TY - JOUR

T1 - Анализ социально-экономического бремени спинальной мышечной атрофии в Российской Федерации

AU - Kolbin, A. S.

AU - Vlodavets, D. V.

AU - Kurylev, A. A.

AU - Balykina, Yu Ye

AU - Proskurin, M. A.

AU - Mishinova, S. A.

AU - Germanenko, O. Yu

AU - Kolbina, N. Yu

N1 - Publisher Copyright: © 2018 Slovenske Divadlo. Copyright: Copyright 2021 Elsevier B.V., All rights reserved.

PY - 2020

Y1 - 2020

N2 - Introduction. Spinal muscular atrophies (SMA) are clinically and genetically heterogeneous congenital orphan diseases that lead to progressive spinal motoneurons degeneration and loss of their function. There are 4 types of SMA with type I being the most severe. SMA patients need lots of services (medical, social etc.) throughout their life and the cost of the care has never been calculated in Russia before. Aim. The study aims to calculate the socio-economic burden of SMA in the Russian Federation for direct medical procedures and indirect costs before the introduction of pathogenetic therapy and after its implementation. The assessment of the impact of pathogenetic therapy on the dynamics of the socio-economic burden of SMA in the Russian Federation was performed. Materials and methods. The data from patient-reported SMA registry, insurance medical companies, epidemiological, and rehabilitation care data were compiled into the model. The authors accounted for direct medical (diagnostics, drugs, surgery, orthopedics, rehabilitation, and family) and nonmedical expenditures including indirect (loss of GDP) costs. Characteristics of the patient population have been taken from the national SMA register. The sources of costs data included governmental healthcare and insurance companies' tariffs, price lists of commercial companies, clinics, and laboratory services. The modeling time horizon was 5 years. The two following calculation scenarios were evaluated: 1) none of the patients with SMA receive pathogenic therapy 2) the share of pathogenic therapy is stable during the time horizon at the current level. Results. National SMA register contains the information about 998 patients; 21% of them have SMA I type. In the first scenario, the total social-economic burden of SMA in Russia was 2.37 billion RUR/year (2.38 million RUR/patient/year); the share of in-patient care and rehabilitation were 30.8% and 32.3%, respectively. SMA type I burden was 1.6 billion RUR/year (1.5 million RUR/patient/year). In the second scenario, the total SMA burden was 5.4 billion RUR/year, the highest share of pathogenetic therapy was 43%. Conclusion. The SMA economic burden in Russia is significant and growing along with the increase in the share of pathogenic therapy, but this growth should be compensated by lowering other direct and indirect costs.

AB - Introduction. Spinal muscular atrophies (SMA) are clinically and genetically heterogeneous congenital orphan diseases that lead to progressive spinal motoneurons degeneration and loss of their function. There are 4 types of SMA with type I being the most severe. SMA patients need lots of services (medical, social etc.) throughout their life and the cost of the care has never been calculated in Russia before. Aim. The study aims to calculate the socio-economic burden of SMA in the Russian Federation for direct medical procedures and indirect costs before the introduction of pathogenetic therapy and after its implementation. The assessment of the impact of pathogenetic therapy on the dynamics of the socio-economic burden of SMA in the Russian Federation was performed. Materials and methods. The data from patient-reported SMA registry, insurance medical companies, epidemiological, and rehabilitation care data were compiled into the model. The authors accounted for direct medical (diagnostics, drugs, surgery, orthopedics, rehabilitation, and family) and nonmedical expenditures including indirect (loss of GDP) costs. Characteristics of the patient population have been taken from the national SMA register. The sources of costs data included governmental healthcare and insurance companies' tariffs, price lists of commercial companies, clinics, and laboratory services. The modeling time horizon was 5 years. The two following calculation scenarios were evaluated: 1) none of the patients with SMA receive pathogenic therapy 2) the share of pathogenic therapy is stable during the time horizon at the current level. Results. National SMA register contains the information about 998 patients; 21% of them have SMA I type. In the first scenario, the total social-economic burden of SMA in Russia was 2.37 billion RUR/year (2.38 million RUR/patient/year); the share of in-patient care and rehabilitation were 30.8% and 32.3%, respectively. SMA type I burden was 1.6 billion RUR/year (1.5 million RUR/patient/year). In the second scenario, the total SMA burden was 5.4 billion RUR/year, the highest share of pathogenetic therapy was 43%. Conclusion. The SMA economic burden in Russia is significant and growing along with the increase in the share of pathogenic therapy, but this growth should be compensated by lowering other direct and indirect costs.

KW - Cost

KW - Disease burden

KW - Spinal muscular atrophy

UR - http://www.scopus.com/inward/record.url?scp=85102186439&partnerID=8YFLogxK

U2 - 10.17749/2070-4909/FARMAKOEKONOMIKA.2020.068

DO - 10.17749/2070-4909/FARMAKOEKONOMIKA.2020.068

M3 - статья

AN - SCOPUS:85102186439

VL - 13

SP - 337

EP - 354

JO - Farmakoekonomika

JF - Farmakoekonomika

SN - 2070-4909

IS - 4

ER -