2017 - Budapest - Hungary

PAGE 2017: Benefit-Risk assessment
Andreas Kouroumalis

European regulatory views on benefit-risk assessment methodologies - role of MCDA and other model-based approaches

Andreas Kouroumalis

European Medicines Agency


  • Provide an overview of the current benefit/risk assessment process of drug evaluation in the EU
  • Describe past and ongoing initiatives to further improve benefit/risk analysis
  • Highlight opportunities to support the shift towards quantitative models in benefit/risk

Overview/Description of presentation: Benefit-risk assessment is the cornerstone of drug licensing decisions. Increased interest in the decision making process behind drug approvals have led to significant changes by which drug regulatory authorities conduct their assessments and communicate their decisions. Over the last 10 years, there has been a clear trend towards a more explicit benefit/risk analysis, using structured frameworks, both in Europe and the USA. The European Medicines Agency has explored the possibility to use quantitative methods in its assessments however this has not yet gained support, mainly because the experience of the European regulatory network is very limited with such models as well as the perceived complexity of such methods in the European regulatory context. To gain more experience, the Agency has invited the pharmaceutical industry to explore alternative methods in their assessment of the benefit/risk balance of new medicinal products, including quantitative methods. Acceptance of such methods will depend to a large extent on their uptake by the pharmaceutical industry within their submissions and the perceived usefulness of such approaches by regulators

Conclusions/Take home message: Quantitative methods in benefit/risk assessment in Europe are currently not required. Further evidence and experience are needed to understand the role of quantitative methods in drug regulation. 

Reference: PAGE 26 (2017) Abstr 8414 [www.page-meeting.org/?abstract=8414]
Oral: Benefit-Risk assessment
Click to open PDF poster/presentation (click to open)