Researchers Propose New Approach to Phase II Clinical Trial Design

Haitao Pan

Haitao Pan, PhD, Assistant Professor, St. Jude Children's Research Hospital

Scientists from St. Jude Children’s Research Hospital have proposed a new method for designing Phase II clinical trials and created a corresponding software to implement the approach.

The work was recently detailed in the journal Pharmaceutical Statistics. The corresponding authors are Haitao Pan, PhD, of the St. Jude Department of Biostatistics, and Jianrong Wu, PhD, of the Markey Cancer Center at University of Kentucky.

“In rare cancers, like pediatric cancers, using historical data is a pragmatic approach for speeding up clinical trials,” Pan said. “The proposed method focuses on a kind of Phase II trial with characteristics of a single-arm trial, using historical control data and a survival endpoint.”

In recent years, drug development in oncology has broadened from mainly considering randomized controlled trials to subtype-specific clinical trials. The latter often rely on historical controls, Pan said.

“Though the randomized controlled trial is the established gold standard for clinical research, in recent years there has been an increasing number of single-arm studies, particularly in the areas of oncology with Phase I expansion cohorts, as well as in rare diseases,” Pan said. “Such an approach is better suited for cases where a randomized trial would be unethical or impractical, and for smaller trials to facilitate quicker access to novel therapies for patients.”

The authors present an event-driven approach for Bayesian one-stage and two-stage, single-arm, Phase II trial designs. Their theoretical and empirical findings aim to help investigators who want to design a trial using the Bayesian approach attain the kinds of operating characteristics often required by regulatory agencies. The software they developed, called BayesDesign, can be accessed at no cost at

The study was funded by the National Cancer Institute (CA177558) and ALSAC, the fundraising and awareness organization of St. Jude.

Edited by Gary Cramer