11th November 2025: Appraisal Committee Meeting
Today is first NICE committee meeting for this joint review of the two drugs, Spinraza (Nusinersen) and Risdiplam (Evrysdi) (known as a Multiple Technology Appraisal, or MTA).
Representatives from SMA UK, Treat SMA and MDUK will be present at this meeting as Patient Experts. They will speak about their personal experiences of the treatments. They will also share the perspectives of the wider community. This review will decide whether NICE will recommend these life-changing treatments for permanent, routine NHS England access. Wales and Northern Ireland usually adopt the same recommendation but may choose to follow Scotland.
Over 2 years ago, in July 2023 SMA UK, Treat SMA and MDUK came together to start to plan how we could ensure the SMA community’s perspective would be heard loud and clear.
Thank you to all of you who have participated by sharing your experiences through our surveys and on our social platforms. Your stories have built our key messages. Your direct quotes have reinforced our points in the most powerful way. Your willingness to participate and share how treatment has impacted your lives has enabled us to build a strong and persuasive argument for retaining these treatments across the spectrum of SMA.
We have a team of 5 patient and clinical experts delivering our collaborative messages at the committee meeting on Tuesday 11th November:
From the patient experts:
Lucy Frost
My name is Lucy, and I am the co-founder of Treat SMA and mother to George, aged 14, who has Spinal Muscular Atrophy and is currently taking Risdiplam.
I played an active role, alongside my colleagues, advocating to launch the Early Access Programme (EAP) at Great Ormond Street Hospital for children with SMA Type 1 to access Nusinersen and across the UK. I was also deeply involved in organising protests, campaigns, and media outreach to ensure Nusinersen became available here in the UK, working closely with NICE and Biogen throughout that process.
In 2021, I served as a patient expert on the NICE appraisal panel for Risdiplam, as a member of Treat SMA.
For this appraisal, I will be speaking about Risdiplam and how it has affected not only George but also his sister and me. I will share insights into the positive impacts the treatment has brought to our family benefits that often aren’t fully reflected in the clinical data.
Ultimately, my hope is that both treatments will be made available to everyone affected by Spinal Muscular Atrophy.
Andi Thorton
My name is Andi and I live with SMA type 2. I will be bringing the adult perspective to the meeting.
Treatment enables adults with SMA to maintain function, continue with careers, and build plans for the future. My aim is to ensure that stabilisation and small, clinically unmeasured gains are appropriately valued by the committee.
Portia Thorman: SMA UK
I am the head of Advocacy and Community at SMA UK. I am also mum to Ezra, a charming 9-year-old young man living with SMA type 1.
Through both my professional and personal life, I have built a solid network of colleagues and friends living with SMA. I will speak up to ensure that all treatment impacts are valued including the social and psychological aspects, and the value of stabilisation.
Ezra is a Spinraza patient, personally, I will be sharing the many and varied ways that Spinraza has not only allowed him to live but has enabled him to thrive. My particular focus will be on the gains that we see at home which go unmeasured by clinical scales.
From the clinical experts:
Dr Channa Hewamadduma,
I’m Channa Hewamadduma, neuromuscular neurologist who cares for adult persons with Neuromuscular disorders. I lead the SMA service in my region for adult SMA patients. I’m attending the MTA as a clinical expert in SMA.
A large number of adults with SMA have benefitted from disease modifying therapies. My hope is to convince the panel attending MTA that the therapeutic unmet needs of SMA patients and secure an approval for both Risdiplam and Nusinersen for paediatric and adult SMA persons.
Dr Mariacristina Scoto
I am a paediatric neuromuscular consultant working at GOSH, with 15 years’ experience in SMA clinical management and translational research; I am also co-PI of the SMA REACH UK network which was chosen to collect the national safety and efficacy data on both Nusinersen and Risdiplam. My role, as nominated clinical expert for the paediatric cohort, to share my knowledge and clinical experience on the impact that both these treatments have on patients and families.
As a clinician I have seen the positive impact these treatments have had on patients and families in the past 5 years, so my hope is for an open access for both treatments; my hope is that clinicians and families will be allowed to have a transparent and free discussion on all treatments about their known risks and benefits, in order to reach an agreement on which one to choose for each patient. I hope that through this process, all voices will be heard and the need to continue providing these treatments to all patients will be understood and accepted.
I hope we will be able to continue collecting meaningful data for patients to answer to many existing unknowns on long term benefits. I hope we will continue to have an open dialogue with NICE and NHSE to improve patients’ satisfaction and care.
We know that news about access to treatment can be a source of great anxiety for everyone in the SMA community. We are committed to advocating for full and equal access to both treatments for everyone living with SMA. This will ensure that you and your clinicians have the choice that best suits your individual needs. We will continue to keep you updated through every stage of this process.
About 3 weeks after this meeting, NICE will issue the draft guidance.
