Nemes Szilárd, Gustavsson Andreas, Jauhiainen Alexandra
BioPharma Early Biometrics and Statistical Innovation, Data Science & AI, BioPharmaceuticals R&D, AstraZeneca, 43183 Gothenburg, Sweden.
Entropy (Basel). 2022 May 16;24(5):713. doi: 10.3390/e24050713.
Restricted Mean Survival Time (RMST), the average time without an event of interest until a specific time point, is a model-free, easy to interpret statistic. The heavy reliance on non-parametric or semi-parametric methods in the survival analysis has drawn criticism, due to the loss of efficacy compared to parametric methods. This assumes that the parametric family used is the true one, otherwise the gain in efficacy might be lost to interpretability problems due to bias. The Focused Information Criterion (FIC) considers the trade-off between bias and variance and offers an objective framework for the selection of the optimal non-parametric or parametric estimator for scalar statistics. Herein, we present the FIC framework for the selection of the RMST estimator with the best bias-variance trade-off. The aim is not to identify the true underling distribution that generated the data, but to identify families of distributions that best approximate this process. Through simulation studies and theoretical reasoning, we highlight the effect of censoring on the performance of FIC. Applicability is illustrated with a real life example. Censoring has a non-linear effect on FICs performance that can be traced back to the asymptotic relative efficiency of the estimators. FICs performance is sample size dependent; however, with censoring percentages common in practical applications FIC selects the true model at a nominal probability (0.843) even with small or moderate sample sizes.
受限平均生存时间(RMST)是指直到某个特定时间点无感兴趣事件发生的平均时间,它是一种无模型且易于解释的统计量。生存分析中对非参数或半参数方法的严重依赖受到了批评,因为与参数方法相比其效能有所损失。这假定所使用的参数族是真实的参数族,否则由于偏差,效能的提升可能会因可解释性问题而丧失。聚焦信息准则(FIC)考虑了偏差和方差之间的权衡,并为选择标量统计量的最优非参数或参数估计量提供了一个客观框架。在此,我们提出用于选择具有最佳偏差 - 方差权衡的RMST估计量的FIC框架。目的不是识别生成数据的真实潜在分布,而是识别最能近似此过程的分布族。通过模拟研究和理论推理,我们突出了删失对FIC性能的影响。通过一个实际例子说明了其适用性。删失对FIC性能有非线性影响,这可以追溯到估计量的渐近相对效率。FIC的性能取决于样本量;然而,在实际应用中常见的删失百分比情况下,即使样本量较小或适中,FIC也能以标称概率(0.843)选择真实模型。