Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy.
J Pediatr
; 227: 274-280.e2, 2020 12.
Article
em En
| MEDLINE
| ID: mdl-32659229
ABSTRACT
OBJECTIVE:
To evaluate the cost-effectiveness of nusinersen with and without universal newborn screening for infantile-onset spinal muscular atrophy (SMA). STUDYDESIGN:
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.RESULTS:
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.CONCLUSION:
Universal newborn screening for SMA provides improved economic value for payers and patients when nusinersen is available.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos
/
Atrofia Muscular Espinal
/
Triagem Neonatal
/
Análise Custo-Benefício
Tipo de estudo:
Diagnostic_studies
/
Health_economic_evaluation
/
Prognostic_studies
/
Screening_studies
Limite:
Humans
/
Newborn
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article