Compressing drug development timelines in oncology using phase '0' trials.
The optimal evaluation of molecularly targeted anticancer agents requires the integration of pharmacodynamic assays into early clinical investigations. Phase '0' trials conducted under the new Exploratory Investigational New Drug Guidance from the US Food and Drug Administration can provide a platform to establish the feasibility of assays for target modulation in human samples, evaluate biomarkers for drug effects and provide pharmacokinetic data. Phase 0 trials could facilitate rational drug selection, identify therapeutic failures early, and might compress timelines for anticancer drug development. We expect that such trials will become a routine part of early-phase oncological drug development in the future.
Kummar
Shivaani
S
Center for Cancer Research, SAIC-Frederick, Inc., NCI-Frederick, Frederick, Maryland, USA.
Kinders
Robert
R
Rubinstein
Larry
L
Parchment
Ralph E
RE
Murgo
Anthony J
AJ
Collins
Jerry
J
Pickeral
Oxana
O
Low
Jennifer
J
Steinberg
Seth M
SM
Gutierrez
Martin
M
Yang
Sherry
S
Helman
Lee
L
Wiltrout
Robert
R
Tomaszewski
Joseph E
JE
Doroshow
James H
JH
D000970:Antineoplastic Agents / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D015195:Drug Design; D004992:Ethics, Medical; D006801:Humans
D000970:Antineoplastic Agents