Tuesday, 14 July 2020

Ceisteanna (1071, 1096, 1154, 1223)

Joan Collins


1071. Deputy Joan Collins asked the Minister for Health if the Spinraza treatment has been stopped for persons (details supplied); and when the treatment will commence [14951/20]

Amharc ar fhreagra

Gerald Nash


1096. Deputy Ged Nash asked the Minister for Health the reason 12 children with SMA are still waiting to be treated with Spinraza one year after the drug was approved; the reason for the delay; when the matter will finally be addressed by the HSE; and if he will make a statement on the matter. [15076/20]

Amharc ar fhreagra

Gino Kenny


1154. Deputy Gino Kenny asked the Minister for Health if his attention has been drawn to the number of children with spinal muscular atrophy eligible for treatment with the approved drug Spinraza; the number of those receiving the treatment; the reason for the delay in treatment for some of these children such as a child (details supplied); the way in which and when these issues will be addressed in order that all children can receive this vital treatment; and if he will make a statement on the matter. [15427/20]

Amharc ar fhreagra

Patricia Ryan


1223. Deputy Patricia Ryan asked the Minister for Health when Spinraza will be made available for children with spinal muscular atrophy; and if he will make a statement on the matter. [15570/20]

Amharc ar fhreagra

Freagraí scríofa (Ceist ar Health)

I propose to take Questions Nos. 1071, 1096, 1154 and 1223 together.

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 effectiveness of this new drug therapy was also reviewed.

The recommendation is to approve access for children with genetically confirmed SMA Type I, II or III, in accordance with the controlled access criteria recommended by the Rare Diseases Technology Review Committee. The rare diseases committee recommendation was clearly targeted at the youngest and most severely affected SMA patients, and this group is the clear priority for the HSE. The actual patient assessment and approval process will be the means for determining access on an individual case by case basis.

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 34 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 18 patients are currently receiving Spinraza.

However, other patients who were waiting to start treatment have been put on hold due to COVID-19. CHI has confirmed that a Clinical Nurse Manager is commencing at the end of July, and that they are currently working through the remaining patients and how they can be accommodated in the midst of COVID-19 restrictions around social distancing. Individual treatment plans are being worked up for each of the remaining patients.

CHI has confirmed that it is in a continuous engagement process with families regarding their child's individual plan for the administration of Spinraza, as well as representatives of SMA Executive and Advocacy Group.