Tobias Polak

The ethics of expanded access research 245 12 ♠ 56. CHMP/EMEA. Guideline on Registry-Based Studies. https://www.ema.europa.eu/en/documents/ scientific-guideline/guideline-registry-basedstudies_en-0.pdf; 2021. 57. Sherman RE, Anderson SA, Dal Pan GJ, et al. Real-World Evidence — What Is It and What Can It Tell Us? N Engl J Med. 2016;375(23):22932297. doi:10.1056/NEJMsb1609216 58. Arlett P, Kjær J, Broich K, Cooke E. Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value. Clin Pharmacol Ther. 2022;111(1):21-23. doi:10.1002/cpt.2479 59. Desai RJ, Matheny ME, Johnson K, et al. Broadening the reach of the FDA Sentinel system: A roadmap for integrating electronic health record data in a causal analysis framework. Npj Digit Med. 2021;4(1):170. doi:10.1038/s41746-021-00542-0 60. 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 61. FDA approves alpelisib for PIK3CA-related overgrowth spectrum | FDA. Accessed January 17, 2023. https://www.fda.gov/drugs/resourcesinformation-approved-drugs/fda-approvesalpelisib-pik3ca-related-overgrowth-spectrum 62. 62. Lynch HF, Morris S, Andrews JA. Access to investigational drugs for patients with amyotrophic lateral sclerosis in the USA. Lancet Neurol. 2022;21(7):593-594. doi:10.1016/ S1474-4422(22)00217-4 63. Gould P, Salam T, Kimberly L, Bateman-House A, Fernandez Lynch H. Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs. JAMA Netw Open. 2022;5(11):e2239766. doi:10.1001/ jamanetworkopen.2022.39766 64. Kang S, Chang S, Ross JS, Miller JE. Implementation of 21st Century Cures Act Expanded Access Policies Requirements. Clin Pharmacol Ther. Published online September 12, 2021. doi:10.1002/cpt.2401

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