دورية أكاديمية
Assessing the value of orphan drugs using conventional cost-effectiveness analysis:Is it fit for purpose?
| العنوان: | Assessing the value of orphan drugs using conventional cost-effectiveness analysis:Is it fit for purpose? |
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| المؤلفون: | Postma, Maarten J., Noone, Declan, Rozenbaum, Mark H., Carter, John A., Botteman, Marc F., Fenwick, Elisabeth, Garrison, Louis P. |
| المصدر: | Postma , M J , Noone , D , Rozenbaum , M H , Carter , J A , Botteman , M F , Fenwick , E & Garrison , L P 2022 , ' Assessing the value of orphan drugs using conventional cost-effectiveness analysis : Is it fit for purpose? ' , Orphanet journal of rare diseases , vol. 17 , no. 1 , 157 . https://doi.org/10.1186/s13023-022-02283-zTest |
| سنة النشر: | 2022 |
| المجموعة: | University of Groningen research database |
| مصطلحات موضوعية: | Health equity, Orphan drug policy, Pharmaceutical policy, Rare diseases, Value of life, Value-based pricing |
| الوصف: | Conventional cost-effectiveness analysis-i.e., assessing pharmaceuticals through a cost per quality-adjusted life year (QALY) framework-originated from a societal commitment to maximize population health given limited resources. This "extra-welfarist" approach has produced pricing and reimbursement systems that are not well- aligned with the unique considerations of orphan drugs. This framework has been slow to evolve along with our increased understanding of the impact of rare diseases, which in turn has complicated the assessment of orphan drugs meant to treat rare diseases. Herein, we (i) discuss the limitations of conventional cost-effectiveness analysis as applied to assessing access to, as well as the pricing and reimbursement of, orphan drugs, (ii) critically appraise alternative and supplemental approaches, and (iii) offer insights on plausible steps forward. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| العلاقة: | https://research.rug.nl/en/publications/dbc4a82a-1d26-44b1-ab98-1021ecdca3feTest |
| DOI: | 10.1186/s13023-022-02283-z |
| الإتاحة: | https://doi.org/10.1186/s13023-022-02283-zTest https://hdl.handle.net/11370/dbc4a82a-1d26-44b1-ab98-1021ecdca3feTest https://research.rug.nl/en/publications/dbc4a82a-1d26-44b1-ab98-1021ecdca3feTest https://pure.rug.nl/ws/files/216918105/Assessing_the_value_of_orphan_drugs_using_conventional_cost_effectiveness_analysis_Is_it_fit_for_purpose_.pdfTest http://www.scopus.com/inward/record.url?scp=85127594287&partnerID=8YFLogxKTest |
| حقوق: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.819C7DB2 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1186/s13023-022-02283-z |
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