Advancing Targeted Therapies for Rare Diseases Act of 2015

“506G. Targeted drugs for rare diseases

“(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 to address an unmet medical need in one or more patient subgroups (or gene variant subpopulations) 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 Act or section 351 of the Public Health Service Act, allow the sponsor of a genetically 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 applications previously approved under this Act or section 351 of the Public Health Service Act,

“(c) Definitions—For purposes of this section—

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

“(A) is the subject of an application under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act for the treatment of a rare disease or condition (as such term is defined in section 526) that is serious or life-threatening;

“(B) incorporates or utilizes a genetically targeted technology or a variant protein targeted technology; and

“(C) may result in the modulation (including suppression, up-regulation, or activation) of the function of a gene or its associated gene product;

“(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 subsets of patients with the same disease, including due to other variants in the same gene; and

“(3) the term variant protein targeted technology means a technology or compound that modulates the function of a variant protein, due to a gene variant, intended to treat one or more subsets of patients with the same disease, due to other variants in the same gene.

“(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 Act or section 351 of the Public Health Service Act (as authorized prior to the date of enactment of the Advancing Targeted Therapies for Rare Diseases Act of 2015), including the standards of evidence, and applicable conditions, for approval under such Act; or

“(2) confer any new rights, beyond those authorized under this section, with respect to the permissibility of referencing information contained in another application submitted under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act.”