References
- Palumbo A, Anderson K. Multiple myeloma. N Engl J Med. 2011;364(11):1046–1060.
- American Cancer Society. Key statistics about multiple myeloma; 2018 [cited 2018 Oct 23]. Available from: https://www.cancer.org/cancer/multiple-myeloma/about/key-statistics.html
- Raab MS, Podar K, Breitkreutz I, et al. Multiple myeloma. Lancet. 2009 July 25;374(9686):324–339.
- American Cancer Society. Signs and symptoms of multiple myeloma; 2018 [cited 2018 Oct 23]. Available from: https://www.cancer.org/cancer/multiple-myeloma/detection-diagnosis-staging/signs-symptoms.html
- Durie BGM Concise review of the disease and treatment options. International Myeloma Foundation (2017 Edition) 2017 [cited 2017 Oct 17]. Available from: https://www.myeloma.org/sites/default/files/images/publications/UnderstandingPDF/concisereview.pdf
- Kumar SK, Therneau TM, Gertz MA, et al. Clinical course of patients with relapsed multiple myeloma. Mayo Clin Proc. 2004 July 1;79(7):867–874.
- Orlowski RZ. Novel agents for multiple myeloma to overcome resistance in phase III clinical trials. Semin Oncol. 2013 Oct 1;40(5):634–651.
- Jakubowiak A. Management strategies for relapsed/refractory multiple myeloma: current clinical perspectives. Semin Hematol. 2012 July 1;49:S16–S32.
- Amgen data on file. 2019. Calculated weighted average by line based on Amgen chart review data.
- Food and Drug Administration. KYPROLIS® (carfilzomib) for injection, for intravenous use: highlights of prescribing information; 2019 [cited 2019 Jan 21]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/202714s025lbl.pdf
- Kim C, Werther W, Iskander KS, et al. Demographic and clinical characteristics of Multiple Myeloma (MM) patients treated with carfilzomib-based regimens in a real world setting. Blood. 2017;130(Suppl 1):5441.
- Medhekar R, Hines D, Panjabi S, et al. Could patients with Multiple Myeloma (MM) derive additional benefit from their treatments? Real-world evidence for carfilzomib dosing intensity on survival and treatment progression. Blood. 2018;132(Suppl 1):836.
- Moreau P, Mateos MV, Berenson JR, et al. Once weekly versus twice weekly carfilzomib dosing in patients with relapsed and refractory multiple myeloma (A.R.R.O.W.): interim analysis results of a randomised, phase 3 study. Lancet Oncol. 2018;19(7):953–964.
- Moreau P, Kumar S, Boccia R, et al. Convenience, satisfaction, health-related quality of life of once-weekly 70 mg/m2 vs. twice-weekly 27 mg/m2 carfilzomib (randomized A.R.R.O.W. study). Leukemia. 2019 May 15;33:2934–2946.
- Jakubowiak AJ, Houisse I, Majer I, et al. Cost-effectiveness of carfilzomib plus dexamethasone compared with bortezomib plus dexamethasone for patients with relapsed or refractory multiple myeloma in the United States. Expert Rev Hematol. 2017;10(12):1107–1119.
- Jakubowiak AJ, Campioni M, Benedict A, et al. Cost-effectiveness of adding carfilzomib to lenalidomide and dexamethasone in relapsed multiple myeloma from a US perspective. J Med Econ. 2016;19(11):1061–1074.
- SEER. Surveillance, epidemiology, and end results program. Stat Fact Sheets: Myeloma; 2016 [cited 2016 Mar 28]. Available from: http://seer.cancer.gov/statfacts/html/mulmy.html
- Human Mortality Database. The United States of America, life tables; 2018.
- Analysource. Wholesale acquisition cost (WAC); 2018 [cited 2018 Oct 8]. Available from: https://www.analysource.com/
- Centers for Medicare & Medicaid Services (CMS). National physician fee schedule 2018 [cited 2018 Oct 8]. Available from: https://www.cms.gov/apps/physician-fee-schedule/
- HCUPnet. National Inpatient Sample (NIS), Agency for Healthcare Research and Quality (AHRQ). National statistics by principal diagnosis code. Rockville, MD: Agency for Healthcare Research and Quality; 2015 [cited 2018 Oct 4]. Available from https://hcupnet.ahrq.gov/#setup
- Latimer NR. Survival analysis for economic evaluations alongside clinical trials—extrapolation with patient-level data: inconsistencies, limitations, and a practical guide. Med Decis Mak. 2013 June 1;33(6):743–754.
- Jackson C, Stevens J, Ren S, et al. Extrapolating survival from randomized trials using external data: a review of methods. Med Decis Making. 2017;37(4):377–390.
- Latimer N NICE DSU technical support document 14: undertaking survival analysis for economic evaluations alongside clinical trials - extrapolation with patient-level data 2013 [updated 2013 Mar; 2017 Jan 30]. Available from: http://nicedsu.org.uk/wp-content/uploads/2016/03/NICE-DSU-TSD-Survival-analysis.updated-March-2013.v2.pdf
- van Agthoven M, Segeren CM, Buijt I, et al. A cost-utility analysis comparing intensive chemotherapy alone to intensive chemotherapy followed by myeloablative chemotherapy with autologous stem-cell rescue in newly diagnosed patients with stage II/III multiple myeloma; a prospective randomised phase III study. Eur J Cancer. 2004;40(8):1159–1169.
- National Institute for Health and Care Excellence. Pomalidomide for relapsed and refractory multiple myeloma previously treated with lenalidomide and bortezomib [TA338] 2015. [cited 2019 Apr 30]. Available from: https://www.nice.org.uk/guidance/ta338
- National Institute for Health and Care Excellence. Daratumumab monotherapy for treating relapsed and refractory multiple myeloma (TA510); 2018 [cited 2018 Oct 11]. Available from: https://www.nice.org.uk/guidance/ta510/
- Bennison C, Stephens S, Lescrauwaet B, et al. Cost-effectiveness of ocriplasmin for the treatment of vitreomacular traction and macular hole. J Mark Access Health Policy. 2016;4:1–11.
- Amgen data on file. Retrospective study of healthcare utilization and costs in patients with multiple myeloma using Truven MarketScan Data through December 2014; 2015.
- Bureau of Labor Statistics. Consumer Price Index (CPI) for medical care services in the U.S. for all urban consumers; 2018 [cited 2018 Oct 10]. Available from: https://data.bls.gov/timeseries/CUSR0000SAM2
- Hillner BE, Smith TJ. Efficacy does not necessarily translate to cost effectiveness: a case study in the challenges associated with 21st-century cancer drug pricing. J clin oncol. 2009;27(13):2111–2113.
- Neumann PJ, Cohen JT, Weinstein MC. Updating cost-effectiveness — the curious resilience of the $50,000-per-QALY threshold. N Engl J Med. 2014;371(9):796–797.
- Majer IM, Castaigne J-G, Palmer S, et al. Modeling covariate-adjusted survival for economic evaluations in oncology. PharmacoEconomics. 2019 May 1;37(5):727–737.
- Hornberger J, Rickert J, Dhawan R, et al. The cost-effectiveness of bortezomib in relapsed/refractory multiple myeloma: Swedish perspective. Eur J Haematol. 2010;85(6):484–491. .
- Möller J, Nicklasson L, Murthy A. Cost-effectiveness of novel relapsed-refractory multiple myeloma therapies in Norway: lenalidomide plus dexamethasone vs bortezomib. J Med Econ. 2011 Jan 01;14(6):690–697.
- Unger JM, Barlow WE, Martin DP, et al. Comparison of survival outcomes among cancer patients treated in and out of clinical trials. J Natl Cancer Inst. 2014;106(3):002.