More needs to be done to bring life-saving cancer trials to Australian children
13 October 2026: A new white paper released today – Building a Future of Life-Saving Clinical Trials for Children with Cancer – sets out four practical reforms to bring more potentially life-saving cancer trials to Australian children.
The white paper shows that Australian children with cancer are not receiving the same opportunities to access clinical trials and emerging treatments as adults.
Australian clinical trial sites are more than twice as likely as US sites, to be selected for global adult oncology trials, on a per-capita basis, with Australia securing around 5 per cent of global industry-sponsored adult oncology trials.
Yet only seven industry-sponsored paediatric oncology trials were active in Australia in the 12 months to June 2026, and 75 per cent of paediatric oncology trials in Australia are not industry sponsored.
The white paper finds children with cancers including neuroblastoma, sarcomas, brain tumours and some leukaemias have fewer clinical trial opportunities in Australia than children in the United States, United Kingdom or Germany.
Building a Future of Life-Saving Clinical Trials for Children with Cancer notes that Australia already has much of the clinical expertise, research capability and infrastructure necessary to become a global destination for paediatric cancer trials — but structural and commercial barriers are preventing that potential from being realised.
Against a further backdrop that risks the future of clinical trials in Australia, with reports that fund managers are postponing investment decisions in local biotech companies due to Government changes to research and development tax incentives.
More than 1,000 Australian children and adolescents are diagnosed with cancer every year. While around 85 per cent survive, three children still die from cancer every week and approximately 70 per cent of survivors experience varying degrees of long-term side effects from their treatment.
For children whose cancer does not respond to existing treatments, access to an early-phase industry-sponsored clinical trial can become one of the few remaining treatment options.
CEO of Medicines Australia Liz de Somer said Australia had already demonstrated that it could compete successfully for global cancer research investment.
“Australia has built a strong reputation for adult cancer clinical trials. The challenge is to make sure Australian children can benefit from similar success.
“Australia must seize the opportunity to build on the ambitions of the Childhood, Adolescent and Young Adult Cancer Roadmap and the Government’s HTA reform agenda. Together, these reforms could help create a stronger pathway from clinical research through to patient access, ensuring more Australian children can benefit from breakthrough cancer treatments.”
The white paper followed the National Paediatric Oncology Industry-Sponsored Clinical Trial Roundtable, convened in July by Children’s Cancer Institute and Medicines Australia’s Oncology Industry Taskforce at the Minderoo Children’s Comprehensive Cancer Centre.
The paper is also timely in the context of the recently released Childhood, Adolescent and Young Adult (CAYA) Cancer Roadmap, which identifies research and clinical trials as a national priority and calls for greater investment, collaboration and equitable access to innovative therapies for children and young people with cancer
Eighteen representatives from patient advocacy, clinical care, research, government and industry examined what would be required to attract more industry-sponsored paediatric oncology trials to Australia.
The paper identifies barriers including limited international industry awareness of Australia’s paediatric oncology capabilities, fragmented data systems, inconsistent infrastructure between states, uncertainty about future reimbursement pathways, and age restrictions that can exclude children and adolescents from trials despite the biology of their cancer making them potentially suitable candidates.
Four practical reforms could make Australia a global destination for children’s cancer trials:
- Leverage existing well-established networks, such as ANZCHOG, to coordinate paediatric oncology clinical trials, bringing together investment, advocacy and engagement with global trial sponsors.
- Connect Australia’s existing cancer data infrastructure, including ZERO, CIRCAA and SACHA, so information can better support clinical decisions, trial matching and evidence generation.
- Expand access nationally through teletrials, hub-and-spoke and shared-care models, so where a child lives does not determine whether they can participate in a trial.
- Better align regulatory and reimbursement settings with paediatric medicine development, including consideration of age-agnostic approaches and clearer treatment of real-world evidence.
Dr Peter Wejbora, Director, Research Development and Partnerships, Children’s Cancer Institute said:
“Cancer is the leading cause of disease-related death for children in Australia. For some children with cancer, a clinical trial may be their best – or only – treatment option, yet too often the right trial is not available in Australia.”
“Access to more targeted, and safer therapies is critical if we want to cure every child of cancer and meaningful change requires a sector-wide effort.”
Australia’s existing infrastructure includes the Zero Childhood Cancer Program, which has already comprehensively analysed cancers from close to 4,000 children and young people, helping identify potential targeted treatments and clinical trial opportunities.
Associate Professor Andy Moore, Chair of the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG) said:
“Phase 3 clinical trials, which are usually large international, cooperative group trials, have driven improved outcomes for childhood cancer over the past 50 years. Australia has played important roles in many of those trials, but too many children still die of their disease or live with long-term side effects of treatment.
“Access to novel treatments, through both industry-sponsored early-phase trials and international cooperative group trials, is critical to improving outcomes further. Australia has the expertise but requires investment and strategic reform to leverage that expertise and bring more trials of the most promising treatments to Australia.”
The white paper concludes that meaningful reform will require cooperation between industry, clinicians, researchers and the Commonwealth, state and territory governments.
Delivering on the vision of the Childhood, Adolescent and Young Adult Cancer Roadmap and Australia’s ongoing HTA reforms will require a coordinated national effort to attract more paediatric cancer trials, generate stronger evidence, and accelerate access to innovative treatments for children and young people with cancer.
The white paper is available here.
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