Abstract

BackgroundIt has been argued that true endpoints (or ‘hard’ endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost. Other endpoints are termed 'surrogate' endpoints and are intended to substitute and predict the true endpoint. A number of trials that describe using surrogate endpoints use the term ‘pilot’ in the title of the paper but the reason for this, as related by the authors, is the use of these surrogate endpoints in the trial. The conduct and reporting of such a trial may follow the traditional pattern for a conventional randomised controlled trial (RCT) as defined by the original CONSORT statement, with power-based sample size calculations, and significance tests of the results. However, this is contrary to the guidelines of the CONSORT extension for the reporting of pilot trials.Main bodyWe review the definition of a surrogate endpoint and the use of surrogate endpoints in clinical trials. We consider to what extent a trial could be considered a pilot trial if it uses a surrogate endpoint and discuss two examples that illustrate current practice.ConclusionTrials which use surrogate endpoints should only be described as ‘pilot’ when a definitive trial is a distinct possibility and the authors consider conditions which would indicate whether the definitive main trial was worthwhile and feasible. Simply because a trial uses a surrogate endpoint is not justification for calling it a pilot trial.

Highlights

  • It has been argued that true endpoints for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost

  • Surrogate endpoints Weintraub et al [4] have argued that the true endpoints for randomised controlled trials (RCTs), those which are meaningful to clinicians, researchers and patients alike, are limited to health status, survival and cost

  • All other measures may be seen as what are known as ‘surrogate’ endpoints. They have argued that even serious events such as myocardial infarction and stroke may be considered surrogates, as their effect is to adversely affect the critical endpoints of health status, survival, and cost

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Summary

Conclusion

Trials which use surrogate endpoints should only be described as ‘pilot’ when a definitive trial is a distinct possibility and the authors consider conditions which would indicate whether the definitive main trial was worthwhile and feasible.

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