§360bb — Title 21 Food and Drugs | U.S. Code

Summary

A drug maker can ask the Secretary to call their medicine a treatment for a rare disease or condition. The request must come before the company files the drug approval or license application. The Secretary will decide if the drug is being or will be studied for a rare disease. A rare disease means it affects fewer than 200,000 people in the United States, or it affects more but sales are not expected to pay back the cost of developing the drug. The Secretary uses the facts that exist when the request is made. If the Secretary gives the rare-disease designation, the maker must meet two conditions. If the drug is approved or licensed, the maker must tell the Secretary at least one year before they stop making it. If the drug is still in preclinical or clinical studies and not yet approved or licensed, the maker must tell the Secretary if they decide to stop seeking approval or a license. The Secretary must make the designation public and create rules to carry out this process.

Title 21, §360bb

Food and Drugs — Source: USLM XML via OLRC

(a)(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,

(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)A designation of a drug under subsection (a) 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.

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

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

Notes & Related Subsidiaries

Editorial Notes

References in Text

Subsection (b), referred to in subsec. (a)(1), was redesignated as subsec. (c) of this section by Pub. L. 100–290, § 2(b), Apr. 18, 1988, 102 Stat. 90.

Amendments

1997—Subsec. (a)(1). Pub. L. 105–115, § 125(b)(2)(H), struck out “the submission of an application for certification of the drug under section 357 of this title,” before “or the submission of an application for licensing of the drug” in introductory provisions, inserted “or” at end of subpar. (A), redesignated subpar. (C) as (B), and struck out former subpar. (B) which read as follows: “if a certification for such drug is issued under section 357 of this title, or”. Subsec. (b)(1). Pub. L. 105–115, § 125(b)(2)(I)(i), struck out “, a certificate was issued for the drug under section 357 of this title,” before “or a license was issued”. Subsec. (b)(2). Pub. L. 105–115, § 125(b)(2)(I)(ii), struck out “, a certificate has not been issued for the drug under section 357 of this title,” before “or a license has not been issued” and “, approval of an application for certification under section 357 of this title,” before “or approval of a license”. 1988—Subsec. (a)(1). Pub. L. 100–290, § 2(a), inserted after first sentence “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, the submission of an application for certification of the drug under section 357 of this title, or the submission of an application for licensing of the drug under section 262 of title 42.” Subsecs. (b) to (d). Pub. L. 100–290, § 2(b), added subsec. (b) and redesignated former subsecs. (b) and (c) as (c) and (d), respectively. 1985—Subsec. (a)(1). Pub. L. 99–91 struck out “or” at end of subpar. (A), struck out subpar. (B) and substituted subpars. (B) and (C), and inserted “, certification,” after “approval”. 1984—Subsec. (a)(2). Pub. L. 98–551 substituted “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” for “which occurs so infrequently in the United States that”.

Statutory Notes and Related Subsidiaries

Effective Date

of 1985 AmendmentAmendment by Pub. L. 99–91 effective Aug. 15, 1985, see section 8(b) of Pub. L. 99–91, set out as a note under section 360aa of this title.

§360bb Designation of drugs for rare diseases or conditions