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Surrogate endpoints in clinical trials: Cardiovascular diseases
Author(s) -
Wittes Janet,
Lakatos Edward,
Probstfield Jeffrey
Publication year - 1989
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.4780080405
Subject(s) - surrogate endpoint , clinical endpoint , endpoint determination , clinical trial , censoring (clinical trials) , sample size determination , surrogate data , medicine , missing data , intensive care medicine , statistics , mathematics , pathology , physics , nonlinear system , quantum mechanics
A surrogate endpoint in a cardiovascular clinical trial is defined as endpoint measured in lieu of some other so‐called ‘true’ endpoint. A surrogate is especially useful if it is easily measured and highly correlated with the true endpoint. Often the ‘true’ endpoint is one with clinical importance to the patient, for example, mortality or a major clinical outcome, while a surrogate is one biologically closer to the process of disease, for example, ejection fraction. Use of the surrogate can often lead to dramatic reductions in sample size and much shorter studies than use of the true endpoint. We discuss several problems common in trials with surrogate endpoints. Most important is the effect of missing data, especially in the face of informative censoring. Possible solutions are the assignment of scores or formal penalties to missing data.