Spinraza – the first treatment for spinal muscular atrophy (SMA) – is due to be made available on the NHS in Scotland for children and adults with SMA Types 2 and 3. Subject to final agreement, people will be able to receive treatment from April 2019. It follows approval through the new ultra-orphan pathway assessment process for the rarest conditions, which was set up in October 2018 by the Scottish Government.
What happens now regarding access to Spinraza in Scotland?
The approval for access for SMA Types 2 and 3 is likely to be for a period of three years. Data on the treatment’s efficacy will be collected during this time with the aim of making a final decision on routine availability at the end.
Spinraza is already available on the NHS in Scotland for SMA Type 1 patients following the Scottish Medicines Consortium approval in May 2018.
Spinraza would be one of the first drugs to be approved through the new pathway. Once finalised, treatment will be routinely available for people with SMA Types 1, 2, and 3 in Scotland.
You can read our FAQs on Spinraza in Scotland, and what this means for the UK, here.
Reaction to the news
Muscular Dystrophy UK and Spinal Muscular Atrophy UK welcome the news, which could benefit up to an estimated 100 people in Scotland. The charities also hope the new pathway will pave the way for more patients with rare, muscle-wasting conditions to have quicker access to emerging treatments in the future.
However, the treatment is not currently available on the NHS elsewhere in the UK for any types of SMA and this decision highlights the unequal access across the UK.
Catherine Woodhead, Chief Executive of Muscular Dystrophy UK, commented:
Today’s announcement is fantastic news for families in Scotland and gives them hope for the future. We know that Spinraza slows down the progression of spinal muscular atrophy, a devastating and sometimes-fatal condition, and this decision has the power to change the lives of up to 100 children and adults and their loved ones.
We now want to see a similar assessment route introduced in the rest of the UK, which is lagging behind Scotland and other countries in approving the treatment. Spinraza is widely available in Europe, but if a child is diagnosed with even the most severe form of the condition they cannot access this treatment in England, Wales or Northern Ireland.
Dr Sheonad MacFarlane’s daughter, Eilidh, 10, has SMA Type 2. Sheonad (pictured with family), from Glasgow, also chairs Muscular Dystrophy UK’s Scottish Council. She said:
Naturally, the Scottish Medicines Consortium’s original decision for restricted use was a huge disappointment. But we vowed to continue fighting, and the decision to make it available for people with all types of SMA now feels like we have been given a second chance. Having access to Spinraza means so much to us and to other families, and we are overjoyed that our hopes have finally been realised.
As a parent, it’s heart-breaking to see your child gradually lose their physical abilities. Nothing can prepare you for the devastating emotional impact this has on you, particularly when there is a treatment out there that is agonisingly out of reach. Now, however, we can look to a future where those living with SMA can have a better quality of life.
Amy Cameron’s three-year-old son, Zac, has SMA Type 1 and has access to Spinraza on the NHS in Scotland. Amy, from Alloa, Clackmannanshire, said:
Our whole life was thrown into a whirlwind the day we heard those three words – spinal muscular atrophy. But Spinraza has bought us valuable time with our little boy and given us hope for the future. Zac has achieved things we never thought possible and continues to amaze us every day.
I’m so pleased that Spinraza will be available for people with other types of SMA. It’s made such a difference to our lives, and other families deserve that same chance.
Next steps in pressing for access across the rest of the UK
Spinraza is not available on the NHS in England, Wales or Northern Ireland for any type of SMA.
Muscular Dystrophy UK and SMA UK will keep campaigning on behalf of families in the rest of the UK. Now more than ever, we need to see leadership and urgent action from NICE, Biogen (the drug’s manufacturer) and NHS England, who must sit down together and show flexibility to find a solution.
Spinraza is currently being appraised by NICE for use on the NHS in England (Wales and Northern Ireland typically follow this guidance). NICE gave the drug a negative opinion for all types of SMA in its initial guidance, which was published on 14 August 2018. NICE also held a committee meeting on 23 October 2018 and has said since then that discussions are ongoing with Biogen.
Some children in these countries with SMA Type 1 can access treatment through a compassionate scheme – known as an Expanded Access Programme – introduced by Biogen in 2016. However, this closed to new patients on 1 November 2018. Infants already on the scheme continue to receive Spinraza.
NICE is due to make a further decision following its next committee meeting to discuss Spinraza on 6 March.
What can I do now to encourage access to Spinraza in the rest of the UK?
If you have the condition, or know someone who has the condition, you can contact your local MP and ask them to put pressure on NICE, Biogen and NHS England. Please email Jonathan Kingsley at: email@example.com to find out more.
We are also encouraging MPs to write to NICE, Biogen and NHS England about Spinraza, and the template letter for MPs to send to the three organisations can be downloaded here.
If you would like information or support, please call our Information and Support Team on our free helpline: 0800 652 6352 (Mon-Fri, 8.30am-6pm) or email us at: firstname.lastname@example.org. You can also find our factsheet on SMA here.