Comparing access to orphan medicinal products in Europe
Abstract Objectives The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2019-05-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s13023-019-1078-5 |
| _version_ | 1818317439608291328 |
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| author | Bernarda Zamora Francois Maignen Phill O’Neill Jorge Mestre-Ferrandiz Martina Garau |
| author_facet | Bernarda Zamora Francois Maignen Phill O’Neill Jorge Mestre-Ferrandiz Martina Garau |
| author_sort | Bernarda Zamora |
| collection | DOAJ |
| description | Abstract Objectives The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service. Methods Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared. Results We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation – but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average. Conclusions Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain. |
| first_indexed | 2024-12-13T09:37:21Z |
| format | Article |
| id | doaj.art-7b612a1ac22c4a4b9a57713dfb10861f |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-12-13T09:37:21Z |
| publishDate | 2019-05-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-7b612a1ac22c4a4b9a57713dfb10861f2022-12-21T23:52:19ZengBMCOrphanet Journal of Rare Diseases1750-11722019-05-0114111210.1186/s13023-019-1078-5Comparing access to orphan medicinal products in EuropeBernarda Zamora0Francois Maignen1Phill O’Neill2Jorge Mestre-Ferrandiz3Martina Garau4Office of Health EconomicsOffice of Health EconomicsOffice of Health EconomicsOffice of Health EconomicsOffice of Health EconomicsAbstract Objectives The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service. Methods Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared. Results We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation – but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average. Conclusions Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.http://link.springer.com/article/10.1186/s13023-019-1078-5Orphan drugsAccessHTAReimbursement |
| spellingShingle | Bernarda Zamora Francois Maignen Phill O’Neill Jorge Mestre-Ferrandiz Martina Garau Comparing access to orphan medicinal products in Europe Orphanet Journal of Rare Diseases Orphan drugs Access HTA Reimbursement |
| title | Comparing access to orphan medicinal products in Europe |
| title_full | Comparing access to orphan medicinal products in Europe |
| title_fullStr | Comparing access to orphan medicinal products in Europe |
| title_full_unstemmed | Comparing access to orphan medicinal products in Europe |
| title_short | Comparing access to orphan medicinal products in Europe |
| title_sort | comparing access to orphan medicinal products in europe |
| topic | Orphan drugs Access HTA Reimbursement |
| url | http://link.springer.com/article/10.1186/s13023-019-1078-5 |
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