随访时间不等的非劣效性和等效性试验中负二项分布率比较的样本量
Sample size for comparing negative binomial rates in noninferiority and equivalence trials with unequal follow-up times
摘要：
我们推导了在随访时间不等的非劣效性和等效性试验中基于相对和绝对率差指标比较两个负二项分布率的样本量公式，并建立了基于两个处理效应指标的治疗比较所需样本量之间的近似关系。所提出的方法允许分散参数根据治疗组而变化。这些方法的准确性进行了评估模拟。结果表明，通过将所有个体的随访时间设置为平均随访时间来忽略受试者之间的随访时间差异，可能会大大低估所需的样本量，导致研究效力不足。提供了基于已公布的汇总结果反算色散参数的方法。
英文摘要：
We derive the sample size formulae for comparing two negative binomial rates based on both the relative and absolute rate difference metrics in noninferiority and equivalence trials with unequal follow-up times, and establish an approximate relationship between the sample sizes required for the treatment comparison based on the two treatment effect metrics. The proposed method allows the dispersion parameter to vary by treatment groups. The accuracy of these methods is assessed by simulations. It is demonstrated that ignoring the between-subject variation in the follow-up time by setting the follow-up time for all individuals to be the mean follow-up time may greatly underestimate the required size, resulting in underpowered studies. Methods are provided for back-calculating the dispersion parameter based on the published summary results.
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