Role of Phase 0 trials in drug development.

Future medicinal chemistry

PubMedID: 21426052

Doroshow JH, Kummar S. Role of Phase 0 trials in drug development. Future Med Chem. 2009;1(8):1375-80.
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.