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Commentary

Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal

1
Cergas (Centre for Research on Health and Social Care Management), SDA Bocconi School of Management, 20136 Milan, Italy
2
Independent Pharmacologist Scientific Advisor in Rare Disease Pharmaceuticals and Registries, 00184 Rome, Italy
3
National Centre for Rare Diseases, Istituto Superiore di Sanità, 00162 Rome, Italy
*
Author to whom correspondence should be addressed.
Int. J. Environ. Res. Public Health 2021, 18(3), 1101; https://doi.org/10.3390/ijerph18031101
Received: 7 December 2020 / Revised: 9 January 2021 / Accepted: 20 January 2021 / Published: 26 January 2021
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. View Full-Text
Keywords: rare diseases; social economic burden; cost of illness; health technology assessment rare diseases; social economic burden; cost of illness; health technology assessment
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MDPI and ACS Style

Armeni, P.; Cavazza, M.; Xoxi, E.; Taruscio, D.; Kodra, Y. Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal. Int. J. Environ. Res. Public Health 2021, 18, 1101. https://doi.org/10.3390/ijerph18031101

AMA Style

Armeni P, Cavazza M, Xoxi E, Taruscio D, Kodra Y. Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal. International Journal of Environmental Research and Public Health. 2021; 18(3):1101. https://doi.org/10.3390/ijerph18031101

Chicago/Turabian Style

Armeni, Patrizio; Cavazza, Marianna; Xoxi, Entela; Taruscio, Domenica; Kodra, Yllka. 2021. "Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal" Int. J. Environ. Res. Public Health 18, no. 3: 1101. https://doi.org/10.3390/ijerph18031101

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