Smith MY, Marsh K, Hauber B, Disantostefano R. Quantitative benefit-risk assessment: What methods are being used? How far has industry come? Poster presented at the 2020 DIA Virtual Conference; June 14, 2020. Washington, DC.


Quantitative benefit risk assessment (qBRA) involves the incorporation of quantitative data on stakeholder preferences into the assessment of the benefit-risk profile of a medicinal product or device. This includes methodological approaches such as multi-criteria decision analysis (MCDA), discrete choice experiments (DCEs), and net-clinical benefit. Often these approaches utilize patients’ preference to weigh risks and benefits, but other stakeholders’ preferences are also used in some instances as well.

Many life science companies are doing more qBRA. This is partly stimulated by regulatory demand for this data, in particular from the FDA. But the uses extend beyond supporting regulatory submissions, including supporting internal decisions and reimbursement submissions.

Implementation of qBRA raises challenges. For what technologies and decisions should it be used? Who should decide this, given the multiple stakeholders interested in qBRA? Whose preferences should be incorporated into the qBRA, and what methods should be used to do so? How should qBRA be implemented, given the multi-disciplinary nature such analysis?

Companies are adopting different approaches to answering these questions. But to date no research has been undertaken to learn the lessons from this experience. To fill this gap, qualitative interviews were undertaken with 20 qBRA leads from a representative sample of life sciences companies. The results of the research describe the extent and nature of qBRA currently being undertaken in companies, decision making around qBRA, how it is implemented, challenges and critical success factors to implementing qBRA, and future directions for qBRA.

This session will be organized into three sections. In section 1 the results of research with qBRA leads in life sciences companies will be presented. In section 2, individuals serving in these positions will reflect on the findings of the research and share and discuss their own experiences in a panel discussion. Lastly, in section 3 the session will conclude with a Questions and Answers session between the speaker panel and the audience.

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