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01.07.20

NHS patients among first in Europe for new cystic fibrosis treatment

After a new landmark deal was struck, NHS patients in England will be some of the first people in Europe to benefit from a new “life-transforming” treatment for cystic fibrosis, the Chief Executive of the NHS Sir Simon Stevens has announced.

Three in five patients with the deadly genetic condition could benefit from the new ‘triple combination’ treatment, meaning nine out of 10 patients with cystic fibrosis – representing more than 7,000 people nationally – would now benefit from a therapy which tackles the underlying causes of the disease and helps the lungs work effectively.

The landmark deal, which has been negotiated ahead of a license being granted by the European Medicines Agency, means clinicians will be free to start prescribing the triple treatment to patients in England as soon as the license is granted in the coming weeks.

As many as 300 patients with rare mutations of the disease, which currently fall outside of the scope of the European licensing body’s current considerations, will also be able to benefit from a range of treatments as part of the terms of the deal.

This, it is hoped, will offer a lifeline to those with currently limited treatment options.

NHS England have also ensured as part of the deal equivalent terms for cystic fibrosis patients in Wales, Northern Ireland and Scotland would be offered should the respective devolved administrations wish to adopt them.

The deal, which is supported by NICE, will last four years and allow for further data to be collected to better understand the impact for patients of this new treatment.

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Three in five patients with the deadly genetic condition could benefit from the new ‘triple combination’ treatment
 

NICE will work with the company and the NHS on continued data collection for all three products involved in advance of the technology appraisal. Prices will then be adjusted as necessary, following full NICE appraisal, to ensure that taxpayers continue to get a good deal.

The new triple combination treatment combines three drugs which perform different functions – ivacaftor, tezacaftor and elexacaftor. It has been described by the UK Cystic Fibrosis Medical Association as showing evidence of being potentially “truly life-transforming”.

Announcing the agreement, Sir Simon Stevens said: “This is a potentially transformational treatment for cystic fibrosis, and I am proud that, thanks to this landmark deal, NHS patients will be among the first in Europe to benefit.

“Staff have pulled out all of the stops to provide specialist hospital care to 100,000 patients with COVID-19 and countless others with the virus, but they have also been working round the clock to help those needing urgent care or with serious long-term conditions such as cystic fibrosis.

“The triple therapy is the latest improvement in care which will help thousands of people with CF lead longer, healthier lives and shows once again that where drug companies are prepared to work flexibly with the NHS we can deliver life-changing medicines.”

Health Secretary Matt Hancock added: “Every family dreams of seeing their children reach their full potential and I am bowled over by the incredible bravery of families living with cystic fibrosis.

“I will always fight for families and loved ones to have access to the best possible treatments so that everyone has the opportunity to flourish.”

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