OBJECTIVES: The objectives were the economic evaluation of the pathogenetic therapy of paroxysmal nocturnal hemoglobinuria (PNH) and the determination of the consequences of its low effectiveness - the development of breakthrough hemolysis episodes (BHE).

METHODS: The population included patients with PNH receiving eculizumab pathogenetic therapy (PT). The following types of pharmacoeconomic analyses were performed: cost of illness (CIA), cost-effectiveness (CEA), and budget impact (BIA). The method for assessing CIA involved modeling costs associated with PT and development of the underlying disease complications. The dynamics of the drug provision cost for patients with PNH requiring a change in eculizumab administration was evaluated, which considered the selected distribution scenario for shares between the options for managing patients with PNH.

RESULTS: In Russia, eculizumab PT for PNH requires up to 16.5 billion rubles for 619 patients. Treatment of patients who exhibit low control over the disease symptoms and require correction of the eculizumab dosage is more expensive than the standard eculizumab use: with an increase of the dose to 1200 mg every 14 ± 2 days, the cost increases from 25.5 mln rubles to 33.9 mln rubles; with a reduction of the interval between injections (900 mg every 10 ± 2 days) - up to 35.1 mln rubles; with a simultaneous increase in dosage and a reduction of the interval between injections (1200 mg every 10 ± 2 days ) - up to 46.4 mln rubles. Modeling of the various frequency distribution of therapeutic approaches demonstrates a budget savings of 100 mln rubles.

CONCLUSIONS: Patients with BHE increase the cost for the target population to 3.4 bln rubles due to the need for therapy correction. The most frequently used approach in Russia for the correction of insufficiently effective eculizumab therapy by reducing the interval between injections is a more resource-intensive technique than the tactics of increasing the dosage.