NICE Announces Start of Review to Consider if People who have SMA Type 3 who're Unable to Walk can Benefit from Nusinersen Treatment

29 October 2020

NICE has today announced that it has begun the process to review data collected as part of the Managed Access Agreement (MAA) for nusinersen. Specifically, it will consider whether people who have SMA Type 3 who are unable to walk can benefit from the treatment and therefore should be included in the MAA. NICE expects to publish a final decision in 6 months.

About the review

The review, involving Biogen, patient groups, clinicians, SMA REACH UK and NHS England and NHS Improvement, will assess whether new evidence has become available to support a change in the MAA treatment eligibility criteria.  The process for conducting the review, which has been timed to enable the best available evidence to be submitted by Biogen, including data from a European registry study, is outlined on the NICE website (scroll down to Commercial Arrangements).

What will Biogen, patient groups and clinicians be doing?

Biogen has until 7th December 2020 to submit its evidence. SMA UK, other patient groups and clinicians will also be submitting any national and international evidence they have collected. All evidence will first be reviewed by an independent External Assessment Centre (EAC) who will then make a recommendation to the Managed Access Oversight Committee (MAOC). Representatives of all the above groups will continue to be part of the MAOC which will then review the evidence and recommendation of the EAC. For more information about what will happen, when and how please see the NICE website (scroll down to Commercial Arrangements and follow the links about the evidence review process where you will find details of the timeline and process).

We want to assist NHS England and Neuromuscular Treatment Centres with their planning should there be a positive recommendation for access. SMA UK will therefore be contacting people who have SMA Type 3 and have lost their walking ability to find out if they would wish to have this treatment.  

Interim arrangements for children who have SMA Type 3 who lost ambulation in the 12 months prior to 28th July 2019 and who are receiving nusinersen treatment

Until the outcome of the evidence review is known, NHS England and NHS Improvement (NHSE&I) has agreed to suspend the stopping criterion for paediatric patients who have not regained independent ambulation after 12 months of treatment - read more.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Assessment at NICE, said:

“There are people with SMA who are not able to access treatment with nusinersen under the terms of the MAA which began in July 2019.

“At the time we said that we would not close the door to these people. Uniquely for this type of arrangement we made a commitment that we would review new evidence on the potential benefits of nusinersen for type III SMA patients who are not currently receiving it. If that review shows those patients would benefit, we will recommend they are included in the MAA to receive nusinersen.

“With today’s announcement we are honouring our commitment to conduct a review and are engaging with the company and other stakeholders to ensure that every piece of relevant evidence can be considered as part of that process.”