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Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent ‘value for money’ for the NHS? A rapid inquiry into suggestions that it may be cost-effective
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Connock, Martin, Andronis, Lazaros, Auguste, Peter, Dussart, Claude and Armoiry, Xavier (2020) Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent ‘value for money’ for the NHS? A rapid inquiry into suggestions that it may be cost-effective. Expert Opinion on Biological Therapy, 20 (7). pp. 823-827. doi:10.1080/14712598.2020.1772747 ISSN 1471-2598.
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WRAP-Will-Onasemnogene-abeparvosec-treatment-spinal-Armoiry-2020.pdf - Accepted Version - Requires a PDF viewer. Download (1046Kb) | Preview |
Official URL: http://dx.doi.org/10.1080/14712598.2020.1772747
Abstract
Objectives
Nusinersen (Spinraza®, Biogen) and onasemnogene abeparvosec (Zolgensma®, Novartis) are novel gene-based therapies for the orphan disease Spinal Muscular Atrophy. Onasemnogene abeparvosec has been allocated an acquisition cost of up to US$5 million per patient. We undertook a rapid inquiry to evaluate if onasemnogene abeparvosec is likely to be cost effective for the UK NHS.
Methods
We used publicly available cost effectiveness data and recommended methodology to perform cost utility evaluation of onasemnogene abeparvosec versus best supportive care and nusinersen.
Results
Our evaluations highlight wide variations in cost and benefit estimates of nusinersen and indicate that onasemnogene abeparvosec is unlikely to represent value for money according to current standards of reimbursement. Results are discussed in the context of reimbursement decisions for orphan diseases.
Conclusion
Commonly implemented commercial confidentiality practices combined with uncertain data obscure scrutiny and justification of past and present reimbursement decisions for orphan drugs. Future cutting edge expensive therapies will be numerous, they will entail very substantial economic strains. We conclude that there is an urgent and increasing need for the development of improved procedures that can lead to equitable, consistent and transparent decision making.
| Item Type: | Journal Article | ||||||||
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| Subjects: | R Medicine > RB Pathology R Medicine > RD Surgery |
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| Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences > Population, Evidence & Technologies (PET) Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
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| Library of Congress Subject Headings (LCSH): | Spinal muscular atrophy, Spinal muscular atrophy -- Gene therapy, Spinal muscular atrophy -- Gene therapy -- Cost effectiveness, Gene therapy -- Effectiveness | ||||||||
| Journal or Publication Title: | Expert Opinion on Biological Therapy | ||||||||
| Publisher: | Taylor & Francis | ||||||||
| ISSN: | 1471-2598 | ||||||||
| Official Date: | 11 June 2020 | ||||||||
| Dates: |
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| Volume: | 20 | ||||||||
| Number: | 7 | ||||||||
| Page Range: | pp. 823-827 | ||||||||
| DOI: | 10.1080/14712598.2020.1772747 | ||||||||
| Status: | Peer Reviewed | ||||||||
| Publication Status: | Published | ||||||||
| Reuse Statement (publisher, data, author rights): | “This is an Accepted Manuscript of an article published by Taylor & Francis in Expert Opinion on Biological Therapy on 20/05/2020, available online: http://www.tandfonline.com/10.1080/14712598.2020.1772747 | ||||||||
| Access rights to Published version: | Open Access (Creative Commons) | ||||||||
| Date of first compliant deposit: | 27 May 2020 | ||||||||
| Date of first compliant Open Access: | 20 May 2021 |
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