Benefit-risk assessment model for medicines – Developing a structured approach to decision making
13-14th June 2005, The Georgetown Inn, Washington DC, USA
As public accountability grows for decisions taken in both public and private sectors, so the need develops for organisations to provide an audit trail for important decisions. Decisions leading to drug approvals demand careful attention with respect to balancing the benefits and risks, which in turn requires a structured process that leaves an audit trail. The Workshop brought together 27 senior participants drawn from regulatory agencies in the USA and Europe, academia and the pharmaceutical industry. The purpose was to explore a structured model for benefit-risk assessment and consider its potential for providing support to decision-makers in both regulatory agencies and pharmaceutical companies.
This was the second Workshop on this topic, hosted by the CMR International Institute for Regulatory Science, the first having been held in March 2004, in the UK. The success of this meeting led recommendations for a further Workshop to be convened in the USA.
The Workshop was organised to encourage frank and open discussions about the way in which important, far-reaching decisions are taken within both companies and regulatory agencies and the potential shortcomings of such processes. The main component of the meeting, however, was an interactive demonstration of the application of the technical model ‘multi-criteria decision analysis’ (MCDA). The model was discussed using a hypothetical scenario based on safety and efficacy data relating to an atypical antipsychotic agent that was clinically tested against placebo and a comparator compound.
The MCDA methodology provides a way of looking at complex problems and breaking them down into more manageable pieces that can be studied using a mixture of data and judgement. The components are then ‘re-assembled’, using computer software, to present a coherent overall picture for the decision makers. The purpose of this tool is to serve as an aid to thinking and decision-making, but not to take the decisions.
At the conclusion of the meeting it was, as at the 2004 meeting, unanimously agreed that the methodology had great potential and it was recommended that it should be explored further as an adjunct to the decision-making process for both companies and regulatory agencies. An important part of this would be to undertake retrospective testing using actual case studies.
It was also recommended that a third Workshop should be convened at a later stage to discuss the results of such studies and explore further the practical application of the methodology and its place in preparing regulatory submissions as well as in the regulatory review process.