Analysis of two binomial proportions in noninferiority confirmatory trials.
binomial distribution
confidence interval
confirmatory trials
difference in proportions
noninferiority
Journal
Pharmaceutical statistics
ISSN: 1539-1612
Titre abrégé: Pharm Stat
Pays: England
ID NLM: 101201192
Informations de publication
Date de publication:
11 Dec 2023
11 Dec 2023
Historique:
revised:
24
09
2023
received:
05
04
2022
accepted:
13
11
2023
medline:
12
12
2023
pubmed:
12
12
2023
entrez:
12
12
2023
Statut:
aheadofprint
Résumé
In this article, we propose considering an approximate exact score (AES) test for noninferiority comparisons and we derive its test-based confidence interval for the difference between two independent binomial proportions. This test was published in the literature, but not its associated confidence interval. The p-value for this test is obtained by using exact binomial probabilities with the nuisance parameter being replaced by its restricted maximum likelihood estimate. Calculated type I errors revealed that the AES method has important advantages for noninferiority comparisons over popular asymptotic methods for adequately powered confirmatory clinical trials, at 80% or 90% statistical power. For unbalanced sample sizes of the compared groups, type I errors for the asymptotic score method were shown to be higher than the nominal level in a systematic pattern over a range of true proportions, but the AES method did not suffer from such a problem. On average, the true type I error of the AES method was closer to the nominal level than all considered methods in the empirical comparisons. In rare cases, type I errors of the AES test exceeded the nominal level, but only by a small amount. Presented examples showed that the AES method can be more attractive in practice than practical exact methods. In addition, p-value and confidence interval of the AES method can be obtained in <30 s of computer time for most confirmatory trials. Theoretical arguments, combined with empirical evidence and fast computation time should make the AES method attractive in statistical practice.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2023 John Wiley & Sons Ltd.
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