```

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

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.

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

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Advancing Targeted Therapies for 
Rare Diseases Act of 2015''.

SEC. 2. TARGETED DRUGS FOR RARE DISEASES.

    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:

``SEC. 506G. 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 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
            ``(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 of the Public 
Health Service Act, allow the sponsor of a genetically 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 applications previously approved under 
        this Act or section 351 of the Public Health Service Act,
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).
    ``(c) Definitions.--For purposes of this section--
            ``(1) the term `genetically targeted drug' means a drug 
        which--
                    ``(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) incorporates or utilizes a genetically 
                targeted technology or a variant protein targeted 
                technology; and
                    ``(C) may result in the modulation (including 
                suppression, up-regulation, or activation) of the 
                function of a gene or its associated gene product;
            ``(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
            ``(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.
    ``(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 
        the Advancing Targeted Therapies for Rare Diseases Act of 
        2015), including the standards of evidence, and applicable 
        conditions, for approval under such Act; or
            ``(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.''.
                                 <all>
```