Efficiency of Adaptive and Individualized Designs Relative to Fixed Treatment

• Activity Number: 616
• Type: Contributed
• Date/Time: Wednesday, August 12, 2015 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Russell Reeve*
• Companies: Quintiles
• Keywords: dose response ; randomized concentration-controlled trial ; recruitment rate ; regional trial

Abstract

Adaptive trials have been touted as a methodology that improves the efficiency of clinical trials, by adaptive the trial to the observed data, and thereby allowing for improved trial design properties. But this is not necessarily the case in many circumstances. The efficiency of the trial is a function of several characteristics, including (1) recruitment rate; (2) site by treatment interactions; (3) data analysis; (4) knowledge of dose-response function. Indeed, if one has good knowledge of the dose-response function, this it may be deleterious to engage in an adaptive design, whereas if one has little accurate knowledge of the dose-response relationship, then the adaptive design will be beneficial. In this talk, we look at the efficiency of two types of adaptive designs: (A) Unblinded sample-size re-estimation; (B) Dose adaptive design. We look at the relationship of efficiency as a function of recruitment rate and degree of site by treatment interaction. We find the recruitment rate at which the adaptive design is optimally efficient, and times when the adaptive design is inefficient. Finally, we compare the design to an individualized design, such as the randomized-concen