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£200,000 cystic fibrosis drug ‘could transform lives’

From the Telegraph 10:25AM BST 30 Jul 2012 (No Reporters name)

A drug which could transform the lives of people with cystic fibrosis has been   developed, as the health watchdog investigates whether it can be provided on   the NHS at an annual cost of £200,000

Trials of the drug Ivacaftor have shown improvement in patients’ breathing and weight gain, with use reducing their need for antibiotics.

It has been shown to help some of those who suffer from cystic fibrosis, and has been hailed by one trial co-ordinator as “remarkable”.

It is now being studied by the National Institute for Clinical Excellence (NICE) to determine whether it is value for money, at a cost of £200,000 per year.

It has already been approved for use in patients over six years of age in the United States, and by EU watchdogs.

It is expected to become available in France, Germany and the Irish Republic shortly, according to the Times newspaper

Cystic fibrosis currently affects more than 9,000 people in Britain and is   incurable, causing internal organs to be “clogged up” with a sticky mucus.

Caused by a faulty gene, it places severe limitations on sufferers and   significantly shortens life expectancy.

Ivacaftor works by targeting a particular mutation, G551D, which is present in   around 600 people with cystic fibrosis in Britain – around six per cent.

Stuart Elborn, lead coordinator of the trials from Queen’s University, Belfast, told the newspaper: “When the first slide went up, we were speechless.

“The hair was standing on the back of my neck.

“It was the moment the drug went from being one that might not even work to   one that will transform thousands of lives.”

According to the Times, the £200,000 a year cost would use more than half of   the £110m UK budget currently used on cystic fibrosis.

Vertex, the company which spent 13 years developing and making the drug, says   the cost will be offset against money spent on patients’ spending time in   hospital or taking time off work.

A spokesman said they were “working with the health authorities to make it   available as quickly as possible”.



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