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. The project facilitated capture of clinical evidence to inform the licensure and funding of new therapeutic products for an initial three rare-diseases.

    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$446,072
    Project durationJune 2018 - November 2020 (Finished)
    ContactProfessor Matt Bellgard, Queensland University of Technology

    Visit Queensland University of Technology for more details.

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