A perspective on surrogate endpoints in controlled clinical trials.
The last couple of decades have seen a large amount of activity in the area of surrogate marker and surrogate endpoint validation, both from a clinical and a statistical perspective. Prentice made a pivotal contribution in the context of a single trial. Subsequently, the framework he proposed has been discussed, criticized, and extended. An important class of extensions considers several rather than a single trial. Recently, a lot of work has been done in this so-called hierarchical or meta-analytic framework. In this paper, we review both the single trial and the hierarchical framework. A number of applications, scattered throughout the literature, are brought together. We outline the statistical issues involved in trying to validate surrogate endpoints. Clearly statistical evidence should only be seen as a component in a decision making process that also involves a number of clinical and biological considerations.
Molenberghs
Geert
G
Center for Statistics, Limburgs Universitair Centrum, Diepenbeek, Belgium.
Burzykowski
Tomasz
T
Alonso
Ariel
A
Buyse
Marc
M
D018931:Antineoplastic Agents, Hormonal / Q000627:therapeutic use; D015415:Biomarkers; D018849:Controlled Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D007093:Imidazoles / Q000627:therapeutic use; D008297:Male; D015233:Models, Statistical; D011237:Predictive Value of Tests; D011471:Prostatic Neoplasms / Q000188:drug therapy; D016896:Treatment Outcome
D018931:Antineoplastic Agents, Hormonal; D015415:Biomarkers; D007093:Imidazoles; C061121:liarozole