Funding approved for life-changing CF drug

Minister for Health James Reilly said today the Government had approved funding for a life-changing drug used in the treatment of cystic fibrosis which will benefit about 120 people suffering from the disease.

Dr Reilly said his department had made provision for "€20 million plus funding a year" to provide for the supply of the drug, Kalydeco, which will become available from March 1st to some 120 children and teenagers with a particular type of CF.

Dr Reilly said that he had received a letter from the HSE chief executive Tony O'Brien seeking a policy directive on providing the new drug, and following discussions with Taoiseach Enda Kenny, they had decided today to approve funding for the drug.

"I think this is a very positive story and it's certainly going to improve the quality of life for 120 Irish citizens with a particular form of cystic fibrosis .... I think this is a good breakthrough and offers more in the way of hope to people with cystic fibrosis in this country," he said.

"I don't want to endanger the deal but we have made provision for it in our budget - there were protracted negotiations and a final figure has been reached - it's a substantial figure but nonetheless, how do you value the life of a child?"

Dr Reilly made the announcement when opening the Mercy Urgent Care Centre in Gurranebraher in Cork where he was accompanied by Minister for State Kathleen Lynch and Cork North West Fine Gael TD Aine Collins, whose daughter, Lily, suffers from CF. "I’m personally delighted. I have a daughter who has cystic fibrosis and she has that particular gene type and this will literally change her life and bring new hope to all of us - as parent, all my hopes and dreams have come true with this announcement," Ms Collins said.

Cystic fibrosis campaigner and Irish Times columnist Orla Tinsley said it is a "wonderful" day for people with cystic fibrosis in Ireland.

"This is a wonderful step forward for people with CF in Ireland. It is a great result after a frustrating wait," she said. "It is actually a miracle drug. There are immediate effects for people who are able to take it. It is the greatest thing to happen in the history of the illness. It's a great day. It's an emotional day."

The announcement was also welcomed by Pamela O'Connor from Hollyhill. She and her brother Jason both have CF. She said the provision of the new drug would help prolong both their lives. "Today my son, Scott is celebrating his 10th birthday - he doesn't have CF but after today's announcement I can imagine being around to see him when he's 20 or maybe when he's 30th so thanks very much, it's great news."

According to a Department of Health statement, Kalydeco was assessed by the National Centre for Pharmacoeconomics (NCPE) in January 2013 and found to have significant benefits for those with CF patients with the G551D mutataion which accounts for about 10 per cent of Irish sufferers.

Among the benefits acknowledged were significant improvements in lung function, increased body weight, improvement in patients' quality of life and a 55 per cent reduction in pulmonary exacerbations, the department said.

The assessment 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 and the NCPE recommended against reimbursing the drug at the submitted price.

However, the NCPE suggested a risk-sharing scheme, alongside a price reduction, could enable patients to gain access to Kalydeco. This led to talks between the HSE Corporate Pharmaceutical Unit and the drug manufacturers Vertex Pharmaceuticals, resulting in a system where the drug could be made available to patients whilst at the same time reducing  the financial impact to the State. The HSE Drugs Group then made a positive recommendation to support the provision of the drug.

The Irish Times understands that Mr O’Brien - on foot of the HSE Drugs Group positive recommendation - then contacted Dr Reilly for a policy directive on the new drug leading to today’s announcement.