A practical approach for eliciting expert prior beliefs about cancer survival in phase III randomized trial

J Clin Epidemiol. 2009 Apr;62(4):431-437.e2. doi: 10.1016/j.jclinepi.2008.04.009. Epub 2008 Nov 17.

Abstract

Objective: To propose and compare practical approaches that allow eliciting and using expert opinions about the benefit effect on a censored endpoint, such as event-free survival (EFS), used in the planning of a clinical trial based on Bayesian methodology.

Study design and setting: Individual interviews of 37 experts. Bayesian normal models on the log hazard ratio (HR) of EFS were implemented. We illustrate our approach by using a trial of autologous stem cell transplantation (ASCT) vs. chemotherapy (CT) in chronic lymphocytic leukemia (CLL). We elicited experts' prior beliefs about the difference in 3-year EFS between the two treatment arms, either roughly or throughout weights over the difference scale. Subsequently, a Bayesian synthesis of the information reported in the trial protocol with that in the experts' prior was performed, using: (1) the postulated treatment effect based on null (skeptical) and alternative (enthusiastic) hypotheses with shared standard error; and (2) the expected difference derived from experts' distributions.

Results: As compared with the priors based on the trial protocol data, expert priors agreed with some average from enthusiastic and skeptical information, with close standard errors.

Conclusion: This case study illustrates a rational approach to construct an expert-based prior. It should be considered as part of the design of future Bayesian trials.

Publication types

  • Comparative Study

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Attitude of Health Personnel*
  • Bayes Theorem
  • Clinical Trials, Phase III as Topic*
  • Expert Testimony*
  • Female
  • Humans
  • Leukemia, Lymphocytic, Chronic, B-Cell / mortality*
  • Leukemia, Lymphocytic, Chronic, B-Cell / therapy
  • Male
  • Randomized Controlled Trials as Topic*
  • Stem Cell Transplantation
  • Surveys and Questionnaires

Substances

  • Antineoplastic Agents