A systematic review of moral reasons on orphan drug reimbursement
Abstract Background The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing number of approved OMPs increases th...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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BMC
2021-06-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-021-01925-y |
| _version_ | 1818724094421499904 |
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| author | Bettina M. Zimmermann Johanna Eichinger Matthias R. Baumgartner |
| author_facet | Bettina M. Zimmermann Johanna Eichinger Matthias R. Baumgartner |
| author_sort | Bettina M. Zimmermann |
| collection | DOAJ |
| description | Abstract Background The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing number of approved OMPs increases their budget impact despite their low prevalence, making it pressing to find solutions to ethical challenges on how to fairly allocate scarce healthcare resources under this context. One potential solution could be to grant OMPs special status when considering them for reimbursement, meaning that they are subject to different, and less stringent criteria than other drugs. This study aims to provide a systematic analysis of moral reasons for and against such a special status for the reimbursement of OMPs in publicly funded healthcare systems from a multidisciplinary perspective. Results With a systematic review of reasons, we identified 39 reasons represented in 243 articles (scientific and grey literature) for and against special status for the reimbursement of OMPs, then categorized them into nine topics. Taking a multidisciplinary perspective, we found that most articles came from health policy (n = 103) and health economics (n = 49). More articles took the position for a special status of OMPs (n = 97) than those against it (n = 31) and there was a larger number of reasons identified in favour (29 reasons) than against (10 reasons) this special status. Conclusion Results suggest that OMP reimbursement issues should be assessed and analysed from a multidisciplinary perspective. Despite the higher occurrence of reasons and articles in favour of a special status, there is no clear-cut solution for this ethical challenge. The binary perspective of whether or not OMPs should be granted special status oversimplifies the issue: both OMPs and rare diseases are too heterogeneous in their characteristics for such a binary perspective. Thus, the scientific debate should focus less on the question of disease prevalence but rather on how the important variability of different OMPs concerning e.g. target population, cost-effectiveness, level of evidence or mechanism of action could be meaningfully addressed and implemented in Health Technology Assessments. |
| first_indexed | 2024-12-17T21:20:57Z |
| format | Article |
| id | doaj.art-c508a3af126c47dba7db2c7e39deaa14 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-12-17T21:20:57Z |
| publishDate | 2021-06-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-c508a3af126c47dba7db2c7e39deaa142022-12-21T21:32:12ZengBMCOrphanet Journal of Rare Diseases1750-11722021-06-0116112110.1186/s13023-021-01925-yA systematic review of moral reasons on orphan drug reimbursementBettina M. Zimmermann0Johanna Eichinger1Matthias R. Baumgartner2Institute for Biomedical Ethics, University of BaselInstitute for Biomedical Ethics, University of BaselDivision of Metabolism and Children’s Research Center, University Children’s Hospital Zurich, University of ZurichAbstract Background The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing number of approved OMPs increases their budget impact despite their low prevalence, making it pressing to find solutions to ethical challenges on how to fairly allocate scarce healthcare resources under this context. One potential solution could be to grant OMPs special status when considering them for reimbursement, meaning that they are subject to different, and less stringent criteria than other drugs. This study aims to provide a systematic analysis of moral reasons for and against such a special status for the reimbursement of OMPs in publicly funded healthcare systems from a multidisciplinary perspective. Results With a systematic review of reasons, we identified 39 reasons represented in 243 articles (scientific and grey literature) for and against special status for the reimbursement of OMPs, then categorized them into nine topics. Taking a multidisciplinary perspective, we found that most articles came from health policy (n = 103) and health economics (n = 49). More articles took the position for a special status of OMPs (n = 97) than those against it (n = 31) and there was a larger number of reasons identified in favour (29 reasons) than against (10 reasons) this special status. Conclusion Results suggest that OMP reimbursement issues should be assessed and analysed from a multidisciplinary perspective. Despite the higher occurrence of reasons and articles in favour of a special status, there is no clear-cut solution for this ethical challenge. The binary perspective of whether or not OMPs should be granted special status oversimplifies the issue: both OMPs and rare diseases are too heterogeneous in their characteristics for such a binary perspective. Thus, the scientific debate should focus less on the question of disease prevalence but rather on how the important variability of different OMPs concerning e.g. target population, cost-effectiveness, level of evidence or mechanism of action could be meaningfully addressed and implemented in Health Technology Assessments.https://doi.org/10.1186/s13023-021-01925-yRare diseasesOrphan diseasesOrphan drugsReimbursementEthicsAllocation of resources |
| spellingShingle | Bettina M. Zimmermann Johanna Eichinger Matthias R. Baumgartner A systematic review of moral reasons on orphan drug reimbursement Orphanet Journal of Rare Diseases Rare diseases Orphan diseases Orphan drugs Reimbursement Ethics Allocation of resources |
| title | A systematic review of moral reasons on orphan drug reimbursement |
| title_full | A systematic review of moral reasons on orphan drug reimbursement |
| title_fullStr | A systematic review of moral reasons on orphan drug reimbursement |
| title_full_unstemmed | A systematic review of moral reasons on orphan drug reimbursement |
| title_short | A systematic review of moral reasons on orphan drug reimbursement |
| title_sort | systematic review of moral reasons on orphan drug reimbursement |
| topic | Rare diseases Orphan diseases Orphan drugs Reimbursement Ethics Allocation of resources |
| url | https://doi.org/10.1186/s13023-021-01925-y |
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