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

Abstract

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.

Keywords:

• Bootstrap-resampling method
• combined unilateral and bilateral data
• confidence interval
• intra-class correlation
• proportion difference

Disclosure statement

No potential conflict of interest was reported by the author(s).

Additional information

Funding

The work of Qiu was supported by the National Natural Science Foundation of China [Grant Nos. 11871124, 11471060] and the Natural Science Foundation of Chongqing [Grant No. cstc2018jcyjAX0241, cstc2020jcyj-msxmX0232].