Benefit-risk methodology

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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. The project aims to identify decision-making models that can 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 consists 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

Public consultation

The Agency will launch a public consultation on the first four work packages, depending on the outcome of the final phase of the project and following the agreement of the Committee for Medicinal Products for Human Use.

The public consultation will include a workshop, which will:

  • inform and engage the project's stakeholders;
  • explore the acceptability and the implementation of the Agency's methodology for benefit-risk assessment;
  • form an integral part of the public consultation phase prior to the adoption of a final, formal position from the Agency.


Until the end of 2011, the Agency conducted the project in collaboration with experts in decision theory from the London School of Economics and Political Science (LSE) and with the University of Groningen

Presentation of outcome of benefit-risk assessments

The working group also recommended improvements to the way in which the outcome of benefit-risk assessments are presented. The Agency made a number of these improvements to its committees’ assessment reports in 2009, to reflect the benefits and risks of medicines in a clearer manner and to describe the sources of uncertainty and variability.

Table of contents

Benefit-risk methodology project

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Related links

CHMP working group on benefit-risk assessment methods

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Comments from Dr William Holden on work package 2 report

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