The first Lauren Poole had heard of epithelioid sarcoma was the day she was told her thumb had to be amputated to save her life.
The tiny lump on the tip of her thumb had been innocuous enough. But her instincts told her to have it removed and tested.
She was diagnosed with the rare soft tissue cancer, and 17 hours later a surgeon was severing her thumb at the main knuckle.
"It was all fast and terrifying," the 23-year-old said.
"As I was waiting for a scan I realised my dad and sister didn't know I had cancer and I was already getting ready for surgery.
"Telling dad was easily one of the worst moment of my life."
Ms Poole considers herself incredibly fortunate. Most epithelioid sarcomas go undiagnosed until the cancer is very advanced.
There are no pharmaceutical treatments for the rare and aggressive condition that predominantly affects 20 to 39-year-olds. The only option is amputation.
"I know people who have lost whole limbs. I know people who were told it was terminal the day they were diagnosed," Ms Poole said.
"It's so strange sitting in a lecture at university surrounded by students worrying about the exam or their essay and I'm worrying about whether I have more cancer," said the archaeology and English undergraduate.
A radical new report offers the federal government a road map for addressing inequitable access to rare and less common cancer treatments.
More than 52,000 people are diagnosed with rare or less common (RLC) cancers and an estimated 25,000 people die from the conditions every year in Australia.
Rare and less common cancers are defined as cancers with fewer than six cases, and six to 12 cases per year per 100,000 population respectively.
The Rare Cancer Australia report recommends radical reforms to research and treatment funding models, including provisional pharmaceutical benefits scheme listings and enrolling RLC cancer patients in clinical trials designed to investigate treatments for more common conditions.
The current system was "hopelessly inequitable whichever way you crunch the numbers," said the chairman of Rare Cancers Australia, Richard Vines.
RLC cancers account for 30 per cent of all cancer diagnoses and almost 50 per cent of all cancer deaths, but attract just 13.5 per cent of research funding and 12.6 per cent of funding for treatments through the PBS.
Medicines for rare cancers can be prohibitively expensive, with some RLC cancer patients having no alternative but to pay exorbitant sums for orphan drugs that attracted no government subsidies.
"Rare cancer patients pay taxes that are used to pay for drugs on the PBS to treat patients with more common cancers, then they have to pay full price for their own drugs," Mr Vines said.
"The level of injustice is beyond comprehension," he said.
The report delivered at the CanForum conference at Parliament House on Wednesday urges the Australian government to invest in more local clinical trials for RLC cancers.
"The fastest way to get serious treatment for TLC cancer patients is to increase government funding for clinical trials," Mr Vines said.
Professor David Thomas, director at the Kinghorn Cancer Centre and head of the cancer division at the Garvan Institute, said trials needed to move away from classifications of cancer based on tissue biology – or which part of the body the cancer was located.
Garvan's MoST Study uses genomic screening to identify variants that could become the basis for treatments, and whether effective therapies for more common cancer may work in rare cancers.
"We recommend the federal government nationalise this program so that is it accessible to all Australians in their own states," Professor Thomas said.
The report also proposed provisional PBS listings be introduced for medicines to treat RLC cancers where additional evidence of effectiveness was still needed.
Mr Vines said meeting the benchmark of gold standard evidence for PBS listings was "almost impossible" for rare cancers with small patient populations and negligible incentives for pharmaceutical companies to fund them.
"Provisional PBS listings would enable researchers to monitor the drug's effects over time in a real world setting and see if it reasonably aligns with patient expectations," he said.
Health Minister foreshadows reforms
Health minister Greg Hunt has charged the Pharmaceutical Benefits Advisory Committee (PBAC) chair Professor Andrew Wilson with the task of developing a new process to give rare cancer patients greater access to subsidised treatments.
Speaking at the CanForum conference, Mr Hunt flagged plans to "bring together" TGA and PBAC "pan-tumour" assessments right across a range of therapeutic outcomes", but did not provide detail about how this would work.
Mr Hunt also told delegates that more than 20 per cent of medical Research Future Funds would be earmarked for research into rare conditions, and would call for proposals for new clinical trials by the end of September as part of a $13 million package.
The RCA report also recommended:
- Pharmaceutical companies tack on RCL cancer indications when applying for TGA approvals and PBS listings for drugs to treat common cancers.
- RLC cancer patients join cohorts of clinical trials designed to test drugs for more common cancers and their data analysed and pooled with data into similar projects internationally.
- Australian trials be designed in consultation with the regulatory bodies to support drugs getting TGA approval and PBS listings for RLC cancers.
- Data collection systems including My Health Record be used to capture patient outcomes for provisional TGA and PBS drugs.
"We can fix this, we just need to have the will," Mr Vines said.
Story originally by Kate Aubusson, appearing in the The Sydney Morning Herald, 9/8/17. The original article can be found here