title: A comparative study of European rare disease and orphan drug markets.
abstract: OBJECTIVE
  This article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom.
  
  
  METHODS
  Information was derived from the international literature, analysis of legal texts, and a survey completed by national experts.
  
  
  RESULTS
  These countries adopted varying approaches towards regulating rare disease and orphan drug markets and, hence, the availability, pricing and reimbursement of orphan drugs vary between countries. Strategies to keep down prices include public procurement in Sweden, profit controls in the United Kingdom, and price comparisons with other countries. To gain reimbursement, the cost-effectiveness and/or budget impact of orphan drugs is considered in some countries. Other societal considerations, such as whether the drug treats a life-threatening disease, are sometimes taken into account.
  
  
  CONCLUSIONS
  Extensive government intervention exists in rare disease and orphan drug markets in the countries studied. Our recommendations are to define priorities for research on rare diseases and orphan drugs at the European level, to set up disease and patient registries with a view to investigating the long-term effectiveness and cost-effectiveness of orphan drugs, to assess the profitability of orphan drugs, and to take into account societal considerations when evaluating orphan drugs.
authors.affiliation: Yellow Window Management Consultants, Antwerp, Belgium. Alain.Denis@yellowwindow.com
authors.forename: Alain
authors.initials: A
authors.lastname: Denis
authors.forename: Lut
authors.initials: L
authors.lastname: Mergaert
authors.forename: Christel
authors.initials: C
authors.lastname: Fostier
authors.forename: Irina
authors.initials: I
authors.lastname: Cleemput
authors.forename: Steven
authors.initials: S
authors.lastname: Simoens
mesh_terms: D003363:Cost Control; D016527:Drug Costs / Q000331:legislation & jurisprudence*; D004335:Drug and Narcotic Control / Q000191:economics*; D005060:Europe; D005252:Fees, Pharmaceutical / Q000331:legislation & jurisprudence*; D006291:Health Policy; D006801:Humans; D007349:Insurance, Health, Reimbursement / Q000331:legislation & jurisprudence*; D040541:Marketing / Q000331:legislation & jurisprudence*; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*