Research overview
In stark contrast to common disease, 95% of the collectively common rare diseases lack a specific drug treatment. Access to a clinical trial is typically the only chance for a child with a rare disease to access a therapeutic intervention. There are around 63,000 children in Western Australia living with a rare disease, with most having no therapeutic intervention available, and only a small minority accessing clinical trials. Where a clinical trial is accessed, children and their families are travelling interstate or overseas, incurring great personal and financial cost. Local trial access is often limited by a lack of rare disease clinical trial infrastructure, perpetuating a lack of clinical trial experience in WA. Solutions to improve clinical trial infrastructure locally are desperately needed.
The Adaptive Rare Disease Platform Trial (ARDaPT) will deliver a streamlined, scalable and reusable infrastructure for multiple therapeutic interventions across all types of rare disease to address inequitable access, and support more timely treatment development.
ARDaPT will determine how an adaptive platform trial design can be used to investigate the efficacy of potential therapeutic treatments in rare diseases whilst improving patient outcomes and reducing time to trial approval. Rare disease clinical trial access will improve dramatically because of this infrastructure, enabling more, and timelier, industry and local trials partners.
The expected outcomes of ARDaPT include the establishment of approved clinical trial infrastructure that has regulatory approval, publication of trial protocol, reduction trial approval duration, and the improvement of clinical trial access for patients suffering from rare diseases.
Project researchers
Dr Bradley MacDonald
Partners
Perth Children’s Hospital
Funders
Women and Infants Research Foundation
Project timeline
2025 – 2026
