[Congressional Record Volume 162, Number 144 (Thursday, September 22, 2016)]
[Senate]
[Pages S6062-S6064]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                       ADVANCING HOPE ACT OF 2015

  Mr. COATS. Mr. President, I ask unanimous consent that the Senate 
proceed to the immediate consideration of Calendar No. 415, S. 1878.
  The PRESIDING OFFICER. The clerk will report the bill by title.
  The senior assistant legislative clerk read as follows:

       A bill (S. 1878) to extend the pediatric priority review 
     voucher program.

  There being no objection, the Senate proceeded to consider the bill, 
which had been reported from the Committee on Health, Education, Labor, 
and Pensions, with an amendment to strike all after the enacting clause 
and insert in lieu thereof the following:

     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.

[[Page S6063]]

       ``(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).

  Mr. COATS. Mr. President, I ask unanimous consent that the Sanders 
amendment, which is at the desk, be agreed to; the committee-reported 
substitute amendment, as amended, be agreed to; that the bill, as 
amended, be read a third time and passed; and that the motion to 
reconsider be considered made and laid upon the table.
  The PRESIDING OFFICER. Without objection, it is so ordered.
  The amendment (No. 5090) was agreed to, as follows:

                     (Purpose: To improve the bill)

       On page 7, strike lines 7 through 17 and insert the 
     following:
       ``(5) Termination of authority.--The Secretary may not 
     award any priority review vouchers under paragraph (1) after 
     December 31, 2016.''; and

  The committee-reported amendment in the nature of a substitute, as 
amended, was agreed to.
  The bill (S. 1878), as amended, was ordered to be engrossed for a 
third reading, was read the third time, and passed, as follows:

                                S. 1878

       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

[[Page S6064]]

     (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).

                          ____________________