ArticleInternational journal of environmental research and public health2021
Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal.
Article in International journal of environmental research and public health, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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Who cites it
8 citing papers in PubMed.
- Model-Based Economic Analyses of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2026Review
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study.Skin health and disease · 2024Article
- Economic Burden of Idiopathic Pulmonary Fibrosis in Spain: A Prospective Real-World Data Study (OASIS Study).PharmacoEconomics · 2023Observational
- Socio-economic costs of rare diseases and the risk of financial hardship: a cross-sectional study.The Lancet regional health. Western Pacific · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Valuing the "Burden" and Impact of Rare Diseases: A Scoping Review.Frontiers in pharmacology · 2022Article
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In the field of rare diseases (RDs), the evidence standard is often lower than that required by health technology assessment (HTA) and payer authorities. In this commentary, we propose that appropriate economic evaluation for rare disease treatments should be initially informed by cost-of-illness (COI) studies conducted using a societal perspective. Such an approach contributes to improving countries' understanding of RDs in their entirety as societal and not merely clinical, or product-specific issues. In order to exemplify how the disease burden's distribution has changed over the last fifteen years, key COI studies for Hemophilia, Fragile X Syndrome, Cystic Fibrosis, and Juvenile Idiopathic Arthritis are examined. Evidence shows that, besides methodological variability and cross-country differences, the disease burden's share represented by direct costs generally grows over time as novel treatments become available. Hence, to support effective decision-making processes, it seems necessary to assess the re-allocation of the burden produced by new medicinal products, and this approach requires identifying cost drivers through COI studies with robust design and standardized methodology.
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