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The social-economic burden of spinal muscular atrophy in Russia

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

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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



References

1. Vlodavets D.V., Kharlamov D.A., Artemyeva S.B., Belousova E.D. Federal clinical guidelines (protocols) for the diagnosis and treatment of spinal muscular atrophy in children. [Electronic resource] URL: http://ulgb3.ru/doc/211218_10-58.pdf. Accessed: 20.07.2020. (in Russ).

2. Sokolova M.G., Alexandrov N.Yu., Lobzin S.V. Spinal muscular atrophy in children: etiology, pathogenesis, diagnosis and treatment principles. Bulletin of the North-Western State Medical University named after I.I. Mechnikov. 2013; 5 (4): 108-113 (in Russ).

3. Mercuri E., et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018; 28 (2): 103-115.

4. Finkel R.S., et al. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscul Disord. 2018; 28 (3): 197-207.

5. Lally C., et al. Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States. Orphanet J Rare Dis. 2017; 12 (1): 175.

6. Oskoui M., et al. The changing natural history of spinal muscular atrophy type 1. Neurology. 2007; 69 (20): 1931-6.

7. Mannaa M.M., et al. Survival probabilities of patients with childhood spinal muscle atrophy. J Clin Neuromuscul Dis. 2009; 10 (3): 85-9.

8. Chung B.H., et al. Spinal muscular atrophy: survival pattern and functional status. Pediatrics. 2004; 114: e548-e553.

9. Gillingwater T.H., et al. Counting the cost of spinal muscular atrophy. J Med Econ. 2016; 19 (8): 827-8.

10. Droege M., et al. Economic burden of spinal muscular atrophy in the United States: a contemporary assessment. J Med Econ. 2020; 23 (1): 70-79.

11. Armstrong E.P., et al. The economic burden of spinal muscular atrophy. J Med Econ. 2016; 19 (8): 822-6.

12. Order of the Ministry of Health of the Russian Federation of December 29, 2012 No. 1741n “On the approval of the standard of primary health care for children with spinal muscular atrophies” (in Russ).

13. General Tariff Agreement for OMS of St. Petersburg, 2020 [Electronic resource] URL: https://spboms.ru/page/docs. Accessed: 20.07.2020 (in Russ).

14. State register of maximum selling prices for medicines. [Electronic resource]. URL: http://grls.rosminzdrav.ru/. Accessed: 20.07.2020 (in Russ).

15. Unified information system in the field of procurement. [Electronic resource] URL: https://zakupki.gov.ru/epz/main/public/home.html. Accessed: 20.07.2020 (in Russ).

16. Information about the availability of drugs in pharmacies in Moscow. [Electronic resource] URL: https://www.medlux.ru/compare?ids=46220. Accessed: 20.07.2020 (in Russ).

17. Decree of the Government of the Russian Federation of December 7, 2019 N 1610 “On the Program of State Guarantees of Free Provision of Medical Care to Citizens for 2020 and for the Planning Period of 2021 and 2022” Accessed: 20.07.2020 (in Russ).

18. Federal Fund of Compulsory Health Insurance. Ministry of Health of the Russian Federation. Methodical recommendations on methods of payment for medical care at the expense of compulsory medical insurance. Moscow, 2019 (in Russ).

19. Laboratory service Helix. [Electronic resource] URL: https://helix.ru/kb/item/42-079. Accessed: 20.07.2020 (in Russ).

20. City multidisciplinary hospital No. 2, price list of services. [Electronic resource] URL: http://gmpb2.ru/platnye-uslugi/prejskuranty-uslug. Accessed: 20.07.2020 (in Russ).

21. St. Petersburg Clinical Hospital of the Russian Academy of Sciences. [Electronic resource] URL: https://poliran.ru/kliniko-diagnosticheskaja-laboratorija/. Accessed: 20.07.2020 (in Russ).

22. St. Petersburg City Pokrovskaya Hospital [Electronic resource] URL: http://www.pokrov.spb.ru/assortiment/medic/laboratory/list_1.html. Accessed: 20.07.2020 (in Russ).

23. Moscow Children’s City Clinical Hospital named after N.F. Filatov DZM. Electronic resource. [Electronic resource] URL: https://filatovmos.ru/plat/pricelist.html. Accessed: 20.07.2020 (in Russ).

24. Medical Genetic Center. [Electronic resource] URL: https://www.spbmgc.ru. Accessed: 20.07.2020 (in Russ).

25. Hospital for war veterans [Electronic resource] URL: http://gvv-spb.ru/preyskurant-na-mrt. Accessed: 20.07.2020 (in Russ).

26. Federal State Budgetary Institution “Russian Rehabilitation Center” Childhood “[Electronic resource] URL: http://rrcdetstvo.ru/Files/%D0%BF%D1%80%D0%B5%D0%B9%D1%81%D0%BA%D1%83%D1%80%D0%B0%D0%BD%D1%82%20%D1%86%D0%B5%D0%BD.pdf. Accessed: 20.07.2020 (in Russ).

27. Psychoneurological dispensary №8 [Electronic resource] URL: http://pnd8.spb.ru/platnye-uslugi.html. Accessed: 20.07.2020 (in Russ).

28. FSBI RosNIIGT FMBA of Russia. [Electronic resource] URL: http://www.bloodscience.ru/patient/price/. Accessed: 20.07.2020 (in Russ).

29. Darba J. Management and current status of spinal muscular atrophy: a retrospective multicentre claims database analysis. Orphanet J Rare Dis. 2020; 15 (1): 8.

30. LLC “Medpribor medical equipment”. Price-list. [Electronic resource] URL: https://medpribor.pro/pc/cpap-bipap-terapiya-i-respiratornaya-podderzhka/cpap-bipap-pribory/ustrojstvo-ventilyatsii-legkih/. Accessed: 20.07.2020 (in Russ).

31. LLC “NBC”. Price-list. [Electronic resource] URL: https://www.oxy2.ru/catalog/apparaty_dlya_otvedeniya_mokroty/. Accessed: 20.07.2020 (in Russ).

32. Federal Law of 15.12.2001 N 166-FZ (as amended on 01.10.2019) “On state pension provision in the Russian Federation” (in Russ).

33. Decree of the President of the Russian Federation of March 7, 2019 N 95 “On amendments to the Decree of the President of the Russian Federation of February 26, 2013 N 175” On monthly payments to persons caring for children with disabilities and persons with disabilities from childhood of group I” (in Russ).

34. Order of January 31, 2011 N 57n “On approval of the procedure for payment of compensation for independently acquired by a disabled person technical means of rehabilitation and (or) the service provided, including the procedure for determining its size and the procedure for informing citizens about the amount of this compensation” (in Russ).

35. Order of the Government of the Russian Federation of December 30, 2005 No. 2347-r “On approval of the classification of technical means of rehabilitation (products) within the federal list of rehabilitation measures, technical means of rehabilitation and services provided to a disabled person” (in Russ).

36. Order of the Ministry of Labor and Social Protection of the Russian Federation of February 13, 2018 N 85n “On approval of the terms of use of technical means of rehabilitation, prostheses and prosthetic and orthopedic devices before their replacement” (in Russ).

37. WHO. The global burden of disease: 2004 update: Geneva: World Health Organization; 2008 2008; 146 p.

38. IHME. The Global Burden of Disease: Generating Evidence, Guiding Policy. Seattle: Institute for Health Metrics and Evaluation; 2013. 50 p.

39. Daems R., Maes E., Mehra M., Carroll B., Thomas A. Pharmaceutical portfolio management: global disease burden and corporate performance metrics. Value Health. 2014; 17 (6): 732-8.

40. Klug C., et al. Disease Burden of Spinal Muscular Atrophy in Germany. Orphanet J Rare Dis. 2016; 11 (1): 58. https://doi.org/10.1186/s13023-016-0424-0.

41. Avksentieva M.V. et al. Economic feasibility of long-term artificial lung ventilation at home in the provision of palliative care for children. Palliative medicine and rehabilitation. 2016; 2: 40-49 (in Russ).

42. Order of the Government of St. Petersburg dated August 28, 2019 N 31-rp “On approval of the St. Petersburg program” Development of the palliative care system for 2019-2024” (in Russ).

43. Law of St. Petersburg “On the Territorial program of state guarantees for free provision of medical care to citizens in St. Petersburg for 2019 and for the planning period of 2020 and 2021” (in Russ).


For citation:


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

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