TY - JOUR

T1 - Фармакоэкономическая оценка лекарственного препарата рисдиплам у пациентов со спинальной мышечной атрофией

AU - Kolbin, A. S.

AU - Kurylev, A. A.

AU - Balykina, Yu

AU - Proskurin, M. A.

AU - Mishinova, S.

N1 - Publisher Copyright: © 2021 Belarusian National Technical University. All rights reserved.

PY - 2021/1/22

Y1 - 2021/1/22

N2 - 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.

AB - 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.

KW - Health economic evaluation

KW - Medical costs

KW - Risdiplam

KW - SMA

KW - Spinal muscular atrophy

UR - http://www.scopus.com/inward/record.url?scp=85118117589&partnerID=8YFLogxK

UR - https://www.mendeley.com/catalogue/ba5093e9-0f25-3616-b395-e2415f715392/

U2 - 10.17749/2070-4909/farmakoekonomika.2021.101

DO - 10.17749/2070-4909/farmakoekonomika.2021.101

M3 - статья

AN - SCOPUS:85118117589

VL - 14

SP - 299

EP - 310

JO - Farmakoekonomika

JF - Farmakoekonomika

SN - 2070-4909

IS - 3

ER -