Powering Progress: A plan to increase clinical trials for mitochondrial disease in Australia

Mitochondrial disease (mito) comprises more than 350 genetic disorders affecting approximately one in 4,300 people, with no cure and few effective treatments. With over 60 therapies in development and more than 25 clinical trials commenced globally, Australia has taken part in some trials but missed out on many others. Developed by Mito Foundation and Research Australia from a literature review, an analysis of recent mito trials and multi-stakeholder consultation, this ten-year action plan aims to make Australia an attractive and sustainable location for mito clinical trials. It identifies three priority areas, each with actions for clinician researchers, industry, governments and patient organisations: building clinical research capacity, supporting the mito community to participate in trials, and strengthening collaboration. Contributing work streams address data infrastructure, non-industry-led trials, registration and reimbursement, and policy improvement. The plan also sets out an implementation approach, roles and responsibilities, and evaluation metrics for monitoring progress.