New cystic fibrosis drug ivacaftor (Kalydeco) to be made available for Irish patients

4th February, 2013


Dr James Reilly TD, Minister for Health today (1st February 2013) announced that Kalydeco, a new innovative medicine for the treatment of cystic fibrosis (CF) in patients who have the G551D mutation, will be made available for patients in Ireland from 1st March next. The decision was made following a request from the Director General Designate of the HSE, Tony O’Brien for a policy direction, prior to its implementation.

Cystic Fibrosis is a genetic condition caused by mutations in the CF transmembrane conductance regulator (CFTR) protein and Kalydeco is the first in a new class of drugs known as CFTR potentiators. As just over 10% of patients with CF in Ireland have the G551D mutation it is estimated that approximately 120 patients will be suitable for treatment with the new drug.

As with all new drugs Kalydeco was assessed by the National Centre for Pharmacoeconomics (NCPE) in January 2013. The assessment acknowledged the benefits of the drug including significant improvements in lung function, increased body weight, improvement in patients’ quality of life and a 55% reduction in pulmonary exacerbations. The assessment did however highlight the high cost of the drug at over € 234,000 per patient per annum and the significant budget impact at over € 28 million per annum. The NCPE recommended against reimbursing the drug at the submitted price but suggested mechanisms such as a risk sharing scheme in addition to a price reduction could enable patients to gain access to Kalydeco.

The NCPE recommendation led to follow-up discussions between the HSE Corporate Pharmaceutical Unit and the Company, Vertex Pharmaceuticals. These discussions provided a mechanism where the drug could be made available to patients whilst at the same time reducing significantly the budget impact of the drug. They were considered by the HSE Drugs Group and a positive recommendation was made supporting the provision of Kalydeco for Irish CF patients.