Mixed-effect models to assess consistency and reliability across multiple evaluations.

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：: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：:Frequentist design for two-arm randomized Phase II clinical trials with outcomes from the exponential dispersion family was proposed previously, where the total sample sizes are minimized under multiple constraints on the standard errors of the estimated group means and their difference. This design was generalized fr...

journal_title：Journal of biopharmaceutical statistics

authors： Jiang W,Wick JA,He J,Mahnken JD,Mayo MS

更新日期：2018-01-01 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：: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：: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：: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 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：: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：: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：: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：:A method is proposed for the estimation of drug or toxicity potencies using in vitro data. A typical experiment in cancer research is presented where cells from a tumor-derived cell line were deposited as fixed volumes in 12-well cell culture plates. After waiting for 72 hours (for further growth), the wells were expo...

journal_title：Journal of biopharmaceutical statistics

authors： Le Chap T,Grambsch PM,Liu A

更新日期：2005-01-01 00:00:00

abstract：:Four multidose animal carcinogenicity assay trend test procedures based on the chi-square, Hoel-Walburg, log-rank, and Peto procedures are compared under conditions of competing risks. A total of 42 different risk populations are simulated in which a simulated animal can contract one or more of five different tumor ty...

journal_title：Journal of biopharmaceutical statistics

authors： Graves TS

更新日期：1994-11-01 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：: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：: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：: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：:We develop a Bayesian approach for estimating vaccine efficacy for susceptibility (VEs) and infectiousness (VEI) using outbreak size household data. Our method allows for heterogeneity in transmission probabilities due to factors that are related to individuals' characteristics, such as age, in addition to vaccination...

journal_title：Journal of biopharmaceutical statistics

authors： Davis XM,Waller LA,Haber M

更新日期：2006-01-01 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：:We present a new computational method for identifying regulated pathway components in transcript profiling (TP) experiments by evaluating transcriptional activity in the context of known biological pathways. We construct a graph representing thousands of protein functional relationships by integrating knowledge from p...

journal_title：Journal of biopharmaceutical statistics

authors： Pradines J,Rudolph-Owen L,Hunter J,Leroy P,Cary M,Coopersmith R,Dancik V,Eltsefon Y,Farutin V,Leroy C,Rees J,Rose D,Rowley S,Ruttenberg A,Wieghardt P,Sander C,Reich C

更新日期：2004-08-01 00:00:00

abstract：:Highly clustered count data are commonly seen in medical device clinical studies such as cardiac rhythm management. For instance, anti-arrhythmia shocks delivered from an implantable cardioverter-defibrillator (ICD) often occur as "storms", i.e., multiple shocks within a short period of time. There are unique challeng...

journal_title：Journal of biopharmaceutical statistics

authors： Wang ZJ

更新日期：2008-01-01 00:00:00

abstract：:Testing for noninferiority and equivalence between an experimental therapy and a standard therapy in terms of the ratio of binomial proportions is considered. New tests based on the Fieller-Hinkley distribution of the ratio of random variables are proposed. Restricted maximum likelihood estimates of the null variances...

journal_title：Journal of biopharmaceutical statistics

authors： Koti KM

更新日期：2007-01-01 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：:When marketed lots of pharmaceutical products produce a number of out-of-specification (OOS) samples, it can be very disruptive. In some cases such lots must be recalled, at substantial expense. We present a model of the probability of OOS samples and show quantitatively how this probability depends on the underlying ...

journal_title：Journal of biopharmaceutical statistics

authors： Fairweather WR

更新日期：2003-08-01 00:00:00

abstract：:Multiple comparisons are commonly seen in clinical trials and many other fields. An example, which is the focus of this paper, is the comparison of several test treatments (possibly different doses of a compound) with placebo (control). It is well known that steps must be taken to control the type I error rate when mu...

journal_title：Journal of biopharmaceutical statistics

authors： Chen M,Kianifard F

更新日期：1997-07-01 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：: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：:Rank-based test procedures in censored survival data differ considerably in their sensitivity to various alternatives to the hypothesis of equality of underlying distributions. Procedures based on the simultaneous use of multiple statistics provide an appealing approach to obtaining more global sensitivity while maint...

journal_title：Journal of biopharmaceutical statistics

authors： Yang P,Fleming TR

更新日期：2006-01-01 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：:The proportion ratio (PR) of a positive response between an experimental treatment and a standard treatment (or placebo) is often used to measure the relative treatment efficacy in a randomized clinical trial (RCT). For ethical reasons, it is almost inevitable to encounter some patients not complying with their assign...

journal_title：Journal of biopharmaceutical statistics

authors： Lui KJ,Chang KC

更新日期：2012-01-01 00:00:00