Statistical controversies in clinical research: assessing pathologic complete response as a trial-level surrogate end point for early-stage breast cancer.

ABSTRACT: BACKGROUND:A trial-level surrogate end point for a randomized clinical trial may allow assessment of the relative benefits of the treatment to be performed at an earlier time point and potentially with a smaller sample size. However, determining whether an end point is a reliable trial-level surrogate based on results of previous trials is not straightforward. The question of trial-level surrogacy is easily confused with the question of individual-level surrogacy, and this confusion can lead to controversy. A recent example concerns the evaluation of pathologic complete response (pCR) as a surrogate for event-free survival (EFS) and overall survival (OS) in early-stage breast cancer. MATERIALS AND METHODS:The differences between individual-level surrogacy (i.e. for patients receiving a spe

SUBMITTER: Korn EL 

PROVIDER: S-EPMC4701120 | biostudies-literature | 2016 Jan

REPOSITORIES: biostudies-literature