(en) [Your money or your life? - Kalydeco®: a milestone towards the “cure” of cystic fibrosis] From February 1st 2016 on, Ivacaftor (KALYDECO™) will be reimbursed in Belgium for the treatment of cystic fibrosis (CF) in patients aged 6 years and older carrying at least one of nine well characterized gating (Class III) cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations, which account for approximately 3% of Belgian CF patients. In most of these patients, this very expensive oral drug (248,000 €/year) substantially improves lung function, lowers sweat chloride levels, and improves body weight by targeting the primary defect, thus representing a fundamental shift in the way the disease is managed. However, clinicians should be aware of the drug's potential for numerous drug interactions.
Lebecque, O., Godding, V., & Lebecque, P. (2016). Innovations en Pneumologie pédiatrique. Que retenir de 2015? Louvain médical, 135(2), 61-64. https://hdl.handle.net/2078.5/184863 (Original work published 2016)