Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries
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 dr...
| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2019-11-01
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| Series: | Frontiers in Pharmacology |
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| Online Access: | https://www.frontiersin.org/article/10.3389/fphar.2019.01279/full |
| _version_ | 1818588102243909632 |
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| author | Ewa Stawowczyk Krzysztof Piotr Malinowski Paweł Kawalec Rafał Bobiński Jacek Siwiec Dimitra Panteli Dimitra Panteli Dimitra Panteli Helene Eckhardt Helene Eckhardt Helene Eckhardt Steven Simoens Antònia Agusti Antònia Agusti Marc Dooms Andrzej Pilc Andrzej Pilc |
| author_facet | Ewa Stawowczyk Krzysztof Piotr Malinowski Paweł Kawalec Rafał Bobiński Jacek Siwiec Dimitra Panteli Dimitra Panteli Dimitra Panteli Helene Eckhardt Helene Eckhardt Helene Eckhardt Steven Simoens Antònia Agusti Antònia Agusti Marc Dooms Andrzej Pilc Andrzej Pilc |
| author_sort | Ewa Stawowczyk |
| collection | DOAJ |
| description | 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). |
| first_indexed | 2024-12-16T09:19:25Z |
| format | Article |
| id | doaj.art-440618f808e04dfa9ef508702e733fe0 |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-12-16T09:19:25Z |
| publishDate | 2019-11-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-440618f808e04dfa9ef508702e733fe02022-12-21T22:36:48ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122019-11-011010.3389/fphar.2019.01279466507Reimbursement Status and Recommendations Related to Orphan Drugs in European CountriesEwa Stawowczyk0Krzysztof Piotr Malinowski1Paweł Kawalec2Rafał Bobiński3Jacek Siwiec4Dimitra Panteli5Dimitra Panteli6Dimitra Panteli7Helene Eckhardt8Helene Eckhardt9Helene Eckhardt10Steven Simoens11Antònia Agusti12Antònia Agusti13Marc Dooms14Andrzej Pilc15Andrzej Pilc16Faculty of Health Sciences, University of Bielsko-Biala, Bielsko-Biała, PolandInstitute of Public Health, Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, PolandInstitute of Public Health, Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, PolandFaculty of Health Sciences, University of Bielsko-Biala, Bielsko-Biała, PolandInstitute of Public Health, Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, PolandDepartment of Health Care Management, Berlin University of Technology, Berlin, GermanyWHO Collaborating Centre for Health Systems Research and Management, Berlin, GermanyResearch Hub of the European Observatory on Health Systems and Policies, Berlin, GermanyDepartment of Health Care Management, Berlin University of Technology, Berlin, GermanyWHO Collaborating Centre for Health Systems Research and Management, Berlin, GermanyResearch Hub of the European Observatory on Health Systems and Policies, Berlin, GermanyDepartment of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, BelgiumClinical Pharmacology Service, Catalan Institute of Pharmacology Foundation, Vall d’hebron University Hospital, Barcelona, SpainDepartment of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, SpainUniversity Hospitals Leuven, Leuven, BelgiumInstitute of Public Health, Faculty of Health Sciences, Jagiellonian University Medical College, Krakow, Poland0Institute of Pharmacology, Polish Academy of Sciences, Krakow, PolandObjective: 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).https://www.frontiersin.org/article/10.3389/fphar.2019.01279/fullhealth technology assessmentdrug policyrare diseasereimbursementorphan |
| spellingShingle | Ewa Stawowczyk Krzysztof Piotr Malinowski Paweł Kawalec Rafał Bobiński Jacek Siwiec Dimitra Panteli Dimitra Panteli Dimitra Panteli Helene Eckhardt Helene Eckhardt Helene Eckhardt Steven Simoens Antònia Agusti Antònia Agusti Marc Dooms Andrzej Pilc Andrzej Pilc Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries Frontiers in Pharmacology health technology assessment drug policy rare disease reimbursement orphan |
| title | Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries |
| title_full | Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries |
| title_fullStr | Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries |
| title_full_unstemmed | Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries |
| title_short | Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries |
| title_sort | reimbursement status and recommendations related to orphan drugs in european countries |
| topic | health technology assessment drug policy rare disease reimbursement orphan |
| url | https://www.frontiersin.org/article/10.3389/fphar.2019.01279/full |
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