Optimal design of trials to demonstrate the utility of genomically‐guided therapy: Putting Precision Cancer Medicine to the test. Issue 5 (15th July 2014)

• Record Type: Journal Article
• Authors: Dienstmann, Rodrigo; Rodon, Jordi; Tabernero, Josep
• Other Names: Mendelsohn John specialEditor.; Ringborg Ulrik specialEditor.; Schilsky Richard specialEditor.
• Is Part Of: Molecular oncology. Volume 9:Issue 5(2015:May)
• Page Start: 940
• Page End: 950
• Publication Date: 2014-07-15
• Subjects: Clinical trial design -- Precision Cancer Medicine -- Targeted therapy; Cancer -- Molecular aspects -- Periodicals; 616.994005
• DOI: 10.1016/j.molonc.2014.06.014
• Languages: English
• ISSNs: 1574-7891

Abstract

Abstract : The new age of Precision Cancer Medicine, with specific biomarkers being used to direct targeted agents, generally concerns only a subset of patients within a certain histopathologically defined tumor type. This paradigm is challenged by the need to perform widespread molecular screening in certified laboratories, with results available to clinicians within reasonable timeframe. Tumor heterogeneity and clonal evolution must be considered in the decision making process. Adaptive and innovative clinical trial designs exploring predictive algorithms and reconsideration of traditional efficacy endpoints are required to rapidly translate scientific discoveries into patient care. Furthermore, international collaboration in cancer research and open discussions on the availability of investigational agents will likely redefine the drug development and approval process in the coming years. Highlights: Co‐developing biomarkers and drugs is crucial in genomically‐guided therapies. Innovative trials have adaptive designs for patient accrual and drug selection. Trials with novel efficacy endpoints in non‐advanced settings are anticipated. Circulating biomarkers may help individualize therapy in acquired resistance setting.