The Health Service Executive has approved Spinraza, the only treatment on the market for the rare muscle-wasting disease spinal muscular atrophy (SMA), after a lengthy campaign by patients.
The HSE’s leadership team this week decided to reimburse the drug, effectively overturning a negative recommendation by its drugs group last month.
The drugs group voted by a narrow majority in May against reimbursing Spinraza, despite a revised price application by the manufacturer, Biogen.
The UK’s national health service subsequently approved the drug, leaving Ireland in an “isolated position”, HSE director general Paul Reid acknowledged in an appearance before the Oireachtas health committee.
The HSE's decision has been welcomed by Minister for Health Simon Harris.
Earlier this year, the HSE said the drug would cost about €600,000 in the first year to treat each of the 25 Irish children with the disease and €380,000 a year thereafter, “with an estimated budget impact in excess of €20 million over a five-year period”.
Biogen subsequently issued a revised price proposal which it said was in line with the final price negotiated in other European countries that have approved the drug.
Spinraza is approved in 26 countries around Europe with just Estonia rejecting it.
As part of its submission, Biogen has previously provided the HSE with pricing for its portfolio of biosimilars - like-for-like therapies for expensive biologic drugs that have come off patent - that it says would offset the cost of Spinraza.
Families of children with SMA protested outside Leinster House in February, with members of most Opposition parties prominent among the attendance.
With just €10 million available for innovation in drugs this year, the HSE had spent virtually all of its new medicines budget by February.
