Planning survival studies to compare a treatment to an active control.

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：:The widely used distinction of Little and Rubin (1) about types of randomness for missing data presents difficulties in its application to dropouts in longitudinal repeated measurement studies. In its place, a new typology of randomness for dropouts is proposed that relies on using a survival model for the dropout pro...

journal_title：Journal of biopharmaceutical statistics

authors： Lindsey JK

更新日期：2000-11-01 00:00:00

abstract：:In this article, a parametric sequential test is proposed under the Weibull model. The proposed test is asymptotically normal with an independent increment structure. The sample size for a fixed sample test is derived for the purpose of group sequential trial design. In addition, a multi-stage group sequential procedu...

journal_title：Journal of biopharmaceutical statistics

authors： Wu J,Xiong X

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：:Due to the importance of precision medicine, it is essential to identify the right patients for the right treatment. Biomarkers, which have been commonly used in clinical research as well as in clinical practice, can facilitate selection of patients with a good response to the treatment. In this paper, we describe a b...

journal_title：Journal of biopharmaceutical statistics

authors： Diao G,Dong J,Zeng D,Ke C,Rong A,Ibrahim JG

更新日期：2018-01-01 00:00:00

abstract：:Recently, two CPMP Points to Consider, one on adjustment for baseline covariates and the other on multiplicity issues in clinical trials, have included recommendations on the use of subgroup analysis for regulatory purposes. However, despite their regular use and regulatory attention, the validity and nature of subgro...

journal_title：Journal of biopharmaceutical statistics

authors： Grouin JM,Coste M,Lewis J

更新日期：2005-01-01 00:00:00

abstract：:Drug-related side effects are one of the leading causes of death and illness in the developed world. Finding genes that modify drug response has the potential to significantly improve drug delivery, by identifying both individuals that can benefit from therapy and those at increased risk of harm. We present a simple a...

journal_title：Journal of biopharmaceutical statistics

authors： He M,Allen A

更新日期：2010-03-01 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：: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：:In confidence interval estimation of the difference between two proportions with overdispersion due to positive correlations, the usual asymptotic normality-based method generally has lower coverage rates than desired, especially when sample size is moderate. Applying the concept of effective sample size to existing m...

journal_title：Journal of biopharmaceutical statistics

authors： Chen C,Li J,Zhou Z

更新日期：2004-05-01 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：:The Log-odds ratio for 2 × 2 contingency tables is often approximated by a normal distribution with an approximated variance. Hwang and Biswas (2008) illustrated that the standard expression for the variance should be modified in the presence of correlation. They also provided an adjustment to this variance expression...

journal_title：Journal of biopharmaceutical statistics

authors： Biswas A,Hwang JS

更新日期：2011-01-01 00:00:00

abstract：:Statistical methodologies for human abuse potential studies are rarely evaluated. Human abuse potential studies assess whether test drugs produce positive and negative subjective responses on abuse-related measures using volunteers with histories of recreational drug use. These studies typically have a randomized, dou...

journal_title：Journal of biopharmaceutical statistics

authors： Chen L,Bonson KR

更新日期：2013-03-11 00:00:00

abstract：:Historical control trials (HCTs) are frequently conducted to compare an experimental treatment with a control treatment from a previous study, when they are applicable and favored over a randomized clinical trial (RCT) due to feasibility, ethics and cost concerns. Makuch and Simon developed a sample size formula for h...

journal_title：Journal of biopharmaceutical statistics

authors： Zhu H,Zhang S,Ahn C

更新日期：2016-01-01 00:00:00

abstract：:For the assessment of biosimilarity of biosimilar products, the United States (US) Food and Drug Administration (FDA) proposed a stepwise approach for providing the totality-of-the-evidence of similarity between a proposed biosimilar product and a US-licensed (reference) product. The stepwise approach starts with the ...

journal_title：Journal of biopharmaceutical statistics

authors： Wang T,Chow SC

更新日期：2017-01-01 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：: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：:This paper introduces exact permutation methods for use when there are independent clusters of data with arbitrary within-cluster correlation. To eliminate the problem of clustering, we randomly select a data point from each cluster and for this now independent data, and calculate our test statistic and the associated...

journal_title：Journal of biopharmaceutical statistics

authors： Follmann D,Fay M

更新日期：2010-07-01 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：:Laboratory data with a lower quantification limit (censored data) are sometimes analyzed by replacing non-quantifiable values with a single value equal to or less than the quantification limit, yielding possibly biased point estimates and variance estimates that are too small. Motivated by a three-period, three-treatm...

journal_title：Journal of biopharmaceutical statistics

authors： Karon JM,Wiegand RE,van de Wijgert JH,Kilmarx PH

更新日期：2015-01-01 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：:Large sample size imbalance is not uncommon in the biosimilar development. At the beginning of a product development, sample sizes of a biosimilar and a reference product may be limited. Thus, a sample size calculation may not be feasible. During the development stage, more batches of reference products may be added a...

journal_title：Journal of biopharmaceutical statistics

authors： Dong XC,Weng YT,Tsong Y

更新日期：2017-01-01 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：:The issue of consistency and reliability arises frequently in applied clinical trials, and there is a considerable amount of literature on this topic. For a continuous random variable, we describe a process to assess whether the mean and variance are the same from one evaluation to the other and we derive the intracla...

journal_title：Journal of biopharmaceutical statistics

authors： Kao TC,Sparling Y,Rochon J

更新日期：2003-08-01 00:00:00

abstract：:In the early stages of traditional drug development, the frequency of dosing (e.g., QD, BID, etc.) is typically determined by the pharmacokinetic properties of a compound. After an appropriate dose frequency is chosen, the magnitude of dose is then evaluated via parallel-group dose-response trials. For some drugs, how...

journal_title：Journal of biopharmaceutical statistics

authors： Hafner KB,Ruberg SJ

更新日期：1996-07-01 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：: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 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：:The design of a three-arm trial including the experimental treatment, an active reference treatment, and a placebo is recommended as a useful approach to the assessment of noninferiority of the experimental treatment. The inclusion of the placebo arm enables the assessment of assay sensitivity and internal validation,...

journal_title：Journal of biopharmaceutical statistics

authors： Hida E,Tango T

更新日期：2013-01-01 00:00:00

abstract：:A common question in clinical studies is how to use historical data from earlier studies, leveraging relevant information into the design and analysis of a new study. Bayesian approaches are particularly well-suited to this task, with their natural ability to borrow strength across data sources. In this paper, we prop...

journal_title：Journal of biopharmaceutical statistics

authors： Li JX,Chen WC,Scott JA

更新日期：2016-01-01 00:00:00