Use of Real-World Data and Real-World Evidence to Support Medical Device Regulatory Decision-Making (DRAFT)
This guidance clarifies FDA's evaluation process for real-world data (RWD) to determine if it can generate real-world evidence (RWE) suitable for medical device regulatory decision-making. It applies to regulatory submissions for medical devices and provides expanded recommendations for sponsors considering using RWE to support submissions.
This is a draft guidance. Not for implementation.
Recommended Actions
- Conduct fit-for-purpose assessment of RWD relevance and reliability
- Document and provide in FDA submission:
- Purpose of using RWE
- Description of RWD sources
- Study protocol and analysis plan
- Data management and quality control plans
- Human subject protections
- Validation/adjudication results if conducted
- Ensure adequate documentation of:
- Data collection procedures
- Quality control processes
- Data linkage methodologies
- Changes in clinical practice
- Missing data assessment
- Prepare regulatory submission including:
- Cover letter identifying RWD/RWE components
- Fit-for-purpose assessment
- Protocol
- Study report
- Additional required documentation
- Consider early engagement with FDA through Q-submission program to discuss RWE approach
Key Considerations
Clinical testing
- Clinical evidence can be generated from RWD alone or combined with traditional clinical studies
- Studies using RWD should be carefully designed to mitigate potential bias
- Protocol and analysis plan should be created prior to analyzing RWD
- Sample size should be adequate to detect clinically meaningful differences
Safety
- Safety assessment should be based on comparison of serious device-related adverse event rates
- Adverse event reporting plans must be included
- Safety endpoints and outcomes should be clearly defined
Other considerations
- RWD must be relevant and reliable for generating RWE
- Data quality and integrity must be demonstrated
- Appropriate data management practices must be in place
- Patient privacy protections must be established
- Study protocols must be documented and followed
- Changes in clinical practice over time must be considered
- Data linkages across sources must use predefined methodologies
- Statistical analysis plans must be pre-specified
- Documentation of previous use of RWD source should be provided
Relevant Guidances
- Use of Real-World Data to Generate Evidence for Medical Device Regulatory Decisions
- Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program
Related references and norms
- ICD-10-CM: International Classification of Diseases, Tenth Revision, Clinical Modification
- LOINC: Logical Observation Identifiers, Names, and Codes
- SNOMED CT: Systematized Nomenclature of Medicine Clinical Terms
- CPT: Current Procedural Terminology
Original guidance
- Use of Real-World Data and Real-World Evidence to Support Medical Device Regulatory Decision-Making
- HTML / PDF
- Issue date: 2023-12-14
- Last changed date: 2024-02-07
- Status: DRAFT
- Official FDA topics: Radiation-Emitting Products, Real World Data / Real World Evidence (RWD/RWE), Medical Devices, Postmarket, Premarket
- ReguVirta summary file ID: e27dd4ae6000d19be8c0d4480b326bc9
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