Standard: HHS - 21 CFR PART 316


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Scope of this part.

(a)This part implements sections 525, 526, 527, and 528 of the act and provides procedures to encourage and facilitate the development of drugs for rare diseases or conditions, including biological products and antibiotics. This part sets forth the procedures and requirements for:

(1) Submissions to FDA of:

(i) Requests for recommendations for investigations of drugs for rare diseases or conditions;

(ii) Requests for designation of a drug for a rare disease or condition; and

(iii) Requests for gaining exclusive approval for a drug for a rare disease or condition.

(2) Allowing a sponsor to provide an investigational drug under a treatment protocol to patients who need the drug for treatment of a rare disease or condition.

(b) This part does not apply to food, medical devices, or drugs for veterinary use.

 (c) References in this part to regulatory sections of the Code of Federal Regulations are to chapter I of title 21, unless otherwise noted.


The purpose of this part is to establish standards and procedures for determining eligibility for the benefits provided for in section 2 of the Orphan Drug Act, including written recommendations for investigations of orphan drugs, a 7-year period of exclusive marketing, and treatment use of investigational orphan drugs. This part is also intended to satisfy Congress’ requirements that FDA promulgate procedures for the implementation of sections 525(a) and 526(a) of the act.

Organization: Food and Drug Administration (Medical Products Quality Assurance)
Document Number: 21 cfr part 316
Publish Date: 2015-04-01
Page Count: 14
Available Languages: EN
DOD Adopted: NO
ANSI Approved: NO
Most Recent Revision: YES
Current Version: YES
Status: Active

Document History

Document # Change Type Update Date Revision Status
21 CFR PART 316 Change Type: COMPLETE REVISION Update Date: 2014-04-01 Status: ACTV
21 CFR PART 316 Change Type: COMPLETE REVISION Update Date: 2017-04-01 Status: ACTV
21 CFR PART 316 Change Type: COMPLETE REVISION Update Date: 2016-04-01 Status: ACTV