Description
The number of medicinal products currently labeled for pediatric use is limited. This guidance is intended to encourage and facilitate timely pediatric medicinal product development internationally. The guidance provides an outline of critical issues in pediatric drug development and approaches to the safe, efficient, and ethical study of medicinal products in the pediatric population.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
2Subject of stability testing guidance
Includes pharmaceuticals, biologics, vaccines, cell or gene therapy products
Stakeholders
10Early discussion is recommended regarding safety dataset size.; Entities with whom the adequacy of RWD and extrapolation plans should be discussed.; early discussion useful when proposed endpoints differ; Entities that should agree upon success criteria in advance.
Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining
Independent Ethics Committee listed in eCTD documentation
Institutional Review Board providing study approvals
Involved in the recruitment process; Person responsible for providing consent for pediatric participants
Shared responsibility for pediatric drug development
Bodies responsible for evaluating trial risks and benefits for pregnant participants.; IRBs experienced in this population may advise on compensation.
Review and define maximum blood volume for 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
Regulatory Context
Document Types
4Documentation and development of dosage forms for children
Written form signed by pediatric participants of appropriate intellectual maturity
Process by which a subject voluntarily confirms his or her willingness to participate in a particular trial; Integral feature of the ethical conduct of a trial
Submissions standardized by ICH guidelines
Attributes
3Factor related to neurodevelopmental vulnerability
Used to assess pubertal development
Factor suggesting urgent initiation of pediatric studies
Technical Details
Substances
3Therapy for respiratory distress syndrome
Used to minimize discomfort during IV catheter placement
Fatal Benzyl Alcohol Poisoning in Neonatal Intensive Care Units
Testing Methods
7PK data used for dose selection
PD data used for dose selection
AI used to reduce the number of animal-based PK studies.
Used to confirm effectiveness when blood levels are unclear
Nonclinical safety data
M&S method included in MIDD; Model coding scripts for base and final models
Practical approach for obtaining pharmacokinetic data in neonatal studies due to blood volume limits.
Processes
3Development of drug forms suitable for pediatric use
Procedure for blood sampling that should be minimized to reduce distress
Evaluation of human data after drug approval
Clinical Concepts
10Frequently underrepresented in trials.
Reporting adverse events when engaging with patients.; changes may be related to benefits, tolerability, and/or unintended effects
Condition unique to neonates
Unique neonatal disease state
Disease predominantly affecting preterm infants
Sub-population within the neonatal period definition
Pediatric age category from 0 to 27 days
Pediatric age category from 28 days to 23 months
Subject population requiring additional safeguards in research
Pediatric population that may be included in adult clinical trials.; inclusion in adult clinical trials recommended when disease is similar
Identified Hazards
Hazards
2Basis for considering juvenile animal studies
Known toxicity risks that investigators must be aware of
Standards & References
Specifications
1Basis for dosing recommendations
ICH References (10)
Clinical Investigation of Medicinal Products in the Pediatric Population.
Referenced for basic considerations in safety data collection.
document remedial actions in the clinical trial report; Structure and Content of Clinical Study Reports standards.
Dose-Response Information to Support Drug Registration
Ethnic Factors in the Accessibility of Foreign Clinical Data.
Referenced for safety data collection and adverse event reporting plans.
General Considerations for Clinical Studies
Statistical Principles for Clinical Trials; Discourages deterministic procedures due to high risk of bias; Notes that the use of Bayesian methods in clinical trials may be considered.
Choice of Control Group in Clinical Trials.
Nonclinical Safety Studies for the Conduct of Human Clinical Trials
Related MFDS Guidelines
Korean regulatory guidelines covering similar topics
See Also (8)
- E11A Pediatric Extrapolation
- M15 General Principles for Model-Informed Drug Development
- Clinical Pharmacology Data to Support a Demonstration of Biosimilarity to a Reference Product
- Q6A Specifications: Test Procedures and Acceptance Criteria for New Drug Substances and New Drug Products: Chemical Substances
- Rare Diseases: Considerations for the Development of Drugs and Biological Products
- Q3C Impurities: Residual Solvents_2011
- Providing Regulatory Submissions in Electronic Format --Content of the Risk Evaluation and Mitigation Strategies Document Using Structured Product Labeling: Guidance for Industry
- Q4B Annex 5: Disintegration Test General Chapter