MEASURING PATIENT AND PHYSICIAN PREFERENCES TO INFORM BENEFIT-RISK ANALYSIS

Format Requirements: This introductory workshop will include lecture, an exercise, and a case study. It will begin with an overview of current benefit-risk approaches under consideration by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It then will provide a detailed review of the numerous preference-elicitation methods currently being used to quantify benefit-risk preferences. Particular emphasis will be places on those methods that have been used or currently are being used in submissions to regulatory agencies. During the exercise, participants will be asked to complete a preference-elicitation survey and discuss the results. The case study will focus on a pilot study commissioned by the FDA Center for Devices and Radiological Health (CDRH). As an introductory workshop, it is expected that participants will have little to moderate knowledge about benefit-risk preference methods. Some basic knowledge of survey methods, patient-reported outcomes, and basic statistical analysis would be advantageous.

Background: Regulators, clinicians, and patients routinely make decisions that require evaluating tradeoffs between treatment safety and desired clinical benefits in the absence of directly comparable metrics. Regulatory agencies have initiated a number of activities designed to assess alternative methods for quantitative benefit-risk analysis. Nearly all of the approaches to quantitative benefit-risk analysis under consideration include determining the relative importance of benefits-and risks by eliciting this information from patients or physicians. In addition, there are numerous methods that can be used to elicit these inputs including, but not limited to: multi-criteria decision analysis (MCDA), analytic hierarchy processing (AHP), conjoint analysis and discrete-choice experiments (DCE), and threshold techniques.

Description and Objectives: This workshop will provide participants with a basic overview of the current state of benefit-risk preference assessment. The course will provide an assessment of the current need for this type of research, an overview of the range of methods being used to evaluate benefits and risks of medical interventions, and provide a number of real-world examples. The workshop will incorporate hands-on activities and several case studies.

 By the end of this course participants will:

• Have a greater understanding of the need for and use of quantitative benefit-risk analysis in regulatory decision making;
• Have an understanding of the range of options for benefit-risk preference elicitation; 
• Develop some basic skills to indentify when a particular benefit-risk preference method is appropriate for a particular research question and know where to find appropriate guidance to apply the methods successfully.