22 thoughts on “Petition for Zolgensma in Ireland”

  1. 100% support this. We deserve to not have to fight for this!
    Theo is beautiful. The very best wishes for the future

  2. Email I’ve sent to the cpu@hse.ie who are doing the pricing negotiations.


    Hope you are well.

    I was disappointed to not receive a reply from my original email to this address on the 13th of Sept from this team.

    I will need to talk with someone at the Corporate Pharmaceutical Unit about Zolgensma gene therapy reimbursement in Ireland. What is the current status of this ? Why are families being left to wait without any indication of status for a therapy that will change their lives ?

    In the Netherlands, a number of children have already been dosed with this drug this year after the HTA (health technology assessment Ireland was involved with was finalized). This therapy in the Netherlands is provided even before pricing negotiations are finalized as an early access program. This is the humane and logical thing to do as this new therapy is inevitably the best drug available with the best prognosis for SMA type 1 infants.

    I’m under the impression after speaking to a financial consultant in the UK that reviewed the HTA conducted by the NCPE that there are unfortunately some questionable assumptions, particularly in the financial calculations. I do understand on the NCPE’s side they handed over this work to the HSE to find out a suitable price as the therapy is no doubt expensive. My question is why is there a price on the life of a child ? Surely the drug should be given if it works better. The HTA reads to me, “yes it’s a better therapy as it’s once off but can we negotiate a better price”.

    The infants with the most severe form will undoubtedly have a better quality of life from this treatment than they will with Nusinersen due to the mechanism of action directly replacing a gene in SMA types that do not have a lot of backup SMN2 genes (targeted by Nusinersen). This is proven in the clinical data over long term studies ie. START LTFU.

    We are talking about a drug that will prevent our children with severe SMA to be permanently ventilated and to avoid death. This is the reality of the situation sitting here in Temple street hospital currently with Theo that had an acute life threatening incident at less than 5 months of age. Zolgensma acts in a systemic nature and has a perceived better ability (inevitably as delivered as a viral vector) that helps to avoid any further deterioration in swallow or breathing muscles in severe SMA. The US, England, Germany, Italy, Netherlands and other countries have made this drug available and with good reason. Ireland will follow suit inevitably, but time is of the essence in these 2 children’s cases, Kate from Dunmanwya and Theo from Killorglin.

    I trust you understand this has been raised directly to the Taoiseach this week passed in the DAIL by more than 3 TD’s and Sean Kelly, member of the European parliament is aware of the need for this process to be sped up on compassionate grounds alone if negotiations are not finalized.

    We want this treatment provided to the two children in Ireland with SMA under the age of 2 to receive the treatment as soon as possible. The clinicians here in the hospital are of the same opinion and I have spoken to them. Public opinion also is supportive, we’ve flown past 5000 signatures this morning https://www.change.org/p/irish-government-approve-zolgensma-miracle-treatment-in-ireland-for-sma-type-1. Politicians also are of the same opinion as I mentioned above.

    There are reasons why these children need it urgently:
    1. Has to be administered under a certain weight range normally around 24 months old. This is because the more weight the larger the viral dose administered.
    2. The more time wasted the more likely it is Theo and Kate could come in contact with Adenovirus and develop antibodies against the treatment.

    Please reply as soon as possible so we can arrange a meeting to discuss the status.


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