The National Institute for Health and Care Excellence (NICE) has published draft guidance that vamorolone, a potential alternative to existing corticosteroids, is not recommended, within its marketing authorisation, for treating Duchenne muscular dystrophy in people four years and over.
The NICE committee that is carrying out the appraisal concluded that vamorolone is an effective treatment for Duchenne muscular dystrophy and that it could offer important benefits because of its potential to reduce adverse side effects associated with corticosteroids. But there were several areas where they felt there isn’t enough evidence to recommend its use by the NHS. These areas include:
- Vamorolone’s relative effectiveness compared with other corticosteroids.
- The extent of the benefit of vamorolone’s potential to reduce adverse side effects associated with corticosteroids because of limited trial evidence.
- The need for more analyses of the impact of vamorolone on carer health-related quality of life.
This is not NICE's final guidance on this medicine. The draft guidance outlines NICE’s current recommendations and this is now open for public consultation.
What does this mean?
If this draft recommendation is accepted, this means people who are not currently receiving vamorolone through the NHS in England will be unable to access it. However, people who are already being treated will be able to continue to receive it until their doctor considers it's appropriate to stop.
The NICE appraisal consultation document can be read in detail here and is now open for consultation until Wednesday 24 April 2024.
Our response and next steps
Our Director of Care, Campaigns and Support, Rob Burley, is a formal Patient Expert to the appraisal, and gave evidence directly to the committee in partnership with a second Patient Expert, Mandy Roe, whose son lives with Duchenne muscular dystrophy and who has experience of receiving vamorolone.
We’re disappointed by the draft recommendation but encouraged that the NICE committee has recognised that vamorolone is an effective treatment and that it has the potential to reduce adverse side effects associated with corticosteroids side effects. As part of the appraisal process we heard directly from the Duchenne community about how important a greater choice of treatments is, and about the urgent need for treatments with less severe side effects.
It's very common for NICE to not recommend a treatment at this stage. The decision often changes after the consultation and a second committee meeting has taken place – for vamorolone, this second committee meeting is scheduled for Tuesday 7 May 2024.
We’ll be taking part in the consultation and we’re currently looking at the key areas where we feel the views of the community will have the most impact.
We also urge NICE and the company that makes vamorolone, Santhera, to work closely together between now and 7 May to address the evidence gaps raised in the draft guidance and to find a solution that means new patients can benefit from vamorolone when clinically appropriate.
How you can share your views
We’ll continue to work with all parties involved and to present the patient voice in partnership with the community and other patient groups. Over the coming weeks, we’ll let you know how to support our consultation response and how to make your own individual response.
In the meantime you can share your views with us by emailing our Campaigns teams
If you’ve been affected by this announcement and would like support, get in touch with our helpline by calling 0800 652 6352 between 10am-2pm Monday to Thursday or email info@musculardystrophyuk.org