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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.

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By ReguVirta
3 min read

This is a draft guidance. Not for implementation.

What You Need to Know? 👇

What are the two methods for submitting electronic orphan drug and HUD designation requests to FDA?

You can submit through the FDA Electronic Submissions Gateway (ESG) as the preferred method, or send physical media (CD-ROMs) directly to OPD with a signed paper cover letter.

What file format should I use for electronic submissions to the Office of Orphan Products Development?

Documents should be submitted in text-based format when possible. References and publications may be submitted in PDF format. Avoid image-based scanned documents unless the original is only available in paper.

Do I need to convert previous paper submissions to electronic format when switching to electronic submissions?

No, FDA does not expect you to provide electronic files for previous paper submissions. You only need to submit subsequent amendments, correspondence, and annual reports electronically once you begin electronic submissions.

What information must be included on physical media labels for orphan drug designation requests?

Labels must include: designation type, request number (if known), company name, drug/device name, submission type, submission date, disk number, and electronic submission point of contact with phone number.

Yes, bookmarks and hypertext links are extremely important for navigation. Provide bookmarks for each table of contents item and hypertext links throughout the document for references, appendices, tables, and figures.

What virus protection requirements apply to electronic submissions sent on physical media?

You must include a statement in the cover letter that the submission is virus-free, along with a description of the antivirus software used, including the software name, version, and company.

What You Need to Do 👇

  1. Determine preferred submission method (ESG or physical media)
  2. If using ESG:
    • Complete preparatory activities
    • Register and obtain digital certificate
    • Follow format requirements
  3. If using physical media:
    • Prepare properly labeled CD-ROMs
    • Include paper cover letter
    • Follow protective packaging requirements
  4. 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
  5. 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:
    1. FDA Electronic Submissions Gateway (ESG) - preferred method
    2. 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 🔗

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 ID: 5e82dfef77d17f5796bf7e03defba18c
FDA guidance, Draft
Medical Devices Administrative / Procedural Drugs Biologics
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