Patient Access to Medicines for Rare Diseases in European Countries

Value Health. 2018 May;21(5):553-560. doi: 10.1016/j.jval.2018.01.007. Epub 2018 Mar 16.


Background: The number of authorized orphan and non-orphan medicines for rare diseases has increased in Europe. Patient access to these medicines is affected by high costs, weak efficacy/safety evidence, and societal value. European health care systems must determine whether paying for expensive treatments for only a few patients is sustainable.

Objectives: This study aimed to evaluate patient access to orphan and non-orphan medicines for rare diseases in 22 European countries during 2005 to 2014.

Methods: Medicines for rare diseases from the Orphanet list, authorized during 2005 to 2014, were searched for in the IMS MIDAS Quarterly Sales Data, January 2005 - December 2014 (IQVIA, Danbury, CT). The following three measures were determined for each country: number of available medicines, median time to continuous use, and medicine expenditure. A medicine was considered available if uninterrupted sales within a 1-year period were detected.

Results: From 2005 to 2014, 125 medicines were authorized and 112 were found in the search. Of those, between 70 (63%) and 102 (91%) were available in Germany, the United Kingdom, Italy, France, and the Scandinavian countries. These countries were also the fastest to enable continuous use (3-9 mo). Only 27% to 38% of authorized medicines were available in Greece, Ireland, Bulgaria, Romania, and Croatia, which took 1 to 2.6 years to begin continuous use. A country's expenditure on medicines for rare diseases in 2014 ranged between €0.2 and €31.9/inhabitant.

Conclusions: Patient access to medicines for rare diseases varies largely across Europe. Patients in Germany, Scandinavian countries, Switzerland, France, and the United Kingdom can access larger numbers of medicines in shorter time.

Keywords: availability; medicine expenditure; orphan medicines; patient access; rare disease.

MeSH terms

  • Drug Costs*
  • Europe
  • Health Services Accessibility / economics*
  • Health Services Accessibility / standards
  • Humans
  • Orphan Drug Production / economics*
  • Pharmaceutical Preparations / economics
  • Pharmaceutical Preparations / supply & distribution*
  • Rare Diseases / drug therapy
  • Rare Diseases / economics*


  • Pharmaceutical Preparations