1. Reflections on the importance of cost of illness analysis in rare diseases: a proposal
- Author
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Yllka Kodra, Marianna Cavazza, Domenica Taruscio, Patrizio Armeni, and Entela Xoxi
- Subjects
medicine.medical_specialty ,Technology Assessment, Biomedical ,Cystic Fibrosis ,Health, Toxicology and Mutagenesis ,Cost-Benefit Analysis ,lcsh:Medicine ,03 medical and health sciences ,Indirect costs ,0302 clinical medicine ,Cost of illness ,Medicine ,Humans ,RARE DISEASES, SOCIAL ECONOMIC BURDEN, COST OF ILLNESS, HEALTH TECHNOLOGY ASSESSMENT ,030212 general & internal medicine ,Intensive care medicine ,Disease burden ,HEALTH TECHNOLOGY ASSESSMENT ,business.industry ,030503 health policy & services ,lcsh:R ,Public Health, Environmental and Occupational Health ,Health technology ,SOCIAL ECONOMIC BURDEN ,Robust design ,Cost driver ,Economic evaluation ,Commentary ,COST OF ILLNESS ,0305 other medical science ,business ,RARE DISEASES ,Rare disease - 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.
- Published
- 2021