Families appeal for vital drug therapy
The weakening and wasting of his muscles will not stop five-year-old Harry Bulman travelling almost 500km by minibus today to join a protest calling on the State to fund a potentially life-changing therapy.
Harry, who has spinal muscular atrophy (SMA), a rare neuromuscular disorder, will travel with family and friends from Youghal, Co Cork, to Leinster House, where parents of the 25 or so children in Ireland affected by SMA will plea for access to Spinraza, the first drug approved for treating the disorder.
Approximately 25 European countries have already approved the drug for reimbursement, but not Ireland, where last week the HSE said it was too expensive.
An evaluation by the National Centre for Pharmacoeconomics found the Year 1 reimbursement price for Spinraza, including Vat, was estimated at €641,466 per patient. The reimbursement price for subsequent years, Vat included, was €321,105. The HSE said the five-year budget impact was an estimated €20m.
Harry’s mum, Rebecca Bulman, is among a number of parents who will give a presentation to politicians in Leinster House at 10am today where they will argue the drug is essential for their children. They will then join a protest outside the gates at 11am, where a petition of over 100,000 signatures demanding access to Spinraza will be handed in to the Government.
Harry was diagnosed with SMA at 18 months of age. Regular visits to hospital and to Enable Ireland are part of his life. He is confined to a wheelchair and the amount of time he can spend sitting on the floor playing is gradually reducing. He attends school with the help of a special needs assistant, but is finding it increasingly challenging to do his homework because of difficulties holding a pencil. Due to muscle wastage, he is developing scoliosis, a curvature of the spine.
“We are all just heartbroken that they have said they are not going to fund the drug,” Rebecca said.
Spinraza’s effectiveness has been established in two pivotal clinical trials and supported by multiple clinical studies.
The HSE said in a statement that it regrets not being able to reach agreement with the manufacturer of Spinraza, Biogen, for reimbursement “at a price that is viable”.
The statement said the HSE is “obligated... to ensure that expenditure of this scale must represent evidence of clinical and cost effectiveness”.
“In comparison, for example, the HSE’s total expenditure for complex paediatric homecare packages in 2018 was €23.8m,” the statement said.
The HSE wrote to Biogen on February 21 “informing them of our proposal to refuse reimbursement at the current price offering”.
Under the requirements of the Health (Pricing and Supply of Medical Goods) Act 2013, the company has 28 days to respond or make representations to the HSE’s proposed decision.
Today’s protest coincides with World Rare Disease Day.
Currently, drugs are approved for only approximately 5% of 6,000-plus rare diseases.
