Cystic fibrosis patients say they are hopeful powerful new drugs to treat all mutations of the disease may be developed within a few years.
A delegation of CF patients visited Leinster House on Wednesday to thank politicians who supported their successful campaign for the approval of two life-saving drugs.
The HSE and Vertex Pharmaceuticals this week reached agreement in principle on the supply of the drugs, Orkambi and Kalydeco, to 600 patients.
Jillian McNulty, who organised two protests by CF patients outside the Dáil in recent months, said the deal came “just in time” for many fellow patients whose condition was worsening.
“My hope now is that their lives will be transformed, just as mine has since I started receiving treatment.”
Ms McNulty, from Co Longford, said that since she began taking Orkambi on a clinical trial four years ago, she has spent just 12 weeks in hospital. In the preceding years, she was hospitalised for up to nine months a year with respiratory problems caused by her condition.
Although a final agreement has not been signed, Ms McNulty said she was confident the agreement was effectively “over the line” and that treatments would become available from next month.
Orkambi and Kalydeco are effective for 70 per cent of CF patients but Ms McNulty said further drugs to treat other, rarer mutations of the disease could be developed within a few years.
Leading role
Cystic Fibrosis Ireland praised Government and Opposition politicians for their support but it was Fianna Fáil TDs who were most prominent in accepting thanks on the Dáil plinth.
Party leader Micheál Martin said the State urgently needed to review its framework for approving drugs for rare diseases and should take a leading role in encouraging research into new treatments.
Asked whether he had any concerns over the price of the deal, which has not been disclosed, Mr Martin said the State may be getting a better deal from Vertex than other countries. The fact the agreement covered a pipeline of present and future drugs was welcome, he added.
