Statistical approaches for evaluating surrogate outcomes in clinical trials: A systematic review |
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| Authors: | Hannah Ensor Robert J. Lee Cathie Sudlow Christopher J. Weir |
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| Affiliation: | 1. Centre for Population Health Sciences, University of Edinburgh Medical School, Edinburgh, UKh.m.ensor@sms.ed.ac.uk;3. Centre for Population Health Sciences, University of Edinburgh Medical School, Edinburgh, UK;4. Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK;5. Edinburgh Health Services Research Unit, University of Edinburgh, Western General Hospital, Edinburgh, UK |
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| Abstract: | The use of surrogate outcomes that predict treatment effect on an unobserved true outcome may have substantial economic and ethical advantages, through reducing the length and size of clinical trials. There has been extensive investigation of the best means of evaluating putative surrogates. We present a systematic review on the evolution of statistical methods for validating surrogates starting from the defining paper of Prentice (1989). We highlight the fundamental differences in the current statistical evaluation approaches, their advantages and disadvantages, and examine the understanding and perceptions of investigators in this area. |
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| Keywords: | Clinical trials statistical methodology surrogate evaluation systematic review |
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