The ‘rare’ case for a reform agenda that changes the reflection

A new policy reform agenda is calling for faster diagnosis, broader access to precision medicine and a national focus on rare cancer survival.

Rare Cancers Australia (RCA) will bring patients, advocates, researchers and industry representatives together in Canberra on 8 September to launch its new report, From rare to recognised: the changing story of cancer. The report seeks to shift the national conversation around patients and families who have too often been left at the margins of the health system.

Among its key recommendations, RCA is calling for governments to establish a national cancer survival target, strengthen personalised supportive care and accelerate diagnosis. It also proposes expanded access to genomic testing, faster pathways to innovative medicines and the establishment of a Rare Cancer Therapies Access Accelerator to dismantle regulatory and funding barriers to emerging treatments.

“We urgently need a fairer approach for the more than 17,000 Australians who die from a rare or less common cancer each year, as well as for all those survivors who currently don’t have the support to live well,” said Christine Cockburn, CEO of Rare Cancers Australia.

Rare cancers account for about 25 % of all cancer diagnoses and are associated with significantly poorer overall survival than more common cancers. Patients frequently receive diagnoses only after disease has advanced, following lengthy and complex diagnostic pathways and amid limited treatment options. For many, that experience is compounded by fear, isolation and a profound sense of uncertainty.

The report also argues that Australia’s understanding of what it means to survive cancer needs to evolve alongside advances in treatment.

“We need to change the cultural mindset around what it means to live with cancer,” said Dr Lisa Briggs, who is living with a rare lung cancer. “The term ‘survivorship’ only works for someone who finishes treatment and goes on to live a long life, often cancer free. But many people living with rare and incurable cancers are surviving years – even decades – while remaining on lifelong treatment. Our experiences are very different, and systems are not designed to support us. They simply haven’t kept pace with change.”

Few advances have altered the trajectory of rare cancers as significantly as genomics. Australia no longer has a genomics evidence gap. It has an implementation gap.

Genomic testing infrastructure has struggled to keep pace with demand, while lengthy turnaround times can have direct consequences for patients with aggressive disease. Many patients are waiting 8-10 weeks for results that can be critical to treatment decisions.

“The tipping point, scientifically, passed about three to five years ago. The major challenge is implementation into routine care through our health system,” stated Professor David Thomas, Chief Science and Strategy Officer at Omico. “We need to move the most mature advances of science into routine healthcare and create a mechanism for access to emerging breakthroughs as a right, not an exception.”

Evidence emerging from Omico’s Molecular Screening & Therapeutics (MoST) study points to the clinical impact of a more genomics-enabled system. Among patients with rare cancers, the study found an extension of survival from 15 to 23 months, alongside improved quality of life, among those receiving therapies supported by at least prospective Phase 2 or higher evidence of clinical benefit.

For patients already navigating the system, however, the case for reform is measured not only in clinical outcomes but in years of life regained.

“I’ve been taking drugs to control my cancer for the best part of 11 years and access to emerging treatments has made that possible. I’ve lived a very productive life, watched my kids grow up,” reflected A/Professor Nigel Marks, who is living with gastrointestinal stromal tumours (GIST). “I just wish it was a little easier for patients: more information, more connection, less arm-twisting and hoop-jumping to get the treatments they need.”

The report argues that the next phase of cancer reform must extend beyond survival to encompass the quality of life of people living with cancer.

“Not only did I have to pay $10,000 every three weeks for my recent treatment while the person sitting next to me with metastatic breast cancer got it on the PBS (our family has paid $250,000 to access treatments so far), but I also wasn’t eligible for the supportive care that the breast cancer patients received. Talk about a double blow!” said Caitlin Delaney AM, who is living with a rare ovarian cancer.

Effective cancer care must be person-centred and responsive to individual circumstances, encompassing physical health, mental wellbeing and quality of life. Delivering that care cannot rest with an individual clinician alone. It requires the health system to operate cohesively around the needs of the patient.

“Delays in accessing innovative cancer medicines don’t just affect health. They affect every part of life. When I was well enough to access effective treatment, I returned to the classroom as a teacher. I advocated for other patients. I volunteered, contributed, paid taxes, and tried to give back to the community that had supported me. My husband could also work uninterrupted as well, and we were both able look after our son,” said Sarah McGoram OAM, who is living with GIST. Access to treatment didn’t just extend my life – it allowed me to keep living it.”

Cancer Australia is currently undertaking a two-year evaluation of the Australian Cancer Plan. The evaluation is assessing progress across the sector against the plan’s two-year goals and identifying areas requiring additional attention to keep implementation on track towards its five-year objectives. It could accelerate progress for people affected by rare and less common cancers.

“To fundamentally change the story of rare cancer, we need to move from project-based to system-level solutions. We need an explicit prioritisation of rare and poor-survival cancers in policy, including in the Australian Cancer Plan, state cancer plans and Medicare programs, to incentivise different ways of working and enable greater agility and focus,” explained Professor Clare Scott AM, Professor of Gynaecological Cancer at the University of Melbourne.

Despite years of consultation and broad consensus that reform is needed, implementation has barely commenced on the Health Technology Assessment (HTA) Review while scientific advances continue to accelerate.

“Government has spent years admiring the problem. The solutions have been workshopped, documented in government reports, tested through government reviews and mapped out in roadmaps,” emphasised Liz de Somer, CEO of Medicines Australia. “We need bold leadership and action. Patients can’t wait. The global geopolitical landscape is definitely not waiting – it is just increasing the pressure. Now is the time to make the changes that will secure equitable access to innovative therapies for Australians for generations to come.”

One significant development came in March with the first pan-tumour Pharmaceutical Benefits Scheme (PBS) listing of BMS’ immune checkpoint inhibitors nivolumab and ipilimumab. Yet the milestone was almost a decade in the making, highlighting the lengthy path from advocacy and scientific evidence to meaningful change.

“We need to move beyond thinking in binaries. It’s not simply government or industry,” commented Professor Robyn Ward AM, Chair of the Pharmaceutical Benefits Advisory Committee (PBAC). “There are other ways to build markets that deliver innovation where traditional commercial incentives don’t exist.”

The future of rare cancer care does not need to mirror the past. The nation holds the data, insights and tools to rewrite the story.

“It’s time for rare cancers to be given the attention and focus that they deserve,” Cockburn stated. “We must take bold and decisive actions now to fix and future-proof our health system. Incremental change and slow adoption of innovation will never deliver what people with rare cancers so desperately need – more time.”

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