Chapter 4 76 REFERENCES 1. Walter RB, Appelbaum FR, Tallman MS, Weiss NS, Larson RA, Estey EH. Shortcomings in the clinical evaluation of new drugs: acute myeloid leukemia as paradigm. Blood. 2010;116(14):2420-2428. 2. Abola M V., Prasad V. The Use of Superlatives in Cancer Research. JAMA Oncol. 2016;2(1):139. 3. Hordijk M, Vermeulen SF, Bunnik EM. The ‘false hope’ argument in discussions on expanded access to investigational drugs: a critical assessment. Med Health Care Philos. 2022;25(4):693-701. 4. Beyar-Katz O, Rowe JM, Townsend LE, Tallman MS, Hadomi R, Horowitz NA. Published abstracts at international meetings often over- or underestimate the initial response rate. Blood. 2017;129(16):2326-2328. 5. Sasaki K, Ravandi F, Kadia TM, et al. De novo acute myeloid leukemia: A population-based study of outcome in the United States based on the Surveillance, Epidemiology, and End Results (SEER) database, 1980 to 2017. Cancer. 2021;127(12):2049-2061. 6. Tran AA, Miljković M, Prasad V. Analysis of estimated clinical benefit of newly approved drugs for US patients with acute myeloid leukemia. Leuk Res. 2020;96:106420. 7. Oliva EN, Franek J, Patel D, Zaidi O, Nehme SA, Almeida AM. The Real-World Incidence of Relapse in Acute Myeloid Leukemia (AML): A Systematic Literature Review (SLR). Blood. 2018;132(Supplement 1):5188-5188. 8. Kantarjian H, Sawyers C, Hochhaus A, et al. Hematologic and Cytogenetic Responses to Imatinib Mesylate in Chronic Myelogenous Leukemia. N Engl J Med. 2002;346(9):645-652. 9. Cherny NI, Dafni U, Bogaerts J, et al. ESMOMagnitude of Clinical Benefit Scale version 1.1. Ann Oncol. 2017;28(10):2340-2366. 10. Kiesewetter B, Cherny NI, Boissel N, et al. EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies. ESMO Open. 2020;5(1):e000611. 11. Paul JE,Trueman P.“Fourth hurdle reviews”, NICE, and database applications. Pharmacoepidemiol Drug Saf. 2001;10(5):429-438. 12. Weinstein MC. A QALY is a QALY is a QALY — Or is it? J Health Econ. 1988;7(3). 13. Weinstein MC, Torrance G, McGuire A. QALYs: The basics. Value Health. 2009;12(SUPPL. 1):S5-S9. 14. Zeckhauser R, Shepard D. Where Now for Saving Lives? Law Contemp Probl. 1976;40(4):5. 15. The National Institute for Health and Care. Guide to the methods of technology appraisal. Nice. 2018;(April 2013):1-93. 16. 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. 17. National Institute for Health and Care Excellence. Single technology appraisal: User guide for company evidence submission. 2018;(January 2015):1-36. 18. Mistry H, Nduka C, Connock M, et al. Venetoclax for Treating Chronic Lymphocytic Leukaemia: An Evidence Review Group Perspective of a NICE Single Technology Appraisal. PharmacoEconomics. 2018;36(4):399-406. 19. Lassen MR, Raskob GE, Gallus A, Pineo G, Chen D, Portman RJ. Apixaban or Enoxaparin for Thromboprophylaxis after Knee Replacement. N Engl J Med. 2009;361(6):594-604. 20. Lassen MR, Raskob GE, Gallus A, Pineo G, Chen D, Hornick P. Apixaban versus enoxaparin for thromboprophylaxis after knee replacement (ADVANCE-2): a randomised double-blind trial. The Lancet. 2010;375(9717):807-815. 21. Lassen MR, Gallus A, Raskob GE, Pineo G, Chen D, Ramirez LM. Apixaban versus Enoxaparin for Thromboprophylaxis after Hip Replacement. N Engl J Med. 2010;363(26):2487-2498. 22. Hwang TJ, Carpenter D, Lauffenburger JC, Wang B, Franklin JM, Kesselheim AS. Failure of Investigational Drugs in Late-Stage Clinical Development and Publication of Trial Results. JAMA Intern Med. 2016;176(12):1826.
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