US Budget Impact Model for Selinexor in Relapsed or Refractory Multiple Myeloma

References

• Kumar SK, Rajkumar V, Kyle RA, et al. Multiple myeloma. Nat Rev Dis Primer. 2017;3(1):17046. doi:10.1038/nrdp.2017.46
   PubMed Web of Science ®Google Scholar
• Surveillance, Epidemiology, and End Results (SEER). Program. Cancer stat facts: myeloma. 2016. Available from: https://seer.cancer.gov/statfacts/html/mulmy.html. Accessed November 27, 2018.
   Google Scholar
• Cancer facts & Figures 2019. Available from: http://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2019/cancer-facts-and-figures-2019.pdf. Accessed October 30, 2019.
   Google Scholar
• Dimopoulos MA, Richardson PG, Moreau P, Anderson KC. Current treatment landscape for relapsed and/or refractory multiple myeloma. Nat Rev Clin Oncol. 2015;12(1):42–54. doi:10.1038/nrclinonc.2014.200
   PubMed Web of Science ®Google Scholar
• Sonneveld P, De Wit E, Moreau P. How have evolutions in strategies for the treatment of relapsed/refractory multiple myeloma translated into improved outcomes for patients? Crit Rev Oncol Hematol. 2017;112:153–170. doi:10.1016/j.critrevonc.2017.02.007
   PubMed Web of Science ®Google Scholar
• Fonseca R, Abouzaid S, Bonafede M, et al. Trends in overall survival and costs of multiple myeloma, 2000–2014. Leukemia. 2017;31(9):1915–1921. doi:10.1038/leu.2016.380
   PubMed Web of Science ®Google Scholar
• Mikhael J. Treatment options for triple-class refractory multiple myeloma. Clin Lymphoma Myeloma Leuk. 2019;S2152265019320087. doi:10.1016/j.clml.2019.09.621
   Web of Science ®Google Scholar
• Bazarbachi AH, Al Hamed R, Malard F, Harousseau J-L, Mohty M. Relapsed refractory multiple myeloma: a comprehensive overview. Leukemia. 2019;33(10):2343–2357. doi:10.1038/s41375-019-0561-2
   PubMed Web of Science ®Google Scholar
• Kumar SK, Dimopoulos MA, Kastritis E, et al. Natural history of relapsed myeloma, refractory to immunomodulatory drugs and proteasome inhibitors: a multicenter IMWG study. Leukemia. 2017;31(11):2443–2448. doi:10.1038/leu.2017.138
   PubMed Web of Science ®Google Scholar
• Cornell RF, Gandhi UH, Lakshman A, et al. Subsequent treatment outcomes of multiple myeloma refractory to CD38-monoclonal antibody therapy. Blood. 2018;132(Supplement 1):2015. doi:10.1182/blood-2018-99-115078
   Web of Science ®Google Scholar
• Gandhi UH, Cornell RF, Lakshman A, et al. Outcomes of patients with multiple myeloma refractory to CD38-targeted monoclonal antibody therapy. Leukemia. 2019;33(9):2266–2275. doi:10.1038/s41375-019-0435-7
   PubMed Web of Science ®Google Scholar
• Pick M, Vainstein V, Goldschmidt N, et al. Daratumumab resistance is frequent in advanced-stage multiple myeloma patients irrespective of CD38 expression and is related to dismal prognosis. Eur J Haematol. 2018;100(5):494–501. doi:10.1111/ejh.13046
   PubMed Web of Science ®Google Scholar
• Usmani S, Ahmadi T, Ng Y, et al. Analysis of real-world data on overall survival in multiple myeloma patients with ≥3 prior lines of therapy Including a Proteasome Inhibitor (PI) and an Immunomodulatory Drug (IMID), or double refractory to a PI and an imid. The Oncologist. 2016;21(11):1355–1361. Doi:10.1634/theoncologist.2016-0104
   PubMed Web of Science ®Google Scholar
• XPOVIO (selinexor) prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/212306s000lbl.pdf. Available from: Accessed July. 8, 2019.
   Google Scholar
• Chari A, Vogl DT, Gavriatopoulou M, et al. Oral selinexor–dexamethasone for triple-class refractory multiple myeloma. N Engl J Med. 2019;381(8):727–738. doi:10.1056/NEJMoa1903455
   PubMed Web of Science ®Google Scholar
• Jagannath S, Vogl DT, Dimopoulos M-A, et al. Phase 2b results of the STORM study: oral selinexor plus low dose dexamethasone (SD) in patients with penta-refractory myeloma (Penta-MM). Clin Lymphoma Myeloma Leuk. 2018;18:S249–S250. doi:10.1016/j.clml.2018.07.149
   PubMed Web of Science ®Google Scholar
• Runyan A, Banks J, Bruni DS. Current and future oncology management in the United States. J Manag Care Spec Pharm. 2019;25(2):272–281. doi:10.18553/jmcp.2019.25.2.272
   PubMed Web of Science ®Google Scholar
• Djatche LM, Goble JA, Chun G, Varga S. Evaluating oncology value-based frameworks in the U.S. marketplace and challenges in real-world application: a multiple myeloma test case. J Manag Care Spec Pharm. 2018;24(1):39–46. doi:10.18553/jmcp.2018.24.1.39
   PubMed Web of Science ®Google Scholar
• Schnipper LE, Davidson NE, Wollins DS, et al. American Society of Clinical Oncology Statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol. 2015;33(23):2563–2577. doi:10.1200/JCO.2015.61.6706
   PubMed Web of Science ®Google Scholar
• Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology value framework: revisions and reflections in response to comments received. J Clin Oncol. 2016;34(24):2925–2934. doi:10.1200/JCO.2016.68.2518
   PubMed Web of Science ®Google Scholar
• Sullivan SD, Mauskopf JA, Augustovski F, et al. Budget impact analysis—principles of good practice: report of the ISPOR 2012 budget impact analysis good Practice II task force. Value Health. 2014;17(1):5–14. doi:10.1016/j.jval.2013.08.2291
   PubMed Web of Science ®Google Scholar
• Mauskopf JA, Sullivan SD, Annemans L, et al. Principles of good practice for budget impact analysis: report of the ISPOR task force on good research practices—budget impact analysis. Value Health. 2007;10(5):336–347. doi:10.1111/j.1524-4733.2007.00187.x
   PubMed Web of Science ®Google Scholar
• The AMCP format for formulary submissions, version 3.1. A format for submission of clinical and economic evidence of pharmaceuticals in support of formulary consideration. December 2012. Available from: http://amcp.org/practice-resources/amcp-format-formulary-submisions.pdf. Accessed July 16, 2018..
   Google Scholar
• MacEwan JP, Batt K, Yin W, et al. Economic burden of multiple myeloma among patients in successive lines of therapy in the United States. Leuk Lymphoma. 2018;59(4):941–949. doi:10.1080/10428194.2017.1361035
   PubMed Web of Science ®Google Scholar
• 2017 National Population Projections Datasets. Available from: https://www.census.gov/data/datasets/2017/demo/popproj/2017-popproj.html. Accessed January 29, 2019.
   Google Scholar
• Karyopharm Therapeutics, Inc. Epidemiology presentation. Data on file. 2018a.
   Google Scholar
• Karyopharm Therapeutics, Inc. Updated MM Epidemiology Presentation based on Symphony claims analysis (2016-2018); FHAD (2011-2018); STORM recruitment; and market research. Data on file; 2019.
   Google Scholar
• Cid Ruzafa J, Merinopoulou E, Baggaley RF, et al. Patient population with multiple myeloma and transitions across different lines of therapy in the USA: an epidemiologic model: multiple Myeloma lines of therapy. Pharmacoepidemiol Drug Saf. 2016;25(8):871–879. doi:10.1002/pds.3927
   Web of Science ®Google Scholar
• Costa LJ, Brill IK, Omel J, Godby K, Kumar SK, Brown EE. Recent trends in multiple myeloma incidence and survival by age, race, and ethnicity in the United States. Blood Adv. 2017;1(4):282–287. doi:10.1182/bloodadvances.2016002493
   PubMed Web of Science ®Google Scholar
• Kazandjian D. Multiple myeloma epidemiology and survival: A unique malignancy. Semin Oncol. 2016;43(6):676–681. doi:10.1053/j.seminoncol.2016.11.004
   PubMed Web of Science ®Google Scholar
• Cubanski J, Damico A, Neuman T. Medicare Part D: A first look at prescription drug plans in 2019. Available from: https://www.kff.org/medicare/issue-brief/medicare-part-d-a-first-look-at-prescription-drug-plans-in-2019/. Accessed February 1, 2019.
   Google Scholar
• Center for Medicare and Medicaid Services, Fast Facts. January 2019. Available from: https://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/CMS-Fast-Facts/index.html. Accessed May 18, 2020. 7, 2019.
   Google Scholar
• Selinexor Treatment of Refractory Myeloma (STORM). August 2018. Available from: https://clinicaltrials.gov/ct2/show/NCT02336815?term=NCT02336815&rank=1. Accessed November 21, 2018.
   Google Scholar
• Micromedex 2.0. Red Book Online. Thomson Reuters. Available from: https://www.micromedexsolutions.com. Accessed December 17, 2018..
   Google Scholar
• Karyopharm Therapeutics, Inc. Email correspondence regarding pricing. Data on file. 2018c.
   Google Scholar
• Mauskopf J, Earnshaw SR, Brogan AJ, Wolowacz S, Brodtkorb T-H Budget-Impact Analysis of Health Care Interventions: A Practical Guide; 2017.
   Google Scholar
• Bureau of Economic Analysis, 2018: Table 2.4.4U. Price Indexes for Personal Consumption Expenditures by Type of Product. December 2018.
   Google Scholar
• Karyopharm Therapeutics, Inc. US Demand Estimation Presentation. Data on file, 2018d.
   Google Scholar
• Karyopharm Therapeutics, Inc. KCP-330-012 CSR. Data on file. 2018b.
   Google Scholar
• Patterns of Health Care Resource Use Required for Treatment of Each TEAE Based on Guidance from James A. Kaye, MD (Hematology, Oncology). DrPH, RTI Health Solutions.
   Google Scholar
• Shao C, Monberg M, Cao X, Zhou W, Zhong Y, Marinello P. Real-world treatment patterns, health care utilization, and costs among relapsed/Refractory Multiple Myeloma (rrMM) patients. Blood. 2016;128(22):3555. doi:10.1182/blood.V128.22.3555.3555
   Web of Science ®Google Scholar
• Bloudek L, Roy A, Kish JK, et al. Estimating the economic impact of adding panobinostat to a U.S. formulary for relapsed and/or refractory multiple myeloma: a budget impact and cost-benefit model. J Manag Care Spec Pharm. 2016;22(8):991–1002. doi:10.18553/jmcp.2016.22.8.991
   Web of Science ®Google Scholar
• Potluri R, Bhandari H, Ranjan S, Chen C. Budget impact analysis of introducing elotuzumab in combination with lenalidomide and dexamethasone for relapsed/refractory multiple myeloma: a US payer perspective. Blood. 2016;128(22):2363. doi:10.1182/blood.V128.22.2363.2363
   Web of Science ®Google Scholar
• Hollmann S, Goyert N, Moldaver D, Grima D, Maiese E. U.S. budget impact analysis for daratumumab in combination with lenalidomide and dexamethasone, bortezomib and dexamethasone, or pomalidomide and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma. In: meeting Abstracts - Academy of Managed Care Pharmacy Nexus 2017. Vol 23. Journal of Managed Care & Specialty Pharmacy. 2017;S32. doi:10.18553/jmcp.2017.23.10-a.s1
   Google Scholar
• Shah N, Chari A, Scott E, Mezzi K, Usmani SZ. B-cell maturation antigen (BCMA) in multiple myeloma: rationale for targeting and current therapeutic approaches. Leukemia. 2020;34(4):985–1005. doi:10.1038/s41375-020-0734-z
   PubMed Web of Science ®Google Scholar