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Reimbursement status and recommendations related to orphan drugs in European countries
Stawowczyk, E. (University of Bielsko-Biala. Faculty of Health Sciences)
Malinowski, K. P. (Jagiellonian University Medical College)
Kawalec, P. (Jagiellonian University Medical College)
Bobiński, R. (Jagiellonian University Medical College)
Siwiec, J. (Jagiellonian University Medical College)
Panteli, D. (European Observatory on Health Systems and Policies)
Eckhardt, H. (European Observatory on Health Systems and Policies)
Simoens, Steven (KU Leuven. Department of Pharmaceutical and Pharmacological Sciences)
Agustí Escasany, M. Antònia (Universitat Autònoma de Barcelona. Departament de Farmacologia, de Terapèutica i de Toxicologia)
Dooms, M. (University Hospitals Leuven (Bèlgica))
Pilc, A. (Polish Academy of Sciences. Institute of Pharmacology)

Date: 2019
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).
Note: Zelei, T., Molnár, M. J., Szegedi, M., and Kaló, Z. (2016). Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries. Orphanet. J. Rare Dis. 11 (1), 72. doi: 10.1186/ s13023-016-0455-6 Conflict of Interest: SS is grant holder of a project on "How to reimburse and pay orphan drugs for rare diseases?" funded by the Scientific Research Foundation Flanders.
Note: This study was conducted within the statutory project: "Reimbursement and clinical aspects of orphan drug policy in Poland and Europe" (project number: K/ZDS/007863). We would like to thank Francis Arickx for help in data acquisition for Belgium.
Rights: Tots els drets reservats.
Language: Anglès
Document: Article ; recerca ; Versió publicada
Published in: Frontiers in Pharmacology, Vol. 10 (2019), p. 1279, ISSN 1663-9812

DOI: 10.3389/fphar.2019.01279
PMID: 31827433


9 p, 929.7 KB

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Articles > Research articles
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 Record created 2020-06-03, last modified 2023-12-03



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