Current development in clinical trials: issues old and new.

abstract：:To study the spread of sexually transmitted diseases (STDs) using social/sexual mixing models, one must have quantitative information about sexual mixing. An unavoidable complication in gathering such information by survey is that members of the surveyed population will almost certainly have sexual contacts outside th...

journal_title：Statistics in medicine

authors： Rubin G,Umbach D,Shyu SF,Castillo-Chavez C

更新日期：1992-09-15 00:00:00

abstract：:Testing the equality of 2 proportions for a control group versus a treatment group is a well-researched statistical problem. In some settings, there may be strong historical data that allow one to reliably expect that the control proportion is one, or nearly so. While one-sample tests or comparisons to historical cont...

journal_title：Statistics in medicine

authors： Gabriel EE,Nason M,Fay MP,Follmann DA

更新日期：2018-03-30 00:00:00

abstract：:We present some practical extensions and applications of a strategy proposed by Thall, Simon and Estey for designing and monitoring single-arm clinical trials with multiple outcomes. We show by application how the strategy may be applied to construct designs for phase IIA activity trials and phase II equivalence trial...

journal_title：Statistics in medicine

authors： Thall PF,Sung HG

更新日期：1998-07-30 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：:Two important qualities of controlled clinical trials are that they reduce dependence on historical standards for evaluating therapy and separate the effect of treatment from the confounding influence of time. Whatever the theory of the clinical trial, however, time has not easily been banished from the analysis of me...

journal_title：Statistics in medicine

authors： Senn SJ

更新日期：1989-11-01 00:00:00

abstract：:We consider the problem of comparing cumulative incidence functions of non-mortality events in the presence of informative coarsening and the competing risk of death. We extend frequentist-based hypothesis tests previously developed for non-informative coarsening and propose a novel Bayesian method based on comparing ...

journal_title：Statistics in medicine

authors： Shardell M,Scharfstein DO,Vlahov D,Galai N

更新日期：2008-12-10 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：: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

authors： Senn S,Julious S

更新日期：2009-11-20 00:00:00

abstract：:For each of 211 arteriosclerosis obliterans patients, the degree of stenosis of arteries at four sites were examined at Hiroshima University Hospital to analyse the relationship between the degree of stenosis and age, sex and site. The generalized estimating equations using a proportional odds model for the stenosis p...

journal_title：Statistics in medicine

authors： Nakashima E,Tsuji S,Fukuoka H,Ohtaki M,Ito K

更新日期：2003-07-15 00:00:00

abstract：:The U.S. Food and Drug Administration (FDA) has proposed new regulations that address the 'prescribability' and 'switchability' of new formulations of already-approved drugs. These new criteria are known, respectively, as population and individual bioequivalence. Two methods have been proposed in the bioequivalence li...

journal_title：Statistics in medicine

authors： McNally RJ,Iyer H,Mathew T

更新日期：2003-01-15 00:00:00

abstract：:The identification of new adverse drug reactions is often tricky. For a given case, the relationship between drug exposure and symptom occurrence is usually questionable. It could be investigated statistically from a series of drug-event association cases with an independence test between the two variables. Analysing ...

journal_title：Statistics in medicine

authors： Tubert P,Begaud B

更新日期：1991-06-01 00:00:00

abstract：:The standard analysis of clinical trials stratified by centre is to include centres as fixed effects, but if many centres contribute small numbers of patients, this approach results in a loss of power. Assuming no treatment by centre interaction, we used simulation to examine power and coverage of confidence intervals...

journal_title：Statistics in medicine

authors： Pickering RM,Weatherall M

更新日期：2007-12-30 00:00:00

abstract：:Parameters for latent transition analysis (LTA) are easily estimated by maximum likelihood (ML) or Bayesian method via Markov chain Monte Carlo (MCMC). However, unusual features in the likelihood can cause difficulties in ML and Bayesian inference and estimation, especially with small samples. In this study we explore...

journal_title：Statistics in medicine

authors： Chung H,Lanza ST,Loken E

更新日期：2008-05-20 00:00:00

abstract：:When estimating the probability of natural conception from observational data on couples with an unfulfilled child wish, the start of assisted reproductive therapy (ART) is a competing event that cannot be assumed to be independent of natural conception. In clinical practice, interest lies in the probability of natura...

journal_title：Statistics in medicine

authors： van Geloven N,Geskus RB,Mol BW,Zwinderman AH

更新日期：2014-11-20 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：:Identification of the latency period for the effect of a time-varying exposure is key when assessing many environmental, nutritional, and behavioral risk factors. A pre-specified exposure metric involving an unknown latency parameter is often used in the statistical model for the exposure-disease relationship. Likelih...

journal_title：Statistics in medicine

authors： Peskoe SB,Spiegelman D,Wang M

更新日期：2019-03-30 00:00:00

abstract：:Assessing the QT prolongation potential of a drug is typically done based on pivotal safety studies called thorough QT studies. Model-based estimation of the drug-induced QT prolongation at the estimated mean maximum drug concentration could increase efficiency over the currently used intersection-union test. However,...

journal_title：Statistics in medicine

authors： Dosne AG,Bergstrand M,Karlsson MO,Renard D,Heimann G

更新日期：2017-10-30 00:00:00

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

authors： Heimann G,Von Tress M,Gasparini M

更新日期：2015-08-30 00:00:00

abstract：:In many studies, interest lies in determining whether members of the study population will undergo a particular event of interest. Such scenarios are often termed 'mover-stayer' scenarios, and interest lies in modelling two sub-populations of 'movers' (those who have a propensity to undergo the event of interest) and ...

journal_title：Statistics in medicine

authors： O'Keeffe AG,Tom BD,Farewell VT

更新日期：2013-02-20 00:00:00

abstract：:In many medical and health studies, high-dimensional data are often encountered. Principal component analysis (PCA) is a commonly used technique to reduce such data to a few components that includes most of the information provided by the original data. However, PCA is known to be very sensitive to some abnormal obser...

journal_title：Statistics in medicine

authors： Fung WK,Gu H,Xiang L,Yau KK

更新日期：2007-06-15 00:00:00

abstract：:Meta analysis is a collection of quantitative methods devoted to combine summary information from related but independent studies. Because research reports usually present only data reductions and summary statistics rather than detailed data, the reviewer must often resort to rather crude methods for constructing summ...

journal_title：Statistics in medicine

authors： Di Pietrantonj C

更新日期：2006-07-15 00:00:00

abstract：:Public health interventions are often designed to target communities defined either geographically (e.g. cities, counties) or socially (e.g. schools or workplaces). The group randomized trial (GRT) is regarded as the gold standard for evaluating these interventions. However, community leaders may object to randomizati...

journal_title：Statistics in medicine

authors： Pennell ML,Hade EM,Murray DM,Rhoda DA

更新日期：2011-07-10 00:00:00

abstract：:We applied a computationally practical form of probit analysis for multiple response variables to data on early childhood development of four psychiatric disorders: disruptive disorders (DD-attention deficit disorders, oppositional defiant disorder, conduct disorder); adjustment disorders (ADJ); emotional disorders (E...

journal_title：Statistics in medicine

authors： Gibbons RD,Lavigne JV

更新日期：1998-11-15 00:00:00

abstract：:Clinical trials utilizing predictive biomarkers have become a research focus in personalized medicine. We investigate the effects of biomarker misclassification on the design and analysis of stratified biomarker clinical trials. For a variety of inference problems including marker-treatment interaction in particular, ...

journal_title：Statistics in medicine

authors： Liu C,Liu A,Hu J,Yuan V,Halabi S

更新日期：2014-08-15 00:00:00

abstract：:The Wilcoxon-Mann-Whitney (WMW) test is often used to compare the means or medians of two independent, possibly nonnormal distributions. For this problem, the true significance level of the large sample approximate version of the WMW test is known to be sensitive to differences in the shapes of the distributions. Base...

journal_title：Statistics in medicine

authors： Fagerland MW,Sandvik L

更新日期：2009-05-01 00:00:00

abstract：:A mixed effect model is proposed to jointly analyze multivariate longitudinal data with continuous, proportion, count, and binary responses. The association of the variables is modeled through the correlation of random effects. We use a quasi-likelihood type approximation for nonlinear variables and transform the prop...

journal_title：Statistics in medicine

authors： Li H,Zhang Y,Carroll RJ,Keadle SK,Sampson JN,Matthews CE

更新日期：2017-11-10 00:00:00

abstract：:A mixture model incorporating long-term survivors has been adopted in the field of biostatistics where some individuals may never experience the failure event under study. The surviving fractions may be considered as cured. In most applications, the survival times are assumed to be independent. However, when the survi...

journal_title：Statistics in medicine

authors： Yau KK,Ng AS

更新日期：2001-06-15 00:00:00

abstract：:Lower urinary tract symptoms can indicate the presence of urinary tract infection (UTI), a condition that if it becomes chronic requires expensive and time consuming care as well as leading to reduced quality of life. Detecting the presence and gravity of an infection from the earliest symptoms is then highly valuable...

journal_title：Statistics in medicine

authors： Barcella W,Iorio MD,Baio G,Malone-Lee J

更新日期：2016-04-15 00:00:00

abstract：:We present a model for describing correlated binocular data from reader-based diagnostic studies, where the same group of readers evaluates the presence or absence of certain diseases on binocular organs (e.g., fellow eyes) of patients. Multiple random effects are incorporated to meaningfully delineate various associa...

journal_title：Statistics in medicine

authors： Withanage N,de Leon AR,Rudnisky CJ

更新日期：2015-12-20 00:00:00

abstract：:Performance of a diagnostic test is ideally evaluated by a comparison of the test results to a gold standard for all the patients in a study. In practice, however, it is common for a subset of study patients to have the gold standard not verified (missing) due to ethical or expense considerations. Sensitivity and spec...

journal_title：Statistics in medicine

authors： Kosinski AS,Chen Y,Lyles RH

更新日期：2010-07-10 00:00:00