NICE has contacted all stakeholders to say that the meeting to discuss the clinical and cost effectiveness of these two drugs will not be this December as planned. It is now likely to be in September 2025. Access to treatments will continue during this time. This will be for people starting the treatments as well as those already taking the treatments.

NICE has listened to, and agrees with, the concerns raised by SMA UK, our partner patient organizations, SMA clinicians, and pharmaceutical companies about the External Assessment Group (EAG)’s report.  This was commissioned by NICE and prepared by Warwick University. It’s purpose was to critically evaluate the clinical and economic evidence for Spinraza and Risdiplam.

NICE is now commissioning a new EAG report. This should be ready by the end of May 2025. NICE will then hold a further 1-month consultation. They anticipate that a first committee discussion will now be September 2025.

What did we say about the EAG report?

Working collaboratively, and in consultation with NICE, SMA UK led on a joint patient organisation response to the EAG report.  We submitted our response on 18th September expressing concerns that:

• Real world evidence was not considered as carefully as evidence from clinical trials.
• The report did not show an understanding of the fact that mobility outcomes do not reflect the holistic impact that SMA has on daily living.
• In a rare disease like SMA, clinical trials will always be small and long term data limited. Therefore, continuing to collect and review real world data is essential.
• The report did not seem to recognise that treatment outcomes can be individualised, therefore having a choice of treatments is essential.
• The report did not value stabilisation of the condition as a hugely positive treatment outcome and as such should be assessed as a highly effective outcome across the SMA population.
• The report did not recognise that 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.

What has NICE said about the comments on the EAG report?

Having reviewed all  stakeholder consultation comments on the EAG’s report, NICE is very concerned about the issues raised. The main ones they highlight are:

• Stakeholders felt that the report demonstrated a lack of understanding of SMA and a lack of clinical input and validation
• A concern that not all the available data had been used to its full potential, including the data collected during the managed access period
• Concerns about the lack of detail about the economic modelling and that it wasn’t clear about the changes made.

What happens now?

NICE has decided that there are so many concerns, they need a new external assessment group report. This will be prepared by Newcastle. It will build on the protocol designed by Warwick and submissions from the companies and other stakeholders but restart the review of the evidence from the beginning. In addition, NICE have asked Newcastle to engage with the patient and professional organisations involved in this appraisal during the creation of the report to ensure its validity.

NICE are confident that this will lead to a more robust report, given Newcastle’s experience of SMA and the data available, due to their involvement in the managed access oversight.

Newcastle will start working on this in the new year and produce a report by the end of May 2025. NICE will then hold a 1-month consultation. They therefore anticipate that a first committee discussion will now be September 2025. 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.

These MAAs allowed access to these treatments on the condition that researchers would collect more information from patients and their clinicians on:

• how well the treatment works,
• how safe it is
• whether or not it is good value for money.

In 2019 – 2020, the NICE committee did not feel there was enough evidence from the clinical trials to answer these questions.

This is why, if you have been 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.

When two or more treatments are used for the same condition, NICE reviews them at the same time via a Multiple Technology Appraisal (MTA).

They 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 This group examines all the evidence submitted by the different stakeholders. For this MTA, Researchers at the University of Warwick are leading this work. The results of their independent study will be used to help the decision makers. You can read their final protocol here.

Wednesday 4th December 2024 Committee meeting.

NICE will meet review all the evidence. This is their opportunity  to ask the SMA clinical and patient experts (open tab below to see who these are) and pharmaceutical companies any last questions they have on the effectiveness, safety and costs of the two drugs..

When to will we know what NICE recommends?

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.

This webinar summarises the process, the role of the patient groups and includes our earlier call for action.

Video recorded: 17th February 2024

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.

Scoping Consultation.

In August 2023 we were all invited to review 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 scoping response.

NICE’s final consultation questions.

At the beginning of January 2024, based on the comments from the patient groups, clinicians, scientific experts and pharmaceutical companies, NICE sent out the final consultation questions

Preparing for our consultation submission – our survey.

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 led a survey to obtain real-life feedback from the community. Thank you to all who responded.

The joint patient groups submission.

On 19th April 2024, SMA UK led on a response as a unified voice from the three patient groups and the SMA Community to the NICE appraisal committee members.

Patient and clinical experts.

NICE usually asks for nominations and selects 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 given there are two different treatments being assessed, which are prescribed to a wide range of ages and ‘SMA Types’.

On 4th March all three patient groups met with the chair of the committee to discuss why a wider representation is so important for this assessment.

Update: 5th September 2024

The EAG report, prepared by Warwick University in consultation with clinicians and patient representatives, critically evaluates the clinical and economic evidence for Spinraza and Risdiplam. This 460-page report compares the two treatments in terms of clinical and cost effectiveness, also considering Zolgensma and best supportive care. It will serve as key evidence for the committee’s decision-making meeting on December 4, 2024.

On August 7, 2024, NICE shared the report with stakeholders, including clinicians, SMA UK, Treat SMA, and MDUK. Patient organisations are invited to comment on whether the report accurately reflects the real-world impact of SMA. SMA UK is leading a joint response, highlighting the importance of real-world evidence, the need for individualised treatment options, and the limitations of current outcome measures.

With a deadline of Wednesday 18 September, working collaboratively, and in consultation with NICE, SMA UK are leading on a joint patient organisation response to the EAG report.  We are focussing on   

• The importance of considering real world evidence carefully alongside the clinical data.  

• Mobility outcomes do not reflect the holistic impact that SMA has on daily living.  

• In a rare disease like SMA, clinical trials will always be small and long term data limited. Therefore, continuing to collect and review real world data is essential.  

• Treatment outcomes can be individualised, therefore having a choice of treatments is essential. 

• Stabilisation of the condition is a hugely positive treatment outcome and as such should be assessed as a highly effective outcome across the SMA population.  

• 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.   
• 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.  

The committee will review all feedback before the December meeting. While an interim negative recommendation is possible, it is not final, as many rare disease treatments later receive positive recommendations after further review. Treatment access will remain unchanged until any final guidance, which may not take effect until mid-2025.

Over the past 2 years, 17 of 21 rare disease medicines (81%) that received an interim negative decision after the first committee meeting eventually received a positive recommendation. This was usually after a second or third committee meeting. 

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 (Evrysdi):

The full Risdiplam Managed Access Agreement

Nusinersen (Spinraza):

The full nusinersen Managed Access Agreement

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.

We asked Biogen UK for their comments. On 25th June  2024 they said:

‘Due to the nature and complexity of a Multiple Technology Appraisal (MTA), a final outcome is not expected until around April – July 2025. Furthermore, the full terms of the Managed Access Agreement (MAA) for nusinersen for new and existing patients, remain in place throughout the appraisal period until new guidance is published following the final outcome.

It is important to understand that the NICE process is designed to evaluate the clinical and cost effectiveness of a medicine. It is possible that following the first committee meeting (currently scheduled for 2nd October), NICE issue an interim negative recommendation for part of, or all of the population being appraised. However, it’s important to note that further committee meetings will take place to either confirm or overturn this interim decision following a period of public consultation and commercial discussions.  For example, over the past 2 years, 17 of 21 rare disease medicines (81%) that received an interim negative decision after the first committee meeting eventually received a positive recommendation. This was usually after a second or third committee meeting*.

Whilst Biogen recognises the outcome of the process may seem uncertain at this time, 32 of 34 medicines (94%) that have been assessed at the end of a Managed Access Agreement have received a positive recommendation following their NICE re-appraisal*.

Please be assured, Biogen is working very hard to ensure the nusinersen NICE appraisal is positive and that we achieve broad access for as many patients as possible in the UK.‘

*Analysis of NICE HTA outcomes from MAP Patient Access Ltd. HTA database for Biogen, April 2024

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

Your views and experiences are always helpful for us. If you missed the survey and would still like to tell us why access to these treatments matter to you, please contact us in one of these ways:

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