ArticleThe European journal of health economics : HEPAC : health economics in prevention and care2016
Social/economic costs and health-related quality of life in patients with Prader-Willi syndrome in Europe.
Article in The European journal of health economics : HEPAC : health economics in prevention and care, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 5 of them syntheses that pooled it.
What it found
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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.
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Who cites it
27 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- The burden of illness in Prader-Willi syndrome: a systematic literature review.Orphanet journal of rare diseases · 2025Pooled it
- Impact of Choice of Tariff When Calculating Clinically Meaningful EQ-5D Scores in Metabolic Dysfunction-Associated Steatohepatitis.Liver international : official journal of the International Association for the Study of the Liver · 2025Pooled it
- Pooled it
- Health-Related Quality of Life in Patients With Different Diseases Measured With the EQ-5D-5L: A Systematic Review.Frontiers in public health · 2021Pooled it
- Cost-of-illness studies in nine Central and Eastern European countries.The European journal of health economics : HEPAC : health economics in prevention and care · 2019Pooled it
- Health-related quality of life and medication use among individuals with Angelman syndrome.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2023Trial
- Trial
- Patient advocacy group perspectives on treatment priorities and clinical trials for the rare neurodevelopmental condition, Prader-Willi syndrome.Orphanet journal of rare diseases · 2026Article
- Assessment of Quality of Life and Psychological Well-Being in Italian Adult Subjects with Prader-Willi Syndrome Using the Health Survey Short Form and the Psychological General Well-Being Index Questionnaires.Healthcare (Basel, Switzerland) · 2025Article
- EndoCompass Project: Research Roadmap for Growth Disorders.Hormone research in paediatrics · 2025Review
- Psychological conditions of caregivers of adult subjects with Prader-Willi syndrome.Orphanet journal of rare diseases · 2024Article
- Health-related quality of life in patients with diverse rare diseases: An online survey.Genetics in medicine open · 2024Article
- Methods used to account for caregivers' sex and gender within studies examining the financial burden of caregivers of children and adolescents : Results from a scoping review.ClinicoEconomics and outcomes research : CEOR · 2024Article
- Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies.PharmacoEconomics · 2023Article
- Caregiver-based perception of disease burden in Schaaf-Yang syndrome.Molecular genetics & genomic medicine · 2023Article
- Progressive resistance training in young people with Prader-Willi syndrome: protocol for a randomised trial (PRESTO).BMJ open · 2022Article
- Valuing the "Burden" and Impact of Rare Diseases: A Scoping Review.Frontiers in pharmacology · 2022Article
- Rare disease emerging as a global public health priority.Frontiers in public health · 2022Review
- What Every Internist-Endocrinologist Should Know about Rare Genetic Syndromes in Order to Prevent Needless Diagnostics, Missed Diagnoses and Medical Complications: Five Years of 'Internal Medicine for Rare Genetic Syndromes'.Journal of clinical medicine · 2021Article
- Cost-of-illness studies in rare diseases: a scoping review.Orphanet journal of rare diseases · 2021Article
Corrections and comments
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Authors and funding
16 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveThe aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe.
methodsWe conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.
resultsA total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00.
conclusionThe main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL.
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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.