Australia attracts more than its share of adult cancer trials, but children are being left behind as experts call for urgent reforms to improve access to emerging treatments.
Australian children with cancer are missing out on potentially life-saving treatments because fragmented research infrastructure, regulatory barriers, and limited commercial incentives are preventing international clinical trials from reaching local patients.
A new white paper released today by Medicines Australia and the Children’s Cancer Institute has called for four major reforms to attract more industry-sponsored paediatric oncology trials to Australia.
The report, Building a Future of Life-Saving Clinical Trials for Children with Cancer, revealed a stark disparity between Australia’s success in attracting adult oncology trials and the opportunities available to children.
Australian trial sites were more than twice as likely as their US counterparts to be selected for global adult oncology trials on a per-capita basis, with Australia attracting around 5% of industry-sponsored adult oncology trials worldwide, the report revealed.
But just seven industry-sponsored paediatric oncology trials were active in Australia in the 12 months to June 2026, while 75% of local paediatric oncology trials were not industry-sponsored.
Children with cancers including neuroblastoma, lymphoma, and acute lymphoblastic leukaemia had fewer trial opportunities than their counterparts in the US, UK, and Germany, the report found.
More than 1000 Australian children and adolescents are diagnosed with cancer each year. Although around 85% survive, cancer remains the leading cause of disease-related death in children, while many survivors experience lifelong complications from treatment.
Children’s Cancer Institute director of research development and partnerships Dr Peter Wejbora said access to clinical trials could be critical for children with limited treatment options.
“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,” he said.
“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.”
The white paper followed a national roundtable convened in July by the Children’s Cancer Institute and Medicines Australia’s Oncology Industry Taskforce, which brought together 18 representatives from clinical care, research, government, industry, and patient advocacy.
Participants identified several barriers to attracting international trials, including poor awareness of Australia’s paediatric oncology capabilities, inconsistent infrastructure and funding between jurisdictions, fragmented patient data, and uncertainty around regulatory approval and reimbursement.
Age restrictions were another concern, with adolescents potentially excluded from adult oncology trials despite having cancers with similar biological characteristics.
The report recommended establishing a nationally coordinated consortium to attract international sponsors, building on existing networks such as the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG).
It also called for better integration of existing cancer data platforms, including the Zero Childhood Cancer Program (ZERO), the Cancer Information Registry for Children and Adolescents in Australia (CIRCAA), and the SACHA registry, to improve patient identification, trial matching, and evidence generation.
Expanding tele-trials, hub-and-spoke arrangements, and shared-care models could reduce geographical barriers and allow more children to participate in trials closer to home, the paper said.
The fourth recommendation focused on aligning regulatory and reimbursement arrangements with paediatric drug development, including age-agnostic approaches to medicine access and greater recognition of real-world evidence.
The report highlighted the potential of Australia’s existing research infrastructure, including ZERO, which had enrolled close to 4000 children and young people and used comprehensive genomic analysis to identify targeted treatment options.
However, it warned that Australia was not making full use of these capabilities to attract international investment.
Medicines Australia CEO Liz de Somer said Australia had already demonstrated its ability to compete 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,” she said.
“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 report also raised concerns about the commercial environment for paediatric drug development, with sponsors weighing the cost of conducting Australian trials against the likelihood of obtaining regulatory approval and subsidised access to treatments.
Uncertainty around reimbursement through the Pharmaceutical Benefits Scheme could make Australia a less attractive destination for international research, particularly where small patient populations made conventional evidence requirements difficult to meet.
The report pointed to the Health Technology Assessment Policy and Methods Review recommendation supporting age-agnostic PBS listings where appropriate as one opportunity to address these barriers.
ANZCHOG chair Associate Professor Andy Moore said international cooperative group trials had been instrumental in improving childhood cancer outcomes over the past five decades.
“Phase III clinical trials, which are usually large international, cooperative group trials, have driven improved outcomes for childhood cancer over the past 50 years,” he said.
“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.”
He said access to novel treatments through both industry-sponsored early-phase trials and international cooperative group trials would be essential to further improving outcomes.
“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,” Professor Moore said.
The white paper came as governments considered implementation of the national Childhood, Adolescent and Young Adult Cancer Roadmap, released in May, which identified equitable access to clinical trials and innovative therapies as national priorities.
Its authors said delivering meaningful change would require coordinated action from the commonwealth, states and territories, clinicians, researchers, and pharmaceutical companies.
The full report is available here.
