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

M. Schlander*, C.C. Adarkwah, A. Gandjour

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


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 (sic)15,660 and (sic)4965 million for approved and pipeline ultra-orphan drugs, respectively (total: (sic)20,625 million). Conclusion: The analysis does not support concerns regarding an uncontrolled growth in expenditures for drugs for UODs.

Original languageEnglish
Pages (from-to)171-179
Number of pages9
JournalExpert Review of Pharmacoeconomics & Outcomes Research
Issue number1
Publication statusPublished - Feb 2015


  • budget impact analysis
  • drugs
  • Europe
  • non-oncological diseases
  • ultra-orphan
  • RARE

Cite this