Editorial: Incorporating Phase 0 microdosing as a powerful tool into a new vision of drug development
Frontiers in Pharmacology July 16, 2024 H. Markus Weiss, Yuichi Sugiyama, Esther van Duijn
Phase 0 microdosing—testing compounds at subtherapeutic doses in humans—enables early decisions in drug development with a reduced preclinical safety package. Its uptake has been slow despite advantages such as better decisions informed by early human data, reduced animal experiments, and access to vulnerable populations. Intratarget microdosing (ITM) enhances this approach by assessing safety or efficacy-related biomarkers beyond pharmacokinetics, including target expression via Positron Emission Tomography. The CIVO platform, an injectable device for surface-accessible tumors, can microdose up to eight drugs simultaneously and generate human pharmacodynamic data safely, improving translation over animal efficacy data.