chow SC

更新日期：2019-01-01 00:00:00

abstract：:Pressures for rapid drug development, especially for treatments that may affect public health significantly, drive a need to reconsider what is necessary to establish the "substantial evidence" of efficacy and safety required for regulatory approval. The concept of substantial evidence of effect can be stated fairly s...

journal_title：Journal of biopharmaceutical statistics

authors： Gould AL

更新日期：2002-02-01 00:00:00

abstract：:The outcome selection of bioequivalence evaluations for abbreviated new drug applications results in bias towards unity of test-reference ratio estimates, and underestimation of intrasubject variability for the test drug product. In this study, the selection bias in the estimates of testreference ratio and intrasubjec...

journal_title：Journal of biopharmaceutical statistics

authors： Wang Y

更新日期：2000-08-01 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：:There are minimal standards for the processing of whole blood components, and to apply those standards requires a system of quality assurances. Excessive indications of failures in compliance trigger inspections and other remedial actions, but the demarcation of what is excessive is a critical issue. Issues of low vol...

journal_title：Journal of biopharmaceutical statistics

authors： Lachenbruch PA,Foulkes MA,Williams AE,Epstein JS

更新日期：2005-01-01 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

authors： Lu Y,Zhang ZZ,Chow SC

更新日期：2014-01-01 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：: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

authors： Whitehead J,Zhou Y,Hampson L,Ledent E,Pereira A

更新日期：2007-01-01 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 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：:It is well known that there is strong relationship between HIV viral load and CD4 cell counts in AIDS studies. However, the relationship between them changes during the course of treatment and may vary among individuals. During treatments, some individuals may experience terminal events such as death. Because the term...

journal_title：Journal of biopharmaceutical statistics

authors： Lu T,Wang M,Liu G,Dong GH,Qian F

更新日期：2016-01-01 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：:Although asymptotically, the empirical covariance estimator is consistent and robust with respect to the selection of the working correlation matrix, when the sample size is small, its bias may not be negligible. This article proposes a small sample correction for the empirical covariance estimator in general Gaussian...

journal_title：Journal of biopharmaceutical statistics

authors： Fan C,Zhang D,Zhang CH

更新日期：2012-01-01 00:00:00

abstract：:Bayesian sequential integration is an appealing approach in drug development, as it allows to recursively update posterior distributions as soon as new data become available, thus considerably reducing the computation time. However, preclinical trials are often characterized by small sample sizes, which may affect the...

journal_title：Journal of biopharmaceutical statistics

authors： La Gamba F,Jacobs T,Serroyen J,Geys H,Faes C

更新日期：2020-06-18 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：:We consider the problem of estimating a biomarker-based subgroup and testing for treatment effect in the overall population and in the subgroup after the trial. We define the best subgroup as the subgroup that maximizes the power for comparing the experimental treatment with the control. In the case of continuous outc...

journal_title：Journal of biopharmaceutical statistics

authors： Joshi N,Fine J,Chu R,Ivanova A

更新日期：2019-01-01 00:00:00

abstract：:Sample size estimation (SSE) is an important issue in the planning of clinical studies. While larger studies are likely to have sufficient power, it may be unethical to expose more patients than necessary to answer a scientific question. Budget considerations may also cause one to limit the study to an adequate size t...

journal_title：Journal of biopharmaceutical statistics

authors： Wu PS,Lin M,Chow SC

更新日期：2016-01-01 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：: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：: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：: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