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Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe

Background/aim: Ultra-orphan diseases (UODs) have been defined by a prevalence of less than 1 per 50,000 persons. However, little is known about budget impact of ultra-orphan drugs. Methods: For analysis, the budget impact analysis (BIA) had a time horizon of 10 years (2012-2021) and a pan-European...

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Bibliographic Details
Main Authors: Schlander, Michael (Author) , Adarkwah-Yiadom, Charles Christian (Author) , Gandjour, Afschin (Author)
Format: Article (Journal)
Language:English
Published: 2015
In: Expert review of pharmacoeconomics and outcomes research
Year: 2015, Volume: 15, Issue: 1, Pages: 171-179
ISSN:1744-8379
DOI:10.1586/14737167.2015.965156
Online Access:Verlag, Volltext: http://dx.doi.org/10.1586/14737167.2015.965156
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Author Notes:Michael Schlander, Charles Christian Adarkwah and Afschin Gandjour
Description
Summary:Background/aim: Ultra-orphan diseases (UODs) have been defined by a prevalence of less than 1 per 50,000 persons. However, little is known about budget impact of ultra-orphan drugs. Methods: For analysis, the budget impact analysis (BIA) had a time horizon of 10 years (2012-2021) and a pan-European payer’s perspective, based on prevalence data for UODs for which patented drugs are available and/or for which drugs are in clinical development. Results: A total of 18 drugs under patent protection or orphan drug designation for non-oncological UODs were identified. Furthermore, 29 ultra-orphan drugs for non-oncological diseases under development that have the potential of reaching the market by 2021 were found. Total budget impact over 10 years was estimated to be €15,660 and €4965 million for approved and pipeline ultra-orphan drugs, respectively (total: €20,625 million). Conclusion: The analysis does not support concerns regarding an uncontrolled growth in expenditures for drugs for UODs.
Item Description:Online veröffentlicht: 14. Oktober 2014
Gesehen am 28.08.2017
Physical Description:Online Resource
ISSN:1744-8379
DOI:10.1586/14737167.2015.965156

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