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

Patrizio Armeni, Marianna Cavazza, Entela Xoxi, Domenica Taruscio and Yllka Kodra
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Patrizio Armeni: Cergas (Centre for Research on Health and Social Care Management), SDA Bocconi School of Management, 20136 Milan, Italy
Marianna Cavazza: Cergas (Centre for Research on Health and Social Care Management), SDA Bocconi School of Management, 20136 Milan, Italy
Entela Xoxi: Independent Pharmacologist Scientific Advisor in Rare Disease Pharmaceuticals and Registries, 00184 Rome, Italy
Domenica Taruscio: National Centre for Rare Diseases, Istituto Superiore di Sanità, 00162 Rome, Italy
Yllka Kodra: National Centre for Rare Diseases, Istituto Superiore di Sanità, 00162 Rome, Italy

IJERPH, 2021, vol. 18, issue 3, 1-18

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.

Keywords: rare diseases; social economic burden; cost of illness; health technology assessment (search for similar items in EconPapers)
JEL-codes: I I1 I3 Q Q5 (search for similar items in EconPapers)
Date: 2021
References: View references in EconPapers View complete reference list from CitEc
Citations: View citations in EconPapers (1)

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