[United States Statutes at Large, Volume 130, 114th Congress, 2nd Session]
[From the U.S. Government Publishing Office, www.gpo.gov]


Public Law 114-229
114th Congress

An Act


 
To extend the pediatric priority review voucher program. <>

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 Hope Act of 2016''.

SEC. 2. REAUTHORIZATION OF PROGRAM FOR PRIORITY REVIEW TO ENCOURAGE
TREATMENTS FOR RARE PEDIATRIC DISEASES.

(a) In General.--Section 529 of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360ff) is amended--
(1) in subsection (a)--
(A) in paragraph (3), by amending subparagraph (A)
to read as follows:
``(A) The disease is a serious or life-threatening
disease in which the serious or life-threatening
manifestations primarily affect individuals aged from
birth to 18 years, including age groups often called
neonates, infants, children, and adolescents.''; and
(B) in paragraph (4)(F), by striking ``Prescription
Drug User Fee Amendments of 2012'' and inserting
``Advancing Hope Act of 2016'';
(2) in subsection (b)--
(A) by striking paragraph (4) and inserting the
following:
``(4) Notification.--
``(A) Sponsor of a rare pediatric disease product.--
``(i)  <> In general.--Beginning on the date that
is 90 days after the date of enactment of the
Advancing Hope Act of 2016, the sponsor of a rare
pediatric disease product application that intends
to request a priority review voucher under this
section shall notify the Secretary of such intent
upon submission of the rare pediatric disease
product application that is the basis of the
request for a priority review voucher.
``(ii) Applications submitted but not yet
approved.--The sponsor of a rare pediatric disease
product application that was submitted and that
has not been approved as of the date of enactment
of the Advancing Hope Act of 2016 shall be
considered eligible for a priority review voucher,
if--
``(I) such sponsor has submitted
such rare pediatric disease product
application--

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``(aa) <> on or after the date
that is 90 days after the date
of enactment of the Prescription
Drug User Fee Amendments of
2012; and
``(bb) on or before the date
of enactment of the Advancing
Hope Act of 2016; and
``(II) such application otherwise
meets the criteria for a priority review
voucher under this section.
``(B) Sponsor of a drug application using a priority
review voucher.--
``(i) <> In general.--The
sponsor of a human drug application shall notify
the Secretary not later than 90 days prior to
submission of the human drug application that is
the subject of a priority review voucher of an
intent to submit the human drug application,
including the date on which the sponsor intends to
submit the application. Such notification shall be
a legally binding commitment to pay the user fee
to be assessed in accordance with this section.
``(ii) Transfer after notice.--The sponsor of
a human drug application that provides
notification of the intent of such sponsor to use
the voucher for the human drug application under
clause (i) may transfer the voucher after such
notification is provided, if such sponsor has not
yet submitted the human drug application described
in the notification.''; and
(B) by striking paragraph (5) and inserting the
following:
``(5) Termination of authority.--The Secretary may not award
any priority review vouchers under paragraph (1) after December
31, 2016.''; and
(3) in subsection (g), by inserting before the period ``,
except that no sponsor of a rare pediatric disease product
application may receive more than one priority review voucher
issued under any section of this Act with respect to the drug
for which the application is made.''

(b) <> Rule of Construction.--Nothing in
this Act, or the amendments made by this Act, shall be construed to
affect the validity of a priority review voucher that was issued under
section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C.
360ff) before the date of enactment of this Act.

SEC. 3. GAO REPORT.

(a) <> Study.--The Comptroller General
of the United States shall conduct a study on the effectiveness of
awarding priority review vouchers under section 529 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360ff) in providing incentives for the
development of drugs that treat or prevent rare pediatric diseases (as
defined in subsection (a)(3) of such section) that would not otherwise
have been developed. <> In conducting such study,
the Comptroller General shall examine the following:
(1) The indications for which each drug for which a priority
review voucher was awarded under such section 529 was approved
under section 505(b)(1) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 355(b)(1)) or section 351(a) of the Public Health
Service Act (42 U.S.C. 262(a)).

[[Page 945]]

(2) Whether the priority review voucher impacted sponsors'
decisions to invest in developing a drug to treat or prevent a
rare pediatric disease.
(3) <> An analysis of the drugs for which
such priority review vouchers were used, which shall include--
(A) the indications for which such drugs were
approved under section 505(b)(1) of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 355(b)(1)) or section
351(a) of the Public Health Service Act (42 U.S.C.
262(a));
(B) whether unmet medical needs were addressed
through the approval of such drugs, including, for each
such drug--
(i) if an alternative therapy was previously
available to treat the indication; and
(ii) if the drug provided a benefit or
advantage over another available therapy;
(C) the number of patients potentially treated by
such drugs;
(D) the value of the priority review voucher if
transferred; and
(E) the length of time between the date on which a
priority review voucher was awarded and the date on
which it was used.
(4) With respect to the priority review voucher program
under section 529 of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360ff)--
(A) the resources used by the Food and Drug
Administration in implementing such program, including
the effect of such program on the Food and Drug
Administration's review of drugs for which a priority
review voucher was not awarded or used;
(B) the impact of the program on the public health
as a result of the review and approval of drugs that
received a priority review voucher and products that
were the subject of a redeemed priority review voucher;
and
(C) alternative approaches to improving such program
so that the program is appropriately targeted toward
providing incentives for the development of clinically
important drugs that--
(i) prevent or treat rare pediatric diseases;
and
(ii) would likely not otherwise have been
developed to prevent or treat such diseases.

(b) Report.--Not later than January 31, 2022, the Comptroller
General of the United States shall submit to the Committee on Health,
Education, Labor, and Pensions of the Senate and the


[[Page 946]]

Committee on Energy and Commerce of the House of Representatives a
report containing the results of the study of conducted under subsection
(a).

Approved September 30, 2016.

LEGISLATIVE HISTORY--S. 1878:
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CONGRESSIONAL RECORD, Vol. 162 (2016):
Sept. 22, considered and passed Senate.
Sept. 27, considered and passed House.