--- kind: "section" citation: "21 U.S.C. § 360ff–1" title: "21" title_heading: "Food and Drugs" number: "360ff–1" heading: "Targeted drugs for rare diseases" release: "119-102" date: "2026-07-12" url: "https://uscodex.org/usc/21/360ff-1" units: - "Chapter 9 — Federal Food, Drug, and Cosmetic Act" - "Subchapter V — Drugs and Devices" - "Part B — Drugs for Rare Diseases or Conditions" --- # §360ff–1. Targeted drugs for rare diseases - (a) **Purpose—** The purpose of this section, through the approach provided for in [subsection (b)](#b), is to— - (1) facilitate the development, review, and approval of genetically targeted [drugs](/usc/21/321.md?p=g-1) and variant protein targeted [drugs](/usc/21/321.md?p=g-1) to address an unmet medical need in one or more patient subgroups, including subgroups of patients with different mutations of a gene, with respect to rare diseases or conditions that are serious or life-threatening; and - (2) maximize the use of scientific tools or methods, including surrogate endpoints and other biomarkers, for such purposes. - (b) **Leveraging of data from previously approved drug application or applications—** The [Secretary](/usc/21/321.md?p=d) may, consistent with applicable standards for approval under this chapter or section 351(a) of the Public Health Service Act [[42 U.S.C. 262(a)](/usc/42/262.md?p=a)], allow the [sponsor](/usc/21/360fff.md?p=8) of an application under [section 355(b)(1) of this title](/usc/21/355.md?p=b-1) or section 351(a) of the Public Health Service Act for a genetically targeted [drug](/usc/21/321.md?p=g-1) or a variant protein targeted [drug](/usc/21/321.md?p=g-1) to rely upon data and information— - (1) previously developed by the same [sponsor](/usc/21/360fff.md?p=8) (or another [sponsor](/usc/21/360fff.md?p=8) that has provided the [sponsor](/usc/21/360fff.md?p=8) with a contractual right of reference to such data and information); and - (2) submitted by a [sponsor](/usc/21/360fff.md?p=8) described in [paragraph (1)](#b-1) in support of one or more previously approved applications that were submitted under [section 355(b)(1) of this title](/usc/21/355.md?p=b-1) or section 351(a) of the Public Health Service Act, for a [drug](/usc/21/321.md?p=g-1) that incorporates or utilizes the same or similar genetically targeted technology as the [drug](/usc/21/321.md?p=g-1) or [drugs](/usc/21/321.md?p=g-1) that are the subject of an application or applications described in [paragraph (2)](#b-2) or for a variant protein targeted [drug](/usc/21/321.md?p=g-1) that is the same or incorporates or utilizes the same variant protein targeted [drug](/usc/21/321.md?p=g-1), as the [drug](/usc/21/321.md?p=g-1) or [drugs](/usc/21/321.md?p=g-1) that are the subject of an application or applications described in [paragraph (2)](#b-2). - (c) **Definitions—** For purposes of this section— - (1) the term “genetically targeted [drug](/usc/21/321.md?p=g-1)” means a [drug](/usc/21/321.md?p=g-1) that— - (A) is the subject of an application under [section 355(b)(1) of this title](/usc/21/355.md?p=b-1) or section 351(a) of the Public Health Service Act [[42 U.S.C. 262(a)](/usc/42/262.md?p=a)] for the treatment of a rare disease or condition (as such term is defined in [section 360bb of this title](/usc/21/360bb.md)) that is serious or life-threatening; - (B) may result in the modulation (including suppression, up-regulation, or activation) of the function of a gene or its associated gene [product](/usc/21/360eee.md?p=13); and - (C) incorporates or utilizes a genetically targeted technology; - (2) the term “genetically targeted technology” means a technology comprising non-replicating nucleic acid or analogous compounds with a common or similar chemistry that is intended to treat one or more patient subgroups, including subgroups of patients with different mutations of a gene, with the same disease or condition, including a disease or condition due to other variants in the same gene; and - (3) the term “variant protein targeted [drug](/usc/21/321.md?p=g-1)” means a [drug](/usc/21/321.md?p=g-1) that— - (A) is the subject of an application under [section 355(b)(1) of this title](/usc/21/355.md?p=b-1) or section 351(a) of the Public Health Service Act [[42 U.S.C. 262(a)](/usc/42/262.md?p=a)] for the treatment of a rare disease or condition (as such term is defined in [section 360bb of this title](/usc/21/360bb.md)) that is serious or life-threatening; - (B) modulates the function of a [product](/usc/21/360eee.md?p=13) of a mutated gene where such mutation is responsible in whole or in part for a given disease or condition; and - (C) is intended to treat one or more patient subgroups, including subgroups of patients with different mutations of a gene, with the same disease or condition. - (d) **Rule of construction—** Nothing in this section shall be construed to— - (1) alter the authority of the [Secretary](/usc/21/321.md?p=d) to approve [drugs](/usc/21/321.md?p=g-1) pursuant to this chapter or section 351 of the Public Health Service Act [[42 U.S.C. 262](/usc/42/262.md)] (as [authorized](/usc/21/360eee.md?p=2) prior to December 13, 2016), including the standards of evidence, and applicable conditions, for approval under such applicable chapter or Act; or - (2) confer any new rights, beyond those [authorized](/usc/21/360eee.md?p=2) under this chapter or the Public Health Service Act [[42 U.S.C. 201](/usc/42/201.md) et seq.] prior to December 13, 2016, with respect to the permissibility of a [sponsor](/usc/21/360fff.md?p=8) referencing information contained in another application submitted under [section 355(b)(1) of this title](/usc/21/355.md?p=b-1) or section 351(a) of the Public Health Service Act [[42 U.S.C. 262(a)](/usc/42/262.md?p=a)]. ## Source credit (June 25, 1938, ch. 675, § 529A, as added Pub. L. 114–255, div. A, title III, § 3012, Dec. 13, 2016, 130 Stat. 1091.) ## Notes ### Editorial Notes ### References in Text The Public Health Service Act, referred to in subsec. (d)(2), is act July 1, 1944, ch. 373, 58 Stat. 682, which is classified generally to chapter 6A (§ 201 et seq.) of Title 42, The Public Health and Welfare. For complete classification of this Act to the Code, see Short Title note set out under section 201 of Title 42 and Tables.