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'We need to do right by families': Call for HSE to fund drug for rare disease after NHS approval

Spinal muscular atrophy weakens muscles and causes problems with movement, breathing and swallowing.

Sam Bailey, who has SMA, at a recent protest in Dublin to demand funding for the drug.
Sam Bailey, who has SMA, at a recent protest in Dublin to demand funding for the drug.
Image: Orla Ryan/TheJournal.ie

THE HSE HAS been urged to approve funding for the use of the drug Spinraza in the treatment of spinal muscular atrophy (SMA) in Ireland following approval by the NHS. 

SMA is a rare, debilitating and life-threatening disease that affects nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. 

Where it develops in babies and toddlers it can significantly reduce life expectancy.

While it is not a cure, trials have shown that Spinraza, also known as nusinersen, can slow the effects of SMA in some cases. 

NHS England today said it has approved the drug for use after an agreement with its makers, Biogaen, to fund treatment for a limited time to allow more data to be collected on its effectiveness.

The treatment will be made available to the youngest and most severely affected patients immediately. 

The HSE has previously said it will not reimburse for the drug at its current price, stating it would cost more than €20 million over a five-year period. Around 26 children in Ireland with SMA could benefit from Spinraza.

Source: SMA Ireland

Labour TD Alan Kelly said it is time now for the HSE to follow the lead of the NHS and sanction funding for the “life-changing” drug. He said families here have been “crying out” for the drug to be made available and the news from the UK “will give many people hope”.

“Ireland is now one of the only countries in the European Union to have not approved this drug. We need to do right by the small number of families whose loved ones have this debilitating condition.”

Also commenting on the announcement, Sinn Féin senator Rose-Conway Walsh pointed out that it appears the price has been lowered compared to previous cases, including the offer that was made to the HSE.

“It is vital that this is taken into consideration as the HSE considers its next move in negotiations with the company,” she said. 

In a statement, Biogen today said it has submitted a revised proposal for reimbursement that it hopes the HSE will respond to in a “similarly positive way” to the NHS.

To date, more than 7,500 patients worldwide have been treated with nusinersen, from infants to adults.

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