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A perspective on surrogate endpoints in controlled clinical trials

Center for Statistics, Limburgs Universitair Centrum, Diepenbeek, Belgium

Tomasz Burzykowski

Center for Statistics, Limburgs Universitair Centrum, Diepenbeek, Belgium

Ariel Alonso

Center for Statistics, Limburgs Universitair Centrum, Diepenbeek, Belgium

Marc Buyse

International Drug Development Institute (IDDI), Brussels, Belgium, marc.buyse{at}iddi.com

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. Prentice¹ 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.

Statistical Methods in Medical Research, Vol. 13, No. 3, 177-206 (2004)
DOI: 10.1191/0962280204sm362ra


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