Phase 0 Clinical Trial Strategies for the Neurosurgical Oncologist

In an era of escalating drug discovery costs, shifting priorities within the pharmaceutical industry, and longstanding challenges in central nervous system drug delivery, surgical trials offer an avenue to identify promising agents with demonstrable tumor penetration and molecular effects. The rise of pharmacodynamic- and pharmacokinetic-driven clinical trials, including phase 0 study designs, creates an opportunity for the neurosurgical oncologist to engage drug development for brain tumor patients directly. Here, we review the phase 0 clinical trial mechanism as well as its current and future applications within neurosurgical oncology.