dc.contributor.author	Stawowczyk, Ewa
dc.contributor.author	Malinowski, Krzysztof Piotr
dc.contributor.author	Kawalec, Paweł
dc.contributor.author	Bobiński, Rafał
dc.contributor.author	Siwiec, Jacek
dc.contributor.author	Panteli, Dimitra
dc.contributor.author	Eckhardt, Helene
dc.contributor.author	Simoens, Steven
dc.contributor.author	Agusti, Antònia
dc.contributor.author	Dooms, Marc
dc.contributor.author	Pilc, Andrzej
dc.date.accessioned	2019-11-28T13:31:36Z
dc.date.available	2019-11-28T13:31:36Z
dc.date.issued	2019-11-27
dc.date.updated	2019-11-27T05:39:39Z
dc.description.abstract	Objective: To review the reimbursement recommendations issued by selected European health technology assessment agencies for orphan drugs and the reimbursement status of these drugs; to assess the relationship between the type of recommendation and reimbursement status. Methods: The list of orphan drugs to be included in the analysis was obtained from the European Medicines Agency and Orphanet. Seven European states were included in the analysis: Belgium, England, France, Germany, Poland, Scotland, and Spain. For all identified orphan drugs, relevant data on the reimbursement status and type of recommendation were collected for each country. The relationship between the type of recommendation and reimbursement status was evaluated separately for each considered country, using Cohen’s kappa coefficient for the measurement of agreement; sub-analyses for oncology and metabolic drugs were performed. Results: Most reimbursement recommendations for orphan drugs were positive (71%), while approximately 17% were negative and almost 13% were conditional. The highest percentage of positive reimbursement recommendations was observed in Spain (97%) and France (95%) and the highest percentage of negative reimbursement recommendations was revealed for Poland (49%). On average, 65% of the 163 analyzed orphan drugs were reimbursed from public funds. The highest number of reimbursed orphan drugs was observed in Germany (n = 148), while the lowest, in Poland (n = 41). Considering all analyzed drugs, the highest agreement between recommendations and reimbursement status was observed for Spain (value of 1), and the lowest, for Germany (κ = -0.03). Conclusions: On average, more than 60% of identified orphan drugs were reimbursed from public funds in the included countries, and the majority of reimbursement recommendations were found to be positive. The agreement between reimbursement recommendations and reimbursement status differed between the countries, but overall, it did not show any patterns, as it ranged from -0.03 to 1 (κ coefficient).	en
dc.identifier.eissn	1663-9812
dc.identifier.uri	https://depositonce.tu-berlin.de/handle/11303/10408
dc.identifier.uri	http://dx.doi.org/10.14279/depositonce-9360
dc.language.iso	en	en
dc.rights.uri	https://creativecommons.org/licenses/by/4.0/	en
dc.subject.ddc	610 Medizin und Gesundheit	de
dc.subject.other	health technology assessment	en
dc.subject.other	drug policy	en
dc.subject.other	rare disease	en
dc.subject.other	reimbursement	en
dc.subject.other	orphan	en
dc.title	Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries	en
dc.type	Article	en
dc.type.version	publishedVersion	en
dcterms.bibliographicCitation.articlenumber	1279	en
dcterms.bibliographicCitation.doi	10.3389/fphar.2019.01279	en
dcterms.bibliographicCitation.journaltitle	Frontiers in Pharmacology	en
dcterms.bibliographicCitation.originalpublishername	Frontiers Media S.A.	en
dcterms.bibliographicCitation.originalpublisherplace	Lausanne	en
dcterms.bibliographicCitation.volume	10	en
tub.accessrights.dnb	free	en
tub.affiliation	Fak. 7 Wirtschaft und Management::Inst. Technologie und Management (ITM)::FG Management im Gesundheitswesen	de
tub.affiliation.faculty	Fak. 7 Wirtschaft und Management	de
tub.affiliation.group	FG Management im Gesundheitswesen	de
tub.affiliation.institute	Inst. Technologie und Management (ITM)	de
tub.publisher.universityorinstitution	Technische Universität Berlin	en

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries

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