NHS patients among first in Europe to benefit from landmark deal for cystic fibrosis treatment

NHS patients in England will be some of the first people in Europe to benefit from a new “truly life-transforming” treatment for cystic fibrosis, health service chief executive Sir Simon Stevens has announced today.

Three in five patients with the deadly genetic condition could benefit from the ‘triple combination’ treatment produced by Vertex Pharmaceuticals.

This means nine out of 10 patients with cystic fibrosis – more than 7,000 people – will now benefit from a therapy which tackles the underlying causes of the disease, by helping the lungs work effectively.

The landmark deal, negotiated ahead of a licence being granted by the European Medicines Agency, means that clinicians will be free to start prescribing the triple treatment to English patients as soon as the licence is finally granted, in the coming weeks.

The terms of the deal also mean that as many as 300 patients with rare mutations, which fall outside of the scope of the European licensing body’s current considerations, will also be able to benefit from the range of treatments produced by Vertex Pharmaceuticals, offering a lifeline to those with limited treatment options.

In negotiating the deal, NHS England has also ensured that Vertex will offer equivalent terms for cystic fibrosis patients in Wales, Northern Ireland and Scotland should those devolved administrations wish to adopt them.

The deal is welcome news for patients with cystic fibrosis who have been advised to shield at home during the COVID-19 pandemic.

NHS England recently announced that thousands of people with the condition will receive devices allowing medics to monitor their condition remotely, as part of the NHS drive to give more people connected, supported and personalised care in their own homes, saving them from having to make trips to hospital.

The news of the breakthrough deal comes in the week of the NHS’ anniversary, which will be marked by events across the country, ahead of the service’s ‘birthday’ on Sunday July 5.

Announcing the agreement, NHS chief executive 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.”

The deal – supported by the National Institute for Health & Care Excellence (NICE), the internationally renowned body for assessing the clinical and cost effectiveness of new treatments and technologies – will last for four years and allow for further data to be collected to better understand the impact for patients of this game-changing 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.

Chief Executive of the Cystic Fibrosis Trust, David Ramsden, said: “It is fantastic news that a deal has been done between NHS England and Vertex Pharmaceuticals and Kaftrio will now be available to thousands of people across England in the coming weeks. This will truly save lives.

“This is a great day, but we know that there is more to do and we will not stop until everyone with cystic fibrosis across the UK has access to life saving drugs.”

Health Secretary, Matt Hancock, said: “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.

“In October we reached a fair deal with Vertex to give patients Orkambi at a price we could afford, transforming the lives of hundreds.

“I’m absolutely thrilled we have now secured a deal for Kaftrio, a cutting-edge medicine which gives hope to families and patients for a longer, healthier and happier future.

“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.”

Ludovic Fenaux, Senior Vice President, Vertex International, said: “This is an important day for patients with CF in England. I am delighted that eligible patients will be able to benefit from access to triple combination regime in the next few months, should the licence be granted by the European Commission. Establishing this agreement, in advance of the medicine being licensed, illustrates the collaboration between Vertex, NHS England and NICE. It shows just what is possible when the biotech industry and the NHS partner together for the benefit of patients.”

Investment in world-class, cutting edge technologies and treatments is at the heart of the NHS Long Term Plan, published last year.

The new triple combination treatment combines three drugs which perform different functions – ivacaftor, tezacaftor and elexacaftor.

The treatment will be a further boost to NHS patients with cystic fibrosis, who already have full access to drugs Orkambi, Symkevi and Kalydeco, also manufactured by Vertex Pharmaceuticals, following a similar agreement negotiated by NHS England at the end of 2019.

As the European licence for the triple combination will only cover patients 12 years and older at this stage, younger children will continue to benefit from the previous deal struck by NHS England giving them continued access to Orkambi, Symkevi and Kalydeco, where clinically appropriate, with access to the triple therapy to follow automatically, if the European regulators approve the move.

Commercial terms of the agreement are confidential, but the approach achieved is consistent with NHS England’s other managed access agreements reached with the pharmaceutical industry.

The drug has been described by the UK Cystic Fibrosis Medical Association as showing evidence of being potentially ‘truly life-transforming.’

Today’s announcement is the test in a growing catalogue of commercial access deals benefiting thousands of patients that have been put in place over the past 18 months, following NHS England’s establishment of a new commercial medicines function led by Blake Dark.