I add to the calls for the availability of treatments for Friedreich's ataxia and Duchenne muscular dystrophy and to recognise the very brave patients and their advocates who have made this case in Leinster House. Skyclarys is the first and only approved treatment for Friedreich's ataxia which around 200 people live with in Ireland. This medication can slow the progression of their condition. The company that makes Skyclarys has put the official pricing proposal to the CPU and now the situation is fully in the hands of the HSE. Patients do not know how long it will take for the HSE to make its final decision. On 26 May, I asked the Minister for Health for a definitive timeline on consideration of Skyclarys for reimbursement. Many patients and their advocates were really upset when it became apparent Skyclarys was not discussed at the 10 June meeting of the HSE's corporate pharmaceutical unit. It is important this process is completed as quickly as possible. I ask the Minister to do all she can to ensure Skyclarys is included on the agenda of the August meeting of the CPU. I would also like to raise the approval of givinostat for people suffering with Duchenne muscular dystrophy. I have raised it as have others on several occasions with the Minister for Health since EMA approval was granted last year. Last week the HSE recommended the drug for approval. This week the SLT signed off on this, bringing children with Duchenne closer to accessing this medication in Ireland. The Minister said the timeline for children getting this medication will depend on supply and the final approval steps. I urge her to use all possible influence to speed up this process so that children who desperately need this medication can avail of it. The approval of this treatment is welcome but it should not have taken this level of concerted lobbying by parents and patients to achieve. The Minister says decisions around pricing are not made by her personally and they are rightly in the hands of experts. That is the right approach but the issue is not with the making of the decision, it is the process, the time it takes and a system that has become overly bureaucratised and needs to be streamlined. A review of that system is going on at the moment. That review is timely and I hope we get the result quickly. I hope we get the changes that are necessary made to the system of reviewing approvals for these orphan drugs because patients and their families deserve better.

Sentiment score: 0.21