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.
What You Need to Know? 👇
What are the key principles for using PRO instruments in medical device evaluation?
FDA recommends four key principles: establish and define the concept of interest the PRO instrument captures; clearly identify the PRO’s role in the clinical study protocol; provide evidence showing the PRO instrument reliably assesses the concept; and effectively communicate PRO-related results in device labeling.
How do I determine if a PRO instrument is fit-for-purpose for my medical device study?
Consider three factors: whether the concept being measured is meaningful to patients and changes would be meaningful; what role the PRO will serve in your clinical study protocol; and whether evidence supports the PRO instrument’s use for measuring your concept of interest in your specific context.
Can I modify an existing PRO instrument instead of developing a new one?
Yes, FDA encourages modifying or adapting existing PRO instruments as a least burdensome approach when feasible. You must clearly describe modifications in your submission documentation and may need new validity evidence depending on the extent of changes made to the original instrument.
What role do cognitive interviews play in PRO instrument development?
Cognitive interviews are qualitative research methods used to determine whether patients understand PRO instrument concepts and items as intended by developers. FDA recommends conducting these interviews to generate evidence supporting the wording of instructions, items, recall periods, and response options before clinical study use.
How can real-world data support PRO instrument validation?
Real-world evidence from electronic health records, claims data, registries, or health-monitoring devices can generate validity evidence for PRO instruments. FDA encourages considering these alternative approaches as potentially less burdensome than traditional validation studies, including nested development within real-world data sources.
What is the MDDT program and how does it relate to PRO instruments?
The Medical Device Development Tools (MDDT) program qualifies tools that medical device sponsors can use in development and evaluation. Qualified PRO instruments under MDDT have been evaluated by CDRH and confirmed to produce scientifically-plausible measurements within their specified context of use.
What You Need to Do 👇
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