A systematic review of cost-effectiveness analyses of gene therapy for hemophilia type A and B

Recently, the US Food and Drug Administration approved 2 novel single-administration gene therapies to treat the rare bleeding disorders hemophilia A and B. The early data look promising for both therapies, but they have been questioned for their high costs. This study showed that the gene therapies...

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Bibliographic Details
Published in:Journal of managed care & specialty pharmacy 2024-10, Vol.30 (10), p.1178-1188
Main Authors: Alshehri, Alaa, Dougherty, John A., Beckman, Linda, Svensson, Mikael
Format: Article
Language:English
Subjects:
Farmakologi och toxikologi
Pharmacology and Toxicology
Systematic Review
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Summary:Recently, the US Food and Drug Administration approved 2 novel single-administration gene therapies to treat the rare bleeding disorders hemophilia A and B. The early data look promising for both therapies, but they have been questioned for their high costs. This study showed that the gene therapies may provide long-term value, but it depends on data used in the economic evaluation and how long the treatment effect is sustained, which is currently uncertain. Novel hemophilia A and B gene therapies can potentially provide long-term value for patients and payers, but there is considerable uncertainty around the magnitude and durability of the treatment effect of these gene therapies. Outcomes-based agreements that tie payments and/or discounts to patient outcomes can be used to share the risk between manufacturers and payers to facilitate patient access while more clinical data are collected.
ISSN:2376-0540
2376-1032
2376-1032
DOI:10.18553/jmcp.2024.30.10.1178