Deputy Broughan welcomes today’s announcement that Spinraza is approved for children with spinal muscular atrophy (SMA) following a long and difficult campaign by families of children affected by this rare, muscle-wasting disease. In Ireland. there are around 70 people, 26 of whom are children, with SMA. It is the number one genetic cause of death for infants worldwide and will affect around 1 in 11,000 babies born worldwide each year. Spinraza or Nusinersen is produced by Biogen and has been the first treatment for Spinal Muscular Atrophy (SMA). It was available in most of the other European countries but not in Ireland.
In April of this year, Deputy Broughan submitted a Private Members’ Motion on Spinraza which called for the Government to meet with children and adults with SMA and their parents; to urgently direct the HSE to speed up negotiations with Biogen because time is of the essence for these patients; to increase the resources available to the National Centre for Pharmacoeconomics (NCPE) and the HSE Corporate Pharmaceutical Unit (HSE-CPU) to allow for quicker assessment, negotiations and approval of new treatments; to reduce the time allowed under the Framework Agreement on the Supply and Pricing of Medicines, and in line with the 2013 Act, for a decision by the HSE from 180 days of receiving the application down to 90 days, with a maximum of 120 days if further information is needed from the company; to revise the Rare Disease Strategy and include greater input from patient advocacy groups and improve the remit of the Rare Diseases Medicinal Products/Technology Review Committee (RDTRC) to be in line with the Scottish PACE model; to introduce an Orphan Drugs levy on pharmaceutical companies and create an Orphan Drugs fund; to provide a report to the Dáil by the end of Quarter 3 2019, to outline the impact of our membership of the BeNeLuxa initiative; and to ensure that the review of the HSE reimbursement and pricing decision-making process is completed by the end of Quarter 3, 2019. Deputy Broughan’s motion highlighted the real life impacts of Spinraza (Nusinersen) and the inadequacy of the health technology assessment for treatments and medicines for rare diseases. Deputy Broughan says “I welcome today’s news that Spinraza will now be made available to children with SMA, but I despair that young adults over 18 and adults who have fought long and hard for access to this treatment will be denied access. It is a very unfair system, but it has been a hard-won battle for the parents of children with SMA and I hope that access and reimbursement is processed speedily. Well done to the families and groups involved in the campaign.”