[Congressional Bills 108th Congress]
[From the U.S. Government Publishing Office]
[S. 650 Enrolled Bill (ENR)]

        S.650

                       One Hundred Eighth Congress

                                 of the

                        United States of America


                          AT THE FIRST SESSION

          Begun and held at the City of Washington on Tuesday,
           the seventh day of January, two thousand and three


                                 An Act


 
To amend the Federal Food, Drug, and Cosmetic Act to authorize the Food 
 and Drug Administration to require certain research into drugs used in 
                           pediatric patients.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Pediatric Research Equity Act of 
2003''.
SEC. 2. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND BIOLOGICAL PRODUCTS.
    (a) In General.--Subchapter A of chapter V of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is amended by inserting 
after section 505A the following:
``SEC. 505B. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND BIOLOGICAL 
PRODUCTS.
    ``(a) New Drugs and Biological Products.--
        ``(1) In general.--A person that submits an application (or 
    supplement to an application)--
            ``(A) under section 505 for a new active ingredient, new 
        indication, new dosage form, new dosing regimen, or new route 
        of administration; or
            ``(B) under section 351 of the Public Health Service Act 
        (42 U.S.C. 262) for a new active ingredient, new indication, 
        new dosage form, new dosing regimen, or new route of 
        administration;
    shall submit with the application the assessments described in 
    paragraph (2).
        ``(2) Assessments.--
            ``(A) In general.--The assessments referred to in paragraph 
        (1) shall contain data, gathered using appropriate formulations 
        for each age group for which the assessment is required, that 
        are adequate--
                ``(i) to assess the safety and effectiveness of the 
            drug or the biological product for the claimed indications 
            in all relevant pediatric subpopulations; and
                ``(ii) to support dosing and administration for each 
            pediatric subpopulation for which the drug or the 
            biological product is safe and effective.
            ``(B) Similar course of disease or similar effect of drug 
        or biological product.--
                ``(i) In general.--If the course of the disease and the 
            effects of the drug are sufficiently similar in adults and 
            pediatric patients, the Secretary may conclude that 
            pediatric effectiveness can be extrapolated from adequate 
            and well-controlled studies in adults, usually supplemented 
            with other information obtained in pediatric patients, such 
            as pharmacokinetic studies.
                ``(ii) Extrapolation between age groups.--A study may 
            not be needed in each pediatric age group if data from one 
            age group can be extrapolated to another age group.
        ``(3) Deferral.--On the initiative of the Secretary or at the 
    request of the applicant, the Secretary may defer submission of 
    some or all assessments required under paragraph (1) until a 
    specified date after approval of the drug or issuance of the 
    license for a biological product if--
            ``(A) the Secretary finds that--
                ``(i) the drug or biological product is ready for 
            approval for use in adults before pediatric studies are 
            complete;
                ``(ii) pediatric studies should be delayed until 
            additional safety or effectiveness data have been 
            collected; or
                ``(iii) there is another appropriate reason for 
            deferral; and
            ``(B) the applicant submits to the Secretary--
                ``(i) certification of the grounds for deferring the 
            assessments;
                ``(ii) a description of the planned or ongoing studies; 
            and
                ``(iii) evidence that the studies are being conducted 
            or will be conducted with due diligence and at the earliest 
            possible time.
        ``(4) Waivers.--
            ``(A) Full waiver.--On the initiative of the Secretary or 
        at the request of an applicant, the Secretary shall grant a 
        full waiver, as appropriate, of the requirement to submit 
        assessments for a drug or biological product under this 
        subsection if the applicant certifies and the Secretary finds 
        that--
                ``(i) necessary studies are impossible or highly 
            impracticable (because, for example, the number of patients 
            is so small or the patients are geographically dispersed);
                ``(ii) there is evidence strongly suggesting that the 
            drug or biological product would be ineffective or unsafe 
            in all pediatric age groups; or
                ``(iii) the drug or biological product--

                    ``(I) does not represent a meaningful therapeutic 
                benefit over existing therapies for pediatric patients; 
                and
                    ``(II) is not likely to be used in a substantial 
                number of pediatric patients.

            ``(B) Partial waiver.--On the initiative of the Secretary 
        or at the request of an applicant, the Secretary shall grant a 
        partial waiver, as appropriate, of the requirement to submit 
        assessments for a drug or biological product under this 
        subsection with respect to a specific pediatric age group if 
        the applicant certifies and the Secretary finds that--
                ``(i) necessary studies are impossible or highly 
            impracticable (because, for example, the number of patients 
            in that age group is so small or patients in that age group 
            are geographically dispersed);
                ``(ii) there is evidence strongly suggesting that the 
            drug or biological product would be ineffective or unsafe 
            in that age group;
                ``(iii) the drug or biological product--

                    ``(I) does not represent a meaningful therapeutic 
                benefit over existing therapies for pediatric patients 
                in that age group; and
                    ``(II) is not likely to be used by a substantial 
                number of pediatric patients in that age group; or

                ``(iv) the applicant can demonstrate that reasonable 
            attempts to produce a pediatric formulation necessary for 
            that age group have failed.
            ``(C) Pediatric formulation not possible.--If a waiver is 
        granted on the ground that it is not possible to develop a 
        pediatric formulation, the waiver shall cover only the 
        pediatric groups requiring that formulation.
            ``(D) Labeling requirement.--If the Secretary grants a full 
        or partial waiver because there is evidence that a drug or 
        biological product would be ineffective or unsafe in pediatric 
        populations, the information shall be included in the labeling 
        for the drug or biological product.
    ``(b) Marketed Drugs and Biological Products.--
        ``(1) In general.--After providing notice in the form of a 
    letter and an opportunity for written response and a meeting, which 
    may include an advisory committee meeting, the Secretary may (by 
    order in the form of a letter) require the holder of an approved 
    application for a drug under section 505 or the holder of a license 
    for a biological product under section 351 of the Public Health 
    Service Act (42 U.S.C. 262) to submit by a specified date the 
    assessments described in subsection (a)(2) if the Secretary finds 
    that--
            ``(A)(i) the drug or biological product is used for a 
        substantial number of pediatric patients for the labeled 
        indications; and
            ``(ii) the absence of adequate labeling could pose 
        significant risks to pediatric patients; or
            ``(B)(i) there is reason to believe that the drug or 
        biological product would represent a meaningful therapeutic 
        benefit over existing therapies for pediatric patients for one 
        or more of the claimed indications; and
            ``(ii) the absence of adequate labeling could pose 
        significant risks to pediatric patients.
        ``(2) Waivers.--
            ``(A) Full waiver.--At the request of an applicant, the 
        Secretary shall grant a full waiver, as appropriate, of the 
        requirement to submit assessments under this subsection if the 
        applicant certifies and the Secretary finds that--
                ``(i) necessary studies are impossible or highly 
            impracticable (because, for example, the number of patients 
            in that age group is so small or patients in that age group 
            are geographically dispersed); or
                ``(ii) there is evidence strongly suggesting that the 
            drug or biological product would be ineffective or unsafe 
            in all pediatric age groups.
            ``(B) Partial waiver.--At the request of an applicant, the 
        Secretary shall grant a partial waiver, as appropriate, of the 
        requirement to submit assessments under this subsection with 
        respect to a specific pediatric age group if the applicant 
        certifies and the Secretary finds that--
                ``(i) necessary studies are impossible or highly 
            impracticable (because, for example, the number of patients 
            in that age group is so small or patients in that age group 
            are geographically dispersed);
                ``(ii) there is evidence strongly suggesting that the 
            drug or biological product would be ineffective or unsafe 
            in that age group;
                ``(iii)(I) the drug or biological product--

                    ``(aa) does not represent a meaningful therapeutic 
                benefit over existing therapies for pediatric patients 
                in that age group; and
                    ``(bb) is not likely to be used in a substantial 
                number of pediatric patients in that age group; and

                ``(II) the absence of adequate labeling could not pose 
            significant risks to pediatric patients; or
                ``(iv) the applicant can demonstrate that reasonable 
            attempts to produce a pediatric formulation necessary for 
            that age group have failed.
            ``(C) Pediatric formulation not possible.--If a waiver is 
        granted on the ground that it is not possible to develop a 
        pediatric formulation, the waiver shall cover only the 
        pediatric groups requiring that formulation.
            ``(D) Labeling requirement.--If the Secretary grants a full 
        or partial waiver because there is evidence that a drug or 
        biological product would be ineffective or unsafe in pediatric 
        populations, the information shall be included in the labeling 
        for the drug or biological product.
        ``(3) Relationship to other pediatric provisions.--
            ``(A) No assessment without written request.--No assessment 
        may be required under paragraph (1) for a drug subject to an 
        approved application under section 505 unless--
                ``(i) the Secretary has issued a written request for a 
            related pediatric study under section 505A(c) of this Act 
            or section 409I of the Public Health Service Act (42 U.S.C. 
            284m);
                ``(ii)(I) if the request was made under section 
            505A(c)--

                    ``(aa) the recipient of the written request does 
                not agree to the request; or
                    ``(bb) the Secretary does not receive a response as 
                specified under section 505A(d)(4)(A); or

                ``(II) if the request was made under section 409I of 
            the Public Health Service Act (42 U.S.C. 284m)--

                    ``(aa) the recipient of the written request does 
                not agree to the request; or
                    ``(bb) the Secretary does not receive a response as 
                specified under section 409I(c)(2) of that Act; and

                ``(iii)(I) the Secretary certifies under subparagraph 
            (B) that there are insufficient funds under sections 409I 
            and 499 of the Public Health Service Act (42 U.S.C. 284m, 
            290b) to conduct the study; or
                ``(II) the Secretary publishes in the Federal Register 
            a certification that certifies that--

                    ``(aa) no contract or grant has been awarded under 
                section 409I or 499 of the Public Health Service Act 
                (42 U.S.C. 284m, 290b); and
                    ``(bb) not less than 270 days have passed since the 
                date of a certification under subparagraph (B) that 
                there are sufficient funds to conduct the study.

            ``(B) No agreement to request.--Not later than 60 days 
        after determining that no holder will agree to the written 
        request (including a determination that the Secretary has not 
        received a response specified under section 505A(d) of this Act 
        or section 409I of the Public Health Service Act (42 U.S.C. 
        284m), the Secretary shall certify whether the Secretary has 
        sufficient funds to conduct the study under section 409I or 499 
        of the Public Health Service Act (42 U.S.C. 284m, 290b), taking 
        into account the prioritization under section 409I.
    ``(c) Meaningful Therapeutic Benefit.--For the purposes of 
paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I) of subsection (a) and 
paragraphs (1)(B)(i) and (2)(B)(iii)(I)(aa) of subsection (b), a drug 
or biological product shall be considered to represent a meaningful 
therapeutic benefit over existing therapies if the Secretary estimates 
that--
        ``(1) if approved, the drug or biological product would 
    represent a significant improvement in the treatment, diagnosis, or 
    prevention of a disease, compared with marketed products adequately 
    labeled for that use in the relevant pediatric population; or
        ``(2) the drug or biological product is in a class of products 
    or for an indication for which there is a need for additional 
    options.
    ``(d) Submission of Assessments.--If a person fails to submit an 
assessment described in subsection (a)(2), or a request for approval of 
a pediatric formulation described in subsection (a) or (b), in 
accordance with applicable provisions of subsections (a) and (b)--
        ``(1) the drug or biological product that is the subject of the 
    assessment or request may be considered misbranded solely because 
    of that failure and subject to relevant enforcement action (except 
    that the drug or biological product shall not be subject to action 
    under section 303); but
        ``(2) the failure to submit the assessment or request shall not 
    be the basis for a proceeding--
            ``(A) to withdraw approval for a drug under section 505(e); 
        or
            ``(B) to revoke the license for a biological product under 
        section 351 of the Public Health Service Act (42 U.S.C. 262).
    ``(e) Meetings.--Before and during the investigational process for 
a new drug or biological product, the Secretary shall meet at 
appropriate times with the sponsor of the new drug or biological 
product to discuss--
        ``(1) information that the sponsor submits on plans and 
    timelines for pediatric studies; or
        ``(2) any planned request by the sponsor for waiver or deferral 
    of pediatric studies.
    ``(f) Scope of Authority.--Nothing in this section provides to the 
Secretary any authority to require a pediatric assessment of any drug 
or biological product, or any assessment regarding other populations or 
uses of a drug or biological product, other than the pediatric 
assessments described in this section.
    ``(g) Orphan Drugs.--Unless the Secretary requires otherwise by 
regulation, this section does not apply to any drug for an indication 
for which orphan designation has been granted under section 526.
    ``(h) Integration With Other Pediatric Studies.--The authority 
under this section shall remain in effect so long as an application 
subject to this section may be accepted for filing by the Secretary on 
or before the date specified in section 505A(n).''.
    (b) Conforming Amendments.--(1) Section 505(b)(1) of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 355(b)(1)) is amended in the 
second sentence--
        (A) by striking ``and (F)'' and inserting ``(F)''; and
        (B) by striking the period at the end and inserting ``, and (G) 
    any assessments required under section 505B.''.
    (2) Section 505A(h) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355a(h)) is amended--
        (A) in the subsection heading, by striking ``Regulations'' and 
    inserting ``Pediatric Research Requirements''; and
        (B) by striking ``pursuant to regulations promulgated by the 
    Secretary'' and inserting ``by a provision of law (including a 
    regulation) other than this section''.
    (3) Section 351(a)(2) of the Public Health Service Act (42 U.S.C. 
262(a)(2)) is amended--
        (A) by redesignating subparagraph (B) as subparagraph (C); and
        (B) by inserting after subparagraph (A) the following:
            ``(B) Pediatric studies.--A person that submits an 
        application for a license under this paragraph shall submit to 
        the Secretary as part of the application any assessments 
        required under section 505B of the Federal Food, Drug, and 
        Cosmetic Act.''.

SEC. 3. TECHNICAL AND CONFORMING AMENDMENTS.

    (a) Abbreviated New Drug Application.--Section 505A of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is amended in 
subparagraphs (A) and (B) of subsection (b)(2) and subparagraphs (A) 
and (B) of subsection (c)(2) by striking ``505(j)(4)(B)'' and inserting 
``505(j)(5)(B)''.
    (b) Pediatric Advisory Committee.--(1) Section 505A(i)(2) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a(i)(2)) is amended 
by striking ``Advisory Subcommittee of the Anti-Infective Drugs'' each 
place it appears.
    (2) Section 14 of the Best Pharmaceuticals for Children Act (42 
U.S.C. 284m note; Public Law 107-109) is amended--
        (A) in the section heading, by striking ``pharmacology'';
        (B) in subsection (a), by striking ``(42 U.S.C. 217a),'' and 
    inserting (42 U.S.C. 217a) or other appropriate authority,'';
        (C) in subsection (b)--
            (i) in paragraph (1), by striking ``and in consultation 
        with the Director of the National Institutes of Health''; and
            (ii) in paragraph (2), by striking ``and 505A'' and 
        inserting ``505A, and 505B''; and
        (D) by striking ``pharmacology'' each place it appears and 
    inserting ``therapeutics''.
    (3) Section 15(a)(2)(A) of the Best Pharmaceuticals for Children 
Act (115 Stat. 1419) is amended by striking ``Pharmacology''.
    (4) Section 16(1)(C) of the Best Pharmaceuticals for Children Act 
(21 U.S.C. 355a note; Public Law 107-109) is amended by striking 
``Advisory Subcommittee of the Anti-Infective Drugs''.
    (5) Section 17(b)(1) of the Best Pharmaceuticals for Children Act 
(21 U.S.C. 355b(b)(1)) is amended in the second sentence by striking 
``Advisory Subcommittee of the Anti-Infective Drugs''.
    (6) Paragraphs (8), (9), and (11) of section 409I(c) of the Public 
Health Service Act (42 U.S.C. 284m(c)) are amended by striking 
``Advisory Subcommittee of the Anti-Infective Drugs'' each place it 
appears.

SEC. 4. EFFECTIVE DATE.

    (a) In General.--Subject to subsection (b), this Act and the 
amendments made by this Act take effect on the date of enactment of 
this Act.
    (b) Applicability to New Drugs and Biological Products.--
        (1) In general.--Subsection (a) of section 505B of the Federal 
    Food, Drug, and Cosmetic Act (as added by section 2) shall apply to 
    an application described in paragraph (1) of that subsection 
    submitted to the Secretary of Health and Human Services on or after 
    April 1, 1999.
        (2) Waivers and deferrals.--
            (A) Waiver or deferral granted.--If, with respect to an 
        application submitted to the Secretary of Health and Human 
        Services between April 1, 1999, and the date of enactment of 
        this Act, a waiver or deferral of pediatric assessments was 
        granted under regulations of the Secretary then in effect, the 
        waiver or deferral shall be a waiver or deferral under 
        subsection (a) of section 505B of the Federal Food, Drug, and 
        Cosmetic Act, except that any date specified in such a deferral 
        shall be extended by the number of days that is equal to the 
        number of days between October 17, 2002, and the date of 
        enactment of this Act.
            (B) Waiver and deferral not granted.--If, with respect to 
        an application submitted to the Secretary of Health and Human 
        Services between April 1, 1999, and the date of enactment of 
        this Act, neither a waiver nor deferral of pediatric 
        assessments was granted under regulations of the Secretary then 
        in effect, the person that submitted the application shall be 
        required to submit assessments under subsection (a)(2) of 
        section 505B of the Federal Food, Drug, and Cosmetic Act on the 
        date that is the later of--
                (i) the date that is 1 year after the date of enactment 
            of this Act; or
                (ii) such date as the Secretary may specify under 
            subsection (a)(3) of that section;
        unless the Secretary grants a waiver under subsection (a)(4) of 
        that section.
    (c) No Limitation of Authority.--Neither the lack of guidance or 
regulations to implement this Act or the amendments made by this Act 
nor the pendency of the process for issuing guidance or regulations 
shall limit the authority of the Secretary of Health and Human Services 
under, or defer any requirement under, this Act or those amendments.

                               Speaker of the House of Representatives.

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