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: The open-source adaptive clinical trial platform has been developed and it being used with three rare disease populations (motor neurone disease, cystic fibrosis and Angelman syndrome). Twelve companies and 194 users are using the platform.

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