Finance Minister Grant Robertson at his photo op for his Wellbeing Budget 2022. Video / Mark Mitchell
Dozens of people are travelling to Wellington for the Budget to highlight concerns about sick children not getting Pharmac-funded treatments.
Two-year-old Zoey Butcher will be among them today. She suffers from spinal muscular atrophy (SMA), which if untreated will leave her unable to walk or even stand.
"I'm hoping to obviously gain access to the treatment for her, just to improve her quality of life," Zoey's mum Chauntel Wedlake told the Herald.
Patient Voice Aotearoa said treatments for SMA are funded in other countries, including Australia.
The genetic disease affected the central and peripheral nervous systems and voluntary muscle movement.
As the disease progresses, it can cause a loss of ability to control speaking, walking, breathing and swallowing.
Chauntel Wedlake says she and daughter Zoey hope to get some good news about Pharmac funding for rare illnesses. Photo / Supplied
In Zoey's case, it means without treatment she will require a wheelchair and could lose any chance to dance, swim, or even stand.
"If all else fails, at least we're raising awareness," Wedlake said after travelling from Auckland. "So other kids don't have to go through the same thing."
What also frustrated Zoey's family was the fact the issue was raised at a high level years ago.
"You shouldn't have to fight to access these treatments," Wedlake said.
Former National Party MP Paula Bennett accepted a petition three years ago, which more than 15,000 people signed asking Pharmac to fund life-saving SMA medicine.
Bennett in a Herald column earlier this month said apart from people with SMA, other children and adults in New Zealand desperately needed access to life-changing medicines.
Patient Voice Aotearoa Chair Dr Malcolm Mulholland said about 30 other patients and supporters planned to visit Parliament today.
"The people coming have a range of diseases including cancers, rare disorders and chronic illnesses," Mulholland added.
"They are coming in the hope that Pharmac receives the funding it requires and their lives are transformed as a result," he said.
Mulholland has experienced the anguish of not being able to get funded treatment for a loved one.
In 2019, Pharmac's rare disorders subcommittee recommended Spinraza be funded with a high priority for the treatment of children with the illness.
But even that recommendation did not guarantee funding.
Spinraza caused the body to produce more Survival Motor Neuron protein, and worked in a specific SMA type.
Zoey Butcher, 2 years old, has a rare illness called spinal muscular atrophy - but her supporters say medicines could help if funding was approved. Photo / Supplied
The Pharmac rare disorders subcommittee in 2019 discussed infusion gene therapy Zolgensma and noted the US Food & Drug Administration approved it earlier that year.
In 2021, Pharmac's rare disorders and neurological combined subcommittee discussed Risdiplam.
Those subcommittees said oral Risdiplam had the potential to reduce health system costs but some health-related quality of life data about it was not available.
Health Minister Andrew Little was asked yesterday about the visiting patients and what news the Budget might have for them but would only say: "The Budget's tomorrow."
The Green Party last week said a big boost for mental health was needed in the Budget and so was more funding for Pharmac.
Pharmac funding was also in the spotlight three weeks ago, after the Cancer Control Agency said New Zealanders were missing out on many potentially life-saving treatments.
The agency identified 20 different gaps across nine different cancer types where the medicine was publicly funded in Australia, but not in New Zealand. Other researchers including Mulholland identified even more gaps.
In March 2021, the Government announced a review of the way Pharmac purchased medicines.
The review panel questioned how Pharmac prioritised medicines for treating rare disorders - including potentially expensive medicines which could offer major health gains to small numbers of people.
The panel, which former Consumer NZ chief executive Sue Chetwin chaired, heard it could take from nine months to 10 years to get a medicine through Pharmac's funding process.
The Health Minister received the review's final report in early March and calls have emerged for him to release it.
Patient Voice Aotearoa trustee Fiona Tolich said Zoey's life, and the lives of 35 other New Zealand children, were now in the hands of the Government.
"Her parents are desperate and their fear is intense and real. They look at their baby girl and worry about what the next day will bring," Tolich said.
Tolich said if Zoey was born across the Tasman, she would have three options to treat her disease and there would be hope for her future.
The fundraisers hoped to improve Zoey's living conditions because she needed space for a wheelchair, and full insulation due to respiratory health troubles.
