Four-year-old Charlotte Bond couldn’t roll over by herself two years ago.
She has type 2 spinal muscular atrophy (SMA), a genetic condition that affects the control of muscle movement and causes a person’s muscles to waste away.
Her family moved to Australia where the drug Spinraza is funded. Because of it, she can now take about 250 steps a day unassisted.
About 35 kids in New Zealand are in need of the life-saving drug — one that’s being funded by 56 other countries around the world. However, it is not funded in New Zealand.
Mum Kristie Yeoman said the drug was life-changing for Charlotte.
She told Breakfast by the second dose, Charlotte was in her bed and able to roll over.
After the eighth dose, she was “blown away” to find Charlotte had started walking.
“She’s doing amazing,” Yeoman said.
Access to the drug in Australia is free. In New Zealand, it would cost upward of $390,000 a year, not including administration fees.
“Fund it. It’s incredible,” Yeoman told drug-buying agency Pharmac.
Fiona Tolich is fighting to make the SMA drug available in New Zealand. She estimated funding Spinraza for all 35 kids in New Zealand who have the condition would cost about $7 million, depending on what Pharmac can negotiate with the drug company.
“It’s not too much to ask,” she said.
“On top of that [$390,000], because it’s not funded, you would then have to pay for the administration … no-one can access it for that very reason.”
The most severe type of SMA — type 1 — has a 95 per cent death rate for children who get it early. Without treatment, many die before they turn two from respiratory failure. For children with SMA type 2, life expectancy is reduced. Most only live to adolescence or young adulthood.
Tolich keeps a notebook of the names of Kiwi kids with SMA. She has a milder version of it herself.
“It’s like an SMA whānau. You’re so connected. I talk to some of these families more than I do my own family - you get this bond.”
In the past 12 months, she'd attended three funerals for three children in the notebook, some she's attended virtually because of the Covid-19 pandemic.
“We need their drug on the market here because there’s no other option,” Tolich said.
Numerous submissions to get Spinraza funded in New Zealand have all failed. That’s despite Pharmac's Rare Disorders Subcommittee recommending the drug “be funded with a high priority” for children who aren’t yet showing symptoms, and “funded with a medium priority” for people showing symptoms.
The subcommittee said there was “good quality evidence” the treatment improved survival and improved motor functions.
It noted that the price proposed by the supplier was high.
Pharmac told RNZ because the patent for the drug won’t expire until 2030, it wouldn’t be able to get a 50 per cent discount. Because of that, it estimated the cost would be “considerably higher” than $7 million, but wouldn’t put a number to it.
Tolich tried to lodge a complaint with the Human Rights Commission, saying the Government was discriminating against children with SMA. The commission told her because Pharmac funds all drugs for treating children with cancer, an argument could be made for SMA patients’ discrimination.
The commission also warned a counter-argument to it could be that Pharmac stop blanket funding for paediatric cancer cases, something RNZ reported Pharmac had already been considering .
“The Human Rights Commission have absolutely no teeth in this fight,” Tolich said.
She planned to go to the UN next, because she had exhausted her options domestically.
Tolich said she would argue New Zealand wasn’t recognising the rights of children, of health, and of people with a disability.
“We’ve signed up to all these UN conventions and covenants. But, clearly, we don’t value them because otherwise we would be implementing that domestically.”