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Health economic evaluation of risdiplam in patients with spinal muscular atrophy

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

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 spinal muscular atrophy.

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 of Clinical Pharmacology and Evidence-Based Medicine; Professor, Chair of Pharmacology, Faculty of Medicine

Scopus Author ID: 19836020100; WoS ResearcherID: G-5537-2015; RSCI SPIN-code: 7966-0845 

6-8 Lev Tolstoy Str., Saint Petersburg 197022, Russia

7-9 Universitetskaya Qy, Saint-Petersburg 199034, Russia



А. А. Kurylev
Pavlov University
Russian Federation

Aleksey A. Kurylev – MD, PhD, Assistant Professor, Chair of Clinical Pharmacology and Evidence-Based Medicine

WoS ResearcherID: O-2630-2015; Scopus Author ID: 56682002300; RSCI SPIN-code: 4470-7845

6-8 Lev Tolstoy Str., Saint Petersburg 197022, Russia



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; RSCI SPIN-code: 1886-5256

7-9 Universitetskaya Qy, Saint-Petersburg 199034, Russia



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

RSCI SPIN-code: 7406-2352

7-9 Universitetskaya Qy, Saint-Petersburg 199034, Russia



S. А. Mishinova
Pavlov University
Russian Federation

Sofia A. Mishinova – Assistant Professor, Chair of Clinical Pharmacology and Evidence-Based Medicine

6-8 Lev Tolstoy Str., Saint Petersburg 197022, Russia



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Review

For citations:


Kolbin А.S., Kurylev А.А., Balykina Yu.Е., Proskurin М.А., Mishinova S.А. Health economic evaluation of risdiplam in patients with spinal muscular atrophy. FARMAKOEKONOMIKA. Modern Pharmacoeconomics and Pharmacoepidemiology. 2021;14(3):299-310. (In Russ.) https://doi.org/10.17749/2070-4909/farmakoekonomika.2021.101

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