All Times EDT
Keywords: Adaptive Design, Platform Trials
Randomized, controlled trials (RCTs) have been the gold standards for clinical development in recent decades - it is statistically rigorous, operationally understandable and trial results are easy to interpret. However, many RCTs face the same dilemmas – they are time-consuming, costly, and limited to investigate one product on one disease area. Clinical trialists have been discovering new methodologies, both statistically and operationally justifiable, in order to improve the timeline and cost on trial design.
Adaptive design, i.e. group sequential design, sample size re-estimation, is one way that can make clinical trials more efficient. Platform design (master protocol) is another method that can also significantly reduce the time and cost of trials by investigating one product on multiple indications (basket trial) or investigating multiple products on the same disease indication (umbrella trial). Many studies have utilized either adaptive design or platform design and approved successfully. However, what if we combine these two innovative designs together to form the “adaptive platform” design trial that would provide even more flexibilities by adding or removing trials to/from the platform and making decision based on predefined algorithms (FDA Guidance on Adaptive Designs for Clinical Trials of Drugs and Biologics).