Perform Patient Preference Studies


A patient preference study is a way to collect and analyze patient and caregiver preferences concerning risks they are willing to accept for a specified benefit provided by a treatment. In fact, patient and caregiver preferences can differ greatly from what clinicians and researchers might expect, especially in the rare disease community. Although performing patient preference studies during each stage of therapy development has become more common, the FDA does not require Sponsors to include these studies. Therefore, you may wish to educate research and industry partners about the advantages of patient preference studies. The data from patient preference studies can:

  • Characterize the burden of disease, unmet medical needs, patient perspective on benefit-risk, and diversity of preferences in the patient population:
    • Identify what is most important to patients.
    • Understand the maximum risk and/or burden patients will accept for different benefits.
    • Learn how patients may compare benefits and risks between 2 therapy options.
    • Determine whether preferences differ based on demographics or geographical location.
    • Investigate other, differentiating factors that may affect patient preferences, such as severity of symptoms or stage of disease.
  • Influence the design of clinical trials
    • Develop endpoints that matter to patients.
    • Select target populations within the patient group.
    • Reveal confounding factors that may require stratification of participants during analysis, for example, participants may be stratified by age or disease stage in order to best determine effectiveness and safety of treatment.
    • Develop meaningful effect sizes for endpoints based on patient and caregiver preferences. You can learn more about Clinical Outcome Assessments (COAs) in NCATS Toolkit section on Help Industry With Clinical Trial Design.
    • Determine how to promote patient participation to make certain enough participants enroll. 
    • Uncover factors that may affect a participant’s decision to discontinue participation in a clinical trial.
  • Impact the FDA marketing review process:
    • Qualified COAs developed through the inclusion of patient preferences can be presented to FDA for consideration during the clinical trial and marketing review process. 
    • Benefit-risk of therapies targeting unmet medical needs may be assessed more tolerantly by FDA.
    • Patient perspective of benefit-risk determined through scientifically performed patient preference studies will be considered by FDA during the marketing review process.
  • Provide patients with information important to their treatment decision-making process after a therapy receives FDA marketing approval.
    • COAs may be used in labeling the therapy to allow better understanding of benefits and risks of use.


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)
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)