Amends the Federal Food, Drug, and Cosmetic Act to direct the Secretary of Health and Human Services (HHS), at the request of the sponsor of a drug, to expedite the drug's development and review if: (1) it is intended, either alone or in combination, to treat a serious life-threatening disease or condition; and (2) preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.
Authorizes the drug's sponsor to request the Secretary to designate the drug as a breakthrough therapy. Requires the Secretary, within 60 days of such request, to determine whether the drug meets such criteria and, if so, make such designation, followed by appropriate actions to expedite its development and review for approval.
Requires an annual report from the Secretary to Congress on drugs for which breakthrough designations were requested and approved.
Directs the Secretary to issue guidance on implementing requirements with respect to breakthrough therapies and to amend promulgated regulations.
Requires the Comptroller General to assess the manner by which the Food and Drug Administration (FDA) has applied the processes established by this Act and their impact on the development and timely availability of innovative treatments for patients affected by serious or life-threatening conditions.
[Congressional Bills 112th Congress]
[From the U.S. Government Publishing Office]
[H.R. 5334 Introduced in House (IH)]
112th CONGRESS
2d Session
H. R. 5334
To amend chapter V of the Federal Food, Drug, and Cosmetic Act to
expedite the development and review of breakthrough therapies.
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
May 7, 2012
Mr. Bilbray (for himself and Ms. DeGette) introduced the following
bill; which was referred to the Committee on Energy and Commerce
_______________________________________________________________________
A BILL
To amend chapter V of the Federal Food, Drug, and Cosmetic Act to
expedite the development and review of breakthrough therapies.
Be it enacted by the Senate and House of Representatives of the
United States of America in Congress assembled,
SECTION 1. BREAKTHROUGH THERAPIES.
(a) In General.--Section 506 (21 U.S.C. 356) is amended--
(1) by redesignating subsection (d) as subsection (f);
(2) by redesignating subsections (a) through (c) as
subsections (b) through (d), respectively;
(3) by inserting before subsection (b), as so redesignated,
the following:
``(a) Designation of a Drug as a Breakthrough Therapy.--
``(1) In general.--The Secretary shall, at the request of
the sponsor of a drug, expedite the development and review of
such drug if the drug is intended, alone or in combination with
1 or more other drugs, to treat a serious or life-threatening
disease or condition and preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement
over existing therapies on 1 or more clinically significant
endpoints, such as substantial treatment effects observed early
in clinical development. (In this section, such a drug is
referred to as a `breakthrough therapy'.)
``(2) Request for designation.--The sponsor of a drug may
request the Secretary to designate the drug as a breakthrough
therapy. A request for the designation may be made concurrently
with, or at any time after, the submission of an application
for the investigation of the drug under section 505(i) or
section 351(a)(3) of the Public Health Service Act.
``(3) Designation.--
``(A) In general.--Not later than 60 calendar days
after the receipt of a request under paragraph (2), the
Secretary shall determine whether the drug that is the
subject of the request meets the criteria described in
paragraph (1). If the Secretary finds that the drug
meets the criteria, the Secretary shall designate the
drug as a breakthrough therapy and shall take such
actions as are appropriate to expedite the development
and review of the application for approval of such
drug.
``(B) Actions.--The actions to expedite the
development and review of an application under
subparagraph (A) may include, as appropriate--
``(i) holding meetings with the sponsor and
the review team throughout the development of
the drug;
``(ii) providing timely advice to, and
interactive communication with, the sponsor
regarding the development of the drug to ensure
that the development program to gather the non-
clinical and clinical data necessary for
approval is as efficient as practicable;
``(iii) involving senior managers and
experienced review staff, as appropriate, in a
collaborative, cross-disciplinary review;
``(iv) assigning a cross-disciplinary
project lead for the Food and Drug
Administration review team to facilitate an
efficient review of the development program and
to serve as a scientific liaison between the
review team and the sponsor; and
``(v) taking steps to ensure that the
design of the clinical trials is as efficient
as practicable, when scientifically
appropriate, such as by minimizing the number
of patients exposed to a potentially less
efficacious treatment.'';
(4) in subsection (f)(1), as so redesignated, by striking
``applicable to accelerated approval'' and inserting
``applicable to breakthrough therapies, accelerated approval,
and''; and
(5) by adding at the end the following:
``(g) Report.--Beginning in fiscal year 2013, the Secretary shall
annually prepare and submit to the Committee on Health, Education,
Labor, and Pensions of the Senate and the Committee on Energy and
Commerce of the House of Representatives, and make publicly available,
with respect to this section for the previous fiscal year--
``(1) the number of drugs for which a sponsor requested
designation as a breakthrough therapy; and
``(2) the number of products designated as a breakthrough
therapy.''.
(b) Guidance; Amended Regulations.--
(1) In general.--
(A) Guidance.--Not later than 18 months after the
date of enactment of this Act, the Secretary of Health
and Human Services (referred to in this section as the
``Secretary'') shall issue draft guidance on
implementing the requirements with respect to
breakthrough therapies, as set forth in section 506(a)
of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
356(a)), as amended by this section. The Secretary
shall issue final guidance not later than 1 year after
the close of the comment period for the draft guidance.
(B) Amended regulations.--
(i) In general.--If the Secretary
determines that it is necessary to amend the
regulations under title 21, Code of Federal
Regulations in order to implement the
amendments made by this section to section
506(a) of the Federal Food, Drug, and Cosmetic
Act, the Secretary shall amend such regulations
not later than 2 years after the date of
enactment of this Act.
(ii) Procedure.--In amending regulations
under clause (i), the Secretary shall--
(I) issue a notice of proposed
rulemaking that includes the proposed
regulation;
(II) provide a period of not less
than 60 days for comments on the
proposed regulation; and
(III) publish the final regulation
not less than 30 days before the
effective date of the regulation.
(iii) Restrictions.--Notwithstanding any
other provision of law, the Secretary shall
promulgate regulations implementing the
amendments made by this section only as
described in clause (ii).
(2) Requirements.--Guidance issued under this section
shall--
(A) specify the process and criteria by which the
Secretary makes a designation under section 506(a)(3)
of the Federal Food, Drug, and Cosmetic Act; and
(B) specify the actions the Secretary shall take to
expedite the development and review of a breakthrough
therapy pursuant to such designation under such section
506(a)(3), including updating good review management
practices to reflect breakthrough therapies.
(c) Independent Review.--Not later than 3 years after the date of
enactment of this Act, the Comptroller General of the United States, in
consultation with appropriate experts, shall assess the manner by which
the Food and Drug Administration has applied the processes described in
section 506(a) of the Federal Food, Drug, and Cosmetic Act, as amended
by this section, and the impact of such processes on the development
and timely availability of innovative treatments for patients affected
by serious or life-threatening conditions. Such assessment shall be
made publicly available upon completion.
(d) Conforming Amendments.--Section 506B(e) (21 U.S.C. 356b) is
amended by striking ``section 506(b)(2)(A)'' each place such term
appears and inserting ``section 506(c)(2)(A)''.
<all>
Introduced in House
Introduced in House
Referred to the House Committee on Energy and Commerce.
Referred to the Subcommittee on Health.
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