Challenges and Opportunities for Improving Benefit-Risk Assessment of Pharmaceuticals from an Economic Perspective

This Briefing reports the discussions of a group of experts at an OHE workshop on the benefit-risk assessments of drugs. The objective was to identify how particular tools and methods used in economic analysis and the decision sciences might improve the methodologies that regulatory authorities apply to evaluate drug benefit and risk. Four methods were presented and discussed: health outcomes modelling using quality-adjusted life years, incremental net health benefit, stated benefit-risk preferences, and multi-criteria decision analysis. Each has the potential to improve regulatory benefit-risk assessment by addressing three major issues inherent in the current regulatory approach: transparency of decision making, alignment of preferences, and consistency. However, no single tool is the clear choice to address three issues. Strong arguments were made during the workshop that regulators should consider the preferences of patients and the community in benefit-risk decision making. The strengths and limitations of each of the four methods as an aid in making such decisions were discussed. It was agreed that further developmental work and research is needed to refine and test each of these and identify the circumstances in which each could be used. Although such methods can help inform and improve regulatory decision making, these difficult decisions will always require judgments based on multiple considerations.