Play the winner for phase II/III clinical trials.

Abstract:

:In comparing two treatments under a typical sequential clinical trial setting, a 50-50 randomization design generates reliable data for making efficient inferences about the treatment difference for the benefit of patients in the general population. However, if the treatment difference is large and the endpoint of the study is potentially fatal, it does not seem appropriate to sacrifice a large number of study patients who are assigned to the inferior arm. An adaptive design is a data-dependent treatment allocation rule that sequentially uses accumulating information about the treatment difference during the trial to modify the allocation rule for new study patients. In this article, we utilize real trials from AIDS and cancer to illustrate the advantage of using adaptive designs. Specifically we show that, with adaptive designs, the loss of power for testing the equality of two treatments is negligible. Moreover, the study patients do not have to pay a handsome price for the benefit of future patients. We also propose multi-stage adaptive rules to relax the administrative burden of implementing the study and to handle continuous response variables, such as the failure time in survival analysis.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Yao Q,Wei LJ

doi

10.1002/(sici)1097-0258(19961130)15:22<2413::aid-s

subject

Has Abstract

pub_date

1996-11-15 00:00:00

pages

2413-23; discussion 2455-8

issue

21-22

eissn

0277-6715

issn

1097-0258

pii

10.1002/(SICI)1097-0258(19961130)15:22<2413::AID-S

journal_volume

15

pub_type

杂志文章
  • A numerical strategy to evaluate performance of predictive scores via a copula-based approach.

    abstract::Assessing and comparing the performance of correlated predictive scores are of current interest in precision medicine. Given the limitations of available theoretical approaches for assessing and comparing the predictive accuracy, numerical methods are highly desired which, however, have not been systematically develop...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8566

    authors: Zhang Y,Shao Y

    更新日期:2020-09-10 00:00:00

  • Statistical comparison of two handwashing protocols.

    abstract::This paper describes statistical procedures for use in an experiment that compares two handwashing protocols. The evaluation of a handwashing protocol entails collection of the wash effluent. Colony counts for the effluent reflect the number of flora removed by the wash protocol. The analysis aims to formulate and est...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1002/sim.4780050412

    authors: Le CT

    更新日期:1986-07-01 00:00:00

  • Simple models for repeated ordinal responses with an application to a seasonal rhinitis clinical trial.

    abstract::In contrast to other models for ordinal data, the continuation ratio model can be fitted with standard statistical software. This makes it particularly appropriate for large clinical trials with ordinal response variables. In addition, when the trials are longitudinal, this model can be applied to individual responses...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1002/(sici)1097-0258(19971230)16:24<2873::aid-s

    authors: Lindsey JK,Jones B,Ebbutt AF

    更新日期:1997-12-30 00:00:00

  • Aggregation of existing geographic regions to diminish spurious variability of disease rates.

    abstract::The availability of large data sets together with the growth in power and storage capabilities of computers have made the analysis of the spatial distribution of disease rates an increasingly important tool in public health research. Use of existing geographic divisions or groupings tends to result either in unstable ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780121916

    authors: Morris RD,Munasinghe RL

    更新日期:1993-10-01 00:00:00

  • Consequences of event rate heterogeneity across non-randomized study sub-groups.

    abstract::Analyses to compare non-randomized groups are more and more common in both post hoc analyses of randomized clinical trials data and in analyses of long-term observational data. In such cases, it is quite likely that there are unknown or uncollected sources of heterogeneity in event rates. Research has shown that an un...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1795

    authors: Eberly LE

    更新日期:2004-07-15 00:00:00

  • Estimating adjusted risk difference (RD) and number needed to treat (NNT) measures in the Cox regression model.

    abstract::In medical research, risk difference (RD) and number needed to treat (NNT) measures for survival times have been mainly proposed without consideration of covariates. In this paper, we develop adjusted RD and NNT measures for use in observational studies with survival time outcomes within the framework of the Cox propo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3793

    authors: Laubender RP,Bender R

    更新日期:2010-03-30 00:00:00

  • A special case of reduced rank models for identification and modelling of time varying effects in survival analysis.

    abstract::Flexible survival models are in need when modelling data from long term follow-up studies. In many cases, the assumption of proportionality imposed by a Cox model will not be valid. Instead, a model that can identify time varying effects of fixed covariates can be used. Although there are several approaches that deal ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7088

    authors: Perperoglou A

    更新日期:2016-12-10 00:00:00

  • Selection of patients for randomized controlled trials: implications of wide or narrow eligibility criteria.

    abstract::This paper discusses the various philosophies that influence the selection of patients for entry into randomized controlled trials. Although a number of different and often competing issues have to be considered depending upon the trial, keeping entry criteria simple, wide and at times even flexible is usually prefera...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780090114

    authors: Yusuf S,Held P,Teo KK,Toretsky ER

    更新日期:1990-01-01 00:00:00

  • Discriminant analysis using a multivariate linear mixed model with a normal mixture in the random effects distribution.

    abstract::We have developed a method to longitudinally classify subjects into two or more prognostic groups using longitudinally observed values of markers related to the prognosis. We assume the availability of a training data set where the subjects' allocation into the prognostic group is known. The proposed method proceeds i...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3849

    authors: Komárek A,Hansen BE,Kuiper EM,van Buuren HR,Lesaffre E

    更新日期:2010-12-30 00:00:00

  • Sample size calculations for comparative studies of medical tests for detecting presence of disease.

    abstract::Technologic advances give rise to new tests for detecting disease in many fields, including cancer and sexually transmitted disease. Before a new disease screening test is approved for public use, its accuracy should be shown to be better than or at least not inferior to an existing test. Standards do not yet exist fo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1058

    authors: Alonzo TA,Pepe MS,Moskowitz CS

    更新日期:2002-03-30 00:00:00

  • What are the statistical implications of treatment non-compliance in cluster randomized trials: A simulation study.

    abstract::Subjects in randomized controlled trials do not always comply to the treatment condition they have been assigned to. This may cause the estimated effect of the intervention to be biased and also affect efficiency, coverage of confidence intervals, and statistical power. In cluster randomized trials non-compliance may ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8351

    authors: Moerbeek M,Schie SV

    更新日期:2019-11-20 00:00:00

  • Identifying representative trees from ensembles.

    abstract::Tree-based methods have become popular for analyzing complex data structures where the primary goal is risk stratification of patients. Ensemble techniques improve the accuracy in prediction and address the instability in a single tree by growing an ensemble of trees and aggregating. However, in the process, individua...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4492

    authors: Banerjee M,Ding Y,Noone AM

    更新日期:2012-07-10 00:00:00

  • Measurement in clinical trials: a neglected issue for statisticians?

    abstract::Biostatisticians have frequently uncritically accepted the measurements provided by their medical colleagues engaged in clinical research. Such measures often involve considerable loss of information. Particularly, unfortunate is the widespread use of the so-called 'responder analysis', which may involve not only a lo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3603

    authors: Senn S,Julious S

    更新日期:2009-11-20 00:00:00

  • Meta-analysis combining parallel and cross-over clinical trials. II: Binary outcomes.

    abstract::We examine different methods to pool binary outcomes used both in parallel and cross-over trials. Odds ratio (OR) estimators obtained from joint conditional probabilities in cross-over trials, such as the Mantel-Haenszel and Peto methods, are compared to an OR estimator using marginal results of cross-over trials. Whe...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1206

    authors: Curtin F,Elbourne D,Altman DG

    更新日期:2002-08-15 00:00:00

  • Analysis of panel data under hidden mover-stayer models.

    abstract::Analysis of panel data is often challenged by the presence of heterogeneity and state misclassification. In this paper, we propose a hidden mover-stayer model to facilitate heterogeneity for a population that consists of two subpopulations each of movers or of stayers and to simultaneously account for state misclassif...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7346

    authors: Yi GY,He W,He F

    更新日期:2017-09-10 00:00:00

  • A mediation analysis for a nonrare dichotomous outcome with sequentially ordered multiple mediators.

    abstract::Mediation analyses can help us to understand the biological mechanism in which an exposure or treatment affects an outcome. Single mediator analyses have been used in various applications, but may not be appropriate for analyzing intricate mechanisms involving multiple mediators that affect each other. Thus, in this a...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8485

    authors: Lai EY,Shih S,Huang YT,Wang S

    更新日期:2020-05-15 00:00:00

  • Chronic disease prevention: public health potential and research needs.

    abstract::This paper, arising out of an event to honour the statistical and scientific contributions of Professor Peter Armitage, is concerned with research strategies and needs for chronic disease prevention. A few highlights from recent intervention trials for the prevention of cancer, cardiovascular disease, fractures and di...

    journal_title:Statistics in medicine

    pub_type:

    doi:10.1002/sim.2045

    authors: Prentice RL

    更新日期:2004-11-30 00:00:00

  • Case-control analysis with a continuous outcome variable.

    abstract::It is not uncommon for a continuous outcome variable Y to be dichotomized and analysed using logistic regression. Moser and Coombs (Statist. Med. 2004; 23:1843-1860) provide a method for converting the output from a standard linear regression analysis using the original continuous outcome Y to give much more efficient...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.3474

    authors: Jiang Y,Scott A,Wild CJ

    更新日期:2009-01-30 00:00:00

  • Exact and asymptotic inference in clinical trials with small event rates under inverse sampling.

    abstract::In this paper, we discuss statistical inference for a 2 × 2 table under inverse sampling, where the total number of cases is fixed by design. We demonstrate that the exact unconditional distributions of some relevant statistics differ from the distributions under conventional sampling, where the sample size is fixed b...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6511

    authors: Heimann G,Von Tress M,Gasparini M

    更新日期:2015-08-30 00:00:00

  • A Naive Bayes machine learning approach to risk prediction using censored, time-to-event data.

    abstract::Predicting an individual's risk of experiencing a future clinical outcome is a statistical task with important consequences for both practicing clinicians and public health experts. Modern observational databases such as electronic health records provide an alternative to the longitudinal cohort studies traditionally ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6526

    authors: Wolfson J,Bandyopadhyay S,Elidrisi M,Vazquez-Benitez G,Vock DM,Musgrove D,Adomavicius G,Johnson PE,O'Connor PJ

    更新日期:2015-09-20 00:00:00

  • Dynamic Cox modelling based on fractional polynomials: time-variations in gastric cancer prognosis.

    abstract::The most popular model used for survival analysis is the proportional hazards regression model proposed by Cox. This is mainly due to its exceptional simplicity. Nevertheless the fundamental assumption of the Cox model is the proportionality of the hazards. For many applications, however, this assumption is doubtful. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1411

    authors: Berger U,Schäfer J,Ulm K

    更新日期:2003-04-15 00:00:00

  • Quantifying the impact of between-study heterogeneity in multivariate meta-analyses.

    abstract::Measures that quantify the impact of heterogeneity in univariate meta-analysis, including the very popular I(2) statistic, are now well established. Multivariate meta-analysis, where studies provide multiple outcomes that are pooled in a single analysis, is also becoming more commonly used. The question of how to quan...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5453

    authors: Jackson D,White IR,Riley RD

    更新日期:2012-12-20 00:00:00

  • Interval estimation for rank correlation coefficients based on the probit transformation with extension to measurement error correction of correlated ranked data.

    abstract::The Spearman (rho(s)) and Kendall (tau) rank correlation coefficient are routinely used as measures of association between non-normally distributed random variables. However, confidence limits for rho(s) are only available under the assumption of bivariate normality and for tau under the assumption of asymptotic norma...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2547

    authors: Rosner B,Glynn RJ

    更新日期:2007-02-10 00:00:00

  • Confidence intervals for an exposure adjusted incidence rate difference with applications to clinical trials.

    abstract::To summarize safety data such as clinical adverse experiences in clinical trials with a moderate to long-term follow-up, we may use a measurement which accounts for the potential differences in the follow-up duration between treatment groups. The incidence rate, which uses the total person-time follow-up in a treatmen...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2335

    authors: Liu GF,Wang J,Liu K,Snavely DB

    更新日期:2006-04-30 00:00:00

  • Structured correlation in models for clustered data.

    abstract::Correlation is always a concern in the analysis of clustered data. One area of interest is to develop a general correlation modelling approach for high dimensional data with unbalanced hierarchical and heterogeneous data structures, e.g. multilevel data. Commonly used correlation structures might have limitation for s...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2368

    authors: Chao EC

    更新日期:2006-07-30 00:00:00

  • Likelihood-based methods for regression analysis with binary exposure status assessed by pooling.

    abstract::The need for resource-intensive laboratory assays to assess exposures in many epidemiologic studies provides ample motivation to consider study designs that incorporate pooled samples. In this paper, we consider the case in which specimens are combined for the purpose of determining the presence or absence of a pool-w...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4426

    authors: Lyles RH,Tang L,Lin J,Zhang Z,Mukherjee B

    更新日期:2012-09-28 00:00:00

  • Designing therapeutic cancer vaccine trials with delayed treatment effect.

    abstract::Arming the immune system against cancer has emerged as a powerful tool in oncology during recent years. Instead of poisoning a tumor or destroying it with radiation, therapeutic cancer vaccine, a type of cancer immunotherapy, unleashes the immune system to combat cancer. This indirect mechanism-of-action of vaccines p...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7157

    authors: Xu Z,Zhen B,Park Y,Zhu B

    更新日期:2017-02-20 00:00:00

  • Exact approaches for testing hypotheses based on the intra-class kappa coefficient.

    abstract::Testing involving the intra-class kappa coefficient is commonly performed in order to assess agreement involving categorical ratings. A number of procedures have been proposed, which make use of the limiting null distribution as the sample size goes to infinity in order to compute the observed significance. As with ma...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6135

    authors: Wilding GE,Consiglio JD,Shan G

    更新日期:2014-07-30 00:00:00

  • Comparison of tests for categorical data from a stratified cluster randomized trial.

    abstract::Two features commonly exhibited by randomized trials of health promotion interventions are cluster randomization and stratification. Ignoring correlations between individuals within clusters can lead to an inflated type I error rate and hence a P-value which overstates the significance of the result. This paper compar...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1256

    authors: Dobbins TA,Simpson JM

    更新日期:2002-12-30 00:00:00

  • Improved tests for a random effects meta-regression with a single covariate.

    abstract::The explanation of heterogeneity plays an important role in meta-analysis. The random effects meta-regression model allows the inclusion of trial-specific covariates which may explain a part of the heterogeneity. We examine the commonly used tests on the parameters in the random effects meta-regression with one covari...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1482

    authors: Knapp G,Hartung J

    更新日期:2003-09-15 00:00:00