II 

113TH CONGRESS 

2D SESSION S. 2996 

To create a limited population pathway for approval of certain antibacterial 

drugs. 

IN THE SENATE OF THE UNITED STATES 

DECEMBER 10, 2014 

Mr. BENNET (for himself 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 create a limited population pathway for approval of 

certain antibacterial 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 ‘‘Promise for Antibiotics 

5 and Therapeutics for Health Act’’ or the ‘‘PATH Act’’. 

6 SEC. 2. LIMITED POPULATION PATHWAY FOR ANTI7 BACTERIAL DRUGS. 

8 Section 506 of the Federal Food, Drug, and Cosmetic 

9 Act (21 U.S.C. 356) is amended— 


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1 (1) by transferring subsection (e) so that it ap2 pears before subsection (f); and 

3 (2) by adding at the end the following: 

4 ‘‘(g) LIMITED POPULATION PATHWAY FOR ANTI5 BACTERIAL DRUGS.— 

6 ‘‘(1) IN GENERAL.—The Secretary shall estab7 lish a program under which the Secretary may, at 

8 the request of a sponsor, approve an antibacterial 

9 drug, alone or in combination with one or more 

10 drugs, as a limited population antibacterial drug, 

11 upon a determination that such drug is intended to 

12 treat a serious or life-threatening disease, condition, 

13 or infection and address an unmet medical need for 

14 such disease, condition, or infection within an identi15 fiable limited population. 

16 ‘‘(2) LIMITED POPULATION PATHWAY.— 

17 ‘‘(A) IN GENERAL.—The sponsor of an 

18 antibacterial drug that the Secretary deter19 mines to be eligible for approval as a limited 

20 population antibacterial drug shall be required 

21 to demonstrate the safety and effectiveness of 

22 such drug, as required under section 505(d) or 

23 section 351(a) of the Public Health Service Act, 

24 for the intended use of the drug. The Secretary 

25 shall determine the safety and effectiveness of 


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1 an antibacterial drug under the limited popu2 lation pathway for antibacterial drugs in ac3 cordance with subparagraph (B). An anti4 bacterial drug shall be eligible for approval 

5 under the limited population pathway only upon 

6 the request of the sponsor. 

7 ‘‘(B) CONSIDERATIONS.— 

8 ‘‘(i) BENEFIT-RISK PROFILE.—The 

9 Secretary’s determination of safety and ef10 fectiveness of a limited population anti11 bacterial drug shall reflect the benefit-risk 

12 profile of the drug in the intended limited 

13 population, taking into account the sever14 ity, rarity, or prevalence of the infection 

15 the drug is intended to treat and the avail16 ability or lack of alternative treatment for 

17 such infection. Approval of a drug under 

18 the limited population antibacterial drug 

19 pathway shall not be denied due to a lack 

20 of evidence to fully establish a favorable 

21 benefit-risk profile in a population that is 

22 broader than the intended limited popu23 lation. 

24 ‘‘(ii) TYPES OF EVIDENCE.—In deter25 mining whether to approve a drug under 


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1 the limited population pathway, the Sec2 retary— 

3 ‘‘(I) shall rely on sufficient evi4 dence, which may include traditional 

5 endpoints, alternate endpoints, or a 

6 combination of traditional and alter7 nate endpoints, and, as appropriate, 

8 small clinical data sets; and 

9 ‘‘(II) may rely on supplemental 

10 data, including preclinical evidence, 

11 pharmacologic or pathophysiologic evi12 dence, nonclinical susceptibility, phar13 macokinetic data, and other such con14 firmatory evidence as the Secretary 

15 determines appropriate. 

16 ‘‘(3) REQUIREMENTS.—With respect to a drug 

17 approved through the limited population pathway, 

18 the Secretary shall require— 

19 ‘‘(A) the labeling of such antibacterial 

20 drug, such as through a logo or other means, 

21 to indicate that the drug has been approved for 

22 use only in a limited population and that the 

23 safety and efficacy of the drug has been dem24 onstrated only with respect to such limited pop25 ulation; and 


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1 ‘‘(B) the sponsor to submit copies of all 

2 promotional materials related to the limited 

3 population antibacterial drug, at least 30 days 

4 prior to dissemination of the materials. 

5 ‘‘(4) OTHER PROGRAMS.—A sponsor of a drug 

6 that seeks approval of a drug through the limited 

7 population pathway for antibacterial drugs may also 

8 seek approval of such drug under subsections (a), 

9 (b), and (c), and sections 505E and 524. 

10 ‘‘(5) GUIDANCE.—Not later than 18 months 

11 after the date of enactment of the Promise for Anti12 biotics and Therapeutics for Health Act, the Sec13 retary shall issue draft guidance describing criteria, 

14 processes, and other general considerations for dem15 onstrating the safety and effectiveness of limited 

16 population antibacterial drugs and how the pathway 

17 can be expanded to other therapeutic areas in addi18 tion to antibacterial infections. The Secretary may 

19 approve antibacterial drugs through such limited 

20 population pathway prior to issuing guidance under 

21 this paragraph. 

22 ‘‘(6) POSTAPPROVAL MONITORING PROGRAMS

23 FOR ANTIBACTERIAL DRUGS.—The Secretary, in 

24 consultation with the Commissioner and other rel25 evant heads of agencies, shall conduct postapproval 


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1 monitoring programs to study how antibacterial 

2 drugs approved through the pathway under this sub3 section are used and to monitor changes in bacterial 

4 resistance to drugs, including drugs approved under 

5 this pathway. 

6 ‘‘(7) ADVICE.—The Secretary shall provide 

7 prompt advice to the sponsor of a drug for which the 

8 sponsor seeks approval through the limited popu9 lation pathway for antibacterial drugs to enable the 

10 sponsor to plan a development program to obtain the 

11 necessary data for approval of such drug through 

12 the limited population pathway for antibacterial 

13 drugs and to conduct any additional studies that 

14 would be required to gain approval of such drug for 

15 use in a broader population. 

16 ‘‘(8) TERMINATION OF LIMITATIONS.—If, after 

17 approval of a drug through the limited population 

18 pathway for antibacterial drugs, the Secretary ap19 proves a broader indication for such drug for which 

20 the sponsor applies under section 505(b) or section 

21 351 of the Public Health Service Act, the Secretary 

22 may remove any postmarketing conditions, including 

23 requirements with respect to labeling and review of 

24 promotional materials under paragraph (3) and 

25 postapproval monitoring under paragraph (6), appliVerDate Sep 11 2014 19:39 Dec 16, 2014 Jkt 049200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S2996.IS S2996

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1 cable to the approval of the drug through the limited 

2 population pathway for antibacterial drugs. 

3 ‘‘(9) RULES OF CONSTRUCTION.— 

4 ‘‘(A) STANDARDS OF EVIDENCE AND AU5 THORITY OF SECRETARY.—Nothing in this sub6 section shall be construed to alter the standards 

7 of evidence applicable to the review and ap8 proval of a drug under this Act or the Public 

9 Health Service Act, or to modify or limit the 

10 authority of the Secretary to approve or mon11 itor drugs pursuant to this Act or the Public 

12 Health Service Act as authorized prior to the 

13 date of enactment of the Promise for Anti14 biotics and Therapeutics for Health Act. 

15 ‘‘(B) PRESCRIBING AUTHORITY.—Nothing 

16 in this subsection shall be construed to restrict 

17 the prescribing of antibiotics or other products, 

18 including drugs approved under the limited pop19 ulation pathway, by health care professionals, 

20 or to limit the practice of health care. 

21 ‘‘(10) EXPANSION OF PATHWAY.—Beginning on 

22 October 1, 2016, the limited population pathway for 

23 antibiotic drugs may be expanded to apply to ap24 proval of other drugs intended to treat a serious or 

25 life-threatening illness. The approval of such drugs 


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1 shall be subject to the considerations and require2 ments described in this subsection, unless the Sec3 retary delivers a report to Congress prior to that 

4 date explaining why such pathway should not be 

5 used for other therapeutic areas in addition to anti6 bacterial infections.’’. 

Æ 


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