The clinical use and cost-effectiveness of domestic blood coagulation factors VIII and IX
Authors: Fokina D. S. , Zhukova O. V. , Khokhlov A. L. , Kudlay D. A. , Borozinets A. Yu. , Pilyus F. G. 
Published: March 2022

According to the data from the Federal Center for Planning and Organization of Medicine Provision for Citizens of Ministry of Healthcare of Russia, as of 2021, a total of 11 151 people have been diagnosed with hemophilia, 35.9% of them being children. Here we aimed to analyze data on the use of domestic recombinant coagulation factors VIII and IX in real clinical practice and to evaluate their cost-effectiveness. For our analysis, we used data from an observational study and a comparative clinical study of moroctocog alfa (Octofactor) in patients with moderate and severe hemophilia A as well as data from a comparative clinical study of nonacog alfa (Innonafactor) in patients with moderate and severe hemophilia B. The observational study of moroctocog alfa (Octofactor) lasted 85 weeks (from 09 January 2016 to 01 September 2017) and included 30 Russian clinical centers with a total of 237 patients diagnosed with moderate or severe hemophilia A. All patients were male, aged between 19 and 78 years (the mean age was 35.2 ± 11.1 years). The comparative study of moroctocog alfa included 18 hemophilia patients receiving prophylactic treatment at 3 Russian clinical centers. The mean age in the study cohort was 38.2 ± 12.9 years. Similarly, the comparative study of nonacog alfa (Innonafactor) included 18 hemophilia patients receiving prophylactic treatment at 3 Russian clinical centers. The mean age in this study cohort was 34.1 ± 9.5 years. The clinical studies were approved by the Ethics Board of the Ministry of Healthcare of Russia. The observational study was approved by the Interuniversity Ethics Committee (Moscow) and local ethics committees. A health economic evaluation was conducted using the following methodologies: a cost-effectiveness analysis, a budget impact analysis, an opportunity cost analysis, and a sensitivity analysis. The main measure of moroctocog alfa (Octofactor) effectiveness was the frequency of spontaneous bleeding within 48–72 hours after the administration of a prophylactic drug. The frequency of bleeding episodes was reported to be 1.4 ± 2.9. The main measure of nonacog alfa (Innonafactor) effectiveness was the mean number of bleeding episodes in patients receiving prophylactic treatment. It was reported to be 0.22 ± 0.44 episodes. Our health economic evaluation showed that the cost-effectiveness ratio for moroctocog alfa (Octofactor) was 1.8 fold lower than that for the comparator (Octanate, a plasma-derived factor VIII product); the cost-effectiveness ratio for nonacog alfa (Innonafactor) was 2.1 fold lower than that for the comparator (Octanine F, a coagulation factor IX) meaning that domestic coagulation factor products allow patients to achieve better treatment results (i.e. fewer bleeding episodes) at lower cost. The budget impact and opportunity cost analyses in two model groups of patients (n = 100 each) revealed that the use of moroctocog alfa (Octofactor) would enable us to treat 36 more patients, while the use of nonacog alfa (Innonafactor) would allow 3 more patients to receive care. The sensitivity analysis showed that switching 50% of patients from the foreign comparator to moroctocog alfa (Octofactor) would save 5.32% of the budget, while switching 50% of patients from the foreign comparator (Octanine F) to nonacog alfa (Innonafactor) would save 0.98% of the budget meaning that more patients could receive treatment. It was also demonstrated that changes in the proportion of patients receiving Octofactor and Octanate within the range of ± 50% would result in cumulative costs ranging from ₽12 994 million to ₽15 233 million that correspond to a budget surplus of 5.32% and a budget deficit of 10.99%, respectively. Similarly, treatment costs for Innonafactor and Octanine F would vary from ₽2 473 million to ₽2 516 million that correspond to a budget surplus of 0.98% and a budget deficit of 0.74%, respectively.