The State Expert Center, the authorized body for state health technology assessment (HTA), recommends considering the possibility of negotiating managed access agreements for the medicinal product ruxolitinib.
The relevant state HTA conclusion is published on the Center's website at: http://surl.li/ulwcp
Ruxolitinib is used for the treatment of patients with primary and secondary myelofibrosis of the intermediate-2 and high-risk groups in the presence of splenomegaly.
Disease priority: for which patients is it important?
Myelofibrosis is an orphan (rare), life-threatening myeloproliferative disease. It is accompanied by dysregulation of signaling involving JAK1 and JAK2, progressive replacement of bone marrow with fibrous tissue, and excessive production of cytokines, which leads to pronounced symptoms of tumor intoxication and splenomegaly.
Cancer treatment is one of the priority areas of healthcare development for 2023–2025 (MoH of Ukraine order of 07.10.2022 No. 1832).
About the medicinal product under assessment
The results of the COMFORT-II study established that the use of ruxolitinib has a clinical advantage in reducing spleen volume by ≥35% at week 48 compared with best available therapy (BAT) in patients with primary and secondary myelofibrosis (developed after polycythemia vera or essential thrombocythemia) from intermediate-2 and high-risk groups according to the IPSS, DIPSS, and DIPSS-Plus scales.
A reduction in spleen size of ⩾35% was maintained during long-term therapy (median duration 3,2 years); the probability of maintaining the response, assessed by spleen size, was 0,51 at 3 years and 0,48 at 5 years.
Based on the results of the final 5-year analysis, overall survival was longer with ruxolitinib compared with BAT, although without a statistically significant difference both in the ITT population and in the results of modeling adjusted for the crossover design.
Disease progression at week 48 occurred in 30% of patients in the ruxolitinib group compared with 26% in the best available therapy (BAT) group, but without a statistically significant difference.
At the same time, the use of ruxolitinib compared with best available therapy (BAT) significantly improved quality of life and alleviated disease-related symptoms during the follow-up period of 48 weeks.
UO recommendations
It is recommended to consider negotiating managed access agreements for the medicinal product ruxolitinib (tablets of 5 mg and 15 mg) for the treatment of patients with primary and secondary myelofibrosis (developed after polycythemia vera or essential thrombocythemia) in the intermediate-2 and high-risk groups according to the IPSS, DIPSS and DIPSS-Plus scales in the presence of splenomegaly, in accordance with the restrictions and conditions in the conclusion.
It is also recommended to collect and accumulate real-world evidence, with subsequent analysis of the indicators of clinical efficacy and safety of ruxolitinib use in local clinical practice in Ukraine in patients with primary and secondary myelofibrosis.
