№ lp_2_1_00781
Regulatory guidance and structured application text outlining statutory and scientific requirements for sponsors seeking orphan-drug designation in the United States under 21 CFR Part 316.
Document Type: Regulatory guidance and application template
Subject: Orphan-drug designation request
Regulatory Framework: Code of Federal Regulations Title 21 (2019)
Jurisdiction: United States
Relevant Authority: Food and Drug Administration
Applicable Regulation: 21 CFR Part 316
Referenced Guidance: M4 Organization of the Common Technical Document for the Registration of Pharmaceuticals for Human Use Guidance for Industry
Module Reference: Module 1.12.17
Intended Applicant: Sponsor of a drug product
Scope: Rare diseases or conditions affecting fewer than 200,000 persons in the United States or meeting cost-recovery criteria
Submission Requirements: Completed, dated, and signed request in duplicate
Content Requirements: Sponsor information, drug description, scientific rationale, regulatory status, prevalence documentation, references
Sections Included: Title Page; Orphan-Drug Designation Request Statement; Information on the Sponsor; Description of Rare Disease and Proposed Use; Description of the Drug and Rationale; Statement Regarding Follow-On Drugs; Disease Subsets/Plausibility; Summary of Regulatory Status; References
Price: 8 / 10 USD
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