Keith Swanick (Fianna Fail) | Oireachtas source

I asked that the Minister come to the House so that I could echo my colleagues, Deputies Calleary and Lisa Chambers, and my Seanad colleague, Senator Conway-Walsh, in calling for the drug Spinraza to be made immediately available. Spinal muscular atrophy, SMA, is a group of inherited diseases of the motor nerves that cause very debilitating muscle weaknesses and wasting. It is life threatening, rare and the leading cause of death in infants and toddlers. There are 25 children in this country with this condition. It presents them with several burdens. They are wheelchair bound, suffer muscle recession and considerable pain and the day-to-day exercise of their lives is virtually impossible in things such as handwriting and other small things. I have met some of their family members. All they want is to give their children the best life they can.

Hope for these children comes in the form of Spinraza, the first treatment of its kind in the world. There is significant evidence from countries across the world, and from clinical trials, that Spinraza is changing children's lives significantly, slowing down muscle wastage and, in many cases, allowing muscle strengthening to occur which allows them to live a much more active, healthy life. Grace O'Malley from Robeen near Ballinrobe in County Mayo, along with her parents Paul and Lorraine, and other family members of those suffering from SMA are begging for access to this drug. They know it will not cure Grace but they also know from the CHERISH trial results, which outline the effectiveness of Spinraza, that these are the children who are most at risk of losing the muscle function which Spinraza has the potential to greatly manage and to improve Grace's condition and afford her some level of independence.

Earlier this year, Ireland entered an international alliance with the Benelux countries which was said would strengthen Ireland's hand in negotiations with big pharmaceutical companies. The Minister, Deputy Harris, hailed it as an agreement that would improve Irish patients' access to new and innovative drugs. I, like many others, am waiting to see the fruits of this arrangement. The Minister's repeated reference to the Netherlands and Belgium negotiating for Spinraza prior to Ireland joining the Benelux countries' initiative is misleading and confusing, considering the statement from Biogen on 20 September which said that in July 2018, Ireland was offered the same terms as other countries, regardless of the time of joining the initiative.

The latest information received by the families is that Spinraza has been referred to the rare diseases technical review committee. They have questions to which I would like answers. Who forms this committee? How does it differ from the National Centre for Pharmacoeconomics, NCPE, assessment? I understand that Professor Michael Barry is the head of the NCPE and the rare disease review committee, which raises a question about the impartiality of this whole process. As there is no new medical information to consider since the original dossier was presented to the NCPE, what exactly will it consider? The evidence will be the same as will the people. Will this review group engage with the patients? Has a timeframe been put on the assessments given the very serious nature of the disease?