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Page last updated: 8th May 2024

The National Institute of Health and Care Excellence (NICE) approved Nusinersen for funding by the NHS in England in July 2019 and for Risdiplam in August 2020.

This was on the condition that researchers would collect more information from patients and their clinicians on how well it works, how safe it is and whether or not it is good value for money. This was because, at that time, the NICE committee did not feel there was enough evidence from the clinical trials to answer these questions. SMA, being a rare disease meant the numbers of participants were not big enough to satisfy a final decision.

Both treatments have been funded via time limited Managed Access Agreements (MAA) which come to an end this year (2024).  If you are prescribed either of these drugs, you will have been completing physio assessments, answering questions and completing patient reported outcomes which will be used as evidence.

SMA UK’s role as the patient organisation

From professional discussions with industry and clinicians, it became clear that the clinical evidence was not really reflecting the real-life experiences of treatment for those living with SMA. Notably, the value of stabilisation for adults and the importance of access for all no matter the severity of the condition.

SMA UK, Muscular Dystrophy UK (MDUK) and TreatSMA are consultees to the appraisal committee; this means we have the opportunity to contribute to the decision-making process in a number of different ways.

  • SMA UK will advocate for all those who have SMA Type 1, 2 or 3 so that they can access whichever of these two drugs their clinician agrees is the safest option for them.
  • SMA UK will also advocate for access to nusinersen for those who have SMA Type 4 and are not currently considered eligible for any treatments. Nusinersen has a licence to treat all with 5qSMA. Although there is no clinical trial evidence of it being used with those with Type 4, the condition has the same genetic cause.

Risdiplam’s licence is only for SMA Type 1, 2 and 3. It is not possible for NICE to make a recommendation that goes beyond the licence.

MTA Submission: 19th April 2024 

Based on the comments from the patient groups, clinicians, scientific experts and pharmaceutical companies, NICE sent out the final consultation questions at the beginning of January 2024. SMA UK led on a response as a unified voice from the SMA Community which was submitted to the NICE appraisal committee members. Read our unified response along with the appendix below.

MTA Submission Document

MTA Appendix Document

Patient and clinical expert nominations

It is standard practice for NICE to invite two patient experts to a committee meeting to give the patient perspective of the treatment being discussed. SMA UK, TreatSMA and MDUK all advocated for the need for more than this. With two different treatments being assessed, which are prescribed to a wide range of ages and SMA Types, all three patient groups met with the chair of the committee to discuss why a wider representation is so important for this assessment.

The chair confirmed that he would allow three patient experts:

  • Portia Thorman, representing children
  • Lucy Frost, representing teenagers
  • Andi Thorton, representing adults

August 2023

We reviewed the background information that was provided to all parties involved in the assessment to ensure it is correct and detailed enough from the community’s perspective. SMA UK and Muscular Dystrophy UK responded jointly. You can read our response here.

Your input is vital.

SMA UK have joined forces with Treat SMA and MDUK to produce a webinar about NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam. An expert committee will gather to assess the new clinical and real-world evidence, along with evidence from clinical trials, to decide whether the two treatments will be recommended for routine use in the NHS.

This webinar will inform you about the process, let you know about the role of the patient groups and will tell you how to get involved. We need your help to get these treatments approved.

Video recorded: 17th February 2024

An explanation of what happens if the committee does not recommend either treatment for NHS funding is written into the Managed Access Agreements. See the relevant extracts from the documents below:


‘2.10: If at the guidance update NICE publish negative final guidance for risdiplam, existing patients may continue to receive treatment until they and their treating clinician consider it appropriate to stop. The company and NHSE&I have agreed how access to treatment will continue in these circumstances: this is detailed within the commercial access agreement.’

You can read the whole MAA document here


‘8.1 If at the termination or expiry of this MAA: (i) NICE does not recommend nusinersen for NHS funding for patients, NHS England funding for nusinersen will cease to be available and treatment will cease (in which case cessation shall be managed between the MAH and NHS England to ensure it is effected in a controlled manner, this will be agreed in collaboration with the MAA Oversight Committee which includes clinicians and patient groups)..’

You can read the whole MAA document here

We understand the anxiety this will bring to Spinraza-treated patients. At SMA UK we are doing all we can to retain NHS funding for both treatments, ensuring everyone living with SMA has the choice of available disease-modifying treatments to suit their individual needs. We will be advocating for full and equal access for all.



When two or more treatments are used for the same condition, NICE reviews them at the same time. They set up a Multiple Technology Appraisal (MTA) and have done this for these two treatments.

They will look at:

  • all the evidence that has been collected while the MAAs have been in place
  • any new evidence from clinical trials
  • international studies

They also invite any groups that have contact with, or are part of, the SMA Community, to submit any evidence they hold and give their views on the specific questions they are looking to answer.

You can see the list of these potential ‘stakeholders’ on this NICE website page.

NICE also commission an external assessment group to review all the information and examine the evidence submitted by the different stakeholders throughout the process. Researchers at the University of Warwick leading this project will investigate how effective and how cost effective the treatments are. The results of their independent study will be used to help the decision makers. You can read their final protocol here.

They will have a committee meeting later in the year to review all the evidence and make a decision about whether to recommend the treatments for further funding by the NHS.

Wales and Northern Ireland have followed NICE’s guidance on access and are likely to follow the recommendations.

Our survey has now closed – thank you to everyone who submitted their responses.

We received 114 responses from people living with SMA, bringing perspectives of both treatments being assessed. We also received responses from 144 carers, sharing their invaluable thoughts and experiences.

Case studies are always helpful for us, so if you would still like to submit one, please choose from one of the following options:

  • Please submit written case studies to
  • Audio/video to WhatsApp 07759210888
  • Use WeTransfer to send to

Between April and October, evidence from all the different experts involved will be carefully considered by the members of the committee. The committee meeting in October is NICE’s opportunity to ask the SMA clinical and patient experts and pharmaceutical companies any last questions they have on the effectiveness, safety and costs of the two drugs.

About three weeks after the committee meeting, NICE will send out their draft recommendations to all consultees. The patient groups and others experts involved will then have 20 working days to submit their comments before the recommendations are published.

If there are no appeals against the draft recommendations, the guidance will be published about 16 weeks after the final committee meeting.

It often takes more than one committee meeting to reach a very final decision.

Date Actions
Friday 5th January 2024 Invitation to participant sent to all stakeholders
Forms for Completion, appraisal guides and submission templates available
The final scope, stakeholder list, response to scoping consultation comments and the Equality Impact Assessment available on NICE website
-Friday 26th January 2024 Deadline for participation and confidentially agreements and expert nomination forms
External Assessment Group (EAG) Protocol sent to stakeholders
Friday 2nd February 2024 Stakeholder Information Meeting (SIM)
Friday 19th April 2024 Deadline for stakeholder submissions
Early August to early September 2024 Consultation on EAG report
Tuesday 2nd October 2024 Committee Meeting