Improving Clinical Trial Efficiency: Thinking outside the box
Abstract
Design strategies have evolved over the past few years as a means to accelerate the drug development process so that the right therapies can be delivered to the right patients. Basket, umbrella and adaptive enrichment strategies represent a class of novel designs for testing targeted therapeutics in oncology. Umbrella trials include a central infrastructure for screening and identification of patients, and focus on a single tumor type or histology with multiple sub-trials each testing a targeted therapy within a molecularly defined subset. Basket trial designs offer the possibility to include multiple molecularly defined sub-populations, often across histology or tumor types, but included in one cohesive design to evaluate the targeted therapy in question. Adaptive enrichment designs offer the potential to enrich for patients with a particular molecular feature predictive of benefit for the test treatment based on accumulating evidence from the trial. This session will aim to discuss the fundamentals of these design strategies, the underlying statistical framework, the logistical barriers with implementation and ultimately, the interpretation of the trial results. New statistical approaches, extensive multi-disciplinary collaboration and state of the art data capture technologies are needed to implement these strategies in practice. Logistical challenges to implementation arising from centralized assay testing, requirement of multiple specimens, multidisciplinary…
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