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Charity backs brave NI boy's campaign to "end cruel postcode lottery" for drug

“The estimated cost that Belfast Trust has produced seems high and does not align with any other figures we have seen from other places," says leading UK charity

Colleen Pentony with her son Alfie from Newry.
Colleen Pentony with her son Alfie from Newry.(Image: Justin Kernoghan / Belfast Live)

A 12-year-old boy's campaign to overturn a "disgusting" decision to deny potentially life changing, free medicine to children in Northern Ireland has been backed by a leading charity.

Alfie Pentony, from from Newry, is one of a number of children in Northern Ireland with a condition known as Duchenne Muscular Dystrophy. It is one of the most severe, genetic muscle wasting conditions ever diagnosed, and until recently meant boys like Alfie were unlikely to live into their 20s.

But breakthrough advances in modern medicine and treatment has changed that outlook. A potential breakthrough has been made recently with a drug known as Givinostat - but health bosses are refusing to make it available in Northern Ireland despite 'early access' arrangements already in place elsewhere in the UK.

Read more: Mum's plea over "ridiculous" drug costs for son with muscle wasting condition

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The charity Duchenne UK, founded in 2011 by two mothers whose children were diagnosed with the condition, has now backed little Alfie's call for health authorities here to end the "cruel postcode lottery".

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Speaking to Belfast Live alongside his mum Colleen earlier this week, Alfie said it is "disgusting" that the drug hasn't been made available here.

"My friend is going to lose the ability to walk in a few weeks," he said. "It is disgusting".

Mum Colleen said: ""Alfie is now 12 and still has good ability walking, but even at that his muscles are breaking down every day and getting weaker. Why should he have to wait? He is a kid that understands his condition and only wants help - for him and for his friends so he can walk longer."

She added: "This drug could give Alfie a better quality of life. Why should I have to constantly fundraise to take Alfie to America for treatment to help slow this cruel disease down?"

Alfie from Newry
Alfie from Newry(Image: Justin Kernoghan)

Emily Reuben OBE and Alex Johnson OBE, founders of Duchenne UK, in a statement to Belfast Live in response to Alfie's brave campaign, said: “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.”

Givinostat was approved in December 2024 by the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) and has since been made available in other parts of the UK as part of an 'early access programme'

But the Belfast Trust, in its response to the Freedom of Information request in April, said it is "not in a position to proceed" with the early access programme. Assuming 13 patients in total, the Trust said it would cost £309,000 to roll the new drug out. It included the cost of various specialist services including psychology, physiotherapy, and play therapy in its estimate.

The estimate differs wildly from those given by Trusts elsewhere in the UK. A business plan from the Oxford University Hospitals Trust in England estimated the cost of rolling out the new drug to 35-40 patients at £66,000, according to a report by Channel Four News last month.

A spokesperson for the Trust told Belfast Live: "Givinostat is a new treatment approved by the Medicines & Healthcare products Regulatory Agency (MHRA) for the treatment of Duchenne Muscular Dystrophy (DMD). The National Institute for Health and Care Excellence (NICE) is currently developing recommendations for use of Givinostat within the NHS in England. Treatments that have been recommended by NICE for routine use in the NHS in England are typically also routinely available in Northern Ireland. Belfast Trust is required to work within the process for commissioning of new medicines following a NICE recommendation for use.

"The drug is available for an Early Access Programme (EAP) for eligible patients, pending decisions about wider access 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. We recognise this will be very disappointing for families and the Belfast Trust sincerely apologises to them."

The spokesperson continued: "Should NICE recommend treatment with Givinostat Belfast Trust will liaise with commissioning colleagues in the Department of Health’s Strategic Performance & Planning Group (SPPG) to establish what is needed to allow the Trust to be in a position to support its delivery.

"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 commence appropriate treatment and regularly review the patient. They can also take the opportunity to refer patients to trials in other UK centres if appropriate."

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The Trust spokesperson added: "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. "

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