Emerging methods in benefit-risk assessment and decision-making for medicinal products

Shahrul Mt-Isa, Statistician
 

The evaluation of the balance between benefits and risks of drugs is fundamental to all stakeholders involved in the development, registration and use of drugs including patients, health care providers, regulators and pharmaceutical companies. Currently diverse methods are being used, some of which are not as transparent as the others. Structured approaches to decision making have recently gained regulators’ attention. More quantitative statistical approaches have evolved over many decades that may draw together disparate data from the many sources and formally synthesise them with relevant preference values from stakeholders and present them in ways that can aid decision-making. Nonetheless, there is an absence of widely accepted methods for the quantification of benefit-risk assessment and the visual representation of benefit-risk profiles. In this short tutorial, the challenges in medical decision-making are addressed, an overview of the emerging methods in benefit-risk assessment is discussed, and the prospects of these formal methods in regulatory decision-making are then demonstrated using real-life published data.