SMA Ireland is today deeply concerned at the ongoing conduct of the HSE and Government with regard to the approval of the Spinraza drug treatment for SMA sufferers.
It emerged last night through an article in TheJournal.ie that the HSE may have been deliberately dishonest in public statements regarding Spinraza, seemingly as a PR exercise to avoid criticism and pushback from families and the public. The HSE and Minister should clarify the situation as soon as possible.
The quotes from the article have disturbing implications for the honesty of the HSE in its statements on Spinraza and other rare disease drug cases.
Anton Mannering of SMA Ireland said “All of this is very disturbing and worrying news for anyone with a child with a rare disease and not just those with SMA. Obviously in the first instance, that the Spinraza treatment has been refused here, when it is approved in almost every other EU country, is a scandal in and of itself.”
“The idea that the HSE may have deliberately tried to mislead parents and in particular the media and public about the cost and efficacy of the treatment is absolutely mind blowing, if confirmed. Some people don’t understand families anger and confusion at the refusal decision because they trust the HSE to be honest about these things. It casts a shadow over the veracity of anything the HSE say about the situation including the statements on budget availability.”
“It is doubly worrying as this seems to have been done as part of a deliberate PR exercise starting on the same day the notification of refusal came out. Some very hard questions need to be asked and answered and we urge our public representatives to do so.”
SMA Ireland urges everyone to contact your local TD and ask them to question the Minister about the matter.
From the TheJournal.ie article below:
“Biogen said it is “genuinely surprised” by the HSE’s statement. “The figures referenced are at least six months out of date. In terms of efficacy, the effectiveness of nusinersen in type II/III SMA patients is supported by a randomised clinical trial in the same way that efficacy has been demonstrated in type I SMA patients,” a statement notes.
It adds that both trials were stopped early on the advice of the US Food and Drug Administration and the European Medicines Agency “given the strength of the interim results”.
The statement also notes that Spinraza has been approved for reimbursement in 25 other European countries, saying the price being paid in those countries is in line with what was offered to the HSE after negotiations.
Biogen recognises that this decision marks a deeply concerning day for SMA patients and their families, and we share that concern: Ireland is now significantly behind many parts of the world in making nusinersen available.
Biogen asked the HSE to re-enter discussions “as a matter of utmost priority”.