“Life-changing” drugs to treat cystic fibrosis will routinely be available on the NHS after an agreement was made on their price.
The National Institute for Health and Care Excellence (Nice) said last November that the treatments were clinically effective but were too expensive at the list price for it to recommend them.
But in a development on Thursday, the pharmaceutical firm Vertex announced it had reached a long-term reimbursement agreement with NHS England over Orkambi, Symkevi and Kaftrio, enabling them to be approved on the NHS.
Nice was also able to use a new method to evaluate the drugs which means it can give extra weight to health benefits from medicines for treating the most severe diseases such as cystic fibrosis.
Vertex, NHS England and Nice have also committed to work together on rapid access for all eligible patients for the new triple therapy vanzacaftor/tezacaftor/deutivacaftor, which is currently being evaluated by medicines regulators.
Vertex said it was working with the NHS in Scotland, Wales and Northern Ireland to finalise similar access agreements as soon as possible.
The Cystic Fibrosis Trust said the move would give people access to the “life-changing” drugs and followed many years of campaigning by people affected by the condition.
David Ramsden, chief executive of the charity, said: “This is a fantastic moment for many people with cystic fibrosis and their families – ending uncertainty and helping to ensure that everyone who can benefit is able to access these vital medicines, now and in the future.
“We should not forget though that these treatments are not a cure and simply don’t work for some people.
“With the support of our incredible community, clinicians and researchers a lot has been achieved but we know there is still lots more to do.”
Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system.
It can cause serious lung infections and people can often have problems digesting food.
Symptoms usually start in early childhood and the condition gets worse over time.
There is no cure for the condition.
Nice has published a full list of who is eligible for the drugs, which can be given in childhood.
Around 8,200 people are already on the treatments in England and will continue to be able to get them on the NHS.
The drugs will also be available to people who are diagnosed with cystic fibrosis in the future.
More than 11,000 people in the UK have the condition.
John Stewart, NHS England director of specialised commissioning, said: “We know this is the news that people living with cystic fibrosis and their families have been waiting for.
“Thanks to this NHS deal thousands of patients including young children will be able to have a much better quality of life, whether that is being able to walk to school or ride a bike for the first time.
“The NHS has a strong track record of offering people with cystic fibrosis life-changing treatments with 8,000 people now benefiting – and today’s deal give families security knowing that they will be guaranteed access to these drugs for years to come.”
