Abstract:
:To confirm results obtained from local evaluation at investigational centers, many oncology studies utilize blinded independent central review (BICR) to make assessments of the primary endpoint, progression-free survival (PFS). The comparison of data often leads to large discordances between these observations, casting doubt on the reliability of the estimated treatment effects from these trials. Here we propose new statistics to measure discordance and evaluate their utility to detect bias in the local evaluation of progression relative to the standard measures of discordance. A new observational error model is proposed that can be used to describe the discordance in patient assessments between multiple readers.
journal_name
J Biopharm Statjournal_title
Journal of biopharmaceutical statisticsauthors
Mannino FV,Amit O,Lahiri Sdoi
10.1080/10543406.2013.813516subject
Has Abstractpub_date
2013-01-01 00:00:00pages
971-85issue
5eissn
1054-3406issn
1520-5711journal_volume
23pub_type
杂志文章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
pub_type: 杂志文章
doi:10.1080/10543406.2016.1265536
更新日期:2017-01-01 00:00:00
abstract::We extend the development of the expression of the Fisher information matrix in nonlinear mixed effects models for designs evaluation. We consider the dependence of the marginal variance of the observations with the mean parameters and assume an heteroscedastic variance error model. Complex models with interoccasions ...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1081/BIP-120019267
更新日期:2003-05-01 00:00:00
abstract::All too often in clinical trials the assessment of quality of life is seen as a bolt-on study. Consequently insufficient consideration is often given to its design, collection, analysis and presentation, and its impact on the trial results and on clinical practice is minimal. In many trials quality of life is a key en...
journal_title:Journal of biopharmaceutical statistics
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400701329497
更新日期:2007-01-01 00:00:00
abstract::I review the designs available for Phase I dose-finding studies of chemotherapeutic agents in cancer patients. The designs are based on the assumption that both efficacy and toxicity increase with dose, and thus attempt to minimize the number of patients treated at low doses, and also to minimize the chance that patie...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543400600860295
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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
pub_type: 杂志文章
doi:10.1081/BIP-120028516
更新日期:2004-02-01 00:00:00
abstract::Sample size calculation formulas for testing equality, noninferiority, superiority, and equivalence based on odds ratio were derived under both parallel and one-arm crossover designs. An example concerning the study of odds ratio between a test compound (treatment) and a standard therapy (control) for prevention of re...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1081/BIP-120016231
更新日期:2002-11-01 00:00:00
abstract::In many clinical trials for chronic conditions a run-in period is used prior to randomization. Often, only those participants who meet certain criteria during the run-in phase go on to get randomized. The others, along with the information that they might have provided, are excluded from the study. This exclusion of t...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2011.550107
更新日期:2011-03-01 00:00:00
abstract::The sequential parallel comparison design (SPCD) is a two-stage design recommended for trials with possibly high placebo response. A drug-placebo comparison in the first stage is followed in the second stage by placebo nonresponders being re-randomized between drug and placebo. We describe how SPCD can be used in tria...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2014.924960
更新日期:2014-01-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
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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
pub_type: 杂志文章
doi:10.1080/10543406.2014.979197
更新日期:2015-01-01 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
pub_type: 杂志文章
doi:10.1081/BIP-200067988
更新日期:2005-01-01 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
pub_type: 杂志文章
doi:10.1080/10543400701645165
更新日期:2007-01-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2013.834916
更新日期:2013-01-01 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
pub_type: 杂志文章
doi:10.1080/10543400601001527
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409808835253
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1081/BIP-120037183
更新日期:2004-05-01 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
pub_type: 杂志文章,评审
doi:10.1080/10543406.2015.1092031
更新日期:2016-01-01 00:00:00
abstract::We assess the QT effect using an exposure-response model in a "thorough QT/QTc study" with a four-period crossover design in which the treatments are placebo, positive control, higher dose of investigational drug, and therapeutic dose of investigational drug. In the study, QTc interval values and the drug concentratio...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400903582026
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journal_title:Journal of biopharmaceutical statistics
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2013.735762
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2017.1289943
更新日期:2017-01-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400802071360
更新日期:2008-01-01 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
pub_type: 杂志文章,评审
doi:10.1080/10543406.2014.972511
更新日期:2015-01-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409608835129
更新日期:1996-05-01 00:00:00
abstract::Fisher's exact test and Pearson's chi-square with continuity correction are frequently employed in the analysis of epidemiological data involving a 2 x 2 contingency table. This paper reviews the concepts and controversies underlying these procedures and discusses their appropriateness and adequacies in analyzing such...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543409508835098
更新日期:1995-03-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
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更新日期:2007-01-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
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更新日期:2006-05-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2014.941985
更新日期:2014-01-01 00:00:00