In recent years, for a number of life-threatening and chronic progressive rare (orphan) diseases, leading to a reduction in life expectancy of citizens or their disability, the powers to provide patients with drugs have been transferred from the regional level to the federal one. The most expensive diseases that have consistently formed the main financial burden for regional budgets since the creation of the List of the Government of the Russian Federation include paroxysmal nocturnal hemoglobinuria (Marchiafava-Mikeli), idiopathic thrombocytopenic purpura (Evans syndrome), other sphingolipidoses: Fabry disease (Fabry-Anderson), Niemann-Pick, as well as pulmonary (arterial) hypertension (idiopathic) (primary). In order to assess the impact on the budget of these diseases, an analytical decision-making model was developed that allows real-time calculation of how much federal budget funds will be required depending on the number of patients, the cost of drug therapy and the proportion of the patient population receiving a particular treatment. depending on the course of the disease. In addition, the developed model will make it possible to estimate at the regional level the necessary costs for drug provision for these diseases, if this authority remains at the level of the constituent entities of the Russian Federation.

rare diseases, orphan drugs, drug maintenance, healthcare, financing, federalization, budget, regions of the Russian Federation, analytical decision-making model.