Electronic Submission Format Requirements for Orphan Drug and Humanitarian Use Device Designation Requests (DRAFT)
This guidance applies to electronic submissions of orphan-drug and humanitarian use device (HUD) designation requests to the Office of Orphan Products Development (OPD), including related submissions such as amendments, correspondence, and annual reports.
This is a draft guidance. Not for implementation.
Recommended Actions
- Determine preferred submission method (ESG or physical media)
- If using ESG:
- Complete preparatory activities
- Register and obtain digital certificate
- Follow format requirements
- If using physical media:
- Prepare properly labeled CD-ROMs
- Include paper cover letter
- Follow protective packaging requirements
- For all submissions:
- Use text-based format where possible
- Include proper bookmarks and hyperlinks
- Follow table of contents structure per regulations
- Ensure virus-free status
- Include all required documentation
- Maintain electronic format for subsequent submissions
- Keep contact information for technical support:
- ESG preparation: esgprep@fda.gov
- Orphan drug format questions: desigesub@fda.hhs.gov
- HUD format questions: hudesub@fda.hhs.gov
Key Considerations
Software
- Documents should be submitted in text-based format when possible
- PDF format should be used for references and publications
- Documents should enable clear viewing, easy navigation, and ability to print page by page
- Documents should allow copying of text, images and data into other common software formats
Cybersecurity
- Submissions must be virus-free with documentation of virus checking software used
- For FDA Gateway submissions, digital certificates are required for electronic signatures
- Secure messaging standards must be followed
Labelling
- Physical media must be properly labeled with:
- Type of designation request
- Request number
- Company name
- Drug/Device name
- Submission type and date
- Number of media units
- Contact information
Other considerations
- Two submission methods available:
- FDA Electronic Submissions Gateway (ESG) - preferred method
- Physical media (e.g., CD-ROMs) sent directly to OPD
- Cover letters must include submission description, electronic media details, virus-free statement
- Table of contents must follow regulatory requirements (21 CFR 316.20 for orphan drugs, 21 CFR 814.102 for HUDs)
- Bookmarks and hypertext links required for efficient navigation
- Once started electronically, subsequent submissions should continue electronically
Relevant Guidances
- Electronic Records and Electronic Signatures - Scope and Application
- Humanitarian Device Exemption (HDE) Program: Operational Requirements and Benefit-Risk Assessment
- Humanitarian Use Device (HUD) Designation Requests: Content and Format
- Electronic Copy (eCopy) Program for Medical Device Submissions: Technical Standards and Requirements
Related references and norms
- 21 CFR Part 11: Electronic Records; Electronic Signatures
- 21 CFR Part 316: Orphan Drugs
- 21 CFR Part 814: Premarket Approval of Medical Devices
Original guidance
- Electronic Submission Format Requirements for Orphan Drug and Humanitarian Use Device Designation Requests
- HTML / PDF
- Issue date: 2006-02-01
- Last changed date: 2021-05-13
- Status: DRAFT
- Official FDA topics: Medical Devices, Administrative / Procedural, Drugs, Biologics
- ReguVirta summary file ID: 5e82dfef77d17f5796bf7e03defba18c
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