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NICE’s Multi Technology Assessment of Nusinersen and Risdiplam  2023 – 2026 >

NICE’s Multi Technology Assessment of Nusinersen and Risdiplam  2023 – 2026 >

 

14th May 2026: NICE recommends nusinersen and risdiplam for permanent NHS England funding

After 10 years of advocacy and campaigning, SMA UK is delighted to celebrate this hard-won milestones with the SMA Community, patient groups and clinicians.

The decision follows the National Institute for Health and Care Excellence’s extensive review of these two the treatments. The recommendations were finalised on 4th June 2026.

NICE Website > 

 

Our Advocacy and Access Diary:

19th February 2026: Statement from NICE

The appraisal was discussed by the technology appraisal committee on 3rd February 2026.
We are unable to release the outcome of the committee meeting discussion at this time as NICE continues to be in discussions with the companies and NHS England. We can confirm that the committee doesn’t need any further information or analysis, and we don’t anticipate any further committee meetings will be required. We are working to finalise the recommendations as soon as possible.
We anticipate being able to update stakeholders by w/c 9 March 2026.”

 

3rd February NICE’s Second Committee meeting

25th November 2025: Positive Interim Outcome as NICE Committee Temporarily Pauses Appraisal

Following the committee meeting on 11 November, NICE paused the appraisal of risdiplam and nusinersen to review additional evidence. They were looking for more data from the pharmaceutical companies in three key areas:

  • Patient health-related quality of life
  • Healthcare resource use
  • Carer health-related quality of life

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.

  • Why the Pause? The committee made good progress on many issues, but they realised they needed more information before they could make their final recommendations. They called for ‘additional analysis’.
  • What Extra Information was Needed? The committee needed a better look at the money side of things (the ‘economic modelling’). This was in three main areas:
    1. How the treatments affect the quality of life of patients.
    2. How much healthcare patients need (like hospital visits or equipment).
    3. How the treatments affect the quality of life of the carers.
  • The Goal: They wanted 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.

 

September 2025: We submit the Joint Patient Group response to the EAG report to NICE

Our mission in collating the joint patient organisation response was steadfast, to secure full access to both drugs for all individuals with SMA, regardless of type, age, or functional status. Our detailed feedback to the EAG and NICE focused on several key areas where we believe the report fell short of fully capturing the true impact of SMA and the full value of the treatments.

Read our Response >

 

July 2025: New External Assessment Group (EAG Report) released

Newcastle University’s report was shared with stakeholders this month. The report reviewed all the clinical and economic data submitted by pharmaceutical companies, clinicians, and, importantly, patient groups. This latest report showed a significant improvement on previous assessments, with a wider recognition of individual differences across the SMA spectrum and a clear effort to incorporate patient perspectives.

Read the New External Assessment Group (EAG) report >

 

November 2024 – Review Delayed

The planned December 2024 NICE committee meeting to discuss the clinical and cost effectiveness of these two drugs was postponed.

NICE listened to, and agreed with, the concerns raised by SMA UK, our partner patient organizations, SMA clinicians, and pharmaceutical companies about the External Assessment Group (EAG)’s report.  The report was to critically evaluate the clinical and economic evidence for Spinraza and Risdiplam.

NICE then commissioned a new EAG report to build on this first one:

  • They asked for engagement with the patient and professional organisations to ensure the report’s validity.
  • The report will be published in July 2025
  • They will then hold a further 2 month consultation.

 

April 2024 Patient Groups respond jointly to NICE

We led the unified voice of the three patient groups and the SMA Community.

Jointly we advocated that:

  • In close consultation with individual’s specialist clinicians, treatment should be available for everyone living with SMA no matter the SMN copy number or clinical type.
  • Measures used to assess quality of life and outcomes from treatments are not sensitive enough to capture the benefits that treatment for SMA brings across the vast and varied spectrum of the condition. Therefore, real world data from patients should be carefully considered.

Read the  submission  and survey results sent to NICE. 

 

January 2024 Launch of Survey of Community Views.

We received responses from:

  • 114 people living with SMA, bringing perspectives of both treatments being assessed
  • 144 carers, sharing their invaluable thoughts and experiences

August 2023 NICE’s Scoping Consultation

We responded with MDUK to NICE’s invitation to review the background information for the review.

Read our joint Scoping Consultation response >