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.
What You Need to Know? 👇
What is the population threshold for Humanitarian Use Device (HUD) designation?
A device qualifies for HUD designation if it treats or diagnoses a disease affecting not more than 8,000 individuals in the United States per year. This threshold was increased from 4,000 by the 21st Century Cures Act.
How do population estimates differ between therapeutic and diagnostic devices?
For therapeutic devices, count new patients diagnosed annually who are eligible for treatment. For diagnostic devices, count all patients who would be tested annually, including both positive and negative results.
What is an “orphan subset” in HUD designation requests?
An orphan subset is a medically justified subset of a non-rare disease where device properties (mechanism, adverse events, clinical experience) preclude its use in the broader population, limiting appropriate use to ≤8,000 patients annually.
How long does FDA take to review a HUD designation request?
OOPD has up to 45 calendar days from receipt to approve, request additional information, or disapprove the request. Additional information submissions may trigger a new 45-day review period.
Can a device be used multiple times per patient and still qualify for HUD designation?
Yes. HUD designation depends on the number of patients who would use the device annually, not the total number of device uses. Multiple uses per patient don’t disqualify the device.
What happens after receiving HUD designation approval?
HUD designation makes you eligible to submit a Humanitarian Device Exemption (HDE) marketing application to CDRH or CBER. The designation doesn’t guarantee HDE approval but is a prerequisite for submission.
What You Need to Do 👇
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