II 

Calendar No. 416 

114TH CONGRESS 

2D 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, Mr. HATCH, and Mr. 

ENZI) introduced the following bill; which was read twice and referred to 

the Committee on Health, Education, Labor, and Pensions 

APRIL 5, 2016 

Reported by Mr. ALEXANDER, with an amendment 

[Strike out all after the enacting clause and insert the part printed in italic] 

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, 


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1 SECTION 1. SHORT TITLE. 

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

3 Therapies for Rare Diseases Act of 2015’’. 

4 SEC. 2. TARGETED DRUGS FOR RARE DISEASES. 

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

6 (21 U.S.C. 351 et seq.) is amended by inserting after sec7 tion 506F the following: 

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

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

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

11 ‘‘(1) facilitate the development, review, and ap12 proval of genetically targeted drugs to address an 

13 unmet medical need in one or more patient sub14 groups (or gene variant subpopulations) with respect 

15 to rare diseases or conditions that are serious or life16 threatening; and 

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

18 methods, including surrogate endpoints and other 

19 biomarkers for such purposes. 

20 ‘‘(b) LEVERAGING OF DATA FROM PREVIOUSLY AP21 PROVED DRUG APPLICATION OR APPLICATIONS.—The 

22 Secretary may, consistent with applicable standards for 

23 approval under this Act or section 351 of the Public 

24 Health Service Act, allow the sponsor of a genetically tar25 geted drug to rely upon data and information— 


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

2 (or another sponsor that has provided the sponsor 

3 with a contractual right of reference to such data 

4 and information); and 

5 ‘‘(2) submitted by a sponsor described in para6 graph (1) in support of one or more applications 

7 previously approved under this Act or section 351 of 

8 the Public Health Service Act, 

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

10 genetically targeted technology, or the same variant pro11 tein targeted technology, as the drug or drugs that are 

12 the subject of an application or applications described in 

13 paragraph (2). 

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

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

16 a drug which— 

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

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

19 of the Public Health Service Act for the treat20 ment of a rare disease or condition (as such 

21 term is defined in section 526) that is serious 

22 or life-threatening; 

23 ‘‘(B) incorporates or utilizes a genetically 

24 targeted technology or a variant protein tar25 geted technology; and 


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1 ‘‘(C) may result in the modulation (includ2 ing suppression, up-regulation, or activation) of 

3 the function of a gene or its associated gene 

4 product; 

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

6 means a technology comprising non-replicating nu7 cleic acid or analogous compounds with a common or 

8 similar chemistry that is intended to treat one or 

9 more subsets of patients with the same disease, in10 cluding due to other variants in the same gene; and 

11 ‘‘(3) the term ‘variant protein targeted tech12 nology’ means a technology or compound that modu13 lates the function of a variant protein, due to a gene 

14 variant, intended to treat one or more subsets of pa15 tients with the same disease, due to other variants 

16 in the same gene. 

17 ‘‘(d) RULE OF CONSTRUCTION.—Nothing in this sec18 tion shall be construed to— 

19 ‘‘(1) alter the authority of the Secretary to ap20 prove drugs pursuant to this Act or section 351 of 

21 the Public Health Service Act (as authorized prior 

22 to the date of enactment of the Advancing Targeted 

23 Therapies for Rare Diseases Act of 2015), including 

24 the standards of evidence, and applicable conditions, 

25 for approval under such Act; or 


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1 ‘‘(2) confer any new rights, beyond those au2 thorized under this section, with respect to the per3 missibility of referencing information contained in 

4 another application submitted under section 

5 505(b)(1) of this Act or section 351(a) of the Public 

6 Health Service Act.’’. 

7 SECTION 1. SHORT TITLE. 

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

9 Therapies for Rare Diseases Act of 2016’’. 

10 SEC. 2. TARGETED DRUGS FOR RARE DISEASES. 

11 Subchapter B of chapter V of the Federal Food, Drug, 

12 and Cosmetic Act (21 U.S.C. 360aa et seq.) is amended by 

13 inserting after section 529 the following: 

14 ‘‘SEC. 529A. TARGETED DRUGS FOR RARE DISEASES. 

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

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

17 ‘‘(1) facilitate the development, review, and ap18 proval of genetically targeted drugs and variant pro19 tein targeted drugs to address an unmet medical need 

20 in one or more patient subgroups, including sub21 groups of patients with different mutations of a gene, 

22 with respect to rare diseases or conditions that are se23 rious or life-threatening; and 


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1 ‘‘(2) maximize the use of scientific tools or meth2 ods, including surrogate endpoints and other bio3 markers for such purposes. 

4 ‘‘(b) LEVERAGING OF DATA FROM PREVIOUSLY AP5 PROVED DRUG APPLICATION OR APPLICATIONS.—The Sec6 retary may, consistent with applicable standards for ap7 proval under this Act or section 351(a) of the Public Health 

8 Service Act, allow the sponsor of an application under sec9 tion 505(b)(1) of this Act or section 351(a) of the Public 

10 Health Service Act for a genetically targeted drug or a vari11 ant protein targeted drug to rely upon data and informa12 tion— 

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

14 (or another sponsor that has provided the sponsor 

15 with a contractual right of reference to such data and 

16 information); and 

17 ‘‘(2) submitted by a sponsor described in para18 graph (1) in support of one or more previously ap19 proved applications that were submitted under section 

20 505(b)(1) of this this Act or section 351(a) of the Pub21 lic Health Service Act, 

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

23 genetically targeted technology as the drug or drugs that 

24 are the subject of an application or applications described 

25 in paragraph (2) or for a variant protein targeted drug 


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1 that is the same or incorporates or utilizes the same variant 

2 protein targeted drug, as the drug or drugs that are the 

3 subject of an application or applications described in para4 graph (2). 

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

6 ‘‘(1) the term ‘genetically targeted drug’ means a 

7 drug that— 

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

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

10 the Public Health Service Act for the treatment 

11 of a rare disease or condition (as such term is 

12 defined in section 526) that is serious or life13 threatening; 

14 ‘‘(B) may result in the modulation (includ15 ing suppression, up-regulation, or activation) of 

16 the function of a gene or its associated gene 

17 product; and 

18 ‘‘(C) incorporates or utilizes a genetically 

19 targeted technology; 

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

21 means a technology comprising non-replicating nu22 cleic acid or analogous compounds with a common or 

23 similar chemistry that is intended to treat one or 

24 more patient subgroups, including subgroups of pa25 tients with different mutations of a gene, with the 


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1 same disease or condition, including a disease or con2 dition due to other variants in the same gene; and 

3 ‘‘(3) the term ‘variant protein targeted drug’ 

4 means a drug that— 

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

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

7 the Public Health Service Act for the treatment 

8 of a rare disease or condition (as such term is 

9 defined in section 526) that is serious or life10 threatening; 

11 ‘‘(B) modulates the function of a product of 

12 a mutated gene where such mutation is respon13 sible in whole or in part for a given disease or 

14 condition; and 

15 ‘‘(C) is intended to treat one or more pa16 tient subgroups, including subgroups of patients 

17 with different mutations of a gene, with the same 

18 disease or condition. 

19 ‘‘(d) RULE OF CONSTRUCTION.—Nothing in this sec20 tion shall be construed to— 

21 ‘‘(1) alter the authority of the Secretary to ap22 prove drugs pursuant to this Act or section 351 of the 

23 Public Health Service Act (as authorized prior to the 

24 date of enactment of this section), including the 


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1 standards of evidence, and applicable conditions, for 

2 approval under such applicable Act; or 

3 ‘‘(2) confer any rights, beyond those authorized 

4 under this Act or the Public Health Service Act prior 

5 to enactment of this section, with respect to the per6 missibility of referencing information contained in 

7 another application submitted under section 505(b)(1) 

8 of this Act or section 351(a) of the Public Health 

9 Service Act.’’. 


srobinson on DSK5SPTVN1PROD with BILLS
Calendar No. 416 

114TH CONGRESS 

2D SESSION S. 2030 

A BILL 

To allow the sponsor of an application for the apviously approved targeted drugs. information with respect to such sponsor’s pre- proval of a targeted drug to rely upon data and 

APRIL 5, 2016 

Reported with an amendment 


srobinson on DSK5SPTVN1PROD with BILLS