ArticleHealthcare (Basel, Switzerland)2015
Showing Value in Newborn Screening: Challenges in Quantifying the Effectiveness and Cost-Effectiveness of Early Detection of Phenylketonuria and Cystic Fibrosis.
Article in Healthcare (Basel, Switzerland), 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 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
23 citing papers in PubMed.
- Cystic Fibrosis Newborn Screening: Cost Trajectory as a Marker to Improve Health Outcomes.Pediatric pulmonology · 2026Article
- Data-Related and Methodological Challenges in Assessing the Cost-Effectiveness of Traditional and Genomic Newborn Screening Programs.Applied health economics and health policy · 2026Review
- Methodological and Procedural Considerations for Developing Decision Analytic Models to Assess the Health Economic Impacts of Newborn Bloodspot Screening: A Systematic Methodological Review.International journal of neonatal screening · 2025Review
- Results of the Hungarian Newborn Screening Pilot Program for Spinal Muscular Atrophy.International journal of neonatal screening · 2025Article
- Rare Disease, Advocacy and Justice: Intersecting Disparities in Research and Clinical Care.The American journal of bioethics : AJOB · 2023Article
- Newborn screening for congenital hypothyroidism and phenylketonuria-Beyond cost savings.The Journal of pediatrics · 2023Article
- Lifetime Cost Effectiveness of Phenylketonuria Screening National Program in Iran.Iranian journal of public health · 2021Article
- Of Screening, Stratification, and Scores.Journal of personalized medicine · 2021Review
- Data Needs for Economic Evaluations of Screening in Pediatric Primary Care: A Research Framework.Pediatrics · 2021Review
- Health economic burden of patients with phenylketonuria (PKU) - A retrospective study of German health insurance claims data.Molecular genetics and metabolism reports · 2021Article
- Newborn Screening and Treatment of Phenylketonuria: Projected Health Outcomes and Cost-Effectiveness.Children (Basel, Switzerland) · 2021Article
- Monetary Valuation of Children's Cognitive Outcomes in Economic Evaluations from a Societal Perspective: A Review.Children (Basel, Switzerland) · 2021Review
- The Methodological Quality and Challenges in Conducting Economic Evaluations of Newborn Screening: A Scoping Review.International journal of neonatal screening · 2020Article
- Challenges in Assessing the Cost-Effectiveness of Newborn Screening: The Example of Congenital Adrenal Hyperplasia.International journal of neonatal screening · 2020Review
- Adverse Outcomes and Economic Burden of Congenital Adrenal Hyperplasia Late Diagnosis in the Newborn Screening Absence.Journal of the Endocrine Society · 2020Article
- Health utilities and parental quality of life effects for three rare conditions tested in newborns.Journal of patient-reported outcomes · 2019Article
- Observational
- What Contribution Did Economic Evidence Make to the Adoption of Universal Newborn Hearing Screening Policies in the United States?International journal of neonatal screening · 2018Article
- The Sweat Metabolome of Screen-Positive Cystic Fibrosis Infants: Revealing Mechanisms beyond Impaired Chloride Transport.ACS central science · 2017Article
- Cost and Cost-Effectiveness Assessments of Newborn Screening for Critical Congenital Heart Disease Using Pulse Oximetry: A Review.International journal of neonatal screening · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
Abstract
Decision makers sometimes request information on the cost savings, cost-effectiveness, or cost-benefit of public health programs. In practice, quantifying the health and economic benefits of population-level screening programs such as newborn screening (NBS) is challenging. It requires that one specify the frequencies of health outcomes and events, such as hospitalizations, for a cohort of children with a given condition under two different scenarios-with or without NBS. Such analyses also assume that everything else, including treatments, is the same between groups. Lack of comparable data for representative screened and unscreened cohorts that are exposed to the same treatments following diagnosis can result in either under- or over-statement of differences. Accordingly, the benefits of early detection may be understated or overstated. This paper illustrates these common problems through a review of past economic evaluations of screening for two historically significant conditions, phenylketonuria and cystic fibrosis. In both examples qualitative judgments about the value of prompt identification and early treatment to an affected child were more influential than specific numerical estimates of lives or costs saved.
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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.