17 May 2019

Parents beg Pharmac to fund drugs as son’s muscles waste away

From Checkpoint, 5:17 pm on 17 May 2019

It's a tale of two children - both four years old, both suffering a rare genetic disorder which slowly eats away at their muscles and limits their mobility. 

But the two children live in different countries - Ivy Old in Australia, Lincoln Woodmass here in New Zealand.

Lincoln and Ivy have spinal muscular atrophy - or SMA - a childhood version of motor neurone disease.

But because they're living on either side of the Tasman, the impact SMA will have on their lives is vastly different.

Ivy's mum Belinda Old said up until recently they had parallel childhoods - both were diagnosed with SMA type 3 around their second birthday, both experienced a decline in their condition and didn't know what the future held.

But 12 months ago Ivy Old, her sister Olive - who also has SMA - and her parents Belinda and Gavin moved to Melbourne, where the life-changing drug Spinraza is publically funded.

Belinda Old is Australian and the girls have dual citizenship. She said accessing Spinraza across the Tasman had been phenomenal for her daughters' health.

The Olds were back in New Zealand this week visiting family and their wider SMA community, including Lincoln.

Belinda Old said it had been bittersweet.

"We feel so lucky that our girls are the ones - but then we feel so guilty that there's children here who we have met that aren't having these opportunities - it's heartbreaking and I don't feel like we can ever truly celebrate wholeheartedly until these children get treatment," she said.

Lincoln's parents Tania and Regan Woodmass said they were ecstatic about how well Ivy and Olive were doing with Spinraza.

But Mr Woodmass said the girls' progress highlighted the awful situation they faced in New Zealand - where Spinraza isn't funded.

"It brings a tear to my eye just seeing that, from little girls that were worse than Lincoln is, now to be able to walk around on their own steam is just fantastic.

"It's excellent, and what the hell do we have to do to get that same sort of treatment?"

Spinraza is the only treatment for SMA and overseas users have described it as a wonder drug - it halts muscle deterioration of people with SMA and can also bring back lost mobility.

It has the greatest impact if used as early as possible and Mr Woodmass said they were racing against time to get it for Lincoln.

"We're at crossroads with Lincy - because he's been sized up for his power chair but if we somehow don't have to, if we could somehow get him Spinraza, if we could halt it there, maybe get some gains, that would be everything."

There are four different types of SMA - Lincoln and Ivy both have type three, putting them at the milder end of the spectrum. Babies with type one aren't expected to live for more than 18 months.

Tania Woodmass has seen the effect SMA has and is terrified for her son.

"They've had lungs collapse, difficulty eating, breathing swallowing, they can't hold their own heads up. 

"It scares the living daylights out of me to think about what could be."

Fewer than 100 people have the condition in New Zealand and this month they worked to get more than 15,000 people to sign a petition calling on Pharmac to fund Spinraza.

Earlier this year Pharmac deferred a decision on funding the drug for under 18-year-olds, saying it was awaiting further evidence.

Tania Woodmass said other countries hadn't needed to wait and Pharmac's deferral was a disgrace.

Overseas estimates put the cost of Spinraza at around $1 million, per person per year - but Gavin Old said that was a totally arbitrary figure as Pharmac hadn't engaged in any price negotiations.

"The million dollars is for the first year in America, so completely different space over there, the insurance companies pay for their medication in America, the drug companies go there first and as they go on the price gets negotiated and comes down 

Pharmac said it was in regular contact with Spinraza's supplier and if they get more evidence from them before their next subcommittee meeting in September, it could be assessed then.

The Woodmass' and the Olds say denying children access to Spinraza is denying them a chance to live - and as they watch SMA children in Australia go from strength to strength, it is also strengthening their resolve to get the drug to New Zealanders - as soon as possible.