Small clinical trials: are they all bad?

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：:The intraclass correlation coefficient rho plays a key role in the design of cluster randomized trials. Estimates of rho obtained from previous cluster trials and used to inform sample size calculation in planned trials may be imprecise due to the typically small numbers of clusters in such studies. It may be useful t...

journal_title：Statistics in medicine

authors： Ukoumunne OC,Davison AC,Gulliford MC,Chinn S

更新日期：2003-12-30 00:00:00

abstract：:We describe the usual statistical concepts and consequent appropriate simulations of a prospective study for the simple case of a single risk variable and an assumed logistic model. We examine the simulations of Lilienfeld and Pyne, and show that they are seriously flawed. Contrary to those authors' claims, the estima...

journal_title：Statistics in medicine

authors： Halperin M,Abbott RD,Blackwelder WC,Jacobowitz R,Lan KK,Verter JI,Wedel H

更新日期：1985-04-01 00:00:00

abstract：:A good measure of field vaccine efficacy should evaluate the direct protective effect of vaccination on the person who receives the vaccine. The conventional estimator for vaccine efficacy depends on population level factors that are either unrelated or indirectly related to the direct biological action of the vaccine...

journal_title：Statistics in medicine

authors： Longini IM Jr,Halloran ME,Haber M,Chen RT

更新日期：1993-02-01 00:00:00

abstract：:Clinical trials studying treatments for rare diseases are challenging to design and conduct due to the limited number of patients eligible for the trial. One design used to address this challenge is the small n, sequential, multiple assignment, randomized trial (snSMART). We propose a new snSMART design that investiga...

journal_title：Statistics in medicine

authors： Fang F,Hochstedler KA,Tamura RN,Braun TM,Kidwell KM

更新日期：2021-02-20 00:00:00

abstract：:We propose a semiparametric method for estimating ROC surfaces for continuous diagnostic tests based on two test measurements. Such a three-class diagnostic problem based on two test measurements arises naturally from some DNA amplification-related diagnostic scenarios. Simulation results show that our proposed semipa...

journal_title：Statistics in medicine

authors： Wan S,Zhang B

更新日期：2009-08-15 00:00:00

abstract：:We propose a two-step procedure to personalize drug dosage over time under the framework of a log-linear mixed-effect model. We model patients' heterogeneity using subject-specific random effects, which are treated as the realizations of an unspecified stochastic process. We extend the conditional quadratic inference ...

journal_title：Statistics in medicine

authors： Zhu X,Qu A

更新日期：2016-10-30 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：:With the increasing availability of detailed clinical information, there is optimism that treatment choices can be selectively directed to those individuals most likely to benefit. While standard clinical trials can establish whether a treatment appears to be effective on average, subsequent work is needed to determin...

journal_title：Statistics in medicine

authors： Sitlani CM,Heagerty PJ

更新日期：2014-07-30 00:00:00

abstract：:Identification of key factors associated with the risk of developing cardiovascular disease and quantification of this risk using multivariable prediction algorithms are among the major advances made in preventive cardiology and cardiovascular epidemiology in the 20th century. The ongoing discovery of new risk markers...

journal_title：Statistics in medicine

authors： Pencina MJ,D'Agostino RB Sr,D'Agostino RB Jr,Vasan RS

更新日期：2008-01-30 00:00:00

abstract：:Frailty models are encountered in many medical applications, yet little research has been devoted to develop measures that quantify the predictive ability of these models. In this paper, we elaborate on the concept of the concordance probability to clustered data, resulting in an 'Overall Conditional C-index' or bfC(O...

journal_title：Statistics in medicine

authors： Van Oirbeek R,Lesaffre E

更新日期：2010-12-30 00:00:00

abstract：:The FDA Guidance, while highly appropriate for industry sponsored trials, need not be imposed on publicly (e.g. NIH) financed clinical trials. While the potential for conflicts of interest exist in the latter, they are in general manageable and pose an acceptable low risk of threatening the integrity of a study. Howev...

journal_title：Statistics in medicine

authors： Lachin JM

更新日期：2004-05-30 00:00:00

abstract：:There is considerable public concern about health disparities among different cultural/racial/ethnic groups. Important process measures that might reflect inequities are treated prevalence and the service utilization rate in a defined period of time. We have previously described a method for estimating N, the distinct...

journal_title：Statistics in medicine

authors： Laska EM,Meisner M,Wanderling J,Siegel C

更新日期：2010-07-20 00:00:00

abstract：:We consider methods for selecting the joint specification of the mean and variance functions in statistical models for rates or counts. Based on analyses of diagnosis-specific hospital discharge rates in Michigan, we show that a Poisson model with an extra variance component for the systematic variation is superior to...

journal_title：Statistics in medicine

authors： Wolfe RA,Petroni GR,McLaughlin CG,McMahon LF Jr

更新日期：1991-09-01 00:00:00

abstract：:A considerable number of the clinical reports which are presented to the Dutch Board for the Evaluation of Drugs, have deficiencies and/or shortcomings. A number of these, including loose description of the target population and sampling method, methodological flaws, incorrect treatment of withdrawals, confounding of ...

journal_title：Statistics in medicine

authors： De Jonge H

更新日期：1983-04-01 00:00:00

abstract：:This paper describes the Stoke Index which has been designed to give a global measure of disease activity in rheumatoid arthritis. The index is based on two objective laboratory measurements, one subjective and two semi-objective clinical measurements, chosen from 13 measurements using clinical judgement. Variable sel...

journal_title：Statistics in medicine

authors： Jones PW,Ziade MF,Davis MJ,Dawes PT

更新日期：1993-06-30 00:00:00

abstract：:The present study investigates the performance of several statistical tests to detect publication bias in diagnostic meta-analysis by means of simulation. While bivariate models should be used to pool data from primary studies in diagnostic meta-analysis, univariate measures of diagnostic accuracy are preferable for t...

journal_title：Statistics in medicine

authors： Bürkner PC,Doebler P

更新日期：2014-08-15 00:00:00

abstract：:We develop parametric maximum likelihood methods to adjust for treatment changes during follow-up in order to assess the causal effect of treatment in clinical trials with time-to-event outcomes. The accelerated failure time model of Robins and Tsiatis relates each observed event time to the underlying event time that...

journal_title：Statistics in medicine

authors： Walker AS,White IR,Babiker AG

更新日期：2004-02-28 00:00:00

abstract：:Meta-analysis has generally been accepted as a fundamental tool for combining effect estimates from several studies. For binary studies with rare events, the Peto odds ratio (POR) method has become the relative effect estimator of choice. However, the POR leads to biased estimates for the OR when treatment effects are...

journal_title：Statistics in medicine

authors： Brockhaus AC,Bender R,Skipka G

更新日期：2014-12-10 00:00:00

abstract：:Using both simulated and real datasets, we compared two approaches for estimating absolute risk from nested case-control (NCC) data and demonstrated the feasibility of using the NCC design for estimating absolute risk. In contrast to previously published results, we successfully demonstrated not only that data from a ...

journal_title：Statistics in medicine

authors： Salim A,Delcoigne B,Villaflores K,Koh WP,Yuan JM,van Dam RM,Reilly M

更新日期：2017-02-10 00:00:00

abstract：:Graphical methods based on the analysis of differences between log cumulative hazard functions are considered for a two-group semi-proportional hazard model which allows for interaction between treatments and covariates. Confidence procedures and test statistics that can be used to test for interaction and for main ef...

journal_title：Statistics in medicine

authors： Dabrowska DM,Doksum KA,Feduska NJ,Husing R,Neville P

更新日期：1992-08-01 00:00:00

abstract：:In many epidemiological studies it is common to resort to regression models relating incidence of a disease and its risk factors. The main goal of this paper is to consider inference on such models with error-prone observations and variances of the measurement errors changing across observations. We suppose that the o...

journal_title：Statistics in medicine

authors： de Castro M,Galea M,Bolfarine H

更新日期：2008-11-10 00:00:00

abstract：:The original article to which this Correction refers was published in Statistics in Medicine 2000 19(14): 1901-1914. ...

journal_title：Statistics in medicine

authors： Edwardes MD

更新日期：2000-11-15 00:00:00

abstract：:Inference for randomized clinical trials is generally based on the assumption that outcomes are independently and identically distributed under the null hypothesis. In some trials, particularly in infectious disease, outcomes may be correlated. This may be known in advance (e.g. allowing randomization of family member...

journal_title：Statistics in medicine

authors： Proschan M,Follmann D

更新日期：2008-03-15 00:00:00

abstract：:In genetic association studies, mixed effects models have been widely used in detecting the pleiotropy effects which occur when one gene affects multiple phenotype traits. In particular, bivariate mixed effects models are useful for describing the association of a gene with a continuous trait and a binary trait. Howev...

journal_title：Statistics in medicine

authors： Zhang Q,Yi GY

更新日期：2020-11-20 00:00:00

abstract：:In the analysis of composite endpoints in a clinical trial, time to first event analysis techniques such as the logrank test and Cox proportional hazard test do not take into account the multiplicity, importance, and the severity of events in the composite endpoint. Several generalized pairwise comparison analysis met...

journal_title：Statistics in medicine

authors： Verbeeck J,Spitzer E,de Vries T,van Es GA,Anderson WN,Van Mieghem NM,Leon MB,Molenberghs G,Tijssen J

更新日期：2019-12-30 00:00:00

abstract：:Standard approaches to analysis of randomized controlled trials (RCTs) using Markov models make it difficult to generalize treatment effects to new patient groups and synthesize evidence across trials. This paper demonstrates how pair-wise and mixed treatment comparison meta-analysis can be applied to event history da...

journal_title：Statistics in medicine

authors： Price MJ,Welton NJ,Ades AE

更新日期：2011-01-30 00:00:00

abstract：:Three-arm trials including the experimental treatment, an active reference treatment and a placebo are recommended in the guidelines of the ICH and EMEA/CPMP as a useful approach to the assessment of assay sensitivity. Generally, the acceptable non-inferiority margin Δ has been defined as the maximum clinically irrele...

journal_title：Statistics in medicine

authors： Hida E,Tango T

更新日期：2011-02-10 00:00:00

abstract：:The continual reassessment method (CRM) is an adaptive design for Phase I trials whose operating characteristics, including appropriate sample size, probability of correctly identifying the maximum tolerated dose, and the expected proportion of participants assigned to each dose, can only be determined via simulation....

journal_title：Statistics in medicine

authors： Braun TM

更新日期：2020-09-16 00:00:00

abstract：:We present an approach for inference on contrasts of clinically meaningful functionals of a survivor distribution (e.g., restricted mean, quantiles) that can avoid strong parametric or semiparametric assumptions on the underlying structure of the data. In this multistage approach, we first use an adaptive predictive m...

journal_title：Statistics in medicine

authors： Rudser KD,LeBlanc ML,Emerson SS

更新日期：2012-07-20 00:00:00