Role of phase 0 trials in drug development

James H. Doroshow, Shivaani Kummar

Research output: Contribution to journalComment/debatepeer-review

2 Scopus citations


Phase 0 trials can provide proof of concept that an agent modulates its intended target without exposing patients to the drug-tolerability testing that precedes efficacy evaluation. Drugs that do not modulate their targets can be discarded, focusing development resources on the most clinically promising agents. However, wider uptake of Phase 0 trials may be hindered by ethical concerns over their lack of therapeutic intent, the substantial resources required to develop and validate target assays or skepticism that such trials save development time.

Original languageEnglish (US)
Pages (from-to)1375-1380
Number of pages6
JournalFuture Medicinal Chemistry
Issue number8
StatePublished - Nov 2009
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology
  • Drug Discovery


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