Thousands of people with cystic fibrosis are finally assured ‘lifechanging’ drugs on the NHS in what has been hailed a ‘monumental day’ by families.
UK regulators reached a deal with drug-makers Vertex over three ‘miracle’ drugs – Orkambi, Symkevi and Kaftrio, which can extend lives by decades.
It follows years of campaigning by the Cystic Fibrosis Trust, which described the agreement as a ‘really important moment’ for thousands of families blighted by the deadly condition.
The treatments will help many to ‘live normal lives’, potentially stopping bouts of severe illness and reducing the need for gruelling daily physio.
David Ramsden, chief executive of the charity, said: ‘This is a really significant day and there are a lot of emotional families today.
‘In a disease as remorseless as cystic fibrosis, you want access to the best treatments available and we now in a position in England where that now seems certain.’
One of those set to benefit is Rufus who, like any child about to turn two, doesn’t like to sit still
His parents Alex and Ben, both 31, described weeping with relief at the news he will now be eligible for the triple therapy, Kaftrio, from next month
Kaftrio is one of trio of drugs recently approved to be given to people with cystic fibrosis on the NHS
Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system, affecting around 11,000 people in the UK with around 1 in 25 carrying the faulty gene.
A progressive disease, symptoms usually start in early childhood and there is no cure. The average life expectancy is still the mid-30s but it is hoped these treatments will extend this.
Known as ‘modulator’ drugs, these are the first to treat the cause of the disease, rather than simply treat the symptoms with ongoing results suggesting they can extend life by decades.
Patients have been able to access the treatments during an interim period in which further data was being collated on their effectiveness.
But at a cost of around £100,000 per patient, per year, NICE said in November that although clinically effective, they were too expensive to recommend at list price.
There were fears that new patients including young children would miss out on the medicines when they became eligible.
Details of the commercial agreement remain confidential, although it is likely to be below list price.
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.
Effective in around 90 per cent of people with the disease, the decision will ends anxieties for those already on the drugs and the families of young children about to become eligible.
Mr Ramsden added: ‘We’ve seen these drugs make such a difference to people’s lives already.
‘There’s been a big spike in women with cystic fibrosis having babies – an unexpected baby boom – which has been a really lovely thing to see. These drugs allow whole new life moments that people are able to take advantage of.
‘But it’s important to say this is still just the start for cystic fibrosis patients. We should not forget though that these treatments are not a cure and simply don’t work for some people.’
Around 8,200 people are already on the treatments in England and will continue to be able to get them on the NHS.
Rufus was diagnosed with cystic fibrosis when he was just days old, a genetic condition which causes sticky mucus to build up in the lungs and digestive system
Such is the severity of his condition, the family from West to East Yorkshire so he could benefit from the sea air of Bridlington
The drugs will also be available to people who are diagnosed with cystic fibrosis in the future.
Negotiations are expected to be finalised with health services in Scotland, Wales and Northern Ireland in the coming days.
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.’
One of those set to benefit is Rufus who, like any child about to turn two, doesn’t like to sit still.
But a simple cough or cold can mean he’s out of action for weeks at a time, unable to play or go to toddler groups.
He must endure gruelling physio three or four times a day when poorly, with little energy for anything else.
Diagnosed when just days old, his parents Alex and Ben, both 31, described weeping with relief at the news he will now be eligible for the triple therapy, Kaftrio, from next month.
They say the drug – which significantly improves lung function, helping people with cystic fibrosis to breathe more easily – will give him a normal life with his older brother Henry, four.
‘It’s been a massive worry – especially for Rufus’s future,’ said Ben, who works in social care.
Two of the other drugs impacted by the new agreement are Orkambi and Symkevi which can which can extend lives of people with cystic fibrosis by decades
‘It’s been bittersweet hearing from other families that are just a bit older than him, hearing just how beneficial it is.
‘We’ve been in limbo thinking this could be really good for him but then having that awful realisation he might not get it.’
Such is the severity of his condition, the family from West to East Yorkshire so he could benefit from the sea air of Bridlington.
They have now put him on a waiting list for nursery and hope the treatment means he can live a full and normal childhood.
Full-time mum Alex said: ‘For Rufus, it means he will be like any other child – which is all we want him to be. We just to have the same chances as anybody else and this should give him that.
‘When he’s okay, he’s like any other child but when he’s ill, he doesn’t have a life.
‘He can’t go out to toddler groups or play because he’s not well enough and is really lethargic. With this medicine, he can go to nursery and he can live like any other toddler.
‘What this means is when he does get ill, he won’t be as ill – he won’t get to the level. He’ll just be like any other child with that gets a cold or virus.’
Source: Mail Online
