Avtor/Urednik		Detiček, Andreja; Locatelli, Igor; Kos, Mitja
Naslov		Patient access to medicines for rare diseases in European countries
Tip		članek
Vol. in št.		Letnik 21, št. 5
Leto izdaje		2018
Obseg		str. 553-560
ISSN		1098-3015 - Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
Jezik		eng
Abstrakt		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.
Deskriptorji		Bolezni Pacienti
Proste vsebinske oznake		zdravstvo redke bolezni Evropa dostop do zdravil health services elderly people Europe analysis