title: Compressing drug development timelines in oncology using phase '0' trials.
abstract: The optimal evaluation of molecularly targeted anticancer agents requires the integration of pharmacodynamic assays into early clinical investigations. Phase '0' trials conducted under the new Exploratory Investigational New Drug Guidance from the US Food and Drug Administration can provide a platform to establish the feasibility of assays for target modulation in human samples, evaluate biomarkers for drug effects and provide pharmacokinetic data. Phase 0 trials could facilitate rational drug selection, identify therapeutic failures early, and might compress timelines for anticancer drug development. We expect that such trials will become a routine part of early-phase oncological drug development in the future.
authors.affiliation: Center for Cancer Research, SAIC-Frederick, Inc., NCI-Frederick, Frederick, Maryland, USA.
authors.forename: Shivaani
authors.initials: S
authors.lastname: Kummar
authors.forename: Robert
authors.initials: R
authors.lastname: Kinders
authors.forename: Larry
authors.initials: L
authors.lastname: Rubinstein
authors.forename: Ralph E
authors.initials: RE
authors.lastname: Parchment
authors.forename: Anthony J
authors.initials: AJ
authors.lastname: Murgo
authors.forename: Jerry
authors.initials: J
authors.lastname: Collins
authors.forename: Oxana
authors.initials: O
authors.lastname: Pickeral
authors.forename: Jennifer
authors.initials: J
authors.lastname: Low
authors.forename: Seth M
authors.initials: SM
authors.lastname: Steinberg
authors.forename: Martin
authors.initials: M
authors.lastname: Gutierrez
authors.forename: Sherry
authors.initials: S
authors.lastname: Yang
authors.forename: Lee
authors.initials: L
authors.lastname: Helman
authors.forename: Robert
authors.initials: R
authors.lastname: Wiltrout
authors.forename: Joseph E
authors.initials: JE
authors.lastname: Tomaszewski
authors.forename: James H
authors.initials: JH
authors.lastname: Doroshow
mesh_terms: D000970:Antineoplastic Agents / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D015195:Drug Design; D004992:Ethics, Medical; D006801:Humans
chemical_list: D000970:Antineoplastic Agents