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Patient Preference Information in Medical Device Development, Clinical Trials, and Regulatory Decision Making

This guidance focuses on Patient Preference Information (PPI) that may be used in FDA decision-making related to PMAs, HDE applications, and de novo requests. It provides recommendations for collecting and submitting PPI to FDA, outlines qualities of patient preference studies, and discusses FDA's inclusion of PPI in decision summaries and device labeling. The guidance is applicable to both diagnostic and therapeutic devices subject to these review processes.

  1. Determine if PPI would be valuable for your specific device type and intended use
  2. Engage with FDA early through pre-submission if planning to collect PPI
  3. Design patient preference studies following recommended qualities:
    • Ensure patient-centeredness
    • Use representative sample
    • Capture preference heterogeneity
    • Follow established research practices
    • Minimize cognitive bias
    • Include validity tests
  4. Consider PPI throughout product lifecycle:
    • During device design and development
    • In clinical trial design
    • For benefit-risk assessments
    • In postmarket monitoring
  5. Develop appropriate labeling that effectively communicates benefit-risk information to patients
  6. Consider conditions of approval that may help mitigate risks based on PPI findings
  7. Document and maintain data integrity for all PPI collected
  8. Include relevant PPI in regulatory submissions and device labeling
  9. Consider post-approval studies to confirm benefit-risk profile in identified patient subgroups

Key Considerations

Clinical testing

  • PPI can inform clinical trial design by helping identify endpoints important to patients
  • PPI can help define minimum clinically meaningful benefit which impacts sample size
  • Patient-informed clinical study design may reduce barriers to participation
  • Patient input may inform development/selection of PRO measures

Non-clinical testing

  • Patient input during discovery phase can inform device design and features
  • Patient-sensitive design inputs help refine device design through human factors engineering
  • Patient input can influence which devices are developed by defining unmet needs

Human Factors

  • Patient-centered design inputs should be considered during development
  • Device design should account for patient end-user needs
  • Human factors engineering should incorporate patient input

Labelling

  • Device labeling must contain sufficient benefit-risk information
  • Information should be written in plain language understandable to patients
  • Visual aids like pictorials and graphics should be included when appropriate
  • Clear indication of appropriate patient population should be provided
  • Potential benefits and risks, including likelihoods, should be described
  • Relevant contraindications, warnings and precautions must be included

Safety

  • Patient tolerance for risk should be evaluated
  • Both willingness and unwillingness to accept identified risks should be assessed
  • Risk communication should use multiple formats (verbal, numeric, graphic)
  • Absolute rather than relative risk should be presented when possible

Other considerations

  • ISO 14971:2007: Medical Devices – Application of Risk Management to Medical Devices

Original guidance

  • Patient Preference Information in Medical Device Development, Clinical Trials, and Regulatory Decision Making
  • HTML / PDF
  • Issue date: 2016-08-24
  • Last changed date: 2020-10-20
  • Status: FINAL
  • Official FDA topics: Medical Devices, Radiation-Emitting Products
  • ReguVirta summary file ID: 5435f862937312f18368adaae3bf23ce
This post is licensed under CC BY 4.0 by the author.