Implementing an internationally accepted framework for the benefit-risk assessment of medicines: How close are we to this objective?
20-21 June 2013, Washington DC, USA
At the annual CIRS Benefit-Risk Workshop in 2012, there was an agreement among those who are developing methodologies for assessing the benefits and risks of medicines that there are four key stages for these evaluations, namely; framing the decision; identifying the benefits and risks; assessing the benefit and risks; and developing interpretations and recommendations. An overarching eight-step framework developed at this workshop underpins the four stages:
- Developing a decision context
- Building a value tree
- Refining the value tree
- Assessing the relative importance of parameters
- Evaluating options
- Assessing uncertainty
- Concisely presenting results – visualisation
- Issuing final recommendations.
The overarching framework provides the basis for a common agreement on the principles for benefit-risk assessment and all the methodologies for evaluation that are currently being developed by regulators and pharmaceutical companies have either implicitly or explicitly incorporated most of the eight steps. Over 2012-2013, an implementation and usage guide was developed for the overarching framework through the CIRS Universal Methodologies for Benefit-Risk Assessment (UMBRA) initiative. Two issues within the conduct of benefit-risk assessment remained to be resolved, one being the assessment of relative importance and the other the evaluation of uncertainty. However, in some methodologies they may not be considered as specific seps. Moreover, the process of determining relative importance was identified as particularly difficult, due to perceived complexity, subjectivity and the lack of standardised methodology.
CIRS investigated these issues with companies and agencies as part of the UMBRA initiative and proposals for consideration with regards to the applicability of the UMBRA Framework were discussed and debated at this workshop. Stakeholders in the development and regulation of medicine sought to determine if the overarching framework and the methodologies that had been developed and were being used routinely within companies and agencies were fit or purpose and if not, what the main concerns were.
Workshop objectives
- Discuss the progress made by the different groups in 2013 in defining and implementing a benefit-risk methodology framework and specific methodologies within their organisations
- Further the thinking around assessing relative importance and uncertainty within the context of making explicit benefit-risk decisions and how these should be approached
- Develop proposals for the implementation of the overarching UMBRA framework and discuss its use from molecule to marketplace in the life cycle of medicines