Confidence intervals for the difference between two median survival times for clustered survival data

Clustered survival data arise often in clinical trial design, where the correlated subunits from the same cluster are randomized to different treatment groups. Under such design, we consider the problem of constructing confidence interval for the difference of two median survival time given the covariates. We use Cox gamma frailty model to account for the within-cluster correlation. Based on the conditional confidence intervals, we can identify the possible range of covariates over which the two groups would provide different median survival times. The associated coverage probability and the expected length of the proposed interval are investigated via a simulation study. The implementation of the confidence intervals is illustrated using a real data set.     

Conservative confidence intervals for the intraclass correlation coefficient for clustered binary data

Asymptotic approaches are traditionally used to calculate confidence intervals for intraclass correlation coefficient in a clustered binary study. When sample size is small to medium, or correlation or response rate is near the boundary, asymptotic intervals often do not have satisfactory performance with regard to coverage. We propose using the importance sampling method to construct the profile confidence limits for the intraclass correlation coefficient. Importance sampling is a simulation based approach to reduce the variance of the estimated parameter. Four existing asymptotic limits are used as statistical quantities for sample space ordering in the importance sampling method. Simulation studies are performed to evaluate the performance of the proposed accurate intervals with regard to coverage and interval width. Simulation results indicate that the accurate intervals based on the asymptotic limits by Fleiss and Cuzick generally have shorter width than others in many cases, while the accurate intervals based on Zou and Donner asymptotic limits outperform others when correlation and response rate are close to their boundaries.     

Confidence intervals for the difference in paired Youden indices

Comparison of accuracy between two diagnostic tests can be implemented by investigating the difference in paired Youden indices. However, few literature articles have discussed the inferences for the difference in paired Youden indices. In this paper, we propose an exact confidence interval for the difference in paired Youden indices based on the generalized pivotal quantities. For comparison, the maximum likelihood estimate‐based interval and a bootstrap‐based interval are also included in the study for the difference in paired Youden indices. Abundant simulation studies are conducted to compare the relative performance of these intervals by evaluating the coverage probability and average interval length. Our simulation results demonstrate that the exact confidence interval outperforms the other two intervals even with small sample size when the underlying distributions are normal. A real application is also used to illustrate the proposed intervals. Copyright © 2012 John Wiley & Sons, Ltd.     

A new revised version of McNemar's test for paired binary data

By considering separately B and C, the frequencies of individuals who consistently gave positive or negative answers in before and after responses, a new revised version of McNemar's test is derived. It improves upon Lu's revised formula, which considers B and C together. When both B and C are 0, the new revised version produces the same results as McNemar's test. When one of B and C is 0, the new revised test produces the same results as Lu's version. Compared to Lu's version, the new revised test is a more complete revision of McNemar's test.     

Robust tests for the equality of variances for clustered data

Tests for the equality of variances are often needed in applications. In genetic studies the assumption of equal variances of continuous traits, measured in identical and fraternal twins, is crucial for heritability analysis. To test the equality of variances of traits, which are non-normally distributed, Levene [H. Levene, Robust tests for equality of variances, in Contributions to Probability and Statistics, I. Olkin, ed. Stanford University Press, Palo Alto, California, 1960, pp. 278–292] suggested a method that was surprisingly robust under non-normality, and the procedure was further improved by Brown and Forsythe [M.B. Brown and A.B. Forsythe, Robust tests for the equality of variances, J. Amer. Statis. Assoc. 69 (1974), pp. 364–367]. These tests assumed independence of observations. However, twin data are clustered – observations within a twin pair may be dependent due to shared genes and environmental factors. Uncritical application of the tests of Brown and Forsythe to clustered data may result in much higher than nominal Type I error probabilities. To deal with clustering we developed an extended version of Levene's test, where the ANOVA step is replaced with a regression analysis followed by a Wald-type test based on a clustered version of the robust Huber–White sandwich estimator of the covariance matrix. We studied the properties of our procedure using simulated non-normal clustered data and obtained Type I error rates close to nominal as well as reasonable powers. We also applied our method to oral glucose tolerance test data obtained from a twin study of the metabolic syndrome and related components and compared the results with those produced by the traditional approaches.     

Confidence intervals for a bounded mean in exponential families

Setting confidence bounds or intervals for a parameter in a restricted parameter space is an important issue in applications and is widely discussed in the recent literature. In this article, we focus on the distributions in the exponential families, and propose general forms of the truncated Pratt interval and rp interval for the means. We take the Poisson distribution as an example to illustrate the method and compare it with the other existing intervals. Besides possessing the merits from the theoretical inferences, the proposed intervals are also shown to be competitive approaches from simulation and real-data application studies.     

Confidence intervals for the difference between independent binomial proportions: comparison using a graphical approach and moving averages

This paper uses graphical methods to illustrate and compare the coverage properties of a number of methods for calculating confidence intervals for the difference between two independent binomial proportions. We investigate both small‐sample and large‐sample properties of both two‐sided and one‐sided coverage, with an emphasis on asymptotic methods. In terms of aligning the smoothed coverage probability surface with the nominal confidence level, we find that the score‐based methods on the whole have the best two‐sided coverage, although they have slight deficiencies for confidence levels of 90% or lower. For an easily taught, hand‐calculated method, the Brown‐Li ‘Jeffreys’ method appears to perform reasonably well, and in most situations, it has better one‐sided coverage than the widely recommended alternatives. In general, we find that the one‐sided properties of many of the available methods are surprisingly poor. In fact, almost none of the existing asymptotic methods achieve equal coverage on both sides of the interval, even with large sample sizes, and consequently if used as a non‐inferiority test, the type I error rate (which is equal to the one‐sided non‐coverage probability) can be inflated. The only exception is the Gart‐Nam ‘skewness‐corrected’ method, which we express using modified notation in order to include a bias correction for improved small‐sample performance, and an optional continuity correction for those seeking more conservative coverage. Using a weighted average of two complementary methods, we also define a new hybrid method that almost matches the performance of the Gart‐Nam interval. Copyright © 2014 John Wiley & Sons, Ltd.     

Confidence intervals for the between group variance in the unbalanced one-way random effects model of anaylsis of variance

A confidence interval for the between group variance is proposed which is deduced from Wald'sexact confidence interval for the rtio of the two variance components in the one-way random effects model and the exact confidence interval for the error variance resp.an unbiased estimator of the error variance. In a simulation study the confidence coeffecients for these two intervals are compared with the confidence coefficients of two other commonly used confidence intervals. There the confidence interval derived here yields confidence coefficiends which are always greater than the prescriped level.     

A comparative study of confidence intervals to assess biosimilarity from analytical data

Assessment of analytical similarity of tier 1 quality attributes is based on a set of hypotheses that tests the mean difference of reference and test products against a margin adjusted for standard deviation of the reference product. Thus, proper assessment of the biosimilarity hypothesis requires statistical tests that account for the uncertainty associated with the estimations of the mean differences and the standard deviation of the reference product. Recently, a linear reformulation of the biosimilarity hypothesis has been proposed, which facilitates development and implementation of statistical tests. These statistical tests account for the uncertainty in the estimation process of all the unknown parameters. In this paper, we survey methods for constructing confidence intervals for testing the linearized reformulation of the biosimilarity hypothesis and also compare the performance of the methods. We discuss test procedures using confidence intervals to make possible comparison among recently developed methods as well as other previously developed methods that have not been applied for demonstrating analytical similarity. A computer simulation study was conducted to compare the performance of the methods based on the ability to maintain the test size and power, as well as computational complexity. We demonstrate the methods using two example applications. At the end, we make recommendations concerning the use of the methods.     

Comparisons of computational methods for clustered binary data

Clustered binary data are common in medical research and can be fitted to the logistic regression model with random effects which belongs to a wider class of models called the generalized linear mixed model. The likelihood-based estimation of model parameters often has to handle intractable integration which leads to several estimation methods to overcome such difficulty. The penalized quasi-likelihood (PQL) method is the one that is very popular and computationally efficient in most cases. The expectation–maximization (EM) algorithm allows to estimate maximum-likelihood estimates, but requires to compute possibly intractable integration in the E-step. The variants of the EM algorithm to evaluate the E-step are introduced. The Monte Carlo EM (MCEM) method computes the E-step by approximating the expectation using Monte Carlo samples, while the Modified EM (MEM) method computes the E-step by approximating the expectation using the Laplace's method. All these methods involve several steps of approximation so that corresponding estimates of model parameters contain inevitable errors (large or small) induced by approximation. Understanding and quantifying discrepancy theoretically is difficult due to the complexity of approximations in each method, even though the focus is on clustered binary data. As an alternative competing computational method, we consider a non-parametric maximum-likelihood (NPML) method as well. We review and compare the PQL, MCEM, MEM and NPML methods for clustered binary data via simulation study, which will be useful for researchers when choosing an estimation method for their analysis.     

Confidence intervals for assessing equivalence of two treatments with combined unilateral and bilateral data

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.     

Confidence intervals for the comparison of two poisson distributions

Confidence interval construction the difference in mean event rates for two Index independent , Poisson samples is discussed. Intervals are derived by considering Bayes estimates of the mean event rates using a family of noninformative priors. The coverage probabilities of the proposed are compared to those of the standard Wald interval for of observed events. A compromise method of constructing interval based on the data is suggested and its properties are evaluated. The method is illustrated in several examples.     

Confidence intervals for coefficients of variation in two-parameter exponential distributions

This article examines confidence intervals for the single coefficient of variation and the difference of coefficients of variation in the two-parameter exponential distributions, using the method of variance of estimates recovery (MOVER), the generalized confidence interval (GCI), and the asymptotic confidence interval (ACI). In simulation, the results indicate that coverage probabilities of the GCI maintain the nominal level in general. The MOVER performs well in terms of coverage probability when data only consist of positive values, but it has wider expected length. The coverage probabilities of the ACI satisfy the target for large sample sizes. We also illustrate our confidence intervals using a real-world example in the area of medical science.     

Confidence intervals for estimating the population signal-to-noise ratio: a simulation study

This paper considered several confidence intervals for estimating the population signal-to-noise ratio based on parametric, non-parametric and modified methods. A simulation study has been conducted to compare the performance of the interval estimators under both symmetric and skewed distributions. We reported coverage probability and average width of the interval estimators. Based on the simulation study, we observed that some of our proposed interval estimators are performing better in the sense of smaller width and coverage probability and have been recommended for the researchers.     

Confidence intervals for the ratio of two variances

In this paper we consider confidence intervals for the ratio of two population variances. We propose a confidence interval for the ratio of two variances based on the t-statistic by deriving its Edgeworth expansion and considering Hall's and Johnson's transformations. Then, we consider the coverage accuracy of suggested intervals and intervals based on the F-statistic for some distributions.     

Testing the homogeneity of proportions for clustered binary data without knowing the correlation structure

A robust generalized score test for comparing groups of cluster binary data is proposed. This novel test is asymptotically valid for practically any underlying correlation configurations including the situation when correlation coefficients vary within or between clusters. This structure generally undermines the validity of the typical large sample properties of the method of maximum likelihood. Simulations and real data analysis are used to demonstrate the merit of this parametric robust method. Results show that our test is superior to two recently proposed test statistics advocated by other researchers.     

Confidence intervals for the mean of a normal distribution with restricted parameter space

For a normal distribution with known variance, the standard confidence interval of the location parameter is derived from the classical Neyman procedure. When the parameter space is known to be restricted, the standard confidence interval is arguably unsatisfactory. Recent articles have addressed this problem and proposed confidence intervals for the mean of a normal distribution where the parameter space is not less than zero. In this article, we propose a new confidence interval, rp interval, and derive the Bayesian credible interval and likelihood ratio interval for general restricted parameter space. We compare these intervals with the standard interval and the minimax interval. Simulation studies are undertaken to assess the performances of these confidence intervals.     

On the robustness of empirical likelihood ratio confidence intervals for location

The authors examine the robustness of empirical likelihood ratio (ELR) confidence intervals for the mean and M‐estimate of location. They show that the ELR interval for the mean has an asymptotic breakdown point of zero. They also give a formula for computing the breakdown point of the ELR interval for M‐estimate. Through a numerical study, they further examine the relative advantages of the ELR interval to the commonly used confidence intervals based on the asymptotic distribution of the M‐estimate.     

Smooth bootstrap-based confidence intervals for one binomial proportion and difference of two proportions

Constructing confidence intervals (CIs) for a binomial proportion and the difference between two binomial proportions is a fundamental and well-studied problem with respect to the analysis of binary data. In this note, we propose a new bootstrap procedure to estimate the CIs by resampling from a newly developed smooth quantile function in [11 Newcombe, R. G. 1998. Two-sided confidence intervals for the single proportion: Comparison of seven methods. Stat. Med., 17: 857–872. (doi:10.1002/(SICI)1097-0258(19980430)17:8<857::AID-SIM777>3.0.CO;2-E)[Crossref], [PubMed], [Web of Science ®] , [Google Scholar]] for discrete data. We perform a variety of simulation studies in order to illustrate the strong performance of our approach. The coverage probabilities of our CIs in the one-sample setting are superior than or comparable to other well-known approaches. The true utility of our new and novel approach is in the two-sample setting. For the difference of two proportions, our smooth bootstrap CIs provide better coverage probabilities almost uniformly over the interval (?1, 1), particularly in the tail region as compared than other published methods included in our simulation. We illustrate our methodology via an application to several different binary data sets.     

Approximate and Fiducial Confidence Intervals for the Difference Between Two Binomial Proportions

The problem of estimating the difference between two binomial proportions is considered. Closed-form approximate confidence intervals (CIs) and a fiducial CI for the difference between proportions are proposed. The approximate CIs are simple to compute, and they perform better than the classical Wald CI in terms of coverage probabilities and precision. Numerical studies indicate that these approximate CIs can be used safely for practical applications under a simple condition. The fiducial CI is more accurate than the approximate CIs in terms of coverage probabilities. The fiducial CIs, the Newcombe CIs, and the Miettinen–Nurminen CIs are comparable in terms of coverage probabilities and precision. The interval estimation procedures are illustrated using two examples.