Sep 28, 2022, 1:43 PM

Rare Disorders NZ is thrilled for those affected by spinal muscular atrophy (SMA) to finally receive news today that Pharmac has reached a provisional agreement with medicine supplier Biogen to fund nusinersen (brand name Spinraza) for people with SMA.

This announcement is a massive win for those affected by SMA, who have advocated for years for this life-saving treatment to be funded. It is thanks to their hard mahi, and in particular Patient Voice Aotearoa advocate Fiona Tolich, that Spinraza will soon be available.

There are approximately 75 New Zealander’s living with SMA, which is a rare, inherited neuromuscular disorder that affects the control of muscle movement and causes the muscles to gradually waste away.

It is the leading genetic cause of infant mortality. Spinraza has proven to be a very effective treatment, improving survival and motor function, and is currently funded in 64 countries, including Australia.  

While Rare Disorders NZ will be celebrating this news with the SMA community, frustration remains that it has taken so long to approve this life-changing medicine, having long called for a separate assessment pathway for medicines for rare disorders. 

“Pharmac’s cost-saving, bulk-buying model may work for high-volume treatments, but it disproportionately disadvantages rare disorder medicines due to their higher costs and lower volumes. With Pharmac’s limited budget, rare disorder medicines cannot compete under this model and change needs to happen to ensure more equitable access to medicines in New Zealand,” says Rare Disorders NZ Chief Executive, Michelle Arrowsmith. 

Rare Disorders NZ looks forward to the release of Pharmac’s response to the Pharmac Review in the coming month to learn how Pharmac intends to improve its approach to rare disorder medicines, and urges Pharmac to ensure rare disorders are given a fair and clear assessment pathway for medicines.

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