Today in the Seanad, Senator Diarmuid Wilson and I persisted in our long quest for Translarna approval. We expressed to Minister Catherine Byrne the urgency for the five boys with Duchenne Muscular Dystrophy. They need the drug before their muscles deteriorate further and ultimately stop working.
The ending of legal proceedings between the HSE and the drugs company, PTC Therapeutics, offers a real chance to provide Translarna without delay. We now have a window to process a fresh application to have the drug covered by the HSE.
What is vital, is that any new application take account of evidence and submissions already made in similar applications. The last application took 2-3 years. Any fresh application must be processed without delays.
The HSE needs to negotiate the price of Translarna with PTC. I cannot understand how any meaningful cost benefit analysis could have been carried out when the HSE did not seek to negotiate the price in the first place.
I am now requesting that Minister Harris gets directly involved in instructing the HSE to outline the clear pathway and timeline in processing the application once it is received from PTC.
The five children who are eligible for Translarna to effectively treat their condition cannot afford to wait any longer. Every day that goes by brings each of these youngsters one step closer to losing their mobility. I hope that we will see a positive outcome this time round for these children and their families.
