Abstract:
:Medical research commonly relies on the combination of 2 x 2 tables of counted data for making inferences about treatment effects or about the causes of disease. This article reviews point estimation and interval estimation for a common odds ratio. Traditional methods for providing these estimates face special challenges, and sometimes break down, when the data are sparse. Recent research provides practical alternatives to the traditional methods, and uses new computer algorithms to make them readily available in microcomputer software packages. This article illustrates and compares the various methods and offers recommendations for using the newer methods when analysing data sets of moderate size.
journal_name
Stat Methods Med Resjournal_title
Statistical methods in medical researchauthors
Emerson JDdoi
10.1177/096228029400300204subject
Has Abstractpub_date
1994-01-01 00:00:00pages
157-78issue
2eissn
0962-2802issn
1477-0334journal_volume
3pub_type
杂志文章,评审abstract::Nonlinear mixed-effects modeling is one of the most popular tools for analyzing repeated measurement data, particularly for applications in the biomedical fields. Multiple integration and nonlinear optimization are the two major challenges for likelihood-based methods in nonlinear mixed-effects modeling. To solve thes...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280220949898
更新日期:2020-08-24 00:00:00
abstract::Many statistical studies report p-values for inferential purposes. In several scenarios, the stochastic aspect of p-values is neglected, which may contribute to drawing wrong conclusions in real data experiments. The stochastic nature of p-values makes their use to examine the performance of given testing procedures o...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217704451
更新日期:2018-12-01 00:00:00
abstract::Cluster randomized and multicentre trials evaluate the effect of a treatment on persons nested within clusters, for instance patients within clinics or pupils within schools. Although equal sample sizes per cluster are generally optimal for parameter estimation, they are rarely feasible. This paper addresses the relat...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280206079018
更新日期:2008-08-01 00:00:00
abstract::Semicompeting risks and interval censoring are frequent in medical studies, for instance when a disease may be diagnosed only at times of visit and disease onset is in competition with death. To evaluate the ability of markers to predict disease onset in this context, estimators of discrimination measures must account...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214531691
更新日期:2016-12-01 00:00:00
abstract::We propose a fully parametric model for the analysis of competing risks data where the types of failure may not be independent. We show how the dependence between the cause-specific survival times can be modelled with a copula function. Features include: identifiability of the problem; accessible understanding of the ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1191/0962280203sm335ra
更新日期:2003-08-01 00:00:00
abstract::Early phase trials of complex interventions currently focus on assessing the feasibility of a large randomised control trial and on conducting pilot work. Assessing the efficacy of the proposed intervention is generally discouraged, due to concerns of underpowered hypothesis testing. In contrast, early assessment of e...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215589507
更新日期:2016-06-01 00:00:00
abstract::In this paper, a new allocation rule for treatment assignments in sequential clinical trials is proposed. The stratified and randomized play-the-winner rule (SRPWR) is an extension of the randomized play-the-winner rule to more than two treatments. It is applicable to cases where the probabilities of success of a trea...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280207081606
更新日期:2008-12-01 00:00:00
abstract::Classification with a large number of predictors and biomarker discovery become increasingly important in biological and medical research. This paper focuses on performing classification of cardiovascular diseases based on electrocardiogram analysis which deals with many variables and a lot of measurements within vari...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217699996
更新日期:2018-11-01 00:00:00
abstract::Clinical trials are expensive and time-consuming and so should also be used to study how treatments work, allowing for the evaluation of theoretical treatment models and refinement and improvement of treatments. These treatment processes can be studied using mediation analysis. Randomised treatment makes some of the a...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280216666111
更新日期:2018-06-01 00:00:00
abstract::Repeated measures and multivariate outcomes are an increasingly common feature of trials. Their joint analysis by means of random effects and latent variable models is appealing but patterns of heterogeneity in outcome profile may not conform to standard multivariate normal assumptions. In addition, there is much inte...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/0962280209105016
更新日期:2010-06-01 00:00:00
abstract::Estimating the long-term health impact of air pollution using an ecological spatio-temporal study design is a challenging task, due to the presence of residual spatio-temporal autocorrelation in the health counts after adjusting for the covariate effects. This autocorrelation is commonly modelled by a set of random ef...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214527384
更新日期:2014-12-01 00:00:00
abstract::With the emergence of rich information on biomarkers after treatments, new types of prognostic tools are being developed: dynamic prognostic tools that can be updated at each new biomarker measurement. Such predictions are of interest in oncology where after an initial treatment, patients are monitored with repeated b...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214535763
更新日期:2016-12-01 00:00:00
abstract::One of the main advantages of Bayesian analyses of clinical trials is their ability to formally incorporate skepticism about large treatment effects through the use of informative priors. We conducted a simulation study to assess the performance of informative normal, Student- t, and beta distributions in estimating r...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215620828
更新日期:2018-01-01 00:00:00
abstract::Statistical methods for carrying out asymptotic inferences (tests or confidence intervals) relative to one or two independent binomial proportions are very frequent. However, inferences about a linear combination of K independent proportions L = Σβ(i)p(i) (in which the first two are special cases) have had very little...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280209347953
更新日期:2011-08-01 00:00:00
abstract::This review provides an overview of forensic inference from genetic markers. Because the judge and jurors are charged with decision-making, the forensic expert's job is to provide a useful summary of the evidence to the court. Hence, this review focuses on the likelihood ratio as a means of summarizing the genetic dat...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228029300200304
更新日期:1993-01-01 00:00:00
abstract::In reliability theory, diagnostic accuracy, and clinical trials, the quantity P ( X > Y ) + ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280220966629
更新日期:2020-12-01 00:00:00
abstract::We review the origins of backcalculation (or back projection) methods developed for the analysis of AIDS (acquired immunodeficiency syndrome) incidence data. These techniques have been used extensively for >15 years to deconvolute clinical case incidence, given knowledge of the incubation period distribution, to obtai...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1191/0962280203sm337ra
更新日期:2003-06-01 00:00:00
abstract::A central assumption in the design and conduct of non-inferiority trials is that the active-control therapy will have the same degree of effectiveness in the planned non-inferiority trial as in the prior placebo-controlled trials used to define the non-inferiority margin. This is referred to as the 'constancy' assumpt...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280218801134
更新日期:2019-10-01 00:00:00
abstract::Pattern-mixture model (PMM)-based controlled imputations have become a popular tool to assess the sensitivity of primary analysis inference to different post-dropout assumptions or to estimate treatment effectiveness. The methodology is well established for continuous responses but less well established for binary res...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280220941880
更新日期:2020-12-01 00:00:00
abstract::Estimation of the effect of a binary exposure on an outcome in the presence of confounding is often carried out via outcome regression modelling. An alternative approach is to use propensity score methodology. The propensity score is the conditional probability of receiving the exposure given the observed covariates a...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280210394483
更新日期:2012-06-01 00:00:00
abstract::Monte Carlo evaluation of resampling-based tests is often conducted in statistical analysis. However, this procedure is generally computationally intensive. The pooling resampling-based method has been developed to reduce the computational burden but the validity of the method has not been studied before. In this arti...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280216661876
更新日期:2018-05-01 00:00:00
abstract::In recent years, there has been a prominent discussion in the literature about the potential for overestimation of the treatment effect when a clinical trial stops at an interim analysis due to the experimental treatment showing a benefit over the control. However, there has been much less attention paid to the conver...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280218795320
更新日期:2019-10-01 00:00:00
abstract::The random effects model in meta-analysis is a standard statistical tool often used to analyze the effect sizes of the quantity of interest if there is heterogeneity between studies. In the special case considered here, meta-analytic data contain only the sample means in two treatment arms and the sample sizes, but no...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217718867
更新日期:2019-01-01 00:00:00
abstract::Spatial resolution plays an important role in functional magnetic resonance imaging studies as the signal-to-noise ratio increases linearly with voxel volume. In scientific studies, where functional magnetic resonance imaging is widely used, the standard spatial resolution typically used is relatively low which ensure...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217752378
更新日期:2019-04-01 00:00:00
abstract::Dependent binary response data arise frequently in practice due to repeated measurements in longitudinal studies or to subsampling primary sampling units as in fields such as teratology and ophthalmology. Several classes of approaches have recently been proposed to analyse such repeated binary outcome data. The differ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228029200100303
更新日期:1992-01-01 00:00:00
abstract::Trials run in either rare diseases, such as rare cancers, or rare sub-populations of common diseases are challenging in terms of identifying, recruiting and treating sufficient patients in a sensible period. Treatments for rare diseases are often designed for other disease areas and then later proposed as possible tre...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280216662070
更新日期:2018-05-01 00:00:00
abstract::In the clinical development of some new infectious disease drugs, early clinical pharmacology trials may predict with high confidence that the efficacious doses are well below the range of the safety margin. In this case, a dose-ranging study may be unnecessary after a proof-of-concept (PoC) study testing the highest ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280218807950
更新日期:2019-12-01 00:00:00
abstract::The analysis of health care costs is complicated by the skewed and heteroscedastic nature of their distribution with possibly additional zero values. Statistical methods that do not adjust for these features can lead to incorrect conclusions. This paper reviews recent developments in statistical methods for the analys...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1191/0962280202sm290ra
更新日期:2002-08-01 00:00:00
abstract::This paper reviews models for incomplete continuous and categorical longitudinal data. In terms of Rubin's classification of missing value processes we are specifically concerned with the problem of nonrandom missingness. A distinction is drawn between the classes of selection and pattern-mixture models and, using sev...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228029900800105
更新日期:1999-03-01 00:00:00
abstract::It is a common practice to analyze longitudinal data frequently arisen in medical studies using various mixed-effects models in the literature. However, the following issues may standout in longitudinal data analysis: (i) In clinical practice, the profile of each subject's response from a longitudinal study may follow...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214544207
更新日期:2017-02-01 00:00:00