[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