Benefit–risk assessment in medicine has been a valuable tool in the regulation of medicines since the 1960s. Benefit–risk assessment takes place in multiple stages during a medicine’s life-cycle and can be conducted in a variety of ways, using methods ranging from qualitative to quantitative. Each benefit–risk assessment method is subject to its own specific strengths and limitations. Despite its widespread and long-time use, benefit–risk assessment in medicine is subject to debate and suffers from a number of limitations and is currently still under development.
This state of the art review paper will discuss the various aspects and approaches to benefit–risk assessment in medicine in a chronological pathway. The review will discuss all types of benefit–risk assessment a medicinal product will undergo during its lifecycle, from Phase I clinical trials to post-marketing surveillance and health technology assessment for inclusion in public formularies. The benefit–risk profile of a drug is dynamic and differs for different indications and patient groups. In the end of this review we conclude benefit–risk analysis in medicine is a developed practice that is subject to continuous improvement and modernisation. Improvement not only in methodology, but also in cooperation between organizations can improve benefit–risk assessment.