Novel Bayesian Quantification of Benefit-Risk Balance in Rare Diseases

Music City Center 

Assessing the benefit-risk profile is essential throughout the entire lifecycle of a medical product to ensure that the benefits outweigh the risks (FDA. 2023. Benefit-risk assessment (BRA) for new drug and biological products: Guidance for industry: Final guidance). Conducting rigorous, evidence-based BRA can be challenging, as it involves making these assessments transparent and entirely measurable during development, approval, and post-marketing stages. These difficulties are more pronounced for rare diseases due to limited availability of clinical data during their development phases. Furthermore, the lack of control data adds complexity to BRA. This presentation addresses how to achieve a transparent and fully measurable benefit-risk balance in such cases using an innovative Bayesian approach. The approach utilizes both stakeholder preference elicitation and clinical trial results and accounts for uncertainties in those measures. A discussion on an R-shiny application for implementing this methodology will also be included.

Quantiative Benefit-Risk Assessment

Bayesian Method

Preference trade-off