[House Report 116-551]
[From the U.S. Government Publishing Office]


116th Congress    }                                    {        Report
                        HOUSE OF REPRESENTATIVES
 2d Session       }                                    {       116-551

======================================================================



 
                   CREATING HOPE REAUTHORIZATION ACT

                                _______
                                

 September 29, 2020.--Committed to the Committee of the Whole House on 
            the State of the Union and ordered to be printed

                                _______
                                

 Mr. Pallone, from the Committee on Energy and Commerce, submitted the 
                               following

                              R E P O R T

                        [To accompany H.R. 4439]

    The Committee on Energy and Commerce, to whom was referred 
the bill (H.R. 4439) to amend the Federal Food, Drug, and 
Cosmetic Act to make permanent the authority of the Secretary 
of Health and Human Services to issue priority review vouchers 
to encourage treatments for rare pediatric diseases, having 
considered the same, reports favorably thereon with amendments 
and recommends that the bill as amended do pass.

                                CONTENTS

                                                                   Page
  I. Purpose and Summary..............................................2
 II. Background and Need for the Legislation..........................2
III. Committee Hearings...............................................3
 IV. Committee Consideration..........................................4
  V. Committee Votes..................................................4
 VI. Oversight Findings...............................................4
VII. New Budget Authority, Entitlement Authority, and Tax Expenditures4
VIII.Federal Mandates Statement.......................................5

 IX. Statement of General Performance Goals and Objectives............5
  X. Duplication of Federal Programs..................................5
 XI. Committee Cost Estimate..........................................5
XII. Earmarks, Limited Tax Benefits, and Limited Tariff Benefits......5
XIII.Advisory Committee Statement.....................................5

XIV. Applicability to Legislative Branch..............................5
 XV. Section-by-Section Analysis of the Legislation...................5
XVI. Changes in Existing Law Made by the Bill, as Reported............6

    The amendments are as follows:
    Strike all after the enacting clause and insert the 
following:

SECTION 1. SHORT TITLE.

  This Act may be cited as the ``Creating Hope Reauthorization Act''.

SEC. 2. EXTENSION OF AUTHORITY TO ISSUE PRIORITY REVIEW VOUCHERS TO 
                    ENCOURAGE TREATMENTS FOR RARE PEDIATRIC DISEASES.

  Section 529(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360ff(b)) is amended--
          (1) by striking ``September 30, 2020'' each place it appears 
        and inserting ``September 30, 2024''; and
          (2) by striking ``September 30, 2022'' and inserting 
        ``September 30, 2026''.

    Amend the title so as to read:
    A bill to amend the Federal Food, Drug, and Cosmetic Act to 
extend the authority of the Secretary of Health and Human 
Services to issue priority review vouchers to encourage 
treatments for rare pediatric diseases.

                         I. Purpose and Summary

    H.R. 4439, the ``Creating Hope Reauthorization Act'', was 
introduced on September 20, 2019, by Representative G.K. 
Butterfield (D-NC) and referred to the Committee on Energy and 
Commerce. H.R. 4439, as reported, will extend the Food and Drug 
Administration (FDA) Pediatric Rare Disease Priority Review 
Voucher (PRV) program for four years. Under this program, 
certain manufacturers of rare pediatric disease drugs can be 
eligible for a voucher that can be used or transferred to 
obtain a priority review for a subsequent drug after the date 
of approval of the rare pediatric disease drug.

              II. Background and Need for the Legislation

    The Pediatric Rare Disease PRV program was originally 
enacted in 2012 with the intent to create an incentive for drug 
manufacturers to develop therapies for rare pediatric diseases 
that affect neonates, infants, children, and adolescents. The 
program requires the FDA to award a PRV to the sponsor of a 
rare pediatric disease product application that receives 
approval as a drug or biological product to treat a rare 
pediatric disease, defined as a serious or life-threatening 
disease in which the serious or life-threatening manifestations 
primarily affect individuals aged from birth to 18 years. The 
incentive value is premised on the ability for the holder of a 
PRV to shorten the FDA review time on another drug application 
from the standard ten months to six months, or for the holder 
of a PRV to sell the voucher to another manufacturer to be used 
on a drug application of the manufacturer's choosing.
    To date, FDA has awarded 22 PRVs for rare pediatric 
diseases.\1\ PRVs have been redeemed to accelerate review of 
applications for treatments of a variety of conditions, 
including human immunodeficiency virus (HIV) and type 2 
diabetes, and have been sold for prices that range between $65 
million and $350 million.\2\ While these facts indicate that 
PRVs carry some level of value, at least one study has shown 
that the Pediatric Rare Disease PRV program has not improved 
the rate of new pediatric drugs starting or completing clinical 
testing, suggesting that the value of PRVs is an insufficient 
incentive for drug development.\3\ Furthermore, observers have 
noted that as more PRVs have been awarded, the price of PRVs 
has reduced, suggesting that additional awards reduce the PRV 
value to developers.\4\ Additionally, FDA has said that the 
program strains agency resources and impairs the agency's 
ability to set public health priorities.\5\
---------------------------------------------------------------------------
    \1\Michael Mezher, Zachary Brennan, and Alexander Gaffney, 
Everything You Need to Know about FDA's Priority Review Vouchers, 
Regulatory Affairs Professionals Society (Feb. 25, 2020) (www.raps.org/
regulatory-focus/news-articles/2017/12/regulatory-explainer-everything-
you-need-to-know-about-fdas-priority-review-vouchers).
    \2\Id.
    \3\Thomas J. Hwang et al., Impact of the Priority Review Voucher 
Program on Drug Development for Rare Pediatric Diseases, Health Affairs 
(Feb. 2019).
    \4\House Committee on Energy and Commerce, Subcommittee on Health, 
Testimony of Aaron S. Kesselheim, M.D., J.D., M.P.H., Professor of 
Medicine, Harvard Medical School, Legislative Hearing on Improving 
Access to Care: Legislation to Reauthorize Key Public Health Programs, 
116th Cong. (July 29, 2020).
    \5\Government Accountability Office, Rare Diseases: Too Early to 
Gauge Effectiveness of FDA's Pediatric Voucher Program (Mar. 2016) 
(GAO-16-319).
---------------------------------------------------------------------------
    Proponents of H.R. 4439 argue that the program is an 
important consideration for drug manufacturers when deciding 
where to invest research and development dollars. According to 
a review of PRV programs conducted by the U.S. Government 
Accountability Office (GAO), six of seven drug manufacturers 
interviewed by the GAO indicated that the PRV was one of a 
number of factors in making drug development decisions, with 
the seventh indicating that the PRV was ``pivotal'' in its 
development of a drug.\6\ Proponents have also suggested that 
the increase in the number of drug applications receiving 
pediatric rare disease designation--from 24 in 2016 to 64 in 
2019--indicates an increased level of drug development for 
pediatric rare diseases under the program.\7\
---------------------------------------------------------------------------
    \6\Government Accountability Office, Drug Development: FDA's 
Priority Review Voucher Programs (Jan. 2020) (GAO-20-251).
    \7\House Committee on Energy and Commerce, Subcommittee on Health, 
Testimony of Nancy Goodman, M.P.P., J.D., Founder and Executive 
Director, Kids v. Cancer, Legislative Hearing on Improving Access to 
Care: Legislation to Reauthorize Key Public Health Programs, 116th 
Cong. (July 29, 2020).
---------------------------------------------------------------------------
    H.R. 4439, as introduced, would remove the sunset on the 
authority of the Secretary of the U.S. Department of Health and 
Human Services (the Secretary) to issue new pediatric rare 
disease designations and PRVs, which is set to expire on 
September 30, 2020. So as not to foreclose on the potential for 
development of additional treatments for pediatric rare 
diseases, but in recognition of concerns about effectiveness of 
the program, the Committee adopted an amendment in the nature 
of a substitute (AINS) that would extend the Pediatric Rare 
Disease PRV program for four years.

                        III. Committee Hearings

    For the purposes of section 103(i) of H. Res. 6 of the 
116th Congress, the following hearing was used to develop or 
consider H.R. 4439:
    The Subcommittee on Health held a legislative hearing on 
July 29, 2020, entitled ``Improving Access to Care: Legislation 
to Reauthorize Key Public Health Programs''' to consider H.R. 
4439, the ``Creating Hope Reauthorization Act'' and four other 
bills. The Subcommittee received testimony from the following 
witnesses:
           Robert Boyd, M.C.R.P., M. Div., President, 
        School-Based Health Alliance;
           Linda Goler Blount, M.P.H., President and 
        CEO, Black Women's Health Imperative;
           Nancy Goodman, M.P.P., J.D., Founder and 
        Executive Director, Kids v. Cancer;
           Aaron Seth Kesselheim, M.D., J.D., M.P.H., 
        Professor of Medicine, Harvard Medical School;
           Brian Lindbergh, Chief Legal Officer and 
        General Counsel, National Bone Marrow Donor Program; 
        and
           Travis T. Tygart, CEO, U.S. Anti-doping 
        Agency.

                      IV. Committee Consideration

    Representative Butterfield (D-NC) introduced H.R. 4439, the 
``Creating Hope Reauthorization Act'', on September 20, 2019, 
and the bill was referred to the Committee on Energy and 
Commerce. The bill was then referred to the Subcommittee on 
Health on September 23, 2019. A legislative hearing was held on 
the bill on July 29, 2020.
    On September 9, 2020, H.R. 4439 was discharged from further 
consideration by the Subcommittee on Health as the bill was 
called up for markup by the full Committee. The Committee on 
Energy and Commerce met in virtual open markup session, 
pursuant to notice, to consider the bill H.R. 4439. During 
consideration of the bill, an amendment in the nature of a 
substitute offered by Mr. Pallone of New Jersey was agreed to 
by a voice vote. Upon conclusion of consideration of the bill, 
the full Committee agreed to a motion on final passage by Mr. 
Pallone, Chairman of the committee, to order H.R. 4439 reported 
favorably to the House, amended, by a voice vote, a quorum 
being present.

                           V. Committee Votes

    Clause 3(b) of rule XIII of the Rules of the House of 
Representatives requires the Committee to list each record vote 
on the motion to report legislation and amendments thereto. The 
Committee advises that there were no record votes taken on H.R. 
4439, including the motion for final passage of the bill.

                         VI. Oversight Findings

    Pursuant to clause 3(c)(1) of rule XIII and clause 2(b)(1) 
of rule X of the Rules of the House of Representatives, the 
oversight findings and recommendations of the Committee are 
reflected in the descriptive portion of the report.

 VII. New Budget Authority, Entitlement Authority, and Tax Expenditures

    Pursuant to 3(c)(2) of rule XIII of the Rules of the House 
of Representatives, the Committee adopts as its own the 
estimate of new budget authority, entitlement authority, or tax 
expenditures or revenues contained in the cost estimate 
prepared by the Director of the Congressional Budget Office 
pursuant to section 402 of the Congressional Budget Act of 
1974.
    The Committee has requested but not received from the 
Director of the Congressional Budget Office a statement as to 
whether this bill contains any new budget authority, spending 
authority, credit authority, or an increase or decrease in 
revenues or tax expenditures.

                    VIII. Federal Mandates Statement

    The Committee adopts as its own the estimate of Federal 
mandates prepared by the Director of the Congressional Budget 
Office pursuant to section 423 of the Unfunded Mandates Reform 
Act.

       IX. Statement of General Performance Goals and Objectives

    Pursuant to clause 3(c)(4) of rule XIII, the general 
performance goal or objective of this legislation is to extend 
the authority of the Secretary of Health and Human Services to 
issue PRVs to encourage the development of treatments for rare 
pediatric diseases.

                   X. Duplication of Federal Programs

    Pursuant to clause 3(c)(5) of rule XIII, no provision of 
H.R. 4439 is known to be duplicative of another Federal 
program, including any program that was included in a report to 
Congress pursuant to section 21 of Public Law 111-139 or the 
most recent Catalog of Federal Domestic Assistance.

                      XI. Committee Cost Estimate

    Pursuant to clause 3(d)(1) of rule XIII, the Committee 
adopts as its own the cost estimate prepared by the Director of 
the Congressional Budget Office pursuant to section 402 of the 
Congressional Budget Act of 1974.

    XII. Earmarks, Limited Tax Benefits, and Limited Tariff Benefits

    Pursuant to clause 9(e), 9(f), and 9(g) of rule XXI, the 
Committee finds that H.R. 4439 contains no earmarks, limited 
tax benefits, or limited tariff benefits.

                   XIII. Advisory Committee Statement

    No advisory committee within the meaning of section 5(b) of 
the Federal Advisory Committee Act was created by this 
legislation.

                XIV. Applicability to Legislative Branch

    The Committee finds that the legislation does not relate to 
the terms and conditions of employment or access to public 
services or accommodations within the meaning of section 
102(b)(3) of the Congressional Accountability Act.

           XV. Section-by-Section Analysis of the Legislation


Section 1. Short title

    Section 1 designates that the bill may be cited as the 
``Creating Hope Reauthorization Act''.

Sec. 2. Extension of authority to issue priority review vouchers to 
        encourage treatments for rare pediatric diseases

    Section 2 amends the Federal Food, Drug, and Cosmetic Act 
to extend the sunset on the Secretary's authority to issue new 
Pediatric Rare Disease PRVs to September 30, 2024, provided 
that the PRV is issued for a drug that, not later than 
September 30, 2024, is designated as a drug for a rare 
pediatric disease; and is, not later than September 30, 2026, 
approved under section 351(b)(1) of the Federal Food, Drug, and 
Cosmetic Act, or section 351(a) of the Public Health Service 
Act.

       XVI. Changes in Existing Law Made by the Bill, as Reported

  In compliance with clause 3(e) of rule XIII of the Rules of 
the House of Representatives, changes in existing law made by 
the bill, as reported, are shown as follows (existing law 
proposed to be omitted is enclosed in black brackets, new 
matter is printed in italics, and existing law in which no 
change is proposed is shown in roman):

                  FEDERAL FOOD, DRUG, AND COSMETIC ACT




           *       *       *       *       *       *       *
CHAPTER V--DRUGS AND DEVICES

           *       *       *       *       *       *       *



Subchapter B--Drugs for Rare Diseases or Conditions

           *       *       *       *       *       *       *



SEC. 529. PRIORITY REVIEW TO ENCOURAGE TREATMENTS FOR RARE PEDIATRIC 
                    DISEASES.

  (a) Definitions.--In this section:
          (1) Priority review.--The term ``priority review'', 
        with respect to a human drug application as defined in 
        section 735(1), means review and action by the 
        Secretary on such application not later than 6 months 
        after receipt by the Secretary of such application, as 
        described in the Manual of Policies and Procedures of 
        the Food and Drug Administration and goals identified 
        in the letters described in section 101(b) of the 
        Prescription Drug User Fee Amendments of 2012.
          (2) Priority review voucher.--The term ``priority 
        review voucher'' means a voucher issued by the 
        Secretary to the sponsor of a rare pediatric disease 
        product application that entitles the holder of such 
        voucher to priority review of a single human drug 
        application submitted under section 505(b)(1) or 
        section 351(a) of the Public Health Service Act after 
        the date of approval of the rare pediatric disease 
        product application.
          (3) Rare pediatric disease.--The term ``rare 
        pediatric disease'' means a disease that meets each of 
        the following criteria:
                  (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.
                  (B) The disease is a rare disease or 
                condition, within the meaning of section 526.
          (4) Rare pediatric disease product application.--The 
        term ``rare pediatric disease product application'' 
        means a human drug application, as defined in section 
        735(1), that--
                  (A) is for a drug or biological product--
                          (i) that is for the prevention or 
                        treatment of a rare pediatric disease; 
                        and
                          (ii) that contains no active 
                        ingredient (including any ester or salt 
                        of the active ingredient) that has been 
                        previously approved in any other 
                        application under section 505(b)(1), 
                        505(b)(2), or 505(j) of this Act or 
                        section 351(a) or 351(k) of the Public 
                        Health Service Act;
                  (B) is submitted under section 505(b)(1) of 
                this Act or section 351(a) of the Public Health 
                Service Act;
                  (C) the Secretary deems eligible for priority 
                review;
                  (D) that relies on clinical data derived from 
                studies examining a pediatric population and 
                dosages of the drug intended for that 
                population;
                  (E) that does not seek approval for an adult 
                indication in the original rare pediatric 
                disease product application; and
                  (F) is approved after the date of the 
                enactment of the Advancing Hope Act of 2016.
  (b) Priority Review Voucher.--
          (1) In general.--The Secretary shall award a priority 
        review voucher to the sponsor of a rare pediatric 
        disease product application upon approval by the 
        Secretary of such rare pediatric disease product 
        application.
          (2) Transferability.--
                  (A) In general.--The sponsor of a rare 
                pediatric disease product application that 
                receives a priority review voucher under this 
                section may transfer (including by sale) the 
                entitlement to such voucher. There is no limit 
                on the number of times a priority review 
                voucher may be transferred before such voucher 
                is used.
                  (B) Notification of transfer.--Each person to 
                whom a voucher is transferred shall notify the 
                Secretary of such change in ownership of the 
                voucher not later than 30 days after such 
                transfer.
          (3) Limitation.--A sponsor of a rare pediatric 
        disease product application may not receive a priority 
        review voucher under this section if the rare pediatric 
        disease product application was submitted to the 
        Secretary prior to the date that is 90 days after the 
        date of enactment of the Prescription Drug User Fee 
        Amendments of 2012.
          (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.
          (5) Termination of authority.--The Secretary may not 
        award any priority review vouchers under paragraph (1) 
        after [September 30, 2020] September 30, 2024, unless 
        the rare pediatric disease product application--
                  (A) is for a drug that, not later than 
                [September 30, 2020] September 30, 2024, is 
                designated under subsection (d) as a drug for a 
                rare pediatric disease; and
                  (B) is, not later than [September 30, 2022] 
                September 30, 2026, approved under section 
                505(b)(1) of this Act or section 351(a) of the 
                Public Health Service Act.
  (c) Priority Review User Fee.--
          (1) In general.--The Secretary shall establish a user 
        fee program under which a sponsor of a human drug 
        application that is the subject of a priority review 
        voucher shall pay to the Secretary a fee determined 
        under paragraph (2). Such fee shall be in addition to 
        any fee required to be submitted by the sponsor under 
        chapter VII.
          (2) Fee amount.--The amount of the priority review 
        user fee shall be determined each fiscal year by the 
        Secretary, based on the difference between--
                  (A) the average cost incurred by the Food and 
                Drug Administration in the review of a human 
                drug application subject to priority review in 
                the previous fiscal year; and
                  (B) the average cost incurred by the Food and 
                Drug Administration in the review of a human 
                drug application that is not subject to 
                priority review in the previous fiscal year.
          (3) Annual fee setting.--The Secretary shall 
        establish, before the beginning of each fiscal year 
        beginning after September 30, 2012, the amount of the 
        priority review user fee for that fiscal year.
          (4) Payment.--
                  (A) In general.--The priority review user fee 
                required by this subsection shall be due upon 
                the notification by a sponsor of the intent of 
                such sponsor to use the voucher, as specified 
                in subsection (b)(4)(A). All other user fees 
                associated with the human drug application 
                shall be due as required by the Secretary or 
                under applicable law.
                  (B) Complete application.--An application 
                described under subparagraph (A) for which the 
                sponsor requests the use of a priority review 
                voucher shall be considered incomplete if the 
                fee required by this subsection and all other 
                applicable user fees are not paid in accordance 
                with the Secretary's procedures for paying such 
                fees.
                  (C) No waivers, exemptions, reductions, or 
                refunds.--The Secretary may not grant a waiver, 
                exemption, reduction, or refund of any fees due 
                and payable under this section.
          (5) Offsetting collections.--Fees collected pursuant 
        to this subsection for any fiscal year--
                  (A) shall be deposited and credited as 
                offsetting collections to the account providing 
                appropriations to the Food and Drug 
                Administration; and
                  (B) shall not be collected for any fiscal 
                year except to the extent provided in advance 
                in appropriations Acts.
  (d) Designation Process.--
          (1) In general.--Upon the request of the manufacturer 
        or the sponsor of a new drug, the Secretary may 
        designate--
                  (A) the new drug as a drug for a rare 
                pediatric disease; and
                  (B) the application for the new drug as a 
                rare pediatric disease product application.
          (2) Request for designation.--The request for a 
        designation under paragraph (1) shall be made at the 
        same time a request for designation of orphan disease 
        status under section 526 or fast-track designation 
        under section 506 is made. Requesting designation under 
        this subsection is not a prerequisite to receiving a 
        priority review voucher under this section.
          (3) Determination by secretary.--Not later than 60 
        days after a request is submitted under paragraph (1), 
        the Secretary shall determine whether--
                  (A) the disease or condition that is the 
                subject of such request is a rare pediatric 
                disease; and
                  (B) the application for the new drug is a 
                rare pediatric disease product application.
  (e) Marketing of Rare Pediatric Disease Products.--
          (1) Revocation.--The Secretary may revoke any 
        priority review voucher awarded under subsection (b) if 
        the rare pediatric disease product for which such 
        voucher was awarded is not marketed in the United 
        States within the 365-day period beginning on the date 
        of the approval of such drug under section 505 of this 
        Act or section 351 of the Public Health Service Act.
          (2) Postapproval production report.--The sponsor of 
        an approved rare pediatric disease product shall submit 
        a report to the Secretary not later than 5 years after 
        the approval of the applicable rare pediatric disease 
        product application. Such report shall provide the 
        following information, with respect to each of the 
        first 4 years after approval of such product:
                  (A) The estimated population in the United 
                States suffering from the rare pediatric 
                disease.
                  (B) The estimated demand in the United States 
                for such rare pediatric disease product.
                  (C) The actual amount of such rare pediatric 
                disease product distributed in the United 
                States.
  (f) Notice and Report.--
          (1) Notice of issuance of voucher and approval of 
        products under voucher.--The Secretary shall publish a 
        notice in the Federal Register and on the Internet Web 
        site of the Food and Drug Administration not later than 
        30 days after the occurrence of each of the following:
                  (A) The Secretary issues a priority review 
                voucher under this section.
                  (B) The Secretary approves a drug pursuant to 
                an application submitted under section 505(b) 
                of this Act or section 351(a) of the Public 
                Health Service Act for which the sponsor of the 
                application used a priority review voucher 
                under this section.
          (2) Notification.--If, after the last day of the 1-
        year period that begins on the date that the Secretary 
        awards the third rare pediatric disease priority 
        voucher under this section, a sponsor of an application 
        submitted under section 505(b) of this Act or section 
        351(a) of the Public Health Service Act for a drug uses 
        a priority review voucher under this section for such 
        application, the Secretary shall submit to the 
        Committee on Energy and Commerce of the House of 
        Representatives and the Committee on Health, Education, 
        Labor, and Pensions of the Senate a document--
                  (A) notifying such Committees of the use of 
                such voucher; and
                  (B) identifying the drug for which such 
                priority review voucher is used.
  (g) Eligibility for Other Programs.--Nothing in this section 
precludes a sponsor who seeks a priority review voucher under 
this section from participating in any other incentive program, 
including under this Act, 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..
  (h) Relation to Other Provisions.--The provisions of this 
section shall supplement, not supplant, any other provisions of 
this Act or the Public Health Service Act that encourage the 
development of drugs for tropical diseases and rare pediatric 
diseases.
  (i) GAO Study and Report.--
          (1) Study.--
                  (A) In general.--Beginning on the date that 
                the Secretary awards the third rare pediatric 
                disease priority voucher under this section, 
                the Comptroller General of the United States 
                shall conduct a study of the effectiveness of 
                awarding rare pediatric disease priority 
                vouchers under this section in the development 
                of human drug products that treat or prevent 
                such diseases.
                  (B) Contents of study.--In conducting the 
                study under subparagraph (A), the Comptroller 
                General shall examine the following:
                          (i) The indications for which each 
                        rare disease product for which a 
                        priority review voucher was awarded was 
                        approved under section 505 or section 
                        351 of the Public Health Service Act.
                          (ii) Whether, and to what extent, an 
                        unmet need related to the treatment or 
                        prevention of a rare pediatric disease 
                        was met through the approval of such a 
                        rare disease product.
                          (iii) The value of the priority 
                        review voucher if transferred.
                          (iv) Identification of each drug for 
                        which a priority review voucher was 
                        used.
                          (v) The length of the period of time 
                        between the date on which a priority 
                        review voucher was awarded and the date 
                        on which it was used.
          (2) Report.--Not later than 1 year after the date 
        under paragraph (1)(A), the Comptroller General shall 
        submit to the Committee on Energy and Commerce of the 
        House of Representatives and the Committee on Health, 
        Education, Labor, and Pensions of the Senate, a report 
        containing the results of the study under paragraph 
        (1).

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