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[Congressional Bills 114th Congress]
[From the U.S. Government Publishing Office]
[S. 1878 Enrolled Bill (ENR)]

        S.1878

                     One Hundred Fourteenth Congress

                                 of the

                        United States of America

                          AT THE SECOND SESSION

           Begun and held at the City of Washington on Monday,
           the fourth day of January, two thousand and sixteen

                                 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--

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

                               Speaker of the House of Representatives.

                            Vice President of the United States and    
                                               President of the Senate.
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