The Duchenne Muscular Dystrophy drug is available elsewhere in the UK but the Belfast Trust has opted against the early access programme
A Newry boy denied a breakthrough medicine for his rare health condition has been told he can’t move his care elsewhere in the UK, where the drug is already available.
Alfie Pentony, who has a form of muscular dystrophy known as Duchenne, is one of a small number of boys in Northern Ireland who have been denied access to a new type of drug known as Givinostat.
While the Belfast Trust has opted not to roll out an early access programme for Givinostat, the breakthrough new medicine is being made available elsewhere by several health Trusts elsewhere in the UK. Alfie’s mum, Colleen, had hoped to transfer his care to Newcastle, where there is a long established history of expertise in muscle disease diagnosis, care and research – and is willing to foot the bill for travel and accommodation to England herself.
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Health chiefs in Northern Ireland, however, have turned down the request in a move branded “cruel” by Alfie. And the 12-year-old boy from Newry is facing a race against time as NHS bosses decide whether to make givinostat available more widely on the NHS.
Duchenne is a degenerative condition, meaning Alfie’s muscles will continue to deteriorate until he loses the ability to walk and ultimately loses his life. It is unlikely he will be given givinostat if he becomes wheelchair bound before it’s granted approval.
His mum, Colleen, told Belfast Live she had submitted what’s known as an ECR – extra contractual referral – for her son so that he could have his care overseen by medics in Newcastle, rather than Belfast. The family, she said, already travels to Newcastle for specialist help that they pay for privately.
But the Belfast Trust has said no.
In a letter from the Strategic Planning and Performance Group at the Department of Health, which has been seen by Belfast Live, it was stated that there are “no clinical exceptional grounds to transfer this patient’s care to Newcastle”.
Colleen said: “I rang them and went nuts. They won’t let him go to where he would get the proper care. Alfie understands what is going on, he’s nearly 13 now, and he himself has said it’s cruel, and it is cruel.
“I know he won’t cope well in the wheelchair. I was speaking to another family in Newcastle, who is getting it [Givinostat], and they said the same thing – it’s cruel.”
She added: “I’m not going to let this lie. I’m not going to back down. It’s not right. I have to do what’s right for my son.”
As a new treatment, Givinostat was first approved in December 2024 by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and has not yet been given the green light for widespread roll-out on the NHS. It’s currently being made available, free of charge, through an Early Access Programme elsewhere in the UK.
But the Belfast Trust claimed in April, in response to a Freedom of Information request, that it would cost £309,000 to roll out the drug in Northern Ireland – a far higher estimate than the costs quoted by Health Trusts elsewhere in the UK.
Health Minister Mike Nesbitt, meanwhile, has said the Belfast Trust should “put plans in place to enable implementation to proceed at pace in the event of a positive recommendation by NICE this Autumn.”
The Trust’s decision not to roll out the early access programme has been criticised by a leading UK charity.
Emily Reuben OBE and Alex Johnson OBE, founders of Duchenne UK, told Belfast Live in June: “Patients in other parts of the UK can now access this treatment, but patients in Northern Ireland are losing out to this cruel postcode lottery.
“The estimated cost that Belfast Trust has produced seems high and does not align with any other figures we have seen from other places.
“Givinostat offers hope to families, and everyone who could benefit deserves access to this free medicine which has already been approved as a safe and effective medicine.”
The Belfast Trust was approached for comment on the refusal to allow Alfie’s care to be transferred to Newcastle. A spokesperson said: “Belfast Trust fully understands how difficult this issue is for Alfie and his family.
“We will continue to care for him and provide his family with all support and advice possible. We sincerely apologise to Alfie and his family for being unable to provide Givinostat at this time.
“While the drug can be made available for an Early Access Programme (EAP) following NICE recommendations, currently Belfast Trust is not in a position to proceed under the EAP as its implementation will need to be managed within defined and agreed protocols and additional staffing resources will also be required to ensure the treatment can be provided safely.
“Should NICE recommend treatment with Givinostat, Belfast Trust will liaise with commissioning colleagues in the Department of Health’s Strategic Performance and Planning Group (SPPG) to establish what is needed to allow the Trust to be in a position to support its delivery.”
The Belfast Trust statement continued: “The Royal Belfast Hospital for Sick Children provides comprehensive care for patients with DMD and is part of the NorthStar clinical network which consists of consultants, physiotherapists and other allied health professionals at 23 specialist paediatric tertiary centres across the UK. At the children’s hospital the neurologist, along with geneticists, makes a diagnosis and commences appropriate treatment and regularly reviews the patient. They can also take the opportunity to refer patients to trials in other UK centres if appropriate.
“A multi-disciplinary team approach is adopted for patients with DMD, and this includes neurology, cardiology and respiratory specialists as well as endocrine specialists. Physiotherapists are also involved with the patient’s care. Any proposal being considered for service development will reflect staffing and infrastructure costs for one full year. “
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