Bioequivalence revisited.

abstract：:The case-control study is a simple and an useful method to characterize the effect of a gene, the effect of an exposure, as well as the interaction between the two. The control-free case-only study is yet an even simpler design, if interest is centered on gene-environment interaction only. It requires the sometimes pl...

journal_title：Statistics in medicine

authors： Lee WC,Wang LY,Cheng KF

更新日期：2010-10-30 00:00:00

abstract：:The objective of a phase II cancer clinical trial is to screen a treatment that can produce a similar or better response rate compared to the current treatment results. This screening is usually carried out in two stages as proposed by Simon. For ineffective treatment, the trial should terminate at the first stage. En...

journal_title：Statistics in medicine

authors： Chen TT

更新日期：1997-12-15 00:00:00

abstract：:Surrogate endpoint validation has been well established by the meta-analytical correlation-based approach as outlined in the seminal work of Buyse et al. (Biostatistics, 2000). Surrogacy can be assumed if strong associations on individual and study levels can be demonstrated. Alternatively, if an effect on a true endp...

journal_title：Statistics in medicine

authors： Schürmann C,Sieben W

更新日期：2016-03-30 00:00:00

abstract：:Sample size planning should reflect the primary objective of a trial. If the primary objective is prediction, the sample size determination should focus on prediction accuracy instead of power. We present formulas for the determination of training set sample size for survival prediction. Sample size is chosen to contr...

journal_title：Statistics in medicine

authors： Götte H,Zwiener I

更新日期：2013-02-28 00:00:00

abstract：:In recent years randomized trials designed to establish non-inferiority of a new treatment as compared to a standard one have been more widely used. Two-sample statistics have been proposed for this equivalence testing problem. However, they are not suited to situations where a long-term survivor fraction is expected....

journal_title：Statistics in medicine

authors： Broët P,Tubert-Bitter P,De Rycke Y,Moreau T

更新日期：2003-03-30 00:00:00

abstract：:In this work, we describe a two-stage sampling design to estimate the infection prevalence in a population. In the first stage, an imperfect diagnostic test was performed on a random sample of the population. In the second stage, a different imperfect test was performed in a stratified random sample of the first sampl...

journal_title：Statistics in medicine

authors： Miller E,Huppert A,Novikov I,Warburg A,Hailu A,Abbasi I,Freedman LS

更新日期：2015-11-10 00:00:00

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

authors： Jiang Y,Scott A,Wild CJ

更新日期：2009-01-30 00:00:00

abstract：:We discuss a sample size calculation for a pair-matched case-control study to test for interaction between a specific exposure and a second risk factor. The second risk factor could be either binary or continuous. An algorithm for the calculation of sample size is suggested which is based on a logistic regression mode...

journal_title：Statistics in medicine

authors： Qiu P,Moeschberger ML,Cooke GE,Goldschmidt-Clermont PJ

更新日期：2000-04-15 00:00:00

abstract：:In this paper, we investigate the impact of time-invariant covariates when fitting transition mixed models. This is carried out by emphasizing on the role of baseline responses on the estimation process. Transition models are allowed for two cases of exogenous and endogenous baseline responses. We illustrate these con...

journal_title：Statistics in medicine

authors： Rikhtehgaran R,Kazemi I,Verbeke G

更新日期：2014-11-30 00:00:00

abstract：:Instrumental variable is an essential tool for addressing unmeasured confounding in observational studies. Two-stage predictor substitution (2SPS) estimator and two-stage residual inclusion (2SRI) are two commonly used approaches in applying instrumental variables. Recently, 2SPS was studied under the additive hazards...

journal_title：Statistics in medicine

authors： Ying A,Xu R,Murphy J

更新日期：2019-05-10 00:00:00

abstract：:We propose a new design for dose finding for cytotoxic agents in two ordered groups of patients. By ordered groups, we mean that prior to the study there is clinical information that would indicate that for a given dose one group would be more susceptible to toxicities than patients in the other group. The designs are...

journal_title：Statistics in medicine

authors： Conaway MR,Wages NA

更新日期：2017-01-30 00:00:00

abstract：:For patients who were previously treated for prostate cancer, salvage hormone therapy is frequently given when the longitudinal marker prostate-specific antigen begins to rise during follow-up. Because the treatment is given by indication, estimating the effect of the hormone therapy is challenging. In a previous pape...

journal_title：Statistics in medicine

authors： Taylor JM,Shen J,Kennedy EH,Wang L,Schaubel DE

更新日期：2014-01-30 00:00:00

abstract：:A method is presented which allows us to adapt the sample size as well as the number and time points of interim analyses to the treatment difference observed at an interim look during the course of a clinical trial with censored survival time as the endpoint. The method allows the inclusion of data inspections during ...

journal_title：Statistics in medicine

authors： Schäfer H,Müller HH

更新日期：2001-12-30 00:00:00

abstract：:Genome-wide association studies are characterized by a huge number of statistical tests performed to discover new disease-related genetic variants [in the form of single-nucleotide polymorphisms (SNPs)] in human DNA. Many SNPs have been identified for cross-sectionally measured phenotypes. However, there is a growing ...

journal_title：Statistics in medicine

authors： Sikorska K,Rivadeneira F,Groenen PJ,Hofman A,Uitterlinden AG,Eilers PH,Lesaffre E

更新日期：2013-01-15 00:00:00

abstract：:Comparative effectiveness research represents the kind of research that arguably more directly affects clinical practice and policy. It includes observational studies, clinical trials, and systematic syntheses of existing literature. In this commentary, I argue for the ongoing and critical role of randomization in com...

journal_title：Statistics in medicine

authors： Lauer MS

更新日期：2012-11-10 00:00:00

abstract：:The matched case-control designs are commonly used to control for potential confounding factors in genetic epidemiology studies especially epigenetic studies with DNA methylation. Compared with unmatched case-control studies with high-dimensional genomic or epigenetic data, there have been few variable selection metho...

journal_title：Statistics in medicine

authors： Sun H,Wang S

更新日期：2013-05-30 00:00:00

abstract：:Prognostic models are used in medicine for investigating patient outcome in relation to patient and disease characteristics. Such models do not always work well in practice, so it is widely recommended that they need to be validated. The idea of validating a prognostic model is generally taken to mean establishing tha...

journal_title：Statistics in medicine

authors： Altman DG,Royston P

更新日期：2000-02-29 00:00:00

abstract：:Many complex diseases are known to be affected by the interactions between genetic variants and environmental exposures beyond the main genetic and environmental effects. Study of gene-environment (G×E) interactions is important for elucidating the disease etiology. Existing Bayesian methods for G×E interaction studie...

journal_title：Statistics in medicine

authors： Ren J,Zhou F,Li X,Chen Q,Zhang H,Ma S,Jiang Y,Wu C

更新日期：2020-02-28 00:00:00

abstract：:In the pharmaceutical industry, some tests for quality assurance before, during and after the manufacture of a drug product involve a two-stage sampling technique. We propose statistical testing procedures in a two-stage sampling problem with large within-class sample sizes. We also derive a two-stage sampling plan by...

journal_title：Statistics in medicine

authors： Shao J,Chow SC

更新日期：1993-11-15 00:00:00

abstract：:Methodology for the meta-analysis of individual patient data with survival end-points is proposed. Motivated by questions about the reliance on hazard ratios as summary measures of treatment effects, a parametric approach is considered and percentile ratios are introduced as an alternative to hazard ratios. The genera...

journal_title：Statistics in medicine

authors： Siannis F,Barrett JK,Farewell VT,Tierney JF

更新日期：2010-12-20 00:00:00

abstract：:Correlated data are obtained in longitudinal epidemiological studies, where repeated measurements are taken on individuals or groups over time. Such longitudinal data are ideally analyzed using multilevel modeling approaches, which appropriately account for the correlations in repeated responses in the same individual...

journal_title：Statistics in medicine

authors： Xanthakis V,Sullivan LM,Vasan RS

更新日期：2013-12-10 00:00:00

abstract：:In this paper we give an informal introduction to a robust method for survival analysis which is based on a modification of the usual partial likelihood estimator (PLE). Large sample results lead us to expect reduced bias for this robust estimator compared with the PLE whenever there are even slight violations of the ...

journal_title：Statistics in medicine

authors： Minder CE,Bednarski T

更新日期：1996-05-30 00:00:00

abstract：:In the recent two decades, data mining methods for signal detection have been developed for drug safety surveillance, using large post-market safety data. Several of these methods assume that the number of reports for each drug-adverse event combination is a Poisson random variable with mean proportional to the unknow...

journal_title：Statistics in medicine

authors： Hu N,Huang L,Tiwari RC

更新日期：2015-08-30 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：:Many observational studies adopt what we call retrospective convenience sampling (RCS). With the sample size in each arm prespecified, RCS randomly selects subjects from the treatment-inclined subpopulation into the treatment arm and those from the control-inclined into the control arm. Samples in each arm are represe...

journal_title：Statistics in medicine

authors： Hu Z,Qin J

更新日期：2018-05-20 00:00:00

abstract：:Confounding is a major concern in observational studies. To adjust for confounding bias, the potential confounder(s) for a study must first be identified and measured. But this is not always possible. The unmeasured factors may also exhibit effect modification, and this further complicates the situation. In this paper...

journal_title：Statistics in medicine

authors： Lee WC

更新日期：2011-04-30 00:00:00

abstract：:The design of clinical trials often requires knowledge of quantities such as between- and within subject variances about which only imprecise information exists. To provide assurance that the study has the desired power to detect a minimum clinically meaningful difference between treatment groups. Gould, Gould and Shi...

journal_title：Statistics in medicine

authors： Shih WJ,Gould AL

更新日期：1995-10-30 00:00:00

abstract：:We develop reinforcement learning trials for discovering individualized treatment regimens for life-threatening diseases such as cancer. A temporal-difference learning method called Q-learning is utilized that involves learning an optimal policy from a single training set of finite longitudinal patient trajectories. A...

journal_title：Statistics in medicine

authors： Zhao Y,Kosorok MR,Zeng D

更新日期：2009-11-20 00:00:00

abstract：:In a phase I clinical trial in cancer patients, the drug involved had one known main adverse effect, which also occurs spontaneously in cancer patients with a fairly high frequency. Experiments in rats have shown marked effects of the drug on tumour growth in high doses, but also dose-dependent toxicity. Consequently,...

journal_title：Statistics in medicine

authors： Møller S

更新日期：1995-05-15 00:00:00

abstract：:When interpreting screening mammograms radiologists decide whether suspicious abnormalities exist that warrant the recall of the patient for further testing. Previous work has found significant differences in interpretation among radiologists; their false-positive and false-negative rates have been shown to vary widel...

journal_title：Statistics in medicine

authors： Woodard DB,Gelfand AE,Barlow WE,Elmore JG

更新日期：2007-03-30 00:00:00