title: Optimal design of trials to demonstrate the utility of genomically-guided therapy: Putting Precision Cancer Medicine to the test.
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.
authors: Sage Bionetworks, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue North, Seattle, WA 98109-1024, USA. Electronic address: rodrigo.dienstmann@sagebase.org.
authors: Rodrigo
authors: R
authors: Dienstmann
authors: Medical Oncology Department, Vall d'Hebron University Hospital and Institute of Oncology, Universitat Autònoma de Barcelona, Barcelona, Spain.
authors: Jordi
authors: J
authors: Rodon
authors: Medical Oncology Department, Vall d'Hebron University Hospital and Institute of Oncology, Universitat Autònoma de Barcelona, Barcelona, Spain.
authors: Josep
authors: J
authors: Tabernero
mesh_terms: D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D023281:Genomics; D006801:Humans; D009369:Neoplasms / Q000628:therapy*
keywords: Clinical trial design; Precision Cancer Medicine; Targeted therapy