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

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 (C...

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Autores principales: Armeni, Patrizio, Cavazza, Marianna, Xoxi, Entela, Taruscio, Domenica, Kodra, Yllka
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7908548/
https://www.ncbi.nlm.nih.gov/pubmed/33530652
http://dx.doi.org/10.3390/ijerph18031101
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author Armeni, Patrizio
Cavazza, Marianna
Xoxi, Entela
Taruscio, Domenica
Kodra, Yllka
author_facet Armeni, Patrizio
Cavazza, Marianna
Xoxi, Entela
Taruscio, Domenica
Kodra, Yllka
author_sort Armeni, Patrizio
collection PubMed
description 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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spelling pubmed-79085482021-02-27 Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal Armeni, Patrizio Cavazza, Marianna Xoxi, Entela Taruscio, Domenica Kodra, Yllka Int J Environ Res Public Health Commentary 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. MDPI 2021-01-26 2021-02 /pmc/articles/PMC7908548/ /pubmed/33530652 http://dx.doi.org/10.3390/ijerph18031101 Text en © 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Commentary
Armeni, Patrizio
Cavazza, Marianna
Xoxi, Entela
Taruscio, Domenica
Kodra, Yllka
Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal
title Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal
title_full Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal
title_fullStr Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal
title_full_unstemmed Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal
title_short Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal
title_sort reflections on the importance of cost of illness analysis in rare diseases: a proposal
topic Commentary
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7908548/
https://www.ncbi.nlm.nih.gov/pubmed/33530652
http://dx.doi.org/10.3390/ijerph18031101
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