Description
This is one in a series of guidance documents intended to assist sponsors making regulatory submissions to the Office of Orphan Products Development (OPD) in electronic format using the FDA Electronic Submissions Gateway (ESG) pathway or directly to OPD on physical media (e.g., CD-ROMs). This guidance discusses issues related to the electronic submission of requests for orphan-drug designation, humanitarian use device designation (HUD), and related submissions.
Scope & Applicability
Product Classes
3Specific designation request for medical devices (HUD)
Specific designation request for drugs treating rare diseases
Medical device intended for small populations
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
2PDF format used for publications and references
Preferred format for electronic documents to enable searching and copying
Related CFR Sections (3)
- 21CFR814.102§ 814.102 Designation of HUD status.
(a) Request for designation. Prior to submitting an HDE application, the applicant shall submit a request for HUD designation to FDA's Office of Orphan Products Development. The request shall contain the following:Read full regulation →
- 21CFR316.30§ 316.30 Annual reports of holder of orphan-drug designation.
Within 14 months after the date on which a drug was designated as an orphan drug and annually thereafter until marketing approval, the sponsor of a designated drug shall submit a brief progress report to the FDA Office of Orphan Products Development on the drug that includes:Read full regulation →
- 21CFR316.20§ 316.20 Content and format of a request for orphan-drug designation.
(a) A sponsor that submits a request for orphan-drug designation of a drug for a specified rare disease or condition shall submit each request in the form and containing the information required in paragraph (b) of this section. A sponsor may request orphan-drug designation of a previously unapproveRead full regulation →
Related Warning Letters (2)
- 2022-06-28
CGMP/Active Pharmaceutical Ingredient (API)/Adulterated
Fagron Group B.V.
- 2020-05-05
CGMP/Dietary Supplement/Adulterated/Misbranded
Hawaii Pharm LLC
See Also (8)
- Evaluation of Devices Used with Regenerative Medicine Advanced Therapies: Guidance for Industry (Status: Final)
- Humanitarian Use Device (HUD) Designations : Guidance for Industry and Food and Drug Administration Staff (Status: Final)
- COVID-19: Developing Drugs and Biological Products for Treatment or Prevention: Guidance for Industry (Status: Final)
- Chapter 1 - General (Status: Final)
- Using Electronic Means to Distribute Certain Product Information: Guidance for Industry (Status: Final)
- CPG Sec. 400.210, Radiofrequency Identification Feasibility Studies and Pilot Programs for Drugs (Status: Final)
- Guidance for Industry: Questions and Answers on Juice HACCP Regulation (2003) (Status: Final)
- Guidance for Industry: Juice Hazard Analysis Critical Control Point Hazards and Controls Guidance, First Edition (Status: Final)