Equal‐tailed confidence intervals for comparison of rates

Several methods are available for generating confidence intervals for rate difference, rate ratio, or odds ratio, when comparing two independent binomial proportions or Poisson (exposure‐adjusted) incidence rates. Most methods have some degree of systematic bias in one‐sided coverage, so that a nominal 95% two‐sided interval cannot be assumed to have tail probabilities of 2.5% at each end, and any associated hypothesis test is at risk of inflated type I error rate. Skewness‐corrected asymptotic score methods have been shown to have superior equal‐tailed coverage properties for the binomial case. This paper completes this class of methods by introducing novel skewness corrections for the Poisson case and for odds ratio, with and without stratification. Graphical methods are used to compare the performance of these intervals against selected alternatives. The skewness‐corrected methods perform favourably in all situations—including those with small sample sizes or rare events—and the skewness correction should be considered essential for analysis of rate ratios. The stratified method is found to have excellent coverage properties for a fixed effects analysis. In addition, another new stratified score method is proposed, based on the t‐distribution, which is suitable for use in either a fixed effects or random effects analysis. By using a novel weighting scheme, this approach improves on conventional and modern meta‐analysis methods with weights that rely on crude estimation of stratum variances. In summary, this paper describes methods that are found to be robust for a wide range of applications in the analysis of rates.     

An asymptotic confidence interval for the process capability index Cpm

Abstract

The use of indices as an estimation tool of process capability is long-established among the statistical quality professionals. Numerous capability indices have been proposed in last few years. C_pm constitutes one of the most widely used capability indices and its estimation has attracted much interest. In this paper, we propose a new method for constructing an approximate confidence interval for the index C_pm. The proposed method is based on the asymptotic distribution of the index C_pm obtained by the Delta Method. Under some regularity conditions, the distribution of an estimator of the process capability index C_pm is asymptotically normal.     

Generalized confidence interval for the slope in linear measurement error model

A generalized confidence interval for the slope parameter in linear measurement error model is proposed in this article, which is based on the relation between the slope of classical regression model and the measurement error model. The performance of the confidence interval estimation procedure is studied numerically through Monte Carlo simulation in terms of coverage probability and expected length.     

An improved score interval with a modified midpoint for a binomial proportion

One of the most basic and important problems in statistical inference is the construction of the confidence interval (CI). In this paper, we propose a novel CI for a binomial proportion by modifying the midpoint of the score interval. The proposed modified interval can solve the ‘downward spikes’ problem of the score interval without enlarging the interval length. Simulation studies are carried out to illustrate the performance of the modified interval. With regard to the criterions of coverage probability, mean absolute error and expected length, our method is competitive among the several commonly used methods for constructing a CI. A real data example is also presented to show the application of our method.     

Simultaneous confidence interval construction for many-to-one comparisons of proportion differences based on correlated paired data

In some medical researches such as ophthalmological, orthopaedic and otolaryngologic studies, it is often of interest to compare multiple groups with a control using data collected from paired organs of patients. The major difficulty in performing the data analysis is to adjust the multiplicity between the comparison of multiple groups, and the correlation within the same patient''s paired organs. In this article, we construct asymptotic simultaneous confidence intervals (SCIs) for many-to-one comparisons of proportion differences adjusting for multiplicity and the correlation. The coverage probabilities and widths of the proposed CIs are evaluated by Monte Carlo simulation studies. The methods are illustrated by a real data example.     

Corrected profile likelihood confidence interval for binomial paired incomplete data

Clinical trials often use paired binomial data as their clinical endpoint. The confidence interval is frequently used to estimate the treatment performance. Tang et al. (2009) have proposed exact and approximate unconditional methods for constructing a confidence interval in the presence of incomplete paired binary data. The approach proposed by Tang et al. can be overly conservative with large expected confidence interval width (ECIW) in some situations. We propose a profile likelihood‐based method with a Jeffreys' prior correction to construct the confidence interval. This approach generates confidence interval with a much better coverage probability and shorter ECIWs. The performances of the method along with the corrections are demonstrated through extensive simulation. Finally, three real world data sets are analyzed by all the methods. Statistical Analysis System (SAS) codes to execute the profile likelihood‐based methods are also presented. Copyright © 2013 John Wiley & Sons, Ltd.     

A note on effective sample size for constructing confidence intervals for the difference of two proportions

Proportion differences are often used to estimate and test treatment effects in clinical trials with binary outcomes. In order to adjust for other covariates or intra-subject correlation among repeated measures, logistic regression or longitudinal data analysis models such as generalized estimating equation or generalized linear mixed models may be used for the analyses. However, these analysis models are often based on the logit link which results in parameter estimates and comparisons in the log-odds ratio scale rather than in the proportion difference scale. A two-step method is proposed in the literature to approximate the calculation of confidence intervals for the proportion difference using a concept of effective sample sizes. However, the performance of this two-step method has not been investigated in their paper. On this note, we examine the properties of the two-step method and propose an adjustment to the effective sample size formula based on Bayesian information theory. Simulations are conducted to evaluate the performance and to show that the modified effective sample size improves the coverage property of the confidence intervals.     

Fixed-width sequential confidence interval for the correlation coefficient of a bivariate normal distribution

A sequential confidence interval of fixed width 2d d > 0, is constructed for the correlation coefficient of a bivariate normal distribution. It is shown that the coverage probability is approximately equal to a preassigned number γ, 0 < γ < as d → 0.     

Optimal cut-off for rare events and unbalanced misclassification costs

This paper develops a method for handling two-class classification problems with highly unbalanced class sizes and misclassification costs. When the class sizes are highly unbalanced and the minority class represents a rare event, conventional classification methods tend to strongly favour the majority class, resulting in very low detection of the minority class. A method is proposed to determine the optimal cut-off for asymmetric misclassification costs and for unbalanced class sizes. Monte Carlo simulations show that this proposal performs better than the method based on the notion of classification accuracy. Finally, the proposed method is applied to empirical data on Italian small and medium enterprises to classify them into default and non-default groups.     

Profile likelihood-based confidence interval for the dispersion parameter in count data

The importance of the dispersion parameter in counts occurring in toxicology, biology, clinical medicine, epidemiology, and other similar studies is well known. A couple of procedures for the construction of confidence intervals (CIs) of the dispersion parameter have been investigated, but little attention has been paid to the accuracy of its CIs. In this paper, we introduce the profile likelihood (PL) approach and the hybrid profile variance (HPV) approach for constructing the CIs of the dispersion parameter for counts based on the negative binomial model. The non-parametric bootstrap (NPB) approach based on the maximum likelihood (ML) estimates of the dispersion parameter is also considered. We then compare our proposed approaches with an asymptotic approach based on the ML and the restricted ML (REML) estimates of the dispersion parameter as well as the parametric bootstrap (PB) approach based on the ML estimates of the dispersion parameter. As assessed by Monte Carlo simulations, the PL approach has the best small-sample performance, followed by the REML, HPV, NPB, and PB approaches. Three examples to biological count data are presented.     

A comparison of two varying coefficient meta-analysis methods for an average risk difference

Two interval estimation methods for a general linear function of binomial proportions have been proposed. One method [Zou GY, Huang W, Zhang X. A note on confidence interval estimation for a linear function of binomial proportions. Comput Statist Data Anal. 2009;53:1080–1085] combines Wilson interval estimates of individual proportions, and the other method [Price RM, Bonett DG. An improved confidence interval for a linear function of binomial proportions. Comput Statist Data Anal. 2004;45:449–456] uses an adjusted Wald interval. Both methods are appropriate in varying coefficient meta-analysis models where the risk differences are allowed to vary across studies. The two methods were compared in a simulation study under realistic meta-analysis conditions and the adjusted Wald method was found to have the best performance characteristics.     

Generalized confidence interval estimation for the mean of delta-lognormal distribution: an application to New Zealand trawl survey data

Highly skewed and non-negative data can often be modeled by the delta-lognormal distribution in fisheries research. However, the coverage probabilities of extant interval estimation procedures are less satisfactory in small sample sizes and highly skewed data. We propose a heuristic method of estimating confidence intervals for the mean of the delta-lognormal distribution. This heuristic method is an estimation based on asymptotic generalized pivotal quantity to construct generalized confidence interval for the mean of the delta-lognormal distribution. Simulation results show that the proposed interval estimation procedure yields satisfactory coverage probabilities, expected interval lengths and reasonable relative biases. Finally, the proposed method is employed in red cod densities data for a demonstration.     

Performance of confidence interval tests for the ratio of two lognormal means applied to Weibull and gamma distribution data

Five estimation approaches have been developed to compute the confidence interval (CI) for the ratio of two lognormal means: (1) T, the CI based on the t-test procedure; (2) ML, a traditional maximum likelihood-based approach; (3) BT, a bootstrap approach; (4) R, the signed log-likelihood ratio statistic; and (5) R*, the modified signed log-likelihood ratio statistic. The purpose of this study was to assess the performance of these five approaches when applied to distributions other than lognormal distribution, for which they were derived. Performance was assessed in terms of average length and coverage probability of the CIs for each estimation approaches (i.e., T, ML, BT, R, and R*) when data followed a Weibull or gamma distribution. Four models were discussed in this study. In Model 1, the sample sizes and variances were equal within the two groups. In Model 2, the sample sizes were equal but variances were different within the two groups. In Model 3, the variances were different within the two groups and the larger variance was paired with the larger sample size. In Model 4, the variances were different within the two groups and the larger variance was paired with the smaller sample size. The results showed that when the variances of the two groups were equal, the t-test performed well, no matter what the underlying distribution was and how large the variances of the two groups were. The BT approach performed better than the others when the underlying distribution was not lognormal distribution, although it was inaccurate when the variances were large. The R* test did not perform well when the underlying distribution was Weibull or gamma distributed data, but it performed best when the data followed a lognormal distribution.     

Tests for homogeneity of risk differences in stratified design with correlated bilateral data

ABSTRACT

Correlated bilateral data arise from stratified studies involving paired body organs in a subject. When it is desirable to conduct inference on the scale of risk difference, one needs first to assess the assumption of homogeneity in risk differences across strata. For testing homogeneity of risk differences, we herein propose eight methods derived respectively from weighted-least-squares (WLS), the Mantel-Haenszel (MH) estimator, the WLS method in combination with inverse hyperbolic tangent transformation, and the test statistics based on their log-transformation, the modified Score test statistic and Likelihood ratio test statistic. Simulation results showed that four of the tests perform well in general, with the tests based on the WLS method and inverse hyperbolic tangent transformation always performing satisfactorily even under small sample size designs. The methods are illustrated with a dataset.     

A new stratified three-stage unrelated randomized response model for estimating a rare sensitive attribute based on the Poisson distribution

This article suggests an efficient method of estimating a rare sensitive attribute which is assumed following Poisson distribution by using three-stage unrelated randomized response model instead of the Land et al. model (2011 Land, M., S. Singh, and S. A. Sedory. 2011. Estimation of a rare sensitive attribute using poisson distribution. Statistics 46 (3):351–60. doi:10.1080/02331888.2010.524300.[Taylor &; Francis Online], [Web of Science ®] , [Google Scholar]) when the population consists of some different sized clusters and clusters selected by probability proportional to size(:pps) sampling. A rare sensitive parameter is estimated by using pps sampling and equal probability two-stage sampling when the parameter of a rare unrelated attribute is assumed to be known and unknown.

We extend this method to the case of stratified population by applying stratified pps sampling and stratified equal probability two-stage sampling. An empirical study is carried out to show the efficiency of the two proposed methods when the parameter of a rare unrelated attribute is assumed to be known and unknown.     

Simultaneous confidence interval for assessing non‐inferiority with assay sensitivity in a three‐arm trial with binary endpoints

A three‐arm trial including an experimental treatment, an active reference treatment and a placebo is often used to assess the non‐inferiority (NI) with assay sensitivity of an experimental treatment. Various hypothesis‐test‐based approaches via a fraction or pre‐specified margin have been proposed to assess the NI with assay sensitivity in a three‐arm trial. There is little work done on confidence interval in a three‐arm trial. This paper develops a hybrid approach to construct simultaneous confidence interval for assessing NI and assay sensitivity in a three‐arm trial. For comparison, we present normal‐approximation‐based and bootstrap‐resampling‐based simultaneous confidence intervals. Simulation studies evidence that the hybrid approach with the Wilson score statistic performs better than other approaches in terms of empirical coverage probability and mesial‐non‐coverage probability. An example is used to illustrate the proposed approaches.     

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.