For several years, myaware has been a stakeholder in the appraisal of efgartigimod (Vyvgart), a treatment for myasthenia gravis (MG). This appraisal has been organised by the National Institute for Health and Care Excellence (NICE) and we have worked closely with Muscular Dystrophy UK (MDUK) throughout this process.

After four committee meetings and appeals from the manufacturer Argenx and both patient groups, NICE has confirmed that the treatment won’t be funded and is not recommended for use on the NHS in England. This decision means that anyone eligible for treatment would need to cover the cost themselves, as doctors can only prescribe it privately. Please note this decision does not affect Scotland, as it has a separate medicine assessment process which is ongoing. Argenx is currently in discussions with the relevant authorities in Scotland, as myaware will continue to work alongside MDUK on this process to ensure that the unmet needs of patients is understood. Wales and Northern Ireland typically follow NICE guidance and so the decision can be expected to be reflected there as well.

Efgartigimod has been proven to provide clear benefit for patients with acetylcholine receptor (AChR) positive MG. We have heard from patients who have had significant improvement in their standard of quality of life and have been able to reduce their dosage of steroids and immunosuppressants, which often come accompanied with side effects and comorbidities. There are patients in the UK that, thanks to treatment with efgartigimod, can carry out activities, such as independent walking and return to work, that were impossible beforehand. Unfortunately, NICE has decided that the benefit it offers isn’t currently enough to justify the cost to the NHS.

Patients who currently receive treatment with efgartigimod should be assured that they will continue to do so under the care of their clinician, as per the statement from Argenx below:

Throughout the process, patients in the UK have been able to access VYVGART through the Early Access to Medicines Scheme (EAMS). argenx will continue to provide free-of-charge access to all patients who are currently receiving VYVGART; we are committed to ensuring that patients benefiting from VYVGART will be able to continuing doing so, irrespective of the NICE decision.

We are incredibly disappointed with this news and concerned about the future of access to care for patients in the UK. As a charity, we will continue to work forward collaboratively with all stakeholders to ensure the unmet needs of myasthenia patients are acknowledged and resolved. We will be planning our next steps as a charity in the coming weeks and look to our membership to work with us as we raise awareness of this issue with decision makers in this country.

Signed,

Trevor Ranson (Chairman), Bethan Peach (Medical Committee Chair), and Charlotte Campbell (Research and Partnerships Manager)