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 package | Status |
---|---|
1. Describing the benefit-risk assessment models already being used in the European Union's regulatory network | Completed March 2010 |
2. Assessing the suitability of the current tools and processes used in benefit-risk assessments | Completed August 2010 |
3. Field-testing the most appropriate models in five European medicine regulatory agencies | Completed June 2011 |
4. Refining the most suitable models for use in medicines regulation to create a new benefit-risk tool | Completed February 2012 |
5. Training European assessors to use the final tool | Started March 2012 |
Presentation of outcome of benefit-risk assessments
Based on the findings of the benefit-risk methodology project, the Agency published a
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.
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Benefit-risk methodology project (PDF/179.39 KB)
Adopted
First published: 26/07/2011
Last updated: 26/07/2011
EMEA/108979/2009 -
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Benefit-risk methodology project: Work package 1 report: Description of the current practice of benefit-risk assessment for centralised procedure products in the European Union regulatory network (PDF/253.68 KB)
Adopted
First published: 26/07/2011
Last updated: 26/07/2011
EMA/227124/2011 -
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Benefit-risk methodology project: Work package 2 report: Applicability of current tools and processes for regulatory benefit-risk assessment (PDF/454.55 KB)
Adopted
First published: 13/10/2010
Last updated: 12/01/2010
EMA/549682/2010 -
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Benefit-risk methodology project: Work package 3 report: Field tests (PDF/432.5 KB)
Adopted
First published: 15/09/2011
Last updated: 15/09/2011
EMA/718294/2011 -
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Benefit-risk methodology project: Work package 4 report: Benefit-risk tools and processes (PDF/414.95 KB)
Adopted
First published: 12/03/2012
Last updated: 13/06/2012
EMA/297405/2012 rev.1 -
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Benefit-risk methodology project: Update on work package 5: Effects table pilot (Phase I) (PDF/105.57 KB)
First published: 24/02/2014
Last updated: 24/02/2014
EMA/74168/2014 -
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Benefit-risk methodology project - Report on risk perception study module (PDF/747.3 KB)
First published: 23/02/2012
Last updated: 23/02/2012
EMA/662299/2011
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Benefit-risk methodology project: Comments received from Dr William Holden on the work package 2 report (PDF/184.94 KB)
First published: 09/06/2011
Last updated: 09/06/2011
EMA/314182/2011 -
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Benefit-risk methodology project: Reply to the comments received from Dr William Holden on the work package 2 report (PDF/115.93 KB)
First published: 09/06/2011
Last updated: 09/06/2011
EMA/61755/2011