A man who's been taking a new haemophilia drug for the past two years says it's changed his life.
Andrew Scott, 20, is excited that from this week some of his "blood brothers" will also have free access to emicizumab, which would otherwise cost $600,000 a year.
"I haven't had any internal bleeding or issues," he told The AM Show on Wednesday. "Previous to that I was in hospital, having internal bleeding, in a wheelchair/crutches, my medication wasn't working."
Scott has haemophilia A, an inherited blood-clotting deficiency. Before going on emicizumab in 2018, he'd regularly have episodes of spontaneous internal bleeding, and taking part in sports - such as his beloved surfing - was impossible.
"Your joints or muscles fill up with blood and it's very, very painful, and it takes ages to reabsorb."
He spent much of his high school years in a wheelchair or on crutches, and had to sit his NCEA exams at home on heavy pain medication.
In 2018, he was having two or three bleeds a week, so Pharmac deemed his condition a special case and paid for emicizumab, a new drug only approved by the US Food and Drug Administration at the end of 2017.
It changed Scott's life, and he's now "at university walking 10,000 steps a day and actually living a proper life", and back on the surfboard, which he had been forced to give up.
He only needs to take one dose of emicizumab a week, compared to the daily dose required of his previous medication, which wasn't really working anymore.
At uni he's studying English and drama, with dreams of being an actor - studies he held off until finding a medication that worked.
"I didn't think that was a realistic career for someone with spontaneous bleeding."
From this week, up to 15 other Kiwis will also have access to the drug, sold under the brand name Hemlibra.
"Our clinical advice indicates that treatment with emicizumab significantly reduces the frequency of bleeding episodes for people with haemophilia A who have severe disease and have developed inhibitors to standard treatment," Pharmac said in a statement. "Emicizumab is an injection that people could self-administer, and would reduce their need to go to hospital and improve their quality of life."
Around 500 people have been diagnosed with hemophilia A, of which about a third are estimated to suffer it severely. Pharmac's current funding covers people with severe haemophilia with "inhibitors of factor VIII", meaning their bodies have developed antibodies which prevent other medications from working.
"Pharmac would welcome an application to widen access to emicizumab for patients with severe haemophilia A with or without inhibitors of factor VIII so that this can be assessed through our usual decision-making processes."
Each dose of emicizumab can cost between $3570 and $17,846, depending on the dosage.
Scott says while he still has to be cautious, he's enjoying having more freedom to live his life as he'd like.
"It's no longer the thing that defines me, it's no longer the central thing in my life. It's been huge in building who I am, but it's no longer what is me.
"That'll be the case for the other 15 who are getting it - I'm already hearing stories of some of my blood brothers coming through, it's been really emotional."
