Benefit–Risk Assessment

Abstract

While medicines have been assessed in terms of their benefits and risks since health authorities started requiring clinical studies in the 1960s, only recently more formal approaches for conducting benefit–risk evaluations have emerged. Both the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) are in the process of implementing qualitative benefit–risk frameworks, but at the same time, the industry and some other stakeholders have started to pilot quantitative benefit–risk methods in regulatory settings. Multi-criteria decision analysis seems to emerge as one of the preferred quantitative methods, in particular for medicines with a complex multifactorial benefit–risk profile. In this chapter a number of case studies of the use of MCDA for benefit–risk assessment during drug development and for regulatory purposes will be described. Based on these case studies, technical and operational progress, learnings, and challenges will be discussed, and recommendations will be made when and how MCDA can be used for the benefit–risk assessment of medicines. These conclusions will be contextualized within the broader debate of the use of formal benefit–risk methods as decision tools mainly for regulatory purposes.