Abstract
Aim: Patients with polycythemia vera (PV), a rare and chronic blood cancer, are at a higher risk for thromboembolic events, progression to myelofibrosis, and leukemic transformation. In 2021, ropeginterferon alfa-2b-njft (BESREMiR○) was approved in the US to treat adults with PV. The purpose of this study is to estimate the cost–effectiveness of ropeginterferon alfa-2b-njft, used as a first-or second-line treatment, for the treatment of patients with PV in the US. Materials & methods: A Markov cohort model was developed from the healthcare system perspective in the United States. Model inputs were informed by the PROUD-PV and CONTINUATION-PV studies and published literature. The model population included both low-risk and high-risk patients with PV. The model compared ropeginterferon alfa-2b-njft used either as first-or second-line versus an alternative treatment pathway of first-line hydroxyurea followed by ruxolitinib. Results: Over the modeled lifetime, ropeginterferon alfa-2b-njft provided an additional 0.4 higher quality-adjusted life years (QALYs) and 0.4 life-years with an added cost of $60,175, resulting in a cost per QALY of $141,783. The model was sensitive to treatment costs, the percentage of patients who discontinue hydroxyurea, the percentage of ropeginterferon alfa-2b-njft users who switch to monthly dosing, the percentage of ropeginterferon alfa-2b-njft users as 2nd line treatment, and the treatment response rates. A younger patient age at baseline and a higher percentage of patients with low-risk disease improved the cost–effectiveness of ropeginterferon alfa-2b-njft. Conclusion: Ropeginterferon alfa-2b-njft is a cost-effective treatment option for a broad range of patients with PV, including both low-and high-risk patients and patients with and without prior cytoreductive treatment with hydroxyurea.
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Gerds, A. T., Castro, C., Snopek, F., Flynn, M. M., Ellis, A. G., Manning, M., & Urbanski, R. (2023). Cost–effectiveness of ropeginterferon alfa-2b-njft for the treatment of polycythemia vera. Journal of Comparative Effectiveness Research, 12(9). https://doi.org/10.57264/cer-2023-0066
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