Rare Disorders NZ (RDNZ) supports patient advocate Fiona Tolich’s Human Rights Commission case against PHARMAC for
failing to fund a rare muscular disorder drug.
“Our organisation whole-heartedly supports Fiona’s case for effective treatment,” says RDNZ Chief Executive Lisa Foster.
“It’s shameful that families are forced to move to Australia to access this life-changing medicine, despite continued
patient submissions and clinical evidence to show its worth.”
Spinal Muscular Atrophy (SMA) is a group of spinal muscular disorders that affect the motor neurons. Tolich lives with
SMA and has been campaigning for years to get the drug, Spinraza, funded on behalf of 35 young people aged 18 years and
under who suffer from the rare disorder. Spinraza has shown incredible results for children with SMA in other countries.
The fact that PHARMAC won’t fund it for New Zealanders is blatant “discrimination”, according to Tolich.
“PHARMAC’s own clinical experts placed Spinraza on their high priority for funding list in February – how long do New
Zealanders have to wait? Are we forcing people with rare disorders to play a lottery game with odds never in their
favour? In a country with a keen sense of fairness, is that acceptable?” says Lisa.
There are more New Zealanders living with a rare disorder than those diagnosed with diabetes - an estimated 300,000
people. Within rare disorders there are over 6,000 different conditions with a wide range of symptoms that impact on
daily life in varied ways but with common barriers.
“PHARMAC needs a significant increase in their budget as well as a well overdue review of their funding processes to
ensure people living with uncommon health conditions are not left out,” says Lisa.
RDNZ, and their collective of 140 rare disorder support groups, are calling for systemic changes system to ensure people
living with a rare disorder are considered within our healthcare system. The Fair for Rare NZ campaign wants
acknowledgement of the common challenges facing the rare disorder community through the development of a National Rare
Disorder Framework. NZ lags behind most OECD countries in supporting people living with rare disorders and their whanau
to access needed health and social care.About RDNZ
Rare Disorders NZ (formerly NZORD) is the connector hub and collective voice for patients and families affected by rare
disorders. Rare diseases affect 6% of New Zealanders.
There is no acknowledged definition of rare disease in NZ, however RDNZ follows the European Union policy which defines
a disorder as rare when it affects fewer than 1 in 2,000. More than 6,000 rare disorders collectively affect around
300,000 New Zealanders. Of this number, 50% are children. www.raredisorders.org.nz