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The statistics of phase 0 trials
Larry V Rubinstein1, Seth M Steinberg, Shivaani Kummar
1Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, MD 20892, USA. rubinsteinl@ctep.nci.nih.gov
Statistics in Medicine
|April 27, 2010
Summary
Phase 0 trials, a novel first-in-man study, assess pharmacodynamic (PD) effects of new agents in few patients. These trials efficiently test biologic activity, speeding drug development, especially in oncology.
Area of Science:
- Pharmacology
- Clinical Trials
- Oncology Drug Development
Background:
- Phase 0 trials represent a novel study design for early-stage drug development.
- These trials focus on assessing pharmacodynamic (PD) effects on molecular targets.
Purpose of the Study:
- To introduce statistically efficient and rigorous designs for PD-driven Phase 0 trials.
- To highlight the utility of Phase 0 trials as a proof-of-principle for new therapeutic agents.
- To emphasize the role of Phase 0 trials in accelerating drug development, particularly in oncology.
Main Methods:
- Utilizing pharmacodynamic (PD) assays to measure drug effect on molecular targets.
- Conducting non-comparative trials with a small patient cohort (10-15 individuals).
- Administering pharmacologically active, non-toxic doses to assess predicted PD effects.
Main Results:
- Phase 0 trials serve as an early test of an agent's biologic effect.
- These studies allow for the early identification and elimination of ineffective agents.
- They facilitate the selection of the most promising agents among competing analogues.
Conclusions:
- PD-driven Phase 0 trials are crucial for efficient and rigorous early drug evaluation.
- These trials promise to significantly speed up the development of new therapeutic agents.
- Phase 0 trials are poised to become an increasingly important tool in oncology research.
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