21 U.S.C. § 355c : US Code - Section 355C: Research into pediatric uses for drugs and biological products

Search 21 U.S.C. § 355c : US Code - Section 355C: 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 355 of this title for a new active
ingredient, new indication, new dosage form, new dosing
regimen, or new route of administration; or
(B) under section 262 of title 42 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 355 of this title or the holder of a
license for a biological product under section 262 of title 42 to
submit by a specified date the assessments described in
subsection (a)(2) of this section 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 355 of this
title unless -
(i) the Secretary has issued a written request for a
related pediatric study under section 355a(c) of this title
or section 284m of title 42;
(ii)(I) if the request was made under section 355a(c) of
this title -
(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 355a(d)(4)(A) of this title; or
(II) if the request was made under section 284m of title 42
-
(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 284m(c)(2) of title 42; and
(iii)(I) the Secretary certifies under subparagraph (B)
that there are insufficient funds under sections 284m and
290b of title 42 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
284m or 290b of title 42; 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 355a(d) of this title or section 284m of title 42,(!1)
the Secretary shall certify whether the Secretary has
sufficient funds to conduct the study under section 284m or
290b of title 42, taking into account the prioritization under
section 284m of title 42.
(c) Meaningful therapeutic benefit
For the purposes of paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I)
of subsection (a) of this section and paragraphs (1)(B)(i) and
(2)(B)(iii)(I)(aa) of subsection (b) of this section, 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) of this section, or a request for approval of a pediatric
formulation described in subsection (a) or (b) of this section, in
accordance with applicable provisions of subsections (a) and (b) of
this section -
(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 333 of this title); 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 355(e) of
this title; or
(B) to revoke the license for a biological product under
section 262 of title 42.
(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 360bb of this
title.
(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
355a(n) of this title.