Clinical Research Facility, National University of Ireland Galway, Galway, Ireland
BMJ Evid Based Med. 2022 Oct;27(5):313-316. doi: 10.1136/bmjebm-2020-111603. Epub 2021 Sep 23.
Commonly accepted statistical advice dictates that large-sample size and highly powered clinical trials generate more reliable evidence than trials with smaller sample sizes. This advice is generally sound: treatment effect estimates from larger trials tend to be more accurate, as witnessed by tighter confidence intervals in addition to reduced publication biases. Consider then two clinical trials testing the same treatment which result in the same p values, the trials being identical apart from differences in sample size. Assuming statistical significance, one might at first suspect that the larger trial offers stronger evidence that the treatment in question is truly effective. Yet, often precisely the opposite will be true. Here, we illustrate and explain this somewhat counterintuitive result and suggest some ramifications regarding interpretation and analysis of clinical trial results.
通常被认可的统计学建议指出,大样本量和高功效的临床试验比小样本量的试验能产生更可靠的证据。这条建议通常是合理的:大试验的治疗效果估计往往更准确,这一点可以从更窄的置信区间以及减少的发表偏倚得到证明。现在考虑两项临床试验,它们检验了相同的治疗方法,得到了相同的 p 值,除了样本量的差异外,这两项试验完全相同。在假设统计学意义的情况下,人们最初可能会怀疑较大的试验提供了更强的证据,表明所讨论的治疗方法确实有效。然而,通常情况恰恰相反。在这里,我们举例说明了这一有些违反直觉的结果,并对临床试验结果的解释和分析提出了一些影响。