Trikafta® for the treatment of patients with cystic fibrosis

Cystic fibrosis is a hereditary metabolic disease, in which a defective gene leads to the formation of mucus in the lungs, pancreas and intestines. If untreated, the disease is associated with reduced life expectancy. In Switzerland, since 2021, the drug Trikafta® has been authorised for the treatment of patients aged 6 years and older who have at least one copy of the F508del mutation in the CFTR gene. It is to be reimbursed under the mandatory health insurance scheme for a limited period (until 31 January 2025). This report analyses the cost-effectiveness and the cost implications of Trikafta® compared to standard care.

A pragmatic systematic literature review identified three studies and a network meta analysis, which evaluated safety and efficacy up to 48 weeks compared to standard care. Most of the reported adverse events (e.g. skin rash, gastrointestinal symptoms, headache and psychiatric disorders) were associated with the underlying disease. No studies are available on long-term effectiveness or on mortality.

The calculation of cost-effectiveness involves major uncertainties, especially for key variables such as the impact on mortality and on pulmonary function, and the associated impact on quality of life and disease management costs. To deal with these uncertainties, several modelling scenarios were developed on the basis of information from previous HTA reports. Across almost all scenarios, when applying the official list price for Trikafta® (as of December 2023), the additional costs per quality-adjusted life year gained (ICER) exceeded CHF 1 million. The high additional costs are due to the recurrent annual treatment costs of CHF 228,000 per patient.

The report concludes that there are uncertainties regarding the cost-effectiveness of treatment with Trikafta® in the scenarios analysed. However, the cost-effectiveness is mainly determined by the recurrent annual treatment costs. The total budget impact of treating all patients aged 6 and over would be around CHF 160 million per year.

Last modification 27.06.2024

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