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Role of Phase 0 trials in drug development

James H Doroshow1, Shivaani Kummar

  • 1Division of Cancer Treatment and Diagnosis, National Cancer Institute, NIH, Bethesda, MD 20892, USA. doroshoj@mail.nih.gov

Future Medicinal Chemistry
|March 24, 2011
PubMed

Insights

Phase 0 trials offer early proof of concept for drug target modulation, enabling the discard of non-modulating agents. This approach can optimize drug development resources by focusing on promising candidates early.

Area of Science:

  • Pharmacology
  • Clinical Trials
  • Drug Development

Background:

  • Phase 0 trials provide proof of concept for target modulation.
  • They precede drug-tolerability and efficacy testing.
  • Early identification of non-modulating agents can save resources.

Purpose of the Study:

  • To evaluate the utility of Phase 0 trials in drug development.
  • To assess the potential of Phase 0 trials to optimize resource allocation.
  • To address barriers to the wider adoption of Phase 0 trials.

Main Methods:

  • Utilizing Phase 0 trials for early target modulation assessment.
  • Discarding agents that fail to modulate targets.
  • Focusing development on agents demonstrating target modulation.

Main Results:

  • Phase 0 trials can confirm target modulation without full tolerability testing.
  • Development resources can be focused on clinically promising agents.
  • Non-modulating drugs can be identified and deprioritized early.

Conclusions:

  • Phase 0 trials offer a valuable early step in drug development.
  • Potential barriers include ethical concerns, resource intensity, and skepticism about time savings.
  • Wider adoption requires addressing these challenges.

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