Randomized vs. Single Arm Phase II Oncology Clinical Trials, as Well as Endpoint Selection

• Activity Number: 172
• Type: Contributed
• Date/Time: Monday, August 4, 2014 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Grace Liu*+
• Companies: Janssen
• Keywords: study design ; randomized ; single arm ; Simulation ; phase II trials

Abstract

The main goal of Phase II clinical trials in oncology is to test whether a new treatment has an anticancer effect and/or whether it works against a certain type of cancer. Common used phase II study design is usually single-arm studies and may select multiple-arm trials. Gregory et al. 2011 indicates using single-arm phase II trials resulted in a higher percentage of phase III trials conducted using active agents when there was minimal standard of care activity, or in the presence of a positive historical bias (estimated result is greater than truth). The success rates for phase III trials based on the single arm phase II studies seem to be decreasing. This decrease of phase III studies has led to considerable scientific discussion of single arm versus randomized designs (Rubinstein et al. 2009). Randomized phase II trials performed better in the presence of a negative historical bias, in the presence of high variability, and were more consistent across variation of historical bias. This presentation illustrates several methods regarding to randomized phase II for both response endpoint as well the time to event endpoint. Simulation studies are conducted for comparison of randomize