Emerging Good Practices for Quantitative Benefit-Risk Assessment: A Step Forward [EDITOR'S CHOICE]
Abstract
Balancing the benefits of a health product against its risks is a complex evaluation requiring the availability of relevant and large sets of clinical data and the elaboration of a well-structured analytical framework. Over the past decade, tools for health product benefit-risk assessments have been developed by health authorities to inform regulatory decisions.For example, the European Medicines Agency (EMA) published 5 work package reports on benefit-risk methodology between 2011 and 2015,1 whereas the US Food & Drug Administration (FDA) published 2 guidance documents, “Benefit-Risk Assessment for New Drug and Biologic Products” in 20212 and “Benefit-Risk Determinations for Medical Devices” in 2019.3
EMA and FDA usually utilize a qualitative benefit-risk process (eg, PrOACT-URL4 or BRAT5) that consists of a textual description of the therapeutic context, the favored effects (benefits), the undesirable effects (risks), and conclusions. These frameworks require qualitative judgment on the importance of different benefit and harm outcomes. The 2021 FDA guidance2 suggested that additional analyses may be very useful for difficult benefit-risk assessments (eg, estimating clinical outcomes and integrating benefits and risks in a combined analysis).
EMA and FDA usually utilize a qualitative benefit-risk process (eg, PrOACT-URL4 or BRAT5) that consists of a textual description of the therapeutic context, the favored effects (benefits), the undesirable effects (risks), and conclusions. These frameworks require qualitative judgment on the importance of different benefit and harm outcomes. The 2021 FDA guidance2 suggested that additional analyses may be very useful for difficult benefit-risk assessments (eg, estimating clinical outcomes and integrating benefits and risks in a combined analysis).
Quantitative benefit-risk analysis (qBRA) with explicit outcome weighting can be a valuable tool to help inform decision making when the benefit-risk decision is preference sensitive and qualitative clinical judgment is insufficient. The qBRA can evaluate and account for the uncertainty of outcomes and preference data. In contrast to published frameworks for qualitative or semiqualitative benefit-risk assessments, this ISPOR Good Practices Report, “Quantitative Benefit-Risk Assessment in Medical Product Decision Making,” provides step-by-step guidance for quantitative analysis.
Authors
Salah Ghabri