Mum of Newry boy Alfie casts doubt on ability of NI health service to roll out new medicine, after Belfast Trust says no due to cost
17:06, 09 Aug 2025Updated 18:26, 09 Aug 2025
Colleen Pentony’s son Alfie from Newry.(Image: Justin Kernoghan / Belfast Live)
Health Trusts have been told to be ready to “proceed at pace” if a new drug that could help NI children with a muscle-wasting condition is approved for use on the NHS.
But a Newry mum has expressed concern about the ability of Trusts here to roll out the new medicine, known as Givinostat, if it’s given the green light – after her son was told by the Belfast Trust he couldn’t get it through an Early Access Programme. That was despite the medicine being made available elsewhere in the UK.
Colleen Pentony’s 12-year-old boy Alfie is one of a small number of children who could benefit from Givinostat, which is designed to help people such as Alfie with a genetic muscle-wasting condition known as Duchenne Muscular Dystrophy.
READ MORE: Mum’s plea over “ridiculous” drug costs for son with muscle wasting conditionREAD MORE: Charity backs brave NI boy’s campaign to “end cruel postcode lottery” for drug
Duchenne causes people with the condition to lose muscle, meaning they will lose the ability to walk before it ultimately claims their lives.
But evidence shows givinostat can slow the progression of the condition. As a new treatment, the breakthrough medicine 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 Alfie has been told he can’t have it. 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.
Colleen Pentony with her son Alfie from Newry.(Image: Justin Kernoghan / Belfast Live)
Meanwhile, children here are facing a race against time for the drug to be made available, with Givinostat not approved for children who have already lost the ability to walk. If Alfie becomes wheelchair bound beforehand, he might never benefit from the breakthroughs in medical science that led to Givinostat.
In the meantime, Health Minister Mike Nesbitt has said he is “acutely aware” of the need to be swift in making it available.
He said he has told the Belfast Trust to “put plans in place” to “proceed at pace” if Givinostat is given the go-ahead for widespread use on the NHS.
Alfie’s mum Colleen, however, has expressed concern about the ability of the health service here to roll out the medicine – given their refusal to allow access through the Early Access Programme, in contrast with health authorities elsewhere in the UK.
“The care is not there for my son,” she said. “That’s because of the state of the health service in Northern Ireland. How can the Health Trust prepare to put this in place, when at the minute they are saying they can’t do it with the Early Access Programme? Are they ever going to be able to roll it out properly? If they can’t handle it now and, say it’s approved in September, October or November, how are they going to do it then?”
Responding to a written request for an update on the early access programme for Givinostat for boys with Duchenne, lodged in writing by the SDLP MLA Mark H Durkan, the Health Minister Mike Nesbitt said: “Compassionate use schemes such as Early Access Programmes (EAP) permit health providers to make new licensed treatments available to patients free of charge while they are being assessed for clinical and cost-effectiveness by the National Institute for Health and Care Excellence (NICE).
“They do not however provide any additional resources which are often required to safely administer the drug and monitor patients, as is the case with givinostat.
“Whilst it is an operational decision for health trusts to participate in an EAP and to identify the additional resources required prior to a recommendation by NICE, all our Health and Social Care Trusts are constrained by the severe budgetary pressures currently facing the Health and Social Care (HSC) system and the urgent need to reduce costs.
“It is my understanding that in light of this it has not been possible for the Belfast Trust to identify the additional funding that would be required to support the EAP for givinostat.”
Alfie from Newry(Image: Justin Kernoghan)
The Minister added: “I am however acutely aware of the time sensitive need to access givinostat promptly, and so I have requested that the Belfast Trust put plans in place to enable implementation to proceed at pace in the event of a positive recommendation by NICE this Autumn.”
In a statement to Belfast Live following the decision to deny Alfie access due to the costs they said would be incurred by allowing children here to take part in the Early Access Programme for Givinostat, a spokesperson for the Belfast Trust said: “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.
“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 Trust spokesperson said: “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.
“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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