[114th Congress Public Law 229]
[From the U.S. Government Publishing Office]



[[Page 130 STAT. 943]]

Public Law 114-229
114th Congress

                                 An Act


 
 To extend the pediatric priority review voucher program. <<NOTE: Sept. 
                        30, 2016 -  [S. 1878]>> 

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled, <<NOTE: Advancing Hope 
Act of 2016.>> 

SECTION 1. <<NOTE: 21 USC 301 note.>>  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)  <<NOTE: Effective date. Time 
                      period.>> 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--

__________
---------------------------------------------------------------------------

     * Editorial note: The original slip law was printed with the wrong 
approval date of Sept. 29, 2016. The correct approval date is Sept. 30, 
2016.

---------------------------------------------------------------------------

[[Page 130 STAT. 944]]

                                            ``(aa) <<NOTE: Time 
                                        period.>> 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) <<NOTE: Deadline.>> 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) <<NOTE: 21 USC 360ff note.>> 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) <<NOTE: Drugs and drug abuse.>> 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. <<NOTE: Examination.>> 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 130 STAT. 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) <<NOTE: Analysis.>> 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 130 STAT. 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:
---------------------------------------------------------------------------

CONGRESSIONAL RECORD, Vol. 162 (2016):
            Sept. 22, considered and passed Senate.
            Sept. 27, considered and passed House.

                                  <all>