Jo Raphael: Trikafta funding huge coup for cystic fibrosis community

Trikafta will be publicly funded in New Zealand. Photo / Andrew Warner

OPINION

“WE DID IT!!!” the flashy red banner reads, as I open up the Cystic Fibrosis New Zealand web page.

Trikafta, the life-changing drug for CF sufferers, has been provisionally funded by Pharmac, it was announced on Sunday.

This simple exclamation belies just how much effort was put in behind the scenes to get this over the line.

I and many of my colleagues have been following this story closely - hoping for a breakthrough.

Now the breakthrough has come but it’s been a rollercoaster.

The Pharmac decision is long-awaited and much celebrated.

The funding has been earmarked to begin on April 1. Unfunded, the drug costs $330,000 a year - out of reach for most people.

We reported this week Pharmac said it had reached a provisional agreement with medicine supplier Vertex to fund Trikafta and it was initiating a consultation on its funding for people aged 6 and older with cystic fibrosis who met certain eligibility criteria.

The Rotorua Daily Post and Bay of Plenty Times have been closely documenting the journeys of some of our local CF sufferers,

One journey that consistently stands out for me is that of Rotorua’s OJ Daniels.

He and his mother had already watched his sister, Santana, lose the fight with CF when she was just 21 and now his mother Trish was watching it all over again with OJ.

I cannot fathom watching helplessly as one child dies while the other continues to suffer.

The good news for OJ is he was accepted into a special funding programme run by Vertex.

The relief was almost overnight.

Trish told us previously that OJ went from being a “sick kid” living on “borrowed time” to waking up suddenly with a “massive burst of energy”.

Brothers Troy and Ashley Watson live together in Whakatāne and both were born with cystic fibrosis. Photo / Andrew Warner

We also highlighted the plight of two Whakatāne brothers - Troy and Ashley Watson.

Both have CF but only Ashley was eligible for Vertex’s special funding programme. Troy described the situation at the time as a “kick in the guts”.

Ashley only qualified for the funding because by the time this insidious condition got a proper grip on him he needed to be on oxygen 24/7 and “struggled to walk from my bedroom to the bathroom”.

Troy’s own symptoms weren’t as bad so he had to wait and watch his brother get better and start living his life.

”It’s quite a hard one to watch when I’m taking it and watching him slowly deteriorate,” Ashley told us.

Ashley reckoned the answer was a Pharmac budget increase - and he was right.

There was a glimmer of hope in this year’s Budget when it was given a 20 per cent boost to a total of $1.2 billion.

Things were also looking up in August when Pharmac said it had received a clinical recommendation that Trikafta should be funded for people aged 6 and older. Pharmac had already re-ranked Trikafta on its Options for Investment list following updated advice from its Pharmacology and Therapeutics Advisory Committee.

Then in September, another blow was dealt when Cystic Fibrosis New Zealand said in a statement it had been told by Pharmac that Trikafta had not moved up its “options for investment” list, meaning it was unlikely to be funded.

But this week we got the long-awaited news. What a stressful, emotional ride for everyone affected. I’m so glad about the outcome of this ordeal and can’t imagine the relief these families must be feeling.

Credit must go to Pharmac and Vertex who were able to come to an agreement on pricing.

But also a huge thank you to those who were brave enough to share their stories, highlighting the need for this drug. Not only can you breathe a sigh of relief - you can just breathe.

You did it.