利用外部数据时,使用多个鲁棒权重减轻倾向评分模型的错误说明。

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY
Jinmei Chen, Guoyou Qin, Yongfu Yu
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引用次数: 0

摘要

倾向得分整合贝叶斯动态借用方法在使用外部数据增强随机对照试验(rct)时提供了有效的协变量调整方法。然而,由于未知的治疗选择过程,确定正确的倾向评分模型可能具有挑战性,可能导致模型规格错误和有偏差的估计。为了提高对模型错误规范的鲁棒性,我们提出了一种创新的贝叶斯推理过程,该过程将多个鲁棒权值纳入信息功率先验的构造中。具体来说,我们指定了一组候选倾向评分模型来导出多个稳健权重,平衡当前数据和外部数据之间的协变量。然后使用贝叶斯幂先验方法将加权的外部数据纳入分析。我们进一步扩展这种方法来利用多个外部数据集。仿真研究表明,当假设的倾向评分模型集包含一个正确指定的模型时,所提出的方法实现了低偏差、低均方根误差(RMSE)、将第一类错误率控制在预定的名义水平和高统计功率等理想的操作特性。这种方法也为研究人员提供了一个强大的策略,谁可能有困难的时间发展或选择单一倾向评分模型。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Mitigating propensity score model misspecification with multiply robust weights when leveraging external data.

Propensity score-integrated Bayesian dynamic borrowing methods offer an effective approach for covariate adjustment when using external data to augment randomized controlled trials (RCTs). However, identifying the correct propensity score model can be challenging due to unknown treatment selection processes, potentially leading to model misspecification and biased estimates. To improve robustness to model misspecification, we propose an innovative Bayesian inference procedure that incorporates multiply robust weights into the construction of informative power priors. Specifically, we specify a set of candidate propensity score models to derive multiply robust weights, balancing covariates between the current data and external data. The weighted external data is then incorporated into the analysis using a Bayesian power prior method. We further extend this approach to leverage multiple external datasets. Simulation studies indicate that when the set of postulated propensity score models include a correctly specified model, the proposed method achieves desirable operating characteristics, including low bias, low root mean squared error (RMSE), controlled type I error rate at the predetermined nominal level, and high statistical power. This method also provides a robust strategy for researchers who may have a difficult time developing or selecting a single propensity score model.

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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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