Parents whose sons are losing ability to walk face ‘nightmare' battle with NHS
The medicine givinostat can slow progress of Duchenne muscular dystrophy – keeping children on their feet for longer – and is being offered for free by a drug firm while it goes through the process of NHS approval.
However, only some NHS trusts across the UK, and few in England, are giving the drug despite it being part of an early access programme since November.
Families are now facing a 'cruel' postcode lottery over who gets the medicine and are in a race against time, according to the charity Duchenne UK.
Some NHS trusts are refusing to supply the drug while others delay it by drawing up lists of who they think should get it first.
NHS trusts say they need to pay for monitoring the drug – around £1,900 per year per patient – out of their own budget.
In desperation, parents have met with Health Secretary Wes Streeting to try and speed up access.
According to the Government, it is up to individual NHS trusts to decide whether to provide the drug.
Duchenne UK is supporting families in their fight for givinostat, including Rosie and Pete Day, an investment manager, and their 10-year-old son Jasper, from Horsham, West Sussex.
The Day family have twins Jasper and Arabella, aged 10, and two older daughters.
Jasper enjoys a range of hobbies (Family handout/PA)
Both Jasper and Arabella have Duchenne, with Arabella a carrier of the genetic disorder and Jasper the most affected, as the condition mostly affects boys.
The drug, developed by ITF Pharma UK, is only being given under the early access programme to children who are still able to walk.
Mrs Day told the PA news agency: 'We try and live in the moment and, in this moment, there's an opportunity to make a massive change to Jasper.
'We're not going to cure it, but this drug gives him the best chance of having the early life that he wants to have and what every parent wants to give their child.
'It's so close – and yet we're stuck.
'We're stuck in this process where there's something in reaching distance that could make a huge difference to keep him walking, keep him active, keep him with his siblings in the garden, keep him walking up the stairs, all of those things, and it just feels that we can't quite get there.
'We're on the finishing line to get something that will make a difference and we can't quite get there because of the system we're in.
'The drug is not going to cure it, but it will give him the childhood you would wish to give your child – to keep them playing football, coming on dog walks, being able to walk up the stairs, playing the French horn, all of those things that you dream your child is going to get to do.
'We can't give it because it we're stuck, even though it's free and it's there – we can't give it.
'It feels like a nightmare, because you know that the doctors want to give it, but when it comes to getting the drug in his mouth and seeing what it would do, we're stuck.
'It's a lottery, basically, depending on where you are in the country … and within NHS trusts themselves.'
Jasper is under the care of the Evelina London Children's Hospital, which is only now starting to contact patients and is drawing up a priority list for who gets the drug first.
'Every day that passes by is a day that we can see Jasper decline and see him struggling with his muscles,' Mrs Day said.
'Two weeks ago, he asked me, 'When will I get the drug, mummy?'
'We're stuck in a situation over who's going to make those decisions regarding who gets the drug – of whether Jasper gets it or another boy.
'For every boy it's heartbreaking, because someone ultimately is not going to get the drug at that moment based on the current criteria.'
She added: 'If Jasper stops walking, we're not going to get that back.
'Every day matters. Six months ago, Jasper could walk up and down the stairs easily. He can't do that now and Pete has to push him up the stairs every night.
'Yet we can't give him something that is as simple as two spoons full of medicine and a blood test, and a little bit of maybe resource looking at the blood test results – that is what is standing in our way.'
Mrs Day said it was unclear how the Evelina is 'going to make a fair choice' because knowing who will lose mobility is not an exact science.
Mr Day said the process is 'hugely subjective' because there is no linear decline in the condition and the decisions made by NHS trusts could end up being 'completely wrong'.
According to Duchene UK, boys whose condition is getting worse risk falling off the list entirely while waiting for the drug.
Around 500 boys in the UK are eligible for givinostat, which can be taken at home like Calpol, with hospitals then doing follow-up blood tests of around eight in the first year, followed by twice a year thereafter.
Children may also need an ECG, though these are already part of routine Duchenne monitoring.
Emily Reuben and Alex Johnson, founders of Duchenne UK, said: 'As time ticks by, more boys are losing out on their chance to access givinostat.
'This is a simple treatment, which can be easily managed at home, and requires uncomplicated blood tests to monitor. It's free to the NHS and could offer real hope for patients and their families.
'The delays are cruel and the postcode lottery is unjustifiable. We are calling on the NHS to urgently make this available to both ambulant (walking) and non-ambulant boys.'
To date, all health boards in Scotland are rolling out the drug, alongside those in Swansea and Cardiff.
Leicester Royal Infirmary was the first trust in England to give the drug, but the Evelina has not yet, and neither have trusts in Manchester, Liverpool and Newcastle.
Great Ormond Street Hospital is working to supply the drug.
A spokesman for the Evelina said: 'We have begun contacting families of all existing Evelina London patients who may be eligible for givinostat and are working to set up appointments in the next few months.
'Our clinical team are working through our patient lists and are currently prioritising children who need the treatment most urgently, or are at a higher risk of losing movement (ambulation) soon.'
An NHS spokesman said: 'The first National Institute for Health and Care Excellence (Nice) committee meeting to consider this treatment is scheduled to be held in July 2025, and if manufacturer ITF Pharma can offer a cost-effective price to enable Nice to recommend its use, the NHS will be ready to work with the company to explore fast-tracking access for patients.
'NHS England has published guidance on manufacturer-led early access schemes, which require trusts to cover substantial costs and find additional clinical resources to administer new treatments, and we understand a number of trusts across the country are preparing to offer givinostat via such a scheme.'
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