Seamless phase 2/3 oncology trial design with flexible sample size determination.

abstract：:Conditional power and predictive power provide estimates of the probability of success at the end of the trial based on the information from the interim analysis. The observed value of the time to event endpoint at the interim analysis could be biased for the true treatment effect due to early censoring, leading to a ...

journal_title：Statistics in medicine

authors： Sun L,Wan Y

更新日期：2018-08-15 00:00:00

abstract：:With reference to a real data on cataract surgery, we discuss the problem of zero-inflated circular-circular regression when both covariate and response are circular random variables and a large proportion of the responses are zeros. The regression model is proposed, and the estimation procedure for the parameters is ...

journal_title：Statistics in medicine

authors： Jha J,Biswas A

更新日期：2018-01-15 00:00:00

abstract：:The correct identification of change-points during ongoing outbreak investigations of infectious diseases is a matter of paramount importance in epidemiology, with major implications for the management of health care resources, public health and, as the COVID-19 pandemic has shown, social live. Onsets, peaks, and infl...

journal_title：Statistics in medicine

authors： Veres-Ferrer EJ,Pavía JM

更新日期：2021-02-20 00:00:00

abstract：:In the last decade or so, pharmaceutical drug development activities in the area of new antibacterial drugs for treating serious bacterial diseases have declined, and at the same time, there are worries that the increased prevalence of antibiotic-resistant bacterial infections, especially the increase in drug-resistan...

journal_title：Statistics in medicine

authors： Huque MF,Valappil T,Soon GG

更新日期：2014-11-10 00:00:00

abstract：:Sequential analysis is frequently employed to address ethical and financial issues in clinical trials. Sequential analysis may be performed using standard group sequential designs, or, more recently, with adaptive designs that use estimates of treatment effect to modify the maximal statistical information to be collec...

journal_title：Statistics in medicine

authors： Emerson SC,Rudser KD,Emerson SS

更新日期：2011-05-20 00:00:00

abstract：:R2 has been criticized as a measure of model performance when predicting a dichotomous outcome, both because its value is often low and because it is sensitive to the prevalence of the event of interest. The C statistic is more widely used to measure model performance in a 0/1 setting. We use a simple parametric famil...

journal_title：Statistics in medicine

authors： Ash A,Shwartz M

更新日期：1999-02-28 00:00:00

abstract：:This paper discusses the benefits and limitations of adaptive sample size re-estimation for phase 3 confirmatory clinical trials. Comparisons are made with more traditional fixed sample and group sequential designs. It is seen that the real benefit of the adaptive approach arises through the ability to invest sample s...

journal_title：Statistics in medicine

authors： Mehta CR,Pocock SJ

更新日期：2011-12-10 00:00:00

abstract：:Many studies have reported large variations in age- and sex-adjusted rates of hospitalizations across small geographic areas. These variations have often been attributed to differences in medical practice style which are not reflected in differences in health care outcomes. There is, however, another potentially impor...

journal_title：Statistics in medicine

authors： Peköz EA,Shwartz M,Iezzoni LI,Ash AS,Posner MA,Restuccia JD

更新日期：2003-05-30 00:00:00

abstract：:In phase 3 clinical trials, ethical and financial concerns motivate sequential analyses in which the data are analyzed prior to completion of the entire planned study. Existing group sequential software accounts for the effects of these interim analyses on the sampling density by assuming that the contribution of subs...

journal_title：Statistics in medicine

authors： Shoben AB,Emerson SS

更新日期：2014-12-20 00:00:00

abstract：:Longitudinal designs are important in medical research and in many other disciplines. Complete longitudinal studies, in which each subject is evaluated at each measurement occasion, are often very expensive and motivate a search for more efficient designs. Recently developed statistical methods foster the use of inten...

journal_title：Statistics in medicine

authors： Helms RW

更新日期：1992-10-01 00:00:00

abstract：:Assay sensitivity has been proposed as a criterion for including psychiatric clinical outcome studies in meta-analyses. The authors assess the performance of assay sensitivity as a method for determining study appropriateness for meta-analysis by calculating expected standard drug vs placebo effect sizes for various c...

journal_title：Statistics in medicine

authors： Gelfand LA,Strunk DR,Tu XM,Noble RE,Derubeis RJ

更新日期：2006-03-30 00:00:00

abstract：:The goal of screening programmes for cancer is early detection and treatment with a consequent reduction in mortality from the disease. Screening programmes need to assess the true benefit of screening, that is, the length of time of extension of survival beyond the time of advancement of diagnosis (lead-time). This p...

journal_title：Statistics in medicine

authors： Xu JL,Prorok PC

更新日期：1995-12-30 00:00:00

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...

journal_title：Statistics in medicine

authors： Yao Q,Wei LJ

更新日期：1996-11-15 00:00:00

abstract：:Competing compartment models of different complexities have been used for the quantitative analysis of dynamic contrast-enhanced magnetic resonance imaging data. We present a spatial elastic net approach that allows to estimate the number of compartments for each voxel such that the model complexity is not fixed a pri...

journal_title：Statistics in medicine

authors： Sommer JC,Gertheiss J,Schmid VJ

更新日期：2014-03-15 00:00:00

abstract：:Residuals in normal regression are used to assess a model's goodness-of-fit (GOF) and discover directions for improving the model. However, there is a lack of residuals with a characterized reference distribution for censored regression. In this article, we propose to diagnose censored regression with normalized rando...

journal_title：Statistics in medicine

authors： Li L,Wu T,Feng C

更新日期：2020-12-13 00:00:00

abstract：:Random forest is a supervised learning method that combines many classification or regression trees for prediction. Here we describe an extension of the random forest method for building event risk prediction models in survival analysis with competing risks. In case of right-censored data, the event status at the pred...

journal_title：Statistics in medicine

authors： Mogensen UB,Gerds TA

更新日期：2013-08-15 00:00:00

abstract：:We examine the common clinical belief that there is an interaction between epilepsy type and the two standard anti-epileptic drugs, valproate and carbamazepine, using data from several randomized clinical trials. Epilepsy type is not always easy to define, and three possible reclassifications are investigated to see w...

journal_title：Statistics in medicine

authors： Williamson PR,Clough HE,Hutton JL,Marson AG,Chadwick DW

更新日期：2002-09-30 00:00:00

abstract：:When analyzing time to disease recurrence, we sometimes need to work with data where all the recurrences are recorded, but no information is available on the possible deaths. This may occur when studying diseases of benign nature where patients are only seen at disease recurrences or in poorly-designed registries of b...

journal_title：Statistics in medicine

authors： Štupnik T,Pohar Perme M

更新日期：2016-03-30 00:00:00

abstract：:We study the competing risks model and show that the cause j cumulative incidence function integrated from 0 to τ has a natural interpretation as the expected number of life years lost due to cause j before time τ. This is analogous to the τ-restricted mean lifetime, which is the survival function integrated from 0 to...

journal_title：Statistics in medicine

authors： Andersen PK

更新日期：2013-12-30 00:00:00

abstract：:Systematic reviews often provide recommendations for further research. When meta-analyses are inconclusive, such recommendations typically argue for further studies to be conducted. However, the nature and amount of future research should depend on the nature and amount of the existing research. We propose a method ba...

journal_title：Statistics in medicine

authors： Roloff V,Higgins JP,Sutton AJ

更新日期：2013-01-15 00:00:00

abstract：:In survival analyses, inverse-probability-of-treatment (IPT) and inverse-probability-of-censoring (IPC) weighted estimators of parameters in marginal structural Cox models are often used to estimate treatment effects in the presence of time-dependent confounding and censoring. In most applications, a robust variance e...

journal_title：Statistics in medicine

authors： Enders D,Engel S,Linder R,Pigeot I

更新日期：2018-10-30 00:00:00

abstract：:Clinical trials with multiple endpoints incur increased familywise type I errors. The Bonferroni correction is a common method used to modify the p-values to account for multiple significance testing. For independent endpoints the Bonferroni method is slightly conservative whereas with high correlation the conservatis...

journal_title：Statistics in medicine

authors： James S

更新日期：1991-07-01 00:00:00

abstract：:Multilevel and longitudinal studies are frequently subject to missing data. For example, biomarker studies for oral cancer may involve multiple assays for each participant. Assays may fail, resulting in missing data values that can be assumed to be missing completely at random. Catellier and Muller proposed a data ana...

journal_title：Statistics in medicine

authors： Ringham BM,Kreidler SM,Muller KE,Glueck DH

更新日期：2016-07-30 00:00:00

abstract：:We present an estimate of the kappa-coefficient of agreement between two methods of rating based on matched pairs of binary responses and show that the estimate depends on the common intraclass correlation coefficient between the pairs. Via Monte Carlo simulation, we investigate power of the test of significance on ka...

journal_title：Statistics in medicine

authors： Shoukri MM,Martin SW,Mian IU

更新日期：1995-01-15 00:00:00

abstract：:Increasing numbers of economic evaluations are conducted alongside randomised controlled trials. Such studies include factorial trials, which randomise patients to different levels of two or more factors and can therefore evaluate the effect of multiple treatments alone and in combination. Factorial trials can provide...

journal_title：Statistics in medicine

authors： Dakin H,Gray A

更新日期：2017-08-15 00:00:00

abstract：:Assessment of equivalence or non-inferiority in accuracy between two diagnostic procedures often involves comparisons of paired areas under the receiver operating characteristic (ROC) curves. With some pre-specified clinically meaningful limits, the current approach to evaluating equivalence is to perform the two one-...

journal_title：Statistics in medicine

authors： Liu JP,Ma MC,Wu CY,Tai JY

更新日期：2006-04-15 00:00:00

abstract：:Conditional power (CP) is the probability that the final study result will be statistically significant, given the data observed thus far and a specific assumption about the pattern of the data to be observed in the remainder of the study, such as assuming the original design effect, or the effect estimated from the c...

journal_title：Statistics in medicine

authors： Lachin JM

更新日期：2005-09-30 00:00:00

abstract：:The generalized estimating equations (GEE) approach is commonly used to model incomplete longitudinal binary data. When drop-outs are missing at random through dependence on observed responses (MAR), GEE may give biased parameter estimates in the model for the marginal means. A weighted estimating equations approach g...

journal_title：Statistics in medicine

authors： Preisser JS,Lohman KK,Rathouz PJ

更新日期：2002-10-30 00:00:00

abstract：:Teaching statistics to medical students is a challenging and often unrewarding task. However, few would argue the need for statistics in the medical school curriculum. In recent years, there has been a growing call for teaching only statistical concepts in medical schools. We strongly oppose this opinion and offer an ...

journal_title：Statistics in medicine

authors： Herman A,Notzer N,Libman Z,Braunstein R,Steinberg DM

更新日期：2007-10-15 00:00:00

abstract：:Modelling disease clustering over space and time can be helpful in providing indications of possible exposures and planning corresponding public health practices. Though a considerable number of studies focus on modelling spatio-temporal patterns of disease, most of them do not directly model a spatio-temporal cluster...

journal_title：Statistics in medicine

authors： Yan P,Clayton MK

更新日期：2006-03-15 00:00:00