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:

Risdiplam:

‘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

Spinraza:

‘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.

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.

In 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.

• Responding to the Consultation

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. We have to respond with our submission by the end of April 2024.

• 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.

Upon reflection, the chair confirmed that he would allow three patient experts, representing child, teenage and adult voices. We will inform you who has been selected as soon as NICE have made it public.

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 office@smauk.org.uk
• Audio/video to WhatsApp 07759210888
• Use WeTransfer to send to office@smauk.org.uk

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.