The social-economic burden of spinal muscular atrophy in Russia
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 (m...
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| Format: | Article |
| Language: | Russian |
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IRBIS LLC
2021-02-01
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| Series: | Фармакоэкономика |
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| Online Access: | https://www.pharmacoeconomics.ru/jour/article/view/450 |
| _version_ | 1797878889185280000 |
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| author | A. S. Kolbin D. V. Vlodavets A. A. Kurylev Yu. Ye. Balykina M. A. Proskurin S. A. Mishinova O. Yu. Germanenko N. Yu. Kolbina |
| author_facet | A. S. Kolbin D. V. Vlodavets A. A. Kurylev Yu. Ye. Balykina M. A. Proskurin S. A. Mishinova O. Yu. Germanenko N. Yu. Kolbina |
| author_sort | A. S. Kolbin |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-10T02:40:12Z |
| format | Article |
| id | doaj.art-91dec5cb41064a93b26b711223acd559 |
| institution | Directory Open Access Journal |
| issn | 2070-4909 2070-4933 |
| language | Russian |
| last_indexed | 2024-04-10T02:40:12Z |
| publishDate | 2021-02-01 |
| publisher | IRBIS LLC |
| record_format | Article |
| series | Фармакоэкономика |
| spelling | doaj.art-91dec5cb41064a93b26b711223acd5592023-03-13T07:48:17ZrusIRBIS LLCФармакоэкономика2070-49092070-49332021-02-0113433735410.17749/2070-4909/farmakoekonomika.2020.068319The social-economic burden of spinal muscular atrophy in RussiaA. S. Kolbin0D. V. Vlodavets1A. A. Kurylev2Yu. Ye. Balykina3M. A. Proskurin4S. A. Mishinova5O. Yu. Germanenko6N. Yu. Kolbina7Pavlov First Saint Petersburg State Medical University; Saint-Petersburg State UniversityVeltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical UniversityPavlov First Saint Petersburg State Medical UniversitySaint-Petersburg State UniversitySaint-Petersburg State UniversityPavlov First Saint Petersburg State Medical UniversityCharitable foundation for aid patients with spinal muscular atrophy and other neuromuscular diseases SMA FamiliesAlmazov National Medical Research CenterIntroduction. 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.https://www.pharmacoeconomics.ru/jour/article/view/450spinal muscular atrophydisease burdencost |
| spellingShingle | A. S. Kolbin D. V. Vlodavets A. A. Kurylev Yu. Ye. Balykina M. A. Proskurin S. A. Mishinova O. Yu. Germanenko N. Yu. Kolbina The social-economic burden of spinal muscular atrophy in Russia Фармакоэкономика spinal muscular atrophy disease burden cost |
| title | The social-economic burden of spinal muscular atrophy in Russia |
| title_full | The social-economic burden of spinal muscular atrophy in Russia |
| title_fullStr | The social-economic burden of spinal muscular atrophy in Russia |
| title_full_unstemmed | The social-economic burden of spinal muscular atrophy in Russia |
| title_short | The social-economic burden of spinal muscular atrophy in Russia |
| title_sort | social economic burden of spinal muscular atrophy in russia |
| topic | spinal muscular atrophy disease burden cost |
| url | https://www.pharmacoeconomics.ru/jour/article/view/450 |
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