Evidence and Summary that led to NICE’s Type 3 Access to Nusinersen Decision Published

04 June 2021

This momentous decision was first announced on 4th May. Over almost two years, SMA UK worked hard to advocate and contribute to this, amplifying the voices of the families and adults who have been impacted so distressingly by their exclusion from treatment.

On NICE’s website here, you can read:

  • Biogen’s submission – which included the submissions from patient groups and clinicians
  • What the independent External Assessment Centre report (commissioned by NICE) said when it reviewed the submission – which was a ‘no’ to changing the access rules for SMA Type 3
  • What the Managed Access Oversight Committee said in response to this review – which was a ‘yes’ to changing the access rules for SMA Type 3
  • The final decision (review outcome summary) – which was a ‘yes’ to changing the access rules for SMA Type 3
  • The new Managed Access Agreement Variation 1

We also explain how scales and measurements are now going to be used in the MAA, 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 considered as part of this review. 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.