Richard Bruton (Dublin Bay North, Fine Gael)
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202. To ask the Minister for Health if spinraza has been evaluated for funding; and if he will make a statement on the matter. [17698/20]

Stephen Donnelly (Wicklow, Fianna Fail)
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The HSE has statutory responsibility for medicine pricing and reimbursement decisions, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.

On 11 June 2019, the HSE Leadership Team approved access to the drug Nusinersen (Spinraza) for children with Spinal Muscular Atrophy (SMA) Type I, II or III on an exceptional and individualised basis.

The HSE decision process in relation to Spinraza involved a full Health Technology Assessment followed by detailed consideration by the HSE expert groups on new drug therapies, including the Technology Review Group for Rare Diseases and the Drugs Committee. Evidence of the clinical and cost effectiveness of this new drug therapy was also reviewed.

The actual delivery of this medicine to approved patients in a safe and sustainable way requires very specific and quite complex service arrangements to be put in place by Children’s Health Ireland (CHI) across all sites.

It is estimated that 1-3 new children will be diagnosed with SMA Type 2 in Ireland each year. CHI has confirmed that there are currently 35 patients with SMA aged 18 years or under who are deemed clinically eligible for treatment with Spinraza by the Paediatric Neurologist in CHI. CHI has further confirmed that 19 patients are currently receiving Spinraza.

However, other patients who are waiting to start treatment have been put on hold due to COVID-19. CHI is currently working through the remaining patients and how they can be accommodated in the midst of COVID-19 restrictions around social distancing.

CHI has confirmed that they have identified a small number of SMA patients whose cases are extremely complex as they have poor lung function. The delivery of the drug to these patients will be potentially more complex and technically difficult and will require complex coordination by CHI.

Children’s Health Ireland is in a continuous engagement process with families regarding their child’s individual treatment plan for the administration of Spinraza as well as representatives of the SMA Executive and Advocacy Group.

I wish to advise the Deputy that funding was provided in 2020 to develop the Spinraza service, including the recruitment of additional staff. A nursing coordinator, who will coordinate the services including communication with families, has been appointed and is commencing the end of July.