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Best Pharmaceuticals for Children Act, January 4, 2002 (Public Law No. 107-109)

<DOC>
[DOCID: f:publ109.107]


[[Page 1407]]

                  BEST PHARMACEUTICALS FOR CHILDREN ACT

[[Page 115 STAT. 1408]]

Public Law 107-109
107th Congress

                                 An Act


 
To amend the Federal Food, Drug, and Cosmetic Act to improve the safety 
     and efficacy of pharmaceuticals for children. <<NOTE: Jan. 4, 
                          2002 -  [S. 1789]>> 

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress <<NOTE: Best Pharmaceuticals for 
Children Act.>> assembled,

SECTION 1. <<NOTE: 21 USC 301 note.>> 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. <<NOTE: 42 USC 284m.>> 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));

[[Page 115 STAT. 1409]]

                    ``(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 <<NOTE: Deadline.>> 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 <<NOTE: Deadline.>> later than 270 days 
        after the date of enactment of this section, the Commissioner of 
        Food and

[[Page 115 STAT. 1410]]

        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 <<NOTE:  Deadline.>> of the anti-infective 
                drugs advisory committee.--Not later than 90 days after 
                receiving a referral

[[Page 115 STAT. 1411]]

                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 <<NOTE: Deadline.>> 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.--

[[Page 115 STAT. 1412]]

                    ``(A) Request <<NOTE: Deadline.>> 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.''.

[[Page 115 STAT. 1413]]

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 <<NOTE: Deadline.>> 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 <<NOTE: Deadline.>> 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.

[[Page 115 STAT. 1414]]

                    ``(C) Consideration <<NOTE: Deadline.>> 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. <<NOTE: 21 USC 393a.>> 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;

[[Page 115 STAT. 1415]]

            ``(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 <<NOTE: Deadline.>> 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

[[Page 115 STAT. 1416]]

        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 <<NOTE: 21 USC 355a note.>> 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. <<NOTE: 42 USC 289 note.>> 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:

[[Page 115 STAT. 1417]]

            (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

[[Page 115 STAT. 1418]]

                          (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

[[Page 115 STAT. 1419]]

            (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. <<NOTE: 42 USC 284m note.>> 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

[[Page 115 STAT. 1420]]

                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

[[Page 115 STAT. 1421]]

                    (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 <<NOTE: Deadline. 21 USC 355 note.>> 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. <<NOTE: 21 USC 355a note.>> REPORT ON PEDIATRIC EXCLUSIVITY 
            PROGRAM.

    Not <<NOTE: Deadline.>> 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;

[[Page 115 STAT. 1422]]

                    (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. <<NOTE: 21 USC 355b.>> ADVERSE-EVENT REPORTING.

    (a) Toll-Free <<NOTE: Deadline. Regulations.>> 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

[[Page 115 STAT. 1423]]

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 <<NOTE: 21 USC 355a note.>> 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.

[[Page 115 STAT. 1424]]

                    (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 <<NOTE: Deadline.>> 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)''.

    Approved January 4, 2002.

LEGISLATIVE HISTORY--S. 1789 (H.R. 2887):
---------------------------------------------------------------------------

HOUSE REPORTS: No. 107-277 accompanying H.R. 2887 (Comm. on Energy and 
Commerce).
CONGRESSIONAL RECORD, Vol. 147 (2001):
            Dec. 12, considered and passed Senate.
            Dec. 18, considered and passed House.

                                  <all>