Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis
| Autor: | Veronica Wiley, Georgina M. Chambers, Sophy T. F. Shih, Michelle A. Farrar |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
Male
Pediatrics medicine.medical_specialty Population level Cost-Benefit Analysis Recombinant Fusion Proteins Oligonucleotides Context (language use) Disease Muscular Atrophy Spinal 03 medical and health sciences Neonatal Screening 0302 clinical medicine medicine Humans Biological Products 0303 health sciences Newborn screening business.industry 030305 genetics & heredity Infant Newborn Cost-effectiveness analysis Spinal muscular atrophy medicine.disease SMA Psychiatry and Mental health Female Surgery Nusinersen Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | Journal of Neurology, Neurosurgery & Psychiatry. 92:1296-1304 |
| ISSN: | 1468-330X 0022-3050 |
| DOI: | 10.1136/jnnp-2021-326344 |
| Popis: | ObjectiveTo assess cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) and early treatment with nusinersen or onasemnogene abeparvovec (gene therapy), compared with nusinersen without SMA screening.MethodsInformed by an Australian state-wide SMA NBS programme, a decision analytical model nested with Markov models was constructed to evaluate costs and quality-adjusted life-years (QALYs) from a societal perspective with sensitivity analyses.ResultsBy treating one presymptomatic SMA infant with nusinersen or gene therapy, an additional 9.93 QALYs were gained over 60 years compared with late treatment in clinically diagnosed SMA. The societal cost was $9.8 million for early nusinersen treatment, $4.4 million for early gene therapy and $4.8 million for late nusinersen treatment. Compared with late nusinersen treatment, early gene therapy would be dominant, gaining 9.93 QALYs while saving $360 000; whereas early nusinersen treatment would result in a discounted incremental cost-effectiveness ratio (ICER) of $507 000/QALY.At a population level, compared with no screening and late treatment with nusinersen, NBS and early gene therapy resulted in 0.00085 QALY gained over 60 years and saving $24 per infant screened (85 QALYs gained and $2.4 million saving per 100 000 infants screened). More than three quarters of simulated ICERs by probability sensitivity analyses showed NBS and gene therapy would be dominant or less than $50 000/QALY, compared with no screening and late nusinersen treatment.ConclusionNBS coupled with gene therapy improves the quality and length of life for infants with SMA and would be considered value-for-money from an Australian clinical and policy context. |
| Databáze: | OpenAIRE |
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