Description
Pediatric drug development has evolved since the original guidance E11 Clinical Investigation of Medicinal Products in the Pediatric Population (ICH E11 (2000)) published, requiring consideration of regulatory and scientific advances relevant to pediatric populations. This addendum does not alter the scope of the original guidance. ICH E11 (2000), including this addendum (R1); is not intended to be comprehensive; other ICH guidances, as well as documents from regulatory authorities worldwide, the World Health Organization (WHO), and pediatric societies, provide additional detail. The purpose of the addendum is to complement and provide clarification and current regulatory perspective on topics in pediatric drug development.
Scope & Applicability
Product Classes
2Subject of stability testing guidance
Age-appropriate dosage forms developed for safe and accurate use in children.
Stakeholders
8Individuals providing information for medical product development; Participants in facilitated discussions and observational studies
Governs top dose in clinical studies
Responsible for submission and communication oversight; Safeguard the rights, safety and well-being of trial participants; Reviewing trial conduct and records
Provides consent or permission for pediatric participation
Informed consent from guardian and/or child participant.
Obtaining child assent appropriate to the child's capability.
Input from stakeholders including clinicians.
Input from stakeholders including experts from academia.
Regulatory Context
Attributes
4Characteristic of orally administered dosage forms for children
Chronologic age alone may not serve as an adequate categorical determinant.
Factors to be considered in determining subgroups.
Biological property that may be altered by manufacturing changes; safety narrative should address bioavailability of the ingredients
Related CFR Sections (5)
- 21CFR56.111§ 56.111 Criteria for IRB approval of research.
(a) In order to approve research covered by these regulations the IRB shall determine that all of the following requirements are satisfied:Read 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 →
- 21CFR50.53§ 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 →
- 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 →
- 21CFR50.3§ 50.3 Definitions.
As used in this part:Read full regulation →
Related Warning Letters (1)
- 2025-03-25
Clinical Investigator
Americo F. Padilla, M.D.
See Also (8)
- Recruiting Study Subjects: Guidance for Institutional Review Boards and Clinical Investigators (Status: Final)
- Non-local IRB Review : Guidance for Institutional Review Boards and Clinical Investigators (Status: Final)
- CHAPTER 48 - 7348.809 Bioresearch Monitoring (Status: Final)
- Changes or Modifications During the Conduct of a Clinical Investigation; Final Guidance for Industry and CDRH Staff (Status: Final)
- Significant Risk and Nonsignificant Risk Medical Device Studies: Guidance For IRBs, Clinical Investigators, and Sponsors (Status: Final)
- IRB Continuing Review After Clinical Investigation Approval: Guidance for IRBs, Clinical Investigators, and Sponsors (Status: Final)
- Guidance for Industry: Acute Bacterial Otitis Media: Developing Drugs for Treatment (Status: Final)
- Exception from Informed Consent Requirements for Emergency Research: Guidance for Institutional Review Boards, Clinical Investigators, and Sponsors (Status: Final)