ArticleNeurology and therapy2025
Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Australian Hospitals.
Article in Neurology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
5 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Economic evidence on the cost-effectiveness of newborn screening for spinal muscular atrophy: a systematic review.European journal of pediatrics · 2025Pooled it
- Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Spain, Greece, and Portugal.Neurology and therapy · 2026Article
- Expanding 5q-SMA Newborn Screening in Latin America: A Brazilian Model for National and Regional Implementation.International journal of neonatal screening · 2026Article
- Article
- Neonatal screening for spinal muscular atrophy: Report of a multicenter study in Brazil.Genetics and molecular biology · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionThis analysis evaluated the cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) from the perspective of Australian state hospital payers.
methodsA cost-utility analysis consisting of a decision tree and Markov cohort designed to calculate the difference in costs and health outcomes between two scenarios: (1) disease-modifying treatment (DMT) for SMA after diagnosis through NBS, and (2) DMT for SMA after diagnosis as symptoms appear. A population of 295,906 newborns was modeled, based on the total number of live births in Australia in 2023. Inputs included screening parameters, epidemiology inputs, SMA natural history data and DMT parameters (nusinersen and onasemnogene abeparvovec), costs, and health-related quality of life parameters. Assumed participation in NBS was 100%. A one-way sensitivity analysis and probabilistic sensitivity analysis were conducted to examine the impact of parameter uncertainty.
resultsThere were 30 patients identified with SMA, of whom 25 patients would be eligible for presymptomatic treatment. NBS for SMA was dominant compared with no NBS for SMA. On a population level, NBS demonstrated a lifetime gain of 267 quality-adjusted life years (QALY) and incremental costs of -AUD$3,983,263 (i.e., cost savings). Every dollar invested in NBS would save hospitals $3.69. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results.
conclusionNBS for SMA was dominant compared with no NBS for SMA in Australia from a state and territory payer perspective. Universal implementation of NBS for SMA would support access equity, as well as early diagnosis and treatment in infants with SMA, potentially leading to improved outcomes.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.