People with spinal muscular atrophy (SMA) and their families were today demonstrating in a bid to see a drug made available on the NHS.
SMA, the number one genetic killer of babies and infants, is a progressive disease of motor neurones that causes early death of nearly 50 UK children a year and leaves hundreds more progressively losing muscle function ending in permanent paralysis.
Today the National Institute of Health and Care Excellence (NICE) was due to meet for a third time to discuss the drug. Previous meetings have resulted in a decision not to fund the drug, Nusinersen, through the NHS, as it was not deemed cost effective.
However according to campaign group TreatSMA the decision caused despair and outrage within the SMA community, forcing hundreds of families to join forces via social media to have their voices heard.
The group says that Nusinersen, which was approved as a breakthrough drug in the US in December 2016 and in the European Union in May 2017, is the only existing treatment that brings about a meaningful change in the lives of those affected by SMA. Developed by Ionis Pharmaceuticals and marketed by Biogen as Spinraza, it is availbale in 43 countries including Scotland.
Campaigners hope their demonstation and vigil for those who have lost their lives waiting for the drug, will make NICE change its mind and make the drug available in England and Wales.
Among the demonstrators is Marni Smyth from Marsden who was diagnosed with SMA when she was just 18 months old.
Marni was feature in The Yorkshire Post when she launched a crowdfunding appeal to raise £22,000 to replace her old electric wheelchair.
She has since hit her target and has the new wheelchair which she says is helping make life a little easier. But she believes Spinraza could help improve her quality of life further.
“Spinraza is most effective when given early when babies are first diagnosed, but it can also help adults like me,” says Marni.
“SMA is a deteriorating condition and Spiranza could give me back some of the movement I have lost and would also reduce the risk of me getting chest infections which is my biggest worry. NICE has been debating this for two years, and time is running out for so many people. I don’t see how they can put a cost on saving a life.”
TreatSMA has continuously advocated that the treatment must be made available for all those with SMA regardless of age, genetic make-up, disease ‘type’ or other arbitrary criteria.
“We, the community, are not prepared to see our lives and the lives of our children taken away from us. Several academic publications confirm that Nusinersen has been proven an effective treatment even in the weakest of patients and across the entire spectrum of the disorder.
“We have closely followed the progress of clinical trials and the subsequent clinical practice and seen babies living and thriving worldwide whilst here in the UK children have been dying.”
Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: “When the committee discussed Spinraza prior to agreeing the previous draft recommendations last August, it noted that the evidence showed a substantial benefit for the treatment, but felt there were significant uncertainties, particularly around its long-term benefits.
“Since then we and NHS England have been discussing with Biogen how they might address the uncertainties identified by the committee, while demonstrating the potential for Spinraza to be considered cost effective and managing the risk to the NHS of allowing access to this treatment.
“It has taken us longer than we would have liked to get to the position we’re in today where our independent committee is able to discuss a revised submission from Biogen, alongside the feedback we received during public consultation on our draft recommendations.
“We recognise that this continuing uncertainty has been concerning for the community but we felt it was important to give all parties the opportunity to get to a point where we could have a meaningful discussion about Spinraza and what it would take to make it available to patients in England.
“We will do our best to ensure that the committee’s decision is made available as quickly as possible.”