Families affected by SMA, a rare genetic condition that can leave children unable to crawl, walk, breathe and swallow, have been given hope by news that Spinraza – a life-changing treatment – looks set to be available on the NHS in Scotland.
Spinraza has helped infants with the condition to live longer, and in some cases crawl and even walk or stand.
Muscular Dystrophy UK’s Chief Executive, Catherine Woodhead, says:
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.
A new decision-making process introduced by the Scottish Government, known as an ultra-orphan pathway, means that patients with the rarest conditions, who are often children with limited treatment options, are more easily and quickly able to access specialised medicines that will help them. Subject to successful sign-off, Spinraza is set to be one of the first drugs to be approved through this new process and is expected to be routinely available for people with SMA Types 1, 2, and 3 from April 2019.
In May last year, the Scottish Medicines Consortium approved the treatment for use on the NHS in Scotland for SMA Type 1 – the most severe form of the condition – but not other types of SMA. The treatment is not currently available on the NHS elsewhere in the UK for any types of SMA.
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.
Dr Sheonad MacFarlane’s daughter, Eilidh, 10, has SMA Type 2. Sheonad, 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.
Catherine Woodhead continued:
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.
SMA UK’s Managing Director, Doug Henderson, said:
This is great news for the SMA community in Scotland giving wider access to this potentially life-changing treatment. We now want similar access across the rest of the UK. NICE is key to this. We want NICE to recommend Spinraza for all with SMA Type 1, 2 or 3 as a matter of urgency. We also want to see a future alignment of the NICE appraisal system with this new Scottish ultra -orphan pathway, which is so much better suited to medicines for rare conditions.
ENDS
For more information about the work of Muscular Dystrophy UK, please contact Laura Burge on 020 7803 2870, l.burge@musculardystrophyuk.org or 07917 768257 out of hours.
