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.     

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.     

Confidence Intervals for Survival Probabilities: A Comparison Study

The confidence interval of the Kaplan–Meier estimate of the survival probability at a fixed time point is often constructed by the Greenwood formula. This normal approximation-based method can be looked as a Wald type confidence interval for a binomial proportion, the survival probability, using the “effective” sample size defined by Cutler and Ederer. Wald-type binomial confidence interval has been shown to perform poorly comparing to other methods. We choose three methods of binomial confidence intervals for the construction of confidence interval for survival probability: Wilson's method, Agresti–Coull's method, and higher-order asymptotic likelihood method. The methods of “effective” sample size proposed by Peto et al. and Dorey and Korn are also considered. The Greenwood formula is far from satisfactory, while confidence intervals based on the three methods of binomial proportion using Cutler and Ederer's “effective” sample size have much better performance.     

Confidence Intervals for the Weighted Sum of Two Independent Binomial Proportions

Confidence intervals for the difference of two binomial proportions are well known, however, confidence intervals for the weighted sum of two binomial proportions are less studied. We develop and compare seven methods for constructing confidence intervals for the weighted sum of two independent binomial proportions. The interval estimates are constructed by inverting the Wald test, the score test and the Likelihood ratio test. The weights can be negative, so our results generalize those for the difference between two independent proportions. We provide a numerical study that shows that these confidence intervals based on large‐sample approximations perform very well, even when a relatively small amount of data is available. The intervals based on the inversion of the score test showed the best performance. Finally, we show that as for the difference of two binomial proportions, adding four pseudo‐outcomes to the Wald interval for the weighted sum of two binomial proportions improves its coverage significantly, and we provide a justification for this correction.     

A comparative study of confidence intervals for negative binomial proportion

In this paper, we investigate four existing and three new confidence interval estimators for the negative binomial proportion (i.e., proportion under inverse/negative binomial sampling). An extensive and systematic comparative study among these confidence interval estimators through Monte Carlo simulations is presented. The performance of these confidence intervals are evaluated in terms of their coverage probabilities and expected interval widths. Our simulation studies suggest that the confidence interval estimator based on saddlepoint approximation is more appealing for large coverage levels (e.g., nominal level≤1% ) whereas the score confidence interval estimator is more desirable for those commonly used coverage levels (e.g., nominal level>1% ). We illustrate these confidence interval construction methods with a real data set from a maternal congenital heart disease study.     

Approximate is Better than “Exact” for Interval Estimation of Binomial Proportions

For interval estimation of a proportion, coverage probabilities tend to be too large for “exact” confidence intervals based on inverting the binomial test and too small for the interval based on inverting the Wald large-sample normal test (i.e., sample proportion ± z-score × estimated standard error). Wilson's suggestion of inverting the related score test with null rather than estimated standard error yields coverage probabilities close to nominal confidence levels, even for very small sample sizes. The 95% score interval has similar behavior as the adjusted Wald interval obtained after adding two “successes” and two “failures” to the sample. In elementary courses, with the score and adjusted Wald methods it is unnecessary to provide students with awkward sample size guidelines.     

Comment on ‘An improved score interval with a modified midpoint for a binomial proportion’

Yu et al. [An improved score interval with a modified midpoint for a binomial proportion. J Stat Comput Simul. 2014;84:1022–1038] propose a novel confidence interval (CI) for a binomial proportion by modifying the midpoint of the score interval. This CI is competitive with the various commonly used methods. At the same time, Martín and Álvarez [Two-tailed asymptotic inferences for a proportion. J Appl Stat. 2014;41:1516–1529] analyse the performance of 29 asymptotic two-tailed CI for a proportion. The CI they selected is based on the arcsin transformation (when this is applied to the data increased by 0.5), although they also refer to the good behaviour of the classical methods of score and Agresti and Coull (which may be preferred in certain circumstances). The aim of this commentary is to compare the four methods referred to previously. The conclusion (for the classic error α of 5%) is that with a small sample size (≤80) the method that should be used is that of Yu et al.; for a large sample size (n?≥?100), the four methods perform in a similar way, with a slight advantage for the Agresti and Coull method. In any case the Agresti and Coull method does not perform badly and tends to be conservative. The program which determines these four intervals are available from the address http://www.ugr.es/local/bioest/Z_LINEAR_K.EXEhttp://www.ugr.es/local/bioest/Z_LINEAR_K.EXE.     

Some Properties of the Exact and Score Methods for Binomial Proportion and Sample Size Calculation

In this article, we point out some interesting relations between the exact test and the score test for a binomial proportion p. Based on the properties of the tests, we propose some approximate as well as exact methods of computing sample sizes required for the tests to attain a specified power. Sample sizes required for the tests are tabulated for various values of p to attain a power of 0.80 at level 0.05. We also propose approximate and exact methods of computing sample sizes needed to construct confidence intervals with a given precision. Using the proposed exact methods, sample sizes required to construct 95% confidence intervals with various precisions are tabulated for p = .05(.05).5. The approximate methods for computing sample sizes for score confidence intervals are very satisfactory and the results coincide with those of the exact methods for many cases.     

A NEW SHORT EXACT GEOMETRIC CONFIDENCE INTERVAL

For discrete distributions, the standard method of producing a two‐sided confidence interval generates an interval that is exact but conservative. This paper proposes a new algorithm to produce a short exact geometric confidence interval with proven properties, and compares the new interval with the standard interval.     

Inference for functions of parameters in discrete distributions based on fiducial approach: Binomial and Poisson cases

In this article, we propose a simple method of constructing confidence intervals for a function of binomial success probabilities and for a function of Poisson means. The method involves finding an approximate fiducial quantity (FQ) for the parameters of interest. A FQ for a function of several parameters can be obtained by substitution. For the binomial case, the fiducial approach is illustrated for constructing confidence intervals for the relative risk and the ratio of odds. Fiducial inferential procedures are also provided for estimating functions of several Poisson parameters. In particular, fiducial inferential approach is illustrated for interval estimating the ratio of two Poisson means and for a weighted sum of several Poisson means. Simple approximations to the distributions of the FQs are also given for some problems. The merits of the procedures are evaluated by comparing them with those of existing asymptotic methods with respect to coverage probabilities, and in some cases, expected widths. Comparison studies indicate that the fiducial confidence intervals are very satisfactory, and they are comparable or better than some available asymptotic methods. The fiducial method is easy to use and is applicable to find confidence intervals for many commonly used summary indices. Some examples are used to illustrate and compare the results of fiducial approach with those of other available asymptotic methods.     

Applications of asymptotic confidence intervals with continuity corrections for asymmetric comparisons in noninferiority trials

A large‐sample problem of illustrating noninferiority of an experimental treatment over a referent treatment for binary outcomes is considered. The methods of illustrating noninferiority involve constructing the lower two‐sided confidence bound for the difference between binomial proportions corresponding to the experimental and referent treatments and comparing it with the negative value of the noninferiority margin. The three considered methods, Anbar, Falk–Koch, and Reduced Falk–Koch, handle the comparison in an asymmetric way, that is, only the referent proportion out of the two, experimental and referent, is directly involved in the expression for the variance of the difference between two sample proportions. Five continuity corrections (including zero) are considered with respect to each approach. The key properties of the corresponding methods are evaluated via simulations. First, the uncorrected two‐sided confidence intervals can, potentially, have smaller coverage probability than the nominal level even for moderately large sample sizes, for example, 150 per group. Next, the 15 testing methods are discussed in terms of their Type I error rate and power. In the settings with a relatively small referent proportion (about 0.4 or smaller), the Anbar approach with Yates’ continuity correction is recommended for balanced designs and the Falk–Koch method with Yates’ correction is recommended for unbalanced designs. For relatively moderate (about 0.6) and large (about 0.8 or greater) referent proportion, the uncorrected Reduced Falk–Koch method is recommended, although in this case, all methods tend to be over‐conservative. These results are expected to be used in the design stage of a noninferiority study when asymmetric comparisons are envisioned. Copyright © 2013 John Wiley & Sons, Ltd.     

The monotone boundary property and the full coverage property of confidence intervals for a binomial proportion

The methodology for deriving the exact confidence coefficient of some confidence intervals for a binomial proportion is proposed in Wang [2007. Exact confidence coefficients of confidence intervals for a binomial proportion. Statist. Sinica 17, 361–368]. The methodology requires two conditions of confidence intervals: the monotone boundary property and the full coverage property. In this paper, we show that for some confidence intervals of a binomial proportion, the two properties hold for any sample size. Based on results presented in this paper, the procedure in Wang [2007. Exact confidence coefficients of confidence intervals for a binomial proportion. Statist. Sinica 17, 361–368] can be directly used to calculate the exact confidence coefficients of these confidence intervals for any fixed sample size.     

Matching pseudocounts for interval estimation of binomial and Poisson parameters

ABSTRACT

For interval estimation of a binomial proportion and a Poisson mean, matching pseudocounts are derived, which give the one-sided Wald confidence intervals with second-order accuracy. The confidence intervals remove the bias of coverage probabilities given by the score confidence intervals. Partial poor behavior of the confidence intervals by the matching pseudocounts is corrected by hybrid methods using the score confidence interval depending on sample values.     

Confidence regions for two proportions from independent negative binomial distributions

The negative binomial distribution offers an alternative view to the binomial distribution for modeling count data. This alternative view is particularly useful when the probability of success is very small, because, unlike the fixed sampling scheme of the binomial distribution, the inverse sampling approach allows one to collect enough data in order to adequately estimate the proportion of success. However, despite work that has been done on the joint estimation of two binomial proportions from independent samples, there is little, if any, similar work for negative binomial proportions. In this paper, we construct and investigate three confidence regions for two negative binomial proportions based on three statistics: the Wald (W), score (S) and likelihood ratio (LR) statistics. For large-to-moderate sample sizes, this paper finds that all three regions have good coverage properties, with comparable average areas for large sample sizes but with the S method producing the smaller regions for moderate sample sizes. In the small sample case, the LR method has good coverage properties, but often at the expense of comparatively larger areas. Finally, we apply these three regions to some real data for the joint estimation of liver damage rates in patients taking one of two drugs.     

Coverage‐adjusted Confidence Intervals for a Binomial Proportion

We consider the classic problem of interval estimation of a proportion p based on binomial sampling. The ‘exact’ Clopper–Pearson confidence interval for p is known to be unnecessarily conservative. We propose coverage adjustments of the Clopper–Pearson interval that incorporate prior or posterior beliefs into the interval. Using heatmap‐type plots for comparing confidence intervals, we show that the coverage‐adjusted intervals have satisfying coverage and shorter expected lengths than competing intervals found in the literature.     

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.     

Confidence curves and improved exact confidence intervals for discrete distributions

The author describes a method for improving standard “exact” confidence intervals in discrete distributions with respect to size while retaining correct level. The binomial, negative binomial, hypergeometric, and Poisson distributions are considered explicitly. Contrary to other existing methods, the author's solution possesses a natural nesting condition: if α < α', the 1 ‐ α' confidence interval is included in the 1 ‐ α interval. Nonparametric confidence intervals for a quantile are also considered.     

A confidence region with multiple intervals in multivariate bioassay

The exact confidence region for log relative potency resulting from likelihood score methods (Williams (1988) An exact confidence interval for the relative potency estimated from a multivariate bioassay, Biometrics, 44:861-868) will very likely consist of two disjoint confidence intervals. The two methods proposed by Williams which aim to select just one (the same) confidence interval from the confidence region are nearly – but not completely – consistent. The likelihood score interval and likelihood ratio interval are asymptotically equivalent. Williams's very strong claim concerning the confidence coefficient in the second selection method is still theoretically unproved; yet, simulations show that it is true for a wide range of practical experimental situations.     

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.     

Confidence interval estimation for negative binomial group distribution

Negative binomial group distribution was proposed in the literature which was motivated by inverse sampling when considering group inspection: products are inspected group by group, and the number of non-conforming items of a group is recorded only until the inspection of the whole group is finished. The non-conforming probability p of the population is thus the parameter of interest. In this paper, the confidence interval construction for this parameter is investigated. The common normal approximation and exact method are applied. To overcome the drawbacks of these commonly used methods, a composite method that is based on the confidence intervals of the negative binomial distribution is proposed, which benefits from the relationship between negative binomial distribution and negative binomial group distribution. Simulation studies are carried out to examine the performances of our methods. A real data example is also presented to illustrate the application of our method.