Tobias Polak

Augmenting treatment arms with external data through propensity-score weighted power-priors with an application in expanded access 213 9♥ 20. Neuenschwander B, Capkun-Niggli G, Branson M, Spiegelhalter DJ. Summarizing historical information on controls in clinical trials. Clin Trials. 2010;7(1):5-18. doi:10.1177/1740774509356002 21. Wang C, Li H, Chen WC, et al. Propensity score-integrated power prior approach for incorporating real-world evidence in singlearm clinical studies incorporating real-world evidence in single-arm clinical studies. J Biopharm Stat. 2019;00(00):1-18. doi:10.1080/1 0543406.2019.1657133 22. Wang C, Rosner GL. A Bayesian nonparametric causal inference model for synthesizing randomized clinical trial and real-world evidence. Stat Med. 2019;38(14):2573-2588. doi:10.1002/sim.8134 23. Lin J, Gamalo-Siebers M, Tiwari R. Propensity score matched augmented controls in randomized clinical trials: A case study. Pharm Stat. Published online July 2018. doi:10.1002/ pst.1879 24. Chen WC, Wang C, Li H, et al. Propensity scoreintegrated composite likelihood approach for augmenting the control arm of a randomized controlled trial by incorporating real- world data. J Biopharm Stat. 2020;30(3):508-520. doi: 10.1080/10543406.2020.1730877 25. Wang X, Suttner L, Jemielita T, Li X. Propensity score-integrated Bayesian prior approaches for augmented control designs: a simulation study. J Biopharm Stat. 2022;32(1):170-190. doi:10.108 0/10543406.2021.2011743 26. Reagan-Udall Foundation. Leveraging RealWorld Treatment Experience from Expanded Access Protocols. Reagan Udall Foundation; 2018:1-26. 27. Polak TB, van Rosmalen J, Uyl – De Groot CA, UylDe Groot CA. Response to Open Peer Commentary “Making It Count: Extracting Real World Data from Compassionate Use and Expanded Access Programs”. Vol 20. Taylor & Francis; 2020:W5. do i:10.1080/15265161.2020.1820113 28. Verde PE,Ohmann C.Combining randomized and nonrandomized evidence in clinical research: A review of methods and applications. Res Synth Methods. 2015;6(1):45-62. doi:10.1002/ jrsm.1122 29. Rosenbaum PR, Rubin DB. The central role of the propensity score in observational studies for causal effects. Biometrika. 1983;70(1):41-55. doi:10.1093/BIOMET/70.1.41 30. Chen J, Kaplan D. Covariate Balance in Bayesian Propensity Score Approaches for Observational Studies. J Res Educ Eff. 2015;8(2):280-302. doi:1 0.1080/19345747.2014.911396 31. Gamalo-Siebers M, Savic J, Basu C, et al. Statistical modeling for Bayesian extrapolation of adult clinical trial information in pediatric drug evaluation. Pharm Stat. 2017;16(4):232249. doi:10.1002/pst.1807 32. Neuenschwander B, Branson M, Spiegelhalter DJ. A note on the power prior. Stat Med. 2009;28(28):3562-3566. doi:10.1002/sim.3722 33. Chen MH, Ibrahim JG, Shao QM. Power prior distributions for generalized linear models. J Stat Plan Inference. 2000;84(1-2):121-137. doi:10.1016/S0378-3758(99)00140-8 34. Duan Y. A Modified Bayesian Power Prior Approach with Applications in Water Quality Evaluation. Popul Engl Ed. Published online 2005:124-124. 35. Ibrahim JG, Chen MH. Power prior distributions for regression models. Stat Sci. 2000;15(1):4660. doi:10.1214/ss/1009212673 36. Lin J, Gamalo-Siebers M, Tiwari R. Propensityscore-based priors for Bayesian augmented control design. Pharm Stat. 2019;18(2):223-238. doi:10.1002/pst.1918 37. Polak TB, Cucchi DGJ, van Rosmalen J, Uylde Groot CA, Darrow JJ. Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations. Front Pharmacol. 2022;13. doi:10.3389/ fphar.2022.913567 38. Polak TB, Fernandez Lynch H. The Ethics of Expanded Access Research. JAMA. 2023;329(13):1057. doi:10.1001/ jama.2023.2204 39. Austin PC. An Introduction to Propensity Score Methods for Reducing the Effects of Confounding in Observational Studies. Multivar Behav Res. 2011;46(3):399-424. doi:10.1080/00 273171.2011.568786

RkJQdWJsaXNoZXIy MTk4NDMw