Robustness of sample size re-estimation with interim binary data for double-blind clinical trials. (English) Zbl 1058.62100
Summary: A central question that arises in clinical trials is how many additional observations, if any, are needed beyond those originally planned? Earlier contributions considered continuous response that assumes a normal distribution. Some treat the portion up to the interim stage as an ‘internal pilot study’ and thus require unblinding. In this paper, for a certain randomized design with interim binary data for double blind clinical trials, we obtain closed-form expressions for the effective level of significance and operating power at the specified alternative. Also considered is the totally unblinded case. Numerical examples indicate a significant increase in the level of significance in the case of the blinded design which is contrary to what W. J. Shih and P. L. Zhao [Stat. Med. 16, 1913–1932 (1997)] have claimed. However, the type I error probability is under control in the unblinded case.
MSC:
| 62P10 | Applications of statistics to biology and medical sciences; meta analysis |
| 62D05 | Sampling theory, sample surveys |
Keywords:
Robustness; Clinical trials; Double-blind experiments; Binary outcomes; Sample size re-estimation; randomized responseReferences:
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