[108th Congress Public Law 155]
[From the U.S. Government Printing Office]


[DOCID: f:publ155.108]

[[Page 1935]]

                  PEDIATRIC RESEARCH EQUITY ACT OF 2003

[[Page 117 STAT. 1936]]

Public Law 108-155
108th Congress

                                 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. <<NOTE: Dec. 3, 2003 -  [S. 650]>> 

    Be it enacted by the Senate and House of <<NOTE: Pediatric Research 
Equity Act of 2003.>> Representatives of the United States of America in 
Congress assembled,

SECTION 1. <<NOTE: 21 USC 301 note.>> 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 <<NOTE: 21 USC 355c.>> 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

[[Page 117 STAT. 1937]]

                      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

[[Page 117 STAT. 1938]]

                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

[[Page 117 STAT. 1939]]

                      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

[[Page 117 STAT. 1940]]

                                    ``(bb) the Secretary does not 
                                receive a response as specified under 
                                section 409I(c)(2) of that Act; and
                          ``(iii)(I) <<NOTE: Certification.>> 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 <<NOTE: Deadline. Certification.>> 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).

[[Page 117 STAT. 1941]]

    ``(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--

[[Page 117 STAT. 1942]]

            (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. <<NOTE: 21 USC 355c note.>> 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--

[[Page 117 STAT. 1943]]

                          (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.

    Approved December 3, 2003.

LEGISLATIVE HISTORY--S. 650:
---------------------------------------------------------------------------

SENATE REPORTS: No. 108-84 (Comm. on Health, Education, Labor, and 
Pensions).
CONGRESSIONAL RECORD, Vol. 149 (2003):
            July 21, considered and passed Senate.
            Nov. 19, considered and passed House.

                                  <all>
