NHS strikes deal with Vertex for transformative cystic fibrosis drugs

After years of negotiations, NHS England has signed an agreement with Vertex Pharmaceuticals to make the company’s suite of cystic fibrosis medicines available to patients in England.

The deal means that NHS patients will have access to Vertex’s current and future cystic fibrosis portfolio with clinicians being able to start prescribing drugs Orkambi, Symkevi and Kalydeco within 30 days.

The UK has the second highest rate of patients with cystic fibrosis in the world and the deal stands to benefit some 5,000 people who may now take up the treatments. With no cap on patient eligibility though, this number increase for any person who may benefit from any of the drugs.

Orkambi in particular was hailed as a transformative medicine when it was approved by the FDA back in 2015. A precision medicine, Orkambi is able to target the underlying cause of cystic fibrosis in patients aged two years or older. The drug works by treating the F508del mutation, which around 50% of people with cystic fibrosis in the UK have. 

Announcing the agreement, NHS chief executive Simon Stevens said: “The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis. That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS. I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.”

Confidential commercial terms were agreed between both parties with Vertex agreeing to submit the three drugs for NICE appraisal. This includes its new triple therapy Trikafta, which could benefit up to 90% of patients with cystic fibrosis and which has recently been approved by the FDA.

Health Secretary Matt Hancock said: “Some wonderful news this morning. We’ve agreed a deal to provide Orkambi and other lifesaving Cystic Fibrosis drugs on the NHS. The deal is great value for money for the NHS, and crucially, will improve thousands of lives. This deal – on the back of several others this summer – shows why we get some of the best value drugs in the world, and is another reason to be so proud of our NHS.”

“Today is a significant day for the cystic fibrosis community in England. This important agreement, reached in collaboration and partnership with NHS England and NICE, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease,” commented Ludovic Fenaux, senior vice president, Vertex International.

David Ramsden, chief executive of the Cystic Fibrosis Trust, added: “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.”