SMA UK, Treat SMA and MDUK 

On November 11th, NICE held its first committee meeting to review whether to recommend permanent routine NHS funding for risdiplam (Evrysdi)  and nusinersen (Spinraza).  The current managed access agreements end in September 2026.   

NICE committees are independent. They are made up of about 25 to 30 members, including health and care professionals and academic researchers. At this committee meeting members heard evidence from clinicians and patient groups, Biogen and Roche.  

We are pleased to report that throughout what turned out to be a very long six-hour meeting, the voices of the patients and clinical experts were very much listened to and highly valued. . 

The Outcome 

Following that long meeting, NICE has temporarily paused the review for risdiplam (Evrysdi) and nusinersen (Spinraza).  

• Why the Pause? The committee made good progress on many issues, but they realised they need more information before they can make their final recommendations. They have called for ‘additional analysis’. 

• What Extra Information is Needed? The committee needs a better look at the money side of things (the ‘economic modelling’). This is in three main areas: 

1. How the treatments affect the quality of life of patients. 

1. How much healthcare patients need (like hospital visits or equipment). 

1. How the treatments affect the quality of life of the carers. 

• The Goal: The want to make sure their final financial review correctly includes all the benefits of the treatments described by SMA patients and clinicians. They felt the true benefits of the treatments had not been fully captured by the current data.  

• Why the Pause is Good News: This pause is a positive sign! It demonstrates that NICE is listening closely to the lived experience shared by us, the people living with SMA every day. The committee is doing all it can to ensure that the economic calculations properly account for all the benefits and gains of these treatments. This especially includes those real-world improvements that might not have been fully measured or captured in the original clinical trials. 

• Partner Organisation Commitment: We will continue to work closely with NICE and with the pharmaceutical companies during this pause to help move the process forward. 

Next Steps and Timeline 

• Who Provides the Data? The economic models are very technical. They are based on the financial plans created by the drug companies. NICE has therefore asked the companies to provide the new data. 

• What Happens Next? Once the companies provide the new information and NICE has reviewed it, they will schedule a second public committee meeting to discuss it. 

• Timeline: We don’t have a date yet, but the second discussion is expected to happen in early 2026. NICE will not release any draft recommendations until after that second meeting. 

Webinar Update and Your Questions 

SMA UK and Treat SMA are planning to update the SMA community with more detail in a webinar. 

• Who will be there? Patient and clinical experts who were part of the committee meeting will be joined by a representative from NICE. 

• Submit Your Questions: If you have any questions about the pause, the next steps, or the process, please submit them through the form here:  

Community update on NICE process for continued access to risdiplam and nusinersen.  – Fill in form 

Access to Treatment Continues 

Crucially, while this review is paused, access to risdiplam (Evrysdi) and nusinersen (Spinraza) will continue. This applies to everyone: people who are already on the treatments and those who are just starting them. 

Statement from NICE: 

Following the committee meeting on 11 November, NICE is pausing this appraisal. This is because while good progress was made in resolving some key issues, the committee has identified some additional analysis that it needs to help it make recommendations. This analysis relates to the economic modelling in 3 broad areas: patient health-related quality of life values, healthcare resource use and carer health-related quality of life. The aim of seeking additional analysis in each of these areas is to make sure that the economic modelling adequately captures the benefits of the treatments that were described by the patient and clinical experts and seen in the clinical data.   

 As this analysis is of a technical nature and related to the company modelling, NICE considers that the companies are best placed to provide it. Once this analysis is available and has been reviewed, a second public committee meeting will be scheduled to discuss it. NICE will not be releasing a draft guidance document until after this second committee meeting. We will be in touch with further details once we are able to, but it is likely that the second committee discussion will be held in early 2026.  

 As a reminder, access to treatments will continue while the appraisal is ongoing. This will be the case for people starting the treatments as well as those already taking the treatments. 

Statement from Biogen 

Biogen welcomes NICE’s decision to pause the appraisal and to seek additional analysis following the most recent committee meeting. We believe that this is the right step to take given the complexity of the issues and the importance of getting the decision right. 

We will be submitting additional evidence, and we hope that this will help to address uncertainties and ensure that the value of these treatments to patients and carers is accounted for at the next committee meeting.      

As stated by NICE, new and existing patients will continue to be able to access treatments while the appraisal is ongoing. Biogen is committed to continuing to work with NICE to reach a positive outcome for people in the UK living with SMA and their families. 

Statement from Roche 

We acknowledge the decision by NICE to pause the Multiple Technology Appraisal for risdiplam to allow for additional economic analysis in key areas, including patient health-related quality of life and carer quality of life. We are grateful to the patient representatives and clinical experts who attended the committee meeting. Their essential contributions ensure that the patient experience remains central to this appraisal process. 

Our primary concern remains with the spinal muscular atrophy community and we fully understand and support NICE’s objective to ensure the economic modelling accurately captures the full benefits of risdiplam as described by patients and clinicians. We are committed to working closely with NICE to provide the requested technical analysis to facilitate a constructive discussion at the next committee meeting, with the shared goal of achieving broad and sustainable access to this important treatment.