General Clinical Pharmacology Considerations for Pediatric Studies of Drugs, Including Biological Products

Description

This guidance assists sponsors of investigational new drug applications (INDs) and applicants of new drug applications (NDAs) under section 505 of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), biologics license applications (BLAs) under section 351(a) of the Public Health Service Act (PHS Act), and supplements to such applications who are planning to conduct clinical studies in pediatric populations. In addition, this guidance assists clinical investigators in the design and planning of, and Institutional Review Boards (IRBs) in the assessment of, clinical studies in pediatric populations.

Scope & Applicability

Regulatory Context

• 21CFR50.53§ 50.53 Clinical investigations involving greater than minimal risk and no prospect of direct benefit to individual subjects, but likely to yield generalizable knowledge about the subjects' disorder or condition.

  Any clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter in which more than minimal risk to children is presented by an intervention or procedure that does not hold out the prospect of direct benefit for the individual subject, or by a monitoring procedure that is Read full regulation →

• 21CFR320.21§ 320.21 Requirements for submission of bioavailability and bioequivalence data.

  (a) Any person submitting a full new drug application to the Food and Drug Administration (FDA) shall include in the application either:Read full regulation →

• 21CFR50.52§ 50.52 Clinical investigations involving greater than minimal risk but presenting the prospect of direct benefit to individual subjects.

  Any clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter in which more than minimal risk to children is presented by an intervention or procedure that holds out the prospect of direct benefit for the individual subject, or by a monitoring procedure that is likely tRead full regulation →

• 21CFR201.57§ 201.57 Specific requirements on content and format of labeling for human prescription drug and biological products described in § 201.56(b)(1) .

  The requirements in this section apply only to prescription drug products described in § 201.56(b)(1) and must be implemented according to the schedule specified in § 201.56(c) , except for the requirement in paragraph (c)(18) of this section to reprint any FDA-approved patient labeling at the end oRead full regulation →

• 21CFR50.51§ 50.51 Clinical investigations not involving greater than minimal risk.

  Any clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter in which no greater than minimal risk to children is presented may involve children as subjects only if the IRB finds that:Read full regulation →

• 21CFR50.54§ 50.54 Clinical investigations not otherwise approvable that present an opportunity to understand, prevent, or alleviate a serious problem affecting the health or welfare of children.

  If an IRB does not believe that a clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter and involving children as subjects meets the requirements of § 50.51 , § 50.52 , or § 50.53 , the clinical investigation may proceed only if:Read full regulation →

• 21CFR312.42§ 312.42 Clinical holds and requests for modification.

  (a) General. A clinical hold is an order issued by FDA to the sponsor to delay a proposed clinical investigation or to suspend an ongoing investigation. The clinical hold order may apply to one or more of the investigations covered by an IND. When a proposed study is placed on clinical hold, subjectRead full regulation →

• 21CFR314.55§ 314.55 Pediatric use information.

  (a) Required assessment. Except as provided in paragraphs (b) , (c) , and (d) of this section, each application for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration shall contain data that are adequate to assess the safety and effectivenessRead full regulation →

• Clinical Investigator

  Shirish M. Gadgeel, M.D.

  2025-09-09

• Clinical Investigator

  Mark J. Savant, M.D

  2025-07-15

• Bioresearch Monitoring Program/Institutional Review Board (IRB)

  United Health Products, Inc.

  2025-05-20

• Clinical Investigator

  Americo F. Padilla, M.D.

  2025-03-25

• Institutional Review Board (IRB)

  Armstrong County Memorial Hospital

  2024-11-05

• Clinical Investigator

  Namita A. Goyal, M.D.

  2024-10-22

• Institutional Review Board (IRB)

  Louisiana State University Health Science Center IRB

  2024-10-08

• Bioresearch Monitoring Program/IRB

  Massachusetts Institute of Technology MIT

  2024-07-16

• Clinical Investigator (Sponsor)

  Angela D. Ritter, M.D.

  2024-06-18

• Institutional Review Board (IRB)

  New York State Psychiatric Institute IRB

  2024-03-26

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