Dara Calleary (Mayo, Fianna Fail) | Oireachtas source

Today is world Rare Disease Day, which is a day to raise awareness among the public and decision makers about the impact of rare diseases on people's lives. On 20 September last, more than six months ago, I raised the issue of spinal muscular atrophy, SMA, with the Tánaiste and the need for the HSE to improve the reimbursement for the drug, Spinraza. This has been raised on a weekly basis by many Deputies across the House since. As we stated, SMA is one of the most horrific diseases imaginable.

We had a meeting this morning, hosted by Deputy Ó Caoláin, in the audiovisual, AV, room where it would have broken a stone heart to hear the stories from the families and from those with the condition. SMA debilitates lives and until the arrival of Spinraza, there was nothing to tackle it effectively. Those living with SMA spoke this morning about the impact of Sprinraza, this miracle drug, and the new opportunities it presented to them. They laid out those testimonies in a painful and direct manner, but along with the pain, there was bravery, courage and a raw ambition to live lives to the fullest. There was an ambition on the part of those older patients with SMA and an ambition on the part of those parents who are seeing lives that they thought they would not see because before the arrival of Sprinraza, the average lifespan of somebody with SMA was two years.

The HSE does not seem to share that ambition or to understand that pain because, on Friday last, it told families again that it would not approve Spinraza for reimbursement and thereby took that chance away. It has sentenced sufferers and their families to continued suffering and to emigration to one of the 25 countries that have approved Sprinraza medically and financially. It was laid out clearly to us this morning and I regret that this decision has potentially sentenced some of those sufferers to death because without Spinraza they will not survive SMA.

Twenty-five countries have approved Sprinraza and can stand over the clinical effectiveness of the drug. Twenty-five countries have come to a deal with Biogen on the pricing of Sprinraza, and yet the Government cannot. A number of Deputies have met Biogen representatives who have engaged with the HSE since September in a detailed manner. They have put a deal on the table, which is considerably less than the prices the HSE is quoting publicly. It strikes me that the HSE is deliberately misleading people about the potential cost of this drug in order to change the course of the debate.

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