Evaluation of new clinical trials designs
Prof. Margaret Kelaher
This research project aims to a) identify, develop and evaluate strategies to improve patient participation in cancer clinical trials at the Victorian Comprehensive Cancer Centre including implementation of novel clinical trial methodologies; b) evaluate and improve the implementation of registry-based randomised controlled trial (RRCT) and; c) assess the characteristics of clinical registries that increase their utility for both RRCT and data-driven clinical and health services research.
Randomised controlled trials (RCTs) are regarded as the gold standard for evaluating the effectiveness of interventions. However, RCTs are complex and expensive to perform. They have highly selected populations, low recruitment rates and limited real-world generalisability. Alternative trial models have been sought to overcome these challenges including RRCTs. In RRCTs, eligible patients who are enrolled in a registry are recruited to participate in RCTs. Patients in routine care are then randomly allocated to alternative treatments. The outcome of the treatment is followed up in the registry, with patients’ existing baseline medical history previously recorded in the registry. RRCTs have the potential to provide a cost-effective clinical trial methodology for RCTs with large numbers of enrolled patients that answer pragmatic but important clinical questions and can change clinical practice. Despite these benefits, there are several challenges in the design and implementation of RRCT that must be address including registry data quality, regulatory and ethical issues, costs and novelty.
Professor Margaret Kelaher
Victorian Comprehensive Cancer Centre