Cost-Effectiveness of Lenalidomide in the Treatment of Patients with Transfusion-Dependent Myelodysplastic Syndromes (MDS) in the United States.
Authors: Goss Thomas F. , Szende Agota , Brueckner Jane , Wang Michael , Schaefer Caroline , List Alan F. 
Published: April 2020

Abstract
Background: Effective treatment for patients with low- or intermediate-1 (IPSS) risk MDS with transfusion-dependent anemia is limited, forcing reliance upon red blood cell (RBC) transfusions and chelation therapy with attendant complications and adverse impact on quality of life (QOL) and health care costs. Lenalidomide is a new immunomodulatory drug, which received fast track designation from the FDA for the treatment of transfusion-dependent MDS associated with chromosome 5q31 deletion. We evaluated the cost-effectiveness of lenalidomide therapy for transfusion-dependent patients with MDS.
Methods: A comprehensive decision analytic model was developed to evaluate and compare the costs and health benefits of lenalidomide with best supportive care (BSC) versus BSC with blood transfusions only over a 1-year period. Data on efficacy and blood transfusion requirements before and after the administration of lenalidomide were estimated from a phase II clinical trial (MDS-003). Health outcome measure used was rate and duration of response as manifested in transfusion-independent time gained, which is associated with important clinical and QOL benefits. The model incorporated direct medical costs related to medication, transfusions (blood components, iron chelation therapy, laboratory procedures, administration of transfusions), diagnostic tests, office visits and other medical resource use. Unit costs were taken from Medicare fee schedules that are standardized and closely reflect provider costs across the US. All costs were expressed in 2005 US dollars.
Results: Major and minor response was observed in 66% and 9% of patients treated with lenalidomide. Time to response was 4.1 weeks. The median duration of response was reached beyond one year (78 weeks). After excluding assessments beyond the 1-year timeframe of the model, the mean duration of transfusion independence among responders was 41.8 weeks. Previous QOL studies in MDS suggest that transfusion-independence is associated with 33% better QOL as measured by the QOL-E. In the first year, an average of 56 % reduction in RBC transfusion requirements was also observed in the lenalidomide group compared to BSC. The costs of lenalidomide therapy were largely offset by savings in blood transfusion costs. Multi-way sensitivity analyses on model parameter estimates showed that the incremental cost-effectiveness ratio of lenalidomide versus BSC is between $1,137 and $9,076 per transfusion-independent year gained. Based on a range of assumptions on QOL associated with living in transfusion independence versus transfusion-dependency, this translates into an incremental cost-effectiveness ratio between $3,240 to $38,799 per quality-adjusted life year (QALY) gained, well within usual acceptable ranges of cost-effectiveness for new technologies.
Conclusions: These results suggest that, through its oral drug administration, reduced transfusion requirements, and increased efficacy, lenalidomide is a cost-effective treatment option in transfusion-dependent MDS associated with chromosome 5q31 deletion in the US. Further research is warranted to confirm these results as longer-term comparative data on efficacy, QOL, and medical resource utilization become available from currently ongoing phase III randomized trials.