ABSTRACT
Safety evaluation in biopharmaceutical product development has three primary objectives. First is the identification of signals that are likely or possibly associated with the candidate drug or vaccine. Second is the development of a comprehensive risk management plan to assure that any potential adverse outcomes to patients are minimized and dealt with in an appropriate manner. Third is the assessment of the benefit-risk tradeoff, which is the main focus of this chapter. A favorable benefit-risk (BR) profile has always been the regulatory standard for approval of new drug and vaccine products. However, there are shortcomings with traditional methodologies for B-R analysis, compounded by limitations in the available data. In the Innovative Medicines Initiative (IMI) PROTECT (Pharmacaepidemiological Research on Outcomes of Therapeutics by a European Consortium), Mt-Isa et al. (2014) have identified 49 methodologies categorized as frameworks, metrics, estimation techniques, and utility survey techniques for the assessment of benefit and risk of medicines. The set is further down to recommend 13 of the 49 methodologies. There is now growing interest in approaching the B-R questions with better approaches; see Jiang and He (2016).
