SynthesisEuropean journal of pediatrics2025
Economic evidence on the cost-effectiveness of newborn screening for spinal muscular atrophy: a systematic review.
Synthesis in European journal of pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
3 citing papers in PubMed.
- Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy.Muscle & nerve · 2026Review
- Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions.Nature reviews. Neurology · 2026Review
- Modeling the Budget Impact of Disease-Modifying Therapies in Infantile-Onset Spinal Muscular Atrophy.Clinical drug investigation · 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
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy (SMA) is a severe neurodegenerative disorder associated with the risk of early infantile death. Newborn screening (NBS) is a vital public health strategy that enables early diagnosis and intervention. Economic evaluations have become essential to support the adoption of NBS programs for SMA. This systematic review aimed to evaluate the cost-effectiveness of NBS across different countries and healthcare settings for SMA. The PubMed, ScienceDirect, and Scopus databases were searched for cost-effectiveness studies of the NBS for SMA from January 2000 to June 2025. A standardized data extraction form was used to collect relevant data, including characteristics, perspectives, economic evaluations, and outcomes. A total of six studies met the predefined inclusion criteria, and all of them reported cost-utility analysis. All studies were economic model-based evaluations. These studies indicated that NBS for SMA is highly likely to demonstrate healthcare and societal cost-effectiveness. All six studies reported favorable economic outcomes, presenting either incremental cost-effectiveness ratios (ICERs) per quality-adjusted life year (QALY) or life-year gained, cost per QALY, or total cost savings. Collectively, these findings demonstrated that NBS for SMA is consistently cost-effective across diverse settings. The systematic review supports the inclusion of this approach in national screening programs as a cost-effective and life-saving public health strategy for SMA.
conclusionThese studies indicated that NBS for SMA is highly likely to demonstrate healthcare and societal cost-effectiveness. All six studies reported favorable economic outcomes, presenting either incremental cost-effectiveness ratios (ICERs) per quality-adjusted life year (QALY) or life-year gained, cost per QALY, or total cost savings. Collectively, these findings demonstrated that NBS for SMA is consistently cost-effective across diverse settings. The systematic review supports the inclusion of this approach in national screening programs as a cost-effective and life-saving public health strategy for SMA.
trial registrationPROSPERO (CRD42024608296). WHAT IS KNOWN: •SMA is a genetic neurodegenerative disorder causing progressive muscle weakness and early infant death if untreated. •Early detection through NBS enables timely intervention, significantly improving survival and quality of life. WHAT IS NEW: •The reviewed literature consistently demonstrates favorable economic outcomes, with most studies reporting either a cost-saving effect or acceptable ICERs per QALY gained. •These findings support the inclusion of SMA in national NBS programs, as early detection enables timely treatment, resulting in improved health outcomes and reduced long-term healthcare costs.
Indexed as
Identifiers
41258205What OpenQuestion holds
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.