Pancreatic Cancer Breakthrough Offers Hope, but Australian Drug Approvals May Leave Patients Waiting

Dee, who is 50 and prefers to be known by that name, weighed just 35 kilograms when she could no longer get off the lounge or take part in family life. A year later, she is celebrating family milestones she feared she would miss, and she calls each morning a gift. What she finds harder to accept is the prospect that the treatment that gave her that year may not reach other patients in Australia in time.

Her path began with a Whipple procedure, a complex operation that removes part of the pancreas and surrounding organs, followed by seven months of chemotherapy. Her first scan afterward showed the cancer had spread to her lungs. Early genomic testing had already identified the genetic characteristics of her tumour, which let doctors find an experimental treatment she was eligible for. At the time, she says, another month or two would have felt extraordinary. Instead her cancer has stayed stable for a year. "It's given me my life back," she says of these new types of treatments.

The context is grim. Pancreatic cancer, often called the silent killer, has long been among the deadliest common cancers, with few advances and a five-year survival rate of 14 per cent. Almost two-thirds of Australians diagnosed die within 12 months, and only 22 per cent live two years.

A new class of drugs has raised hopes. More than 80 companies are developing treatments aimed at the RAS pathway, a signalling system that tells cells when to grow and divide and, when faulty, can drive cancer. Michelle Stewart, chief executive of Pankind Pancreatic Cancer Australia, says options have barely changed in three decades. "The cupboard is kind of bare," she says, describing the emerging treatment as the start of a new era, though not a cure. For a terminally ill patient, a few extra months could mean lasting long enough to benefit from another therapy.

Medical oncologist Michael Lee says early trial results suggest the approach could extend survival substantially compared with conventional chemotherapy, with a better quality of life, since chemotherapy can leave patients debilitated by side effects. Dee's story, the report notes, is not evidence that the specific drug Pankind is championing works, but it shows what access to experimental treatment can mean.

The obstacle is time and money. The drug has not yet been submitted for Australian approval, and Stewart fears the system will not move fast enough once it is. Listing a new medicine on the Pharmaceutical Benefits Scheme takes a median of 22 months. "We don't have two years," Dee tells AAP, calling the wait for drugs that could give patients more time "inhumane."

The anticipated price could exceed $400,000 per patient. Dr Lee points to melanoma, where immunotherapy costs the government about half a billion dollars a year. A decade ago, patients with metastatic melanoma often died within a year; now more than half can survive 10 years. That kind of long-term survival makes the economic case easier. Pancreatic cancer treatments might add months rather than years. "The healthcare dollars do only stretch so far," Lee says, and for pancreatic cancer the line falls "on the wrong side of the fence."

Pankind is asking the federal government to act on a recommendation from its 2024 Health Technology Assessment Review that could speed PBS access to treatments addressing urgent unmet needs. The group says the government has signalled interest to the drug's manufacturer on behalf of pancreatic cancer patients, and has launched an Every Week Matters campaign. Dee intends to be one of its voices while she can. "We die waiting," she says. The next marker to watch is when the drug is submitted in Australia.