title: Role of Phase 0 trials in drug development.
abstract: 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.
authors.affiliation: Division of Cancer Treatment and Diagnosis, National Cancer Institute, NIH, Bethesda, MD 20892, USA. doroshoj@mail.nih.gov
authors.forename: James H
authors.initials: JH
authors.lastname: Doroshow
authors.forename: Shivaani
authors.initials: S
authors.lastname: Kummar
mesh_terms: D000970:Antineoplastic Agents / Q000737:chemistry / Q000627:therapeutic use; D001562:Benzimidazoles / Q000737:chemistry / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D055808:Drug Discovery; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy
chemical_list: D000970:Antineoplastic Agents; D001562:Benzimidazoles; C521013:veliparib