Life-extending cystic fibrosis drugs coming to England

Life-changing cystic fibrosis drugs will be available on the NHS in England, health bosses say.

NHS England struck a deal with US company Vertex Pharmaceuticals today to make all three of their UK-licensed cystic fibrosis medicines, Orkambi, Symkevi and Kalydeco, available to patients in England on the NHS.

Cystic fibrosis is a genetic condition that causes sticky mucus to build up in the lungs and digestive system, causing lung infections and problems with digestion. The UK has the second-highest prevalence of cystic fibrosis of any country in the world, with only around half of those with the condition live to the age of 40.

A spokesman for the Department of Health said the drugs, which improve lung function and reduces breathing difficulties, will be available to patients within 30 days.

The treatments will not work for all patients with cystic fibrosis, only those with certain mutations, but health bosses estimate that half of the 10,000 patients in the UK with the condition will benefit from the drugs, the spokesman said.

Vertex originally wanted to charge £100,000 per patient per year for the treatment, but The National Institute for Health and Care Excellence (Nice), which helps decide which treatments should be available on the NHS in England and Wales, rejected the drug on cost grounds.

A compromise has since been reached in a confidential deal that health bosses say will be good value for British taxpayers and will include future access to Vertex’s new triple therapy Trikafta, which 90 % of Cystic Fibrosis sufferers could stand to benefit from.

NICE has backed the agreement under the condition that Vertex will submit its full portfolio, including the forthcoming triple therapy Trikafta, for comprehensive appraisal. Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said:

‘We are pleased an agreement has been reached between NHS England and Vertex that brings these medicines to patients. It is also welcome that the company will now re-engage with the NICE process.

‘We look forward to working with the company and patient organisations to get ready for the appraisals, including developing the approach to the collection of evidence.’

David Ramsden, chief executive of the Cystic Fibrosis Trust, welcomed the news, saying the charity would continue campaigning for people in Wales and Northern Ireland to have access to the drugs. He said:

‘This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting.

‘We know it doesn’t end here – we will continue to fight for similar access in Wales and Northern Ireland so that all people with cystic fibrosis in the UK are able to benefit from these life-saving drugs.

‘Also, with news that the new triple therapy, Trikafta, which 90% of people with cystic fibrosis could stand to benefit from, could be licenced for use in the coming year, we will continue to campaign so that people with cystic fibrosis never have to wait again for the best available medicines.’

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