21 U.S.C. § 360ff-1 Targeted Drugs For Rare Diseases

• Library: United States Statutes
• Edition: 2023
• Currency: Current through P.L. 118-22 with Court Rules updates (published on www.congress.gov on 12/01/2023)
• Year: 2023
• Citation: 21 U.S.C. § 360ff-1

(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) 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;