The National Institute for Health and Care Excellence’s (NICE) have published draft guidance ‒ following their recent appraisal of the treatment ravulizumab ‒ to say they will not be recommending rivulizumab as an add-on to standard treatment for generalised myasthenia gravis.
This is not NICE's final guidance on ravulizumab, as there will now be a period of public consultation. NICE does not recommend many treatments for use at this stage but often does after this public consultation period.
What this means
If NICE’s draft guidance becomes their final position, it would mean that people in England will not be able to receive ravulizumab.
The NICE appraisal consultation document is now open for consultation.
In their draft guidance, NICE recognises that evidence from clinical trials suggest ravulizumab plus standard treatment (when compared to standard treatment alone) does improve the symptoms of generalised myasthenia gravis, as well as people’s ability to do their normal activities. However, NICE felt it was unclear whether the people in the trial reflected the people who would receive ravulizumab in the NHS were it to be approved.
NICE has also raised concerns around the economic model used by the company that manufacturers ravulizumab. Their own estimates of its cost effectiveness are above what they consider an acceptable use of NHS resources. So, ravulizumab is not recommended.
The committee noted that because ravulizumab improves symptoms for people with generalised myasthenia gravis, it may also improve the health-related quality of life of carers.
Our response and next steps
It's very common for NICE to not recommend a treatment at this stage, and their decision can often change after the consultation stage and a second committee meeting. For ravulizumab, this committee meeting is scheduled for 16 November 2023.
NICE’s draft guidance recognises there is a clinical benefit from ravulizumab. Strong evidence was presented to NICE from clinicians and patients about both the impact of generalised myasthenia gravis on people with the condition and on caregivers, as well as about the need for a range of treatment options.
We urge all parties to work closely together between now and 16 November 2023 to address the challenges raised in the draft guidance and to find a solution that means people can benefit from ravulizumab when clinically appropriate.
Have your say
This announcement about ravulizumab follows news last month that NICE’s draft guidance on another potential treatment for generalised myasthenia gravis, efgartigimod, was also not recommended.
We’re now working in partnership with Myaware on a response to consultations for both treatments. If you, a family member or friend, live with generalised myasthenia gravis we would love to hear your views through this short survey.
To contact us directly to share your views, email our Campaigns team.
For those affected by this announcement who wish to speak to us, you can call, 0800 652 6352 between 10am-2pm on Monday to Friday. Alternatively, you can email us on info@musculardystrophyuk.org