Volume 22, Issue 3 pp. 492-507
MAIN PAPER

An improved score-type confidence interval for stratified risk differences involving rare events

Yetao Hu

Corresponding Author

Yetao Hu

Stony Brook University, Applied Math and Statistics, Stony Brook, New York, USA

Correspondence

Yetao Hu, Stony Brook University, Applied Math and Statistics, John S. Toll Drive, Stony Brook, New York, USA.

Email: [email protected]

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Kaifeng Lu

Kaifeng Lu

Global Statistics and Data Science, BeiGene US, San Mateo, California, USA

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Lei Xie

Lei Xie

SPARC Inc, Princeton, New Jersey, USA

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Wei Zhu

Wei Zhu

Stony Brook University, Applied Math and Statistics, Stony Brook, New York, USA

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First published: 30 December 2022

Abstract

A stratified analysis of the differences in proportions has been widely employed in epidemiological research, social sciences, and drug development. It provides a useful framework for combining data across strata to produce a common effect. However, for rare events with incidence rates close to zero, popular confidence intervals for risk differences in a stratified analysis may not have appropriate coverage probabilities that approach the nominal confidence levels and the algorithms may fail to produce a valid confidence interval because of zero events in both the arms of a stratum. The main objective of this study is to evaluate the performance of certain methods commonly employed to construct confidence intervals for stratified risk differences when the response probabilities are close to a boundary value of zero or one. Additionally, we propose an improved stratified Miettinen–Nurminen confidence interval that exhibits a superior performance over standard methods while avoiding computational difficulties involving rare events. The proposed method can also be employed when the response probabilities are close to one.

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