A treatment for the rare muscle wasting condition myasthenia gravis has not been approved for use on the NHS in England.
Despite acknowledging that efgartigimod (Vyvgart), can ‘improve symptoms and people’s ability to carry out their normal activities’, the National Institute for Health and Care Excellence (NICE) has decided that the benefit it offers isn’t currently enough to justify the cost to the NHS in an announcement made recently.
The news comes as a huge blow to so many living with the condition and to charities Muscular Dystrophy UK and Myaware who represented patients throughout the treatment assessment process, especially as it has been announced during Myasthenia Gravis Awareness Month, a time meant to raise awareness and highlight the progress being made.
Myasthenia gravis is a long term chronic autoimmune condition that causes fatigable muscle weakness, weakness that gets worse with repeated muscle use and improves with rest. Symptoms include droopy eyelids, blurred or double vision, difficulty making facial expressions, difficulties chewing and swallowing, slurred speech, shortness of breath and weakness in the neck, arms and legs. Symptoms usually start in women under 40 and men over 60, but the condition can affect people of all ages. There is no cure for the condition.
Adults who received efgartigimod as part of the clinical trial reported regaining the ability to walk daily, dress independently, and take part in family life. Others spoke about being able to return to work, study again, and feel hope for the future.
A patient, living with myasthenia gravis, Andy, said:
“I’m privileged and honoured to be lucky enough to be included in the efgartigimod journey after 22 years of trying several different immunotherapy treatments including regular plasma exchanges, rituximab and mycophenolate, to name a few.
“I was disappointed and deflated with the news of efgartigimod not being made available to NHS patients. As a very refractory generalised Myasthenic with over 32 crisis’s requiring invasive ventilation, this is the best medication at this time to have made a huge impact and stop me from needing hospitalisations and ventilations. I really hope that in the future the people involved in the decision-making look at the clinicians reports and get these next generation medications made available for the patients, like myself, that have had no luck with the “standard” treatments.”
Andy Fletcher is Chief Executive of the charity Muscular Dystrophy UK, the leading charity for more than 110,000 children and adults in the UK living with one of over 60 muscle wasting and weakening conditions.
He said:
“We’re beyond disappointed with this decision. People living with myasthenia gravis have been let down by a system that continues to put up barriers to accessing treatments that could significantly improve their quality of life. Despite evidence that efgartigimod can improve symptoms, and strong support from clinicians and the patient community, NICE has chosen not to make it available on the NHS. This decision sends a disheartening message to those affected by rare and complex conditions — treatments are within their grasp, but their needs are not being prioritised.”
Charlotte Campbell, Research and Partnerships Manager, at Myaware said:
“We are deeply disappointed and disheartened by this decision, which impacts not only myasthenia patients, but all who suffer from a rare and poorly understood disease. June is myasthenia awareness month, and during a time where we should be empowering patients in the UK, we are instead reminded of the sincere unmet need of a patient community that has had little in terms of treatment innovation and choice over the years. In face of this uncertainty, we will continue to champion myasthenia patients and their right to clean, effective, and quick treatment.”
Although efgartigimod has not been recommended for use on the NHS, doctors can prescribe it privately meaning patients would need to cover the cost themselves. For many, this simply won’t be possible, and it risks creating a system where only those who can afford to pay ‘out of pocket’ can access a treatment that could significantly improve their quality of life.
Wales and Northern Ireland typically follow NICE guidance, so the decision is likely to apply there as well. Scotland has a separate medicines assessment process, so this ruling does not apply to people with myasthenia gravis in Scotland. Argenx, the manufacturer of the treatment, is currently in discussions with the relevant authorities in Scotland.
If you’ve been affected, please contact Muscular Dystrophy UK’s FREE helpline on 0800 652 6352 (open Mon – Thu 10am – 2pm) or visit: musculardystrophyuk.org.
