NICE Agrees to Access to Nusinersen for Those who have SMA Type 3 and Aren’t Able to Walk

04 May 2021

SMA UK is delighted with this decision announced today. It comes after almost two years of advocacy and hard work by families and adults - who have been impacted so distressingly by their exclusion from treatment - their clinicians, patient groups and Biogen. The decision also removes the rules which have meant that those who lost the ability to walk after 12 months of treatment and children who had lost walking ability after July 2018 and did not regain this ability, wouldn’t be eligible for treatment to continue. Read the NICE press release, here.

Thank you

Our thanks to all in the Community who responded to our surveys and requests to tell us about the impact these rules had on you. All of this was submitted to Biogen and went forward as part of the evidence that was reviewed by NICE. You can see our summary of what we said, here.

Many also worked with national and local media to raise awareness, and patient groups TreatSMA and MDUK also worked tirelessly on this.

Our thanks also to the clinicians who gathered evidence and advocated strongly for a change to this rule; to Biogen for their work on gathering and collating all the evidence and making the overall submission to NICE; and to NICE and NHS England for seeing this process through in a way that has never happened previously.

New SMA UK CEO, Angela Smith-Morgan said:

"This is significant and much anticipated progress for people living with SMA Type 3. I am incredibly proud of the role that SMA UK has been able to play in advocating for the need for the treatment to be accessbile to all for whom it is clinically safe. I am delighted for our Community that their voices have been listened to."

What you can do now

These are the Paediatric Treatment Centres

These are the Adult Treatment Centres 

If you / your child are already under the care of a Treatment Centre that is, or should be, delivering nusinersen and wish for an assessment to see if this treatment could be clinically safe and potentially beneficial, you may want to make contact now to let them know you want to arrange this.

If you / your child are not already known to a Treatment Centre or haven’t been seen recently by a Treatment Centre that is, or should be, delivering nusinersen and wish for an assessment to see if this treatment could be clinically safe and potentially beneficial, contact either the hospital team that are currently delivering your care or your GP and ask for a referral to your nearest Treatment Centre. 

If you are an adult and your SMA care should be delivered by a specialist neuromuscular centre that has not signed up to deliver nusinersen treatment, let that centre and its NHS Trust know you want to be assessed and to have them make a suitable arrangement for this.

All the Patient Groups (SMA UK, MDUK, TreatSMA) have been working hard on access for all. If you are not already in contact with one or more of us and need support for access, please do get in touch.

What else is happening now?

We understand that:

  • Treatment Centres were recently made aware that a decision could be announced this week so that they could start preparing for requests for assessment for treatment if the decision was positive.
  • Though the majority of Centres are now providing treatment, there are Centres for adults where services are still being developed and will expand further. NHS England is working as a matter of urgency with Centres with treatment contracts to progress service delivery. There is also ongoing work to address how people living in any area of the country where a Specialist Neuromuscular Centre does not yet provide the service can access treatment.

SMA UK, MDUK and TreatSMA are working with NICE, NHS England and the Managed Access Oversight Committee to update the Managed Access Agreement document to reflect the inclusion of the newly eligible group of patients. We will communicate further details of these changes as they become available and it is anticipated that the revised Managed Access Agreement document and review documentation will be published on the NICE website by June 2021.

This is all the information we have at the moment. We have asked for more clarity and detail as soon as possible to answer any questions people may have and will publish this as soon as we have it. If you have particular questions, do please contact us or phone 01789 267520 and we will pass them on.