The Department of Health has received correspondence from this individual and an acknowledgement of its receipt was issued on 03 April 2019.
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 Nusinersen involved a full Health Technology Assessment followed by detailed consideration by the HSE expert groups on new drug therapies, including the HSE Drugs Group and the Rare Diseases Technology Review Committee. Evidence of the clinical effectiveness of this new drug therapy was also reviewed.
After a review of all the data it was decided 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 actual patient assessment and approval process is the means for determining access, on an individual case-by-case basis, to this complex treatment.