[Congressional Bills 107th Congress]
[From the U.S. Government Publishing Office]
[S. 838 Reported in Senate (RS)]






                                                       Calendar No. 184
107th CONGRESS
  1st Session
                                 S. 838

                          [Report No. 107-79]

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


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                              May 7, 2001

    Mr. Dodd (for himself, Mr. DeWine, Ms. Mikulski, Mr. Frist, Mr. 
  Jeffords, Mr. Bond, Ms. Collins, Ms. Landrieu, Mrs. Feinstein, Mr. 
   Corzine, Mr. Carper, Mrs. Murray, Mr. Reed, Mr. Bingaman, and Mr. 
   Roberts) introduced the following bill; which was read twice and 
  referred to the Committee on Health, Education, Labor, and Pensions

                            October 4, 2001

               Reported by Mr. Kennedy, with an amendment
 [Strike out all after the enacting clause and insert the part printed 
                               in italic]

_______________________________________________________________________

                                 A BILL


 
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,

<DELETED>SECTION 1. SHORT TITLE.</DELETED>

<DELETED>    This Act may be cited as the ``Best Pharmaceuticals for 
Children Act''.</DELETED>

<DELETED>SEC. 2. PEDIATRIC STUDIES OF ALREADY-MARKETED DRUGS.</DELETED>

<DELETED>    Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) is amended--</DELETED>
        <DELETED>    (1) by striking subsection (b); and</DELETED>
        <DELETED>    (2) in subsection (c)--</DELETED>
                <DELETED>    (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</DELETED>
                <DELETED>    (B) by striking ``concerning a drug 
                identified in the list described in subsection 
                (b)''.</DELETED>

<DELETED>SEC. 3. RESEARCH FUND FOR THE STUDY OF OFF-PATENT 
              DRUGS.</DELETED>

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

<DELETED>``SEC. 409I. PROGRAM FOR PEDIATRIC STUDIES OF OFF-PATENT 
              DRUGS.</DELETED>

<DELETED>    ``(a) List of Off-Patent Drugs for Which Pediatric Studies 
are Needed.--</DELETED>
        <DELETED>    ``(1) In general.--Not later than 1 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 (including United States 
        Pharmacopoeia), shall develop, prioritize, and publish a list 
        of approved drugs for which--</DELETED>
                <DELETED>    ``(A) there is no patent or market 
                exclusivity protection; and</DELETED>
                <DELETED>    ``(B) additional studies are needed to 
                assess the safety and effectiveness of the use of the 
                drug in the pediatric population.</DELETED>
        <DELETED>    ``(2) Consideration of available information.--In 
        developing the list under paragraph (1), the Secretary shall 
        consider, for each drug on the list--</DELETED>
                <DELETED>    ``(A) the availability of information 
                concerning the safe and effective use of the drug in 
                the pediatric population;</DELETED>
                <DELETED>    ``(B) whether additional information is 
                needed; and</DELETED>
                <DELETED>    ``(C) whether new pediatric studies 
                concerning the drug may produce health benefits in the 
                pediatric population.</DELETED>
<DELETED>    ``(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).</DELETED>
<DELETED>    ``(c) Process for Contracts and Labeling Changes.--
</DELETED>
        <DELETED>    ``(1) Written request to holders of approved 
        applications for off-patent drugs.--</DELETED>
                <DELETED>    ``(A) In general.--The Commissioner of 
                Food and Drugs, in consultation with the Director of 
                National Institutes of Health, may issue a written 
                request for pediatric studies concerning a drug 
                identified in the list described in subsection (a) to 
                all holders of an approved application for the drug 
                under section 505 of the Federal Food, Drug, and 
                Cosmetic Act. Such a request shall be made in 
                accordance with section 505A of the Federal Food, Drug, 
                and Cosmetic Act.</DELETED>
                <DELETED>    ``(B) Publication of request.--If the 
                Commissioner of Food and Drugs does not receive a 
                response to a written request issued under subparagraph 
                (A) within 30 days of the date on which a request was 
                issued, the Secretary, acting through the Director of 
                National Institutes of Health, shall publish a request 
                for contract proposals to conduct the pediatric studies 
                described in the written request.</DELETED>
        <DELETED>    ``(2) 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.</DELETED>
        <DELETED>    ``(3) Reporting of studies.--</DELETED>
                <DELETED>    ``(A) Upon 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 National Institutes of 
                Health and the Commissioner of Food and Drugs. The 
                report shall include all data generated in connection 
                with the study.</DELETED>
                <DELETED>    ``(B) Availability of reports.--Each 
                report submitted under subparagraph (A) shall be 
                considered to be in the public domain, 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 the drug.</DELETED>
                <DELETED>    ``(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 
                (4).</DELETED>
        <DELETED>    ``(4) Request for labeling changes.--During the 
        180-day period after the date on which a report is submitted 
        under paragraph (3)(A), the Commissioner of Food and Drugs 
        shall--</DELETED>
                <DELETED>    ``(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; 
                and</DELETED>
                <DELETED>    ``(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</DELETED>
                <DELETED>    ``(C)(i) place in the public docket file a 
                copy of the report and of any requested labeling 
                changes; and</DELETED>
                <DELETED>    ``(ii) publish in the Federal Register a 
                summary of the report and a copy of any requested 
                labeling changes.</DELETED>
        <DELETED>    ``(5) Dispute resolution.--If, not later than the 
        end of the 180-day period specified in paragraph (4), 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--</DELETED>
                <DELETED>    ``(A) the Commissioner of Food and Drugs 
                shall immediately refer the request to the Pediatric 
                Advisory Subcommittee of the Anti-Infective Drugs 
                Advisory Committee; and</DELETED>
                <DELETED>    ``(B) not later than 60 days after 
                receiving the referral, the Subcommittee shall--
                </DELETED>
                        <DELETED>    ``(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</DELETED>
                        <DELETED>    ``(ii) make a recommendation to 
                        the Commissioner of Food and Drugs as to 
                        appropriate labeling changes, if any.</DELETED>
        <DELETED>    ``(6) FDA determination.--Not later than 30 days 
        after receiving a recommendation from the Subcommittee under 
        paragraph (5)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.</DELETED>
        <DELETED>    ``(7) 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 (6), 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.</DELETED>
<DELETED>    ``(d) Authorization of Appropriations.--</DELETED>
        <DELETED>    ``(1) In general.--There are authorized to be 
        appropriated to carry out this section--</DELETED>
                <DELETED>    ``(A) $200,000,000 for fiscal year 2002; 
                and</DELETED>
                <DELETED>    ``(B) such sums as are necessary for each 
                of the 5 succeeding fiscal years.</DELETED>
        <DELETED>    ``(2) Availability.--Any amount appropriated under 
        paragraph (1) shall remain available to carry out this section 
        until expended.''.</DELETED>

<DELETED>SEC. 4. TIMELY LABELING CHANGES FOR DRUGS GRANTED EXCLUSIVITY; 
              DRUG FEES.</DELETED>

<DELETED>    (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--</DELETED>
        <DELETED>    (1) by striking subparagraph (F); and</DELETED>
        <DELETED>    (2) by redesignating subparagraph (G) as 
        subparagraph (F).</DELETED>
<DELETED>    (b) Labeling Changes.--Section 505A of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355a) is amended by adding at the end 
the following:</DELETED>
<DELETED>    ``(l) Labeling Supplements.--</DELETED>
        <DELETED>    ``(1) Priority status for pediatric supplements.--
        Any supplement to a human drug application submitted under this 
        section--</DELETED>
                <DELETED>    ``(A) shall be considered to be a priority 
                supplement; and</DELETED>
                <DELETED>    ``(B) shall be subject to the performance 
                goals established by the Commissioner for priority 
                drugs.</DELETED>
        <DELETED>    ``(2) Dispute resolution.--If the Commissioner 
        determines that a supplemental application submitted under this 
        section is approvable and that the only open issue for final 
        action on the supplement 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--</DELETED>
                <DELETED>    ``(A) not later than 180 days after the 
                date of submission of the supplemental application--
                </DELETED>
                        <DELETED>    ``(i) the Commissioner shall 
                        request that the sponsor of the application 
                        make any labeling change that the Commissioner 
                        determines to be appropriate; and</DELETED>
                        <DELETED>    ``(ii) if the sponsor of the 
                        application does not agree to make a labeling 
                        change requested by the Commissioner by that 
                        date, the Commissioner shall immediately refer 
                        the matter to the Pediatric Advisory 
                        Subcommittee of the Anti-Infective Drugs 
                        Advisory Committee;</DELETED>
                <DELETED>    ``(B) not later than 60 days after 
                receiving the referral, the Pediatric Advisory 
                Subcommittee of the Anti-Infective Drugs Advisory 
                Committee shall--</DELETED>
                        <DELETED>    ``(i) review the pediatric study 
                        reports; and</DELETED>
                        <DELETED>    ``(ii) make a recommendation to 
                        the Commissioner concerning appropriate 
                        labeling changes, if any;</DELETED>
                <DELETED>    ``(C) 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; 
                and</DELETED>
                <DELETED>    ``(D) if the sponsor of the application, 
                within 30 days after receiving a request under 
                subparagraph (D), 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.''.</DELETED>

<DELETED>SEC. 5. OFFICE OF PEDIATRIC THERAPEUTICS.</DELETED>

<DELETED>    (a) Establishment.--The Secretary of Health and Human 
Services shall establish an Office of Pediatric Therapeutics within the 
Office of the Commissioner of Food and Drugs.</DELETED>
<DELETED>    (b) Duties.--The Office of Pediatric Therapeutics shall be 
responsible for oversight and coordination 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.</DELETED>
<DELETED>    (c) Staff.--The staff of the Office of Pediatric 
Therapeutics shall include--</DELETED>
        <DELETED>    (1) 1 or more individuals with expertise 
        concerning ethical issues presented by the conduct of clinical 
        research in the pediatric population; and</DELETED>
        <DELETED>    (2) 1 or more individuals with expertise in 
        pediatrics who shall consult with all components of the Food 
        and Drug Administration concerning activities described in 
        subsection (b).</DELETED>

<DELETED>SEC. 6. NEONATES.</DELETED>

<DELETED>    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''.</DELETED>

<DELETED>SEC. 7. SUNSET.</DELETED>

<DELETED>    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:</DELETED>
<DELETED>    ``(j) Sunset.--A drug may not receive any 6-month period 
under subsection (a) or (c) unless--</DELETED>
        <DELETED>    ``(1) on or before October 1, 2007, the Secretary 
        makes a written request for pediatric studies of the 
        drug;</DELETED>
        <DELETED>    ``(2) on or before October 1, 2007, an application 
        for the drug is submitted under section 505(b)(1); 
        and</DELETED>
        <DELETED>    ``(3) all requirements of this section are 
        met.''.</DELETED>

<DELETED>SEC. 8. DISSEMINATION OF PEDIATRIC INFORMATION.</DELETED>

<DELETED>    Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C 355a) (as amended by section 4(b)) is amended by adding at 
the end the following:</DELETED>
<DELETED>    ``(m) Dissemination of Pediatric Information.--</DELETED>
        <DELETED>    ``(1) In general.--Not later than 180 days after 
        the date of submission of a supplemental application 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.</DELETED>
        <DELETED>    ``(2) Effect of subsection.--Nothing in this 
        subsection alters or amends in any way section 552 of title 5 
        or section 1905 of title 18, United States Code.''.</DELETED>

<DELETED>SEC. 9. TECHNICAL AND CONFORMING AMENDMENTS.</DELETED>

<DELETED>    Section 505A of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355a) (as amended by sections 2(1), 4(b), 7, and 8) is 
amended--</DELETED>
        <DELETED>    (1) by redesignating subsections (a), (g), (h), 
        (i), (j), (l), and (m) as subsections (b), (a), (g), (h), (l), 
        (i), and (j), respectively;</DELETED>
        <DELETED>    (2) by moving the subsections so as to appear in 
        alphabetical order; and</DELETED>
        <DELETED>    (3) in paragraphs (1), (2), and (3) of subsection 
        (d) and subsections (e), (g) (as redesignated by paragraph 
        (1)), and (l) (as redesignated by paragraph (1)), by striking 
        ``subsection (a) or (c)'' and inserting ``subsection (b) or 
        (c)''.</DELETED>

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 LACKING EXCLUSIVITY.

    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 LACKING 
              EXCLUSIVITY.

    ``(a) List of Drugs Lacking Exclusivity for Which Pediatric Studies 
are Needed.--
            ``(1) In general.--Not later than 1 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)); or
                    ``(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.); and
                    ``(B) 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 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.--
                    ``(A) In general.--The Commissioner of Food and 
                Drugs, in consultation with the Director of 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) to 
                all holders of an approved application for the drug 
                under section 505 of the Federal Food, Drug, and 
                Cosmetic Act. Such a request shall be made in 
                accordance with section 505A of the Federal Food, Drug, 
                and Cosmetic Act.
                    ``(B) Publication of request.--If the Commissioner 
                of Food and Drugs does not receive a response to a 
                written request issued under subparagraph (A) within 30 
                days of the date on which a request was issued, the 
                Secretary, acting through the Director of 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.
                    ``(C) Disqualification.--A holder that receives a 
                first right of refusal shall not be entitled to respond 
to a request for contract proposals under subparagraph (B).
                    ``(D) 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 subparagraph (A).
            ``(2) 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.
            ``(3) Reporting of studies.--
                    ``(A) Upon 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 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, 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 (4).
            ``(4) Request for labeling changes.--During the 180-day 
        period after the date on which a report is submitted under 
        paragraph (3)(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; and
                    ``(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.
            ``(5) Dispute resolution.--If, not later than the end of 
        the 180-day period specified in paragraph (4), 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--
                    ``(A) the Commissioner of Food and Drugs shall 
                immediately refer the request to the Pediatric Advisory 
                Subcommittee of the Anti-Infective Drugs Advisory 
                Committee; and
                    ``(B) not later than 90 days after receiving the 
                referral, the Subcommittee 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.
            ``(6) FDA determination.--Not later than 30 days after 
        receiving a recommendation from the Subcommittee under 
        paragraph (5)(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.
            ``(7) 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 (6), 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.
            ``(8) 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 5 
                succeeding fiscal years.
            ``(2) Availability.--Any amount appropriated under 
        paragraph (1) shall remain available to carry out this section 
        until expended.''.

SEC. 4. 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.--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--
                    ``(A) 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 by that date, the 
                        Commissioner shall immediately refer the matter 
                        to the Pediatric Advisory Subcommittee of the 
                        Anti-Infective Drugs Advisory Committee;
                    ``(B) not later than 90 days after receiving the 
                referral, 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) 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; and
                    ``(D) 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.''.

SEC. 5. OFFICE OF PEDIATRIC THERAPEUTICS.

    (a) Establishment.--The Secretary of Health and Human Services 
shall establish an Office of Pediatric Therapeutics within the Office 
of the Commissioner of Food and Drugs.
    (b) Duties.--The Office of Pediatric Therapeutics shall be 
responsible for oversight and coordination 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 
include--
            (1) employees of the Department of Health and Human 
        Services who, as of the date of enactment of this Act, exercise 
        responsibilities relating to pediatric therapeutics;
            (2) 1 or more additional individuals with expertise 
        concerning ethical issues presented by the conduct of clinical 
        research in the pediatric population; and
            (3) 1 or more additional individuals with expertise in 
        pediatrics who shall consult and collaborate with all 
        components of the Food and Drug Administration concerning 
        activities described in subsection (b).

SEC. 6. 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. 7. 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 approvable 
        application for the drug is submitted under section 505(b)(1); 
        and
            ``(3) all requirements of this section are met.''.

SEC. 8. DISSEMINATION OF PEDIATRIC INFORMATION.

    Section 505A of the Federal Food, Drug, and Cosmetic Act (21 U.S.C 
355a) (as amended by section 4(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 in any way section 552 of title 5 or section 
        1905 of title 18, United States Code.''.

SEC. 9. CLARIFICATION OF INTERACTION OF MARKET EXCLUSIVITY UNDER 
              SECTION 505A OF THE FEDERAL FOOD, DRUG, AND COSMETIC ACT 
              AND MARKET 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 8) 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).--
            ``(1) In general.--If a 180-day period under section 
        505(j)(5)(B)(iv) overlaps with a 6-month extension 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--
                    ``(A) if the 180-day period would, but for this 
                subsection, expire after the 6-month extension, by the 
                number of days of the overlap; or
                    ``(B) if the 180-day period would, but for this 
                subsection, expire during the 6-month extension, by 6 
                months.
            ``(2) Effect of subsection.--Under no circumstances shall 
        application of this section result in an applicant for approval 
        of a drug under section 505(j) being enabled to commercially 
        market the drug to the exclusion of a subsequent applicant for 
        approval of a drug under section 505(j) for more than 180 
        days.''.

SEC. 10. 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 appropriate committees of 
        Congress, by not later than 2 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. 11. 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), 4(b)(2), 8, and 9) 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), and (n) as subsections (b), (a), (g), (h), (m), 
        (l), (i), (j), and (k), 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 (1)), by striking ``subsection (a) or (c)'' and 
        inserting ``subsection (b) or (c)''; and
            (5) in subsection (g) (as redesignated by paragraph (1)), 
        by striking ``subsection (a) or (b)'' and inserting 
        ``subsection (b) or (c)''.




                                                       Calendar No. 184

107th CONGRESS

  1st Session

                                 S. 838

                          [Report No. 107-79]

_______________________________________________________________________

                                 A BILL

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

_______________________________________________________________________

                            October 4, 2001

                       Reported with an amendment