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 ranks were studied. All nonparametric step-down procedures controlled familywise error rate under normal, double exponential, and exponential distributions. Specific recommendations based on power and bias comparisons of nonparametric methods among themselves, with Shirley's (2) test, and with parametric step-down multiple-test procedures are given.
journal_name
J Biopharm Statjournal_title
Journal of biopharmaceutical statisticsauthors
Sidik K,Morris RWdoi
10.1081/BIP-100101173keywords:
subject
Has Abstractpub_date
1999-05-01 00:00:00pages
217-40issue
2eissn
1054-3406issn
1520-5711journal_volume
9pub_type
杂志文章abstract::A randomized, active-control clinical trial setting with the objective of testing noninferiority for a continuous response variable is considered. Noninferiority margin is based on the concept of preserving a certain fraction of the active control effect. Noninferiority is established if the ratio of the lower (upper)...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543400600609478
更新日期:2006-05-01 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
pub_type: 杂志文章
doi:10.1080/10543400802287230
更新日期:2008-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2014.979197
更新日期:2015-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2017.1289943
更新日期:2017-01-01 00:00:00
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, castin...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2013.813516
更新日期:2013-01-01 00:00:00
abstract::Rubinstein et al. presented a procedure for determining the required duration of accrual for a clinical trial comparing the survival distributions of two treatments using a classical hypothesis testing formulation. Here their testing procedure is modified in two ways. First, the asymptotic variances used in computatio...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409308835056
更新日期:1993-09-01 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
pub_type: 杂志文章
doi:10.1081/BIP-200025678
更新日期:2004-08-01 00:00:00
abstract::How long a dementia patient is cared for in the home before admission to a nursing home depends on the state of the patient and the state of the caregiver. Using 5-year follow-up data, the times until entry to nursing home and until death are modeled using a Cox survival model in which patient and caregiver variables ...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409408835076
更新日期:1994-03-01 00:00:00
abstract::We propose a new adaptive threshold detection and enrichment design in which the biomarker threshold is adaptively estimated and updated by optimizing a trade-off between the size of the biomarker positive population and the magnitude of the treatment effect in that population. Enrichment is based on an enrollment cri...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2020.1832110
更新日期:2020-11-11 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
abstract::Nonparametric methods are presented for the analysis of the two-treatment, two-period crossover design with multivariate response. After forming within-subject sums and differences, the usual tests, including those for carry-over effects and direct treatment effects, can be constructed using a multivariate analysis of...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409308835045
更新日期:1993-03-01 00:00:00
abstract::A main challenge in molecular diagnostic research is to accurately evaluate the performance of a new nucleic acid amplification test when the reference standard is imperfect. Several approaches, such as discrepant analysis, composite reference standard (CRS) method, or latent class analysis (LCA), are commonly applied...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2018.1428613
更新日期:2018-01-01 00:00:00
abstract::Dichotomizing a continuous biomarker is a common practice in medical research. Various methods exist in the literature for dichotomizing continuous biomarkers. The most widely adopted minimum p-value approach uses a sequence of test statistics for all possible dichotomizations of a continuous biomarker, and it chooses...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2012.756503
更新日期:2013-05-01 00:00:00
abstract::It is well known that there is strong relationship between HIV viral load and CD4 cell counts in AIDS studies. However, the relationship between them changes during the course of treatment and may vary among individuals. During treatments, some individuals may experience terminal events such as death. Because the term...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2015.1052493
更新日期:2016-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2019.1657142
更新日期:2019-01-01 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
pub_type: 临床试验,杂志文章
doi:10.1080/10543409408835078
更新日期:1994-07-01 00:00:00
abstract::An ability to predict the metabolic fate of a drug is important to drug design. Programs for predicting drug metabolites are becoming available, as are databases that will facilitate the development of such programs. Objective analysis of the performance of these programs will require statistical methods. The developm...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543409108835004
更新日期:1991-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2016.1226324
更新日期:2016-01-01 00:00:00
abstract::We consider the problem of estimating a biomarker-based subgroup and testing for treatment effect in the overall population and in the subgroup after the trial. We define the best subgroup as the subgroup that maximizes the power for comparing the experimental treatment with the control. In the case of continuous outc...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2019.1633655
更新日期:2019-01-01 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
pub_type: 杂志文章
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543409408835089
更新日期:1994-11-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章
doi:10.1080/10543406.2014.901340
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
doi:10.1080/10543406.2013.834916
更新日期:2013-01-01 00:00:00
abstract::The world of medical devices while highly diverse is extremely innovative, and this facilitates the adoption of innovative statistical techniques. Statisticians in the Center for Devices and Radiological Health (CDRH) at the Food and Drug Administration (FDA) have provided leadership in implementing statistical innova...
journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
doi:10.1080/10543406.2015.1092037
更新日期:2016-01-01 00:00:00
abstract::For noninferiority testing with the maximum allowable noninferiority margin being prespecified, one can perform valid statistical testing at the same alpha level for multiple noninferiority hypotheses with margins being smaller than this maximum margin. This is easily comprehensible because only one confidence level i...
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::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
pub_type: 杂志文章,评审
doi:10.1080/10543409208835036
更新日期:1992-01-01 00:00:00
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journal_title:Journal of biopharmaceutical statistics
pub_type: 杂志文章,评审
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更新日期:2012-01-01 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
pub_type: 杂志文章
doi:10.1080/10543409508835115
更新日期:1995-11-01 00:00:00
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
pub_type: 杂志文章
doi:10.1080/10543406.2017.1399901
更新日期:2018-01-01 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
pub_type: 杂志文章
doi:10.1080/10543406.2017.1377728
更新日期:2018-01-01 00:00:00