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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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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. |
format | Online Article Text |
id | pubmed-7908548 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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