Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment
Objective: To assess shares of reimbursed orphan drugs and agreement in reimbursement decision-making in different European Union member states as well as to define odds for reimbursement influenced by the presence of conditional approval or exceptional circumstances granted by the European Medicine...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2018-11-01
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| Series: | Frontiers in Pharmacology |
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| Online Access: | https://www.frontiersin.org/article/10.3389/fphar.2018.01263/full |
| _version_ | 1818338214028509184 |
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| author | Krzysztof Piotr Malinowski Paweł Kawalec Wojciech Trabka Christoph Sowada Andrzej Pilc Andrzej Pilc |
| author_facet | Krzysztof Piotr Malinowski Paweł Kawalec Wojciech Trabka Christoph Sowada Andrzej Pilc Andrzej Pilc |
| author_sort | Krzysztof Piotr Malinowski |
| collection | DOAJ |
| description | Objective: To assess shares of reimbursed orphan drugs and agreement in reimbursement decision-making in different European Union member states as well as to define odds for reimbursement influenced by the presence of conditional approval or exceptional circumstances granted by the European Medicines Agency (EMA) or by type of the disease.Methods: The list of authorized drugs with current orphan designations was collected from the website of the EMA. For each drug, the information regarding conditional approval or approval under exceptional circumstances was collected. The reimbursement statuses were available on national reimbursement or HTA agencies websites. The agreement for reimbursement decisions between selected countries was assessed using the κ coefficient for the measurement of agreement. The impact of the EMA's conditional approval as well as approval under exceptional circumstances was assessed using the logistic regression and presented as odds ratio.Results: The percentage of reimbursed orphan drugs varied significantly from 27% in Poland to 88% in Denmark, with an average value of 51% (p < 0.0001). Regarding the reimbursement status, the highest, substantial agreement was observed between Spain and Italy, and the lowest agreement was observed between Germany and England, with κ of 0.64 and 0.01, respectively. Conditional approval status significantly decreased the chance for reimbursement in France, Italy, and Spain by 77–80%; however, approval granted under exceptional circumstances had significant impact only in Germany with 85% decrease in chances for reimbursement. The type of the disease (oncology or metabolic) was significantly associated with both conditional approval (p of 0.03—oncology drugs were more likely to be conditionally approved then the rest of analyzed drugs) and exceptional circumstances (p of 0.02—drugs for metabolic diseases were more likely to be approved under exceptional circumstances).Conclusions: Access to reimbursed orphan drugs varies significantly across EU countries. The highest, substantial agreement in reimbursement decisions was observed between Italy and Spain and the lowest between Germany and England. Conditional approval and approval under exceptional circumstances were significant negative predictors of reimbursement in some countries and they were significantly associated with the type of the disease (oncology or metabolic). |
| first_indexed | 2024-12-13T15:07:33Z |
| format | Article |
| id | doaj.art-fe5d4fecc29b4ae8a378341a5139b056 |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-12-13T15:07:33Z |
| publishDate | 2018-11-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-fe5d4fecc29b4ae8a378341a5139b0562022-12-21T23:40:58ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122018-11-01910.3389/fphar.2018.01263418166Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology AssessmentKrzysztof Piotr Malinowski0Paweł Kawalec1Wojciech Trabka2Christoph Sowada3Andrzej Pilc4Andrzej Pilc5Faculty of Health Sciences, Institute of Public Health, Jagiellonian University Medical College, Krakow, PolandFaculty of Health Sciences, Institute of Public Health, Jagiellonian University Medical College, Krakow, PolandBioinformatics and Public Health Department, Faculty of Medicine and Health Sciences, Andrzej Frycz Modrzewski Krakow University, Krakow, PolandFaculty of Health Sciences, Institute of Public Health, Jagiellonian University Medical College, Krakow, PolandFaculty of Health Sciences, Institute of Public Health, Jagiellonian University Medical College, Krakow, PolandInstitute of Pharmacology, Polish Academy of Sciences, Krakow, PolandObjective: To assess shares of reimbursed orphan drugs and agreement in reimbursement decision-making in different European Union member states as well as to define odds for reimbursement influenced by the presence of conditional approval or exceptional circumstances granted by the European Medicines Agency (EMA) or by type of the disease.Methods: The list of authorized drugs with current orphan designations was collected from the website of the EMA. For each drug, the information regarding conditional approval or approval under exceptional circumstances was collected. The reimbursement statuses were available on national reimbursement or HTA agencies websites. The agreement for reimbursement decisions between selected countries was assessed using the κ coefficient for the measurement of agreement. The impact of the EMA's conditional approval as well as approval under exceptional circumstances was assessed using the logistic regression and presented as odds ratio.Results: The percentage of reimbursed orphan drugs varied significantly from 27% in Poland to 88% in Denmark, with an average value of 51% (p < 0.0001). Regarding the reimbursement status, the highest, substantial agreement was observed between Spain and Italy, and the lowest agreement was observed between Germany and England, with κ of 0.64 and 0.01, respectively. Conditional approval status significantly decreased the chance for reimbursement in France, Italy, and Spain by 77–80%; however, approval granted under exceptional circumstances had significant impact only in Germany with 85% decrease in chances for reimbursement. The type of the disease (oncology or metabolic) was significantly associated with both conditional approval (p of 0.03—oncology drugs were more likely to be conditionally approved then the rest of analyzed drugs) and exceptional circumstances (p of 0.02—drugs for metabolic diseases were more likely to be approved under exceptional circumstances).Conclusions: Access to reimbursed orphan drugs varies significantly across EU countries. The highest, substantial agreement in reimbursement decisions was observed between Italy and Spain and the lowest between Germany and England. Conditional approval and approval under exceptional circumstances were significant negative predictors of reimbursement in some countries and they were significantly associated with the type of the disease (oncology or metabolic).https://www.frontiersin.org/article/10.3389/fphar.2018.01263/fullEMA (European Medicines Agency)orphan drugs for rare diseasesreimbursementauthorizationHTA (Health Technology Assessment) |
| spellingShingle | Krzysztof Piotr Malinowski Paweł Kawalec Wojciech Trabka Christoph Sowada Andrzej Pilc Andrzej Pilc Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment Frontiers in Pharmacology EMA (European Medicines Agency) orphan drugs for rare diseases reimbursement authorization HTA (Health Technology Assessment) |
| title | Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment |
| title_full | Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment |
| title_fullStr | Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment |
| title_full_unstemmed | Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment |
| title_short | Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment |
| title_sort | reimbursement of orphan drugs in europe in relation to the type of authorization by the european medicines agency and the decision making based on health technology assessment |
| topic | EMA (European Medicines Agency) orphan drugs for rare diseases reimbursement authorization HTA (Health Technology Assessment) |
| url | https://www.frontiersin.org/article/10.3389/fphar.2018.01263/full |
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