OBJECTIVES: The aim of the study was to determine the prospects for the use of ravulizumab in pathogenetic therapy (PT) of Paroxysmal Nocturnal Hemoglobinuria (PNH).

METHODS: The study population included PNH patients receiving PT. The focus was on patients with insufficient control over the disease symptoms, when the change of the eculizumab dose was required. Cost minimization (CMA), cost of illness (CIA), and budget impact (BIA) analyses were performed. "Days per year outside treatment" indicator was calculated, which reflects the number of days per year without the use of any therapy associated with PNH.

RESULTS: PNH patient management is characterized by high costs. The lowest costs were observed in the group of patients with compensation who were undergoing eculizumab therapy - 25.5 million rubles; ravulizumab treatment was more expensive - 26.5 million rubles. The use of ravulizumab is the least expensive among approaches for patients with improvement, since the dosage regimen adjustment of eculizumab determines the treatment cost increase: with respect to ravulizumab therapy, the cost difference was 7,431,805 rubles per year in case of increasing dosages, 8,627,957 rubles in the case of a reduction in the administration interval, and 19,944,588 rubles in case of therapy correction in two ways at the same time. The introduction of ravulizumab for patients with the phenomena of pharmacokinetic hemolysis (PKH) will lead to a reduction in the PT cost in Russia by 616.1 million rubles. “Days per year, out of treatment” indicator was considered as an effectiveness criterion. Its highest value was observed when using ravulizumab - 355 days. When using eculizumab, the indicator ranged from 261 to 335.

CONCLUSIONS: The use of ravulizumab in comparison with eculizumab in patients with symptoms of PNH and insufficient control over the disease symptoms is appropriate from the CMA point of view, and preferred from the BIA perspective.