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.About Rare Disorders NZ
RDNZ is the only national organisation supporting all New Zealanders who live with a rare condition, and the people who
care for them. RDNZ offers a central starting point for patients and families affected by rare disorders, and helps
families, patients and healthcare providers find essential information and support groups. https://www.raredisorders.org.nz/