First ever treatment for life-devastating condition is approved by European Commission!
Yet the UK children still don’t even have access to compassionate programme.
Spinal muscular atrophy (SMA) is a neuromuscular, debilitating and life-limiting genetic condition and is one of the most common rare diseases with 1 in 40 people being a carrier of faulty gene. The actual occurrence between of SMA is 1 in 6–10 thousands live births and is the leading genetic cause of infant mortality with 95% of children diagnosed with type 1 SMA expected to pass away before their second birthday. Children diagnosed with type 2 SMA have reduced life span and progressively become weaker, losing various physical abilities as muscles deteriorate. People with late onset of disease, consistent with type 3 and 4 have normal lifespans, but the disease has dramatic impact on the quality of life. Further information is available on our website www.treatsma.uk.
Since it was first described over a hundred years ago, the disease had no treatment. Today European Commission has approved the first ever treatment, nusinersen (marketed by Biogen Pharmaceuticals as Spinraza) for this rare and devastating condition for people with ALL types of the disease. This first ever treatment for SMA to make to the market has shown such excellent clinical results that it was expedited through the process to become available as soon as possible.
Whilst EC has granted broad licence, each country has own review process and in the UK it could take up to 2 further years before the drug is approved for use on NHS. This time scale means that many lives will be lost in the process and many other lives will be severely damaged whilst patients wait for a decision to be made.
The urgency of this treatment is so high that Biogen decided to open an Expanded Access Programme which enables ALL children with SMA Type 1 diagnosis to access the treatment before approval process is completed. Furthermore, Biogen decided to provide the drug free of charge to all of these children for the duration of the approval process. NHS was expected to pick up the costs of administration. This program became available in September 2016. To this day only 17 children out of estimated 100 are on the program. NHS has refused to provide resources needed to ensure that all legible children benefit from the program and as a direct result of this a number of children have passed away, a further significant number of children continue to deteriorate and is in mortal danger.
In proactive trusts with larger resources EAP is funded directly by the Trust. However, these hospitals will only treat children within their attachment area thereby restricting the numbers and leave other, less fortunate families to watch their loved ones to continue deteriorating. This is a clear example of post-code lottery and goes against the principles on which NHS has been founded. Furthermore, EAP is available only to patients with Type 1 SMA, leaving other vulnerable children to continue losing physical abilities.
We request that Health Secretary requests an urgent review of the drug by NICE to ensure that a broadest possible approvals are granted to give everybody opportunity to access the drug.
We request that NICE exposures the process and takes into accounts that FDA and EMA have granted broad label which covers the whole spectrum of SMA.
We request that NHS and Biogen negotiate now in order to remove all the barriers to EAP and help our vulnerable children to get a treatment now in the whole of the country and not just in few selected hospitals.
We call to all NHS Trust to step up and talk to Biogen in order to identify what is needed to ensure that their hospitals are able to provide much needed EAP.
We ask the people of Great Britain to show solidarity with us in this cause.