Increasing clinical trial activity in mitochondrial disease in Australia

This stakeholder consultation summary documents the evidence base underpinning the Mito Foundation and Research Australia action plan for increasing clinical trials in mitochondrial disease. Consultation comprised 14 semi-structured interviews with Australian and international clinician researchers, patient organisations, industry and a clinical trials network; online surveys of the mito community, clinicians and researchers, and industry; and workshops at the 2024 AussieMit conference and Mito Community Summit. Participants viewed Australia as an attractive trial location because of its health data quality, trial governance and skilled workforce. Reported barriers included industry focus on larger markets, unfunded pre-trial activities, unsustainable per-visit funding models, limited protected research time and dedicated trial teams, variable site capacity, multi-site governance delays, disease heterogeneity, low rates of genetic diagnosis and the absence of a comprehensive patient registry. Community respondents showed high interest in trial participation. Low response rates from New Zealand limited the scope to Australia, though the findings may inform efforts for other rare diseases.