Tobias Polak

Data collection for the sake of data collection 173 8♦ REFERENCES 1. Reagan-Udall Foundation. Leveraging RealWorld Treatment Experience from Expanded Access Protocols. Reagan Udall Foundation; 2018:1-26. 2. Kimberly LL, Beuttler MM, Shen M, Caplan AL, Bateman-House A. Pre-approval Access Terminology: A Cause for Confusion and a Danger to Patients. Ther Innov Regul Sci. 2017;51(4):494500. doi:10.1177/2168479017696267 3. Young FE, Norris JA, Levitt JA, Nightingale SL. The FDA’s New Procedures for the Use of Investigational Drugs in Treatment. JAMA J Am Med Assoc. 1988;259(15):2267-2270. doi:10.1001/jama.1988.03720150043034 4. 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 5. Balasubramanian G, Morampudi S, Chhabra P, Gowda A, Zomorodi B. An overview of compassionate use programs in the european union member states. Intractable Rare Dis Res. 2016;5(4):244-254. doi:10.5582/ irdr.2016.01054 6. Jarow JP, Lemery S, Bugin K, Khozin S, Moscicki R. Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period. Ther Innov Regul Sci. 2016;50(6):705-709. doi:10.1177/2168479016656030 7. 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 8. Stower H. The promise of real-world data. Nat Med. Published online March 20, 2019. doi:10.1038/d41591-019-00010-z 9. Food and Drug Administration. Submitting Documents Using Real-World Data and RealWorld Evidence to FDA for Drugs and Biologics Guidance for Industry DRAFT GUIDANCE. 2019;(May 2019). 10. Blommestein H. The Added Value of Real-World Evidence. Erasmus University Rotterdam; 2016. 11. 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 12. Bunnik EM, Aarts N, van de Vathorst S. Little to lose and no other options: Ethical issues in efforts to facilitate expanded access to investigational drugs. Health Policy Amst Neth. 2018;122(9):977-983. doi:10.1016/j. healthpol.2018.06.005 13. Fountzilas E, Said R, Tsimberidou AM. Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits. Expert Opin Investig Drugs. 2018;27(2):155-162. doi:10.1080/13543784.20 18.1430137 14. Rawson K. Expanded Access Data Can Support Approval Decisions, US FDA Says. Accessed June 19, 2019. https://pink.pharmaintelligence. informa.com/PS124296/Expanded-AccessData-Can-Support-Approval-Decisions-USFDA-Says 15. Sutter S. Expanded Access Programs Eyed For Data-Gathering Purposes. https://pink. pharmaintelligence.informa.com/PS122926/ Expanded-Access-Programs-Eyed-ForDataGathering-Purposes%0D 16. S. Usdin. Beyond compassionate use: the case for using expanded access protocols to generate real world data. Accessed June 9, 2019. https://www.biocentury.com/biocentury/ regulation/2017-09-29/case-using-expandedaccess-protocols-generate-real-world-data 17. EMA. EMA: Download medicine data. Accessed May 1, 2019. https://www.ema.europa.eu/en/ medicines/download-medicine-data 18. FDA. Drugs@FDA. Accessed May 1, 2019. https://www.fda.gov/drugs/drug-approvalsand-databases/drugsfda-data-files 19. Smith R.An overviewof the tesseract OCR engine. In: Proceedings of the International Conference on Document Analysis and Recognition, ICDAR. ; 2007. doi:10.1109/ICDAR.2007.4376991 20. EMA. Viread: European Public Assessment Report. 21. FDA. NDA 20-645: Ammonul - Medical Review(s).; 2005.

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