Humanitarian Use Device (HUD) Designation Requests: Content and Format
This guidance document assists applicants in preparing and submitting Humanitarian Use Device (HUD) designation requests to FDA's Office of Orphan Products Development (OOPD). A HUD designation is required before submitting a Humanitarian Device Exemption (HDE) marketing application. The guidance explains how to demonstrate that a device is designed to treat or diagnose a disease/condition affecting not more than 8,000 individuals in the US per year.
Recommended Actions
- Determine if device is therapeutic or diagnostic to properly estimate affected population
- Prepare comprehensive device description including:
- Detailed specifications and dimensions
- Visual representations
- Mechanistic and operative aspects
- Compile robust scientific rationale with:
- All relevant nonclinical and clinical data
- Proof-of-principle data
- Both positive and negative results
- Document population estimates through:
- Literature citations
- Medical journals/textbooks
- Government statistics
- Expert consultations (minimum 3 independent experts if needed)
- For orphan subsets:
- Justify based on device properties/limitations
- Provide scientific/medical rationale for restricted use
- Submit complete request with:
- Cover letter
- Table of contents
- All required sections
- Supporting documentation
- Bibliography and references
- Include electronic copy (eCopy) as one of two required submission copies
Key Considerations
Non-clinical testing
- Nonclinical information on device verification and validation against design specifications is reviewed in HDE application, not HUD request
Labelling
- OOPD designates a device for a rare disease/condition, not for specific indications or labeling
- HUD designation may be broader than eventual HDE approved indications
Other considerations
- Population estimates differ for therapeutic vs diagnostic devices:
- Therapeutic: Number of new patients per year diagnosed and eligible for treatment
- Diagnostic: Number of patients per year subjected to diagnosis (positive and negative results)
- For orphan subsets of non-rare conditions:
- Must demonstrate device properties preclude use outside subset
- Cannot be based solely on clinical trial criteria, unmet need, standard of care, or price
- Pediatric considerations: Population under 22 years old
- Supporting documentation required through literature or expert consultation
- 45-day review timeline for OOPD
Relevant Guidances
- Humanitarian Device Exemption (HDE) Program: Operational Requirements and Benefit-Risk Assessment
- Meetings with the Office of Orphan Products Development: Procedures and Best Practices
Original guidance
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