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.