PLoS ONE (Jan 2018)

Bayesian sample size determination for cost-effectiveness studies with censored data.

  • Daniel P Beavers,
  • James D Stamey

DOI
https://doi.org/10.1371/journal.pone.0190422
Journal volume & issue
Vol. 13, no. 1
p. e0190422

Abstract

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Cost-effectiveness models are commonly utilized to determine the combined clinical and economic impact of one treatment compared to another. However, most methods for sample size determination of cost-effectiveness studies assume fully observed costs and effectiveness outcomes, which presents challenges for survival-based studies in which censoring exists. We propose a Bayesian method for the design and analysis of cost-effectiveness data in which costs and effectiveness may be censored, and the sample size is approximated for both power and assurance. We explore two parametric models and demonstrate the flexibility of the approach to accommodate a variety of modifications to study assumptions.