Numbers or Noise? The Patients' Voice in Medical Device Regulatory Decisionmaking

• Activity Number: 473
• Type: Topic Contributed
• Date/Time: Wednesday, August 7, 2013 : 8:30 AM to 10:20 AM
• Sponsor: Section on Bayesian Statistical Science
• Author(s): Reed Johnson*+
• Companies: Research Triangle Institute
• Keywords: benefit-risk analysis ; regulatory decision making ; patient preferences ; discrete-choice experiments ; conjoint analysis

Abstract

FDA invites stakeholders to provide testimony to scientific advisory committees evaluating new devices. Such testimony often includes compelling, emotional details of patients' positive or negative experience with a particular treatment. Nevertheless, anecdotal data, however compelling, is not evidence. This presentation describes and evaluates methods for systematically eliciting and quantifying patient benefit-risk preferences. Such data arguably must satisfy quality standards comparable to other evidence submitted on product efficacy and safety. Recent published guidelines indicate a growing consensus among stated-preference researchers on best-practice methods. Examples from recent patient-preference studies are used to illustrate challenges in establishing the validity and reliability of data on patients' benefit-risk tradeoff preferences. Challenges include verifying the extent to which patients are sufficiently informed about clinical endpoints, are competent to evaluate probabilistic information in view of low numeracy levels in the general population, and provide trade-off preference data that satisfy basic requirements of consistency and theoretical validity.