Save Our Sons Duchenne Foundation have commissioned the McKell Institute and Equity Economics to produce a report on the current issues with access to treatments, services and supports for children with Duchenne muscular dystrophy in Australia.  

 This important report will inform Government and key stakeholders on:

  • The costs of health and other care for children with Duchenne muscular dystrophy.
  • The current issues with access to treatments for children, including an assessment of the regulatory barriers to clinical trials in Australia versus comparator jurisdictions which is impacting the establishment of clinical trials for new treatments.
  • The experience of families with Duchenne muscular dystrophy accessing timely and adequate services and supports under the NDIS.

This report will provide key decision-makers with an accurate and true understanding of the system wide costs of the condition and how robust policy and innovative initiatives can help reduce this cost whilst maximising the benefit and positive impact for persons with Duchenne and their families.

Thank you to members of the Duchenne community, our families and children, who participated in the recent family and carer survey which will help to ensure that the report incorporates up-to-date information reflective of the lived experience of families and carers of children with Duchenne.

The report also includes a review of international regulations for clinical trials and differences in time to approve clinical trials in order to answer the important question: what will it take to give Australian children access to clinical trials for new Duchenne treatments?

In recent months Save Our Sons have significantly strengthened our government relations and stakeholder engagement strategies and efforts, including securing a high number of formal meetings at Parliament House in Canberra to discuss issues including regulatory reform required to address the inequitable regulatory and reimbursement process for rare and ultra-rare diseases, approval timeframes, and streamlining the processes for ensuring that clinical trials are undertaken in Australia and that as many Australian children as practicable have access to these clinical trials. Our strong advocacy, government relations and media campaigns continue to gain momentum and critical traction across the board.

Save Our Sons intend to officially launch this report in March this year through a public event. The Duchenne community will be kept closely informed.