FDA, NIH Award Grant to Study Adaptive Clinical Trial Design

William G. Barsan (University of Michigan)

Researchers from academia and business aim to improve the process for testing therapies and medical devices by investigating a new type of clinical trial called adaptive clinical trials. This kind of clinical study makes adjustments to the trial using information accumulated as patients are enrolled, with the goal of improving clinical trials’ efficiency as a whole.

The three-year project is funded by a grant from the National Institutes of Health/Food and Drug Administration Joint Leadership Council to encourage new research in regulatory science. The project team is led by William G. Barsan (pictured right), chair and director of the University of Michigan Health System’s Department of Emergency Medicine. The team includes Donald A. Berry, senior statistical scientist and founder of Berry Consultants, and Roger J. Lewis, co-chair of the Regulatory and Ethics Knowledge and Research Program and professor of emergency medicine at the University of California–Los Angeles.

Researchers cannot predict when a specific drug or treatment will show therapeutic results during a clinical study. Once a standard trial is underway, characteristics such as inclusion criteria cannot be modified or changed even if data collected before the trials end-date demonstrates that such changes may be necessary.

Patients enrolled later in a study who are randomized to the placebo group, for example, cannot benefit from the therapeutic effect of the treatment, and money will continue to be spent until the trial ends. Current trials often include periodic review of data to search for early benefit or early harm but changes are usually made only when an overwhelming effect is seen.

Adaptive clinical trials can more seamlessly make use of partial data collected using Bayesian statistics, which allow analysts to predict trial success based on early patient responses and other accumulating information.