Welcome for cystic fibrosis drug u-turn

Longford/Westmeath Fine Gael TD Nicky McFadden has welcomed the announcement by Minister for Health James Reilly 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 March 1 next. "I am delighted that kalydeco is to be made available. It will make a huge difference to CF patients. I have been receiving correspondence about the availability of this drug over the last few weeks and I know that the news today will bring great relief and hope to many people. "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," Deputy McFadden said. Kalydeco was assessed by the National Centre for Pharmacoeconomics (NCPE) in January. 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. Minister James Reilly pointed out, however, that the assessment also highlighted 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 price but suggested that mechanisms such as a risk sharing scheme in addition to a price reduction could enable patients to gain access to Kalydeco. This led to follow-up discussions between the HSE Corporate Pharmaceutical Unit and the company, Vertex Pharmaceuticals. And Deputy McFadden explained: "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."