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[Congressional Bills 114th Congress]
[From the U.S. Government Publishing Office]
[S. 185 Reported in Senate (RS)]

<DOC>

                                                       Calendar No. 425
114th CONGRESS
  2d Session
                                 S. 185

    To create a limited population pathway for approval of certain 
                          antibacterial drugs.

_______________________________________________________________________

                   IN THE SENATE OF THE UNITED STATES

                            January 16, 2015

Mr. Hatch (for himself, Mr. Bennet, Ms. Ayotte, Mr. Isakson, Mr. Kirk, 
 Mr. Carper, and Mr. Blumenthal) introduced the following bill; which 
  was read twice and referred to the Committee on Health, Education, 
                          Labor, and Pensions

                             April 18, 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 create a limited population pathway for approval of certain 
                          antibacterial drugs.

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

<DELETED>SECTION 1. SHORT TITLE.</DELETED>

<DELETED>    This Act may be cited as the ``Promise for Antibiotics and 
Therapeutics for Health Act'' or the ``PATH Act''.</DELETED>

<DELETED>SEC. 2. LIMITED POPULATION PATHWAY FOR ANTIBACTERIAL 
              DRUGS.</DELETED>

<DELETED>    Section 506 of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 356) is amended--</DELETED>
        <DELETED>    (1) by transferring subsection (e) so that it 
        appears before subsection (f); and</DELETED>
        <DELETED>    (2) by adding at the end the following:</DELETED>
<DELETED>    ``(g) Limited Population Pathway for Antibacterial 
Drugs.--</DELETED>
        <DELETED>    ``(1) In general.--The Secretary shall establish a 
        program under which the Secretary may, at the request of a 
        sponsor, approve an antibacterial drug, alone or in combination 
        with one or more drugs, as a limited population antibacterial 
        drug, upon a determination that such drug is intended to treat 
        a serious or life-threatening disease, condition, or infection 
        and address an unmet medical need for such disease, condition, 
        or infection within an identifiable limited 
        population.</DELETED>
        <DELETED>    ``(2) Limited population pathway.--</DELETED>
                <DELETED>    ``(A) In general.--The sponsor of an 
                antibacterial drug that the Secretary determines to be 
                eligible for approval as a limited population 
                antibacterial drug shall be required to demonstrate the 
                safety and effectiveness of such drug, as required 
                under section 505(d) or section 351(a) of the Public 
                Health Service Act, for the intended use of the drug. 
                The Secretary shall determine the safety and 
                effectiveness of an antibacterial drug under the 
                limited population pathway for antibacterial drugs in 
                accordance with subparagraph (B). An antibacterial drug 
                shall be eligible for approval under the limited 
                population pathway only upon the request of the 
                sponsor.</DELETED>
                <DELETED>    ``(B) Considerations.--</DELETED>
                        <DELETED>    ``(i) Benefit-risk profile.--The 
                        Secretary's determination of safety and 
                        effectiveness of a limited population 
                        antibacterial drug shall reflect the benefit-
                        risk profile of the drug in the intended 
                        limited population, taking into account the 
                        severity, rarity, or prevalence of the 
                        infection the drug is intended to treat and the 
                        availability or lack of alternative treatment 
                        for such infection. Approval of a drug under 
                        the limited population antibacterial drug 
                        pathway shall not be denied due to a lack of 
                        evidence to fully establish a favorable 
                        benefit-risk profile in a population that is 
                        broader than the intended limited 
                        population.</DELETED>
                        <DELETED>    ``(ii) Types of evidence.--In 
                        determining whether to approve a drug under the 
                        limited population pathway, the Secretary--
                        </DELETED>
                                <DELETED>    ``(I) shall rely on 
                                sufficient evidence, which may include 
                                traditional endpoints, alternate 
                                endpoints, or a combination of 
                                traditional and alternate endpoints, 
                                and, as appropriate, small clinical 
                                data sets; and</DELETED>
                                <DELETED>    ``(II) may rely on 
                                supplemental data, including 
                                preclinical evidence, pharmacologic or 
                                pathophysiologic evidence, nonclinical 
                                susceptibility, pharmacokinetic data, 
                                and other such confirmatory evidence as 
                                the Secretary determines 
                                appropriate.</DELETED>
        <DELETED>    ``(3) Requirements.--With respect to a drug 
        approved through the limited population pathway, the Secretary 
        shall require--</DELETED>
                <DELETED>    ``(A) the labeling of such antibacterial 
                drug, such as through a logo or other means, to 
                indicate that the drug has been approved for use only 
                in a limited population and that the safety and 
                efficacy of the drug has been demonstrated only with 
                respect to such limited population; and</DELETED>
                <DELETED>    ``(B) the sponsor to submit copies of all 
                promotional materials related to the limited population 
                antibacterial drug, at least 30 days prior to 
                dissemination of the materials.</DELETED>
        <DELETED>    ``(4) Other programs.--A sponsor of a drug that 
        seeks approval of a drug through the limited population pathway 
        for antibacterial drugs may also seek approval of such drug 
        under subsections (a), (b), and (c), and sections 505E and 
        524.</DELETED>
        <DELETED>    ``(5) Guidance.--Not later than 18 months after 
        the date of enactment of the Promise for Antibiotics and 
        Therapeutics for Health Act, the Secretary shall issue draft 
        guidance describing criteria, processes, and other general 
        considerations for demonstrating the safety and effectiveness 
        of limited population antibacterial drugs and how the pathway 
        can be expanded to other therapeutic areas in addition to 
        antibacterial infections. The Secretary may approve 
        antibacterial drugs through such limited population pathway 
        prior to issuing guidance under this paragraph.</DELETED>
        <DELETED>    ``(6) Postapproval monitoring programs for 
        antibacterial drugs.--The Secretary, in consultation with the 
        Commissioner and other relevant heads of agencies, shall 
        conduct postapproval monitoring programs to study how 
        antibacterial drugs approved through the pathway under this 
        subsection are used and to monitor changes in bacterial 
        resistance to drugs, including drugs approved under this 
        pathway.</DELETED>
        <DELETED>    ``(7) Advice.--The Secretary shall provide prompt 
        advice to the sponsor of a drug for which the sponsor seeks 
        approval through the limited population pathway for 
        antibacterial drugs to enable the sponsor to plan a development 
        program to obtain the necessary data for approval of such drug 
        through the limited population pathway for antibacterial drugs 
        and to conduct any additional studies that would be required to 
        gain approval of such drug for use in a broader 
        population.</DELETED>
        <DELETED>    ``(8) Termination of limitations.--If, after 
        approval of a drug through the limited population pathway for 
        antibacterial drugs, the Secretary approves a broader 
        indication for such drug for which the sponsor applies under 
        section 505(b) or section 351 of the Public Health Service Act, 
        the Secretary may remove any postmarketing conditions, 
        including requirements with respect to labeling and review of 
        promotional materials under paragraph (3) and postapproval 
        monitoring under paragraph (6), applicable to the approval of 
        the drug through the limited population pathway for 
        antibacterial drugs.</DELETED>
        <DELETED>    ``(9) Rules of construction.--</DELETED>
                <DELETED>    ``(A) Standards of evidence and authority 
                of secretary.--Nothing in this subsection shall be 
                construed to alter the standards of evidence applicable 
                to the review and approval of a drug under this Act or 
                the Public Health Service Act, or to modify or limit 
                the authority of the Secretary to approve or monitor 
                drugs pursuant to this Act or the Public Health Service 
                Act as authorized prior to the date of enactment of the 
                Promise for Antibiotics and Therapeutics for Health 
                Act.</DELETED>
                <DELETED>    ``(B) Prescribing authority.--Nothing in 
                this subsection shall be construed to restrict the 
                prescribing of antibiotics or other products, including 
                drugs approved under the limited population pathway, by 
                health care professionals, or to limit the practice of 
                health care.</DELETED>
        <DELETED>    ``(10) Expansion of pathway.--Beginning on October 
        1, 2016, the limited population pathway for antibiotic drugs 
        may be expanded to apply to approval of other drugs intended to 
        treat a serious or life-threatening illness. The approval of 
        such drugs shall be subject to the considerations and 
        requirements described in this subsection, unless the Secretary 
        delivers a report to Congress prior to that date explaining why 
        such pathway should not be used for other therapeutic areas in 
        addition to antibacterial infections.''.</DELETED>

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Promise for Antibiotics and 
Therapeutics for Health Act'' or the ``PATH Act''.

SEC. 2. ANTIBACTERIAL RESISTANCE MONITORING.

    Section 319E of the Public Health Service Act (42 U.S.C. 247d-5) is 
amended--
            (1) by redesignating subsections (f) and (g) as subsections 
        (k) and (l), respectively; and
            (2) by inserting after subsection (e), the following:
    ``(f) Monitoring at Federal Health Care Facilities.--The Secretary 
shall encourage reporting on aggregate antibacterial drug use and 
bacterial resistance to antibacterial drugs and the implementation of 
antibiotic stewardship programs by health care facilities of the 
Department of Defense, the Department of Veterans Affairs, and the 
Indian Health Service and shall provide technical assistance to the 
Secretary of Defense and the Secretary of Veterans Affairs, as 
appropriate and upon request.
    ``(g) Report on Antibacterial Resistance in Humans and Use of 
Antibacterial Drugs.--Not later than 1 year after the date of enactment 
of this subsection, and annually thereafter, the Secretary shall 
prepare and make publically available data and information concerning--
            ``(1) aggregate national and regional trends of bacterial 
        resistance in humans to antibacterial drugs, including those 
        approved under section 506(g) of the Federal Food, Drug, and 
        Cosmetic Act;
            ``(2) antibacterial stewardship, which may include 
        summaries of State efforts to address bacterial resistance in 
        humans to antibacterial drugs and antibacterial stewardship; 
        and
            ``(3) coordination between the Director of the Centers for 
        Disease Control and Prevention and the Commissioner of Food and 
        Drugs with respect to the monitoring of--
                    ``(A) any applicable resistance under paragraph 
                (1); and
                    ``(B) drugs approved under section 506(g) of the 
                Federal Food, Drug, and Cosmetic Act.
    ``(h) Information Related to Antibiotic Stewardship Programs.--The 
Secretary shall, as appropriate, disseminate guidance, educational 
materials, or other appropriate materials related to the development 
and implementation of evidence-based antibiotic stewardship programs or 
practices at health care facilities, such as nursing homes and other 
long-term care facilities, ambulatory surgical centers, dialysis 
centers, and community and rural hospitals.
    ``(i) Supporting State-based Activities to Combat Antibacterial 
Resistance.--The Secretary shall continue to work with State and local 
public health departments on statewide or regional programs related to 
antibacterial resistance. Such efforts may include activities to 
related to--
            ``(1) identifying patterns of bacterial resistance in 
        humans to antibacterial drugs;
            ``(2) preventing the spread of bacterial infections that 
        are resistant to antibacterial drugs; and
            ``(3) promoting antibiotic stewardship.
    ``(j) Antibacterial Resistance and Stewardship Activities.--
            ``(1) In general.--For the purposes of supporting 
        stewardship activities, examining changes in bacterial 
        resistance, and evaluating the effectiveness of section 506(g) 
        of the Federal Food, Drug, and Cosmetic Act, the Secretary 
        shall--
                    ``(A) provide a mechanism for facilities to report 
                data related to their antimicrobial stewardship 
                activities (including analyzing the outcomes of such 
                activities); and
                    ``(B) evaluate--
                            ``(i) antimicrobial resistance data using a 
                        standardized approach; and
                            ``(ii) trends in the utilization of drugs 
                        approved under such section 506(g) with respect 
                        to patient populations.
            ``(2) Use of systems.--The Secretary shall use available 
        systems, including the National Healthcare Safety Network or 
        other systems identified by the Secretary, to fulfill the 
        requirements or conduct activities under this section.
            ``(3) Availability of data.--The Secretary shall make the 
        data collected pursuant to this subsection public. Nothing in 
        this subsection shall be construed as authorizing the Secretary 
        to disclose any information that is a trade secret or 
        confidential information subject to section 552(b)(4) of title 
        5, United States Code, or section 1905 of title 18, United 
        States Code.''.

SEC. 3. LIMITED POPULATION PATHWAY FOR ANTIBACTERIAL DRUGS.

    Section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
356) is amended--
            (1) by transferring subsection (e) so that it appears 
        before subsection (f); and
            (2) by adding at the end the following:
    ``(g) Limited Population Pathway for Antibacterial Drugs.--
            ``(1) In general.--The Secretary may approve an 
        antibacterial drug, alone or in combination with one or more 
        other drugs, as a limited population drug pursuant to this 
        subsection only if--
                    ``(A) the drug is intended to treat a serious or 
                life-threatening infection in a limited population of 
                patients with unmet needs;
                    ``(B) the standards for approval under section 
                505(c) and (d), or the standards for licensure under 
                section 351 of the Public Health Service Act, as 
                applicable, are met; and
                    ``(C) the Secretary receives a written request from 
                the sponsor to approve the drug as a limited population 
                drug pursuant to this subsection.
            ``(2) Benefit-risk consideration.--The Secretary's 
        determination of safety and effectiveness of a limited 
        population antibacterial drug shall reflect the benefit-risk 
        profile of the drug in the intended limited population, taking 
        into account the severity, rarity, or prevalence of the 
        infection the drug is intended to treat and the availability or 
        lack of alternative treatment in such limited population. Such 
        drug may be approved under this subsection notwithstanding a 
        lack of evidence to fully establish a favorable benefit-risk 
        profile in a population that is broader than the intended 
        limited population.
            ``(3) Additional requirements.--A drug approved under this 
        subsection shall be subject to the requirements of this 
        paragraph, in addition to any other applicable requirements of 
        this Act:
                    ``(A) Labeling.--To indicate that the safety and 
                effectiveness of a drug approved under this subsection 
                has been demonstrated only with respect to a limited 
                population--
                            ``(i) all labeling and advertising of an 
                        antibacterial drug approved under this 
                        subsection shall contain the statement `Limited 
                        Population' in a prominent manner and adjacent 
                        to, and not more prominent than--
                                    ``(I) the proprietary name of such 
                                drug, if any; or
                                    ``(II) if there is no proprietary 
                                name, the established name of the drug, 
                                if any, as defined in section 
                                503(e)(3), or for drugs which are 
                                biological products, the proper name, 
                                as defined by regulation; and
                            ``(ii) the prescribing information for such 
                        antibacterial drug required by section 201.57 
                        of title 21, Code of Federal Regulations (or 
                        any successor regulation) shall also include 
                        the following statement: `This drug is 
                        indicated for use in a limited and specific 
                        population of patients.'.
                    ``(B) Promotional material.--The sponsor of an 
                antibacterial drug subject to this subsection shall 
                submit to the Secretary copies of all promotional 
                materials related to such drug at least 30 calendar 
                days prior to dissemination of the materials.
            ``(4) Other programs.--A sponsor of a drug that seeks 
        approval of a drug under this subsection for antibacterial 
        drugs may also seek designation or approval, as applicable, of 
        such drug under other applicable sections or subsections of 
        this Act of the Public Health Service Act.
            ``(5) Guidance.--Not later than 18 months after the date of 
        enactment of the Promise for Antibiotics and Therapeutics for 
        Health Act, the Secretary shall issue draft guidance describing 
        criteria, processes, and other general considerations for 
        demonstrating the safety and effectiveness of limited 
        population antibacterial drugs. The Secretary shall publish 
        final guidance within 18 months of the close of the public 
        comment period on such draft guidance. The Secretary may 
        approve antibacterial drugs under this subsection prior to 
        issuing guidance under this paragraph.
            ``(6) Advice.--The Secretary shall provide prompt advice to 
        the sponsor of a drug for which the sponsor seeks approval 
        under this subsection for antibacterial drugs to enable the 
        sponsor to plan a development program to obtain the necessary 
        data for approval of such drug under this subsection for 
        antibacterial drugs and to conduct any additional studies that 
        would be required to gain approval of such drug for use in a 
        broader population.
            ``(7) Termination of limitations.--If, after approval of a 
        drug under this subsection, the Secretary approves a broader 
        indication for such drug for which the sponsor applies under 
        section 505(b) or section 351(a) of the Public Health Service 
        Act, the Secretary may remove any postmarketing conditions, 
        including requirements with respect to labeling and review of 
        promotional materials under paragraph (3), applicable to the 
        approval of the drug under this subsection.
            ``(8) Rules of construction.--Nothing in this subsection 
        shall be construed to alter the authority of the Secretary to 
        approve drugs pursuant to this Act and section 351 of the 
        Public Health Service Act, including the standards of evidence, 
        and applicable conditions, for approval under such Acts, the 
        standards of approval of a drug under this Act or the Public 
        Health Service Act, or to alter the authority of the Secretary 
        to monitor drugs pursuant to this Act or the Public Health 
        Service Act.
            ``(9) Reporting and accountability.--
                    ``(A) Biannual reporting.--The Secretary shall 
                report to Congress not less often than once every 2 
                years on the number of requests for approval, and the 
                number of approvals, of an antibacterial drug under 
                this subsection.
                    ``(B) Gao report.--Not later than December 2021, 
                the Comptroller General of the United States shall 
                report on the coordination of activities required under 
                section 319E of the Public Health Service Act, a review 
                of such activities, and the extent to which the use of 
                the pathway established under this subsection has 
                streamlined premarket approval for antibacterial drugs 
                for limited populations, if such pathway has functioned 
                as intended, if such pathway has helped provide for 
                safe and effective treatment for patients, if such 
                premarket approval would be appropriate for other 
                categories of drugs, and if the authorities under this 
                subsection have affected antibiotic resistance.''.

SEC. 4. PRESCRIBING AUTHORITY.

    Nothing in this Act, or an amendment made by this Act, shall be 
construed to restrict the prescribing of antibacterial drugs or other 
products, including drugs approved under section 506(g) of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 356(g)), by health care 
professionals, or to limit the practice of health care.
                                                       Calendar No. 425

114th CONGRESS

  2d Session

                                 S. 185

_______________________________________________________________________

                                 A BILL

    To create a limited population pathway for approval of certain 
                          antibacterial drugs.

_______________________________________________________________________

                             April 18, 2016

                       Reported with an amendment
```