Duchenne muscular dystrophy access
Deputies Crowe, Coppinger and Moynihan urged early access to Duchenne muscular dystrophy medicines, arguing families need urgent action and that a shared-island approach could help. The Tánaiste said he would raise the issue on North-South Council margins, sought an urgent HSE update, and noted increased funding and staff for new medicines, while agreeing the system must be more agile.
I wish to ask about Duchenne muscular dystrophy. We recently had young people in the AV room who talked about the challenges they face. It is a wasting disease. There is a drug but it is not available here yet. It is available in the North. Would the Government look at early access as a shared island? The problem in the North is that it does not have the money to administer the scheme. The problem here is we do not have access to the drug. It seems logical to move ahead on that. I am aware of the Tánaiste's work as regards nurses in the past. Would he be open to looking at this as a possibility for the children on the island of Ireland?
Comment on this
I thank Deputy Crowe for his very constructive suggestion. Tomorrow, we will have a meeting of the North-South Ministerial Council, which will be an opportunity for colleagues across the island of Ireland to get together. I will raise the issue of DMD on the meeting's margins. If it is possible for professionals throughout the island to work together in the interests of patients with a rare disease, I am sure that is something everyone would like to see happen. We need to do more regarding rare diseases and quick and timely access to medications. The Minister for Health is very committed to this. There will be a new strategy in respect of rare diseases shortly also. The HSE has launched an application tracker to increase transparency in the medicines assessment process. I will look into this issue and revert to the Deputy.
Comment on this
On that same issue, Duchenne muscular dystrophy is a muscle-wasting progressive illness that normally starts affecting boys aged between two to three years. It is a life-altering condition. One parent said her four-year-old could be knocked down by gust of wind. The condition is progressive. Walking can sometimes become difficult and the patient can become a wheelchair user. The disorder has no cure and needs to be acted on extremely quickly. Givinostat is a drug that has been shown in other trial areas to be extremely effective. Parents are desperate to get access to this drug. The EU approved the drug and fast-tracked it on 5 June. The Irish State needs to do the same. The National Centre for Pharmacoeconomics needs to give approval to the drug. The difficulty is that drugs can take up to two years to be approved in Ireland. This is largely due to understaffing issues. The UK has fast-tracked and approved the drug. This is vital. Parents have marched and have been outside due to this issue. The State must do all it can to approve this drug so that these young boys do not have their conditions deteriorate and do not wait any longer.
Comment on this
I agree with a lot of what the Deputy said. The idea that any parent has to watch his or her young child in such a fragile state when the parent knows there is medication available that could significantly improve the child's quality of life and health is not right. The HSE is engaging on this matter. In light of it being raised in the House today by a number of Deputies, I will seek an urgent update as well.
On the issue of staffing, the Deputy is right. We have had to increase the number of staff allocated to the assessment process. My understanding is there are 34 additional staff being hired across the pricing and reimbursement process. I will get an urgent update and revert to the Deputies concerned.