Original Title: Ruxolitinibe para tratamento de pacientes com mielofibrose primária, mielofibrose pós policitemia vera ou mielofibrose pós trombocitemia essencial, de risco intermediário-2 ou alto

Authors' objectives: Is ruxolitinib effective, safe and cost-effective for the treatment of intermediate-2 or high-risk myelofibrosis, classified according to the International Prognostic Scoring System (IPSS), when compared with placebo or the best available therapy?

Authors' results and conclusions: The Conitec’s members present at the 87th Ordinary Meeting, on June 4th, 2020, unanimously decided not to recommend the incorporation of ruxolitinib for the treatment of patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis, in people with intermediate-2 or high-risk disease and with a platelet count of more than 50,000/mm3. The Deliberation Record No. 523/2020 was signed.

Authors' recommendations: Scientific evidence: The evidence demonstrated that ruxolitinib provided benefits in relieving symptoms of the disease (odds ratio [OR] 15.3; 95% Confidence Interval [CI] 6.9-33.7). The long-term data from COMFORT-I and II trials showed a statistically significant difference in overall survival for ruxolitinib (about five years). Reduction in spleen size ≥35% was observed in all subtypes of myelofibrosis and in patients with intermediate-2 or high-risk disease. Anaemia was the most common grade 3 or 4 Adverse Event (AE) in both COMFORT-I and II trials, followed by thrombocytopenia. These AEs rarely led to discontinuation and were generally managed by dose modifications or blood transfusions. Budget impact analysis: The incremental value of incorporating ruxolitinib was estimated to be approximately BRL 44 million in the first year, and BRL 300 million after five years. The main limitation of the data was the eligible population. There were no epidemiological data published for Brazil, therefore, the applicant made some assumptions, such as considering the prevalence data for the Federal District representative for the entire Brazilian territory. In the international literature, the prevalence of any myelofibrosis ranged from 0.51 to 2.7/100,000 patients. The impact of incorporating ruxolitinib into the Brazilian Public Health System (SUS) was unclear. Considerations: Low-quality evidence showed that ruxolitinib demonstrated superiority compared to placebo or the best available therapy in the treatment of PMF and post-PV/ET MF, with higher rates of splenomegaly control, improvement of symptoms and quality of life. There is uncertainty on overall survival, but in general, patients treated with ruxolitinib had longer survival than control patients. The responses were sustained for a median period lower than five years, and the benefit occurred regardless of the myelofibrosis subtype, age group, presence or absence of the JAK2 V617F mutation, haemoglobin levels, and platelet counts.

Project Status: Completed

URL for project: http://conitec.gov.br/images/Consultas/Relatorios/EN2020/20210106_Report531_Ruxolitinib_PrimaryMyelofibrosis-PostpolycythaemiaVeraMyelofibrosis-PostessentialThrombocythaemiaMyelofibrosis.pdf

Year Published: 2020

URL for published report: http://conitec.gov.br/images/Relatorios/2020/Ruxolitinibe_mielofibrose_531_2020_FINAL.pdf

URL for additional information: http://conitec.gov.br/recomendation-reports

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Brazil

Organisation Name: National Committee for Technology Incorporation (CONITEC)

Contact Address: Esplanada dos Ministérios, Bl. G, Ed. Sede, 8º andar, CEP: 70058-900

Contact Name: Clarice Moreira Portugal

Contact Email: clarice.portugal@saude.gov.br