Refining the benefit-risk framework for the assessment of medicines: Valuing and weighting benefit and risk parameters
17-18th June 2010, Washington DC, USA
Determining the benefit-risk balance of a medicine is one of the most important steps in its development, review and post-approval re-assessment. The need for and establishment of a benefit-risk framework is recognised as critical from both a regulatory agency and pharmaceutical company perspective in order that there is a transparent articulation of the benefits and risks considered in making the final regulatory decision. Moreover, by applying the parameters derived from new assessment models, regulatory agencies and sponsors have a framework that allows a scientific discussion of benefit-risk.
There is a consensus regarding the essential elements that should comprise any framework as well as regarding the five key steps that take data through a systematic process prior to making the final decision. These steps are construction of summary tables; development of a value tree of benefits and risks; assessment of importance and prioritisation of benefits and risks; assignment of value and weightings for the benefits and risks; and finally, benefit-risk assessment through the application of expert judgement.
Although there is agreement concerning the steps that are required for the framework, the assignment of weighting and values to each component parameter is more difficult, owing to different views, methods and stakeholder perspectives, not just on what should be used in weighting, but how it should be used.
In order to address this specific issue and based on the workshops held by the CMR International Institute for Regulatory Science (the Institute) in 2007 and 2008, the institute held this Executive Forum facilitated around real case studies and focused on refining the beneft-risk framework through a discussion of the assignment of value and weightings.
Workshop objectives
- Present the opportunity to refine the Institute’s benefit-risk framework by making it more robust, understandable and practical.
- Develop an agreement on the principles that should be used in the weighting and values step of the framework.
- Provide a forum for regulatory agencies and industry to review case studies and discuss the utilisation of weighting and values in making a benefit-risk decision.
- Discover agency and industry perspectives of which weighting and values can be used and which issues need to be addressed within the benefit-risk framework.