Workshop on the methods for benefit-risk analysis in drug regulation and health technology assessment

Registration now closed

 

With the support of funding from the Medical Research Council Network of Hubs for Trial Methodological Research (MRC HTMR), we are pleased to announce a 2-day, intensive workshop on the methods for benefit-risk analysis in drug regulation and health technology assessment.

 

Date:

January 24th & 25th, 2013

 

Venue:

Bangor University’s Management Centre http://www.bangor.ac.uk/management_centre/

 

Background:

The focus of benefit-risk analysis has been mainly on regulatory decisions concerning pharmaceuticals. However, the extent to which it is appropriate to make benefit-risk assessments in clinical trials, in reviews of clinical trials, and in public health policy needs further clarification.

The relative benefits and harms of new health technologies are already considered implicitly by bodies such as NICE when generating guidance about their use within health care systems. Such bodies will make recommendations upon clinical- and cost-effectiveness, where ‘effectiveness’ is usually a composite measure combining benefit and harm domains. While NICE has guidance for the methods to be used when creating its guidance, it is yet to develop specific guidance for benefit-risk analysis.

Within clinical trials, judgments on the acceptability of harms in relation to benefits are often limited to measures of net clinical benefit, usually based on composite outcomes that do not weight the relative importance of each constituent measure of outcome.  A particular limitation of this approach is that “harms” often include both a lack of effect (i.e. a manifestation of the under-treated disease) and adverse drug reactions.  As both efficacy and adverse drug reactions are expected to increase with dose (or adherence), the resulting measure of net clinical benefit is of limited value.

Problems associated with the quantification of benefit-risk analyses arise from a range of sources, including whether and how multi-attribute dimensions of benefits and harms should be combined into a single index, how non-trial based evidence and parameter uncertainty may be incorporated into the analysis, how to assess causality (e.g. in relation to rare adverse drug reactions), and how to select and implement a decision rule, and the extent to which formal approaches to decision-making should be incorporated into the decision-making processes.

Workshop Aim:

The aim of the workshop is to develop a research agenda on the assessment of benefits in relation to harm in the context of health technology assessment.

Outputs:

• a paper on the state of the art of benefit-risk analysis in HTA

• ideas for a research grant application

 

Workshop Format:

 A mixture of talks and breakout sessions.

Final Programme

 

 

Day 1 – January 24th
10.00	Refreshments on arrival
10.30	Welcome, introductions and aims of the workshop	Dyfrig Hughes, Bangor University
11.00	The EMA Benefit-Risk Methodology Project	Lawrence Phillips, LSE
11.45	IMI-PROTECT benefit risk project	Deborah Ashby, Imperial College London
12.30	Lunch
1.30	Benefit risk assessment in epilepsy	Tony Marson, University of Liverpool
2.15	Core outcome measures of benefits and harms	Paula Williamson, University of Liverpool
3.00	Coffee break
3.30	If nothing happens, is everything alright?	Yoon Loke, University of East Anglia
4.15	Breakout discussions & feedback
5.30	Close
7.00	Dinner at “1884 Brasserie”

 

 

 

 

Day 2 – January 25th
8.45	Tea & Coffee
9.00	Benefit-risk analysis in economic evaluations	Dyfrig Hughes, Bangor University
9.45	Quantitative approaches to modeling benefit-risk balance in regulatory health technology assessment	Lou Garrison, University of Washington
10.30	Tea & Coffee
11.00	Methods for preference elicitation	Warren Linley, Emily Fargher, Bangor University
11.45	NICE Guidelines	Mark Baker, NICE
12.30	Lunch
1.30	Conclusions and plans for: Workshop report Paper on research priorities Research grant application
2.30	Depart