Periodic benefit-risk assessment using Bayesian stochastic multi-criteria acceptability analysis.

Journal Article (Journal Article)

Benefit-risk (BR) assessment is essential to ensure the best decisions are made for a medical product in the clinical development process, regulatory marketing authorization, post-market surveillance, and coverage and reimbursement decisions. One challenge of BR assessment in practice is that the benefit and risk profile may keep evolving while new evidence is accumulating. Regulators and the International Conference on Harmonization (ICH) recommend performing periodic benefit-risk evaluation report (PBRER) through the product's lifecycle. In this paper, we propose a general statistical framework for periodic benefit-risk assessment, in which Bayesian meta-analysis and stochastic multi-criteria acceptability analysis (SMAA) will be combined to synthesize the accumulating evidence. The proposed approach allows us to compare the acceptability of different drugs dynamically and effectively and accounts for the uncertainty of clinical measurements and imprecise or incomplete preference information of decision makers. We apply our approaches to two real examples in a post-hoc way for illustration purpose. The proposed method may easily be modified for other pre and post market settings, and thus be an important complement to the current structured benefit-risk assessment (sBRA) framework to improve the transparent and consistency of the decision-making process.

Full Text

Duke Authors

Cited Authors

  • Li, K; Yuan, SS; Wang, W; Wan, SS; Ceesay, P; Heyse, JF; Mt-Isa, S; Luo, S

Published Date

  • April 2018

Published In

Volume / Issue

  • 67 /

Start / End Page

  • 100 - 108

PubMed ID

  • 29505866

Pubmed Central ID

  • PMC5972390

Electronic International Standard Serial Number (EISSN)

  • 1559-2030

Digital Object Identifier (DOI)

  • 10.1016/j.cct.2018.02.016


  • eng

Conference Location

  • United States