Muscular dystrophy drug rejected for use by NICE
A man with a rare muscle-wasting condition said he feels "deflated" that a drug that improves his symptoms has not been approved for use on the NHS.Andy Stephenson, from Doncaster, has myasthenia gravis and has been admitted to hospital 32 times because muscles involved in his breathing stopped working.But since taking the drug efgartigimod, the 45-year-old has not needed to go on a ventilator once and said he had seen a "huge improvement" in his symptoms.The National Institute for Health and Care Excellence (NICE) recommended the drug not be used by the NHS due to its "substantial" cost of more than £6,500 per infusion vial, despite it improving people's symptoms.
Myasthenia gravis causes fatigue and muscle weakness, double vision, slurred speech, and difficulty with chewing, swallowing and making facial expressions."If you weigh up the cost of the drug against the hospitalisations, invasive procedures, surgeons and one-to-one care I have needed at times - I think prevention [with efgartigimod] is a cost-effective option," Mr Stephenson said."This is the first drug which has made a huge improvement and, unless you are a multimillionaire, you are not going to be able to afford to get it."
'Immense relief'
Mr Stephenson was diagnosed in 2003 when, after leaving the armed forces, he began to struggle to exercise, close his eyes or smile."Food would get stuck in my throat and I would have to reach down and pull it out," he said."When I was drinking, I was choking, and water was pouring out of my nose."You worry, 'how am I going to live like this, what am I going to do?'"The father-of-four's symptoms fitted in with a recent car crash, but physiotherapists referred him to a neurologist who tested for the condition.Mr Stephenson said: "The diagnosis was an immense relief."If you have a chronic illness, it's good to learn about it, because you're going to be stuck with it forever."Adults who took part in a trial for efgartigimod reported regaining the ability to walk daily, dress independently, and some returned to work or education."Anybody who is on the drug at the minute can stay on it, but for new patients, it's not going to be available to them."I feel so lucky to be on it," Mr Stephenson said."I feel honoured and privileged to have been on the trial."Doctors will be able to privately prescribe the drug, also known as Vyvgart, meaning patients who can pay for it will still be able to benefit.According to drug company Argenx, the price for one 400mg infusion vial is £6,569.73.The number of vials used for one infusion, which Mr Stephenson said he has fortnightly, depends on the individual.
'Disheartening message'
Andy Fletcher, chief executive of charity Muscular Dystrophy UK, said the decision not to approve the drug for NHS use "sends a disheartening message to those affected by rare and complex conditions"."Treatments are within their grasp, but their needs are not being prioritised," he said.NICE said efgartigimod was an add-on alongside other treatments, rather than a replacement.Argenx appealed against the decision on the basis that the drug is a replacement, but NICE rejected the appeal."They got ignored and that, for everybody, was so frustrating," Mr Stephenson said."Our story should've made some sort of impact, but it feels it got bypassed and overlooked."NICE said drugs like efgartigimod replace other treatments only temporarily, providing "small benefits".A spokesperson said effective and fast-acting treatments like efgartigimod would be "welcomed" by patients, given the side effects and slow-acting nature of current treatments, but NICE could not justify the cost."The cost-effectiveness estimates [for efgartigimod] are substantially above what NICE considers an acceptable use of NHS resources," they said."We know this decision will be disappointing for people living with this debilitating condition and those who care for them."Muscular Dystrophy UK said Wales and Northern Ireland tend to follow NICE guidance, meaning patients there will most likely also be affected.A separate decision process is under way in Scotland.
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