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Cystic Fibrosis Treatment Advancement with Trikafta®

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Cystic fibrosis is a genetic metabolic disorder resulting from a defective gene that causes mucus buildup in the lungs, pancreas, and intestines. Without treatment, this condition significantly reduces life expectancy. In Switzerland, the drug Trikafta® has been approved since 2021 for individuals aged six and above who possess at least one F508del mutation in the CFTR gene. The drug is covered by compulsory health insurance until January 31, 2025. This report evaluates the cost-benefit ratio and the financial implications of Trikafta® compared to standard therapies.

A pragmatic systematic literature search identified three studies and one network meta-analysis that examined the safety and efficacy of Trikafta® for up to 48 weeks compared to standard treatment. The adverse events noted, including skin rashes, gastrointestinal complaints, headaches, and mental disorders, were primarily related to the underlying disease rather than the treatment itself. However, there are no studies available on the long-term efficacy or mortality impacts of Trikafta®.

Cost-Benefit Uncertainties of Trikafta® in Managing Cystic Fibrosis: A Financial Perspective

The cost-benefit analysis of Trikafta® is fraught with significant uncertainties, especially concerning critical factors like mortality, lung function, associated quality of life, and the costs of managing cystic fibrosis. To address these uncertainties, models with varying scenarios based on previous Health Technology Assessment (HTA) reports were employed. When Trikafta® is used at its official price as of December 2023, the additional costs per year of life gained in full health exceed 1 million Swiss francs in nearly all scenarios. This high cost is largely due to the annual recurring treatment expenses of 228,000 Swiss francs per person.

The report concludes that there are considerable uncertainties in the cost-benefit ratio of Trikafta® across the examined scenarios. The annual recurring treatment costs significantly contribute to the high additional costs per year of life gained with full health. Treating all affected individuals aged six and above with Trikafta® would impact the cost budget by approximately 160 million Swiss francs annually.

Cystic Fibrosis

Innovative Treatments for Cystic Fibrosis: The Promises and Costs of Trikafta®

In summary, cystic fibrosis, a serious genetic disorder, requires innovative treatments to manage its symptoms and improve patient outcomes. Trikafta®, a relatively new treatment option, has shown promise but comes with substantial costs and uncertainties. The literature search included three studies and one network meta-analysis, all highlighting safety and efficacy concerns over 48 weeks. Adverse events noted were predominantly linked to the underlying disease.

There are no long-term studies on the effectiveness or mortality benefits of Trikafta®. The cost-benefit analysis shows significant uncertainties, particularly in mortality, lung function, quality of life, and overall disease management costs. Despite the potential benefits, the high treatment costs of 228,000 Swiss francs per person per year result in additional costs per year of life gained in full health, often exceeding 1 million Swiss francs.

Models based on previous HTA reports were used to mitigate some uncertainties. Nevertheless, the annual treatment costs remain a critical factor. Treating all eligible individuals with Trikafta® would cost around 160 million Swiss francs per year. This significant financial impact raises questions about the sustainability and practicality of widespread Trikafta® use.

In conclusion, while Trikafta® represents a significant advancement in the treatment of cystic fibrosis, its high cost and associated uncertainties pose challenges for healthcare systems. The financial burden, especially with annual treatment costs of 228,000 Swiss francs per person, must be carefully considered. Comprehensive, long-term studies are needed to better understand the full impact of Trikafta® on mortality, quality of life, and overall healthcare costs. Only with this information can a more accurate assessment of the cost-benefit ratio be made, ensuring that patients receive the most effective and sustainable care possible.

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Resource: Federal Office of Public Health , June 27, 2024

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