2024-03-29T04:36:11Zhttp://repisalud.isciii.es/oai/requestoai:repisalud.isciii.es:20.500.12105/126832022-11-22T13:47:06Zcom_20.500.12105_2102com_20.500.12105_2052com_20.500.12105_2051col_20.500.12105_2103
Repisalud
author
García-Pérez, Lidia
author
Linertová, Renata
author
Valcárcel-Nazco, Cristina
author
Posada De la Paz, Manuel
author
Gorostiza, Inigo
author
Serrano-Aguilar, Pedro
2021-04-19T15:20:44Z
2021-04-19T15:20:44Z
2021
Orphanet J Rare Dis . 2021 Apr 13;16(1):178.
http://hdl.handle.net/20.500.12105/12683
33849613
10.1186/s13023-021-01815-3
1750-1172
Orphanet Journal of Rare Diseases
Objective: The aim of this scoping review was to overview the cost-of-illness studies conducted in rare diseases.
Methods: We searched papers published in English in PubMed from January 2007 to December 2018. We selected cost-of-illness studies on rare diseases defined as those with prevalence lower than 5 per 10,000 cases. Studies were selected by one researcher and verified by a second researcher. Methodological characteristics were extracted to develop a narrative synthesis.
Results: We included 63 cost-of-illness studies on 42 rare diseases conducted in 25 countries, and 9 systematic reviews. Most studies (94%) adopted a prevalence-based estimation, where the predominant design was cross-sectional with a bottom-up approach. Only four studies adopted an incidence-based estimation. Most studies used questionnaires to patients or caregivers to collect resource utilisation data (67%) although an important number of studies used databases or registries as a source of data (48%). Costs of lost productivity, non-medical costs and informal care costs were included in 68%, 60% and 43% of studies, respectively.
Conclusion: This review found a paucity of cost-of-illness studies in rare diseases. However, the analysis shows that the cost-of-illness studies of rare diseases are feasible, although the main issue is the lack of primary and/or aggregated data that often prevents a reliable estimation of the economic burden.
eng
Cost-of-illness
Economic burden
Rare diseases
Scoping review.
Cost-of-illness studies in rare diseases: a scoping review
journal article
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URL
https://repisalud.isciii.es/bitstream/20.500.12105/12683/1/CostOfIllnessStudies_2021.pdf
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CostOfIllnessStudies_2021.pdf
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https://repisalud.isciii.es/bitstream/20.500.12105/12683/4/CostOfIllnessStudies_2021.pdf.txt
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CostOfIllnessStudies_2021.pdf.txt