Designing phase II studies in the context of a programme of clinical research.

Abstract:

:Conventional statistical determinations of sample size in phase II studies typically lead to sample sizes of the order of 25 (Schoenfeld, 1980, International Journal of Radiation Oncology, Biology and Physics 6, 371-374). When the development of new treatments is proceeding rapidly relative to the recruitment of suitable patients, such requirements can prove to be too demanding. As a result, either sample sizes are reduced by a rather arbitrary weakening of the risk specifications, or certain new treatments go untested. In this paper, the phase II testing of a number of treatments will be considered as a single study which has the objective of identifying the most promising treatment for phase III investigation. It is seen to be advantageous to test more treatments, with fewer subjects receiving each, than the conventional methods would allow.

journal_name

Biometrics

journal_title

Biometrics

authors

Whitehead J

subject

Has Abstract

pub_date

1985-06-01 00:00:00

pages

373-83

issue

2

eissn

0006-341X

issn

1541-0420

journal_volume

41

pub_type

杂志文章
  • On modelling microbial infections.

    abstract::Several models for the course of microbial infections during the incubation period are examined. Each model fits sore throat incubation data very well. Together with the lack of precision of the resulting parameter estimates, this suggests that incubation data alone are insufficient for elucidating the finer details o...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Morgan BJ,Watts SA

    更新日期:1980-06-01 00:00:00

  • Bayesian modeling of multiple episode occurrence and severity with a terminating event.

    abstract::An individual's health condition can affect the frequency and intensity of episodes that can occur repeatedly and that may be related to an event time of interest. For example, bleeding episodes during pregnancy may indicate problems predictive of preterm delivery. Motivated by this application, we propose a joint mod...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00720.x

    authors: Herring AH,Yang J

    更新日期:2007-06-01 00:00:00

  • Multivariate survival analysis using piecewise gamma frailty.

    abstract::In this note we propose a frailty model called piecewise gamma frailty for correlated survival data with random effects having a nested structure. In frailty models, a dependence function defined as a hazard ratio of one member given the failure time of another member in a unit is determined by the distributional assu...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Paik MC,Tsai WY,Ottman R

    更新日期:1994-12-01 00:00:00

  • Power and sample size for testing homogeneity of relative risks in prospective studies.

    abstract::Power and sample-size formulas for testing the homogeneity of relative risks using the score method are presented. The homogeneity score test (Gart, 1985, Biometrika 72, 673-677) is formally equivalent to the Pearson chi-square test, although they look different. Results of this paper may be useful in assessing the va...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.1999.00289.x

    authors: Nam JM

    更新日期:1999-03-01 00:00:00

  • Bayesian model-averaged benchmark dose analysis via reparameterized quantal-response models.

    abstract::An important objective in biomedical and environmental risk assessment is estimation of minimum exposure levels that induce a pre-specified adverse response in a target population. The exposure points in such settings are typically referred to as benchmark doses (BMDs). Parametric Bayesian estimation for finding BMDs ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12340

    authors: Fang Q,Piegorsch WW,Simmons SJ,Li X,Chen C,Wang Y

    更新日期:2015-12-01 00:00:00

  • Capture-recapture when time and behavioral response affect capture probabilities.

    abstract::We consider a capture-recapture model in which capture probabilities vary with time and with behavioral response. Two inference procedures are developed under the assumption that recapture probabilities bear a constant relationship to initial capture probabilities. These two procedures are the maximum likelihood metho...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2000.00427.x

    authors: Chao A,Chu W,Hsu CH

    更新日期:2000-06-01 00:00:00

  • A model-based approach for making ecological inference from distance sampling data.

    abstract::We consider a fully model-based approach for the analysis of distance sampling data. Distance sampling has been widely used to estimate abundance (or density) of animals or plants in a spatially explicit study area. There is, however, no readily available method of making statistical inference on the relationships bet...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2009.01265.x

    authors: Johnson DS,Laake JL,Ver Hoef JM

    更新日期:2010-03-01 00:00:00

  • Bayesian calibration of a stochastic kinetic computer model using multiple data sources.

    abstract::In this article, we describe a Bayesian approach to the calibration of a stochastic computer model of chemical kinetics. As with many applications in the biological sciences, the data available to calibrate the model come from different sources. Furthermore, these data appear to provide somewhat conflicting informatio...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2009.01245.x

    authors: Henderson DA,Boys RJ,Wilkinson DJ

    更新日期:2010-03-01 00:00:00

  • Robust tests for treatment effects based on censored recurrent event data observed over multiple periods.

    abstract::We derive semiparametric methods for estimating and testing treatment effects when censored recurrent event data are available over multiple periods. These methods are based on estimating functions motivated by a working "mixed-Poisson" assumption under which conditioning can eliminate subject-specific random effects....

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2005.00357.x

    authors: Cook RJ,Wei W,Yi GY

    更新日期:2005-09-01 00:00:00

  • Regression calibration in semiparametric accelerated failure time models.

    abstract::In large cohort studies, it often happens that some covariates are expensive to measure and hence only measured on a validation set. On the other hand, relatively cheap but error-prone measurements of the covariates are available for all subjects. Regression calibration (RC) estimation method (Prentice, 1982, Biometri...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2009.01295.x

    authors: Yu M,Nan B

    更新日期:2010-06-01 00:00:00

  • A Bayesian approach to the analysis of quantal bioassay studies using nonparametric mixture models.

    abstract::We develop a Bayesian nonparametric mixture modeling framework for quantal bioassay settings. The approach is built upon modeling dose-dependent response distributions. We adopt a structured nonparametric prior mixture model, which induces a monotonicity restriction for the dose-response curve. Particular emphasis is ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12120

    authors: Fronczyk K,Kottas A

    更新日期:2014-03-01 00:00:00

  • Variable selection for logistic regression using a prediction-focused information criterion.

    abstract::In biostatistical practice, it is common to use information criteria as a guide for model selection. We propose new versions of the focused information criterion (FIC) for variable selection in logistic regression. The FIC gives, depending on the quantity to be estimated, possibly different sets of selected variables....

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00567.x

    authors: Claeskens G,Croux C,Van Kerckhoven J

    更新日期:2006-12-01 00:00:00

  • Marginal modeling of binary cross-over data.

    abstract::A model specified in terms of linear models for marginal logits and linear models for log-odds ratios is proposed for the analysis of two-period binary cross-over experiments. Hypothesis testing and parameter estimation are facilitated by standard likelihood methodology. Two examples are used to illustrate how the mod...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Becker MP,Balagtas CC

    更新日期:1993-12-01 00:00:00

  • Comparing the performances of Diggle's tests of spatial randomness for small samples with and without edge-effect correction: application to ecological data.

    abstract::Diggle's tests of spatial randomness based on empirical distributions of interpoint distances can be performed with and without edge-effect correction. We present here numerical results illustrating that tests without the edge-effect correction proposed by Diggle (1979, Biometrics 35, 87-101) have a higher power for s...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.1999.00156.x

    authors: Gignoux J,Duby C,Barot S

    更新日期:1999-03-01 00:00:00

  • Paired versus two-sample design for a clinical trial of treatments with dichotomous outcome: power considerations.

    abstract::For the same number of observations in a small-sample clinical trial with dichotomous outcome, the statistical power associated with a two-sample design, analyzed by Fisher's exact test, is slightly greater than that associated with a matched design, analyzed by McNemar's test, and hence of the matched design, is mono...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Wacholder S,Weinberg CR

    更新日期:1982-09-01 00:00:00

  • First passage times as environmental safety indicators: carboxyhemoglobin from cigarette smoke.

    abstract::The concentration of carbon monoxide in the blood of a cigarette smoker varies in response to the frequency and dose of CO delivered by the cigarettes he smokes and by the rate at which CO washes out of his blood. Moments of first passage times or exit times above a nominal threshold can be calculated using a stochast...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Marcus AH,Czajkowski S Jr

    更新日期:1979-09-01 00:00:00

  • Exact inference on the random-effects model for meta-analyses with few studies.

    abstract::We describe an exact, unconditional, non-randomized procedure for producing confidence intervals for the grand mean in a normal-normal random effects meta-analysis. The procedure targets meta-analyses based on too few primary studies, ≤ 7 , say, to ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12998

    authors: Michael H,Thornton S,Xie M,Tian L

    更新日期:2019-06-01 00:00:00

  • An improved life table method.

    abstract::A life table estimates probabilities of surviving and of dying as well as death rates, as these would apply in a stationary population with the same underlying continuous mortality curve as the observed population. We have derived approximations to the probability of surviving that require no iteration, do not depend ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Keyfitz N,Frauenthal J

    更新日期:1975-12-01 00:00:00

  • Impact of time to start treatment following infection with application to initiating HAART in HIV-positive patients.

    abstract::We estimate how the effect of antiretroviral treatment depends on the time from HIV-infection to initiation of treatment, using observational data. A major challenge in making inferences from such observational data arises from biases associated with the nonrandom assignment of treatment, for example bias induced by d...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2011.01738.x

    authors: Lok JJ,DeGruttola V

    更新日期:2012-09-01 00:00:00

  • Extension of the rank sum test for clustered data: two-group comparisons with group membership defined at the subunit level.

    abstract::The Wilcoxon rank sum test is widely used for two-group comparisons for nonnormal data. An assumption of this test is independence of sampling units both between and within groups. In ophthalmology, data are often collected on two eyes of an individual, which are highly correlated. In ophthalmological clinical trials,...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00582.x

    authors: Rosner B,Glynn RJ,Lee ML

    更新日期:2006-12-01 00:00:00

  • Joint modeling of progression of HIV resistance mutations measured with uncertainty and failure time data.

    abstract::Development of HIV resistance mutations is a major cause for failure of antiretroviral treatment. This article proposes a method for jointly modeling the processes of viral genetic changes and treatment failure. Because the viral genome is measured with uncertainty, a hidden Markov model is used to fit the viral genet...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00635.x

    authors: Hu C,De Gruttola V

    更新日期:2007-03-01 00:00:00

  • Capture-recapture estimation using finite mixtures of arbitrary dimension.

    abstract::Reversible jump Markov chain Monte Carlo (RJMCMC) methods are used to fit Bayesian capture-recapture models incorporating heterogeneity in individuals and samples. Heterogeneity in capture probabilities comes from finite mixtures and/or fixed sample effects allowing for interactions. Estimation by RJMCMC allows automa...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2009.01289.x

    authors: Arnold R,Hayakawa Y,Yip P

    更新日期:2010-06-01 00:00:00

  • An improved method for measuring heart-rate variability: assessment of cardiac autonomic function.

    abstract::Heart rate oscillates in synchrony with respiration. Several methods have been employed to assess this 'sinus arrhythmia', as an index of autonomic nervous system function. This paper proposes a new, easily computed measure, R, which is relatively resistant to the major nonrespiratory sources of variation, including p...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Weinberg CR,Pfeifer MA

    更新日期:1984-09-01 00:00:00

  • Prediction approaches to sequentially searching for an optimal dose.

    abstract::The search for an optimal dose of a drug has been investigated in a sequential setting to ensure the safety level by Eichhorn and Zacks (1973, Journal of the American Statistical Association 68, 594-598; 1981, Communications in Statistics--Theory and Methods A10, 931-953), Eichhorn (1974, Communications in Statistics ...

    journal_title:Biometrics

    pub_type: 临床试验,杂志文章

    doi:

    authors: Shih WJ

    更新日期:1989-06-01 00:00:00

  • Additive gamma frailty models with applications to competing risks in related individuals.

    abstract::Epidemiological studies of related individuals are often complicated by the fact that follow-up on the event type of interest is incomplete due to the occurrence of other events. We suggest a class of frailty models with cause-specific hazards for correlated competing events in related individuals. The frailties are b...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12326

    authors: Eriksson F,Scheike T

    更新日期:2015-09-01 00:00:00

  • Selecting differentially expressed genes from microarray experiments.

    abstract::High throughput technologies, such as gene expression arrays and protein mass spectrometry, allow one to simultaneously evaluate thousands of potential biomarkers that could distinguish different tissue types. Of particular interest here is distinguishing between cancerous and normal organ tissues. We consider statist...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/1541-0420.00016

    authors: Pepe MS,Longton G,Anderson GL,Schummer M

    更新日期:2003-03-01 00:00:00

  • Semiparametric regression of multidimensional genetic pathway data: least-squares kernel machines and linear mixed models.

    abstract::We consider a semiparametric regression model that relates a normal outcome to covariates and a genetic pathway, where the covariate effects are modeled parametrically and the pathway effect of multiple gene expressions is modeled parametrically or nonparametrically using least-squares kernel machines (LSKMs). This un...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2007.00799.x

    authors: Liu D,Lin X,Ghosh D

    更新日期:2007-12-01 00:00:00

  • Sensitivity analysis for nonrandom dropout: a local influence approach.

    abstract::Diggle and Kenward (1994, Applied Statistics 43, 49-93) proposed a selection model for continuous longitudinal data subject to nonrandom dropout. It has provoked a large debate about the role for such models. The original enthusiasm was followed by skepticism about the strong but untestable assumptions on which this t...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2001.00007.x

    authors: Verbeke G,Molenberghs G,Thijs H,Lesaffre E,Kenward MG

    更新日期:2001-03-01 00:00:00

  • A group sequential procedure for all-pairwise comparisons of k treatments based on the range statistic.

    abstract::In this paper, a group sequential procedure for all-private comparisons of the means of k independent normal populations with a common known variance is proposed. A repeated range test is defined and its critical points are tabulated. The power function is studied and minimum group size needed to achieve a desirable p...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Liu W

    更新日期:1995-09-01 00:00:00

  • Comment on "Wang et al. (2005), Robust estimating functions and bias correction for longitudinal data analysis".

    abstract::This note provides a discussion on the manuscript by Wang et al. (2005) who aim to robustify inference for longitudinal data analysis by replacing the ordinary generalized estimating function with an influence-bounded, possibly biased, version. To adjust for the bias of the ensuing robust estimator, the authors provid...

    journal_title:Biometrics

    pub_type: 信件

    doi:10.1111/biom.13263

    authors: Lunardon N,Menardi G

    更新日期:2020-09-01 00:00:00