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NICE publishes second draft guidance on Efgartigimod for treating generalised myasthenia gravis

1 March 2024

In September we shared the news that NICE had published draft guidance on the use of the treatment efgartigimod for generalised myasthenia gravis, which didn’t recommend it for NHS use.

NICE consulted on this draft guidance and a second committee meeting took place on 16 November. Usually, publication of final guidance on the treatment being appraised would follow this, but in this case, NICE has taken the rare step of publishing a second draft guidance with a third committee meeting scheduled for May 2024. 

This second draft guidance still finds that efgartigimod should not be recommended for NHS use, but the fact that there will be a third committee meeting suggests NICE feels some of the issues around the treatment that led to this position could be addressed through further discussion and evidence gathering. 

What does the draft guidance say? 

This second draft guidance repeats the conclusions of the first that: 

  • Efgartigimod is not recommended, within its marketing authorisation, as an add-on to standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies. 
  • This recommendation is not intended to affect treatment with efgartigimod that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before the guidance publication, until they and their NHS clinician consider it right to stop. 

What does this mean? 

If accepted, this means people not currently receiving efgartigimod through the NHS in England will be unable to access it. However, people already being treated with efgartigimod will be able to continue to do so until their doctor considers it’s no longer necessary. 

The NICE appraisal consultation document can be read in detail here and is now open for consultation. 

The draft guidance acknowledges that ‘clinical trial evidence suggests that efgartigimod plus standard treatment improves symptoms and people’s ability to carry out their normal activities compared with standard treatment alone’ but highlights uncertainties that led the appraising committee to conclude it wasn’t clear efgartigimod was cost-effective enough to be recommended for NHS use. 

Some of the remaining areas of uncertainty include to what degree evidence gathered from clinical trials applies to the group of patients who the company wants to make the treatment available to; the degree to which efgartigimod would reduce the need for ‘maintenance intravenous immunoglobulin (IVIg)’, a treatment that can be used to boost antibody levels in people with myasthenia gravis; how the possible placebo effect from clinical trials has been factored into assessments of the treatment’s effectiveness; and the impact of myasthenia gravis on carers. 

Our response and next steps 

We’re disappointed and frustrated that some of the uncertainties highlighted in the first draft guidance remain unresolved. We’re particularly concerned that the impact of myasthenia gravis on carers is still considered uncertain, when much of the evidence we, patient experts and the charity MyAware, have put forward has focused on this issue. 

We’ve received compelling testimony from people who have received efgartigimod through an Early Access to Medicines Scheme (EAMS) about its positive impact on them and their families, and we’re calling on all parties to work together to address the uncertainties that remain −we’ve met with NICE and the company since the last committee meeting. 

While delays in securing wider access to efgartigimod are concerning, the fact that there will be a third NICE committee meeting is a positive sign that they feel the uncertainties could still be addressed and a positive recommendation might be made. We’ll continue to engage in the formal NICE process and will work with all parties in the hope that the process can be sped up. 

How you can share your views 

If you’re currently receiving efgartigimod and are affected by this news, we’re keen to hear from you so we can include your views in our work. Email our Campaigns teams

We will again be making a joint consultation submission with MyAware. You can make your own direct submission to the process through the NICE website

If you’ve been affected by this announcement, contact us on 0800 652 6352, Monday to Friday, 10am-2pm, or email: 

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