§360ff–1 — Title 21 Food and Drugs | U.S. Code

Summary

Makes it easier to develop and get approval for genetically targeted drugs and variant protein drugs that treat rare, serious, or life‑threatening illnesses in specific patient groups (including groups with different gene mutations). It also encourages using scientific tools like surrogate endpoints and biomarkers. The Secretary may allow a drug sponsor to use data they already developed (or data they have a legal right to use) and data that supported earlier approved drug applications when applying for these kinds of drugs. Genetically targeted drug: a drug for a rare, serious illness that changes a gene’s function or its product and uses genetically targeted technology. Genetically targeted technology: non‑replicating nucleic acid or similar compounds with common chemistry aimed at treating subgroups of the same disease. Variant protein targeted drug: a drug for a rare, serious illness that changes the function of a mutated gene product and targets patient subgroups. This does not change the Secretary’s approval authority or the rules about evidence or data‑referencing as they were before December 13, 2016.

Title 21, §360ff–1

Food and Drugs — Source: USLM XML via OLRC

(a)The purpose of this section, through the approach provided for in subsection (b), is to—

(1)facilitate the development, review, and approval of genetically targeted drugs and variant protein targeted drugs 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)The Secretary 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)], allow the sponsor of an application under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act for a genetically targeted drug or a variant protein targeted drug to rely upon data and information—

(1)previously developed by the same sponsor (or another sponsor that has provided the sponsor with a contractual right of reference to such data and information); and

(2)submitted by a sponsor described in paragraph (1) in support of one or more previously approved applications that were submitted under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act,

(c)For purposes of this section—

(1)the term “genetically targeted drug” means a drug that—

(A)is the subject of an application under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] for the treatment of a rare disease or condition (as such term is defined in section 360bb of this title) 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; 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” means a drug that—

(B)modulates the function of a product 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)Nothing in this section shall be construed to—

(1)alter the authority of the Secretary to approve drugs pursuant to this chapter or section 351 of the Public Health Service Act [42 U.S.C. 262] (as authorized 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 under this chapter or the Public Health Service Act [42 U.S.C. 201 et seq.] prior to December 13, 2016, with respect to the permissibility of a sponsor referencing information contained in another application submitted under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)].

Notes & Related Subsidiaries

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.

§360ff–1 Targeted drugs for rare diseases