Efficacy Testing as a Primary Purpose of Phase 1 Clinical Trials: Is it Applicable to First-in-Human Bionics and Optogenetics Trials?

In her article, Pascale Hess raises the issue of whether her proposed modelmay be extrapolated and applied to clinical research fields other than stem cell-based interventions in the brain (SCBI-B) (Hess 2012). Broadly summarized, Hess's model suggests prioritizing efficacy over safety in phase 1 trials involving irreversible interventions in the brain, when clinical criteria meet the appropriate population suffering from "degenerative brain diseases" (Hess 2012). Although there is a need to reconsider the traditional phase 1 model, especially with respect to first-in-human clinical trials involving novel technologies, the question arises as to whether it is appropriate to advocate for a new model that prioritizes efficacy over safety across all phase 1 clini- cal research trials involving irreversible interventions in the brain.