A cost-effectiveness dossier was submitted to the HAS for a novel innovative treatment likely to have a significant impact on the health insurance expenditure in cystic fibrosis (CF).

Modelling of CF implied the extrapolation of short-term respiratory, nutritional and infectious exacerbations outcomes from large-scale RCTs to a lifetime time-horizon using applying a survival Cox-model to parametric survival modelling. Other challenges faced were due to the exhaustive approach of cost-effectiveness assessment in the context of rare diseases.