II 

114TH CONGRESS 

1ST SESSION S. 2030 

To allow the sponsor of an application for the approval of a targeted drug 

to rely upon data and information with respect to such sponsor’s previously approved targeted drugs. 

IN THE SENATE OF THE UNITED STATES 

SEPTEMBER 15, 2015 

Mr. BENNET (for himself, Mr. BURR, Ms. WARREN, and Mr. HATCH) introduced the following bill; which was read twice and referred to the Committee on Health, Education, Labor, and Pensions 

A BILL 

To allow the sponsor of an application for the approval 

of a targeted drug to rely upon data and information 

with respect to such sponsor’s previously approved targeted drugs. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Advancing Targeted 

5 Therapies for Rare Diseases Act of 2015’’. 


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1 SEC. 2. TARGETED DRUGS FOR RARE DISEASES. 

2 Title V of the Federal Food, Drug, and Cosmetic Act 

3 (21 U.S.C. 351 et seq.) is amended by inserting after sec4 tion 506F the following: 

5 ‘‘SEC. 506G. TARGETED DRUGS FOR RARE DISEASES. 

6 ‘‘(a) PURPOSE.—The purpose of this section, through 

7 the approach provided for in subsection (b), is to— 

8 ‘‘(1) facilitate the development, review, and ap9 proval of genetically targeted drugs to address an 

10 unmet medical need in one or more patient sub11 groups (or gene variant subpopulations) with respect 

12 to rare diseases or conditions that are serious or life13 threatening; and 

14 ‘‘(2) maximize the use of scientific tools or 

15 methods, including surrogate endpoints and other 

16 biomarkers for such purposes. 

17 ‘‘(b) LEVERAGING OF DATA FROM PREVIOUSLY AP18 PROVED DRUG APPLICATION OR APPLICATIONS.—The 

19 Secretary may, consistent with applicable standards for 

20 approval under this Act or section 351 of the Public 

21 Health Service Act, allow the sponsor of a genetically tar22 geted drug to rely upon data and information— 

23 ‘‘(1) previously developed by the same sponsor 

24 (or another sponsor that has provided the sponsor 

25 with a contractual right of reference to such data 

26 and information); and 


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1 ‘‘(2) submitted by a sponsor described in para2 graph (1) in support of one or more applications 

3 previously approved under this Act or section 351 of 

4 the Public Health Service Act, 

5 for a drug that incorporates or utilizes the same or similar 

6 genetically targeted technology, or the same variant pro7 tein targeted technology, as the drug or drugs that are 

8 the subject of an application or applications described in 

9 paragraph (2). 

10 ‘‘(c) DEFINITIONS.—For purposes of this section— 

11 ‘‘(1) the term ‘genetically targeted drug’ means 

12 a drug which— 

13 ‘‘(A) is the subject of an application under 

14 section 505(b)(1) of this Act or section 351(a) 

15 of the Public Health Service Act for the treat16 ment of a rare disease or condition (as such 

17 term is defined in section 526) that is serious 

18 or life-threatening; 

19 ‘‘(B) incorporates or utilizes a genetically 

20 targeted technology or a variant protein tar21 geted technology; and 

22 ‘‘(C) may result in the modulation (includ23 ing suppression, up-regulation, or activation) of 

24 the function of a gene or its associated gene 

25 product; 


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1 ‘‘(2) the term ‘genetically targeted technology’ 

2 means a technology comprising non-replicating nu3 cleic acid or analogous compounds with a common or 

4 similar chemistry that is intended to treat one or 

5 more subsets of patients with the same disease, in6 cluding due to other variants in the same gene; and 

7 ‘‘(3) the term ‘variant protein targeted tech8 nology’ means a technology or compound that modu9 lates the function of a variant protein, due to a gene 

10 variant, intended to treat one or more subsets of pa11 tients with the same disease, due to other variants 

12 in the same gene. 

13 ‘‘(d) RULE OF CONSTRUCTION.—Nothing in this sec14 tion shall be construed to— 

15 ‘‘(1) alter the authority of the Secretary to ap16 prove drugs pursuant to this Act or section 351 of 

17 the Public Health Service Act (as authorized prior 

18 to the date of enactment of the Advancing Targeted 

19 Therapies for Rare Diseases Act of 2015), including 

20 the standards of evidence, and applicable conditions, 

21 for approval under such Act; or 

22 ‘‘(2) confer any new rights, beyond those au23 thorized under this section, with respect to the per24 missibility of referencing information contained in 

25 another application submitted under section 


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1 505(b)(1) of this Act or section 351(a) of the Public 

2 Health Service Act.’’. 

Æ 


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