SMA Ireland are planning a Day of Action at Dáil Éireann on Thursday 28th February, World Rare Disease Day. Meeting at 11am at the intersection of Molesworth Street and Frederick Street, SMA families will march to the Dáil to present a petition, now totalling nearly 100 thousand signatories, asking the government to fund the Spinraza drug treatment for SMA sufferers immediately. Minister Simon Harris has been asked to receive the petition which is addressed to him but he has not yet responded.
This protest is at the recent refusal by the HSE to fund Spinraza, the only approved treatment for the rare degenerative condition SMA. Without treatment the prognosis for many SMA patients is deterioration and early death. A meeting will be held to plan future action afterwards.
SMA & Spinraza – First EVER Treatment
SMA is somewhat different to most other conditions in that there has never before been any treatment for SMA. Spinraza is the first and only treatment available. There are competitor drugs and treatments in the pipeline, which seem to look promising, but they could take years for approval and face the same funding battle. In the meantime people with SMA must count the cost.
Spinraza is now available in every other EU country with the exception of Estonia and the UK. It is worth noting that Scotland which has its own health system has started to fund Spinraza treatment for ALL people with SMA.
The terrible toll of SMA
60% of SMA sufferers are diagnosed as Type 1, the most severe type, and 95% of those untreated die before age 2. With Spinraza treatment available elsewhere and through the original trials, not only are many of these children surviving and thriving, but some are even walking. This is unheard of for Type 1 SMA.
Video of Spinraza Treated Type 1 Walking: https://www.youtube.com/watch?v=3EKs8GJnkrc
The prognosis for most Type 2 SMA sufferers is to never walk and rarely live beyond their early 20s and often in a very weakened state as this degenerative condition takes its toll.
Video of Spinraza Treated Type 2 Walking: https://www.youtube.com/watch?v=PGbVbg-YejA
Those with Type 3 can often look forward to losing the ability to walk and be self sufficient. As well as the many other consequences attached to muscle degeneration including scoliosis and poor balance leading to falls.
White Shirt Protest & Minute’s Silence for those lost to SMA.
SMA Ireland would ask anyone who can, to come and support SMA sufferers, and to wear a White T-Shirt or Shirt as a memorial of all those who are missing from this demonstration because they had the misfortune to be born at a time when no treatment existed (more than 60% of those diagnosed historically). How cruel it would be to watch more white shirts be added in future when we finally have a means to prevent it. There will also be a minutes silence held at the Dáil for all those lost to SMA and their families.
Initial Rejection and Review
The drug was initially rejected for funding by the HSE in early 2018 and since protests at Leinster House by parents last September has been going through the Rare Disease Technology Review Committee process. This process has been ongoing with Patient Impact submission being a part of that process. On Friday the HSE communicated to families that it would not reimburse Spinraza at the offered rate.
Biogen made the following statement on Monday to the Irish Times “Biogen provided the HSE with a significant pricing proposal,” the company said in a statement, “absolutely in line with the final price negotiated in countries aligned with Ireland in the Beneluxa initiative, which have each decided to reimburse Spinraza.”
Speaking for SMA Ireland Anton Mannering said ”According to the OECD Ireland has the second highest per capita GDP in the world and the highest GDP per employed person. How can it be, that as one of the wealthiest nations on the planet, we can’t afford something that 20+ EU nations ranked below us can?”
“It is clear enough that it’s not a matter of affordability. So it must be either a philosophical reason of some sort or a management issue. Unless we’re going to say that every other EU country is completely unreasonable we must conclude that, unlike their EU counterparts. Either the Irish Government and the HSE don’t think people with SMA are worth saving or they have processes that are deeply broken. Either of these things is utterly unacceptable”
Does someone have to die?
Mr Mannering added, “It is a matter of open discussion in fearful tone amongst people with children and family that have SMA across this country. Does someone have to die? Does someone have to die, that could have lived and thrived with treatment, in order for the Government to face enough political pressure to fund this drug? Is that the type of government we have? That only the horrific, unnecessary and very predictable death of a vulnerable person, with all the pain associated for families and communities, can gain enough political capital to provoke some ordinary humanity.”