{"title":"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。","authors":"Yuansong Zhao, Peng Yang, Glen Laird, Josh Chen, Ying Yuan","doi":"10.1080/10543406.2025.2489284","DOIUrl":null,"url":null,"abstract":"<p><p>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\".</p>","PeriodicalId":54870,"journal":{"name":"Journal of Biopharmaceutical Statistics","volume":" ","pages":"1-16"},"PeriodicalIF":1.2000,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"PS-SAM: propensity-score-integrated self-adapting mixture prior to dynamically and efficiently borrow information from historical data.\",\"authors\":\"Yuansong Zhao, Peng Yang, Glen Laird, Josh Chen, Ying Yuan\",\"doi\":\"10.1080/10543406.2025.2489284\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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\\\".</p>\",\"PeriodicalId\":54870,\"journal\":{\"name\":\"Journal of Biopharmaceutical Statistics\",\"volume\":\" \",\"pages\":\"1-16\"},\"PeriodicalIF\":1.2000,\"publicationDate\":\"2025-04-17\",\"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/10.1080/10543406.2025.2489284\",\"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/10.1080/10543406.2025.2489284","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
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".
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.