Improving the Odds for Better Clinical Trials

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Improving the Odds for Better Clinical Trials

William Rosenberger would like to see more favorable odds for patients in clinical trials. So the University Professor and chair of Mason Engineering’s Department of Statistics is helping medical researchers apply the concepts used in personalized medicine to better design studies—and beneft patients. In personalized medicine, patients get targeted treatments based on their genetics, specifc illness or disease, and other characteristics.

For years, medical researchers have conducted large-scale studies on the general population. They use statistical analyses to evaluate the safety and efectiveness of experimental treatments, such as new medications or medical devices. Many studies have failed because the therapies didn’t work on the population at large, but they may have worked on a subgroup of patients, Rosenberger says.

Instead of using the older protocol, Rosenberger and his doctoral students are developing enrichment design methodology to help scientists pinpoint new experimental treatments that work for some people, as is done in personalized medicine. Here’s one way it works:

Researchers start out by testing several new medical therapies on a large population. At an interim point of the study, they look at the initial results, do some statistical decision-making with the help of statisticians like Rosenberger, and narrow down the treatments to those that seem to be working the best.

Adaptive designs can also identify the patients who are responding best to treatments and weight the study to favor assigning treatments that work most efectively for the patients in the clinical trials. This is called covariate-adjusted response-adaptive (CARA) randomization, Rosenberger says.

He is particularly interested in designing clinical trials for rare diseases, which led him to serve as a member of an international advisory board for a European Union consortium on small population clinical trials. Changing how the studies are conducted could make it easier for people to get new therapies earlier and also potentially save lives.

“The methodology of CARA enrichment designs has the potential to impact the way we think about designing clinical trials in the future,” he says. “It could lead to more efcient study designs that beneft patients.”

—Nanci Hellmich

Assistant Professor Elizabeth Johnson contributed to the U.S. Census Bureau’s Statistics in Schools program for K-12 teachers and students.

Photo by Evan Cantwell