Most Cost-Effective Treatment Regimen Identified for Relapsed, Refractory Multiple Myeloma


A recent cost-effectiveness analysis evaluated treatments for relapsed or refractory multiple myeloma from a United States health system perspective, published in the Journal of Managed Care & Specialty Pharmacy (January 2018;24[1]:29-38).

In recent years, there have been a few three-drug regimens developed and approved by the US Food and Drug Administration for the treatment of multiple myeloma. However, there is a general lack of direct comparative data for these costly regimens, which supports the need for assessment of the clinical and economic outcomes across all approved regimens.

Josh J Carlson, MPH, PhD, Pharmaceutical Outcomes Research and Policy Program, University of Washington, and colleagues evaluated the cost-effectiveness of multiple myeloma regimens by developing a partition survival model with three health states: progression-free, progression, and death. Among the evaluated regimens were carfilzomib, elotuzumab, ixazomib, daratumumab, and panobinostat, all in combination with either lenalidomide or bortezomib plus dexamethasone in the second or third line of  therapy.

Researchers developed a network meta-analysis and applied progression-free survival (PFS) hazard ratios to baseline parametric PFS functions observed from pooled data on lenalidomide plus dexamethasone. Overall survival was estimated by using data on the relationship between PFS and overall survival (OS) from a meta-analysis of patients with multiple myeloma.

Model costs included in the analysis were those related to drug treatment, administration, monitoring, adverse events, and progression.

Results of the analysis showed that regimens containing daratumumab had the highest expected life years (range, 6.71-7.38) and quality-adjusted life years (range, 4.38-5.44). Daratumumab plus bortezomib and dexamethasone in the second line, as well as panobinostat plus bortezomib and dexamethasone in the third line, were the most cost-effective regimens (incremental cost-effectiveness ratio: $50,700). Researchers noted that the applicability of the latter regimen result may be challenging, due to ongoing toxicity concerns.


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A probabilistic sensitivity analysis revealed second-line daratumumab plus bortezomib and dexamethasone, as well as third-line panobinostat plus bortezomib and dexamethasone, had an 89% and 87% probability of being cost-effective at the $150,000 per quality-adjusted life years threshold, respectively.

Dr Carlson and colleagues concluded that only the addition of daratumumab or panobinostat to multiple myeloma treatment regimens may be considered cost-effective options, according to commonly used thresholds. “Achieving levels of value more closely aligned with patient benefit would require substantial discounts from the remaining agents evaluated,” they wrote.—Zachary Bessette