PP409 Cost-Effectiveness Of Ruxolitinib For Patients With Myelofibrosis: A Review Of The Literature

Autor: Gizem Karakuleli, Leela Barham
Rok vydání: 2021
Předmět:
Zdroj: International Journal of Technology Assessment in Health Care. 37:35-35
ISSN: 1471-6348
0266-4623
DOI: 10.1017/s0266462321001549
Popis: IntroductionMyelofibrosis (MF) is a rare (annual incidence estimated to be 1/100,000 in Europe), chronic hematologic disorder associated with morbidity and mortality as well as the risk of evolution to acute myeloid leukemia. Ruxolitinib (Jakavi®, Novartis) is the first JAK 1/2 inhibitor approved by the FDA and EMA in 2011 in treating MF. Ruxolitinib is considered a high-cost and life-time treatment. UK-based estimates of the cost of treatment are in the region of GBP43,000/year/patient (in 2013). Against the background of the challenge of treatments for rare diseases reaching cost-effectiveness thresholds, this study identified, collected, and appraised the available evidence on the cost-effectiveness of ruxolitinib in the treatment of MF.MethodsA systematic approach was taken to conducting the literature review. Databases searched included PubMed, EMBASE, MEDLINE, and the Cochrane Library based on search terms informed by PICO: myelofibrosis, ruxolitinib, best available therapy/standard of care, and cost-effectiveness/cost-utility/pharmacoeconomics. The search was limited to studies published in the English language. A narrative synthesis was used to evaluate studies and the CHEERS checklist to explore the quality of reporting of the cost-effectiveness analysis.ResultsThe narrative synthesis included five studies conducted in the UK, Portugal, Chile, Canada, and Finland. All cost-effectiveness analyses used data from the same two large, randomized controlled, double-blind, phase III studies (COMFORT-I and -II). Ruxolitinib was compared to the best available therapy (BAT), including hydroxyurea, no medication, and prednisone/prednisolone. Perspectives and included costs varied among analyses. Markov models and discrete state cohort models were used to evaluate the cost-effectiveness and clinical benefit was measured in quality-adjusted life years (QALY) or life years (LY) gained.These analyses estimated the base-case incremental cost-effectiveness ratios (ICER) per QALY of (converted into USD, if appropriate, at the historic average annual exchange rate) GBP44,905 in the UK (2013; USD 70,226), EUR40,000 in Portugal (2016; USD44,272), USD54,500 (2016), CAD61,444 in Canada (2012; USD61,474), and EUR42,367 in Finland (2015; USD42,027). Based upon the cost-effectiveness thresholds applied in each of these countries, ruxolitinib was found to be universally cost-effective, albeit with price adjustments as part of the wider pricing and reimbursement processes used in these countries.ConclusionsRuxolitinib was found to be cost-effective in treating MF informed by different types of models and from different perspectives; however, there was some uncertainty around available data due to limited data sources.
Databáze: OpenAIRE
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