title: Prentice's approach and the meta-analytic paradigm: a reflection on the role of statistics in the evaluation of surrogate endpoints.
abstract: We put a perspective on the strengths and limitations of statistical methods for the evaluation of surrogate endpoints. Whereas using several trials overcomes some of the limitations of a single-trial framework (Prentice, 1989, Statistics in Medicine 8, 431-440), arguably the evaluation of surrogate endpoints can never be done using only statistical evidence but such evidence should be seen as but one component in a decision-making process that involves, among others, a number of clinical and biological considerations. We briefly present a hierarchical framework that incorporates ideas from Prentice's work and is uniformly applicable to different types of surrogate and true clinical outcomes.
authors.affiliation: Center for Statistics, Limburgs Universitair Centrum, Diepenbeek, Belgium. ariel.alonso@luc.ac.be
authors.forename: Ariel
authors.initials: A
authors.lastname: Alonso
authors.forename: Geert
authors.initials: G
authors.lastname: Molenberghs
authors.forename: Tomasz
authors.initials: T
authors.lastname: Burzykowski
authors.forename: Didier
authors.initials: D
authors.lastname: Renard
authors.forename: Helena
authors.initials: H
authors.lastname: Geys
authors.forename: Ziv
authors.initials: Z
authors.lastname: Shkedy
authors.forename: Fabián
authors.initials: F
authors.lastname: Tibaldi
authors.forename: José Cortiñas
authors.initials: JC
authors.lastname: Abrahantes
authors.forename: Marc
authors.initials: M
authors.lastname: Buyse
mesh_terms: D001699:Biometry / Q000379:methods*; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D023381:Endpoint Determination / Q000706:statistics & numerical data*; D006801:Humans; D007370:Interferon Type I / Q000627:therapeutic use; D016013:Likelihood Functions; D008268:Macular Degeneration / Q000188:drug therapy / Q000503:physiopathology; D015201:Meta-Analysis as Topic; D015233:Models, Statistical; D011994:Recombinant Proteins; D014792:Visual Acuity / Q000187:drug effects
chemical_list: D007370:Interferon Type I; D011994:Recombinant Proteins