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The social-economic burden of spinal muscular atrophy in Russia

https://doi.org/10.17749/2070-4909/farmakoekonomika.2020.068

Abstract

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.

About the Authors

A. S. Kolbin
Pavlov First Saint Petersburg State Medical University; Saint-Petersburg State University
Russian Federation

Aleksei S. Kolbin - MD, Dr Sci Med, Professor, Head of the Department of Clinical Pharmacology and Evidence-based Medicine Pavlov SMU; Professor, Department of Pharmacology Saint-Petersburg SU;  President of the St. Petersburg branch of ISPOR (International Society for Pharmacoeconomic Research and Scientific Analysis of Outcomes).

6-8 L'va Tolstogo Str., Saint Petersburg197022; 7-9 Universitetskaya Emb., Saint Petersburg199034; 154 riv. Fontanka Emb., Saint Petersburg190103



D. V. Vlodavets
Veltischev Research and Clinical Institute for Pediatrics of the Pirogov Russian National Research Medical University
Russian Federation

Dmitry V. Vlodavets - MD, PhD, Head of the Russian Children's Neuromuscular Center; Senior Researcher, Department of Psychoneurology and Epileptology; Associate Professor of the Department of Neurology, Neurosurgery and Medical Genetics named after academician L.O. Badalyan Faculty of Pediatrics Pirogov Russian NRMU.

2 Taldomskaya Str., Moscow 125412



A. A. Kurylev
Pavlov First Saint Petersburg State Medical University
Russian Federation

Aleksey A. Kurylev - MD, PhD, Assistant at the Department of Clinical Pharmacology and Evidence-based Medicine.

6-8 L'va Tolstogo Str., Saint Petersburg197022



Yu. Ye. Balykina
Saint-Petersburg State University
Russian Federation

Yuliya E. Balykina - PhD (Physics & Math), Associate Professor, Department of Mathematical Modeling of Energy Systems, Faculty of Applied Mathematics and Control Processes.

7-9 Universitetskaya Emb., Saint Petersburg199034; 154 riv. Fontanka Emb., Saint Petersburg190103



M. A. Proskurin
Saint-Petersburg State University
Russian Federation

Maxim A. Proskurin - Researcher, Department of Mathematical Modeling of Energy Systems, Faculty of Applied Mathematics and Control Processes.

7-9 Universitetskaya Emb., Saint Petersburg199034; 154 riv. Fontanka Emb., Saint Petersburg190103



S. A. Mishinova
Pavlov First Saint Petersburg State Medical University
Russian Federation

Sofya A. Mishinova - Assistant at the Department of Clinical Pharmacology and Evidence-based Medicine.

6-8 L'va Tolstogo Str., Saint Petersburg197022



O. Yu. Germanenko
Charitable foundation for aid patients with spinal muscular atrophy and other neuromuscular diseases SMA Families
Russian Federation

Olga Y. Germanenko - Founder and director of the SMA Family Charitable Foundation for Patients with Spinal Muscular Atrophy and Other Neuromuscular Diseases, member of the Assembly of the European Association "SMA Europe", member of the Expert Council on Rare (Orphan) Diseases of the Health Protection Committee of the State Duma of the Federal Assembly of the Russian Federation.

48 Borisovskie Prudy Str., Moscow 115408



N. Yu. Kolbina
Almazov National Medical Research Center
Russian Federation

Natalia Yu. Kolbina - Head of the department of pediatrics and medical rehabilitation for children №1.

2 Akkuratova Str., St Petersburg 197341



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Review

For citations:


Kolbin A.S., Vlodavets D.V., Kurylev A.A., Balykina Yu.Ye., Proskurin M.A., Mishinova S.A., Germanenko O.Yu., Kolbina N.Yu. The social-economic burden of spinal muscular atrophy in Russia. FARMAKOEKONOMIKA. Modern Pharmacoeconomics and Pharmacoepidemiology. 2020;13(4):337-354. (In Russ.) https://doi.org/10.17749/2070-4909/farmakoekonomika.2020.068

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