Patient Access to Medicines for Rare Diseases in European Countries

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Andreja DetičekMitja Kos

Abstract

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

Citations

Mar 19, 2019·American Journal of Medical Genetics. Part a·Carlos R Ferreira
May 6, 2019·Orphanet Journal of Rare Diseases·Bernarda ZamoraMartina Garau
Feb 20, 2019·Orphanet Journal of Rare Diseases·Marion BourdoncleFlorence Taboulet
Jan 7, 2020·PharmacoEconomics·Franziska Worm, Charalabos-Markos Dintsios
Feb 18, 2020·Current Pharmaceutical Biotechnology·Mohammed H AldosariHuub Schellekens
Jan 31, 2019·Expert Review of Pharmacoeconomics & Outcomes Research·Patricia Vella BonannoBrian Godman
Mar 3, 2020·Frontiers in Public Health·Marcin CzechJolanta Sykut-Cegielska
May 19, 2019·Journal for Immunotherapy of Cancer·Howard L KaufmanWendy K D Selig
Dec 22, 2021·Expert Review of Pharmacoeconomics & Outcomes Research·Giovanni TafuriJens Grueger

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