Surrogate Endpoints: Application in Pediatric Clinical Trials

Abstract

This chapter examines the use of surrogate endpoints in clinical research in general and the role that they can play in pediatric research in particular. A surrogate endpoint does not directly measure clinical impact, but rather reflects the desired therapeutic treatment effect. With the anticipated increase in pediatric clinical trials, the need for surrogate endpoints will increase concomitantly. One must proceed cautiously to ensure that the selection of surrogate endpoints is based on sound scientific rationales. Noted failures of surrogate endpoints to assess treatment effects adequately in adults must not be repeated in pediatric clinical trials, and the concern over inadequately assessing the benefit risk ratio with a surrogate endpoint is of even greater concern in the pediatric population. Trial designs should facilitate the evaluation of proposed surrogate endpoints, including standardizing collection methods and the frequency of biomarker measurements, to allow for across-study analyses to be performed.