Latest news with #Libmeldy
Daily Mail
12 hours ago
- Health
- Daily Mail
Revealed: The most eye-wateringly pricey drugs that the NHS is paying some £3million a dose for...while dementia and cancer patients are denied cheaper treatments
NHS bosses are paying millions of pounds for just a single dose of medication to treat some of the most devastating health conditions in the world. The drugs—some of which cost just shy of £3million per patient—can revolutionise and even save the lives of a handful of people born with rare illnesses each year. But it comes as Government officials have deemed cheaper treatments for far more common conditions like dementia and breast cancer as not worth the money. Libmeldy is widely cited as the most expensive drug available on the NHS, coming in at an eye-watering £2.875million per dose. It's used to treat babies with a fatal genetic disease called metachromatic leukodystrophy (MLD). While born seemingly perfectly healthy, children with MLD have a faulty gene that leads to a destructive build-up of fat around their nerves. Over time, this severely damages their brain and nervous system, with parents forced to watch helplessly as their child gradually stops walking, talking and then eating. Roughly four babies are born with MLD every year in the UK, and have a life expectancy of just five to eight years. But Libmeldy, a one-off treatment administered as an IV drip in hospital, uses a specially designed harmless virus to alter a patients' cells and remove the faulty gene that drives MLD. It's a bespoke treatment that uses a patient's individual cells, meaning a dose is unique to each individual. Another treatment that works in a similar way is Hemgenix, which MailOnline revealed was administered for the first time by the NHS earlier week. The drug —which costs an estimated £2.6million per patient—is also a gene therapy, delivered as one-off IV drip. It is the only treatment of its kind for haemophilia B, a bleeding disorder where the body doesn't make enough—or any—of a vital protein critical to clotting. Clotting stops wounds from bleeding, so those with the disorder risk suffering severe and even life-threatening blood loss from even minor injuries. Patients also run the risk of what are called 'spontaneous bleeds', which can be triggered without a direct injury and even prove deadly if they occur in a vital organ. Prior to Hemgenix, all haemophilia B patients needed regular weekly injections of an artificial clotting agent to keep their risk of catastrophic injury to a minimum. This meant many patients were effectively tethered to their scheduled injections, unable to live their lives without worry and anxiety about every potential nick and scrape. Similar to Libmeldy, Hemgenix works by replacing a patient's defective gene—which is incapable of producing the clotting protein—with one that can, eliminating the need for regular injections. Studies suggest the protective effect lasts for at least three years, but the hope is that it could work for even longer. There are approximately 2,000 people with haemophilia B in the UK. Yet, only around 260 with 'moderately severe or severe haemophilia B' are currently eligible for Hemgenix on the NHS. This puts the potential total bill to the taxpayer at roughly £676million. While a cost of £2.6million per patient may seem prohibitive, medics claim the treating patients this way actually saves the NHS money in the long term. The lifetime cost of providing a patient the alternative weekly clotting injections has been estimated to be £8million. This sum doesn't include the cost of life-saving interventions and surgeries haemophilia B patients may also need. Another multi-million drug approved for use on the NHS is Zolgensma, which comes in at £1.8million per dose. It's designed to help babies with spinal muscular atrophy (SMA), a genetic disease that typically kills within two years if left untreated. Around 56 babies are born with SMA each year, with the condition caused by a defective gene that plays a critical role in allowing nerves in the spinal cord to control muscles movement. SMA causes muscles to waste and gets worse over time, making it difficult for patients to breathe, move and eat. Nine in 10 of those who have the most severe form of the disease, known as type 1, die by the age of two if they do not receive treatment. Zolgensma is given as one off infusion—that like the previously discussed gene therapies—also fixes the faulty gene that drives SMA. Gene therapies are incredibly expensive medications due to the intense design and manufacturing process that goes into their creation. Additionally, because many of them benefit rare health conditions with small patient populations there is an argument that companies need to charge high prices to recoup costs given the drug may only be used a handful of times per year. But exactly how much the NHS pays for these drugs isn't clear. While the companies which make the drugs do charge millions for them, the health service often acquires the medications at an undisclosed discount, so the actual cost is likely to be less than it first appears. Which drugs get funded on the NHS in England and Wales is determined by the NHS spending watchdog, The National Institute for Health and Care Excellence (NICE). In making its decisions, NICE considers a multitude of factors. These include a drug's clinical impact—how much it improves or alleviates a patient's condition—as well as risk of side effects, practical issues like specialist storage, if there are alternatives, and, critically, how much it costs. It then judges if funding the drug will overall provides value for taxpayer money. This is often summarised using a metric called a quality-adjusted life year (QALY), essentially how much it costs to give a patient a healthy year of life. Lecanemab (pictured) and donanemab are currently only available to Brits who can afford to pay around £60,000 privately every year at select clinics Generally, if a drug costs about £20,000 to £30,000 per QALY NICE usually assesses it as a good use of taxpayer funding. This isn't a hard limit. The watchdog can rule drugs that are expensive and only work for a limited time, like end-of-life medications as worth the cost, recognising small amounts of time can be worth a lot to patients and their families. However, this process isn't an an exact science and can attract controversy. For example, the breast cancer drug Enhertu, described as a lifeline by campaigners, has been deemed too expensive by NICE for what it does. However, Scotland's equivalent of NICE—the Scottish Medicines Consortium—has deemed the £10,000 per patient per month drug as value for money. Research suggests Enhertu extends the lives of patients with one of the hardest to treat forms of breast cancer, buying them an extra year or more of life. Given as an infusion, it helps patients with an aggressive and fast-growing type of the cancer called HER2-positive. NICE previously accused the firm behind the drug, AstraZeneca, of refusing to 'offer a fair price'. About 57,000 cases of breast cancer are diagnosed in the UK each year with HER2-positive cancers accounting for roughly one in five of these, some 11,500. Other drugs that have attracted controversy after being rejected by NICE are donanemab and lecanemab. Both medications are designed slow down the early stages of Alzheimer's disease, the leading cause of dementia. The drugs bind to amyloid, a protein which builds up in the brains of people living with Alzheimer's, helping to clear out the substance and slowing cognitive decline. However—in its most recent ruling—NICE said while the treatments worked they only delayed the progression from mild to moderate Alzheimer's by four to six months. As such, the body ruled the medications cannot be provided on the NHS because they are not good value for money and 'only provide modest benefits at best'. Charities described the decision as 'disappointing' and a 'painful setback' for patients, while the firms Lilly, which makes donanemab, and Eisai, which makes lecanemab, said they would appeal the ruling. Alzheimer's is the most common cause of dementia in the UK with 944,000 Britons estimated to be living with the memory robbing disorder. NHS England published a briefing paper last year suggesting the cost of bringing the drugs to the health service could be £500 million to £1 billion per year. While multi-million purchases of single dose drugs using taxpayer funds—as people with far more common condition are denied cheaper medications—may raise eyebrows, the cost is worth putting into context. The NHS purchasing a one off dose of a £2.875million to save a child who would otherwise die, costs roughly 4p per person in the UK. This sum is about half of £5million the NHS in England spends on dishing out the over-the-counter painkiller paracetamol every month, despite the health service banning GPs from prescribing such cheap drugs to patients in 2018.
BBC News
05-04-2025
- Health
- BBC News
Hertfordshire girl with MLD 'could have been treated if screened'
Imagine if there was a treatment available that could save your child's life, but as soon as you discover that they need it, it is too late to is the heart-breaking reality faced by Emily and Sean Stock, from Hertfordshire, regarding their two-year-old daughter Lily. She has metachromatic leukodystrophy (MLD), a rare genetic disease which attacks the brain and spinal cord and is progressively robbing her of the ability to move, speak, see, swallow and eat. It will likely lead to the loss of her life between the ages of five and 2023, the world's most expensive drug, Libmeldy, became available on the £2.8m treatment is a gene therapy that can halt the progress of MLD and allow a child to go on to live a full and healthy life, but only if it is used before the child is symptomatic. Lily's parents are calling on the government, for the NHS and National Screening Committee to add MLD to the list of serious genetic conditions that are screened for at birth."The only way we could have saved Lily would have been to know she had MLD before it started to take effect", Emily says."If Lily had been screened at birth, we could have gone on to have treatment in six to eight weeks and prevented everything we're going through now. "To know that is devastating - I never want another family to face this."Libmeldy was first used in the UK in 2023 when it successfully halted the progress of MLD in 19-month-old had been diagnosed and treated in time because her older sister had become symptomatic and was beyond the point of benefiting from treatment."At the moment, this treatment is only really helping families who are already going through the heartbreak of knowing they are going to lose one of their children anyway," Emily adds: "This isn't just about Lily; it's about the many more children that will come to suffer with this if something doesn't change. "We're going to do everything we can to make sure it does change." Emily and Sean now face an anxious wait for the result of screening on Lily's five-year-old brother Arthur. The odds of carrying MLD are 40,000 to one. It's only if both parents carry it that MLD can be passed onto their child, with a 25% chance of that happening per and Sean became aware they were carriers when Lily was the disease takes hold, an infant's regression is July, Lily, aged 19 months, was developing as expected; she was able to sit up unsupported and was working hard to stand. She would laugh, smirk and smile when praised for her efforts. Eight months on, she is unable to support herself and has to be fed through a tube."She's a beautiful girl, she was a bright little button, she had a wicked laugh", says Emily. Lily's first symptoms were spotted at nursery; she suffered Nystagmus (an involuntary movement of the eyes), her legs were completely stiff with her toes continuously pointing and she wasn't learning to walk."We know there are so many more symptoms to come, that life will only get harder for Lily and that eventually MLD will take her from us far too early," says Emily. Alex, The Leukodystrophy Charity (Alex TLC) supports families with all forms of leukodystrophies, of which there are more than 100 Harrison, its director of support, says only MLD and adrenoleukodystrophy (ALD) are treatable. "Treatment is expensive, but so is the financial cost of caring for these children for the remainder of their lives," she says. "We're talking about young children losing their lives in horrible ways, experiencing some of the worst symptoms imaginable while they are alive."Josh Dean, the Labour MP for Hertford and Stortford, has raised Lily's case in parliament. "I would urge the government, the NHS and the UK National Screening Committee to seriously consider adding MLD to new-born screening", he says."This will do so much to reduce the chances of this happening to other young babies and children"The National Screening Committee is in the process of reviewing the evidence for a screening October, the government launched a study to test over 100,000 newborns for more than 200 conditions, including MLD. The findings "will help improve testing and treatment and help predict, diagnose and treat future illnesses," the government says. Sean's lifelong friend, David Kiddie, from Hertford, is embarking on a 190-mile fundraising Light - between the 10 southern Premier League football grounds and Wembley - aims to raise funds and awareness about the condition. He is due to finish the challenge by facing his fear of heights and abseiling down the Tottenham Hotspur Stadium."Tackling my fear is nothing compared to what Lily and the family are going through", he says. "The financial and psychological turmoil Emily, Sean and their family are facing will continue to escalate."Sean, who is a Tottenham fan, says David's support "means the world" and strangers have been backing the family and the "whole Hertford community". Follow Beds, Herts and Bucks news on BBC Sounds, Facebook, Instagram and X.
