Act now or babies will needlessly die – that is the message from Muscular Dystrophy UK and Spinal Muscular Atrophy UK ahead of a fourth NICE committee meeting next week about Spinraza.
The charities, along with clinicians and families, are urging decision-makers and the government to take urgent action to make the treatment available for all people with all types of SMA in England, Wales and Northern Ireland.
It has been 16 months since NICE started its appraisal of Spinraza, the first treatment for SMA. Without access, children with the most severe form of the condition are unlikely to reach their second birthday, while others will lose the ability to walk, and may have increasing difficulties breathing and swallowing.
NICE’s meeting on Wednesday 8 May will be held in private. We are calling on NICE, drug company Biogen and NHS England to show flexibility and reach an agreement ahead of that meeting. A joint letter has been sent from MDUK and SMA UK to all three parties.
What we are saying
Catherine Woodhead, Chief Executive of Muscular Dystrophy UK, said:
The continued delays are unacceptable. The stark reality is some children will die without access to Spinraza. This is devastating for families, who know there is a treatment that could help but is agonisingly out of reach. In the 16 months it has taken for this drug to be assessed, lives may needlessly have been cut short, and children and adults have lost the ability to walk, breathe and swallow. No more time can be wasted. The Government must recognise that the system is broken and urgently act to overhaul the appraisal process for treatments for rare diseases. But right now, NICE, Biogen and NHS England must end families’ heartbreak by showing flexibility on cost and coming to an agreement urgently.
Doug Henderson, Managing Director of Spinal Muscular Atrophy UK, said:
The SMA community has waited long enough. NICE began appraising Spinraza 16 months ago in January 2018. In the meantime, England has gone from being a world leader in health delivery to people with SMA to 47th, behind many far less wealthy countries.I urge Biogen, NHS England and NICE to complete negotiations by 8 May with a Managed Access Agreement for all with SMA Types 1, 2 and 3. As each week rolls by without access to this life-changing treatment, muscle function deteriorates for children and adults with SMA and newly diagnosed babies are left with a very uncertain future.
Rachel Jamieson’s four-year-old daughter, Matilda, has SMA Type 3. This form of the condition does not affect life expectancy, but leaves children less able to stand and walk over time. Rachel, from York, said:
My heart breaks when my gorgeous, bubbly daughter Matilda asks me why she has wobbly legs, or whether her children will have SMA. Since starting school last year, Matilda has struggled more with her mobility. I believe that, if she had received Spinraza, she would not be having those difficulties. Spinraza gives us hope, but we need it to be made available – and fast. Without this treatment, I fear for Matilda’s independence when she’s older.
Dr Sheonad MacFarlane’s daughter, Eilidh, 10, has SMA Type 2. Without treatment, children with SMA Type 2 are never able to stand unaided, and this continues on into adulthood. Eilidh has now received Spinraza after the drug was approved for use in Scotland for children and adults with SMA Types 2 and 3 earlier this year. Sheonad, from Glasgow, also chairs Muscular Dystrophy UK’s Scottish Council. She said:
Having access to Spinraza means so much to us and to other families; we are overjoyed that Eilidh now has access to this treatment. We still don’t know what the future holds, but we, at last, have hope. 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. It’s unthinkable that children and adults still don’t have access to this treatment in England, Wales and Northern Ireland. My heart goes out to families, and I just hope NICE can reach a decision soon.
Professor Francesco Muntoni, Paediatric Neurologist at Great Ormond Street Institute of Child Health, said:
SMA patients have been let down and now we are essentially the last country in the civilised world to adopt this drug. Spinraza is an effective treatment for SMA but, once the condition has progressed in its course, children are unable to regain lost function. Delays in treatment will mean babies with SMA Type 1 face early death. As a physician, there is nothing more frustrating than knowing a medicine that can help your patients in England is out of reach, while it’s available in Scotland and other European countries. NICE, Biogen and NHS England must come to a compromise and make this drug available.
Spinraza is available in 24 European countries and 46 globally, including Scotland. Some 80 children in England, Wales and Northern Ireland with SMA Type 1 – the most severe form of the condition – access the drug through a free global compassionate scheme put in place by Biogen in 2016. These children continue to receive the treatment via the NHS with Biogen continuing to fund the actual drug costs. However, this scheme closed on 1 November last year to all patients diagnosed after that date.
Clinical trials have shown significant improvement in life expectancy and motor function. Just last week, a three-year study of 28 children aged between two and 15, which was published in the scientific journal Neurology, highlighted meaningful improvements that extended over time. Some of these improvements were remarkably different to the natural progression of the condition when left untreated. In particular, a two-year-old child with SMA Type 2 gained the ability to walk independently after receiving Spinraza, while two children with SMA Type 3 who had lost the ability to walk before taking the drug regained it during the study.
You can help us in keeping the pressure on by:
- Writing to your local MP and asking for their support. We have written a template letter you can use and a handout that you can send which includes more background about the fight for access. You can find your MP’s contact details on the Parliament website.
- Tweeting your MP with a link to our video of Zac and his family talking about the difference Spinraza has made to their lives.
For more information
- 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: email@example.com
- To find out how you can join our campaign, email Clare Lucas at: firstname.lastname@example.org. And if you would like to share your story, email Laura Burge at: email@example.com
- You can also find our factsheet on SMA here and more information on Spinraza here.