U.S. Department of Health and Human ServicesHHS National Institutes of HealthNIH National Center for Advancing Translational SciencesNCATS
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Perform Patient Preference Studies

Tips for Success

  • Know what decisions you want to influence and whether the questions you are asking will help you reach that goal. 
  • Explore whether there are other ways that are less demanding on time and resources to achieve your goals, such as focus groups or interviews.
  • Develop collaborations with a shared understanding of the purpose and use of information generated.
  • Understand the significance of using sound science to develop and analyze patient preference studies.
  • A clear understanding of objectives translates into choosing the appropriate methodologies to achieve those objectives.
  • Engage the FDA throughout the process.
  • Remember patient and caregiver preferences may change over time as new therapies become available.
  • Read Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study (2016), a report providing information to help you decide if and when your group wants to conduct a patient preference study. The report also provides more details about the different types of studies and analyses.
  • Review Benefit Risk Bootcamp (2014), an overview of sessions from an FasterCures event that describes effective participation in benefit-risk assessment. Descriptions and slide presentations from each session are available. Topics discussed include understanding benefit-risk assessment, common methods used, and key FDA programs.

Resources

Engage with the FDA
FDA Patient Affairs U. S. Food and Drug Administration (FDA) (link)
FDA For Patients Website U. S. Food and Drug Administration (FDA) (link)
Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Project Report Medical Device Innovation Consortium (MDIC) (link)
FDA-led Patient-Focused Drug Development (PFDD) Public Meetings U. S. Food and Drug Administration (FDA) (link)
Enhancing Benefit-Risk Assessment in Regulatory Decision-Making U. S. Food and Drug Administration (FDA) (link)
Tips for Success
Key Considerations in Developing & Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) (link)
Benefit-Risk Boot Camp FasterCures (link)