[Congressional Bills 108th Congress]
[From the U.S. Government Publishing Office]
[S. 650 Referred in House (RFH)]
108th CONGRESS
1st Session
S. 650
_______________________________________________________________________
IN THE HOUSE OF REPRESENTATIVES
July 24, 2003
Referred to the Committee on Energy and Commerce
_______________________________________________________________________
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 1 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 1 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.
Passed the Senate July 23 (legislative day, July 21), 2003.
Attest:
EMILY J. REYNOLDS,
Secretary.