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Scottish Medicines Consortium (SMC) Decision on Access to Nusinersen

Page last updated 9th October 2018.

On 7th May 2018, Scotland agreed to fund the treatment for those with SMA Type 1. Though this was good news for families affected by SMA Type 1, it was devastating for families and adults affected by SMA Type 2 or 3. However, on 8th October the Scottish Government announced their new pathway for ultra-orphan treatments which gives some hope. To be eligible for this pathway, a company needs to establish that:

  • The condition has a prevalence of 1 in 50,000 or less in Scotland
  • The medicine has an EMA orphan designation for the condition and this is maintained at time of marketing authorisation
  • The condition is chronic and severely disabling, and
  • The condition requires highly specialised management.

SMA has been designated an ultra-rare condition.

The good and hopeful news is that medicines that fall under the new definition and which have been recently reviewed by SMC but not recommended for routine use, will be admitted to the new pathway. This should include nusinersen. If then the Scottish Medicines Consortium (SMC) consider it clinically effective, it will be made available on the NHS in Scotland for at least three years while further information on its effectiveness is gathered. Following this period, the SMC would then review the evidence and make a final decision on its availability on the NHS in Scotland. You can read more about the process on the SMC website

We will continue to work hard with the other charities, Biogen, and members of the SMC and Scottish Parliament to ensure that nusinersen is re-assessed under the new pathway as soon as possible.

More about 7th May announcement and what followed:

This outcome was despite the hard work of the patient groups and the powerful testimonies from the SMA Community and clinicians via their submissions (you can read ours here) and the PACE meeting.  

The SMC committee's announcement stated:

 "the committee heard compelling evidence on the benefits of nusinersen, supported by the views of experts and patient groups and was able to apply a very high degree of flexibility in accepting this medicine for type 1 SMA. Unfortunately, the evidence presented suggested that nusinersen was substantially less cost effective when used in types 2 and 3 and therefore could not be accepted by the committee. These are incredibly difficult decisions. The medicine is extremely expensive and there is a need to consider all those who need treatment by NHS Scotland. We would welcome a resubmission from the company which addresses the issues raised.”

You can read Biogen’s reaction in their Community Update.

What did we do?:

We wrote jointly with the other charities to Biogen to ask them to review their pricing, work further on gathering evidence and re-submit as soon as possible. You can read our letter and their reply.

In May, we contacted the Cabinet Secretary for Health and Sport to ask that Dr Brian Montgomery’s 2016 Review of Access to New Medicines Report recommendation for the development of an alternative assessment pathway for ultra-orphan medicines be implemented urgently so that nusinersen could be reassessed for Types 2 and 3 under the new process as soon as possible. You can read our letter here. The new system was announced in June and, as of 8th October, is now in place.

What you can do:

Watch for further updates in our monthly E-news.