Evaluating benefit-risk: an Agency perspective

In order for a medicinal product to get marketing authorization in Europe, it has to demonstrate a positive benefit-risk balance. Although this has been the cornerstone of the evaluation process, there is no standardised methodology that is used in this context. Recognising the need for a structured approach that can enhance the transparency and consistency in assessing the benefit-risk balance, the European Medicines Agency (EMA) began a three-year project in early 2009. This project consists of five consecutive work packages. The first four work packages form a research phase that aims to develop and test tools and methods for balancing benefits and risks of medicinal products. The fifth work package is intended for training and initial implementation. Currently, the project has completed its research phase, which recognised the usefulness of two levels of gradation. The first level is a qualitative approach, mainly consisting of a table listing the key effects of the benefit-risk balance and their uncertainty in a common and concise format. The second level, recommended for more complex situations, is a quantitative method that utilises Multi-Criteria Decision Analysis (MCDA) to derive a numerical value for the benefit-risk balance. However, the implementation of MCDA in the assessment of a medicine poses some practical challenges that remain to be addressed in the last work package of the project.