Leveraging Existing Clinical Data for Pediatric Medical Device Indications
This guidance outlines the framework for leveraging existing clinical data to support pediatric indications for medical devices in PMAs, de novo requests, and HDEs. It explains when and how clinical data from adult populations or different pediatric subpopulations can be extrapolated to demonstrate safety and effectiveness for pediatric use. The guidance does not apply to 510(k) submissions.
Recommended Actions
- Engage with FDA early in development process when considering extrapolation
- Follow the Pediatric Extrapolation Decision Tree to determine if extrapolation is appropriate:
- Assess relevancy of existing data
- Evaluate similarities/differences between populations
- Consider data quality
- Document rationale for extrapolation approach including:
- Justification for full vs partial extrapolation
- Analysis of population similarities/differences
- Statistical methodology
- Data quality assessment
- Consider separate evaluations for safety and effectiveness extrapolation
- Plan appropriate supplemental pediatric studies if needed
- Develop comprehensive labeling that reflects extrapolation approach and limitations
- Consider post-market surveillance needs, especially for safety
- Ensure statistical methodology is appropriate and agreed upon with FDA
- Maintain focus on providing valid scientific evidence to support reasonable assurance of safety and effectiveness
- Document all assumptions and limitations of extrapolation approach
Key Considerations
Clinical testing
- Extrapolation decisions for safety and effectiveness are made independently
- Full extrapolation of safety data is expected to occur rarely
- When prospective pediatric data are needed, study design and analysis should be appropriate for the circumstances
- Post-market surveillance may be required, particularly when full extrapolation of safety data is used
Human Factors
- Must consider differences in human factors between adult and pediatric populations that could impact device safety or effectiveness
- Special considerations needed for pediatric-specific challenges like growth during device use period
Labelling
- Must provide appropriate labeling to support safe and effective pediatric use
- Labeling should reflect any limitations of extrapolated data
Safety
- Safety extrapolation requires careful consideration of physiological differences between adults and children
- Full extrapolation of safety data is rare
- Additional safety studies in pediatric populations may be needed even when effectiveness data can be extrapolated
Other considerations
- Must consider similarities/differences in:
- Disease characteristics between populations
- Device characteristics and use between populations
- Location and duration of device implantation/contact
- Patient characteristics unique to pediatrics
- Statistical methodology must be appropriate for extrapolation approach
- Quality of existing data must be sufficient
- Early engagement with FDA recommended when considering extrapolation
Relevant Guidances
- Sex-Specific Considerations in Medical Device Clinical Studies: Patient Enrollment, Data Analysis and Reporting
- Benefit-Risk Determinations for Medical Device Premarket Review
- Premarket Assessment of Medical Devices for Pediatric Use
- Statistical Guidance for Reporting Diagnostic Test Results with Qualitative Outcomes
Related references and norms
- 21 CFR 860.7: Valid scientific evidence requirements
- 21 CFR 860.7(c)(1)&(2): Definition of valid scientific evidence
Original guidance
- Leveraging Existing Clinical Data for Pediatric Medical Device Indications
- HTML / PDF
- Issue date: 2016-06-21
- Last changed date: 2024-04-08
- Status: FINAL
- Official FDA topics: Medical Devices, Pediatric Product Development, Good Clinical Practice (GCP), Premarket Approval (PMA), Premarket, Biologics, HUD/HDE
- ReguVirta summary file ID: 4271e4766f3c157420c5b6a2bd02efbe
This post is licensed under CC BY 4.0 by the author.