Beyond medicines’ barriers: exploring the true cost of multiple myeloma

Figures & data

Table 1. Complete list of questions administered in the online survey.

Number	Question
Question 1	In addition to treatment expenses, please kindly indicate what you believe are direct or indirect expenses to MM patients?
Question 2	Notwithstanding cost effectiveness, how do you assess novel combination therapies? And, what additional factors may be considered?
Question 3	How receptive are payers to considering beyond drug cost and cost of progression?
Question 4	How affected are MM subpopulations (frail, elderly, renal impairment, cytogenic risk groups) on the use of resources?
Question 5	How different is access to treatment and interventions across countries? (please explain your country situation)
Question 6	How affected are MM subpopulations (frail, elderly, renal impairment, cytogenic risk groups) on the use of resources?
Question 7	How different is access to treatment and interventions across countries? (please explain your country situation)
Question 8	In addition to treatment expenses, what other type of expenses can be considered for an HTA assessment?
Question 9	Please describe the current assessment framework used to assess MM therapies and if there are missing considerations. How open are payers to change the current methodology of assessment to assess double branded combinations?
Question 10	Novel therapies are upcoming, especially combinations therapies and BCMA/CAR-T. How are governments prepared to improve access?
Question 11	After seeing the results of the research, do you believe there are any policy issues?
Question 12	After seeing the results of the research, do you recommend any call-to-actions?

Figure 1. Prisma Flow Chart.

Table 2. Proportion of drug costs in total costs.

Author, Year	Research Question	Key Costs	Country
Kalff, 2014	Prednisolone maintenance alone (control group) or in combination with 12 months of thalidomide consolidation (thalidomide group)	Drug therapies (63-71%)	Australia and New Zealand
Touati, 2016	Combined treatment of outpatient hospital care and at home chemotherapy with injectable bortezomib vs. outpatient hospital care alone	Drug cost (28-34%) + administration (27-35%)	France
Djebbari, 2019	Cost aspects of bortezomib therapy	Drug costs (64%)	United Kingdom
Gooding, 2013	Medical resource utilizations costs for doublerefractory/ intolerant patients	Drug costs (31%) + clinical attendances (31%)	United Kingdom
Gooding, 2015	Medical resource utilization costs for patients with double relapsed and/or refractory MM	Drug cost (48%)	United Kingdom
Gonzalez, 2018	Total HRU costs associated with relapsed MM (by treatment line and received treatment regimen)	Drug cost: 94% in the UK 91% in France 95% in Italy	United Kingdom, France, Italy

Abbreviations. HRU, Healthcare Resource Utilization; MM, Multiple Myeloma.

Table 3. Cost aspects of being on maintenance therapy post-transplant.

Total healthcare resource utilization–associated costs by maintenance and non-maintenance patients and by country
Total cost per month across treatment pathway, €	All (n = 91)	Maintenance patients (n = 14)	Non-maintenance patients (n = 77)
	Mean (SD) €	Mean (SD) €	Mean (SD) €
UK unit costs^a	945.80 (1060.88)	638.14 (547.80)	1001.74 (1122.88)
France unit costs^a	446.51 (810.25)	260.86 (235.50)	480.26 (872.04)
Germany unit costs^a	562.58 (1005.59)	402.86 (412.07)	591.62 (1078.37)
Italy unit costs^a	407.96 (453.77)	238.29 (222.78)	438.81 (478.63)
Spain unit costs^a	620.07 (943.31)	301.00 (219.56)	678.08 (1011.61)

Abbreviations. SD, Standard deviation.

Adapted from Ashcroft et al.¹³

^aData on healthcare-resource use (HCRU) per patient per month were collected during the study period in all participating countries and then unit costs for UK, France, Germany, Italy and Spain were applied to the collected HCRU data to determine country-specific total cost.

Figure 2. Leading Burdens on Patients with MM and their Caregivers. Adapted from Robinson et al.²⁷

Ashcroft J, Judge D, Dhanasiri S, et al. Chart review across EU5 in MM post-ASCT patients. Int J Hematol Oncol. 2018;7(1):IJH05.

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Robinson D, Jr Orlowski RZ, Stokes M, et al. Economic burden of relapsed or refractory multiple myeloma: Results from an international trial. Eur J Haematol. 2017;99(2):119–132.

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