Thousands of cystic fibrosis patients will finally get access to ‘life-changing’ drugs from the NHS

Thousands of people with cystic fibrosis have finally been guaranteed ‘life-changing’ drugs from the NHS, in what has been hailed as a ‘monumental day’ by families.

British regulators have reached an agreement with drugmaker Vertex on three ‘miracle drugs’ – Orkambi, Symkevi and Kaftrio, which could extend lives by decades.

It follows years of campaigning by the Cystic Fibrosis Trust, which described the deal as a ‘really important moment’ for thousands of families affected by the deadly condition.

The treatments will help many live a ‘normal life’, halting serious illness and reducing the need for grueling daily physiotherapy.

David Ramsden, chief executive of the charity, said: ‘This is a very important day and there are many emotional families out there today.

‘With a disease as brutal as cystic fibrosis you want access to the best treatments available and we are now in a position in England where that now seems certain.’

His parents Alex and Ben, both 31, described crying with relief at the news that he will be eligible for the triple therapy, Kaftrio, from next month.

Kaftrio is one of three drugs recently approved by the NHS for use in people with cystic fibrosis

Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system. Around 11,000 people in Britain are affected, with around 1 in 25 carrying the faulty gene.

It is a progressive disease. Symptoms usually begin in early childhood and there is no cure. Average life expectancy is still in the mid-30s, but it is hoped that these treatments will extend this.

Known as ‘modulator’ drugs, these are the first to treat the cause of the disease, rather than simply treating the symptoms, with sustained results suggesting they can extend life by decades.

Patients were given access to the treatments during an interim period during which further data was collected on their effectiveness.

But at a cost of around £100,000 per patient per year, NICE said in November that while they were clinically effective, they were too expensive to recommend at list price.

There were concerns that new patients, including young children, would miss out on the drugs if they were eligible.

Details of the commercial agreement remain confidential, although it will likely be below list price.

Nice was also able to use a new method to evaluate the drugs, allowing it to give extra weight to the health benefits of drugs for the treatment of the most serious diseases such as cystic fibrosis.

The decision is effective in about 90 percent of people with the disease and will end the concerns of those already on medication and the families of young children who are about to become eligible.

Mr Ramsden added: ‘We have already seen these medicines make such a difference to people’s lives.

‘There has been a big spike in the number of women with cystic fibrosis having children – an unexpected baby boom – which is really nice to see. These medicines make whole new life moments possible that people can benefit from.

‘But it’s important to say that this is just the beginning for patients with cystic fibrosis. However, we must remember that these treatments are not a cure and simply do not work for some people.”

Around 8,200 people are already receiving treatments in England and will continue to be able to receive them through the NHS.

Rufus was diagnosed with cystic fibrosis when he was just a few days old, a genetic condition that causes sticky mucus to build up in the lungs and digestive system

Such is the severity of his condition that the family from West to East Yorkshire can benefit from Bridlington’s sea air

The drugs will also be available in the future for people who have been diagnosed with cystic fibrosis.

Negotiations with healthcare services in Scotland, Wales and Northern Ireland are expected to be completed in the coming days.

John Stewart, director of specialist commissioning at NHS England, said: ‘We know this is the news people 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 enjoy a much better quality of life, whether that is by walking or cycling to school for the first time.

‘The NHS has a strong track record of providing life-changing treatments to people with cystic fibrosis, now benefiting 8,000 people – and today’s deal gives families peace of mind knowing they will have guaranteed access to these medicines for years to come. ‘

One of those who will benefit from this is Rufus, who, like any child about to turn two, does not like to sit still.

But a simple cough or cold could mean he can’t play or go to toddler groups for weeks.

He has to undergo grueling physical therapy three or four times a day when he’s under the weather, with little energy for anything else.

The diagnosis was made when he was just a few days old. His parents, Alex and Ben, both 31, described crying with relief at the news that he will be eligible for the triple therapy, Kaftrio, from next month.

They say the drug – which significantly improves lung function and helps people with cystic fibrosis breathe more easily – will give him a normal life with his older brother Henry, four years old.

“It was a huge concern, especially for Rufus’s future,” says Ben, who works in social care.

Two of the other drugs covered by the new agreement are Orkambi and Symkevi, which could extend the lives of people with cystic fibrosis by decades.

“It was bittersweet to hear from other families just a little older than him and how helpful it is.

“We were in the dark, thinking this could be a really good thing for him, but when we had that horrible realization, maybe he didn’t get it.”

Such is the severity of his condition that the family from West to East Yorkshire can benefit from the sea air of Bridlington.

They have now put him on a waiting list for nursery and hope the treatment will allow him to have a full and normal childhood.

Full-time mum Alex said: ‘For Rufus this means he will be just like any other child – and that’s all we want him to be. We just need to have the same opportunities as everyone else and this should give him that.

‘When he is well he is just like any other child, but when he is ill he has no life.

‘He cannot go to toddler groups or play because he is not well enough and is very lethargic. With this medicine he can go to daycare and live like any other toddler.

‘What this means is that if he gets sick, he won’t be that sick – he won’t make it to the level. He will be like any other child who has a cold or a virus.”