Considerations in Designing Clinical Trials of Novel Antibacterial Agents Using a Superiority Design.

Abstract

Randomized controlled trials for the regulatory approval of antibacterial agents almost always use a noninferiority design. Indeed, this should always be the default option when clinical development of new antibacterial agents is being planned. However, a superiority design may need to be considered for "nontraditional" antibacterial agents that are added to standard of care therapy. Therefore, it is necessary to consider how superiority trials might best be designed. Based on Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) antibacterial development discussions, we outline some key considerations for evaluable study populations, trial design, endpoints, and regulatory liaison in the context of superiority trials for new antibacterial agents.