Opportunities for children to benefit from novel therapies have increased substantially over the past decade. Change is needed to maximise these opportunities, particularly in the paediatric trial environment. Investment and a coordinated national approach are needed to prevent Australian children falling behind their international peers. There is international recognition that collaborative approaches and strategic investment in paediatric clinical trials reap benefits in terms of access to clinical trials and new therapies. This article exemplifies a multi‐state collaboration and presents a united call to action to prioritise paediatric clinical trials, by strategically investing in effective governance and infrastructure, and a shift in culture that embeds clinical trials in the core business of paediatric health care and academic institutions.
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The Murdoch Children's Research Institute and Sydney Children's Hospitals Network have received funding from Roche to aid in building capacity for gene therapy in Australia. All authors’ hospitals and/or institutions are active sites for pharmaceutical company‐sponsored clinical trials and receive enrolment‐based reimbursement on a trial‐by‐trial basis, but do not receive direct funding for clinical trials capacity or capability building unless stated above.