RESUMEN
A stratified study is often designed for adjusting a confounding effect or effect of different centers/groups in two treatments or diagnostic tests, and the risk difference is one of the most frequently used indices in comparing efficiency between two treatments or diagnostic tests. This article presented five simultaneous confidence intervals (CIs) for risk differences in stratified bilateral designs accounting for the intraclass correlation and developed seven CIs for the common risk difference under the homogeneity assumption. The performance of the CIs is evaluated with respect to the empirical coverage probabilities, empirical coverage widths and ratios of mesial noncoverage probability and the noncoverage probability under various scenarios. Empirical results show that Wald simultaneous CI, Haldane simultaneous CI, Score simultaneous CI based on Bonferroni method and simultaneous CI based on bootstrap-resampling method perform satisfactorily and hence be recommended for applications, the CI based on the weighted-least-square (WLS) estimator, the CIs based on Mantel-Haenszel estimator, the CI based on Cochran statistic and the CI based on Score statistic for the common risk difference behave well even under small sample sizes. A real data example is used to demonstrate the proposed methodologies.
Asunto(s)
Intervalos de Confianza , Modelos Estadísticos , Ensayos Clínicos Controlados Aleatorios como Asunto/métodos , Ensayos Clínicos Controlados Aleatorios como Asunto/estadística & datos numéricos , Proyectos de Investigación/estadística & datos numéricos , Simulación por Computador , Humanos , Análisis de los Mínimos Cuadrados , Probabilidad , Riesgo , Tamaño de la MuestraRESUMEN
A sufficient number of participants should be included to adequately address the research interest in the surveys with sensitive questions. In this paper, sample size formulas/iterative algorithms are developed from the perspective of controlling the confidence interval width of the prevalence of a sensitive attribute under four non-randomized response models: the crosswise model, parallel model, Poisson item count technique model and negative binomial item count technique model. In contrast to the conventional approach for sample size determination, our sample size formulas/algorithms explicitly incorporate an assurance probability of controlling the width of a confidence interval within the pre-specified range. The performance of the proposed methods is evaluated with respect to the empirical coverage probability, empirical assurance probability and confidence width. Simulation results show that all formulas/algorithms are effective and hence are recommended for practical applications. A real example is used to illustrate the proposed methods.
RESUMEN
Responses from the paired organs are generally highly correlated in bilateral studies, statistical procedures ignoring the correlation could lead to incorrect results. Note the intraclass correlation in the study of combined unilateral and bilateral outcomes; 11 confidence intervals (CIs) including 7 asymptotic CIs and 4 Bootstrap-resampling CIs for assessing the equivalence of 2 treatments are derived under Rosner's correlated binary data model. Performance is evaluated with respect to the empirical coverage probability (ECP), the empirical coverage width (ECW) and the ratio of the mesial non-coverage probability to the non-coverage probability (RMNCP) via simulation studies. Simulation results show that (i) all CIs except for the Wald CI and the bias-corrected Bootstrap percentile CI generally produce satisfactory ECPs and hence are recommended; (ii) all CIs except for the bias-corrected Bootstrap percentile CI provide preferred RMNCPs and are more symmetrical; (iii) as the measurement of the dependence increases, the ECWs of all CIs except for the score CI and the profile likelihood CI show increasing patterns that look like linear, while there is no obvious pattern on the ECPs of all CIs except for the profile likelihood CI. A data set from an otolaryngologic study is used to illustrate the proposed methods.
RESUMEN
Studies with sensitive questions should include a sufficient number of respondents to adequately address the research interest. While studies with an inadequate number of respondents may not yield significant conclusions, studies with an excess of respondents become wasteful of investigators' budget. Therefore, it is an important step in survey sampling to determine the required number of participants. In this article, we derive sample size formulas based on confidence interval estimation of prevalence for four randomized response models, namely, the Warner's randomized response model, unrelated question model, item count technique model and cheater detection model. Specifically, our sample size formulas control, with a given assurance probability, the width of a confidence interval within the planned range. Simulation results demonstrate that all formulas are accurate in terms of empirical coverage probabilities and empirical assurance probabilities. All formulas are illustrated using a real-life application about the use of unethical tactics in negotiation.
Asunto(s)
Modelos Estadísticos , Humanos , Tamaño de la Muestra , Prevalencia , Psicometría , Probabilidad , Simulación por Computador , Intervalos de ConfianzaRESUMEN
A disease prevalence can be estimated by classifying subjects according to whether they have the disease. When gold-standard tests are too expensive to be applied to all subjects, partially validated data can be obtained by double-sampling in which all individuals are classified by a fallible classifier, and some of individuals are validated by the gold-standard classifier. However, it could happen in practice that such infallible classifier does not available. In this article, we consider two models in which both classifiers are fallible and propose four asymptotic test procedures for comparing disease prevalence in two groups. Corresponding sample size formulae and validated ratio given the total sample sizes are also derived and evaluated. Simulation results show that (i) Score test performs well and the corresponding sample size formula is also accurate in terms of the empirical power and size in two models; (ii) the Wald test based on the variance estimator with parameters estimated under the null hypothesis outperforms the others even under small sample sizes in Model II, and the sample size estimated by this test is also accurate; (iii) the estimated validated ratios based on all tests are accurate. The malarial data are used to illustrate the proposed methodologies.