Ropeginterferon Alfa-2B Is Cost-Effective to Manage Low-Risk Patients With Polycythemia Vera As Compared to Phlebotomy Only

OBJECTIVES: Polycythemia vera (PV) is a rare myeloproliferative neoplasm associated with high risk of thrombosis, progression to myelofibrosis, acute myeloid leukemia (AML) and reduced survival. Patients younger than 60 years without prior thrombosis are defined at “low risk”. In a phase 2 trial, ropeginterferon alfa-2b (ropeg) at a dose of 100 µg every 2 weeks, on top of the standard regimen, was superior to phlebotomy alone in steadily maintaining hematocrit (HCT) on target (<45%) in low-risk PV patients. The aim of the present analysis was to assess ropeg cost-effectiveness from the Austrian healthcare system perspective over 30 years.

METHODS: We combined a 12-month decision tree with a semi-Markov cohort model with five health states: Myelofibrosis-free survival (MFS) with HCT control, MFS without HCT control, Post-PV Myelofibrosis, AML, and Death. Outcomes were cumulative costs, quality adjusted life years (QALYs) and incremental cost-utility ratio (ICUR). Probabilities and utilities were derived from published literature and costs from Austrian-specific databases. A 5% discount was applied to costs and QALYs. One-way and probabilistic sensitivity analyses (SA) assessed the robustness of findings.

RESULTS: Ropeg led to 23% overall higher costs (+50,960 EUR, mainly due to drug costs) and 1.43 higher QALYs compared to phlebotomy alone, resulting in an ICUR of 35,525 EUR/QALY. Thrombosis, myelofibrosis, and AML costs decreased for the ropeg group by 12%, 30% and 16% respectively. In the one-way SA, ropeg costs and discount rates mostly impacted on results. The probabilistic SA showed a 100% probability of cost-effectiveness at willingness-to-pay threshold aligned to the Austrian GDP per capita (52,372 EUR).

CONCLUSIONS: Ropeg is a cost-effective treatment option for patients with low-risk PV. These findings suggest that early treatment with ropeg could ensure optimal resource allocation by preventing costly thrombotic events and progression to myelofibrosis whilst increasing patient quality of life.