The cystic fibrosis drug Kalydeco is to be made available on the NHS despite costing about £200,000 a year, it was announced yesterday.
The drug, which has been shown to transform the lives of patients affected by a genetic variation of cystic fibrosis, was approved by an advisory panel.
There had been concerns that Kalydeco, which is already available in the US, would be blocked here because of the expense. The cost of making it available to the 270 patients in England who are eligible will account for about half of the NHS budget for cystic fibrosis.
Cystic fibrosis is one of Britain’s most common life-threatening inherited diseases, affecting more than 9,000 people. There is no cure. It is caused by a faulty gene that controls the movement of salt and water in and out of the body’s cells.
The disease affects the internal organs, particularly the lungs and digestive system, by clogging them with a thick, sticky mucus. This makes it difficult to breathe and digest food.
Until the 1960s people with cystic fibrosis rarely survived childhood. Advances in treatment have extended life expectancy and a child born with cystic fibrosis today is likely to live until their late 40s, although the median age of death is in the mid-20s. Daily treatment is intense and the disease imposes severe limitations on how patients live.
Trials of Kalydeco, also known by the generic name ivacaftor, have shown that within days patients with the genetic mutation known as G551D can experience dramatic improvements.
Ed Owen, Chief Executive at the Cystic Fibrosis Trust, said: “Today’s decision is a fantastic early Christmas present for those affected by the G551D mutation. But it’s good news for the whole of the CF community too, as it provides a strong signal that the NHS is willing to fund future transformational therapies — at a time when the future pipeline in drug development is looking so promising.”
The Kalydeco review is seen as a test of the ability of the NHS to accept a new generation of personalised medication, made possible by the genetics revolution. These new and highly effective drugs are much more expensive because they will only ever be used to treat small numbers of patients.