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11 Dec 2019
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Using clinical registries to inform trial design

  • Victorian Comprehensive Cancer Centre

Approaches to clinical trials are changing with the trend towards personalised medicine, as the discovery of new biomarkers and genetic mutations for smaller populations create a difficult environment for designing clinical trials.

Increasingly, cancer treatments target small populations, are given in combination, and at times, pose a significant cost to the health system. Through the VCCC’s alliance collective expertise, there is an opportunity to develop smart, adaptive clinical trials to translate discoveries and enable improved patient health outcomes.

Building evidence for optimal treatment

University of Melbourne, Chair of Cancer Health Services Research, Professor Maarten IJzerman recently joined VCCC thought-leaders to discuss new ways and opportunities for new trials such as the use of single-arm trials, adaptative trials and registry trials.

“The key challenge is to learn from patients, including those who are not on a trial. So, the ideal would be to enrol all patients onto a registry allowing design of adaptive trials within the registry, so we can collect data on all treatments and outcomes and therefore we can learn from every single patient,” Professor IJzerman said.

"There is a trend is to approve drugs based on single-arm trials, for instance in studies where it is difficult to randomise patients, such as those with a rare cancer or undergoing expensive treatments like CAR-T."

The VCCC-hosted discussion led to opportunities for trial design collaborations and novel research methodologies, “It is possible to emulate trials using synthetic control arms and registries, we could match patients from the registry to a single-arm trial,” said Professor IJzerman.

Although these methods are being tested, there remains a concern that many drugs are approved without sufficient comparative evidence. The group therefore was concerned about the validity of new study designs without randomization schemes, such as in SMART or adaptive randomised designs.

Combining conventional trial methodology with registry systems

The VCCC Registry Trials Program has gained significant interest nationally, led by VCCC Research and Education Lead, Gastro-intestinal Cancers, Professor Peter Gibbs. Four trials have been piloted across multiple regional and metropolitan clinical trial sites. The program aims to accelerate recruitment and produce real-world data from clinical registries; ultimately to evaluate the efficacy of commonly used treatments in large patient cohorts.

Professor IJzerman said, “Several of the VCCC members have high-quality clinical registries, and it would be fantastic if every tumour stream could have an associated registry that collects clinical data in a standardised way and links with comprehensive data analysis. Clinical registries can inform trial design, increase efficiencies and optimise treatments for patients.”

The group will meet again in the future to assess progress in clinical trial design, as well as investigate new opportunities in the field.