Ruxolitinib Has Cost-effectiveness, Survival Benefits for Myelofibrosis vs Competitors

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Research presented at EHA2021 shows that the JAK 1/2 inhibitor ruxolitinib produced cost-effective benefits per quality-adjusted life-year and increased the overall survival rate for patients with myelofibrosis.

A pair of abstracts presented at this year’s European Hematology Association 2021 Virtual Congress (EHA2021) show treatment with Jakafi (ruxolitinib; Novartis) is both cost-effective and beneficial for overall survival (OS), vs other treatment options, for myelofibrosis (MF).

In the first study,1 the investigators analyzed the economic utility of ruxolitinib following its 2011 approval by the FDA and the European Medicines Agency. They searched PubMed, Embase, MEDLINE, and the Cochrane Library—all studies had to be published in English—using the terms “myelofibrosis,” “ruxolitinib,” “best available therapy/standard of care,” and “cost-effectiveness/cost-utility/pharmacoeconomics.”

“UK-based estimates of the cost of treatment are in the region of £43,000 per patient, per year,” the authors wrote. “Against the background of the challenge of treatments for rare diseases reaching cost-effectiveness thresholds, this study identified, synthesized and appraised the available evidence on the cost-effectiveness of ruxolitinib in the treatment of MF.”

Their final analysis included trials from the United Kingdom, Portugal, Chile, Canada, and Finland.

The comparison of ruxolitinib against best available therapy (BAT; hydroxyurea, no medication, and prednisone/prednisolone) produced the following incremental cost-effectiveness ratios per quality-adjusted life-year (QALY), “converted into USD, if appropriate, at the historic average annual exchange rate”:

  • £44,905 in the UK (in 2013; 70,262 USD), for 2.51 additional QALYs vs BAT
  • €40,000 in Portugal (in 2016; 44,272 USD), for 2.43 additional QALYs vs BAT
  • 54,500 USD in Chile (in 2016), for 0.98 additional QALYs vs BAT
  • 61,444 CAD in Canada (in 2012; 61,474 USD), for 1.19 additional QALYs vs BAT
  • €42,367 in Finland (in 2015; 42,027 USD), for 2.43 additional QALYs vs BAT

Although the few sources of data were determined to be a limitation on the generalizability of these findings, “ruxolitinib was found to be cost-effective for the treatment of patients with myelofibrosis informed by different types of models and from different perspectives,” the authors concluded.

In the ERNEST project2—originally started in 2013 to gather data on patients with MF on ruxolitinib treatment—investigators analyzed the survival improvement of ruxolitinib vs hydroxyurea, because this outcome remains uncertain, they note. “The aim was to collect estimates of clinical outcome of contemporary myelofibrosis patients in real-life settings,” they added.

ERNEST participants from Italy, Spain, and Sweden who were alive between November 2014 and December 31, 2018, were followed from treatment initiation to the first of death or last study contact/study end date. All had a history of previous cytoreductive therapy. Propensity score matching for age at first administration, gender, type of MF, Dynamic International Prognostic Scoring System (DIPSS) at first administration, year of diagnosis, palpable spleen at first administration, and time to first administration ensured patient exposure to hydroxyurea or ruxolitinib.

Most of the 1010 patients in this analysis had primary MF (PMF; 68%), followed by post–polycythemia vera (22%), and post–essential thrombocythemia (20%). For the period evaluated, most diagnoses occurred between 2009 and 2012 (40%), followed by 2005 to 2008 (37%) and 2001 to 2004 (23%). The median follow-up was 5.2 (95% CI, 2.3-8.2) years, during which there were 625 deaths, and the median OS was 6.2 (95% CI, 2.8-12.6) years.

In addition, during the follow-up period, patients receiving ruxolitinib at first administration were younger, had massive splenomegaly, and more constitutional symptoms compared with patients receiving hydroxyurea. In addition, 64% of patients receiving ruxolitinib had a hydroxyurea treatment history.

Overall, the patients receiving ruxolitinib had a 31% longer OS (6.7 vs 5.1 years; P = .001), which increased to more than double the rate of patients receiving hydroxyurea with a higher DIPSS category (6.4 vs 3.0 years; P = .003). Age, male gender, and high DIPSS category had a negative correlation with OS, while a more recent diagnosis and ruxolitinib exposure positively correlated with OS.

“Within the limitations of an observational study, these real-life data support an overall survival benefit of ruxolitinib over hydroxyurea alone in patients with primary and secondary ML,” the authors concluded.


1. Karakuleli G, Barham L. Cost-effectiveness of ruxolitinib for patients with myelofibrosis: a review of the literature. Presented at: EHA2021; June 10-13, 2021; virtual. Abstract PB1750.

2. Guglielmelli P, Ghirardi A, Carobbio A, et al. Impact of ruxolitinib on survival of patients with myelofibrosis in real world—update of ERNEST (European Registry for Myeloproliferative Neoplasms) Study. Presented at: EHA2021; June 10-13, 2021; virtual.