Today, The Scottish Medicines Consortium Committee announced their decision to accept Spinraza for restricted use in NHS Scotland in infantile onset Type I SMA. Whilst we recognise this is a positive step forward and a lifeline for these children and families, it is hugely disappointing that children, young people and adults with Type 2 and Type 3, who wish to access and could potentially benefit from this treatment, still don’t have this opportunity.
Following the decision, 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.”
Following the SMC decision, Biogen issued a statement in response.
We, along with other SMA charities, have been calling for equitable access for those with all these types of SMA and this was made very clear in all the charities’ patient submissions and representations to the SMC. We will continue to work with all involved to push for early reappraisal of Spinraza for all childhood onset types of 5qSMA, with focus on the SMCs feedback.
In particular, we will be lobbying for early introduction of the new appraisal process for ultra-rare disease treatments, which, although accepted by the Scottish Government, following the Montgomery Review on Access to New Medicines, has yet to be implemented 18 months later.
You can show your support for fast-track reappraisal of Spinraza, by writing to your MP.