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.
Recommended Actions
- Establish and define the concept of interest the PRO instrument will measure
- Clearly identify the role of the PRO instrument in clinical study protocol and statistical analysis plan
- Conduct cognitive interviews to ensure patient understanding before implementation
- Consider using existing validated PRO instruments rather than developing new ones when possible
- Generate appropriate validity evidence based on intended use of PRO instrument
- Engage with FDA through Q-submission process regarding PRO instrument approach
- Consider collaborative development with relevant stakeholders
- Document modifications made to existing PRO instruments
- Consider alternative platforms and parallel development approaches for validation
- 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
- Measure concepts important to patients through effective patient engagement
- Consider leveraging existing PRO instruments and validity evidence
- Consider alternative platforms and parallel development for generating validity evidence
- Collaborate with stakeholders in pre-competitive space
- Consider submitting PRO instruments for qualification under MDDT Program
Relevant Guidances
- Patient Preference Information in Medical Device Development, Clinical Trials, and Regulatory Decision Making
- Patient Engagement in Medical Device Clinical Studies: Roles, Benefits and Best Practices
- Medical Device Development Tools (MDDT) Qualification Program
- Design Considerations for Medical Device Pivotal Clinical Studies
Related references and norms
- AERA/APA/NCME: Standards for Educational and Psychological Testing
Original guidance
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