Compressing drug development timelines in oncology using phase '0' trials

Shivaani Kummar, Robert Kinders, Larry Rubinstein, Ralph E. Parchment, Anthony J. Murgo, Jerry Collins, Oxana Pickeral, Jennifer Low, Seth M. Steinberg, Martin Gutierrez, Sherry Yang, Lee Helman, Robert Wiltrout, Joseph E. Tomaszewski, James H. Doroshow

Research output: Contribution to journalReview articlepeer-review

173 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)131-139
Number of pages9
JournalNature Reviews Cancer
Issue number2
StatePublished - Feb 2007
Externally publishedYes

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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