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Research Article
2 August 2023

Cost–effectiveness of ropeginterferon alfa-2b-njft for the treatment of polycythemia vera

Authors: Aaron T Gerds https://orcid.org/0000-0002-3422-1309, Claudia Castro, Frank Snopek, Megan M Flynn, Alexandra G Ellis https://orcid.org/0000-0002-5128-0255, Meredith Manning, and Ray Urbanski [email protected]Author Info & Affiliations
Volume 12, Number 9
https://doi.org/10.57264/cer-2023-0066
PDFFormats

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 (BESREMi®) 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 USD60,175, resulting in a cost per QALY of USD141,783. 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.

Plain language summary

What is the article about?

This research analyzed the costs and benefits of a therapy called ropeginterferon alfa-2b-njft used to treat patients with polycythemia vera (PV), which is a rare type of blood cancer.

How was the research carried out?

A model was created that compared ropeginterferon alfa-2b-njft used either as the first or second therapy option for patients with PV versus an alternative treatment pathway without ropeginterferon alfa-2b-njft. The data from randomized clinical trials and from real-world sources were included. The model followed patients from treatment initiation through their lifetime and tracked blood clot events and disease progression. Patients who experienced blood clots or disease progression had higher risk of death, higher healthcare costs, and lower health-related quality of life. The lifetime total costs, years alive, and quality-adjusted life years (QALYs) were compared between the alternative treatment pathways.

What were the results?

Over a lifetime, the model showed that patients who received ropeginterferon alfa-2b-njft had more years alive (0.4), and higher QALYs (0.4), and higher cost (USD60,175). Weighing the additional costs versus the additional QALY gains results in a cost per QALY of USD141,783.

What do the results mean?

The results suggest the benefits of treatment with ropeginterferon alfa-2b-njft outweigh the costs for a broad range of patients with PV.

Supplementary Material

File (supplementary figure.docx)

References

Papers of special note have been highlighted as: • of interest; •• of considerable interest
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