{"title":"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。","authors":"Jinmei Chen, Guoyou Qin, Yongfu Yu","doi":"10.1080/10543406.2025.2547593","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1-14"},"PeriodicalIF":1.2000,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Mitigating propensity score model misspecification with multiply robust weights when leveraging external data.\",\"authors\":\"Jinmei Chen, Guoyou Qin, Yongfu Yu\",\"doi\":\"10.1080/10543406.2025.2547593\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":54870,\"journal\":{\"name\":\"Journal of Biopharmaceutical Statistics\",\"volume\":\" \",\"pages\":\"1-14\"},\"PeriodicalIF\":1.2000,\"publicationDate\":\"2025-08-28\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Biopharmaceutical Statistics\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/10543406.2025.2547593\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Biopharmaceutical Statistics","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/10543406.2025.2547593","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
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.
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.