A Systematic Review of Cost-Effectiveness Analyses of Novel Agents in the Treatment of Multiple Myeloma

Abstract

Background: Novel therapies for multiple myeloma (MM) promise to improve outcomes but are also associated with substantial increasing costs. Evidence regarding cost-effectiveness of novel treatments is necessary, but a comprehensive up-to-date overview of the cost-effectiveness evidence of novel treatments is currently lacking. Methods: We searched Embase, Medline via Ovid, Web of Science and EconLIT ProQuest to identify all cost-effectiveness evaluations of novel pharmacological treatment of MM reporting cost per quality-adjusted life year (QALY) and cost per life year (LY) gained since 2005. Quality and completeness of reporting was assessed using the Consolidated Health Economic Evaluation Reporting Standards. Results: We identified 13 economic evaluations, comprising 32 comparisons. Our results show that novel agents generate additional LYs (range: 0.311–3.85) and QALYs (range: 0.1–2.85) compared to backbone regimens and 0.02 to 1.10 LYs and 0.01 to 0.91 QALYs for comparisons between regimens containing two novel agents. Lifetime healthcare costs ranged from USD 60,413 to 1,434,937 per patient. The cost-effectiveness ratios per QALY gained ranged from dominating to USD 1,369,062 for novel agents compared with backbone therapies and from dominating to USD 618,018 for comparisons between novel agents. Conclusions: Cost-effectiveness ratios of novel agents were generally above current willingness-to-pay thresholds. To ensure access, cost-effectiveness should be improved or cost-effectiveness ratios above current thresholds should be accepted.

Clinical and economic studies of pharmacotherapy for multiple myeloma: literature review data

“…для того, чтобы сделать медицинскую помощь таким пациентам более доступной, требуется фармакоэкономическое обоснование применяемых схем фармакотерапии [35]. согласно международным литературным данным, полученным из medline, Scopus, web of Science, pubmed, в настоящее время отсутствуют актуальные сводные данные по оценке экономической эффективности лечения мм [36].…”

Carfilzomib, thalidomide, and dexamethasone is safe and effective in relapsed and/or refractory multiple myeloma: final report of the single arm, multicenter phase II ALLG MM018/AMN002 study.

“…4,5 In parallel, however, the associated healthcare costs are steadily increasing and while treatment-related drug costs account for less than a third of the total, the increased dependency on inpatient and outpatient services required for delivery of novel therapies is expected to further jeopardise their affordability and accessibility for all patients. 2,6 The management of relapsed or refractory multiple myeloma (RRMM) is complex, taking into consideration prior drug exposure, response, and toxicity profile, as well as disease-and patient-related factors. The challenge of managing RRMM is compounded by limited access to novel agents outside of clinical trials.…”

Treatment sequences and drug costs from diagnosis to death in multiple myeloma

“…1 This increasingly puts pressure on healthcare budgets, [2][3][4][5][6][7][8] and potentially endangers (future) access to health care. 9 Thus, sensibly utilising (expensive) drugs and applying these agents in the right patient at the appropriate timeincluding timely cessation of treatment-is crucial to ensure affordability, which supports the goal of achieving and retaining global drug access for MM patients.…”

“…With combination regimens being the cornerstone of MM treatment, these drug costs now represent the most important component of overall healthcare expenses in MM treatment 1 . This increasingly puts pressure on healthcare budgets, 2–8 and potentially endangers (future) access to health care 9 . Thus, sensibly utilising (expensive) drugs and applying these agents in the right patient at the appropriate time—including timely cessation of treatment—is crucial to ensure affordability, which supports the goal of achieving and retaining global drug access for MM patients.…”