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Interval estimation of binomial proportion in clinical trials with a two‐stage design
Author(s) -
Tsai WeiYann,
Chi Yunchan,
Chen ChiaMin
Publication year - 2007
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.2930
Subject(s) - binomial proportion confidence interval , statistics , mathematics , binomial distribution , estimator , interval estimation , confidence interval , interval (graph theory) , negative binomial distribution , continuity correction , binomial (polynomial) , tolerance interval , beta binomial distribution , combinatorics , poisson distribution
Generally, a two‐stage design is employed in Phase II clinical trials to avoid giving patients an ineffective drug. If the number of patients with significant improvement, which is a binomial response, is greater than a pre‐specified value at the first stage, then another binomial response at the second stage is also observed. This paper considers interval estimation of the response probability when the second stage is allowed to continue. Two asymptotic interval estimators, Wald and score, as well as two exact interval estimators, Clopper–Pearson and Sterne, are constructed according to the two binomial responses from this two‐stage design, where the binomial response at the first stage follows a truncated binomial distribution. The mean actual coverage probability and expected interval width are employed to evaluate the performance of these interval estimators. According to the comparison results, the score interval is recommended for both Simon's optimal and minimax designs. Copyright © 2007 John Wiley & Sons,Ltd.