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

Shivaani Kummar1, Robert Kinders, Larry Rubinstein

  • 1Center for Cancer Research, SAIC-Frederick, Inc., NCI-Frederick, Frederick, Maryland, USA.

Nature Reviews. Cancer
|January 26, 2007
PubMed

Insights

Phase 0 trials integrate pharmacodynamic assays into early cancer drug development. These trials assess assay feasibility, biomarkers, and pharmacokinetics, potentially accelerating anticancer drug discovery.

Area of Science:

  • Oncology
  • Pharmacology
  • Clinical Trials

Background:

  • Evaluating molecularly targeted anticancer agents needs pharmacodynamic assays in early clinical studies.
  • Current early-phase trials may not fully leverage pharmacodynamic data.

Purpose of the Study:

  • To explore the utility of Phase 0 trials in optimizing the evaluation of novel anticancer agents.
  • To establish Phase 0 trials as a platform for assay feasibility and biomarker assessment.

Main Methods:

  • Utilizing the US Food and Drug Administration's Exploratory Investigational New Drug Guidance.
  • Integrating pharmacodynamic assays and pharmacokinetic data collection within Phase 0 trials.
  • Assessing target modulation and drug effects in human samples.

Main Results:

  • Phase 0 trials can establish the feasibility of pharmacodynamic assays in human samples.
  • These trials provide valuable pharmacokinetic data and biomarker information.
  • Early identification of therapeutic failures and rational drug selection are facilitated.

Conclusions:

  • Phase 0 trials offer a valuable platform for early assessment of molecularly targeted anticancer agents.
  • Integrating these trials can potentially compress anticancer drug development timelines.
  • Phase 0 trials are expected to become a standard in early-phase oncology research.

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