Leveraging external evidence using Bayesian hierarchical model and propensity score in the presence of covariates

Contemporary Clinical Trials - Tập 132 - Trang 107301 - 2023
Xiaotian Chen1, Yi Yao1, Li Wang1, Saurabh Mukhopadhyay1
1Data and Statistical Sciences, AbbVie Inc., 1 N Waukegan Rd, North Chicago, IL 60064, USA

Tài liệu tham khảo

Pocock, 1976, The combination of randomized and historical controls in clinical trials, J. Chronic Dis., 29, 175, 10.1016/0021-9681(76)90044-8 Lim, 2020, Reducing patient burden in clinical trials through the use of historical controls: appropriate selection of historical data to minimize risk of bias, Ther. Innov. Regul. Sci., 54, 850, 10.1007/s43441-019-00014-4 Rosenbaum, 1983, The central role of the propensity score in observational studies for causal effects, Biometrika, 70, 41, 10.1093/biomet/70.1.41 Wang, 2019, Propensity score-integrated power prior approach for incorporating real-world evidence in single-arm clinical studies, J. Biopharm. Stat., 29, 731, 10.1080/10543406.2019.1657133 Chen, 2020, Propensity score-integrated composite likelihood approach for augmenting the control arm of a randomized controlled trial by incorporating real-world data, J. Biopharm. Stat., 30, 508, 10.1080/10543406.2020.1730877 Wang, 2020, Propensity score-integrated composite likelihood approach for incorporating real-world evidence in single-arm clinical studies, J. Biopharm. Stat., 30, 495, 10.1080/10543406.2019.1684309 Yuan, 2019, Design of randomized controlled confirmatory trials using historical control data to augment sample size for concurrent controls, J. Biopharm. Stat., 29, 558, 10.1080/10543406.2018.1559853 Lin, 2018, Propensity score matched augmented controls in randomized clinical trials: a case study, Pharm. Stat., 17, 629 Xu, 2020, A study design for augmenting the control group in a randomized controlled trial: a quality process for interaction among stakeholders, Ther. Innov. Regul. Sci., 54, 269, 10.1007/s43441-019-00053-x Lin, 2021, Incorporating propensity scores for evidence synthesis under bayesian framework: review and recommendations for clinical studies, J. Biopharm. Stat., 1 Liu, 2021, Propensity-score-based meta-analytic predictive prior for incorporating real-world and historical data, Stat. Med, 40, 4794, 10.1002/sim.9095 Neuenschwander, 2010, Summarizing historical information on controls in clinical trials, Clin. Trials, 7, 5, 10.1177/1740774509356002 Ibrahim, 2000, Power prior distributions for regression models, Stat. Sci., 46 Duan, 2006, Evaluating water quality using power priors to incorporate historical information, Environmetrics, 17, 95, 10.1002/env.752 Neuenschwander, 2009, A note on the power prior, Stat. Med., 28, 3562, 10.1002/sim.3722 Hobbs, 2011, Hierarchical commensurate and power prior models for adaptive incorporation of historical information in clinical trials, Biometrics, 67, 1047, 10.1111/j.1541-0420.2011.01564.x Han, 2017, Covariate-adjusted borrowing of historical control data in randomized clinical trials, Pharm. Stat., 16, 296, 10.1002/pst.1815 Fu, 2023, Covariate handling approaches in combination with dynamic borrowing for hybrid control studies, Pharm. Stat., 22, 585, 10.1002/pst.2297 Pirracchio, 2015, Improving propensity score estimators’ robustness to model misspecification using super learner, Am. J. Epidemiol., 181, 108, 10.1093/aje/kwu253 Lee, 2010, Improving propensity score weighting using machine learning, Stat. Med., 29, 337, 10.1002/sim.3782 Austin, 2011, An introduction to propensity score methods for reducing the effects of confounding in observational studies, Multivar. Behav. Res., 46, 399, 10.1080/00273171.2011.568786 Rosenbaum, 1987, Model-based direct adjustment, J. Am. Stat. Assoc., 82, 387, 10.1080/01621459.1987.10478441 Lunceford, 2004, Stratification and weighting via the propensity score in estimation of causal treatment effects: a comparative study, Stat. Med., 23, 2937, 10.1002/sim.1903 McCandless, 2009, Bayesian propensity score analysis for observational data, Stat. Med., 28, 94, 10.1002/sim.3460 Spanakis, 2022, Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing, J. Biopharm. Stat., 1, 10.1080/10543406.2022.2152833 Lin, 2022, Matching within a hybrid RCT/RWD: framework on associated causal estimands, J. Biopharm. Stat., 1, 10.1080/10543406.2022.2036992 Inman, 1989, The overlapping coefficient as a measure of agreement between probability distributions and point estimation of the overlap of two normal densities, Commun. Stat. Theor. Methods, 18, 3851, 10.1080/03610928908830127 Schmidli, 2014, Robust meta-analytic-predictive priors in clinical trials with historical control information, Biometrics, 70, 1023, 10.1111/biom.12242