Abdus Wahed received funding to develop SMART patient-centered clinical trial methods

Randomized clinical trials are usually considered the “gold standard” when it comes to testing treatments for patients. A newly funded project led by a Pitt School of Public Health biostatistics professor may change that.

With over $1 million from the Patient-Centered Outcomes Research Institute (PCORI) Abdus Wahed, professor of biostatistics, and his team will launch a three-year project to develop methodological and statistical guidance for a new way of testing treatment sequences through adaptive and sequential clinical trials that better center patient needs and interests.

Called “Sequential Multiple Assignment Randomized Trials,” or SMART, these trials allow for more than one treatment to be given to a patient, or for the treatment to change partway through the trial. They also allow for the trial to change while still underway, so that if one treatment is performing better, more patients are assigned to receive it.

“The goal is to optimize each patient’s outcome with the best therapy or sequence of therapies,” said Wahed. “We want clinical researchers to be able to adjust the treatment at each decision point in their clinical trial without sacrificing the validity of the trial. This way we’ll be able to treat more patients with more effective therapies, exposing less patients to ineffective treatments during the trial.”

SMART trials are generally more statistically complicated than traditional randomized clinical trials that assign half of the trial participants to one treatment and half to another. They also have more opportunities for data to be inadvertently not collected or reported. Wahed and his team will overcome these challenges by developing algorithms that account for missing data, ensuring the trial results are statistically sound.

Co-investigators on this project include Yu Cheng and Zhao Ren of the Department of Statistics, Meredith Wallace of the School of Medicine and Jordan Karp of the University of Arizona.