How Are Adaptive Trial Designs Improving Efficiency In Clinical Research?

By Jennifer Dennis-Wall, Ph.D.

The ability to adapt is a valued attribute in today’s constantly changing world, and clinical trial designs are no exception. Adaptive trial designs can save sponsors significant time and money, but they need to be approached with caution and planning.

Historical context

Study design used to be straightforward and included only a few main types: randomized controlled trials (RCTs), cohort studies, case studies, and the like. The need for adaptive designs, or designs that have dynamic treatment protocols guided by study progress and interim data, arose from the limitations of RCTs. Even though RCTs are considered to be the gold standard, they are demanding financially when large sample sizes and long periods of time are needed to see the desired effect. On top of that, the traditional phase I-IV schema for novel drug development means that recruitment restarts at the beginning of each phase.

Adaptive designs can allow for quicker decisions based on interim analyses. They also often feature shorter study durations (when phases are combined), minimized recruiting efforts (when the same patients from one stage can continue on into the next stage), and a lower overall cost. The FDA even supports these innovations and is putting out new guidance all the time about different trial design options to accommodate ethical and practical considerations.