Drug brings ‘whole new OJ’ —mum
Son with cystic fibrosis can plan for future
In the past two weeks, cystic fibrosis sufferer OJ Daniels has become “a whole different person”. Instead of being confined to his Rotorua home, the 19-year-old can now walk, go outside and breathe without fear of “choking to death”.
He can “start thinking about a future” — exercising, getting a job, dating and travelling overseas.
“Before, I couldn’t quite think of one. I was just trying to take it day by day,” OJ said.
“It’s like a whole new OJ.”
OJ has been approved for free access to Trikafta — a $330,000-a-year “miracle drug” that treats the underlying cause of cystic fibrosis.
His mother, Trish Daniels, said OJ went from being a “sick kid” living on “borrowed time” to waking up suddenly with a “massive burst of energy”.
“Before Trikafta, he didn’t have the option to do anything. Now he does.”
Cystic fibrosis is a genetically inherited condition that produces a thick and sticky mucus that mainly affects a person’s lungs and digestive system. Specialists believe the average life expectancy of a person with cystic fibrosis is mid-to-late 30s.
Trikafta is not publicly funded in New Zealand. A petition seeking to change this has been presented to
Parliament, claiming clinical trials have shown “significant improvement” in health outcomes for people with the condition.
In April, OJ and Trish told the Rotorua Daily Post they were “desperate” to get access to Trikafta. Trish lost her daughter, Santana Daniels, to cystic fibrosis in 2017.
“If we don’t get Trikafta I’m likely to lose my son,” Trish said through tears at the time.
Yesterday, Trish and OJ spoke about the “life-changing moment” they found out OJ was approved for Trikafta.
OJ has access to Trikafta through a compassionate managed access programme run by the drug’s supplier and manufacturer Vertex.
They submitted an application to the programme on May 19 and found out OJ had been approved on June 4.
“Now that he’s on it, he stays on it,” Trish said.
The day Trish received the phone call from OJ’S doctor, OJ was “very sick”.
Trish told the doctor she was worried and wanted OJ to go to hospital.
The doctor said, “Well, that might be a good idea because he got approved for Trikafta.
“After that I heard nothing else.” OJ was curled up on the couch, coughing and tired, when his mother “came running down the hallway in tears,” OJ said.
“That’s when she told me we got approved . . . It made me very pleased because we definitely needed it.
“Mum was on a high — I was too sick to be on a high.”
The next day, OJ was admitted to Rotorua Hospital as he was “struggling to breathe”.
“I could barely even move without running quite low on oxygen. I also had massive headaches — it felt like someone had literally smacked me over the head,” OJ said.
“The cough was very, very aggressive.”
Once OJ had stabilised, he was transferred to Auckland Hospital on June 10 so that he could be monitored when he started taking Trikafta.
On June 16, when he took Trikafta for the first time, “I just started
coughing — all the mucus started coming all up . . . very uncontrollably”.
But he noticed a difference the next day.
“I was bouncing off the walls . . . I wanted to get out right there and then out of hospital because I had too much energy.”
Trikafta had also helped him put on weight.
“I’ve actually got a puku now.
Before I was very skinny . . . I hated actually putting my T-shirt [on] because of that.”
After spending nearly three weeks in hospital, OJ returned home on Thursday and celebrated Matariki with wha¯nau on Friday.
“I honestly just enjoyed breathing for once because that’s fun without choking to death,” he said.
OJ used to start his day by spending an hour in the bathroom choking on mucus, trying to bring it up. “Now that’s all changed.”
Instead, he wakes up and takes three daily pills.
“I can leave the house. I can actually leave this chair . . . I can actually stand, go outside and do whatever I want.”
OJ said he and Trish were planning to go to Rarotonga this year. He will also start boxing again as he takes part in Sweatember — a fundraising event in September for those living with cystic fibrosis.
Trish said OJ had become “a whole different person” and she wanted him to experience everything he had missed as a teenager.
“I want him to go to the movies, I want him to go out and have a beer at a nightclub.
“Some of his friends are dads already. And he’s not even gone out on a real date or anything.”
The pair will continue campaigning for Trikafta to be publicly funded for the rest of the cystic fibrosis community.
“It’s called the miracle drug for a reason — it certainly is. The difference has just been phenomenal.”
Last month, a month’s supply of Trikafta was anonymously delivered to their home. Trish said they still have the box of Trikafta which is unused. They do not know who sent it, but now OJ has the drug, they are trying to track down the donor to see what they would like to do with it.
Cystic Fibrosis New Zealand chief executive Lisa Burns said the organisation was “delighted and relieved” for OJ and his family.
“Trikafta is life-changing and gives someone a chance to have a future, to have improved quality of life and the same opportunities as everyone else.”
While this was “incredible news”, Burns said the health of the cystic fibrosis community should not have to deteriorate to the point where they needed to rely on a managed access programme to get medicine.
Burns said Trikafta should be fully funded by Pharmac and available in New Zealand, as it was in more than
30 countries in the world including Australia.
Pharmac has previously said it would like to fund Trikafta and was in talks with its manufacturer, but the cost was a barrier within the agency’s set government funding.
A Vertex spokesperson said Vertex introduced a managed access programme for Trikafta in New Zealand last year “in recognition of the severe and progressive nature of cystic fibrosis resulting in high unmet medical need in some patients”.
The programme was aimed at providing access to the “most critically ill” patients in countries where medicines were not yet funded, but where there was a “clear path to reimbursement”.
“Through this programme, we provide Trikafta free of charge to patients who meet certain objective criteria, if requested by their doctor, under strict conditions, where all other medicinal options have been exhausted, in line with the law.”
It was not designed to replace the reimbursement process in New Zealand.