Tobias Polak

Discussion 261 REFERENCES 1. 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 2. Senn S. Statistical pitfalls of personalized medicine. Nature. 2018;563(7733):619-621. doi:10.1038/d41586-018-07535-2 3. Abola MV, Prasad V. The Use of Superlatives in Cancer Research. JAMA Oncol. 2016;2(1):139139. doi:10.1001/jamaoncol.2015.3931 4. Greenland S, Senn SJ, Rothman KJ, et al. Statistical tests, P values, confidence intervals, and power: a guide to misinterpretations. Eur J Epidemiol. 2016;31(4):337-350. doi:10.1007/ s10654-016-0149-3 5. Senn S. Statistical Issues in Drug Development. Third edition. John Wiley and Sons, Ltd; 2021. 6. La Caze A, Djulbegovic B, Senn S. What does randomisation achieve? Evid Based Med. 2012;17(1):1-2. doi:10.1136/ebm.2011.100061 7. Polak TB, Fernandez Lynch H. The Ethics of Expanded Access Research. JAMA. 2023;329(13):1057. doi:10.1001/ jama.2023.2204 8. Senn S. Seven myths of randomisation in clinical trials. Stat Med. 2013;32(9):1439-1450. doi:10.1002/sim.5713 9. Harrell F. Statistical Thinking - Randomized Clinical Trials Do Not Mimic Clinical Practice, Thank Goodness. Published February 14, 2023. Accessed May 5, 2023. https://www.fharrell. com/post/rct-mimic/ 10. Harrell F. Statistical Thinking - Statistical Errors in the Medical Literature. Published April 8, 2017. Accessed May 5, 2023. https://www. fharrell.com/post/errmed/ 11. National Institute for Health and Clinical Excellence (NICE). Guide to the Single Technology Appraisal Process.; 2009. 12. The National Institute for Health and Care. Guide to the methods of technology appraisal. Nice. 2018;(April 2013):1-93. 13. Latimer NR, Adler AI. Extrapolation beyond the end of trials to estimate long term survival and cost effectiveness. BMJ Med. 2022;1(1):e000094. doi:10.1136/bmjmed-2021-000094 14. Blommestein H. The Added Value of Real-World Evidence. 2016. 15. Polak TB, Cucchi DG, van Rosmalen J, Uyl-de Groot CA. Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals. BMJ Open. 2022;12(1):e052186-e052186. doi:10.1136/bmjopen-2021-052186 16. 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 17. 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 18. Senn S. Disappointing dichotomies. Pharm Stat. 2003;2(4):239-240. doi:10.1002/pst.90 19. 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 20. Sarp S, Reichenbach R, Aliu P. An approach to data collection in compassionate use/managed access. Front Pharmacol. 2022;13. doi:10.3389/ fphar.2022.1095860 21. Brown EG. Using MedDRA: Implications for Risk Management. Drug Saf. 2004;27(8):591-602. doi:10.2165/00002018-200427080-00010 22. Bunnik EM, Aarts N. What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands. BMC Med Ethics. 2019;20(1):80-80. doi:10.1186/s12910-0190420-8

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