Kulikov A.Y., Protsenko M.V., Serpik V.G. 1119

The effectiveness of public spending on the treatment of spinal muscular atrophy in terms of budget impact analysis

We conducted an budget impact analysis was made of the treatment of spinal muscular atrophy (SMA) with drugs Nusinersen and Risdiplam. Its results showed that from the point of view of the organization of drug provision for patients with SMA at current prices, treatment of patients under 18 years of age within the framework of the responsibility of the Circle of Kindness Foundation, given the current distribution of patients, in which 57.6% receive Nusinersen, and 42.4% Risdiplam, is characterized by a smaller budget already in the first year – 18.82 billion rubles, in comparison with the budget of the simulated distribution, in which 42.4% of patients receive Nusinersen, and 57.6% of Risdiplam – 19.27 billion rubles. Thus, with the current distribution over 5 years, budget savings in comparison with the simulated distribution reaches 3.24 billion rubles, excluding the discount factor, and 2.86 billion rubles, when discounting. Analyzing the long-term effect of the budget impact of the use of Nusinersen drugs per patient at the beginning of therapy at the age of two months, it was found that the total savings before the patient switches to provision through regional funding (reaching 18 years of age) can reach 59.72 million. rub. From the point of view of drug provision for patients with SMA at the regional level, that is, patients over 18 years old, the main pool of patients will be formed due to the successive transfer of patients from the Circle of Kindness Fund, since most of the patients (1039 people) are now provided precisely for the account of the Circle of Kindness Fund, and upon reaching the age of 18, this cohort of patients will be transferred to provision at the expense of the budgets of the regions of the Russian Federation. At the same time, it is noted that the costs of initiation of these patients with Nusinersen were covered by the Circle of Kindness Foundation, and therefore, from the point of view of the regions of the Russian Federation, savings on Nusinersen in comparison with Risdiplam will be noted already in the first year of therapy, based on the fact that the annual costs for Nusinersen maintenance mode are 16,957,680 rubles. per patient, and the annual cost of treatment with Risdiplam drug for one adult patient (weighing more than 20 kg) exceeds 20 million rubles. Thus, the stated results of the budget impact analysis revealed the economic feasibility of Nusinersen in the treatment of SMA in comparison with the Risdiplam.
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