Tama Hubbard, 3, has a degenerative disease called spinal muscular atrophy. Photo / Supplied
"Hi!" a bright young voice says down the phone.
Tama Hubbard is back to his talkative, happy self – for now.
The 3-year-old Taupō toddler was yesterday having one of his increasingly rare good days. A couple of weeks ago he was in Rotorua Hospital breathing through a machine.
Tama has a degenerative disease called spinal muscular atrophy (SMA), which sometimes leaves him so weak he can't lift his head, or lift his hand to his mouth to eat.
A small cough can cause him to go downhill quickly.
Last week, the Rotorua Daily Post shared Tama's story – about how fast he was deteriorating, about how the only FDA-approved drug that could help him wasn't publicly funded in New Zealand, and about how his mum, Lisa Geddes, couldn't afford to access it.
The drug, Spinraza, reportedly costs hundreds of thousands of dollars a year.
Read more: Tama is deteriorating fast. But the drug that can help him is too expensive
Today, the Rotorua Daily Post can share the response of the American drug company that generates hundreds of millions of US dollars in sales supplying Spinraza. And Pharmac's reply to that response.
Pharmac, New Zealand's medicine-buying agency, has the ultimate say on whether Spinraza gets funded or not. It is currently evaluating an application for its funding.
When the Rotorua Daily Post called to check up on Tama and his mum yesterday morning, the little fella was handed the phone.
He chatted about cows – the family lives on a dairy farm in rural Taupō – and about playing games outside in the sun. His voice was animated.
When his mum asked for the phone back, so she could continue talking, he said, "Bye!" and then, "Mum, say bye".
Geddes, back on the phone and laughing, said Tama was doing a lot better this week.
"They've just finished calving here, so he's always talking about checking the cows."
She said since the media attention last week, with several stories published nationwide about Tama's condition and the fight to get Spinraza funding, there had been a lot of comments and offers of donations and help.
Geddes said they reopened Tama's Givealittle page, which, as of yesterday afternoon, had received about $2000.
"I've had a couple of messages from people just saying really lovely words and just reaching out, letting us know that they're thinking of us and also wanting to donate to his Givealittle as well, which is really nice," she said.
"Even reading some of them when they came through, it was really overwhelming. The support was overwhelming and I would just find myself ... I would get tears in my eyes from reading these messages, they were just so lovely."
The Rotorua Daily Post contacted American drug company Biogen last week about Tama's case and the Spinraza drug.
In response, a media spokeswoman said in a statement that the company was "always saddened" to hear of people being unable to access Spinraza, "as it's our intention to work towards making it as widely available as possible for those who could benefit from it".
She said Biogen had worked as quickly as possible within New Zealand's regulatory and legal frameworks and were following the process laid out by Pharmac for it to consider reimbursing Spinraza.
"We made a submission to Pharmac as soon as we received Medsafe (regulatory) approval in August 2018, but unfortunately they requested further clinical data which we have subsequently supplied."
The spokeswoman said Biogen had stated and requested to discuss pricing as it was ready and willing to negotiate, "but first Pharmac has to accept the clinical benefit of this product".
"Over 50 countries have registered Spinraza and provide some level of reimbursement and Pharmac is the only reimbursement agency in the world to have questioned the clinical data," she said.
The spokeswoman said Biogen had been advised to follow the Pharmac process, "which we are doing and we will continue to do so, to hopefully make Spinraza available to the New Zealand spinal muscular atrophy community".
Questions about the price of Spinraza – why the drug is so expensive, and whether Biogen could not afford to make it cheaper, considering the amount of profit it is making from it – were not addressed in the spokeswoman's response.
A media spokeswoman for Pharmac, in response to the comments from Biogen, said while some medicines may be available in other countries, "the funding and reimbursement systems are often not comparable".
She said 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."
Meanwhile, Tama and his family continue to wait.
"I can't even explain how much he needs this, I think that's the hardest thing," Geddes said yesterday.
"They're always talking about the price and I get that, I really do, but when it's your child, you just want to do as much as you can, not only for him but everybody else."
Yesterday Tama was happy and talking. Yesterday was a good day.
It's today, tomorrow, the next day, and the day, week, and month after that, that are currently in the balance.
As the call ended, Tama had to have the last word.
"Can I say bye?" he could be heard asking in the background.
And then, "Bye!" Tama shouted down the phone, with the energy and enthusiasm of a boy excited about the sunny day ahead.
The circumstances around the death are being treated as unexplained.