WebMD News Archive
Special funding for cystic fibrosis drug in Scotland
18th January 2013 - Ivacaftor (Kalydeco) is the first medicine to act on one of the genetic mutations that causes cystic fibrosis.
The Scottish Medicines Consortium, which appraises all newly licensed medicines, has refused to recommend the drug saying it isn't value for money'. However, the Scottish Government has stepped in to make sure it is available from March for all patients with the G551D, or Celtic Gene, mutation by announcing a new Rare Conditions Medicines Fund.
The fund will be available from March this year through to April 2014.
Health Secretary Alex Neil says: "This fund bridges the period to the establishment of next year’s value based pricing for medicines and any changes that are made following the completion of the ongoing access to new medicines review."
Cystic fibrosis (CF) is one of the UK's most common life-threatening inherited diseases affecting more than 9,000 people. It's caused by a faulty gene that disrupts the movement of salt and water in and out of the cells within the body. As a result, the internal organs, especially the lungs and digestive system, become clogged with thick sticky mucus resulting in chronic infections and inflammation in the lungs and difficulty digesting food.
Ivacaftor (Kalydeco) helps thin the mucus so it can hydrate and protect the airways and keeps them from getting clogged and then infected. It is effective for the four percent of patients who have the G551D mutation.
It is given orally as a tablet once every 12 hours and patients who have taken it reported a significant improvement in their respiratory symptoms and quality of life.
Rest of UK
The NHS in England decided to provide funding for Ivacaftor (Kalydeco) from January 1st this year for patients who are six and over and who have at least one copy of the G551D mutation.
Decisions are yet to be made in Wales and Northern Ireland.