Preview

FARMAKOEKONOMIKA. Modern Pharmacoeconomic and Pharmacoepidemiology

Advanced search

Health-economic evaluation of risdiplam (Evrysdi®) in patients with spinal muscular atrophy

https://doi.org/10.17749/2070-4909/farmakoekonomika.2021.101

Full Text:

Abstract

Background. Spinal muscular atrophies (SMA) are clinically and genetically heterogenous inherited orphan diseases leading to progressive spinal motoneurons degeneration and loss of function. Risdiplam and nusinersen are both authorized in Russia for pathogenic SMA treatment and included in the list of vital and essential drugs.

Objective: health-economic evaluation of risdiplam (Evrysdi®) in patients with SMA.

Material and methods. The health-economic analysis was done in accordance with local Russian regulation. Cost-minimization analysis was used. We accounted for direct medical costs of pathogenic treatment of SMA, adverse events correction, and supportive care. Decision tree analysis was used. In budget impact analysis the rise of risdiplam share from 0% to 8.5% during 3 years was modelled. Time horizon was 3 years. Probabilistic sensitivity analysis was done.

Results. Total direct medical costs for risdiplam (53,372,153 rubles for one patient in 3 years) were by 21.1% (14,968,427.82 rubles) lower comparing to nusinersen. The most pronounced difference in favor of risdiplam (41,9%) was during first treatment year. The rise of risdiplam share from 0% to 8.5% during 3 years is associated with lower budget costs by 13.9% in 3 years.

Conclusion. Risdiplam is economically more effective comparing to nusinersen, because having equal effectiveness, it’s use is associated with lower direct medical costs.

About the Authors

А. S. Kolbin
Pavlov University; Saint Petersburg State University
Russian Federation

Aleksey S. Kolbin – Dr. Med. Sc., Professor, Chief of Chair Clinical Pharmacology and Evidence-Based Medicine; Professor, Chair of Pharmacology, Faculty of Medicine;
Scopus Author ID: 19836020100; WoS ResearcherID: G-5537-2015; РИНЦ SPIN-код: 7966-0845.

6-8 Lev Tolstoy Str., Saint Petersburg 197022;
7-9 Universitetskaya Naberezhnaya, Saint-Petersburg 199034



А. А. Kurylev
Pavlov University
Russian Federation

Aleksey A. Kurylev – MD, PhD, Assistant Professor, Chair Clinical Pharmacology and Evidence-Based Medicine;
WoS ResearcherID: O-2630-2015; Scopus Author ID: 56682002300; РИНЦ SPIN-код: 4470-7845. 

6-8 Lev Tolstoy Str., Saint Petersburg 197022



Yu. Е. Balykina
Saint Petersburg State University
Russian Federation

Yulia E. Balykina – PhD (Phys.-Math.), Associate Professor, Chair of Mathematical Modeling of Energy Systems, Faculty of Applied Mathematics – Control Processes;
WoS ResearcherID: K-2125-2013; Scopus Author ID: 56028436400; РИНЦ SPIN-код: 1886-5256.

7-9 Universitetskaya Naberezhnaya, Saint-Petersburg 199034



М. А. Proskurin
Saint Petersburg State University
Russian Federation

Maksim A. Proskurin – Specialist, Chair of Mathematical Modeling of Energy Systems, Faculty of Applied Mathematics – Control Processes
РИНЦ SPIN-код: 7406-2352.

7-9 Universitetskaya Naberezhnaya, Saint-Petersburg 199034



S. А. Mishinova
Pavlov University
Russian Federation

Sofia A. Mishinova – Assistant Professor, Chair Clinical Pharmacology and Evidence-Based Medicine
РИНЦ SPIN-код: 3719-8737.

6-8 Lev Tolstoy Str., Saint Petersburg 197022



References

1. Proximal spinal muscular atrophy 5q. Clinical recommendations. Rubricator of clinical recommendations. Available at: https://cr.minzdrav.gov.ru/recomend/593_2 (in Russ.) (accessed 12.06.2021).

2. Sokolova M.G., Aleksandrov N.Yu., Lobzin S.V. Spinal muscular atrophy in children: etiology, pathogenesis, diagnostics and tretment guidelines. Herald of NorthWestern State Medical University named after I.I. Mechnikov. 2013; 5 (4): 108–13 (in Russ.).

3. Mercuri E., Finkel R.S., Muntoni F., et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord. 2018; 28 (2): 103–15. https://doi.org/10.1016/j.nmd.2017.11.005.

4. Instruction for the medical use of nusinersen in the dosage form of a solution for intrathecal administration. State Register of Medicinal Products. Available at: https://grls.rosminzdrav.ru/Grls_View_v2.aspx?routingGuid=c9b96a83-5cd3-46a9-accd1c5ffb70f785&t (in Russ.) (accessed 12.06.2021).

5. Instruction for the medical use of risdiplam in the dosage form of a powder for the preparation of solution for oral administration. State Register of Medicinal Products. Available at: https://grls.rosminzdrav.ru/Grls_View_v2.aspx?routingGuid=997acb67-4bc6-48f0-b173-886c29314b5a&t (in Russ.) (accessed 12.06.2021).

6. Methodological recommendations for budget impact analysis for the implementation of the program of state free medical care provision to citizens. Мoscow; 2018. Available at: https://rosmedex.ru/wp-content/uploads/2019/06/MR-AVB_novayaredaktsiya_2018-g..pdf (in Russ.) (accessed 12.06.2021).

7. Methodological recommendations for the comparative clinical and economic evaluation of a medicinal product. Мoscow; 2018. Available at: https://rosmedex.ru/wpcontent/uploads/2016/12/MR-KE%60I-23.12.2016.pdf (in Russ.) (дата обращения 12.06.2021).

8. Belousov Yu.B. (Ed.) Evaluation of medical technologies. Мoscow: OKI Publishing; 2013 (in Russ.).

9. State Register of Medicinal Products. Available at: https://grls.rosminzdrav.ru/default.aspx (in Russ.) (accessed 12.06.2021).

10. Decree of the Government of the Russian Federation No. 871 of 28.08.2014 (as amended on 03.12.2020) “On approval of the Rules for the formation of lists of medicines for medical use and the minimum range of medicines necessary for the provision of medical care”. Available at: http://www.consultant.ru/document/cons_doc_LAW_167999/ (in Russ.) (accessed 12.06.2021).

11. Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy (FIREFISH). NCT02913482. Available at: https://clinicaltrials.gov/ct2/show/NCT02913482 (accessed 12.06.2021).

12. Baranello G., Servais L., Masson R., et al. FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA). Eur Respir J. 2020; 56: 1172. https://doi.org/10.1183/13993003.congress-2020.1172.

13. A study to investigatethe safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of risdiplam (RO7034067) in Type 2 and 3 spinal muscular atrophy participants (SUNFISH). NCT02908685. Available at: https://clinicaltrials.gov/ct2/show/NCT02908685 (accessed 12.06.2021).

14. Mercuri E., Barisic N., Boespflug-Tanguy O., et al. SUNFISH Part 2: Efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Available at: https://ir.ptcbio.com/static-files/4425c89f-1648-4346-9769-2a29ad30b179 (accessed 12.06.2021).

15. Finkel R.S., Mercuri E., Darras B.T., et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017; 377 (18): 1723–32. https://doi.org/10.1056/NEJMoa1702752.

16. Mercuri E., Darras B.T., Chiriboga C.A., et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018; 378: 625–35. https://doi.org/10.1056/NEJMoa1710504.

17. State Register of Maximum Selling Prices. Available at: http://grls.rosminzdrav.ru/pricelims.aspx (in Russ.) (accessed 12.06.2021).

18. Ministry of Health of the Russian Federation. Federal Compulsory Health Insurance Fund. Methodological recommendations on ways to pay for medical care at the expense of compulsory health insurance. Available at: http://www.ffoms.gov.ru/upload/iblock/e10/e100830c786400232b841cec2865e44d.pdf (in Russ.) (accessed 12.06.2021).

19. Normative reference information of the Federal Compulsory Health Insurance Fund. Available at: http://nsi.ffoms.ru/#refbookList?refbookList:$active=1&refbookList:$selectedId=566 (in Russ.) (accessed 12.06.2021).

20. Decree of the Government of the Russian Federation No. 1610 of 07.12.2019 “On the Program of state guarantees of free medical care for citizens for 2020 and for the planning period of 2021 and 2022”. Available at: https://base.garant.ru/73187132/ (in Russ.) (accessed 12.06.2021).

21. Unified Information System for Procurement. Available at: https://zakupki.gov.ru/epz/main/public/home.html (in Russ.) (accessed 12.06.2021).

22. 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. Sovremennaya farmakoekonomika i farmakoepidemiologiya / FARMAKOEKONOMIKA. Modern Pharmacoeconomics and Pharmacoepidemiology. 2020; 13 (4): 337–54 (in Russ.). https://doi.org/10.17749/2070-4909/farmakoekonomika.2020.068.

23. Vasilyeva Е.I. Physical development of children: an educational and methodological guide for foreign students. Irkutsk: 2013 (in Russ.).

24. General tariff agreement for payment of medical care provided under the Saint Petersburg territorial program of compulsory health insurance for 2020. Available at: http://www.pol24.ru/doc/gts2020.pdf (in Russ.) (accessed 12.06.2021).


For citation:


Kolbin А.S., Kurylev А.А., Balykina Yu.Е., Proskurin М.А., Mishinova S.А. Health-economic evaluation of risdiplam (Evrysdi®) in patients with spinal muscular atrophy. FARMAKOEKONOMIKA. Modern Pharmacoeconomic and Pharmacoepidemiology. 0;. (In Russ.) https://doi.org/10.17749/2070-4909/farmakoekonomika.2021.101

Views: 203


ISSN 2070-4909 (Print)
ISSN 2070-4933 (Online)