Terrified parents fear their toddlers will never get ‘miracle’ cystic fibrosis drug, with NHS watchdogs poised to cut-off supply due to its £200,000 price-tag

Parents of toddlers battling cystic fibrosis have today told how they fear their kids will be denied a ‘miracle’ drug on the NHS, with one mother terrified that it may be cruelly ‘snatched away’ from her 19-month-old daughter.

Kaftrio was this week approved for all children aged two and above, giving hope to hundreds of families affected by the incurable disorder. 

Studies have shown it can extend the life-expectancy of sufferers by 30 years.

Yet, despite its life-changing benefits, NHS watchdogs are poised to reject the drug for widespread use due to its cost. A year’s supply is around £200,000, the National Institute for Health and Care Excellence (NICE) says.

NICE will announce its final decision in March, MailOnline understands. 

If Kaftrio isn’t recommended, as expected, only children already taking the drug will definitely be allowed to continue. 

It raises the prospect that kids who don’t turn two until after NICE’s decision will be denied the opportunity, unless manufacturer Vertex Pharmaceuticals agrees to sell supplies to the NHS at a fraction of its current list price.

Acknowledging the news ‘may be difficult for some families’, the Cystic Fibrosis Trust said: ‘We don’t know exactly what will happen.’

Until this week, the Medicines and Healthcare products Regulatory Agency (MHRA) had only given Kaftrio the green light for kids over six.

Although many parents are overjoyed their cystic fibrosis-affected children will now have access to Kaftrio, others are terrified their kids will miss out because of the two-tier system.

Laura Beck and husband Daniel, parents to Florence, 19 months, are ‘scared’ NICE’s outcome will not go in her favour.

Florence turns two in April 2024, just one month after NICE is expected to reveal its final decision. In draft guidance last week, it claimed Kaftrio’s cost-benefit ratio was above what was considered ‘an acceptable use of NHS resources’.

Discussing Kaftrio’s sudden approval for kids aged between two and five, Mrs Beck, from Portsmouth, said: ‘It is bittersweet.

‘When I heard the news yesterday, I was in M&S and I nearly cried, because it was that amazing. 

‘I am grateful for all those children between two and five that can now access it.’

‘However, Florence is so close to being two, but also so close it could be snatched away from her.’

Although Florence appears healthy, Mrs Beck fears she could become unwell in an instant. 

After quitting her job to care for Florence, Mrs Beck says Kaftrio could enable her to go to nursery without getting sick. 

She said: ‘Everyone I know that’s got a child in the two to five age bracket have been speaking to their doctors already, to find out the next steps and to start taking it as soon as it’s ready.’

Angry at the high prices set by the pharmaceutical company, Mrs Beck believes it’s ‘ridiculous’ that access ‘all comes down to money’.

Although its list price is in the region of £200,000, NHS agreed a cut-price deal with Vertex Pharmaceuticals to make it available in 2020. 

Yet studies suggest manufacturing a year’s supply costs close to £5,000. 

Drug firms mark-up the prices of drugs to claw back the millions of pounds spent on researching breakthroughs.

Aimee Sawyer was always told that cystic fibrosis was a completely different illness to what it was 10 years ago because of new life-saving drugs available. Kaftrio is one of four game-changing medicines, along with Kalydeco, Orkambi and Symkevi. 

But she fears her 21-month-old daughter Skye may never be able to take Kaftrio if the supply is suddenly cut off, even though she will be two when NICE’s decision is set to be announced.

Mrs Sawyer told MailOnline her daughter has a cough, is constantly on antibiotics and does physio twice a day, just to stay well, but Kaftrio could change this.  

The mother-of-three, from Nottingham, wasn’t aware that the drug was even under review and was shocked to find out it could be take away because of costs. 

‘At first, we were devastated and now we are angry,’ she said. ‘What about the offset of the cost if they don’t have the drug?

‘We think about what the cost would be [but] if she doesn’t get this drug, she will have a shorter life. She may not ever be able to work to contribute to society. 

‘If she doesn’t have this drug, she will need more hospital visits, more antibiotics.’

Mrs Sawyer added: ‘My first thought is, it’s so unfair. But I feel selfish feeling like that. Because, actually, I’m so happy that those families are seeing the benefits of these drugs.’

Kaftrio — made-up of ivacaftor, tezacaftor and elexacaftor — is only currently available on the NHS through a temporary deal with London-based manufacturer Vertex Pharmaceuticals. 

Despite being given Kaftrio slightly cheaper than its listed price-tag, NICE last week announced the triple-combination therapy and Symkevi and Orkambi weren’t ‘cost-effective’. 

Draft NICE guidance revealed its cost-benefit ratio was above what was considered ‘an acceptable use of NHS resources’. 

Up to 600 children could benefit from the MHRA’s move to approve Kaftrio for kids aged between two and five, it is estimated. 

And for many, the news that children under six could now take Kaftrio has been life-changing. 

Sarah Pinnington, who is now in ‘touching distance’ of her four-year-old Lucy getting Kaftrio, told MailOnline: ‘When I heard the news I literally sat down on the floor and burst into tears.’

The mother-of-two, who lives in Bridge, Kent, with husband Richard and five-year-old son Frankie, said Kaftrio is ‘perfect’ for her because of her genetic mutations. 

Kaftrio is only eligible for patients with a specific quirk in their DNA, which makes it difficult for cells to move water and salt in and out of cells.

As a result, the body produces thick, sticky mucus that clogs the digestive system, creating problems with food absorption, and the lungs, which results in infections and scarring that gradually reduces patients’ ability to breathe.

This slow suffocation is often what ultimately kills.

Although pleased her daughter will be able to take Kaftrio, she worries about kids who are under two and the prospect of a ‘two tier system’.

She told MailOnline: ‘It is horrific that because of the year in which you were born, you will have access to medication that will completely change your life, your life opportunities, your life expectancy.

‘I understand that there is not a bottomless pit of money in the NHS, and there are very big demands everywhere, but we’re talking about future generations of children who have a genetic condition that they’ve inherited.’

Amy Benson, from Plymouth, is now confident her four-year-old son Willem will have Kaftrio at Christmas.

Willem has just started reception. Every day starts at 6am, so he can get through all of his treatments and medication before school.

People not on the drug may have to take a cocktail of up to 40 drugs a day. 

With a little girl on the way, Mrs Benson and husband Dan were counting on Kaftrio to be made available.

Speaking to MailOnline ahead of the decision to licence Kaftrio for younger kids, Mrs Benson said it felt like they had ‘put a price on his head’.

Now he can get the drug, she doesn’t want to stop campaigning until everyone with cystic fibrosis has ‘equal access’.

‘Any child who hasn’t reached two by that point, or hasn’t even yet been born, hasn’t even yet been planned, they’re not going to benefit, and to not make it accessible makes absolutely no sense,’ said Mrs Benson.

Megan Verney, of Devon, is ‘angry at the pharmaceutical company’ for their ‘greed’ and setting the prices so high.

‘People were literally dying, and then now they’re able to completely turn their life around,’ she said. 

‘So, in my eyes, it’s a miracle drug and It’s the closest thing to a cure. I can’t accept that it’s not going to be an option.’

Her son Charlie, aged three, is not eligible for any of the other modulators and the news he will now be able to take Kaftrio has given the family ‘hope’ again. 

For Mrs Verney and husband Edward, Kaftrio has meant that although Charlie could be on medication for the rest of his life, ‘things can be positive for him’.

She said: ‘I don’t think we will truly be excited until the drug is in our hands and our children’s bodies are responding well.’

Due to the ‘uncertainty’ surrounding NICE’s decision, Mrs Verney admits to feeling ‘nervous’ to start Charlie on Kaftrio, but is still feeling ‘more positive for his future now’.

However, the mother-of two wants to ‘keep up the momentum’ ‘and keep applying pressure’ to make sure children under two also get access. 

For Fiona Stewart and husband Calum, the news their two-year-old daughter Zara would be able to take Kaftrio left them ‘completely in shock’ and ‘happy’.

The family, who lives in Glasgow, have been holding onto the hope the drug would ‘transform her life’.

‘As soon as you find out there is a drug out there that is life changing for your child you just want to get them on it as soon as possible’, Mrs Stewart told MailOnline.  

However, she said it’s ‘heartbreaking’ to ‘think about all the other children that could be left behind’.

Scotland is expected to follow the NICE advice at the end of this review and Mrs Stewart believes not providing children the drug will also cost the NHS a lot of money.

She said: ‘I get that it’s an expensive drug, but I think what needs to be thought of is the expense if a child’s not on the drug. They’re likely to have more intensive care admissions and it’s all the things that come with that, like their blood tests, your scans, all the money that adds up over time, and it’s just distressing for these children.’ 

Cystic fibrosis — which affects 11,000 people in the UK — is usually detected at birth using a newborn screening heel prick test.

Treatment advances over the past four decades have boosted life expectancy – as recently as the 1970s, few people with cystic fibrosis lived beyond their teens.

Kaftrio corrects the underlying problem by helping to create thinner mucus, thereby relieving symptoms.

Experts are hopeful the triple therapy — which combines ivacaftor, tezacaftor and elexacaftor — will add decades to the lives of people with cystic fibrosis.