During the March 2008 plenary meeting, the Committee for Human Medicinal Products (CHMP) adopted the Reflection paper on benefit-risk assessment methods in the context of the evaluation of marketing authorisation applications of medicinal products for human use (EMEA/CHMP/15404/2007). One of the main recommendations for the CHMP was to explore further methodologies for benefit-risk analysis, including a wide range of quantitative and semi-quantitative tools, and involving assessors and experts.

Between June and September 2009, the project team was invited to visit the five participating agencies (France, The Netherlands, Spain, Sweden and UK). At a later point, Paul-Ehrlich-Institut volunteered to participate in the project and was included in the visits in February 2010. At present, there are no common definitions on the concepts of benefits and risks and no formal guidance on the process of their balancing. As a result, interviewees, between agencies and within the same agency, expressed divergent views on the meaning of benefits and risks, and on their weighing. Especially with regard to risks, there was a great variance of responses. Most notably, the uncertainty of realising a benefit from the use of a pharmaceutical product was frequently mentioned as a risk. In practice, formulating a benefit-risk balance for a medicinal product is an intuitive and implicit process based on expert judgement. In all the six agencies there was no mention of the use of a validated and structured process for assessing benefits and risks. In contrast, most of the interviewes acknowledged the need for a more systematic approach and the value it can add. A few assessors mentioned the use of a self-constructed and simplistic method for balancing benefits and risks. The benefit-risk balance is generally considered as the most difficult part of the assessment process, even for experienced assessors. It was mentioned that the most challenging situations are those in which there is substantial uncertainty about the benefits and the risks of a product, the products belong to a new class of drugs, or the products belong to the therapeutic area of oncology.

Outcome:

In the future, EMAs project team will focus on developing  tools and processes, that can add value without disrupting the current workflow. Conclusions can be expected by the end of 2011.

Link:

EMA/ Benefit-Risk Summary