Ruxolitinib May Be More Cost-Effective Than BAT for Patients With Hydroxyurea Resistant/Intolerant PV

Ruxolitinib was found to be more cost-effective than the best available therapy (BAT) when the willingness-to-pay threshold for payers was at $150,000 and applied to patients with hydroxyurea resistant/intolerant polycythemia vera (PV) without splenomegaly, according to a new study.

Ruxolitinib for the treatment of hydroxyurea resistant/intolerant polycythemia vera (PV) without splenomegaly is considered cost-effective compared with the best available therapy (BAT) when a payer’s willingness to pay threshold is at $150,000 per quality-adjusted life year (QALY) gained.

PV is a rare blood cancer characterized by an abnormal red blood cell increase that is caused by a Janus kinase 2 (JAK2) gene mutation. Hydroxyurea chemotherapy resistance or intolerance occurs in 15% to 24% of patients with PV, according to study results presented at the recent Virtual ISPOR 2020 conference.

When the willingness-to-pay threshold was at $150,000, the probability that ruxolitinib was most cost-effective was 73%. Researchers chose this threshold because Institute for Clinical and Economic Review standards depict that costs per QALY and adopting thresholds should be between $100,000 and $150,000.

“These thresholds are commonly cited in US economic analyses. The higher threshold is commonly used for rare conditions, such as [PV],” said Sam Hong, PharmD, co-author and CHOICE fellow at the School of Pharmacy at the University of Washington in Seattle.

The researchers used a 3-state Markov model (hematocrit control, no hematocrit control, and death) to analyze a 15-year period in which 3-month cycles of treatment were used to estimate the economic and health-related quality of life impact of ruxolitinib versus BAT. Costs and outcomes were discounted by 3% annually. The phase 3 RESPONSE-2 trial provided the clinical data, and the published literature were sourced for the cost, utility, and overall survival data.

Investigators found that patients taking ruxolitinib accumulated a mean of 28.5 QALYs (95% CI, 24.9-30.6), resulting in an additional 1.2 QALYs (95% CI, 0.5-2.8) compared with those accumulated by patients in the BAT group, who gained a mean of 27.3 QALYs (95% CI, 23.7-27.0).

Mean total costs for the ruxolitinib arm were $526,100 (95% CI, $428,700-$623,800) compared with $371,700 (95% CI, $310,800-$381-600) for the BAT arm. The mean incremental cost effectiveness ratio (ICER) was $128,600 (95% CI, $82,700-$215,400).

The ICER was most sensitive to change when individual variations were applied to the efficacy of ruxolitinib, the cost of ruxolitinib, and the control state of red blood cell volume.

Previous studies have shown ruxolitinib, a JAK 1/2 inhibitor, to have 7.3 times greater odds (95% CI, 3.4-15.5) than BAT in achieving red blood cell volume control in patients with PV who are resistant or have an intolerance to hydroxyurea.

“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,” the authors concluded.

They noted, however, that additional research is needed to define health state utility values specific to patients with PV and to establish an appropriate surrogate endpoint for survival.


Hong S, Veenstra DL. Cost-utility analysis of ruxolitinib versus best available therapy for the treatment of hydroxyurea resistant/intolerant polycythemia vera without splenomegaly in the United States. Presented at: Virtual ISPOR 2020; May 18-20, 2020.

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