Search results for "Sample Size"

Comparison between splines and fractional polynomials for multivariable model building with continuous covariates: a simulation study with continuous…

2012

In observational studies, many continuous or categorical covariates may be related to an outcome. Various spline-based procedures or the multivariable fractional polynomial (MFP) procedure can be used to identify important variables and functional forms for continuous covariates. This is the main aim of an explanatory model, as opposed to a model only for prediction. The type of analysis often guides the complexity of the final model. Spline-based procedures and MFP have tuning parameters for choosing the required complexity. To compare model selection approaches, we perform a simulation study in the linear regression context based on a data structure intended to reflect realistic biomedica…

Power and Type I Error of the Mean and Covariance Structure Analysis Model for Detecting Differential Item Functioning in Graded Response Items.

2016

In this simulation study, we investigate the power and Type I error rate of a procedure based on the mean and covariance structure analysis (MACS) model in detecting differential item functioning (DIF) of graded response items with five response categories. The following factors were manipulated: type of DIF (uniform and non-uniform), DIF magnitude (low, medium and large), equality/inequality of latent trait distributions, sample size (100, 200, 400, and 800) and equality or inequality of the sample sizes across groups. The simulated test was made up of 10 items, of which only 1 contained DIF. One hundred replications were generated for each simulated condition. Results indicate that the MA…

Adaptive designs with correlated test statistics

2009

In clinical trials, the collected observations such as clustered data or repeated measurements are often correlated. As a consequence, test statistics in a multistage design are correlated. Adaptive designs were originally developed for independent test statistics. We present a general framework for two-stage adaptive designs with correlated test statistics. We show that the significance level for the Bauer-Köhne design is inflated for positively correlated test statistics from a bivariate normal distribution. The decision boundary for the second stage can be modified so that type one error is controlled. This general concept is expandable to other adaptive designs. In order to use these de…

Statistical inference as a decision problem: the choice of sample size

1997

Performance of adaptive sample size adjustment with respect to stopping criteria and time of interim analysis

2006

The benefit of adjusting the sample size in clinical trials on the basis of treatment effects observed in interim analysis has been the subject of several recent papers. Different conclusions were drawn about the usefulness of this approach for gaining power or saving sample size, because of differences in trial design and setting. We examined the benefit of sample size adjustment in relation to trial design parameters such as 'time of interim analysis' and 'choice of stopping criteria'. We compared the adaptive weighted inverse normal method with classical group sequential methods for the most common and for optimal stopping criteria in early, half-time and late interim analyses. We found …

Nearly exact sample size calculation for powerful non-randomized tests for differences between binomial proportions

2015

In the case of two independent samples, it turns out that among the procedures taken in consideration, BOSCHLOO'S technique of raising the nominal level in the standard conditional test as far as admissible performs best in terms of power against almost all alternatives. The computational burden entailed in exact sample size calculation is comparatively modest for both the uniformly most powerful unbiased randomized and the conservative non-randomized version of the exact Fisher-type test. Computing these values yields a pair of bounds enclosing the exact sample size required for the Boschloo test, and it seems reasonable to replace the exact value with the middle of the corresponding inter…

Inferential tools in penalized logistic regression for small and sparse data: A comparative study.

2016

This paper focuses on inferential tools in the logistic regression model fitted by the Firth penalized likelihood. In this context, the Likelihood Ratio statistic is often reported to be the preferred choice as compared to the ‘traditional’ Wald statistic. In this work, we consider and discuss a wider range of test statistics, including the robust Wald, the Score, and the recently proposed Gradient statistic. We compare all these asymptotically equivalent statistics in terms of interval estimation and hypothesis testing via simulation experiments and analyses of two real datasets. We find out that the Likelihood Ratio statistic does not appear the best inferential device in the Firth penal…

Sample size in cluster-randomized trials with time to event as the primary endpoint

2011

In cluster-randomized trials, groups of individuals (clusters) are randomized to the treatments or interventions to be compared. In many of those trials, the primary objective is to compare the time for an event to occur between randomized groups, and the shared frailty model well fits clustered time-to-event data. Members of the same cluster tend to be more similar than members of different clusters, causing correlations. As correlations affect the power of a trial to detect intervention effects, the clustered design has to be considered in planning the sample size. In this publication, we derive a sample size formula for clustered time-to-event data with constant marginal baseline hazards…

Diseño muestral optimo en el caso de no respuesta

1982

Discussed here are several aspects of a simple model for dealing with nonresponse. The model is, in a sense, a sequential one and is developed from a Bayesian decision theory point of view. Within this framework we examine how formalization and combination of one's opinions, and past experience concerning the proportion of nonrespondents, the differences and relations between respondents and nonrespondents, the cost of obtaining information from nonrespondents, etc. We examine the decisions concerning the selection of sampling size m and n, both in the nonrespondent population and in the overall population

On powerful exact nonrandomized tests for the Poisson two-sample setting.

2020

In the case of two independent samples from Poisson distributions, the natural target parameter for hypothesis testing is the ratio of the two population means. The conditional tests which have been derived for this class of problems already in the 1940s are well known to be optimal in terms of power only when randomized decisions between hypotheses are admitted at the boundary of the respective rejection regions. The major objective of this contribution is to show how the approach used by Boschloo in 1970 for constructing a powerful nonrandomized version of Fisher’s exact test for hypotheses about the odds ratio between two binomial parameters can successfully be adapted for the Poisson c…