COST-UTILITY ANALYSIS OF RUXOLITINIB VERSUS BEST AVAILABLE THERAPY FOR THE TREATMENT OF HYDROXYUREA RESISTANT/INTOLERANT POLYCYTHEMIA VERA WITHOUT SPLENOMEGALY IN THE UNITED STATES

Author(s)

Hong S1, Veenstra DL2
1University of Washington School of Pharmacy, SEATTLE, WA, USA, 2University of Washington, Seattle, WA, USA

Presentation Documents

BACKGROUND: Polycythemia vera (PV) is a myeloproliferative neoplasm characterized by an abnormal increase in red blood cell mass. Ruxolitinib is a JAK 1/2 inhibitor which showed 7.3 times greater odds than best available therapy (BAT) (95% confidence interval 3.4-15.5) in achieving hematocrit control in patients with hydroxyurea resistant/intolerant PV. To date, there are no published US economic analyses of ruxolitinib in PV.

OBJECTIVES: To assess the cost-utility of ruxolitinib versus BAT for the treatment of hydroxyurea resistant/intolerant PV without splenomegaly.

PERSPECTIVE: US commercial payer

METHODS: A Markov model comprised of three health states (hematocrit control, no hematocrit control, and death) was used to estimate the economic and health-related quality of life impact of ruxolitinib versus BAT. A 15-year time horizon and 3% discount rate were considered for costs and outcomes. Clinical data was sourced from the phase III RESPONSE-2 trial. Costs, utilities and overall survival were obtained from published literature. Weibull and lognormal distributions were fitted to model beyond trial period. A one-way sensitivity analysis (OWSA) and probabilistic sensitivity analysis (PSA) were performed to assess parameter uncertainty.

RESULTS: Patients on ruxolitinib accumulated 28.5 quality-adjusted life years (QALYs), resulting in 1.2 additional QALYs compared to patients on BAT (27.3 QALYs). Total costs for ruxolitinib ($526,100) were $154,400 higher than for BAT ($371,700). The overall incremental cost-effectiveness ratio (ICER) was $128,600/QALY. The ICER was most sensitive to the efficacy of ruxolitinib, utility in the hematocrit control state, and cost of ruxolitinib. Results of the PSA showed that ruxolitinib would be cost-effective 73% of the time using a willingness-to-pay (WTP) threshold of $150,000/QALY.

CONCLUSIONS: Our results suggest ruxolitinib is cost-effective when compared to best available therapy in patients with hydroxyurea resistant/intolerant PV without splenomegaly using a WTP threshold of $150,000/QALY. Additional research is needed in defining PV-specific utilities and establishing an appropriate surrogate endpoint for survival.

Conference/Value in Health Info

2020-05, ISPOR 2020, Orlando, FL, USA

Code

PCN153

Topic

Economic Evaluation, Methodological & Statistical Research

Topic Subcategory

Modeling & Simulation

Disease

Drugs, Oncology, Rare and Orphan Diseases

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