Accelerating precision therapies through digital infrastructure for adaptive trials and trial-ready cohort studies

The project is a feasibility study for the development of a national digital infrastructure to support adaptive clinical trials and ‘trial-ready’ natural history cohort studies for a number of rare diseases.

The digital study is an open-source solution that will improve efficiencies and seamlessly capture patient-reported data in drug evaluation through trial enrolment and data collection. It will establish linkage of clinician-entered, health system administrative data into a patient record, supporting the efficient systematic capture of data for trials, and enabling real-time Bayesian analysis for novel trial designs. It is specifically intended to facilitate capture of clinical evidence to inform the licensure and funding of new therapeutic products for an initial three rare-diseases.

Outcomes (FY2020):

  • The open source adaptive clinical trial platform has ben developed and it being used with three rare disease populations (Motor Neurone Disease, Cystic Fibrosis and Angelman Syndrome) and a separate project has been commenced to use the system for COVID-19 clinical trial analysis.

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Consortium leadQueensland University of Technology
Consortium membersMurdoch University, Telethon Kids Institute, MNDi Foundation & Foundation for Angelman Syndrome Therapeutics Australia
MTPConnect grant$200,000
Industry contribution$200,000
Project durationJune 2018- November 2020
ContactProfessor Matt Bellgard, Queensland University of Technology