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Patient-Reported Outcome Instruments in Medical Device Evaluation

This guidance outlines principles and best practices for selecting, developing, modifying, and adapting Patient-Reported Outcome (PRO) instruments used in medical device evaluation across the Total Product Life Cycle (TPLC). It provides recommendations to ensure PRO instruments are relevant, reliable, and sufficiently robust while using the least burdensome approach. The guidance does not detail specific methods but rather communicates best practices for PRO instrument implementation in medical device evaluation.

  1. Establish and define the concept of interest the PRO instrument will measure
  2. Clearly identify the role of the PRO instrument in clinical study protocol and statistical analysis plan
  3. Conduct cognitive interviews to ensure patient understanding before implementation
  4. Consider using existing validated PRO instruments rather than developing new ones when possible
  5. Generate appropriate validity evidence based on intended use of PRO instrument
  6. Engage with FDA through Q-submission process regarding PRO instrument approach
  7. Consider collaborative development with relevant stakeholders
  8. Document modifications made to existing PRO instruments
  9. Consider alternative platforms and parallel development approaches for validation
  10. Ensure PRO results are appropriately communicated in device labeling

Key Considerations

Clinical testing

  • PRO instruments can be used in early feasibility, feasibility, pivotal, and postmarket clinical studies
  • Can be used to determine patient eligibility, measure primary/secondary endpoints, or as part of composite endpoints
  • The role of PRO instrument must be clearly defined in clinical study protocol and statistical analysis plan
  • Evidence needed depends on the role of the PRO instrument in the study

Human Factors

  • Conduct cognitive interviews to ensure PRO instruments are understandable to patients
  • Use plain language appropriate for varying literacy levels
  • Ensure response options are consistent with item wording
  • Consider patient burden when designing PRO instruments
  • Consider offering instruments in different languages where appropriate

Labelling

  • PRO results should be effectively communicated in labeling to inform healthcare provider and patient decision making
  • The concept of interest and context of use should be clearly defined
  • Changes measured by PRO instrument should be clinically meaningful

Safety

  • PRO instruments can be used to assess safety endpoints
  • Different validity evidence may be needed for safety vs effectiveness endpoints

Other considerations

  • AERA/APA/NCME: Standards for Educational and Psychological Testing

Original guidance

  • Patient-Reported Outcome Instruments in Medical Device Evaluation
  • HTML / PDF
  • Issue date: 2022-01-26
  • Last changed date: 2022-01-25
  • Status: FINAL
  • Official FDA topics: Medical Devices, Postmarket, Premarket, Biologics
  • ReguVirta summary file ID: d4276a4ccf5ebe0f677489b72e6b26c0
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