21 U.S.C. § 360ff–1: Targeted drugs for rare…

Summary

Helps speed up the development and approval of drugs that aim at specific genes or abnormal proteins for rare, serious, or life‑threatening diseases. It lets the Secretary allow a drug sponsor to use data and information from one or more earlier approved applications by the same sponsor or by another sponsor that gave permission. It also encourages using scientific tools, like surrogate measures and biomarkers, to help show a drug works for specific patient groups, including groups with different mutations in the same gene. Key terms (short): genetically targeted drug — a drug for a rare, serious disease that changes a gene or its product and uses gene‑targeting technology; genetically targeted technology — non‑replicating genetic material or similar compounds with the same chemistry meant to treat patient subgroups; variant protein targeted drug — a drug for a rare, serious disease that changes a mutated protein and is aimed at patient subgroups. Nothing here changes the Secretary’s approval power or the evidence rules, and it does not create new rights beyond those in place before December 13, 2016.

Title 21, §360ff–1

Food and Drugs, Source: USLM XML via OLRC

(a)Purpose

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)Leveraging of data from previously approved drug application or applications

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,

for a drug that incorporates or utilizes the same or similar genetically targeted technology as the drug or drugs that are the subject of an application or applications described in paragraph (2) or for a variant protein targeted drug that is the same or incorporates or utilizes the same variant protein targeted drug, as the drug or drugs that are the subject of an application or applications described in paragraph (2).

(c)Definitions

For purposes of this section

(1)

(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—

(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)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)Rule of construction

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