基于逆概率加权的目标种群方差估计。

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY Journal of Biopharmaceutical Statistics Pub Date : 2024-08-01 Epub Date: 2023-08-24 DOI:10.1080/10543406.2023.2244593
Jinmei Chen, Rui Chen, Yuhao Feng, Ming Tan, Pingyan Chen, Ying Wu
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引用次数: 0

摘要

在观察性研究中,逆概率加权(IPW)经常用于减少或最小化观察到的混杂因素。IPW通过用接收他/她实际接受的治疗水平的条件概率的倒数对每个个体进行加权来创建伪样本。在伪样本中,通过对原始样本中的同一个体进行加权而产生的多个个体之间没有变化。这将减少数据的可变性,从而使目标人群中的方差估计存在偏差。IPW估计量的传统方差估计方法通常忽略这种低估,并倾向于产生有偏差的方差估计。我们在这里提出了一种更合理的方法,通过使用基于地层内变异性估计的参数自举,将这种变异性来源纳入其中。该方法首先使用倾向得分分层和层内标准差来近似基于倾向得分位于相应层内的单个个体生成的多个个体之间的可变性。然后,通过在原始数据中添加随机误差项后重新生成结果,使用参数自举来合并目标可变性。在仿真部分,将所提出的方法的性能与现有的三种方法进行了比较,包括基于模型的天真方差估计、非参数自举方差估计和稳健方差估计。以少肌症患者为例说明所提出方法的实施。根据结果,所提出的方法具有期望的统计特性,并且可以使用所提供的R码来容易地实现。
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On variance estimation of target population created by inverse probability weighting.

Inverse probability weighting (IPW) is frequently used to reduce or minimize the observed confounding in observational studies. IPW creates a pseudo-sample by weighting each individual by the inverse of the conditional probability of receiving the treatment level that he/she has actually received. In the pseudo-sample there is no variation among the multiple individuals generated by weighting the same individual in the original sample. This would reduce the variability of the data and therefore bias the variance estimate in the target population. Conventional variance estimation methods for IPW estimators generally ignore this underestimation and tend to produce biased estimates of variance. We here propose a more reasonable method that incorporates this source of variability by using parametric bootstrapping based on intra-stratum variability estimates. This approach firstly uses propensity score stratification and intra-stratum standard deviation to approximate the variability among multiple individuals generated based on a single individual whose propensity score falls within the corresponding stratum. The parametric bootstrapping is then used to incorporate the target variability by re-generating outcomes after adding a random error term to the original data. The performance of the proposed method is compared with three existing methods including the naïve model-based variance estimator, the nonparametric bootstrap variance estimator, and the robust variance estimator in the simulation section. An example of patients with sarcopenia is used to illustrate the implementation of the proposed approach. According to the results, the proposed approach has desirable statistical properties and can be easily implemented using the provided R code.

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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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