Paul Kehoe (Wexford, Fine Gael) | Oireachtas source

I thank the Senator for raising this very important issue and for giving me, on behalf of the Minister for Health, the opportunity to clarify the position on the availability of Spinraza for patients with spinal muscular atrophy. The Minister for Health appreciates the desire of families to see their children who suffer from this debilitating and painful condition begin treatment with this medicine.As the Senator will be aware, the HSE has statutory responsibility for medicine pricing and reimbursement decisions under the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies criteria for decisions on the reimbursement of medicines. The Minister for Health has no role in this statutory process.

On 11 June 2019, the HSE leadership team approved access to the drug Spinraza for children with spinal muscular atrophy, SMA, type I, II or III on an exceptional and individualised basis. The HSE decision process for 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 HSE drugs group. Evidence of the clinical effectiveness of this new drug therapy was also reviewed. After a thorough review, 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 11 children have been approved for Spinraza treatment in Temple Street Hospital since July. 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 who have been approved for Spinraza treatment are being treated under special care, according to Children's Health Ireland, at Temple Street Hospital. Detailed clinical guidelines have been developed for this service, which includes specific patient criteria that 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 specific and 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 patient for the administration of Spinraza. Category 1 is where the administration of Spinraza is deemed not too technically difficult. In that case, the consultant paediatric neurologist and lead clinician need access to the day ward as administration is performed under local anaesthetic. Each of these patients will also require a physiotherapy assessment prior to administration and recovery time in the day ward. Category 2 cases are 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 Senator will appreciate, therefore, that a considerable amount of service planning and assessment of patients at an individual level is required for treatment with Spinraza to be safely undertaken. The Minister for Health has been advised by the HSE that Children's Health Ireland is engaging on an individual basis with the families involved regarding each child's treatment plan. He has asked to be updated further as soon as possible.