Comparison of concordance correlation coefficient and coefficient of individual agreement in assessing agreement.

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：: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：:An integral part of routine health checkups involves laboratory measurements on various analytes in the blood. It is then common to compare the value of two consecutive measurements sampled at different times from the same patient. A "significant" change requires an action (additional sample and/or clinical action). T...

journal_title：Journal of biopharmaceutical statistics

authors： Cohen A,Doveh E,Umansky T

更新日期：2001-01-01 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 clinical trials, selection of appropriate study endpoints is critical for an accurate and reliable evaluation of safety and effectiveness of a test treatment under investigation. In practice, however, there are usually multiple endpoints available for measurement of disease status and/or therapeutic effect of the t...

journal_title：Journal of biopharmaceutical statistics

authors： Chow SC,Huang Z

更新日期：2019-01-01 00:00:00

abstract：:Dunnett's many-to-one test is used frequently today, especially in dose-finding studies. Using Dunnett's test, the Type I error level for the comparison between the raw mean of the control and the raw means of the study drug groups can be exactly calculated for the normal data. However, this computability depends on t...

journal_title：Journal of biopharmaceutical statistics

authors： Shun Z,Silverberg A,Chang CK,Ouyang P

更新日期：2003-02-01 00:00:00

abstract：:One of the most critical decision points in clinical development is Go/No-Go decision-making after a proof-of-concept study. Traditional decision-making relies on a formal hypothesis testing with control of type I and type II error rates, which is limited by assessing the strength of efficacy evidence in a small isola...

journal_title：Journal of biopharmaceutical statistics

authors： Huang B,Talukder E,Han L,Kuan PF

更新日期：2019-01-01 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：: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：: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：: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：: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：: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：: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：:During a clinical trial, balancing statistical and ethical considerations are important. Response-adaptive randomization methods use the information from past patients to increase the probability of the next patient receiving the better treatment while avoiding the statistical concern of selection bias. We compared th...

journal_title：Journal of biopharmaceutical statistics

authors： Piccorelli AV,Fraker SA

更新日期：2018-01-01 00:00:00

abstract：:In this study, a Bayesian approach was suggested to estimate a change-point according to a covariate threshold when some patients never experienced the event of interest. Gibbs sampler algorithm with latent binary cure indicators was used to simplify the implementation of Markov chain Monte Carlo method. Then, the acc...

journal_title：Journal of biopharmaceutical statistics

authors： Shamsi F,Baghestani AR,Binesh F

更新日期：2020-03-01 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：: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 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：: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

abstract：:A framework is proposed for making quality predictions in situations for which only systematically inaccurate data are available. The predictions are based on the systematically inaccurate data, complete data from similar situations, and expert knowledge. The proposed predictive model is well suited to functional data...

journal_title：Journal of biopharmaceutical statistics

authors： Haaland B,Chiang AY

更新日期：2014-01-01 00:00:00

abstract：:This article discusses the design of a clinical trial where a new treatment will be compared to a control. For a specific type of endpoint, there are a wide variety of test statistics that can be used. Also, the investigator must decide how many patients to accrue in each arm as well as the duration of the study. Afte...

journal_title：Journal of biopharmaceutical statistics

authors： Lawrence J

更新日期：2002-05-01 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：: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：: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：: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：: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：:In recent years, development of biosimilar products has attracted considerable attention. Because of the structural complexity of biologics, it is difficult to demonstrate that a biosimilar product is identical to the reference product. Therefore, for the development of biosimilar products, we need to adopt an approac...

journal_title：Journal of biopharmaceutical statistics

authors： Nagasaki M,Ando Y

更新日期：2014-01-01 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：: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