Gaohong Dong, Ying Cui, Margaret Gamalo-Siebers, Ran Liao, Dacheng Liu, David C Hoaglin, Ying Lu
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引用次数: 0
摘要
Dong 等人(2023 年)的研究表明,胜出统计量(胜出率、胜出几率和净收益)可以互为补充,在优先考虑多种结果的随机试验中证明治疗效果的强度。这一结果建立在三个统计量的点和方差估计值之间的联系以及统计检验中 Z 值的近似相等之上。然而,这一近似值的影响并不明确。本讨论完善了这一方法,并表明赢家统计的 Z 值近似相等在更大范围内成立。因此,三种胜出统计量都能得出非常接近的 p 值。此外,我们的模拟还举例说明,不对删减偏倚进行调整的天真方法可能会得出与真实结果完全相反的结论,而 IPCW(反删减概率加权)方法可以有效地将胜出统计量调整为相应的真实值(即 IPCW 调整后的胜出统计量是无偏的治疗效果估计值)。
On approximate equality of Z-values of the statistical tests for win statistics (win ratio, win odds, and net benefit).
Dong et al. (2023) showed that the win statistics (win ratio, win odds, and net benefit) can complement each another to demonstrate the strength of treatment effects in randomized trials with prioritized multiple outcomes. This result was built on the connections among the point and variance estimates of the three statistics, and the approximate equality of Z-values in their statistical tests. However, the impact of this approximation was not clear. This Discussion refines this approach and shows that the approximate equality of Z-values for the win statistics holds more generally. Thus, the three win statistics consistently yield closely similar p-values. In addition, our simulations show an example that the naive approach without adjustment for censoring bias may produce a completely opposite conclusion from the true results, whereas the IPCW (inverse-probability-of-censoring weighting) approach can effectively adjust the win statistics to the corresponding true values (i.e. IPCW-adjusted win statistics are unbiased estimators of treatment effect).
期刊介绍:
The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers:
Drug, device, and biological research and development;
Drug screening and drug design;
Assessment of pharmacological activity;
Pharmaceutical formulation and scale-up;
Preclinical safety assessment;
Bioavailability, bioequivalence, and pharmacokinetics;
Phase, I, II, and III clinical development including complex innovative designs;
Premarket approval assessment of clinical safety;
Postmarketing surveillance;
Big data and artificial intelligence and applications.