PS-SAM:倾向-分数集成自适应混合,动态有效地从历史数据中获取信息。

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY
Yuansong Zhao, Peng Yang, Glen Laird, Josh Chen, Ying Yuan
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引用次数: 0

摘要

人们对纳入历史数据以提高随机对照试验(rct)的效率或减少所需样本量的兴趣越来越大。一个关键的挑战是,历史数据的患者特征可能与当前的RCT不同。为了解决这个问题,一种众所周知的方法是采用倾向得分匹配或逆概率加权来调整基线异质性,从而将历史数据纳入RCT的推断中。然而,当存在无法测量的混杂因素时,这种方法容易产生偏差。我们通过将自适应混合(SAM)先验与倾向得分匹配和逆概率加权相结合来解决这个问题,以便在存在未测量混杂因素的情况下对信息借用进行额外的适应。由此产生的倾向得分整合SAM (PS-SAM)先验是稳健的,因为如果没有未测量的混杂因素,它们会导致对治疗效果的无偏因果估计;如果存在未测量的混杂因素,它们会提供明显更少的偏倚治疗效果和更好地控制I型误差。仿真研究表明,PS-SAM先验具有良好的操作特性,能够实现自适应信息借用。建议的方法可以作为R包“SAMprior”免费获得。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
PS-SAM: propensity-score-integrated self-adapting mixture prior to dynamically and efficiently borrow information from historical data.

There has been growing interest in incorporating historical data to improve the efficiency of randomized controlled trials (RCTs) or reduce their required sample size. A key challenge is that the patient characteristics of the historical data may differ from those of the current RCT. To address this issue, a well-known approach is to employ propensity score matching or inverse probability weighting to adjust for baseline heterogeneity, enabling the incorporation of historical data into the inference of RCT. However, this approach is subject to bias when there are unmeasured confounders. We address this issue by incorporating a self-adapting mixture (SAM) prior with propensity score matching and inverse probability weighting to enable additional adaptation for information borrowing in the presence of unmeasured confounders. The resulting propensity score-integrated SAM (PS-SAM) priors are robust in the sense that if there are no unmeasured confounders, they result in an unbiased causal estimate of the treatment effect; and if there are unmeasured confounders, they provide a notably less biased treatment effect with better-controlled type I error. Simulation studies demonstrate that the PS-SAM prior exhibits desirable operating characteristics enabling adaptive information borrowing. The proposed methodology is freely available as the R package "SAMprior".

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来源期刊
Journal of Biopharmaceutical Statistics
Journal of Biopharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.50
自引率
18.20%
发文量
71
审稿时长
6-12 weeks
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.
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