[Congressional Bills 114th Congress]
[From the U.S. Government Publishing Office]
[S. 1878 Reported in Senate (RS)]

<DOC>





                                                       Calendar No. 415
114th CONGRESS
  2d Session
                                S. 1878

        To extend the pediatric priority review voucher program.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                             July 28, 2015

     Mr. Casey (for himself, Mr. Isakson, Mr. Brown, and Mr. Kirk) 
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 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,

<DELETED>SECTION 1. SHORT TITLE.</DELETED>

<DELETED>    This Act may be cited as the ``Advancing Hope Act of 
2015''.</DELETED>

<DELETED>SEC. 2. REAUTHORIZATION OF PROGRAM FOR PRIORITY REVIEW TO 
              ENCOURAGE TREATMENTS FOR RARE PEDIATRIC 
              DISEASES.</DELETED>

<DELETED>    Section 529 of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 360ff) is amended--</DELETED>
        <DELETED>    (1) in subsection (a)--</DELETED>
                <DELETED>    (A) by amending paragraph (3) to read as 
                follows:</DELETED>
        <DELETED>    ``(3) Rare pediatric disease.--The term `rare 
        pediatric disease' means any of the following:</DELETED>
                <DELETED>    ``(A) A disease that meets each of the 
                following criteria:</DELETED>
                        <DELETED>    ``(i) The disease primarily 
                        affects individuals aged from birth to 18 
                        years, including age groups often called 
                        neonates, infants, children, and 
                        adolescents.</DELETED>
                        <DELETED>    ``(ii) The disease is a rare 
                        disease or condition, within the meaning of 
                        section 526.</DELETED>
                <DELETED>    ``(B) Any form of sickle cell 
                disease.</DELETED>
                <DELETED>    ``(C) Any pediatric cancers.''; 
                and</DELETED>
                <DELETED>    (B) in paragraph (4)--</DELETED>
                        <DELETED>    (i) in subparagraph (E), by 
                        striking ``and'' at the end;</DELETED>
                        <DELETED>    (ii) in subparagraph (F), by 
                        striking the period at the end and inserting 
                        ``; and''; and</DELETED>
                        <DELETED>    (iii) by adding at the end the 
                        following:</DELETED>
                <DELETED>    ``(G) is for a drug or biological product 
                for which a priority review voucher has not been issued 
                under section 524 (relating to tropical disease 
                products).'';</DELETED>
        <DELETED>    (2) in subsection (b), by striking paragraph (5); 
        and</DELETED>
        <DELETED>    (3) in subsection (d)(2), in the second sentence, 
        by striking the period and inserting ``, but the sponsor of a 
        drug that intends to request a priority review voucher under 
        this section shall notify the Secretary of such intent upon 
        submission of an application under section 505 or section 351 
        of the Public Health Service Act that is the basis of such 
        request.''.</DELETED>

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 
        September 30, 2022, unless the rare pediatric disease product 
        application--
                    ``(A) is for a drug that, not later than September 
                30, 2022, is designated under subsection (d) as a drug 
                for a rare pediatric disease; and
                    ``(B) is, not later than September 30, 2027, 
                approved under section 505(b)(1) of this Act or section 
                351(a) of the Public Health Service Act.''; 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).
                                                       Calendar No. 415

114th CONGRESS

  2d Session

                                S. 1878

_______________________________________________________________________

                                 A BILL

        To extend the pediatric priority review voucher program.

_______________________________________________________________________

                             April 5, 2016

                       Reported with an amendment