New cystic fibrosis drug approved
Published 12/03/2013 | 16:31
A new drug to treat a rare form of cystic fibrosis which could cost more than £5 million has been approved for use in Northern Ireland's health service, it was revealed.
Around 30-35 people may benefit from the Ivacaftor treatment for those with the Celtic gene, a more common mutation in Ireland. The drug carries a list price of £182,625 per patient per year, the region's Health and Social Care Board said.
Cystic fibrosis affects cells that secrete mucus in the lungs and those that produce digestive juices in the gut and pancreas, causing those secretions to thicken and creating long-term problems.
Professor Stuart Elborn from Queen's University Belfast led the original trials of the drug.
"This is a drug that really makes a huge impact on the patients suitable for it. It also gives hope to the whole cystic fibrosis community that the defect in cystic fibrosis is correctable with the right drug," he said.
"This is a transformative drug for these young people and I think the cost of drugs is a debate we have to have as a society rather than for individual people or individual drugs."
Professor Elborn's study found significant improvement in lung function, quality of life and a reduction in disease flare up for those receiving the new treatment.
In Scotland the drug was not approved for use on the NHS after a discount on the cost given to the NHS in England was not offered to its counterpart north of the border, leading to the Scottish Medicines Consortium recommending against its use largely on cost grounds. A special fund was established by the Scottish Government to pay for access instead.
In Northern Ireland, where the genetic mutation is slightly more common, the amount paid by the health service for the drug has not been disclosed. Around 500 people have cystic fibrosis in Northern Ireland, around 7% with the mutation targeted by this new treatment.
A spokesman for the Board said: "Commissioners here considered the available evidence on the efficacy and cost effectiveness of Ivacaftor. In particular commissioners took account of the work undertaken by the North of England Specialised Commissioning Group as the national commissioning lead for CF (cystic fibrosis)."