Exact method of sample size calculation and statistical inference in single-arm clinical trials with objective performance criteria

Abstract

Abstract: AIM: To explore the exact method of sample size estimation and statistical inference in single-arm clinical trials with Objective Performance Criteria (OPC) based on binomial distribution principle.METHODS: This article will systematically and comprehensively discuss the exact method of sample size calculation, confidence interval estimation and hypothesis testing which the single-arm clinical trial method involves in.RESULTS: With the starting point of OPC at 75% by an interval of 1% increasing and the starting point of expected event rate at 76% by an interval of 1% increasing, corresponding exact sample sizes are calculated. And two sets of results under the condition of β of 0.20 and α of 0.05 (one sided and two sided) are tabulated respectively. The exact calculating formulas of confidence interval estimation and hypothesis testing are provided.CONCLUSION: Systematic methodology support is provided for OPC method in single-arm clinical trials in calculating sample size, estimating confidence interval and hypothesis testing.

Key words: Single-arm clinical trial, Objective Performance Criteria, Exact method, Sample size, Confidence interval, Hypothesis testing

CLC Number:

R311

CHENG Qi, LIU Yu-xiu, CHEN Lin, LIU Li-xia. Exact method of sample size calculation and statistical inference in single-arm clinical trials with objective performance criteria[J]. Chinese Journal of Clinical Pharmacology and Therapeutics, 2011, 16(5): 517-522.

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