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Newly-funded haemophilia drug gives young aspiring actor the chance to 'dream again'

Twenty-year-old Andrew Scott is an aspiring actor and teacher. But, there was a time in his life he nearly gave up on performing because of his haemophilia.

Aspiring actor and teacher Andrew Scott at his first stand-up comedy performance in 2019. Source: 1 NEWS

"I’ve always had a passion for drama. I kind of gave up on that as a career option because I thought, for a person with a disability, it was unrealistic," the University of Auckland English and drama student said.

"I thought no one would hire an actor who might, the next day, spontaneously be on crutches."

"But, since going on Emicizumab, I’ve been able to actually consider that dream again."

From today, Government drug-buying agency Pharmac is funding access to Emicizumab, a preventative treatment for haemophilia, for up to 15 people with severe haemophilia.

Without Pharmac funding, the drug would cost a person about $600,000 a year.

Scott was diagnosed at birth with haemophilia A, an inherited blood condition caused by a shortage of a protein that clots blood. This impairs the body’s ability to stop a bleed and increases the risk of internal bleeding.

It's difficult to tell two years on, but Scott said 2018 was the worst in his life, health-wise. 

By the end of that year, he was experiencing two to three spontaneous bleeding episodes a week into his joints or muscles. When the pain got particularly bad, he would use a wheelchair or crutches as he waited for his body to heal.

Scott estimated he missed about a quarter of his final school year while he was in and out of hospital in 2018.

Andrew Scott at the start of 2018. Source: 1 NEWS

"I have a lot of people that keep me humble in that I would forget about how bad my life was in 2018.

"I would be getting on with uni and just doing my thing, and people I haven’t seen in a while would go, 'You’re looking so good, how’s your health?'"

Scott said with haemophilia increasingly at the back of his mind, he no longer had to explain what haemophilia was to someone he’d just met, and he wouldn’t have to worry about making multiple back-up plans whenever he went somewhere in case something went wrong.

In September, Scott performed in his first theatre show. 

"That freedom is something that I never anticipated and really something I can’t quantify. 

"It’s genuinely gone from being at the mercy of my own body and making do and trying to live a good life despite that, to being in charge of my own body and being in charge of my life."

He said the "freedom" Emicizumab granted him why he wanted the drug to eventually be publicly funded for all people with haemophilia, not just for those with severe cases.

"That’s just the process of financial justification," he said of funding the drug more widely.

"But, for the fifteen coming through on the 1st of December … I’m so excited for the families that are getting it."

'I WAS BAD ENOUGH AND BLEEDING ENOUGH'

Scott first got access to Emicizumab through Pharmac at the end of 2018. He lodged a special case application at the time because other treatments for haemophilia weren’t working for him.

For some people with severe haemophilia, like Scott, their immune system can develop a resistance (antibodies called inhibitors) to standard treatments designed to replace their lacking blood-clotting protein. It’s this group of about 10 to 15 people in New Zealand that Pharmac is targeting its funding of Emicizumab towards.

He said he was lucky enough to have access to a "great" medical team who advocated for him to get the drug early.

"I was also fortunate that I was bad enough and bleeding enough to get it," he said.

"That is messed up that we have to have this competing system of 'Are you sick enough to need this?'

"It creates really unhealthy mental pictures of how illness works, that you have to be at a certain level of unwell to deserve treatment. That shouldn’t be how it works."

But Scott said funding Emicizumab for the most severe cases was "a step in the right direction".

"It’s a strange thing because I really wouldn’t wish to not have haemophilia," he added.

"Having a disability, I think, always improves your moral character because it teaches you perseverance and teaches you that, sometimes, you just have to get on with life. 

"Absolutely, there were times in my life when I hated my health. But, the fact is, so much of it has contributed to who I am."

Dr Pete Murray, Pharmac’s deputy medical director, said funding Emicizumab aimed to "significantly" improve the lives of people with severe haemophilia A. 

"This medicine is expected to reduce the frequency of bleeding episodes and as a result, hospital visits, which will have important impacts on improving the quality of life for people who use it," Murray said.

He said Emicizumab added to the range of medicines Pharmac already funded for haemophilia, including two long-acting treatments listed in 2019. 

"It is a high-cost medicine, and funded access is targeted at those who will most benefit from it."

In its decision, Pharmac said it "would welcome a funding application for the wider group of patients" beyond severe cases of haemophilia A with inhibitors.