Dáil Éireann Debate, Tuesday - 7 July 2020
728. Deputy Fergus O'Dowd asked the Minister for Health the position regarding the availability of Spinraza to children with spinal muscular atrophy type I, II or III on an exceptional and individualised basis; his plans to facilitate broader provision of the scheme; and if he will make a statement on the matter. [14012/20]
Amharc ar fhreagraThe 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.
To date, the total number of patients deemed eligible to receive Spinraza is thirty five. Children's Health Ireland (CHI) has confirmed that 18 patients are currently receiving Spinraza. Due to Covid-19, a number of patients who were due to commence treatment have been put on hold.
Individual applications for each patient are prioritised based on clinical need and a treatment plan and a date for administering the drug is agreed with their treating clinician.
The actual delivery of this drug to approved patients in a safe and sustainable way requires very specific and quite complex service arrangements to be put in place in CHI across all sites.
CHI has confirmed that in order to support the SMA service arrangements, a clinical nurse specialist has been appointed who will liaise with families and is the first point of contact for care for patients. A physiotherapist has also been appointed to support these patients ongoing care.
CHI is in continuous engagement process with families regarding their child's individual plan for the administration of Spinraza.
