Chapter 9 214 40. Ibrahim JG, Chen MH, Gwon Y, Chen F. The power prior: Theory and applications. Stat Med. 2015;34(28):3724-3749. doi:10.1002/sim.6728 41. Sosman JA, Kim KB, Schuchter L, et al. Survival in BRAF V600–Mutant Advanced Melanoma Treated with Vemurafenib. N Engl J Med. 2012;366(8):707-714. doi:10.1056/ NEJMoa1112302 42. Flaherty L, Hamid O, Linette G, et al. A Single-Arm, Open-Label, Expanded Access Study of Vemurafenib in Patients With Metastatic Melanoma in the United States. Cancer J. 2014;20(1):18-24. doi:10.1097/ PPO.0000000000000024 43. Han B, Zhan J, John Zhong Z, Liu D, Lindborg S. Covariate-adjusted borrowing of historical control data in randomized clinical trials. Pharm Stat. 2017;16(4):296-308. doi:10.1002/pst.1815 44. Darrow JJ, Sarpatwari A, Avorn J, Kesselheim AS. Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs. Hamel MB, ed. N Engl J Med. 2015;372(3):279-286. doi:10.1056/ NEJMhle1409465 45. Chapman CR, Moch KI, McFadyen A, et al. What compassionate use means for gene therapies. Nat Biotechnol. 2019;37(4):352-355. doi:10.1038/s41587-019-0081-7 46. 46. Rozenberg O, Greenbaum D. Making It Count: Extracting Real World Data from Compassionate Use and Expanded Access Programs. Am J Bioeth AJOB. 2020;20(7):89-92. doi:10.1080/15265161.2020.1779857 47. Caplan AL, Ray A. The Ethical Challenges of Compassionate Use. JAMA. 2016;315(10):979980. doi:10.1001/jama.2016.0416 48. DreyerNA.AdvancingaFrameworkforRegulatory Use of Real-World Evidence: When Real Is Reliable. Ther Innov Regul Sci. 2018;52(3):362368. doi:10.1177/2168479018763591 49. Banbeta A, Lesaffre E, van Rosmalen J. The power prior with multiple historical controls for the linear regression model. Pharm Stat. 2022;21(2):418-438. doi:10.1002/pst.2178 50. Kaplan D, Chen J. A Two-Step Bayesian Approach for Propensity Score Analysis: Simulations and Case Study. Psychometrika. 2012;77(3):581-609. doi:10.1007/s11336-012-9262-8
RkJQdWJsaXNoZXIy MTk4NDMw