PURPOSE: Discuss the challenges of incorporating patient-reported outcome (PRO) measures in stated preference (SP) studies, solutions to these challenges, and use to support regulatory decision making.
DESCRIPTION: Understanding the value patients place on changes in PROs is especially important when improvements in PROs are used as endpoints in clinical trials. These values can support benefit-risk preference assessments and regulatory decision making. In this workshop, attendees who sponsor, conduct, interpret, or use SP study results will learn the advantages of using PROs in SP studies, the challenges, and approaches to address these challenges. The presenters will represent regulatory, pharmaceutical industry (TBD), and research perspectives.
The first presentation (20 min) will compare and contrast PRO and preferences, summarize how PRO and preferences are used in regulatory decisionmaking at FDA in general and in benefit-risk assessment in particular. Relevant regulations and guidelines will be briefly described and the value of including PROs in preference research in various ways will be discussed (e.g., determining eligibility and stratifying sample, describing respondents’ experiences, treatment attributes).
The second presentation (25 min) will identify and discuss several challenges that arise when incorporating PRO into SP studies and discuss alternative ways to address these challenges. Examples will illustrate how PROs have been used in previous SP studies, including sample eligibility and stratification, sample characterization, and as a treatment attribute.
The third presentation (10 min) will step back and discuss how different approaches to addressing the challenges of using PROs in SP research may affect the use of the results of SP studies to support regulatory decision making. In this section, the discussion leader will lead a discussion about some of the factors that may be considered when designing SP studies that use PROs. The remaining time (5 min) will be allotted to discussion of the session and future research.
AUDIENCE INTERACTIVE ELEMENT: The workshop will use real-time polling to evaluate both the challenges of using patient-reported outcomes in stated preference studies and alternative approaches to addressing these challenges.
