Anne Rabbitte (Galway East, Fianna Fail) | Oireachtas source

I thank the Deputy for raising this matter tonight. I am taking this debate on behalf of the Minister, Deputy Donnelly. I do not know what context the Deputy has or how much he knows, so I will read out the script that has been prepared but I do so while very conscious that there are two families watching us tonight, those of the parents of Kate and Theo, whom the Deputy is representing in raising this Topical Issue matter.

I thank the Deputy for raising this important matter regarding the availability of, and reimbursement for, Zolgensma in Ireland. As the Deputy is aware, the HSE has been given statutory responsibility for medicine pricing and reimbursement decisions under the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies certain criteria for deciding whether the State will reimburse the cost of medicines. These include the health needs of the public, the cost-effectiveness of the drug and total budget impact of the drug. HSE decisions on which medicines are reimbursed by the taxpayer are facilitated by the advice of the National Centre for Pharmacoeconomics, NCPE.

In May 2020, EU marketing authorisation was granted to the medicine Zolgensma for treating spinal muscular atrophy, SMA. The Minister for Health is advised by the HSE that, in April 2020, the NCPE received a reimbursement application for Zolgensma for the treatment of patients with a specific SMA diagnosis. In May 2020, the NCPE completed a rapid review for this application and recommended that a full health technology assessment, HTA, be completed to assess the clinical effectiveness and cost-effectiveness of Zolgensma compared with the current standard of care. The HTA was undertaken as a part of the Beneluxa collaboration between Ireland, the Netherlands and Belgium, with Austria acting as a reviewer in the Belgian procedure. This initiative represents a very positive step in addressing the funding challenges posed by new drugs with very high budget impacts, which are shared by all Beneluxa members.

The joint Beneluxa HTA was completed in May 2021 and it recommended that Zolgensma should not be considered for reimbursement unless cost-effectiveness could be improved relative to existing treatments. Pricing and reimbursement negotiations commenced in July 2021 and are currently ongoing. The purpose of challenging pharmaceutical companies' pricing, through negotiations, is to arrive at a position of value for money. In doing so, we can be confident that public resources are used as effectively as possible to reimburse as many medicines as possible and for as many patients as possible.

The Minister fully appreciates that families want to see their children who suffer from SMA get access to new treatments as soon as possible. However, the Minister for Health has no role in the statutory process under the 2013 Act. The Government's allocation of €50 million for new medicines in budget 2021 has enabled the HSE to approve 32 new medicines or expanded uses of existing medicines to date this year. This has included 12 medicines for the treatment of rare diseases. The Minister nonetheless fully understands that this is a worrying time for the families concerned and is hopeful that the HSE can arrive at a positive outcome with respect to Zolgensma.

I am very conscious that this may not be the answer the Deputy would have wished to hear this evening but, at the same time, it is positive that conversations are still ongoing and that no doors whatsoever have been closed. He can see how the €50 million announced in last year's budget has been spent.

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