[ad_1]
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/
© Scoop Media
[ad_2]
Source link