Rose Conway Walsh (Sinn Fein)
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I thank the Minister of State for coming to the House. He will know that I had requested the Minister for Health, Deputy Harris, to do so, but I appreciate the Minister of State's presence. I hope the Minister has communicated with him on the issue of Spinraza. The Minister of State will know that we have been fighting for Spinraza to be made available for the past two years. We were absolutely thrilled when Spinraza was approved on 11 June. We accepted that four to six weeks, or however long necessary, would be required to do the paperwork in order to have the drug administered. Then, on 23 August, there was a sign-off on it. At that point, the parents of the children who need Spinraza were told that everything would be put in place and that they could expect the drug to be administered at the end of October.

The end of October is approaching and the parents have been given another update stating that, due to resource issues, together with bed shortages, the drug that has been approved and that is necessary cannot be administered. I look at these children, and their parents look at them, and they tell me that this is almost worse than the drug not being approved. This pertains to Mayo in particular because we have Grace O'Malley and her friends and Cillian Mearns, who have done work on this. We need to know when the drug will be administered. It was cruel in the first place for them to have to wait for so long for Spinraza to be approved, but there is an additional cruelty in knowing that it has been approved and is there. The drug has been purchased, and now the HSE tells us it does not have the resources to have it administered. We are not talking about a staffed bed for a long period. The Minister of State will know that Spinraza can be administered to day patients, so all we need is a bed for the day, or even half the day, to get the drug administered. That, we are told, is not available. Over the first two months the drug is administered four times and then the dosage decreases. We are talking about just 11 patients who are ready and suitable for this treatment. I hope the Minister of State can give me very specific answers today as to when these children, including Grace and Cillian, will get a specific date to start on Spinraza.

Paul Kehoe (Wexford, Fine Gael)
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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.

Rose Conway Walsh (Sinn Fein)
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I do not take heart from the statement as it outlined what I already know. The issue is one of resources and the Minister for Health has not given a date for the administration of Spinraza. While the Minister does not have a role in the workings of the HSE, he has a role in ensuring that drugs that are approved are then administered. We need a date for these children notwithstanding the complexities the Minister of State outlined.

It is not true that all of the patients who have been approved for Spinraza treatment are being treated under special care because they are not. The Minister of State referred to spinal rods and what is already being done. I appreciate the complexity involved in those cases but children who have not had spinal rods inserted have not been given the treatment either. I ask that the Minister take up this matter and ring-fence resources available to Temple Street Hospital in order that these 11 children can get the drug. After all, it has been purchased and is available and the consultants are ready to do the work. They need the resources to do so. I ask for a date. I am not going to let this matter go. Grace is just ten years old and I will not repeat what she is asking but we owe her answers.

Paul Kehoe (Wexford, Fine Gael)
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The Senator feels very strongly about this matter. The Minister for Health fully recognises that this condition places enormous strain on SMA sufferers and their families and carers and that this is a very worrying time for them.

The complexity of this treatment requires appropriate service arrangements to be put in place in Temple Street Hospital so as to ensure that the treatment and delivery of this medicine are conducted in a safe and sustainable manner appropriate to the standard of care which has been developed for this particular treatment. While the Minister hopes that access to this treatment will be provided shortly for all concerned, it should be remembered that the care of individual patients is a matter for treating clinicians in line with patient safety, protocols and guidelines. These are being developed by the HSE to administer Spinraza. As I said, the Minister hopes to receive a further update on this issue as soon as possible.

This is a clearly a major issue for the Senator and I know she is personally involved and has a personal connection. I do not have answers to hand because I do not have responsibility for this area. However, I will ask the Minister or one of his officials to communicate with her directly and address the specific concerns she raised. I admire the Senator for the manner in which she has raised this issue on behalf of patients who are awaiting treatment with Spinraza.