Description
The purpose of this guidance is to provide a framework for considering whether and what type of long-term neurologic, sensory and developmental evaluations could be useful to support a determination of safety of a drug, biological product, or device (referred to as ‘medical product’ in this guidance) for use in neonates, and if so, which domains of neurodevelopment may be most applicable.
Key Topics
Terms and concepts identified from this document
Scope & Applicability
Product Classes
4Defined in section 201(g) of the FD&C Act; Articles intended for use in diagnosis, cure, mitigation, treatment, or prevention of disease; Product classification based on chemical action or specific intended uses like altering pH
Regulated under section 351(i) of the PHS Act; Virus, therapeutic serum, toxin, vaccine, or protein applicable to prevention or treatment; Alternative regulation category for products meeting device definition
Defined in section 201(h) of the FD&C Act; Instrument or apparatus which does not achieve primary purpose through chemical action; Product classification based on primary intended purpose and chemical action
A type of ATMP involving recombinant nucleic acids or viral vectors.
Stakeholders
2Entity responsible for submitting applications under section 524B
Source of safety information in clinical trials
Regulatory Context
Regulatory Activities
6The overall process of developing medical products for neonates
Alternate strategy for data collection
Alternate strategy if long-term follow-up studies are not feasible
Legislation made permanent under FDASIA
Legislation made permanent under FDASIA
FDASIA Title V made BPCA and PREA permanent
Document Types
1Defines the standard of veterinary practice and limits for anesthetic regimens
Attributes
4Factor related to neurodevelopmental vulnerability
Minimum duration for evaluating neurodevelopmental safety outcomes
Chronological age reduced by the number of weeks born before 40 weeks of gestation
Information to be recorded in the Comments element in case of prematurity.
Technical Details
Substances
2Excipient to be considered for toxicity
Excipient to be considered for toxicity
Testing Methods
8Neurophysiologic testing method
Adjunctive assessments used in safety evaluations
Adjunctive neurological assessment
Adjunctive neurological assessment; Neurophysiologic testing for safety signals
Used to quantify systemic exposure
Nonclinical toxicity testing in young animals
Tools used to measure neurologic function and daily life functioning
Neuroimaging study used to assess anatomical evidence of toxicity
Processes
1Assessment for devices contacting human tissues
Clinical Concepts
10Target population for long-term neurodevelopmental safety studies
Primary focus of the long-term clinical evaluations; Primary outcome being evaluated in neonatal product development
Sub-population within the neonatal period definition
Target population for long-term safety evaluations
Condition treated with high-dose corticosteroids
Condition unique to neonates
Condition unique to neonates
Comorbidity factor affecting neurodevelopmental outcomes
Comorbidity factor to be considered as a covariate
Physical health domain assessment
Identified Hazards
Hazards
1Potential risk from active ingredients or excipients
ICH References (3)
Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population
Nonclinical Safety Testing in Support of Development of Pediatric Pharmaceuticals.
Clinical Investigation of Medicinal Products in the Pediatric Population.
Related MFDS Guidelines
Korean regulatory guidelines covering similar topics