Three-year-old New Zealander Charlotte Bond is showing signs she may walk again on her own with the help of a special drug, but she's having to achieve her milestones across the ditch because of barriers set by Pharmac.
Lifesaving drug Spinraza hasn't been approved for funding in New Zealand, making the country unsafe for her to return home, her mum Kirstie Yeoman told TVNZ1's Breakfast today.
Charlotte and her family have uprooted their lives from Auckland, leaving behind extended family and friends, to start a life in Australia so that she can get access to the treatment for her motor neuron disease spinal muscular atrophy (SMA).
The condition causes basic movements to be exhausting as muscles around her body slowly die.
Ms Yeoman said, despite the financial challenges and being away from home, it was "a no-brainer" to move to Australia as Charlotte's condition worsened with age.
"There's a chance she could die in New Zealand and I'm not ready to bring her back to New Zealand until Pharmac and the health system has made it clear to me that it's safe to bring her back and right now it's not."
Ms Yeoman said small signs most people take for granted, like a child lifting their head, were big achievements for Charlotte. But it's expected after 10 more doses of Spinraza she'll be able to walk.
Fiona Tolich, who has asymptomatic SMA, joined Ms Yeoman on Breakfast this morning and said in the most severe cases, people with the condition will die at 13 months old.
"Internationally those same children, those same type, over 80 per cent are showing no clinical signs of SMA. they're walking, they're dancing, they're skipping; whereas in New Zealand they won't ever achieve the milestone of being able to lift their head."
Spinraza wasn't a cure for the condition, which causes nerves to die off, but Ms Tolich said even in the worst cases it halts deterioration. In the best cases people are able to regain movement they'd lost if the nerves had just "gone to sleep".
Ms Tolich said Pharmac is waiting on more clinical data and evidence about the drug, but said New Zealand was the only country to question the data.
Pharmac's website says it's been seeking clinical advice on Nusinersen (Spinraza) since September last year. They had received advice from the Rare Disorders Subcommittee at meeting on November 5 last year and from Pharmacology and Therapeutics Advisory Committee (PTAC) at meeting on May 23.
"Pharmac seems to be making their decisions completely on financial basis."
Taking action into their own hands, they have led action including submitting a 15,000-signature petition to Parliament with National's Paula Bennett and presented to the health select committee.
"We've also realised that the problem is bigger than SMA so you can't meet these other patient groups and realise that there's 250,000 people that are waiting for a drug to be funded right now in New Zealand.
"There's over 100 drugs on the waiting list, if they're marked high priority, some of those are taking up to five years to come through and for those that aren't given a high priority, they are waiting up to 15 years.
"The problem is our Pharmac system, whether or not it's not funded enough - it's definitely not funded enough, so we've got a petition at the moment to double Pharmac's funding and to reform Pharmac."
Pharmac operations director Lisa Williams told 1 NEWS that having a fixed budget means the agency needs to make careful and considered funding choices in the interests of all New Zealanders.
“Pharmac works to get the best health for New Zealanders we can, by funding medicines from within the available budget," she said.
"While some medicines may be available in other countries, the funding and reimbursement systems are often not comparable. New Zealand must make its own decisions, carefully assessing the available evidence and thinking about medicines use in the New Zealand health context.
"Pharmac will continue making the best choices we can, expanding available treatments for all New Zealanders using a robust, evidence-based approach.”
The rare disorders subcommittee met last month and considered supplementary information provided by the supplier of Spinraza, she said. Pharmac intends to publish a record of the subcommittee’s advice and recommendations "in due course".