Cystic fibrosis: New drug Kalydeco 's go-ahead for Welsh NHS

http://www.bbc.co.uk/news/uk-wales-22484992

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A drug that could transform the lives of some cystic fibrosis sufferers will be available on the Welsh NHS after the health minister went against advisors.

It was feared Mark Drakeford would follow a recommendation not to approve Kalydeco because of its cost.

It would have set Welsh patients apart from those in the rest of the UK, where the drug is available.

The Cystic Fibrosis Trust had warned patients in Wales could have been faced with a second class health service.

The life-threatening genetic disorder damages the lungs and digestive system.

Kalydeco is said to be the first drug to treat the root cause of cystic fibrosis for people with the G551D mutation and could dramatically improve the life expectancy of sufferers.

About 4% of patients across the UK have this mutation, equating to around 13 patients in Wales who could benefit.

'Transformational medicine'

The All Wales Medicines Strategy Group (AWMSG), which advises Mr Drakeford, announced on Wednesday that it did not recommend the approval of the drug for use on the Welsh NHS.

At an estimated cost of up to £180,000 per patient per year, it said the drug, also known as Ivacaftor, was not cost-effective.

But the Cystic Fibrosis Trust said the medicine was "transformational", had a "dramatic effect" and that experts say "no other medicine addresses the basic genetic defect in cystic fibrosis".

It also said the development of future medicines to help patients with more common mutations of cystic fibrosis depended on the use of Kalydeco, also known as Ivacaftor.

Mr Drakeford - who made the final decision about the drug - said on Friday that he would make it available on the Welsh NHS.

He also said he was setting up a review to look into how such new, innovative medicines for rare diseases can be effectively evaluated in Wales.

"I will report back to the National Assembly on progress as it becomes available," he added.

'Common sense prevails'

The Cystic Fibrosis Trust said it was "delighted" by the decision.

"We look forward to this being made available to patients as quickly as possible, so that that those who could benefit will have access to the medicine," said chief executive Ed Owen.

"These are exciting times for research into new medicines for cystic fibrosis.

"We are encouraged by the minister's intention to review the appraisal process for new and orphan medicines. The trust will look to contribute to this review."

Peter Black, Welsh Liberal Democrat assembly member for south Wales west, had written to Mr Drakeford on behalf of a number of constituents to ask him to overrule the AWMSG recommendation.

He said he was pleased that "common sense had prevailed".