Policy developments in regulatory approval.

abstract：:The first randomized clinical trial at the National Cancer Institute (NCI), planned in 1954, commenced in 1955 for the treatment of patients with acute leukaemia. The programme in clinical trials at NCI had strong influence from the clinician and administrator, C. Gordon Zubrod, who introduced the randomized clinical ...

journal_title：Statistics in medicine

authors： Gehan EA,Schneiderman MA

更新日期：1990-08-01 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：:The weighted average treatment effect is a causal measure for the comparison of interventions in a specific target population, which may be different from the population where data are sampled from. For instance, when the goal is to introduce a new treatment to a target population, the question is what efficacy (or ef...

journal_title：Statistics in medicine

authors： Tao Y,Fu H

更新日期：2019-02-10 00:00:00

abstract：BACKGROUND:It has been recommended that onset of antidepressant action be assessed using survival analyses with assessments taken at least twice per week. However, such an assessment schedule is problematic to implement. The present study assessed the feasibility of comparing onset of action between treatments using a ...

journal_title：Statistics in medicine

authors： Mallinckrodt CH,Detke MJ,Kaiser CJ,Watkin JG,Molenberghs G,Carroll RJ

更新日期：2006-07-30 00:00:00

abstract：:In the comparison of two or more treatment groups to a control group, consider a study with non-decreasing repeated measurements of the same characteristic taken over a common set of time points for each subject. Based on the vector of possibly incomplete responses from each subject, this paper considers asymptoticall...

journal_title：Statistics in medicine

authors： Davis CS

更新日期：1996-12-15 00:00:00

abstract：:In drug-drug interaction (DDI) research, a two drug interaction is usually predicted by individual drug pharmacokinetics (PK). Although subject-specific drug concentration data from clinical PK studies on inhibitor/inducer or substrate's PK are not usually published, sample mean plasma drug concentrations and their st...

journal_title：Statistics in medicine

authors： Li L,Yu M,Chin R,Lucksiri A,Flockhart DA,Hall SD

更新日期：2007-09-10 00:00:00

abstract：:In observational studies with censored data, exposure-outcome associations are commonly measured with adjusted hazard ratios from multivariable Cox proportional hazards models. The difference in restricted mean survival times (RMSTs) up to a pre-specified time point is an alternative measure that offers a clinically m...

journal_title：Statistics in medicine

authors： Conner SC,Sullivan LM,Benjamin EJ,LaValley MP,Galea S,Trinquart L

更新日期：2019-09-10 00:00:00

abstract：:The concept of broad sense agreement (BSA) has recently been proposed for studying the relationship between a continuous measurement and an ordinal measurement. They developed a nonparametric procedure for estimating the BSA index, which is only applicable to completely observed data. In this work, we consider the pro...

journal_title：Statistics in medicine

authors： Dai T,Guo Y,Peng L,Manatunga A

更新日期：2020-06-30 00:00:00

abstract：:Orthogonal polynomial scores (OPS) is a simple, biologically meaningful approach to characterize longitudinal data in phase I and II clinical pharmacology trials. It describes average, linear, quadratic and higher order polynomial characteristics of each subject's response over time with use of composite scores comput...

journal_title：Statistics in medicine

authors： Bradstreet TE

更新日期：1993-04-15 00:00:00

abstract：:Reference ranges which take time (such as age) into account are often required in medicine, but simple, systematic and efficient statistical methods for constructing them are lacking. A method is described which is based on low order polynomial curves (linear, quadratic or occasionally cubic), together with guidelines...

journal_title：Statistics in medicine

authors： Royston P

更新日期：1991-05-01 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：:Intraclass correlation coefficient (ICC) estimates must be provided when reporting the results of a cluster randomized trial. This study demonstrates that estimating this parameter with one-way ANOVA and an underlying mixed-effects statistical model leads to biased estimates. The bias depends on the effect size of the...

journal_title：Statistics in medicine

authors： Giraudeau B

更新日期：2006-03-30 00:00:00

abstract：:Observational studies provide a rich source of information for assessing effectiveness of treatment interventions in many situations where it is not ethical or practical to perform randomized controlled trials. However, such studies are prone to bias from hidden (unmeasured) confounding. A promising approach to identi...

journal_title：Statistics in medicine

authors： Lin NX,Henley WE

更新日期：2016-12-10 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：:In England and Wales, a large-scale multiple statistical surveillance system for infectious disease outbreaks has been in operation for nearly two decades. This system uses a robust quasi-Poisson regression algorithm to identify abberrances in weekly counts of isolates reported to the Health Protection Agency. In this...

journal_title：Statistics in medicine

authors： Noufaily A,Enki DG,Farrington P,Garthwaite P,Andrews N,Charlett A

更新日期：2013-03-30 00:00:00

abstract：:There is a single-minded focus on events in survival analysis, and we often ignore longitudinal data that are collected together with the event data. This is due to a lack of methodology but also a result of the artificial distinction between survival and longitudinal data analyses. Understanding the dynamics of such ...

journal_title：Statistics in medicine

authors： Aalen OO

更新日期：2012-08-15 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 investigates the design and analysis of dose-finding trials with two agents. The set of doses for each agent is fixed in advance. The goal of the trial is to find the set of dose combinations with probability of toxicity closest to a pre-specified value. For each of the two agents we assume that the probabi...

journal_title：Statistics in medicine

authors： Ivanova A,Wang K

更新日期：2004-06-30 00:00:00

abstract：:A Non-Parametric Maximum Likelihood approach to the estimation of relative risks in the context of disease mapping is discussed and a NPML approximation to conditional autoregressive models is proposed. NPML estimates have been compared to other proposed solutions (Maximum Likelihood via Monte Carlo Scoring, Hierarchi...

journal_title：Statistics in medicine

authors： Biggeri A,Marchi M,Lagazio C,Martuzzi M,Böhning D

更新日期：2000-09-15 00:00:00

abstract：:Missing values in the covariates are a widespread complication in the statistical inference of regression models. The maximum likelihood principle requires specification of the distribution of the covariates, at least in part. For categorical covariates, log-linear models can be used. Additionally, the missing at rand...

journal_title：Statistics in medicine

authors： Vach W,Blettner M

更新日期：1995-06-30 00:00:00

abstract：:We analyze data obtained from a study designed to evaluate training effects on the performance of certain motor activities of Parkinson's disease patients. Maximum likelihood methods were used to fit beta-binomial/Poisson regression models tailored to evaluate the effects of training on the numbers of attempted and su...

journal_title：Statistics in medicine

authors： Lora MI,Singer JM

更新日期：2008-07-30 00:00:00

abstract：:The proposed guidelines for the assessment of the effect of new pharmaceutical agents on the QT interval (beginning of QRS complex to end of T wave on the electrocardiogram) are based on the maximum of a series over time of simple one-sided 95 per cent upper confidence bounds. This procedure is typically very conserva...

journal_title：Statistics in medicine

authors： Boos DD,Hoffman D,Kringle R,Zhang J

更新日期：2007-09-10 00:00:00

abstract：:This paper develops methods of analysis for active extension clinical trials. Under this design, patients are randomized to treatment or placebo for a period of time (period 1), and then all patients receive treatment for an additional period of time (period 2). We assume a continuous outcome is measured at baseline a...

journal_title：Statistics in medicine

authors： Hu Z,Follmann D

更新日期：2007-05-30 00:00:00

abstract：:We consider the estimation of parameters in a particular segmented generalized linear model with additive measurement error in predictors, with a focus on linear and logistic regression. In epidemiologic studies segmented regression models often occur as threshold models, where it is assumed that the exposure has no i...

journal_title：Statistics in medicine

authors： Küchenhoff H,Carroll RJ

更新日期：1997-01-15 00:00:00

abstract：:Biomarkers, increasingly used in biomedical studies for the diagnosis and prognosis of acute and chronic diseases, provide insight into the effectiveness of treatments and potential pathways that can be used to guide future treatment targets. The measurement of these markers is often limited by the sensitivity of the ...

journal_title：Statistics in medicine

authors： Lee M,Kong L,Weissfeld L

更新日期：2012-07-30 00:00:00

abstract：:Log-linear models for capture-recapture type data are widely used for estimating sizes of populations. Log-linear methods model conditional interactions between the sources. Often, however, the marginal associations are more appropriate and easier for the practitioner to conceptualize. Analyses here of previously publ...

journal_title：Statistics in medicine

authors： Regal RR,Hook EB

更新日期：1998-01-15 00:00:00

abstract：:Zero-inflated Poisson regression is a popular tool used to analyze data with excessive zeros. Although much work has already been performed to fit zero-inflated data, most models heavily depend on special features of the individual data. To be specific, this means that there is a sizable group of respondents who endor...

journal_title：Statistics in medicine

authors： Lin TH,Tsai MH

更新日期：2013-04-30 00:00:00

abstract：:We present a new approach to training back-propagation artificial neural nets (BP-ANN) based on regularization and cross-validation and on initialization by a logistic regression (LR) model. The new approach is expected to produce a BP-ANN predictor at least as good as the LR-based one. We have applied the approach to...

journal_title：Statistics in medicine

authors： Ciampi A,Zhang F

更新日期：2002-05-15 00:00:00

abstract：:Crossover studies have been successfully conducted in the case of continuous responses. Existing procedures of analysis for ordinal responses, on the other hand, are rarely satisfactory unless strict, usually unrealistic, assumptions are made. In this paper we investigate a random effects model and show that the model...

journal_title：Statistics in medicine

authors： Ezzet F,Whitehead J

更新日期：1991-06-01 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