Cost-Effective Analysis Compares Multiple Therapy Options for Pretreated Multiple Myeloma


A recent study confirmed the most cost-effective treatment option in heavily pretreated patients with relapsed or refractory multiple myeloma, published in Clinical Therapeutics (October 2017;39[10]:1986-2005).

Based on the findings of three different clinical trials, pomalidomide plus low-dose dexamethasone, daratumumab monotherapy, and carfilzomib monotherapy have been FDA approved for the treatment of patients with heavily pretreated relapsed or refractory multiple myeloma in the United States. However, the cost-effectiveness of these treatments relative to one another has yet to be determined.

Christopher G Pelligra, BA, Evidera (Waltham, MA), and colleagues conducted a cost-effectiveness analysis of these three treatment options for patients with pretreated relapsed or refractory multiple myeloma for a US payer’s perspective. Researchers designed a model to estimate the cost and health outcomes over a 3-year time span in three health states: progression-free survival (PFS), post-progression, and death. Direct medical costs were considered, including treatment acquisition and administration, pre- and post-medication, prophylaxis treatment, adverse event management, and health care resource utilization.

Researchers estimated costs, life-years, and quality-adjusted life-years.

Over the 3-year span, researchers reported that the use of pomalidomide plus low-dose dexamethasone yielded comparable life-years and quality-adjusted life years gained with daratumumab and carfilzomib (incremental: life-years, 0.02-0.07, respectively; quality-adjusted life-years, 0.01-0.05). The cost of pomalidomide plus low-dose dexamethasone was less than that of daratumumab (-$8,919) and similar to that of carfilzomib (-$195).


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Additionally, sensitivity analyses showed that results were sensitive to PFS, treatment duration, and therapy costs. An equal efficacy scenario resulted in cost-savings for pomalidomide plus low-dose dexamethasone relative to those of both daratumumab and carfilzomib (-$11,779 and -$12,595, respectively).

Researchers noted that the study’s results were not statistically significant, due to a small sample size in their computer model.

Results of the study imply that pomalidomide plus low-dose dexamethasone is the most cost-effective therapy option for patients with heavily pretreated relapsed or refractory multiple myeloma. Researchers acknowledged that head-to-head clinical trials are needed to validate this finding.—Zachary Bessette