Today, (Wednesday, 12th June 2019), the SMA Ireland Executive welcome the announcement that the HSE/Department of Health have agreed to grant access for the drug Nusinersen (Spinraza) on an exceptional and individualised basis for people under 18 years of age with Spinal Muscular Atrophy (SMA) Type I, II or III. We now call on the HSE to roll out Nusinersen, with implementation plans, timeline and responibilities, as a matter of urgency in the most efficient way so that children with SMA don’t suffer any longer.
For the 25 children in Ireland with SMA, this is of course very welcome news following months of uncertainty and worry. However for the wider SMA community it feels like a hollow victory, as the small number of adults with SMA have been let down by this short-sighted decision.
Whilst we accept that there must be some criteria for access to this life changing drug, having an arbitrary age cap is a crude method with little scientific support. We will not stop campaigning until access to Spinraza is made available for all sufferers of SMA in Ireland, as it is in so many other countries across Europe.
It is estimated that there are as many as 25 adults living with SMA in Ireland.
Concerns over basis for exclusion of Adults
The HSE state they have relied on clinical trial data to make this decision. Obviously this is a valid and welcome criteria for assessing treatment efficacy. The use of this data to justify the exclusion of adults, however, is not supported by the data. We find it unlikely that the Spinraza treatment ceases to be effective when people become eligible to vote.
Formal adult trials for Spinraza have only started and will not be completed until 2022/2023 at the earliest. If it is the HSE position that treatment cannot be considered without this data, this may be too late for some of our adults with SMA.
SMA Ireland is concerned that real world experiences of adult treatment in other countries were not properly considered. Spinraza is now an accepted treatment available to adults in multiple countries in Europe and the world. Efficacy in adults can be assessed from clinician and patient testimony available right now. SMA Ireland is currently working with our international counterparts on collating this data and will share it with the HSE and Public Representatives in the coming weeks and months as it becomes available.
SMA Ireland is also exploring other options internationally to ensure Irish patients do not suffer due to HSE/Government short sightedness.
Effects of arbitrary age cutoff on vulnerable adults
Anton Mannering Chairman of SMA Ireland said “It is simply not good enough that up to half the people with SMA will not have access to treatment. There are several young people with SMA who are in their late teens and a few who are in their early twenties. If treatment will be given to a girl who is 17 continuing til later in life, perhaps to her 40s or beyond, how can it be justifiable to not treat someone only a year or two older? It is intolerably cruel and discriminatory to inflict that type of injustice on a person suffering from a degenerative disease. Especially when the only difference between a treated an untreated individual might be which side of voting eligibility they might fall.”
He continued “That is without considering those weaker older adults now starting to experience difficulty chewing and swallowing as this condition robs them of function. As I have said before, does somebody have to die before sense is seen? As a nation we must be better than this. We must do better than this.”
SMA Ireland welcome the HSEs comments regarding funding for treatment in future years and we urge the Minister, his Department and the Government to adjust their budgetary priorities to accommodate treatment for ALL SMA patients.
SMA Ireland remains open to dialogue with all parties to ensure that up to 50% of Irelands SMA population do not remain untreated without reasonable justification.
Anton Mannering, Chairman, SMA Ireland
p: +3531442 9884