Extension of Kalydeco (Ivacaftor): Update from NCPE

Extension of Kalydeco (Ivacaftor): Update from NCPE

Today (12 October), the National Centre for Pharmacoeconomics (NCPE) have issued their recommendation following the Health Technology Assessment (HTA) of Kalydeco for children aged 2- 5years. Kalydeco for children aged 2–5 year olds* has not been recommended for reimbursement at the submitted price. (Note: this is not related to the forthcoming Orkambi decision)

CFI notes that the NCPE ruling leaves a door open to further price negotiations, but the decision will be very disappointing and frustrating for the 18 young children with CF and their families, who could benefit greatly from access to this ground-breaking therapy.

Philip Watt, CEO of Cystic Fibrosis Ireland says “Accessing this life-changing treatment is essential as eligible patients stand to benefit so much at such a crucial time in their development, fighting a progressive, life-limiting disease. We will be urging the government to enter into price negotiations as soon as possible. We would also urge the pharma company, Vertex, to reduce the price of this important drug during these negotiations”.

The treatment, which is currently only available for those over six years old, has been shown to significantly increase lung function, slow the progression of lung disease and significantly reduce hospital admissions.

Research published earlier this year in ‘The Lancet Respiratory Medicine’ journal suggested that pre-school children with one of the nine mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) could also benefit from improved pancreatic function, weight gain and movement of salt between cells.


*This assessment refers to children aged 2-5years who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P,S549N or S549R.