Analysis of recurrent hypoglycemic events.

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abstract：:The Food and Drug Administration of the United States issued a draft guidance on adaptive design clinical trials in February 2010. This draft guidance has attracted a lot of attention because of the increasing interest in adaptive trials by the pharmaceutical industry in recent years.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：:The use of adaptive methods in clinical development has become very popular in recent years due to its flexibility in modifying trial procedures and/or statistical procedures of on-going clinical trials. Modifications to trial procedures are usually documented by protocol amendments. However, the actual patient popula...

journal_title：Journal of biopharmaceutical statistics

authors： Chow SC,Shao J

更新日期：2005-01-01 00:00:00

abstract：:In this paper, a propensity score-integrated composite likelihood (PSCL) approach is developed for cases in which the control arm of a two-arm randomized controlled trial (RCT) (treated vs control) is augmented with patients from real-world data (RWD) containing both clinical outcomes and covariates at the patient-lev...

journal_title：Journal of biopharmaceutical statistics

authors： Chen WC,Wang C,Li H,Lu N,Tiwari R,Xu Y,Yue LQ

更新日期：2020-05-03 00:00:00

abstract：:The small n, Sequential, Multiple Assignment, Randomized Trial (snSMART) is a two-stage clinical trial design for rare diseases motivated by the comparison of three active treatments for isolated skin vasculitis in the ongoing clinical trial ARAMIS (a randomized multicenter study for isolated skin vasculitis, NCT09239...

journal_title：Journal of biopharmaceutical statistics

authors： Wei B,Braun TM,Tamura RN,Kidwell K

更新日期：2020-09-06 00:00:00

abstract：:A test and a reference analytical method are usually compared for agreement based on paired data obtained from several independent subjects. Bias between two methods can be classified as constant and proportional. In this article, we provide an approach for maximum likelihood estimation of total bias between two metho...

journal_title：Journal of biopharmaceutical statistics

authors： Magari RT

更新日期：2004-11-01 00:00:00

abstract：:This article deals with seven special issues related to the assumptions, applicability, and practical use of formulas for calculating power or sample size, respectively, for comparative clinical trials with time-to-event endpoints, with particular focus on the well-known Freedman and Schoenfeld methods. All problems a...

journal_title：Journal of biopharmaceutical statistics

authors： Abel UR,Jensen K,Karapanagiotou-Schenkel I,Kieser M

更新日期：2015-01-01 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：: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 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：:A model-based approach is developed to estimate the distribution of time from seroconversion to diagnosis with acquired immunodeficiency syndrome (AIDS) as a function of selected time-dependent covariates. The approach is applied to longitudinal data collected over 4 years of follow-up from 450 men seropositive for th...

journal_title：Journal of biopharmaceutical statistics

authors： Dunlop DD,Tamhane AC,Chmiel JS,Phair JP

更新日期：1994-07-01 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：:A new three-arm parallel design was recently proposed to investigate the biosimilarity between a biological product and a reference product by using the relative distance. The purpose of this article is to extend their results to binary endpoints for three popular metrics: the risk difference, the log relative risk, a...

journal_title：Journal of biopharmaceutical statistics

authors： Shin W,Kang SH

更新日期：2016-01-01 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 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：:Predictive probability is particularly useful in aiding a decision-making process related to drug development. This is especially true for decisions occurring as the result of interim analyses of clinical trials. Examples of clinical trial applications of Bayesian predictive probability and the use of the beta-binomia...

journal_title：Journal of biopharmaceutical statistics

authors： Johns D,Andersen JS

更新日期：1999-03-01 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：: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：:Recently, a design was proposed for the Simultaneous Global Drug Development Program (SGDDP) to assess the impact of ethnic factors on the effect of a new treatment for a targeted ethnic (TE) population. It used weighted Z tests to combine the information collected from the TE and non-TE (NTE) subgroups in the SGDDP b...

journal_title：Journal of biopharmaceutical statistics

authors： Liu K,Yuan Z,Chen G,Huang Q,Wenrich J

更新日期：2015-01-01 00:00:00

abstract：:In a clinical dose finding study with active control a new drug with several dose levels is compared with an active comparator drug. The main focus of such studies often lies on the estimation of a target dose that leads to the same efficacy as the control. This article investigates the finite sample properties of the...

journal_title：Journal of biopharmaceutical statistics

authors： Helms HJ,Benda N,Friede T

更新日期：2015-01-01 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：:We introduce the idea of a design to detect signals of efficacy in early phase clinical trials. Such a design features three possible decisions: to kill the compound; to continue with staged development; or to continue with accelerated development of the compound. We describe how such studies improve the trade-off bet...

journal_title：Journal of biopharmaceutical statistics

authors： Brown MJ,Chuang-Stein C,Kirby S

更新日期：2012-01-01 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：: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：:In clinical trials of drug development, patients are often followed for a certain period of time, and the outcome variables are measured at scheduled time intervals. The main interest of the trial is the treatment efficacy at a prespecified time point, which is often the last visit. In such trials, patient dropout is ...

journal_title：Journal of biopharmaceutical statistics

authors： Kong F,Chen YF,Jin K

更新日期：2009-11-01 00:00:00

abstract：:Failure to recognize the serious implications of heterogeneous correlations and disregard of the multiple test problem in interpreting the results from repeated measurements ANOVA of any single primary outcome measure can produce false-positive error rates that are more than five times the alpha level that is reported...

journal_title：Journal of biopharmaceutical statistics

authors： Overall JE,Doyle SR

更新日期：1996-03-01 00:00:00

abstract：:The United States Pharmacopeia (USP) content uniformity sampling acceptance plan consisting of a two-stage sampling plan with criteria on sample mean and number of out-of-range tablets is the standard for compendium. It is, however, often used mistakenly for lot quality assurance. In comparison to the Japan Phamacopei...

journal_title：Journal of biopharmaceutical statistics

authors： Tsong Y,Shen M

更新日期：2007-01-01 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：:For administrative convenience or cost efficiency, we may often employ a cluster randomized trial (CRT), in which randomized units are clusters of patients rather than individual patients. Furthermore, because of ethical reasons or patient's decision, it is not uncommon to encounter data in which there are patients no...

journal_title：Journal of biopharmaceutical statistics

authors： Lui KJ,Chang KC

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