US CODE: Title 21,355c. Research into pediatric uses for drugs and biological products

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TITLE 21 > CHAPTER 9 > SUBCHAPTER V > Part A > § 355c

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§ 355c. Research into pediatric uses for drugs and biological products

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(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.

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.

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

(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.

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.

[1] So in original. A closing parenthesis probably should precede the comma.

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