Patient Preference Information in Medical Device Development and Benefit-Risk Assessments (DRAFT)
This guidance provides recommendations on incorporating voluntary Patient Preference Information (PPI) throughout the total product lifecycle of medical devices. It focuses on how PPI may be considered by FDA in decision-making, outlines qualities of valid patient preference studies, provides practical recommendations for collecting and submitting PPI, and discusses inclusion of PPI in decision summaries and device labeling.
This is a draft guidance. Not for implementation.
What You Need to Know? 👇
What is patient preference information and how does it differ from patient-reported outcomes?
Patient preference information (PPI) is qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives among health interventions. Unlike patient-reported outcomes which measure health status at a point in time, PPI captures what patients value and their willingness to make tradeoffs between different treatment attributes.
When should sponsors consider conducting patient preference studies for medical devices?
PPI studies are particularly useful for devices with direct patient interface, life-saving but high-risk devices, devices for unmet medical needs, or novel technologies. They’re most valuable when patient decisions are preference-sensitive, meaning multiple treatment options exist without clear superiority, or when patients may value benefits and risks differently than healthcare professionals.
Is submission of patient preference information required for FDA device submissions?
No, submission of PPI to FDA is completely voluntary. However, it may be useful for certain device types and diseases where usage decisions are preference-sensitive. PPI can provide valuable insights to support benefit-risk assessments throughout the total product lifecycle, from IDE applications to post-market decisions.
What are the key quality requirements for patient preference studies to be considered valid scientific evidence?
Studies should be patient-centered, use representative populations, reflect preference heterogeneity, employ appropriate methods, communicate benefits/risks effectively, minimize cognitive bias, include logical soundness checks, and follow established good research practices from recognized professional organizations like ISPOR. Robust analysis and proper study conduct are essential.
How can sponsors seek FDA feedback on their patient preference study plans?
Sponsors are encouraged to engage with FDA early through the Q-Submission Program. Key engagement points include protocol development, attribute selection, survey instrument design, and recruitment strategies. Early FDA interaction helps ensure alignment on research questions, study objectives, and regulatory relevance before study implementation.
How does FDA incorporate patient preference information into device labeling and decision summaries?
When FDA considers PPI in premarket submissions, it’s typically included in public decision summaries for PMAs, HDEs, and De Novo requests. PPI supporting FDA’s approval should be described in device labeling using plain language, visual aids when appropriate, and clear communication of benefits, risks, and the intended patient population.
What You Need to Do 👇
Recommended Actions
- Engage early with FDA during PPI study planning and design
- Conduct qualitative research to inform quantitative PPI study design
- Pre-test survey instruments and communication formats with target population
- Ensure study population is representative of intended device users
- Document study methodology, including statistical analysis plan
- Include comprehensive data quality checks and validation
- Submit complete study information to FDA including:
- Scientific question and objectives
- Study design and methods
- Eligibility criteria and recruitment approach
- Survey instrument design
- Results with demographics
- Incorporate PPI findings appropriately in device labeling
- Follow established good research practices from recognized organizations
- Maintain data integrity throughout the study process
Key Considerations
Non-clinical testing
- Patient input can inform device design and features during discovery and ideation phase
- Patient-sensitive design inputs may help refine device design to better meet patient end-user needs
Human Factors
- Studies should ensure patient comprehension with minimal cognitive bias
- Pre-testing of communication formats and survey instruments recommended
- Study design should minimize potential cognitive biases
Labeling
- Patient labeling should be written in plain language
- Visual aids like pictorials, graphics, or tables should be included when appropriate
- Clear statement about intended population required
- Information about benefits, risks, contraindications, warnings and precautions should be included
- Likelihoods of benefits and risks should be expressed in absolute terms
Safety
- Critical aspects of benefit, risk and uncertainty should be included in preference elicitation
- Risk attributes descriptions should appropriately convey severity and impact
- Study should measure preferences of representative sample to generalize to population
Other considerations
- Patient-centeredness should be ensured in study design
- Study population should be representative and support generalizability
- Appropriate methods for eliciting preferences should be selected
- Attributes and levels should be carefully selected and justified
- Study should reflect heterogeneity of patient preferences
- Study should follow good research practices by recognized organizations
- Data integrity and validity should be maintained
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
- Applying Human Factors Engineering and Usability Engineering to Medical Devices
- Human Factors Information Needed in Medical Device Marketing Submissions (Draft)
- Device Labeling Requirements and Content for Premarket Approval Applications
Related references and norms 📂
- 21 CFR Parts 50, 56, and 812: Protection of Human Subjects, Institutional Review Boards, Investigational Device Exemptions
- 21 CFR Parts 801 and 809: Labeling Requirements
- 21 CFR 860.7: Determination of Safety and Effectiveness