Challenges for gene therapy in the financial sustainability of health systems: a scoping review




Ossandon, Hugo
Armijo, Nicolás
Vargas, Constanza
Espinoza, Manuel

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Aim: To review the available evidence about the strategies implemented or proposed for coverage or reimbursement for currently approved gene therapies. Methods: A scoping review was conducted to analyze the evidence published during the years 2016 to 2023. The main search criteria were coverage or reimbursement of gene therapy by healthcare systems. The eligible articles were those that described or proposed a financing model used to provide coverage in the various systems around the world. Results: The study identified 279 publications, and after removing duplicates and screening for eligibility, 10 were included in the study. The results show that various financing models have been proposed, including subscription-based payment models, outcome-based payment models, and amortization strategies. However, several barriers to implementing these models were identified, such as deficiencies in informatics systems for data collection, changes in laws or regulations, the lack of accessible clinical endpoints and administrative costs. Conclusion: This scoping review provides an overview of financing strategies for gene therapies. Gene therapies can cure rare or previously intractable diseases, but their high cost can make access difficult. Publishing experiences with these models can help evaluate their use and gather more evidence for their effectiveness.



Delivery of Health Care / economics, Genetic Therapy* / economics, Humans


Ossandon, H., Armijo, N., Vargas, C., Repetto, G. M., & Espinoza, M. A. (2024). Challenges for gene therapy in the financial sustainability of health systems: a scoping review. Orphanet journal of rare diseases, 19(1), 243.