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

Timing

The timing of patient preference studies depends on the stage of therapy development you are hoping to influence. 

  • Early patient preference studies focused on understanding unmet medical needs, burden of disease, and general patient benefit-risk perspectives can provide crucial information during the therapy discovery phase to academic and industry researchers.
    • Influence symptoms to be targeted for treatment development.
    • Attract interest of academic and industry researchers.
    • Determine priority of therapies in early development from a patient perspective.
  • In the preclinical phase of therapy development, when researchers have completed initial analyses of the possible benefit-risk of a potential therapy, patient preference studies can obtain information about more specific preferences and can use this data to develop clear COAs.
    • Ask about more specific benefit-risk scenarios that may influence clinical trial design and patient subpopulations to target for enrollment.
    • Develop a COA or choose one that already exists. You can learn more about COAs in NCATS Toolkit section on Help Industry with Clinical Trial Design.
    • Determine information that may be important to patients during their decision process of whether or not to enter a specific clinical trial. 
  • During Phase 2 and 3 clinical trials, patient preference studies can be focused on actual benefits and risks seen among trial participants.
    •  The data from studies performed now can be submitted to the FDA to be included in the marketing review process.
  • After therapy approval, patient preference studies can be used to detect any difference between stated preferences (what a person believes they will choose) and revealed preferences (what a person actually chooses).

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)