title: Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe.
abstract: OBJECTIVE
  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).
  
  
  CONCLUSIONS
  The analysis does not support concerns regarding an uncontrolled growth in expenditures for drugs for UODs.
authors.affiliation: Institute for Innovation and Valuation in Health Care (InnoValHC), An der Ringkirche 4, D-65197 Wiesbaden, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Schlander
authors.forename: Charles Christian
authors.initials: CC
authors.lastname: Adarkwah
authors.forename: Afschin
authors.initials: A
authors.lastname: Gandjour
mesh_terms: D002017:Budgets; D005060:Europe / Q000453:epidemiology; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics*; D015995:Prevalence; D035583:Rare Diseases / Q000188:drug therapy* / Q000453:epidemiology
keywords: Europe; budget impact analysis; drugs; non-oncological diseases; ultra-orphan