Estimation of ascertainment bias and its effect on power in clinical trials with time-to-event outcomes

Stat Med. 2021 Feb 28;40(5):1306-1320. doi: 10.1002/sim.8842. Epub 2020 Dec 14.

Abstract

While the gold standard for clinical trials is to blind all parties-participants, researchers, and evaluators-to treatment assignment, this is not always a possibility. When some or all of the above individuals know the treatment assignment, this leaves the study open to the introduction of postrandomization biases. In the Strategies to Reduce Injuries and Develop Confidence in Elders (STRIDE) trial, we were presented with the potential for the unblinded clinicians administering the treatment, as well as the individuals enrolled in the study, to introduce ascertainment bias into some but not all events comprising the primary outcome. In this article, we present ways to estimate the ascertainment bias for a time-to-event outcome, and discuss its impact on the overall power of a trial vs changing of the outcome definition to a more stringent unbiased definition that restricts attention to measurements less subject to potentially differential assessment. We found that for the majority of situations, it is better to revise the definition to a more stringent definition, as was done in STRIDE, even though fewer events may be observed.

Keywords: ascertainment bias; cluster-randomized trial; study power; time-to-event data.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Aged
  • Bias*
  • Humans