U.S. CODE

U.S. Code

§ 360bb. Designation of drugs for rare diseases or conditions

(a) Request by sponsor; preconditions; “rare disease or condition” defined

(1) The manufacturer or the sponsor of a drug may request the Secretary to designate the drug as a drug for a rare disease or condition. A request for designation of a drug shall be made before the submission of an application under section 355 (b) of this title for the drug, or the submission of an application for licensing of the drug under section 262 of title 42. If the Secretary finds that a drug for which a request is submitted under this subsection is being or will be investigated for a rare disease or condition and—

(A) if an application for such drug is approved under section 355 of this title, or

(B) if a license for such drug is issued under section 262 of title 42,

the approval, certification, or license would be for use for such disease or condition, the Secretary shall designate the drug as a drug for such disease or condition. A request for a designation of a drug under this subsection shall contain the consent of the applicant to notice being given by the Secretary under subsection (b) of this section respecting the designation of the drug.

(2) For purposes of paragraph (1), the term “rare disease or condition” means any disease or condition which

(A) affects less than 200,000 persons in the United States, or

(B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug. Determinations under the preceding sentence with respect to any drug shall be made on the basis of the facts and circumstances as of the date the request for designation of the drug under this subsection is made.

(b) Notification of discontinuance of drug or application as condition

A designation of a drug under subsection (a) of this section shall be subject to the condition that—

(1) if an application was approved for the drug under section 355 (b) of this title or a license was issued for the drug under section 262 of title 42, the manufacturer of the drug will notify the Secretary of any discontinuance of the production of the drug at least one year before discontinuance, and

(2) if an application has not been approved for the drug under section 355 (b) of this title or a license has not been issued for the drug under section 262 of title 42 and if preclinical investigations or investigations under section 355 (i) of this title are being conducted with the drug, the manufacturer or sponsor of the drug will notify the Secretary of any decision to discontinue active pursuit of approval of an application under section 355 (b) of this title or approval of a license under section 262 of title 42.

Notice respecting the designation of a drug under subsection (a) of this section shall be made available to the public.

(d) Regulations

The Secretary shall by regulation promulgate procedures for the implementation of subsection (a) of this section.

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