Economic Burden of Relapsed or Refractory Multiple Myeloma: Results from an International Trial
Authors: Robinson Donald , Orlowski Robert Z. , He Jianming , Lam Annette , Zhao Ning , Cakana Andrew Z 
Published: October 2019

Abstract
Background: The direct (medical utilization) cost of relapsed or refractory multiple myeloma (RRMM) has been documented; recent studies are now exploring indirect costs as well (Goodwin et al, Cancer Nurs 2013;36(4):301-8; Moreau et al, Haematologica 2014;99(suppl 1):214-5). Healthcare decision makers are interested in identifying meaningful cost offsets from new therapies providing substantially better clinical outcomes (Cook, J Manag Care Pharm 2008;14(7 Suppl):19-25).
Methods: Data from a patient economic questionnaire inquiring about recent disease-specific medical utilization, workforce status, and caregiver burden was analyzed from RRMM patients in a Phase 2, multi-site, international, randomized, controlled study (n = 307; Orlowski et al, Am J Hematol 2015;90(1):42-9). Direct and indirect costs of illness were characterized using standard descriptive statistics among those at study entry who were randomized and subsequently completed a baseline economic questionnaire. Patient-reported health questionnaires included the Brief Pain Inventory (BPI) pain intensity and the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scales; severity thresholds based on literature and developer recommendations for each were applied in analysis.
Results: Patients who answered the questionnaire (n = 267) were 54% male, 92% Caucasian, mean and median age of 63 years, and median ECOG status of 1; while 31% reported moderate to severe pain, 71% reported moderate to severe fatigue. At least 1 hospital visit was reported by 107 (42%) respondents over the last 6 months, while 185 (71%) and 86 (33%) reported specialist and family physician visits, respectively, over the last 3 months (Table). While only 11% of the reporting population was working, 48% of these who weren't working indicated it was due to their disease and 39% of those working reported disease-driven absenteeism of at least 1 day over the last 4 weeks. Of the 80% that were retired, 32% indicated it was an early retirement caused by RRMM.
Conclusions: These data document the substantial direct medical costs for RRMM patients but also the notable indirect, social costs associated with this disease. The clinical and demographic profile of these patients were similar to other multi-site, international RRMM studies, suggesting these results are broadly applicable (Richardson et al, Blood 2014;123(12):1826-32; Siegel et al, Blood 2012;120(14):2817-25; Kumar et al, Leukemia 2012;26(1):149-57). Longitudinal studies tracking treatment impact on economic outcomes, including direct and indirect cost offsets, are needed to understand the full social impact of new therapies in MM (Cook, 2008;14(7 Suppl):19-25).
Table. Patient-reported Economic Impact of RRMM N (%) of patients Disease-driven hospital visits (prior 6 months) 0: 151 (58%)≥ 1: 107 (42%) Disease-driven specialist visits (prior 3 months) 0: 75 (28.9%)≥ 1: 185 (71.1%) Disease-driven family practice visits (prior 3 months) 0: 174 (66.9%)≥ 1: 86 (33.1%) Working now Yes: 28 (11.6%)No: 236 (89.4%) Disease driven absentee days (prior 4 weeks) 0: 17 (61%)≥ 1: 10 (39%) Not working now due to disease Yes: 113 (48.7%)No: 119 (51.3%) Retired Yes: 188 (80%)No: 47 (20%) Early retirement due to disease Yes: 61 (32.4%)No: 127 (67.6%) Disease-driven disability compensation Yes: 96 (36.9%)No: 164 (63.1%) Working days missed by caregiver 0: 201 (80.1%)≥ 1: 50 (19.9%)

Disclosures
Robinson: Janssen: Employment. Orlowski:Janssen: Membership on an entity's Board of Directors or advisory committees; Onyx Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Acetylon Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Bristol-Myers Squibb: Membership on an entity's Board of Directors or advisory committees, Research Funding; Array Biopharma: Membership on an entity's Board of Directors or advisory committees, Research Funding; Millennium Pharaceuticals: Membership on an entity's Board of Directors or advisory committees, Research Funding; Spectrum: Research Funding; Biotheryx: Membership on an entity's Board of Directors or advisory committees; Forma Therapeutics: Membership on an entity's Board of Directors or advisory committees; Genentech: Membership on an entity's Board of Directors or advisory committees. He:Janssen: Employment. Lam:Janssen: Employment. Zhao:Janssen: Employment. Cakana:Janssen: Employment.