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[Congressional Bills 114th Congress]
[From the U.S. Government Publishing Office]
[S. 2030 Reported in Senate (RS)]

<DOC>

                                                       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.

    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 ``Advancing Targeted 
Therapies for Rare Diseases Act of 2015''.</DELETED>

<DELETED>SEC. 2. TARGETED DRUGS FOR RARE DISEASES.</DELETED>

<DELETED>    Title V of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 351 et seq.) is amended by inserting after section 506F the 
following:</DELETED>

<DELETED>``SEC. 506G. TARGETED DRUGS FOR RARE DISEASES.</DELETED>

<DELETED>    ``(a) Purpose.--The purpose of this section, through the 
approach provided for in subsection (b), is to--</DELETED>
        <DELETED>    ``(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</DELETED>
        <DELETED>    ``(2) maximize the use of scientific tools or 
        methods, including surrogate endpoints and other biomarkers for 
        such purposes.</DELETED>
<DELETED>    ``(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--</DELETED>
        <DELETED>    ``(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</DELETED>
        <DELETED>    ``(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,</DELETED>
<DELETED>for a drug that incorporates or utilizes the same or similar 
genetically targeted technology, or the same variant protein targeted 
technology, as the drug or drugs that are the subject of an application 
or applications described in paragraph (2).</DELETED>
<DELETED>    ``(c) Definitions.--For purposes of this section--
</DELETED>
        <DELETED>    ``(1) the term `genetically targeted drug' means a 
        drug which--</DELETED>
                <DELETED>    ``(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;</DELETED>
                <DELETED>    ``(B) incorporates or utilizes a 
                genetically targeted technology or a variant protein 
                targeted technology; and</DELETED>
                <DELETED>    ``(C) may result in the modulation 
                (including suppression, up-regulation, or activation) 
                of the function of a gene or its associated gene 
                product;</DELETED>
        <DELETED>    ``(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</DELETED>
        <DELETED>    ``(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.</DELETED>
<DELETED>    ``(d) Rule of Construction.--Nothing in this section shall 
be construed to--</DELETED>
        <DELETED>    ``(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</DELETED>
        <DELETED>    ``(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.''.</DELETED>

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Advancing Targeted Therapies for 
Rare Diseases Act of 2016''.

SEC. 2. TARGETED DRUGS FOR RARE DISEASES.

    Subchapter B of chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 360aa et seq.) is amended by inserting after section 529 
the following:

``SEC. 529A. 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 and variant protein targeted drugs 
        to address an unmet medical need in one or more patient 
        subgroups, including subgroups of patients with different 
        mutations of a gene, 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(a) of the Public 
Health Service Act, allow the sponsor of an application under section 
505(b)(1) of this Act or section 351(a) of the Public Health Service 
Act for a genetically targeted drug or a variant protein 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 previously approved applications that 
        were submitted under section 505(b)(1) of this this Act or 
        section 351(a) of the Public Health Service Act,
for a drug that incorporates or utilizes the same or similar 
genetically targeted technology as the drug or drugs that are the 
subject of an application or applications described in paragraph (2) or 
for a variant protein targeted drug that is the same or incorporates or 
utilizes the same variant protein targeted drug, as the drug or drugs 
that are the subject of an application or applications described in 
paragraph (2).
    ``(c) Definitions.--For purposes of this section--
            ``(1) the term `genetically targeted drug' means a drug 
        that--
                    ``(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) may result in the modulation (including 
                suppression, up-regulation, or activation) of the 
                function of a gene or its associated gene product; and
                    ``(C) incorporates or utilizes a genetically 
                targeted technology;
            ``(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 patient subgroups, including subgroups of 
        patients with different mutations of a gene, with the same 
        disease or condition, including a disease or condition due to 
        other variants in the same gene; and
            ``(3) the term `variant protein targeted drug' means a drug 
        that--
                    ``(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) modulates the function of a product of a 
                mutated gene where such mutation is responsible in 
                whole or in part for a given disease or condition; and
                    ``(C) is intended to treat one or more patient 
                subgroups, including subgroups of patients with 
                different mutations of a gene, with the same disease or 
                condition.
    ``(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 
        this section), including the standards of evidence, and 
        applicable conditions, for approval under such applicable Act; 
        or
            ``(2) confer any rights, beyond those authorized under this 
        Act or the Public Health Service Act prior to enactment of 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.''.
                                                       Calendar No. 416

114th CONGRESS

  2d Session

                                S. 2030

_______________________________________________________________________

                                 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.

_______________________________________________________________________

                             April 5, 2016

                       Reported with an amendment
```