28-05-2025
‘Time is muscle' – children trapped in postcode lottery for life-extending drug
In the Duchenne community they say time is muscle.
So the clock is always ticking for the loved ones of those suffering with this form of
muscular dystrophy
. Six months ago those families were given hope – only for it to be dashed.
Duchenne causes progressive muscle degeneration and weakness.
Becoming non-ambulatory
– not being able to walk – can happen anytime between the ages of 8 and 12.
People with Duchenne
live into their twenties and thirties on average, but access to better treatments and care can help extend their lives.
And in December, there was potential good news when the Medicines and Healthcare Products Regulatory Agency (the MHRA) approved a new drug. Givinostat is the first drug to show a slowing down of that degeneration – to give boys – and it is mainly boys affected – longer literally on their feet. And the drug company that makes it said it would provide it to patients free.
But Channel 4 News has found that across England though hospitals are either delaying or are refusing to give the new treatment.
This has become a postcode lottery.
Great Ormond Street Hospital (GOSH) has said it will begin to treat Duchenne boys next week with the drug, but said its introduction has been challenging at a time when resources across the whole NHS are more stretched than ever, and they are working hard to identify the funding required.
In South London, at the Evelina children's hospital, they have told parents treatment will begin in late summer. Meanwhile other centres have not even gone that far – and patients are left waiting to see if they will get the drug at all.
We spoke to Alex Clarke, whose son Ben has Duchenne. Their local trust, Oxford University Hospitals, has told families that despite the drug itself being provided free, they cannot afford to use it to treat their patients.
Channel 4 News has now seen internal emails from the Oxford Trust, discussing the potential costs of introducing the drug.
Professor Andrew Brent, the trust's medical officer, writes:
'Unfortunately, however, given the current financial climate and the expectation … to make significant savings, we are not in a position to fund services/treatments that are not nationally approved without stopping doing something else.'
The trust's chief finance officer responded, stating:
'It is not our job to fund new medicines that are not NICE approved.'
Later in a statement Professor Brent said:
'It is a measure of the importance we place on Givinostat that we have been working on a business case before the drug has been approved by NICE. We will continue working towards being able to offer the treatment once approved by NICE, as we hope it will be.'
That business plan seen by Channel 4 News said it will cost just over £66,000 for about 35-40 patients.
While waiting for evaluation, and hopefully approval, from the National institute for Health and Care Excellence (NICE), patients can be treated under the early access programme.
This was set up at the same time as the MHRA approval but it took until March this year for the first trust to agree – that was the Leicester Royal Infirmary, followed by Cardiff and Swansea.
Under the early access programme trusts have to pay out of their own budget for the administration and monitoring of the drug.
In March, parents met with the Health Secretary Wes Streeting but today the Department of Health and Social Care said: 'We are aware of the immense challenges faced by people living with rare diseases such as Duchenne's muscular dystrophy, and their families.
'The department is committed to supporting access to specialist care, treatment and drugs for those with these conditions.'
It would appear their hands are tied.
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