Andreja Detiček1, Igor Locatelli1, Mitja Kos2. 1. Faculty of Pharmacy, University of Ljubljana, Slovenia. 2. Faculty of Pharmacy, University of Ljubljana, Slovenia. Electronic address: mitja.kos@ffa.uni-lj.si.
Abstract
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.
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.
Authors: Howard L Kaufman; Michael B Atkins; Prasun Subedi; James Wu; James Chambers; T Joseph Mattingly; Jonathan D Campbell; Jeff Allen; Andrea E Ferris; Richard L Schilsky; Daniel Danielson; J Leonard Lichtenfeld; Linda House; Wendy K D Selig Journal: J Immunother Cancer Date: 2019-05-17 Impact factor: 13.751
Authors: Jurgen Sota; Antonio Vitale; Ewa Więsik-Szewczyk; Micol Frassi; Giuseppe Lopalco; Giacomo Emmi; Marcello Govoni; Amato de Paulis; Achille Marino; Antonio Gidaro; Sara Monti; Daniela Opris-Belinski; Rosa Maria R Pereira; Karina Jahnz-Rózyk; Carla Gaggiano; Francesca Crisafulli; Florenzo Iannone; Irene Mattioli; Francesca Ruffilli; Ilaria Mormile; Katarzyna Rybak; Valeria Caggiano; Paolo Airò; Abdurrahman Tufan; Stefano Gentileschi; Gaafar Ragab; Ibrahim A Almaghlouth; Adham Aboul-Fotouh Khalil; Marco Cattalini; Francesco La Torre; Maria Tarsia; Henrique A Mayrink Giardini; Moustafa Ali Saad; Monica Bocchia; Federico Caroni; Teresa Giani; Elisa Cinotti; Piero Ruscitti; Pietro Rubegni; Marília A Dagostin; Bruno Frediani; Aslihan Avanoglu Guler; Francesca Della Casa; Maria Cristina Maggio; Andreas Recke; Dagmar von Bubnoff; Karoline Krause; Alberto Balistreri; Claudia Fabiani; Donato Rigante; Luca Cantarini Journal: Front Med (Lausanne) Date: 2022-07-18