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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 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.

Authors:   Michael Schlander; Charles Christian Adarkwah; Afschin Gandjour
Journal:   Expert Review of Pharmacoeconomics & Outcomes Research
Year:   2014
DOI:   10.1586/14737167.2015.965156
Publication date:   14-Oct-2014
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