Wednesday, 23 October 2019

Questions (136)

Paul Kehoe

Question:

136. Deputy Paul Kehoe asked the Minister for Health when a medication (details supplied) will be made available; and if he will make a statement on the matter. [43679/19]

View answer

Written answers (Question to Health)

The HSE is the decision-making body on the reimbursement of medicines under 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 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 thorough 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.

The HSE has indicated that since July, 11 children have been approved for Spinraza treatment in Temple Street Hospital.  Application for approval is based on clearly defined criteria and clinical guidelines being followed in each case.  All patients who have been recommended clinically have been approved to date.

All of the patients which have been approved for Spinraza treatment are being treated under special care under Children’s Health Ireland at Temple Street Hospital.  Detailed clinical guidelines have been developed  for this service which include specific patient criteria which must be fulfilled  in order for the treatment to safely commence and continue. 

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 Temple Street Hospital.

Spinraza is injected during a procedure known as lumbar puncture. There are two categories of patients with regard to the administration of Spinraza.  For category 1, where the administration of Spinraza is deemed not too technically difficult, the Consultant Paediatric Neurologist and Lead Clinician need access to the day ward as administration is performed under local anaesthetic.  Each of these patient will also require a physiotherapy assessment pre- administration and recovery time in the Day Ward.

Category 2 are the more complex patients who have already undergone spinal fusion surgery and insertion of spinal rods, which makes the administering of Spinraza potentially more complex.  For these patients, the delivery of Spinraza will require specialist care to include input by an Anaesthetist and an Interventional Radiologist with associated nursing and the availability of capacity in the Paediatric Intensive Care Unit.

The Deputy will appreciate therefore that a considerable amount of service planning and assessment of patients at an individual level is required in order for treatment with Spinraza to be safely undertaken.

I have been advised by the HSE  that Children’s Health Ireland  is currently engaging on an individual basis with the families  involved regarding each child’s treatment plan.  I have asked the HSE to keep me updated on this matter.