Conducting a pre-confirmatory clinical trial in case of insufficient participant accrual for an investigator-initiated confirmatory clinical trial

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  • Hagihara Aya
    Division of biostatistics Department of Social/Community Medicine and Health Science, Kobe University School of Medicine
  • Omori Takashi
    Department of Clinical Biostatistics, Graduate School of Medicine, Kyoto University

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Other Title
  • 研究者主導試験におけるプレ検証試験の実施−不十分な登録数で終了する検証試験を防ぐために−

Abstract

<p>Conducting confirmatory clinical trials initiated and managed by nonpharmaceutical company researchers is often unfeasible because the calculated sample size is excessively large. We propose an alternative approach: conducting a pre-confirmatory clinical trial with a relatively high type I error rate and limited sample size. Our approach involves the use of a significance level calculated based on the conditional probability (P2), which is the probability of having a true treatment effect, given that the statistical test shows statistical significance, and its application to sample size calculation for pre-confirmatory clinical trials.</p><p>We examined the performance and operational characteristics of our approach using a case study. For a confirmatory clinical trial that required 100 participants in each group, when our method was applied, a pre-confirmatory clinical trial required a considerably small number of participants. For example, for a P2 of 80 %, with no prior information on the treatment effect, and a power of 80 %, the significance level of our approach was calculated as 20 %. Based on these values, the sample size in each group was 52. Although insufficient participant accrual during confirmatory clinical trials may affect the conclusion of the trials, our approach can ameliorate the situation.</p>

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