[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[S. 1789 Enrolled Bill (ENR)]

        S.1789

                      One Hundred Seventh Congress

                                 of the

                        United States of America


                          AT THE FIRST SESSION

         Begun and held at the City of Washington on Wednesday,
             the third day of January, two thousand and one


                                 An Act


 
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety 
              and efficacy of pharmaceuticals for children.

    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 ``Best Pharmaceuticals for Children 
Act''.

SEC. 2. PEDIATRIC STUDIES OF ALREADY-MARKETED DRUGS.

    Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a) is amended--
        (1) by striking subsection (b); and
        (2) in subsection (c)--
            (A) by inserting after ``the Secretary'' the following: 
        ``determines that information relating to the use of an 
        approved drug in the pediatric population may produce health 
        benefits in that population and''; and
            (B) by striking ``concerning a drug identified in the list 
        described in subsection (b)''.

SEC. 3. RESEARCH FUND FOR THE STUDY OF DRUGS.

    Part B of title IV of the Public Health Service Act (42 U.S.C. 284 
et seq.) is amended--
        (1) by redesignating the second section 409C, relating to 
    clinical research (42 U.S.C. 284k), as section 409G;
        (2) by redesignating the second section 409D, relating to 
    enhancement awards (42 U.S.C. 284l), as section 409H; and
        (3) by adding at the end the following:

``SEC. 409I. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.

    ``(a) List of Drugs for Which Pediatric Studies Are Needed.--
        ``(1) In general.--Not later than one year after the date of 
    enactment of this section, the Secretary, acting through the 
    Director of the National Institutes of Health and in consultation 
    with the Commissioner of Food and Drugs and experts in pediatric 
    research, shall develop, prioritize, and publish an annual list of 
    approved drugs for which--
            ``(A)(i) there is an approved application under section 
        505(j) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
        355(j));
            ``(ii) there is a submitted application that could be 
        approved under the criteria of section 505(j) of the Federal 
        Food, Drug, and Cosmetic Act (21 U.S.C. 355(j));
            ``(iii) there is no patent protection or market exclusivity 
        protection under the Federal Food, Drug, and Cosmetic Act (21 
        U.S.C. 301 et seq.); or
            ``(iv) there is a referral for inclusion on the list under 
        section 505A(d)(4)(C) of the Federal Food, Drug, and Cosmetic 
        Act (21 U.S.C. 355a(d)(4)(C)); and
            ``(B) in the case of a drug referred to in clause (i), 
        (ii), or (iii) of subparagraph (A), additional studies are 
        needed to assess the safety and effectiveness of the use of the 
        drug in the pediatric population.
        ``(2) Consideration of available information.--In developing 
    and prioritizing the list under paragraph (1), the Secretary shall 
    consider, for each drug on the list--
            ``(A) the availability of information concerning the safe 
        and effective use of the drug in the pediatric population;
            ``(B) whether additional information is needed;
            ``(C) whether new pediatric studies concerning the drug may 
        produce health benefits in the pediatric population; and
            ``(D) whether reformulation of the drug is necessary.
    ``(b) Contracts for Pediatric Studies.--The Secretary shall award 
contracts to entities that have the expertise to conduct pediatric 
clinical trials (including qualified universities, hospitals, 
laboratories, contract research organizations, federally funded 
programs such as pediatric pharmacology research units, other public or 
private institutions, or individuals) to enable the entities to conduct 
pediatric studies concerning one or more drugs identified in the list 
described in subsection (a).
    ``(c) Process for Contracts and Labeling Changes.--
        ``(1) Written request to holders of approved applications for 
    drugs lacking exclusivity.--The Commissioner of Food and Drugs, in 
    consultation with the Director of the National Institutes of 
    Health, may issue a written request (which shall include a 
    timeframe for negotiations for an agreement) for pediatric studies 
    concerning a drug identified in the list described in subsection 
    (a)(1)(A) (except clause (iv)) to all holders of an approved 
    application for the drug under section 505 of the Federal Food, 
    Drug, and Cosmetic Act. Such a written request shall be made in a 
    manner equivalent to the manner in which a written request is made 
    under subsection (a) or (b) of section 505A of the Federal Food, 
    Drug, and Cosmetic Act, including with respect to information 
    provided on the pediatric studies to be conducted pursuant to the 
    request.
        ``(2) Requests for contract proposals.--If the Commissioner of 
    Food and Drugs does not receive a response to a written request 
    issued under paragraph (1) within 30 days of the date on which a 
    request was issued, or if a referral described in subsection 
    (a)(1)(A)(iv) is made, the Secretary, acting through the Director 
    of the National Institutes of Health and in consultation with the 
    Commissioner of Food and Drugs, shall publish a request for 
    contract proposals to conduct the pediatric studies described in 
    the written request.
        ``(3) Disqualification.--A holder that receives a first right 
    of refusal shall not be entitled to respond to a request for 
    contract proposals under paragraph (2).
        ``(4) Guidance.--Not later than 270 days after the date of 
    enactment of this section, the Commissioner of Food and Drugs shall 
    promulgate guidance to establish the process for the submission of 
    responses to written requests under paragraph (1).
        ``(5) Contracts.--A contract under this section may be awarded 
    only if a proposal for the contract is submitted to the Secretary 
    in such form and manner, and containing such agreements, 
    assurances, and information as the Secretary determines to be 
    necessary to carry out this section.
        ``(6) Reporting of studies.--
            ``(A) In general.--On completion of a pediatric study in 
        accordance with a contract awarded under this section, a report 
        concerning the study shall be submitted to the Director of the 
        National Institutes of Health and the Commissioner of Food and 
        Drugs. The report shall include all data generated in 
        connection with the study.
            ``(B) Availability of reports.--Each report submitted under 
        subparagraph (A) shall be considered to be in the public domain 
        (subject to section 505A(d)(4)(D) of the Federal Food, Drug, 
        and Cosmetic Act (21 U.S.C. 355a(d)(4)(D)) and shall be 
        assigned a docket number by the Commissioner of Food and Drugs. 
        An interested person may submit written comments concerning 
        such pediatric studies to the Commissioner of Food and Drugs, 
        and the written comments shall become part of the docket file 
        with respect to each of the drugs.
            ``(C) Action by commissioner.--The Commissioner of Food and 
        Drugs shall take appropriate action in response to the reports 
        submitted under subparagraph (A) in accordance with paragraph 
        (7).
        ``(7) Requests for labeling change.--During the 180-day period 
    after the date on which a report is submitted under paragraph 
    (6)(A), the Commissioner of Food and Drugs shall--
            ``(A) review the report and such other data as are 
        available concerning the safe and effective use in the 
        pediatric population of the drug studied;
            ``(B) negotiate with the holders of approved applications 
        for the drug studied for any labeling changes that the 
        Commissioner of Food and Drugs determines to be appropriate and 
        requests the holders to make; and
            ``(C)(i) place in the public docket file a copy of the 
        report and of any requested labeling changes; and
            ``(ii) publish in the Federal Register a summary of the 
        report and a copy of any requested labeling changes.
        ``(8) Dispute resolution.--
            ``(A) Referral to pediatric advisory subcommittee of the 
        anti-infective drugs advisory committee.--If, not later than 
        the end of the 180-day period specified in paragraph (7), the 
        holder of an approved application for the drug involved does 
        not agree to any labeling change requested by the Commissioner 
        of Food and Drugs under that paragraph, the Commissioner of 
        Food and Drugs shall refer the request to the Pediatric 
        Advisory Subcommittee of the Anti-Infective Drugs Advisory 
        Committee.
            ``(B) Action by the pediatric advisory subcommittee of the 
        anti-infective drugs advisory committee.--Not later than 90 
        days after receiving a referral under subparagraph (A), the 
        Pediatric Advisory Subcommittee of the Anti-Infective Drugs 
        Advisory Committee shall--
                ``(i) review the available information on the safe and 
            effective use of the drug in the pediatric population, 
            including study reports submitted under this section; and
                ``(ii) make a recommendation to the Commissioner of 
            Food and Drugs as to appropriate labeling changes, if any.
        ``(9) FDA determination.--Not later than 30 days after 
    receiving a recommendation from the Pediatric Advisory Subcommittee 
    of the Anti-Infective Drugs Advisory Committee under paragraph 
    (8)(B)(ii) with respect to a drug, the Commissioner of Food and 
    Drugs shall consider the recommendation and, if appropriate, make a 
    request to the holders of approved applications for the drug to 
    make any labeling change that the Commissioner of Food and Drugs 
    determines to be appropriate.
        ``(10) Failure to agree.--If a holder of an approved 
    application for a drug, within 30 days after receiving a request to 
    make a labeling change under paragraph (9), does not agree to make 
    a requested labeling change, the Commissioner may deem the drug to 
    be misbranded under the Federal Food, Drug, and Cosmetic Act (21 
    U.S.C. 301 et seq.).
        ``(11) No effect on authority.--Nothing in this subsection 
    limits the authority of the United States to bring an enforcement 
    action under the Federal Food, Drug, and Cosmetic Act when a drug 
    lacks appropriate pediatric labeling. Neither course of action (the 
    Pediatric Advisory Subcommittee of the Anti-Infective Drugs 
    Advisory Committee process or an enforcement action referred to in 
    the preceding sentence) shall preclude, delay, or serve as the 
    basis to stay the other course of action.
        ``(12) Recommendation for formulation changes.--If a pediatric 
    study completed under public contract indicates that a formulation 
    change is necessary and the Secretary agrees, the Secretary shall 
    send a nonbinding letter of recommendation regarding that change to 
    each holder of an approved application.
    ``(d) Authorization of Appropriations.--
        ``(1) In general.--There are authorized to be appropriated to 
    carry out this section--
            ``(A) $200,000,000 for fiscal year 2002; and
            ``(B) such sums as are necessary for each of the five 
        succeeding fiscal years.
        ``(2) Availability.--Any amount appropriated under paragraph 
    (1) shall remain available to carry out this section until 
    expended.''.
SEC. 4. WRITTEN REQUEST TO HOLDERS OF APPROVED APPLICATIONS FOR DRUGS 
THAT HAVE MARKET EXCLUSIVITY.
    Section 505A(d) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355a(d)) is amended by adding at the end the following:
        ``(4) Written request to holders of approved applications for 
    drugs that have market exclusivity.--
            ``(A) Request and response.--If the Secretary makes a 
        written request for pediatric studies (including neonates, as 
        appropriate) under subsection (c) to the holder of an 
        application approved under section 505(b)(1), the holder, not 
        later than 180 days after receiving the written request, shall 
        respond to the Secretary as to the intention of the holder to 
        act on the request by--
                ``(i) indicating when the pediatric studies will be 
            initiated, if the holder agrees to the request; or
                ``(ii) indicating that the holder does not agree to the 
            request.
            ``(B) No agreement to request.--
                ``(i) Referral.--If the holder does not agree to a 
            written request within the time period specified in 
            subparagraph (A), and if the Secretary determines that 
            there is a continuing need for information relating to the 
            use of the drug in the pediatric population (including 
            neonates, as appropriate), the Secretary shall refer the 
            drug to the Foundation for the National Institutes of 
            Health established under section 499 of the Public Health 
            Service Act (42 U.S.C. 290b) (referred to in this paragraph 
            as the `Foundation') for the conduct of the pediatric 
            studies described in the written request.
                ``(ii) Public notice.--The Secretary shall give public 
            notice of the name of the drug, the name of the 
            manufacturer, and the indications to be studied made in a 
            referral under clause (i).
            ``(C) Lack of funds.--On referral of a drug under 
        subparagraph (B)(i), the Foundation shall issue a proposal to 
        award a grant to conduct the requested studies unless the 
        Foundation certifies to the Secretary, within a timeframe that 
        the Secretary determines is appropriate through guidance, that 
        the Foundation does not have funds available under section 
        499(j)(9)(B)(i) to conduct the requested studies. If the 
        Foundation so certifies, the Secretary shall refer the drug for 
        inclusion on the list established under section 409I of the 
        Public Health Service Act for the conduct of the studies.
            ``(D) Effect of subsection.--Nothing in this subsection 
        (including with respect to referrals from the Secretary to the 
        Foundation) alters or amends section 301(j) of this Act or 
        section 552 of title 5 or section 1905 of title 18, United 
        States Code.
            ``(E) No requirement to refer.--Nothing in this subsection 
        shall be construed to require that every declined written 
        request shall be referred to the Foundation.
            ``(F) Written requests under subsection (b).--For drugs 
        under subsection (b) for which written requests have not been 
        accepted, if the Secretary determines that there is a 
        continuing need for information relating to the use of the drug 
        in the pediatric population (including neonates, as 
        appropriate), the Secretary shall issue a written request under 
        subsection (c) after the date of approval of the drug.''.
SEC. 5. TIMELY LABELING CHANGES FOR DRUGS GRANTED EXCLUSIVITY; DRUG 
FEES.
    (a) Elimination of User Fee Waiver for Pediatric Supplements.--
Section 736(a)(1) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 379h(a)(1)) is amended--
        (1) by striking subparagraph (F); and
        (2) by redesignating subparagraph (G) as subparagraph (F).
    (b) Labeling Changes.--
        (1) Definition of priority supplement.--Section 201 of the 
    Federal Food, Drug, and Cosmetic Act (21 U.S.C. 321) is amended by 
    adding at the end the following:
        ``(kk) Priority supplement.--The term `priority supplement' 
    means a drug application referred to in section 101(4) of the Food 
    and Drug Administration Modernization Act of 1997 (111 Stat. 
    2298).''.
        (2) Treatment as priority supplements.--Section 505A of the 
    Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is amended by 
    adding at the end the following:
    ``(l) Labeling Supplements.--
        ``(1) Priority status for pediatric supplements.--Any 
    supplement to an application under section 505 proposing a labeling 
    change pursuant to a report on a pediatric study under this 
    section--
            ``(A) shall be considered to be a priority supplement; and
            ``(B) shall be subject to the performance goals established 
        by the Commissioner for priority drugs.
        ``(2) Dispute resolution.--
            ``(A) Request for labeling change and failure to agree.--If 
        the Commissioner determines that an application with respect to 
        which a pediatric study is conducted under this section is 
        approvable and that the only open issue for final action on the 
        application is the reaching of an agreement between the sponsor 
        of the application and the Commissioner on appropriate changes 
        to the labeling for the drug that is the subject of the 
        application, not later than 180 days after the date of 
        submission of the application--
                ``(i) the Commissioner shall request that the sponsor 
            of the application make any labeling change that the 
            Commissioner determines to be appropriate; and
                ``(ii) if the sponsor of the application does not agree 
            to make a labeling change requested by the Commissioner, 
            the Commissioner shall refer the matter to the Pediatric 
            Advisory Subcommittee of the Anti-Infective Drugs Advisory 
            Committee.
            ``(B) Action by the pediatric advisory subcommittee of the 
        anti-infective drugs advisory committee.--Not later than 90 
        days after receiving a referral under subparagraph (A)(ii), the 
        Pediatric Advisory Subcommittee of the Anti-Infective Drugs 
        Advisory Committee shall--
                ``(i) review the pediatric study reports; and
                ``(ii) make a recommendation to the Commissioner 
            concerning appropriate labeling changes, if any.
            ``(C) Consideration of recommendations.--The Commissioner 
        shall consider the recommendations of the Pediatric Advisory 
        Subcommittee of the Anti-Infective Drugs Advisory Committee 
        and, if appropriate, not later than 30 days after receiving the 
        recommendation, make a request to the sponsor of the 
        application to make any labeling change that the Commissioner 
        determines to be appropriate.
            ``(D) Misbranding.--If the sponsor of the application, 
        within 30 days after receiving a request under subparagraph 
        (C), does not agree to make a labeling change requested by the 
        Commissioner, the Commissioner may deem the drug that is the 
        subject of the application to be misbranded.
            ``(E) No effect on authority.--Nothing in this subsection 
        limits the authority of the United States to bring an 
        enforcement action under this Act when a drug lacks appropriate 
        pediatric labeling. Neither course of action (the Pediatric 
        Advisory Subcommittee of the Anti-Infective Drugs Advisory 
        Committee process or an enforcement action referred to in the 
        preceding sentence) shall preclude, delay, or serve as the 
        basis to stay the other course of action.''.

SEC. 6. OFFICE OF PEDIATRIC THERAPEUTICS.

    (a) Establishment.--The Secretary of Health and Human Services 
shall establish an Office of Pediatric Therapeutics within the Food and 
Drug Administration.
    (b) Duties.--The Office of Pediatric Therapeutics shall be 
responsible for coordination and facilitation of all activities of the 
Food and Drug Administration that may have any effect on a pediatric 
population or the practice of pediatrics or may in any other way 
involve pediatric issues.
    (c) Staff.--The staff of the Office of Pediatric Therapeutics shall 
coordinate with employees of the Department of Health and Human 
Services who exercise responsibilities relating to pediatric 
therapeutics and shall include--
        (1) one or more additional individuals with expertise 
    concerning ethical issues presented by the conduct of clinical 
    research in the pediatric population; and
        (2) one or more additional individuals with expertise in 
    pediatrics as may be necessary to perform the activities described 
    in subsection (b).

SEC. 7. NEONATES.

    Section 505A(g) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355a(g)) is amended by inserting ``(including neonates in 
appropriate cases)'' after ``pediatric age groups''.

SEC. 8. SUNSET.

    Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a) is amended by striking subsection (j) and inserting the 
following:
    ``(j) Sunset.--A drug may not receive any 6-month period under 
subsection (a) or (c) unless--
        ``(1) on or before October 1, 2007, the Secretary makes a 
    written request for pediatric studies of the drug;
        ``(2) on or before October 1, 2007, an application for the drug 
    is accepted for filing under section 505(b); and
        ``(3) all requirements of this section are met.''.

SEC. 9. DISSEMINATION OF PEDIATRIC INFORMATION.

    Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a) (as amended by section 5(b)(2)) is amended by adding at the end 
the following:
    ``(m) Dissemination of Pediatric Information.--
        ``(1) In general.--Not later than 180 days after the date of 
    submission of a report on a pediatric study under this section, the 
    Commissioner shall make available to the public a summary of the 
    medical and clinical pharmacology reviews of pediatric studies 
    conducted for the supplement, including by publication in the 
    Federal Register.
        ``(2) Effect of subsection.--Nothing in this subsection alters 
    or amends section 301(j) of this Act or section 552 of title 5 or 
    section 1905 of title 18, United States Code.''.
  SEC. 10. CLARIFICATION OF INTERACTION OF PEDIATRIC EXCLUSIVITY UNDER 
      SECTION 505A OF THE FEDERAL FOOD, DRUG, AND COSMETIC ACT AND 180-
      DAY EXCLUSIVITY AWARDED TO AN APPLICANT FOR APPROVAL OF A DRUG 
      UNDER SECTION 505(j) OF THAT ACT.
    Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a) (as amended by section 9) is amended by adding at the end the 
following:
    ``(n) Clarification of Interaction of Market Exclusivity Under This 
Section and Market Exclusivity Awarded to an Applicant for Approval of 
a Drug Under Section 505(j).--If a 180-day period under section 
505(j)(5)(B)(iv) overlaps with a 6-month exclusivity period under this 
section, so that the applicant for approval of a drug under section 
505(j) entitled to the 180-day period under that section loses a 
portion of the 180-day period to which the applicant is entitled for 
the drug, the 180-day period shall be extended from--
        ``(1) the date on which the 180-day period would have expired 
    by the number of days of the overlap, if the 180-day period would, 
    but for the application of this subsection, expire after the 6-
    month exclusivity period; or
        ``(2) the date on which the 6-month exclusivity period expires, 
    by the number of days of the overlap if the 180-day period would, 
    but for the application of this subsection, expire during the six-
    month exclusivity period.''.
  SEC. 11. PROMPT APPROVAL OF DRUGS UNDER SECTION 505(j) WHEN PEDIATRIC 
      INFORMATION IS ADDED TO LABELING.
    (a) In General.--Section 505A of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355a) (as amended by section 10) is amended by 
adding at the end the following:
    ``(o) Prompt Approval of Drugs Under Section 505(j) When Pediatric 
Information Is Added to Labeling.--
        ``(1) General rule.--A drug for which an application has been 
    submitted or approved under section 505(j) shall not be considered 
    ineligible for approval under that section or misbranded under 
    section 502 on the basis that the labeling of the drug omits a 
    pediatric indication or any other aspect of labeling pertaining to 
    pediatric use when the omitted indication or other aspect is 
    protected by patent or by exclusivity under clause (iii) or (iv) of 
    section 505(j)(5)(D).
        ``(2) Labeling.--Notwithstanding clauses (iii) and (iv) of 
    section 505(j)(5)(D), the Secretary may require that the labeling 
    of a drug approved under section 505(j) that omits a pediatric 
    indication or other aspect of labeling as described in paragraph 
    (1) include--
            ``(A) a statement that, because of marketing exclusivity 
        for a manufacturer--
                ``(i) the drug is not labeled for pediatric use; or
                ``(ii) in the case of a drug for which there is an 
            additional pediatric use not referred to in paragraph (1), 
            the drug is not labeled for the pediatric use under 
            paragraph (1); and
            ``(B) a statement of any appropriate pediatric 
        contraindications, warnings, or precautions that the Secretary 
        considers necessary.
        ``(3) Preservation of pediatric exclusivity and other 
    provisions.--This subsection does not affect--
            ``(A) the availability or scope of exclusivity under this 
        section;
            ``(B) the availability or scope of exclusivity under 
        section 505 for pediatric formulations;
            ``(C) the question of the eligibility for approval of any 
        application under section 505(j) that omits any other 
        conditions of approval entitled to exclusivity under clause 
        (iii) or (iv) of section 505(j)(5)(D); or
            ``(D) except as expressly provided in paragraphs (1) and 
        (2), the operation of section 505.''.
    (b) Effective Date.--The amendment made by subsection (a) takes 
effect on the date of enactment of this Act, including with respect to 
applications under section 505(j) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355(j)) that are approved or pending on that 
date.

SEC. 12. STUDY CONCERNING RESEARCH INVOLVING CHILDREN.

    (a) Contract With Institute of Medicine.--The Secretary of Health 
and Human Services shall enter into a contract with the Institute of 
Medicine for--
        (1) the conduct, in accordance with subsection (b), of a review 
    of--
            (A) Federal regulations in effect on the date of the 
        enactment of this Act relating to research involving children;
            (B) federally prepared or supported reports relating to 
        research involving children; and
            (C) federally supported evidence-based research involving 
        children; and
        (2) the submission to the Committee on Health, Education, 
    Labor, and Pensions of the Senate and the Committee on Energy and 
    Commerce of the House of Representatives, not later than two years 
    after the date of enactment of this Act, of a report concerning the 
    review conducted under paragraph (1) that includes recommendations 
    on best practices relating to research involving children.
    (b) Areas of Review.--In conducting the review under subsection 
(a)(1), the Institute of Medicine shall consider the following:
        (1) The written and oral process of obtaining and defining 
    ``assent'', ``permission'' and ``informed consent'' with respect to 
    child clinical research participants and the parents, guardians, 
    and the individuals who may serve as the legally authorized 
    representatives of such children (as defined in subpart A of part 
    46 of title 45, Code of Federal Regulations).
        (2) The expectations and comprehension of child research 
    participants and the parents, guardians, or legally authorized 
    representatives of such children, for the direct benefits and risks 
    of the child's research involvement, particularly in terms of 
    research versus therapeutic treatment.
        (3) The definition of ``minimal risk'' with respect to a 
    healthy child or a child with an illness.
        (4) The appropriateness of the regulations applicable to 
    children of differing ages and maturity levels, including 
    regulations relating to legal status.
        (5) Whether payment (financial or otherwise) may be provided to 
    a child or his or her parent, guardian, or legally authorized 
    representative for the participation of the child in research, and 
    if so, the amount and type of payment that may be made.
        (6) Compliance with the regulations referred to in subsection 
    (a)(1)(A), the monitoring of such compliance (including the role of 
    institutional review boards), and the enforcement actions taken for 
    violations of such regulations.
        (7) The unique roles and responsibilities of institutional 
    review boards in reviewing research involving children, including 
    composition of membership on institutional review boards.
    (c) Requirements of Expertise.--The Institute of Medicine shall 
conduct the review under subsection (a)(1) and make recommendations 
under subsection (a)(2) in conjunction with experts in pediatric 
medicine, pediatric research, and the ethical conduct of research 
involving children.
  SEC. 13. FOUNDATION FOR THE NATIONAL INSTITUTES OF HEALTH.
    Section 499 of the Public Health Service Act (42 U.S.C. 290b) is 
amended--
        (1) in subsection (b), by inserting ``(including collection of 
    funds for pediatric pharmacologic research)'' after ``mission'';
        (2) in subsection (c)(1)--
            (A) by redesignating subparagraph (C) as subparagraph (D); 
        and
            (B) by inserting after subparagraph (B) the following:
            ``(C) A program to collect funds for pediatric 
        pharmacologic research and studies listed by the Secretary 
        pursuant to section 409I(a)(1)(A) of this Act and referred 
        under section 505A(d)(4)(C) of the Federal Food, Drug, and 
        Cosmetic Act (21 U.S.C. 355a(d)(4)(C)).'';
        (3) in subsection (d)--
            (A) in paragraph (1)--
                (i) in subparagraph (B)--

                    (I) in clause (ii), by striking ``and'' at the end;
                    (II) in clause (iii), by striking the period and 
                inserting ``; and''; and
                    (III) by adding at the end the following:

                ``(iv) the Commissioner of Food and Drugs.''; and
                (ii) by striking subparagraph (C) and inserting the 
            following:
            ``(C) The ex officio members of the Board under 
        subparagraph (B) shall appoint to the Board individuals from 
        among a list of candidates to be provided by the National 
        Academy of Science. Such appointed members shall include--
                ``(i) representatives of the general biomedical field;
                ``(ii) representatives of experts in pediatric medicine 
            and research;
                ``(iii) representatives of the general biobehavioral 
            field, which may include experts in biomedical ethics; and
                ``(iv) representatives of the general public, which may 
            include representatives of affected industries.''; and
            (B) in paragraph (2), by realigning the margin of 
        subparagraph (B) to align with subparagraph (A);
        (4) in subsection (k)(9)--
            (A) by striking ``The Foundation'' and inserting the 
        following:
            ``(A) In general.--The Foundation''; and
            (B) by adding at the end the following:
            ``(B) Gifts, grants, and other donations.--
                ``(i) In general.--Gifts, grants, and other donations 
            to the Foundation may be designated for pediatric research 
            and studies on drugs, and funds so designated shall be used 
            solely for grants for research and studies under subsection 
            (c)(1)(C).
                ``(ii) Other gifts.--Other gifts, grants, or donations 
            received by the Foundation and not described in clause (i) 
            may also be used to support such pediatric research and 
            studies.
                ``(iii) Report.--The recipient of a grant for research 
            and studies shall agree to provide the Director of the 
            National Institutes of Health and the Commissioner of Food 
            and Drugs, at the conclusion of the research and studies--

                    ``(I) a report describing the results of the 
                research and studies; and
                    ``(II) all data generated in connection with the 
                research and studies.

                ``(iv) Action by the commissioner of food and drugs.--
            The Commissioner of Food and Drugs shall take appropriate 
            action in response to a report received under clause (iii) 
            in accordance with paragraphs (7) through (12) of section 
            409I(c), including negotiating with the holders of approved 
            applications for the drugs studied for any labeling changes 
            that the Commissioner determines to be appropriate and 
            requests the holders to make.
            ``(C) Applicability.--Subparagraph (A) does not apply to 
        the program described in subsection (c)(1)(C).'';
        (5) by redesignating subsections (f) through (m) as subsections 
    (e) through (l), respectively;
        (6) in subsection (h)(11) (as so redesignated), by striking 
    ``solicit'' and inserting ``solicit,''; and
        (7) in paragraphs (1) and (2) of subsection (j) (as so 
    redesignated), by striking ``(including those developed under 
    subsection (d)(2)(B)(i)(II))'' each place it appears.

SEC. 14. PEDIATRIC PHARMACOLOGY ADVISORY COMMITTEE.

    (a) In General.--The Secretary of Health and Human Services shall, 
under section 222 of the Public Health Service Act (42 U.S.C. 217a), 
convene and consult an advisory committee on pediatric pharmacology 
(referred to in this section as the ``advisory committee'').
    (b) Purpose.--
        (1) In general.--The advisory committee shall advise and make 
    recommendations to the Secretary, through the Commissioner of Food 
    and Drugs and in consultation with the Director of the National 
    Institutes of Health, on matters relating to pediatric 
    pharmacology.
        (2) Matters included.--The matters referred to in paragraph (1) 
    include--
            (A) pediatric research conducted under sections 351, 409I, 
        and 499 of the Public Health Service Act and sections 501, 502, 
        505, and 505A of the Federal Food, Drug, and Cosmetic Act;
            (B) identification of research priorities related to 
        pediatric pharmacology and the need for additional treatments 
        of specific pediatric diseases or conditions; and
            (C) the ethics, design, and analysis of clinical trials 
        related to pediatric pharmacology.
    (c) Composition.--The advisory committee shall include 
representatives of pediatric health organizations, pediatric 
researchers, relevant patient and patient-family organizations, and 
other experts selected by the Secretary.
  SEC. 15. PEDIATRIC SUBCOMMITTEE OF THE ONCOLOGIC DRUGS ADVISORY 
      COMMITTEE.
    (a) Clarification of Authorities.--
        (1) In general.--The Pediatric Subcommittee of the Oncologic 
    Drugs Advisory Committee (referred to in this section as the 
    ``Subcommittee''), in carrying out the mission of reviewing and 
    evaluating the data concerning the safety and effectiveness of 
    marketed and investigational human drug products for use in the 
    treatment of pediatric cancers, shall--
            (A) evaluate and, to the extent practicable, prioritize new 
        and emerging therapeutic alternatives available to treat 
        pediatric cancer;
            (B) provide recommendations and guidance to help ensure 
        that children with cancer have timely access to the most 
        promising new cancer therapies; and
            (C) advise on ways to improve consistency in the 
        availability of new therapeutic agents.
        (2) Membership.--
            (A) In general.--The Secretary shall appoint not more than 
        11 voting members to the Pediatric Subcommittee from the 
        membership of the Pediatric Pharmacology Advisory Committee and 
        the Oncologic Drugs Advisory Committee.
            (B) Request for participation.--The Subcommittee shall 
        request participation of the following members in the 
        scientific and ethical consideration of topics of pediatric 
        cancer, as necessary:
                (i) At least two pediatric oncology specialists from 
            the National Cancer Institute.
                (ii) At least four pediatric oncology specialists 
            from--

                    (I) the Children's Oncology Group;
                    (II) other pediatric experts with an established 
                history of conducting clinical trials in children; or
                    (III) consortia sponsored by the National Cancer 
                Institute, such as the Pediatric Brain Tumor 
                Consortium, the New Approaches to Neuroblastoma Therapy 
                or other pediatric oncology consortia.

                (iii) At least two representatives of the pediatric 
            cancer patient and patient-family community.
                (iv) One representative of the nursing community.
                (v) At least one statistician.
                (vi) At least one representative of the pharmaceutical 
            industry.
    (b) Pre-Clinical Models To Evaluate Promising Pediatric Cancer 
Therapies.--Section 413 of the Public Health Service Act (42 U.S.C. 
285a-2) is amended by adding at the end the following:
    ``(c) Pre-Clinical Models To Evaluate Promising Pediatric Cancer 
Therapies.--
        ``(1) Expansion and coordination of activities.--The Director 
    of the National Cancer Institute shall expand, intensify, and 
    coordinate the activities of the Institute with respect to research 
    on the development of preclinical models to evaluate which 
    therapies are likely to be effective for treating pediatric cancer.
        ``(2) Coordination with other institutes.--The Director of the 
    Institute shall coordinate the activities under paragraph (1) with 
    similar activities conducted by other national research institutes 
    and agencies of the National Institutes of Health to the extent 
    that those Institutes and agencies have responsibilities that are 
    related to pediatric cancer.''.
    (c) Clarification of Availability of Investigational New Drugs for 
Pediatric Study and Use.--
        (1) Amendment of the federal food, drug, and cosmetic act.--
    Section 505(i)(1) of the Federal Food, Drug, and Cosmetic Act (21 
    U.S.C. 355(i)(1)) is amended--
            (A) in subparagraph (B), by striking ``and'' at the end;
            (B) in subparagraph (C), by striking the period at the end 
        and inserting ``; and''; and
            (C) by adding at the end the following:
            ``(D) the submission to the Secretary by the manufacturer 
        or the sponsor of the investigation of a new drug of a 
        statement of intent regarding whether the manufacturer or 
        sponsor has plans for assessing pediatric safety and 
        efficacy.''.
        (2) Amendment of the public health service act.--Section 
    402(j)(3)(A) of the Public Health Service Act (42 U.S.C. 
    282(j)(3)(A)) is amended in the first sentence--
            (A) by striking ``trial sites, and'' and inserting ``trial 
        sites,''; and
            (B) by striking ``in the trial,'' and inserting ``in the 
        trial, and a description of whether, and through what 
        procedure, the manufacturer or sponsor of the investigation of 
        a new drug will respond to requests for protocol exception, 
        with appropriate safeguards, for single-patient and expanded 
        protocol use of the new drug, particularly in children,''.
    (d) Report.--Not later than January 31, 2003, the Secretary of 
Health and Human Services, acting through the Commissioner of Food and 
Drugs and in consultation with the Director of the National Institutes 
of Health, 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 on patient access to new 
therapeutic agents for pediatric cancer, including access to single 
patient use of new therapeutic agents.

SEC. 16. REPORT ON PEDIATRIC EXCLUSIVITY PROGRAM.

    Not later than October 1, 2006, the Comptroller General of the 
United States, in consultation with the Secretary of Health and Human 
Services, shall submit to Congress a report that addresses the 
following issues, using publicly available data or data otherwise 
available to the Government that may be used and disclosed under 
applicable law:
        (1) The effectiveness of section 505A of the Federal Food, 
    Drug, and Cosmetic Act and section 409I of the Public Health 
    Service Act (as added by this Act) in ensuring that medicines used 
    by children are tested and properly labeled, including--
            (A) the number and importance of drugs for children that 
        are being tested as a result of this legislation and the 
        importance for children, health care providers, parents, and 
        others of labeling changes made as a result of such testing;
            (B) the number and importance of drugs for children that 
        are not being tested for their use notwithstanding the 
        provisions of this legislation, and possible reasons for the 
        lack of testing; and
            (C) the number of drugs for which testing is being done, 
        exclusivity granted, and labeling changes required, including 
        the date pediatric exclusivity is granted and the date labeling 
        changes are made and which labeling changes required the use of 
        the dispute resolution process established pursuant to the 
        amendments made by this Act, together with a description of the 
        outcomes of such process, including a description of the 
        disputes and the recommendations of the Pediatric Advisory 
        Subcommittee of the Anti-Infective Drugs Advisory Committee.
        (2) The economic impact of section 505A of the Federal Food, 
    Drug, and Cosmetic Act and section 409I of the Public Health 
    Service Act (as added by this Act), including an estimate of--
            (A) the costs to taxpayers in the form of higher 
        expenditures by medicaid and other Government programs;
            (B) sales for each drug during the 6-month period for which 
        exclusivity is granted, as attributable to such exclusivity;
            (C) costs to consumers and private insurers as a result of 
        any delay in the availability of lower cost generic equivalents 
        of drugs tested and granted exclusivity under the Federal Food, 
        Drug, and Cosmetic Act (21 U.S.C. 301 et seq.), and loss of 
        revenue by the generic drug industry and retail pharmacies as a 
        result of any such delay; and
            (D) the benefits to the government, to private insurers, 
        and to consumers resulting from decreased health care costs, 
        including--
                (i) decreased hospitalizations and fewer medical 
            errors, due to more appropriate and more effective use of 
            medications in children as a result of testing and re-
            labeling because of the amendments made by this Act;
                (ii) direct and indirect benefits associated with fewer 
            physician visits not related to hospitalization;
                (iii) benefits to children from missing less time at 
            school and being less affected by chronic illnesses, 
            thereby allowing a better quality of life;
                (iv) benefits to consumers from lower health insurance 
            premiums due to lower treatment costs and hospitalization 
            rates; and
                (v) benefits to employers from reduced need for 
            employees to care for family members.
        (3) The nature and type of studies in children for each drug 
    granted exclusivity under the Federal Food, Drug, and Cosmetic Act 
    (21 U.S.C. 301 et seq.), including--
            (A) a description of the complexity of the studies;
            (B) the number of study sites necessary to obtain 
        appropriate data;
            (C) the number of children involved in any clinical 
        studies; and
            (D) the estimated cost of each of the studies.
        (4) Any recommendations for modifications to the programs 
    established under section 505A of the Federal Food, Drug, and 
    Cosmetic Act (21 U.S.C. 355a) and section 409I of the Public Health 
    Service Act (as added by section 3) that the Secretary determines 
    to be appropriate, including a detailed rationale for each 
    recommendation.
        (5) The increased private and Government-funded pediatric 
    research capability associated with this Act and the amendments 
    made by this Act.
        (6) The number of written requests and additional letters of 
    recommendation that the Secretary issues.
        (7) The prioritized list of off-patent drugs for which the 
    Secretary issues written requests.
        (8)(A) The efforts made by the Secretary to increase the number 
    of studies conducted in the neonate population; and
        (B) the results of those efforts, including efforts made to 
    encourage the conduct of appropriate studies in neonates by 
    companies with products that have sufficient safety and other 
    information to make the conduct of studies ethical and safe.

SEC. 17. ADVERSE-EVENT REPORTING.

    (a) Toll-Free Number in Labeling.--Not later than one year after 
the date of the enactment of this Act, the Secretary of Health and 
Human Services shall promulgate a final rule requiring that the 
labeling of each drug for which an application is approved under 
section 505 of the Federal Food, Drug, and Cosmetic Act (regardless of 
the date on which approved) include the toll-free number maintained by 
the Secretary for the purpose of receiving reports of adverse events 
regarding drugs and a statement that such number is to be used for 
reporting purposes only, not to receive medical advice. With respect to 
the final rule:
        (1) The rule shall provide for the implementation of such 
    labeling requirement in a manner that the Secretary considers to be 
    most likely to reach the broadest consumer audience.
        (2) In promulgating the rule, the Secretary shall seek to 
    minimize the cost of the rule on the pharmacy profession.
        (3) The rule shall take effect not later than 60 days after the 
    date on which the rule is promulgated.
    (b) Drugs With Pediatric Market Exclusivity.--
        (1) In general.--During the one year beginning on the date on 
    which a drug receives a period of market exclusivity under 505A of 
    the Federal Food, Drug, and Cosmetic Act, any report of an adverse 
    event regarding the drug that the Secretary of Health and Human 
    Services receives shall be referred to the Office of Pediatric 
    Therapeutics established under section 6 of this Act. In 
    considering the report, the Director of such Office shall provide 
    for the review of the report by the Pediatric Advisory Subcommittee 
    of the Anti-Infective Drugs Advisory Committee, including obtaining 
    any recommendations of such subcommittee regarding whether the 
    Secretary should take action under the Federal Food, Drug, and 
    Cosmetic Act in response to the report.
        (2) Rule of construction.--Paragraph (1) may not be construed 
    as restricting the authority of the Secretary of Health and Human 
    Services to continue carrying out the activities described in such 
    paragraph regarding a drug after the one-year period described in 
    such paragraph regarding the drug has expired.
  SEC. 18. MINORITY CHILDREN AND PEDIATRIC-EXCLUSIVITY PROGRAM.
    (a) Protocols for Pediatric Studies.--Section 505A of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is amended in subsection 
(d)(2) by inserting after the first sentence the following: ``In 
reaching an agreement regarding written protocols, the Secretary shall 
take into account adequate representation of children of ethnic and 
racial minorities.''.
    (b) Study by General Accounting Office.--
        (1) In general.--The Comptroller General of the United States 
    shall conduct a study for the purpose of determining the following:
            (A) The extent to which children of ethnic and racial 
        minorities are adequately represented in studies under section 
        505A of the Federal Food, Drug, and Cosmetic Act; and to the 
        extent ethnic and racial minorities are not adequately 
        represented, the reasons for such under representation and 
        recommendations to increase such representation.
            (B) Whether the Food and Drug Administration has 
        appropriate management systems to monitor the representation of 
        the children of ethnic and racial minorities in such studies.
            (C) Whether drugs used to address diseases that 
        disproportionately affect racial and ethnic minorities are 
        being studied for their safety and effectiveness under section 
        505A of the Federal Food, Drug, and Cosmetic Act.
        (2) Date certain for completing study.--Not later than January 
    10, 2003, the Comptroller General shall complete the study required 
    in paragraph (1) and submit to the Congress a report describing the 
    findings of the study.

SEC. 19. TECHNICAL AND CONFORMING AMENDMENTS.

    Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355a) (as amended by sections 2(1), 5(b)(2), 9, 10, 11, and 17) is 
amended--
        (1)(A) by striking ``(j)(4)(D)(ii)'' each place it appears and 
    inserting ``(j)(5)(D)(ii)'';
        (B) by striking ``(j)(4)(D)'' each place it appears and 
    inserting ``(j)(5)(D)''; and
        (C) by striking ``505(j)(4)(D)'' each place it appears and 
    inserting ``505(j)(5)(D)'';
        (2) by redesignating subsections (a), (g), (h), (i), (j), (k), 
    (l), (m), (n), and (o) as subsections (b), (a), (g), (h), (n), (m), 
    (i), (j), (k), and (l) respectively;
        (3) by moving the subsections so as to appear in alphabetical 
    order;
        (4) in paragraphs (1), (2), and (3) of subsection (d), 
    subsection (e), and subsection (m) (as redesignated by paragraph 
    (2)), by striking ``subsection (a) or (c)'' and inserting 
    ``subsection (b) or (c)''; and
        (5) in subsection (g) (as redesignated by paragraph (2)), by 
    striking ``subsection (a) or (b)'' and inserting ``subsection (b) 
    or (c)''.

                               Speaker of the House of Representatives.

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