How to develop treatments for biologically heterogeneous "diseases".
The standard paradigm for the design of phase III clinical trials is not suitable for evaluation of molecularly targeted treatments in biologically heterogeneous groups of patients. Here, we comment on alternative clinical trial designs and propose a prospective discovery/evaluation framework for using tumor genomics in the design of phase III trials.
Biometric Research Branch, Division of Cancer Treatment & Diagnosis, National Cancer Institute, Bethesda, Maryland, USA. rsimon@nih.gov
Richard M
RM
Simon
D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D002289:Carcinoma, Non-Small-Cell Lung / Q000188:drug therapy*; D000068818:Cetuximab; D017326:Clinical Trials, Phase III as Topic / Q000379:methods*; D006801:Humans; D008175:Lung Neoplasms / Q000188:drug therapy*; D012107:Research Design
D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000068818:Cetuximab