Muscular dystrophy: Drug not being made available for children is 'cruel'
A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available in Northern Ireland.Alfie, from Newry, County Down, was diagnosed with Duchenne muscular dystrophy (DMD) when he was four years old.His dad Jamie Pentony said the new drug, Givinostat, could slow his son's incurable condition down, but the Belfast Trust did "not have the capacity" to offer it.The Belfast Trust said it was not in a position to proceed with the implementation of Givinostat as "additional staffing resources would be required to ensure the treatment could be provided safely".
There are many types of muscular dystrophy. They are inherited genetic conditions that gradually cause the muscles to weaken, leading to an increasing level of disability.DMD is one of the most common and severe forms - it usually affects boys in early childhood and many people with the condition will only live into their 20s or 30s.Speaking on BBC Radio Ulster's Nolan Show, Mr Pentony said there were 13 boys in Northern Ireland who could benefit from the drug."Who is going to tell the 13 kids that could avail of this that some of them might not be walking next year?"Time is muscle when it comes to Duchenne. Your lung is a muscle, your heart is a muscle and most of the boys die from heart failure."They [the Belfast Trust] are telling us they do not have the capacity, they do not have the nurses to take the tests, that they do not have the neurologists to look after the boys whilst on this drug."
Alfie said his condition could stop him from walking, but doctors have told him that because he was "doing so well, it will take a while for that to happen"."The drug would really help me, it would help me a lot, but there are other children that will stop walking before me, so it's important for them to get it as well," he said."It's cruel they are not giving this drug to kids in Northern Ireland with this disability. We all have the right to have something that could help."
The anti-inflammatory drug, Givinostat, has been approved for use in the UK by the medicines regulator and is available early for people who meet strict criteria, on the NHS.Individual trusts must apply to take part in the Early Access Programme (EAP).The Belfast Trust said: "The drug is available for an Early Access Programme (EAP) for eligible patients, pending decisions about wider access following the National Institute for Health and Care Excellence (NICE) recommendations."Currently, Belfast Trust is not in a position to proceed under the EAP. We recognise this will be very disappointing for families and the Belfast Trust sincerely apologises to them."
'Time is muscle'
Una Farrell, from the charity Duchenne UK, said: "It's just hard to understand with a disease that is so cruel and progressive, and time is muscle, why children cannot be given access to it now."We understand that resources are a huge issue, but it's been known for several years that this drug was on its way."There is no cure for muscular dystrophy - finally there is a treatment that the data suggests can slow down the progression."
Routine health service access to Givinostat will depend on guidance from health assessment body NICE.Northern Ireland's Department of Health said it fully understood that patients and families want to access the best available health service treatments and drugs."The department maintains a formal link with NICE. In practice, this means that treatments that have been recommended by NICE for routine use in the NHS in England are also routinely available in Northern Ireland."NICE are developing guidance on the clinical and cost effectiveness of Givinostat for the treatment of DMD in people six years and over, with a committee meeting scheduled for May 2025."
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Scottish Sun
2 days ago
- Scottish Sun
Our brave Jasper is slowly losing the ability to walk – a drug could change that but the NHS cruelly won't fund it
Find out everything you need to know about Jasper's condition below LIVING NIGHTMARE Our brave Jasper is slowly losing the ability to walk – a drug could change that but the NHS cruelly won't fund it Click to share on X/Twitter (Opens in new window) Click to share on Facebook (Opens in new window) THE family of a 10-year-old boy who's losing the ability to walk have described the 'nightmare' battle they face with the NHS to access a free drug for help. Rosie and Pete Day from Horsham, West Sussex have been fighting for the drug givinostat for their son Jasper who has Duchenne muscular dystrophy (DMD). Sign up for Scottish Sun newsletter Sign up 4 Jasper and his twin sister both have Duchenne muscular dystrophy - but Arabella is a carrier while Jasper is most affected Credit: PA 4 The Day family are fighting for Jasper to get givinostat, which can slow progression of the condition Credit: PA Duchenne is a genetic muscle wasting condition that causes progressive muscle weakness. In the UK, an estimated 2,500 people are living with the condition The Day family have twins Jasper and Arabella, and two older daughters. Both Jasper and his twin sister Arabella have Duchenne, with Arabella a carrier and Jasper the most affected - as the condition mostly affects boys. Read more on muscle conditions BEHIND BARS Woman left battling fatal 'muscle death' triggered by first ever spin class Givinostat, developed by ITF Pharma UK, can slow progress of Duchenne - 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. Only some NHS trusts across the UK, and few in England, are giving out 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. Rosie said: "We try and live in the moment and, in this moment, there's an opportunity to make a massive change to Jasper. Toddler, 3, left fighting for life after 'going off his food' - as killer cocktail of common viruses attacked his heart "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.' She added: "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." 'When will I get the drug, mummy?' 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 continued: "If Jasper stops walking, we're not going to get that back. 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 Rosie Day "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." Rosie 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. And she 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. 4 Jasper is under the care of the Evelina London Children's Hospital, but it's unclear whether they'll give him the drug Credit: PA 4 The family say the NHS needs to urgently make the drug available to both ambulant (walking) and non-ambulant boys Credit: PA 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." Parents have met with Health Secretary Wes Streeting to try and speed up access.
The Sun
2 days ago
- The Sun
Our brave Jasper is slowly losing the ability to walk – a drug could change that but the NHS cruelly won't fund it
THE family of a 10-year-old boy who's losing the ability to walk have described the 'nightmare' battle they face with the NHS to access a free drug for help. Rosie and Pete Day from Horsham, West Sussex have been fighting for the drug givinostat for their son Jasper who has Duchenne muscular dystrophy (DMD). 4 4 Duchenne is a genetic muscle wasting condition that causes progressive muscle weakness. In the UK, an estimated 2,500 people are living with the condition The Day family have twins Jasper and Arabella, and two older daughters. Both Jasper and his twin sister Arabella have Duchenne, with Arabella a carrier and Jasper the most affected - as the condition mostly affects boys. Givinostat, developed by ITF Pharma UK, can slow progress of Duchenne - 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. Only some NHS trusts across the UK, and few in England, are giving out 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. Rosie said: "We try and live in the moment and, in this moment, there's an opportunity to make a massive change to Jasper. Toddler, 3, left fighting for life after 'going off his food' - as killer cocktail of common viruses attacked his heart "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.' She added: "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." 'When will I get the drug, mummy?' 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 continued: "If Jasper stops walking, we're not going to get that back. 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 Rosie Day "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." Rosie 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. And she 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. 4 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." Parents have met with Health Secretary Wes Streeting to try and speed up access. Duchenne muscular dystrophy (DMD) Muscular dystrophies (MD) are a group of inherited genetic conditions that gradually cause the muscles to weaken, leading to an increasing level of disability. Duchenne MD (DMD) is one of the most common and severe forms, it usually affects boys in early childhood; people with the condition will usually only live into their 20s or 30s. The condition primarily affects boys because it's an X-linked recessive condition. This means the faulty gene responsible for DMD is located on the X chromosome, and boys have only one X chromosome. Early symptoms often involve delays in walking, difficulty getting up, and enlarged calves. Over time, as the disease progresses, it can impact various parts of the body, including the heart and lungs. Treatment focuses on managing symptoms and slowing the progression of the disease, as there is no cure. Corticosteroids are a mainstay of treatment, and new gene therapies are also available for certain patients. Givinostat (trade name Duvyzat) is a treatment for DMD that has been approved for use in boys aged six and older, regardless of the specific genetic mutation they have. It's a non-steroidal drug that works by inhibiting histone deacetylase (HDAC), which helps to reduce inflammation, muscle loss, and scarring. While not a cure, givinostat can potentially slow down the progression of DMD and preserve muscle function.
BBC News
2 days ago
- BBC News
Belfast Trust: Questions over cost of muscular dystrophy drug
Questions are being asked over why Belfast Trust is quoting ten times the amount of money to treat young muscular dystrophy sufferers with a new drug as a trust in issue was raised in the House of Commons by Chris McDonald, MP for Stockton North, who is concerned patients across the UK are facing a "postcode lottery".The drug, Givinostat, has been approved as safe for use, and the National Institute for Health and Care Excellence (NICE) is due to make a decision on whether to recommend it for use on the NHS later this drug manufacturers offering it for free, Belfast Health Trust, NI's specialist centre for the condition, has so far declined to begin the treatment. The Belfast Trust has cited the need for "additional staffing resources" as the reason for not offering the drug. Some trusts in England, Scotland and Wales have already started offering treatment under an early access programme (EAP).Under a Freedom of Information request, Belfast Trust revealed it had estimated it would cost £309,000 annually to treat 13 patients with the new drug. That's more than £23,000 per a separate FOI request, the Oxford University Hospitals said they estimated the cost at £66,000 for 35 to 40 patients: just under £2,000 a head. 'It's like a bottle of Calpol' 12 year old Alfie Pentony from Newry has Duchenne Muscular Dystrophy. It is a life-limiting muscle wasting disease which affects will eventually need a wheelchair and there is currently no cure but this new drug Givinostat has been shown to keep patients on their feet for mother Colleen Pentony said she cannot understand how the Belfast Trust came to their cost estimate."It's ridiculous. To me, all they need is a nurse and a consultant."They take the medicine at home, I would administer it to Alfie in the morning and night at home. "It's a stable medicine, you don't even have to keep it refrigerated. It's like a bottle of Calpol." Mrs Pentony said the only additional resources needed would be to facilitate the blood tests the boys need, at first every two weeks, then every three said Belfast Trust had included a physiotherapist, play therapist and psychologist in the costings."These job roles should not be included in this costing. We are supposed to have a physiotherapist already, why would these boys need a play therapist, the boys are all over 6?""And any time in the past I asked for psychiatric help for Alfie, they refer me to CAHMS (Children and Adolescent Mental Health Services)", she added. Speaking in the House of Commons on Thursday, Chris McDonald MP, said there should be consistency in approach across the UK."Currently the Belfast Trust is not offering this drug to lads in Northern Ireland because they're claiming it will cost £309,000," he said."That's an order of magnitude out with the rest of the UK so I'd be grateful if the Minister would look very carefully at the situation in NI." 'Cruel postcode lottery' A spokesperson for Belfast Trust apologised to patients, saying said it was "not in a position" to offer the spokesperson said a multi-disciplinary team approach is adopted for patients with Duchenne Muscular Dystrophy which includes physiotherapists, neurology, cardiology and respiratory specialists as well as endocrine asked why their costing was higher than Oxford University Hospitals Trust, a spokesperson said: "It is not possible for the Belfast Trust to comment on costs associated with service delivery within another organisation as individual providers will have different infrastructure and staffing levels in place."A statement from the charity Duchenne UK said patients in Northern Ireland are losing out to a "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."
