title: Surrogate endpoint validation: statistical elegance versus clinical relevance.
abstract: A variety of approaches have been proposed to provide formal and informal validation of proposed surrogate markers. To achieve true clinical impact, the validation must convince both the statistical and clinical communities. In this paper, we argue that the best approach is not a single method but a multi-faceted exploration, using multiple approaches, including those that directly appeal to clinicians but with less statistical foundation and those arising from statistical considerations but more difficult to interpret clinically. We illustrate our approach using data from clinical trials in both early and advanced colorectal cancer.
authors.affiliation: Division of Biostatistics, Mayo Clinic, Rochester, MN, USA.
authors.forename: Em
authors.initials: E
authors.lastname: Green
authors.forename: G
authors.initials: G
authors.lastname: Yothers
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Sargent
mesh_terms: D015415:Biomarkers; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D015179:Colorectal Neoplasms / Q000503:physiopathology / Q000628:therapy; D006801:Humans; D016016:Proportional Hazards Models; D016019:Survival Analysis
chemical_list: D015415:Biomarkers