NICE recommends nusinersen and risdiplam for permanent NHS England funding
After 10 years of advocacy and campaigning, SMA UK and MDUK are delighted to celebrate this hard-won milestones with the SMA Community. NICE’s recommendation ends the dark cloud of anxiety for the future that has been there for so many.
The decision follows the National Institute for Health and Care Excellence’s extensive review of these two the treatments. Along with Zolgensma, they have transformed the lives of many people living with SMA.
This outcome marks the result of years of dedicated advocacy and teamwork. SMA UK, Muscular Dystrophy UK, Treat SMA, clinicians, SMA REACH UK, and the SMA community all played key roles in this effort.
- Wales and Northern Ireland usually follow England.
- This does not change Scotland’s separate guidance
A Community Effort Driving Change
NICE announced this MTA review of SMA in 2023. Since then patient organisations have worked tirelessly. They have ensured that the voices and experiences of those living with SMA were central to the decision-making process.
SMA UK and our partner organisations:
- Coordinated national responses to consultations and evidence reviews
- Submitted detailed patient evidence to demonstrate the real-world impact of treatments
- Mobilised the community to share lived experiences through surveys and engagement activities
- Worked collaboratively with clinicians and researchers to strengthen the evidence base
Community insight was vital for showing decision-makers how SMA impacts daily life. It demonstrated the real benefits that treatments offer, beyond just clinical measures.
Influencing the MTA Process
Throughout this, SMA UK, Treat SMA, MDUK and specialist clinicians presented a unified voice to NICE.
We made evidence submissions in response to consultations and spoke up at committee meetings. We supported individuals and families to share their experiences. We made sure the realities of living with SMA were fully understood.
Towards the end of the consultation NICE identified some important information gaps. SMA UK, Treat SMA and MDUK surveyed 113 people in the UK to find the answers. We presented real life data that had not been captured through clinical trials. Thank you to everyone who responded to this and all our surveys over the last 10 years. Your voice made a difference.
The committee recognised the significant efforts of the SMA community and the importance of this latest survey. It is referred to in the final guidance published today.
Giles Lomax, CEO at SMA UK said, “This is a historic moment for the SMA community. After years of uncertainty, people can finally feel confident that these treatments will remain available on the NHS. It reflects the strength of patient voices and the power of lived experience in shaping decisions.”
A Step Forward, But Not the End
This positive MTA outcome is a big win. However, we must and will continue to advocate for long-term, equitable access to treatments for everyone who may benefit.
We will continue working with partners, clinicians, and the SMA community. We will monitor how this decision works out in practice. We will contribute to any future treatment consultations. We will push for further progress, including advances in research, access, and care.
