Benefit-risk methodology

The European Medicines Agency's opinions are based on balancing the desired effects or 'benefits' of a medicine against its undesired effects or 'risks'. The Agency can recommend the authorisation of a medicine whose benefits are judged to be greater than its risks. In contrast, a medicine whose risks outweigh its benefits cannot be recommended for marketing.

Weighing up the benefits and risks of a medicine is a complex process, since it involves the evaluation of a large amount of data. In addition, there is always some uncertainty around the actual benefits and risks of a medicine, because they can only be determined by looking at the information that is available at a given point in time.

The benefit-risk methodology project

The Agency strives towards making its opinions on the balance of benefits and risks as consistent and transparent as possible. To date, however, there is no standard methodology that is used to aid regulatory decisions on the benefits and risks of medicines.

To help address this problem, the Agency began a three-year project on benefit-risk methodology in early 2009, in collaboration with experts in decision theory from the London School of Economics and Political Science (LSE) and with the University of Groningen.

The aims of the project were to identify decision-making models that could be used in the Agency's work, to make the assessment of the benefits and risks of medicines more consistent, more transparent and easier to audit.

The project began on the recommendation of a working group of the Committee for Medicinal Products for Human Use (CHMP) on benefit-risk assessment methods, which met between 2006 and 2008. The working group's conclusions were published in a reflection paper in March 2008.

The project consisted of five 'work packages':

Work packageStatus
1. Describing the benefit-risk assessment models already being used in the European Union's regulatory networkCompleted March 2010
2. Assessing the suitability of the current tools and processes used in benefit-risk assessmentsCompleted August 2010
3. Field-testing the most appropriate models in five European medicine regulatory agenciesCompleted June 2011
4. Refining the most suitable models for use in medicines regulation to create a new benefit-risk toolCompleted February 2012
5. Training European assessors to use the final toolStarted March 2012

Presentation of outcome of benefit-risk assessments

Based on the findings of the benefit-risk methodology project, the Agency published a File guidance document incorporating a number of improvements in the way committees' assessment reports present the outcomes of benefit-risk assessments. Amongst these is the use of the 'effects table' for all new marketing authorisation and extension of indication applications, considered the most suitable available tool to facilitate the benefit-risk assessment of medicinal products.

Benefit-risk methodology project


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