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Review
. 2021 Nov 9;13(22):5606.
doi: 10.3390/cancers13225606.

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

Maarten R Seefat  1 , David G J Cucchi  1 , Stijn Dirven  1 , Kaz Groen  1 , Sonja Zweegman  1 , Hedwig M Blommestein  2
Affiliations
Review

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

Maarten R Seefat et al. Cancers (Basel). .

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.

Keywords: carfilzomib; cost-effectiveness; daratumumab; economic evaluation; elotuzumab; ixazomib; multiple myeloma; panobinostat; pomalidomide.

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Conflict of interest statement

Sonja Zweegman; Consulting or Advisory Role: Janssen-Cilag, Takeda, Celgene, Sanofi, Oncopeptides; Research Funding: Janssen-Cilag, Takeda, Celgene; Travel, Accommodations, Expenses: Janssen-Cilag, Takeda, Celgene; Hedwig Blommestein; Consulting or Advisory Role: Pfizer, Scientific Advisory Board National Health Care Institute the Netherlands. Research Funding: Celgene, CADTH (Canadian Agency for Drugs and Technologies in Health). No other potential conflicts of interest were reported.

Figures

Figure 1
Figure 1
PRISMA flowchart. * Excluded on basis of exclusion criteria, e.g., no multiple myeloma, no active anti-MM treatment, no costs described, etc.
Figure 2
Figure 2
Incremental Costs, incremental QALYs and ICERs. Abbreviations: Dara: Daratumumab (monotherapy or in combination with backbone therapy), Pom: Pomalidomide monotherapy, Pom-d: Pomalidomide-dexamethasone, Kd: Carfilzomib-dexamethasone, DVd: Daratumumab-bortezomib-dexamethasone, Vd: Bortezomib-dexamethasone, DRd: Daratumumab-lenalidomide-dexamethasone, Rd: Lenalidomide-dexamethasone, KRd: Carfilzomib-lenalidomide-dexamethasone, Erd: Elotuzumab-lenalidomide-dexamethasone, Ird: Ixazomib-lenalidomide-dexamethasone, Pano-Vd: Panobinostat-bortezomib-dexamethasone, HiDex: High dose dexamethasone monotherapy, Kd70 QW: Kd 70 mg/m2 weekly, Kd27 BIW: Kd 27 mg/m2 twice per week.
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