A perspective on surrogate endpoints in controlled clinical trials

Stat Methods Med Res. 2004 Jun;13(3):177-206. doi: 10.1191/0962280204sm362ra.

Abstract

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents, Hormonal / therapeutic use
  • Biomarkers*
  • Controlled Clinical Trials as Topic / methods*
  • Humans
  • Imidazoles / therapeutic use
  • Male
  • Models, Statistical*
  • Predictive Value of Tests
  • Prostatic Neoplasms / drug therapy
  • Treatment Outcome

Substances

  • Antineoplastic Agents, Hormonal
  • Biomarkers
  • Imidazoles
  • liarozole