Sally Sara: While the cost of living has almost everyone feeling the pinch, spare a thought for those waiting for drugs to be listed on the Pharmaceutical Benefit Scheme. 200 Australians living with VHL disease are anxiously waiting to see if a $12,000 a month drug will be included in the PBS in July. Elizabeth Cramsie reports.
Elizabeth Cramsie: When 25-year-old Giulia Di Mauro started getting chronic headaches, she put it down to stress. But when they didn't let up, her GP decided to investigate.
Giulia Di Mauro: So I had an MRI on my brain and I got a call within probably like an hour or two of having the scan from my GP.
Elizabeth Cramsie: After further testing, Julia was diagnosed with Von Hippel-Lindau syndrome, or VHL for short. It's a rare genetic condition which causes tumours to grow in various places in the body.
Giulia Di Mauro: Typically they'll grow on the spinal cord, in the brain and also in your eyes.
Elizabeth Cramsie: Within two weeks, she was having surgery to remove the tumour on her spinal cord.
Giulia Di Mauro: There was a risk of paralysis, so it wasn't something that we went into lightly, but I didn't really have much of a choice.
Elizabeth Cramsie: Julia now has another tumour, this time higher in her neck. So surgery comes with the risk of quadriplegia, and it's the only treatment option available. But a new drug approved by the TGA in 2022 could change that. The only catch, it's not yet listed on the PBS and costs $12,000 a month. Dr James Lynam is a medical oncologist. He says Belzutifan is undergoing extensive testing.
Dr James Lynam: We saw that it could shrink tumours and keep those tumours dormant for a period of time.
Elizabeth Cramsie: The drug is costly, but so is surgery, Dr Lynam says.
Dr James Lynam: So one of the challenges with VHL disease is that a lot of these people are crippled by their surgeries and their cancers. A drug like this would actually enable people to continue to live full active lives.
Elizabeth Cramsie: The Pharmaceutical Benefits Advisory Committee, or PBAC, will consider whether to list Belzutifan in July, which would make the drug significantly cheaper. But it could take some time. Liz de Somer is the CEO of Medicines Australia, the body which represents the research-based pharma industry.
Liz de Somer: Our research shows that it takes an average of 466 days from the time a new medicine is approved for use in Australia to when it's actually made available on the PBS.
Elizabeth Cramsie: The application sometimes go to the PBAC two to three times before a positive result. Liz de Somer says it causes delays, but she's hopeful a review of the system might change that. Giulia Di Mauro hopes the drug could give her an opportunity to plan her future.
Giulia Di Mauro: It really just gives me hope that I could potentially live a normal life like everybody else.
Elizabeth Cramsie: In a statement, the Department of Health told the ABC the review included possible options to streamline and simplify HTA processes and pathways, including changes that can reduce the need for multiple PBAC resubmissions before a medicine is listed on the PBS.
Sally Sara: That's Elizabeth Cramsie reporting there.