Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy.
cost-effectiveness analysis
economic evaluation
newborn screening
nusinersen
orphan drugs
spinal muscular atrophy
Journal
The Journal of pediatrics
ISSN: 1097-6833
Titre abrégé: J Pediatr
Pays: United States
ID NLM: 0375410
Informations de publication
Date de publication:
12 2020
12 2020
Historique:
received:
08
01
2020
revised:
03
07
2020
accepted:
08
07
2020
pubmed:
14
7
2020
medline:
29
1
2021
entrez:
14
7
2020
Statut:
ppublish
Résumé
To evaluate the cost-effectiveness of nusinersen with and without universal newborn screening for infantile-onset spinal muscular atrophy (SMA). A Markov model using data from clinical trials with US epidemiologic and cost data was developed. The primary interventions studied were nusinersen treatment in a screening setting, nusinersen treatment in a nonscreening setting, and standard care. Analysis was conducted from a societal perspective. Compared with no screening and no treatment, the incremental cost-effectiveness ratio (ICER) for nusinersen with screening was $330 558 per event-free life year (LY) saved, whereas the ICER for nusinersen treatment without screening was $508 481 per event-free LY saved. For nusinersen with screening to be cost-effective at a willingness-to-pay (WTP) threshold of $50 000 per event-free LY saved, the price would need to be $23 361 per dose, less than one-fifth its current price of $125 000. Preliminary data from the NURTURE trial indicated an 85.7% improvement in expected LYs saved compared with our base results. In probabilistic sensitivity analysis, nusinersen and screening was a preferred strategy 93% of the time at a $500 000 WTP threshold. Universal newborn screening for SMA provides improved economic value for payers and patients when nusinersen is available.
Identifiants
pubmed: 32659229
pii: S0022-3476(20)30876-3
doi: 10.1016/j.jpeds.2020.07.033
pmc: PMC7686158
mid: NIHMS1611136
pii:
doi:
Substances chimiques
Oligonucleotides
0
nusinersen
5Z9SP3X666
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
274-280.e2Subventions
Organisme : NHGRI NIH HHS
ID : RM1 HG009037
Pays : United States
Informations de copyright
Copyright © 2020 Elsevier Inc. All rights reserved.
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