Figure 6. A novel model for clinical development.
During the exploratory phase of development, this model uses all available knowledge and tools, including biomarkers, modeling, and simulation, as well as advanced statistical methodology. Trials are designed to determine proof-of-concept (POC) and to establish dose selection to a level of rigor that will enhance the likelihood of success in the confirmatory phase. During the confirmatory phase, modern designs, tools, and knowledge are applied to larger-scale studies with the goal of identifying the target patient population in which the drug is efficacious, establishing the benefit-to-risk ratio and confirming the optimal dose and dosing regimen. During this phase, innovative clinical trial designs, such as adaptive or seamless studies, compress timelines, improve dose and regimen selection, and reduce the number of patients assigned to non-viable dosing regimens.
(Reproduced with permission from Orloff J, Douglas F, Pinheiro J, et al. The future of drug development: advancing clinical trial design. Nature reviews. Drug discovery. 2009;8:949-957)