Economic evaluation of CFTR modulator therapies in cystic fibrosis: a cost-benefit and cost-effectiveness perspective from the REMEDIA European commission project.

BACKGROUND: Cystic fibrosis (CF) is a rare, life-limiting genetic disorder characterized by progressive respiratory decline and multisystem complications. The introduction of CFTR (cystic fibrosis transmembrane conductance regulator) modulator therapies has transformed disease management, improving quality of life and survival. However, these therapies are associated with extremely high costs, raising questions regarding their economic value and sustainability within public healthcare systems. The objective of this study was to perform a detailed cost-benefit and cost-effectiveness analysis of CFTR modulators compared to standard CF care, incorporating both clinical outcomes and economic impacts.

METHODS: The study employed probabilistic simulation models over a 10-year horizon using Monte Carlo techniques. Costs and benefits were derived from published literature and real-world datasets, with a discount rate of 3% applied. Economic metrics included avoided complication costs, cost per life-year saved, and relative/absolute cost-benefit ratios.

RESULTS: CFTR modulators reduced complication costs by an average of €75,493 compared to standard care. The average absolute cost-benefit was €2,001,964, and the relative cost-benefit ratio showed that therapy costs were 60 times higher than savings. The cost-effectiveness analysis yielded €239,336 per life-year saved.

CONCLUSION: While CFTR modulators provide significant clinical advantages, their very high costs don't allow to establish any cost-benefit or cost-effectiveness advantages. These findings support the need for price realignment and strategic health policy to enhance access and sustainability of these innovative therapies targeting CF patients.