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Considerations for Developmental Toxicity Studies for Preventive and Therapeutic Vaccines for Infectious Disease Indications: Guidance for Industry

FinalCenter for Biologics Evaluation and Research02/01/2006
Developmental ToxicityGood Laboratory PracticecGMP

Description

This guidance is intended to provide you, sponsors, with recommendations for the conduct of developmental toxicity2studies for investigational preventive and therapeutic vaccines for infectious disease indications. The recommendations set forth in this document pertain to the assessment of the developmental toxicity potential of preventive and therapeutic vaccines for infectious diseases indicated for females of childbearing potential and pregnant individuals.3This guidance applies prospectively to investigational vaccines, i.e., vaccines under investigational new drug applications (IND) and vaccines the subject of a new biologics licenseapplication (BLA). These recommendations do not apply retrospectively to already licensed vaccines except those the subject of additional INDs. This guidance document finalizes the draft guidance entitled “Guidance for Industry: Considerations for Reproductive Toxicity Studies for Preventive Vaccines for Infectious Disease Indications” dated August 2000 (65 FR 54534; September 8, 2000).

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

4
Preventive Vaccine

animal models used to demonstrate prevention of disease

Therapeutic Vaccine

Vaccines intended for the treatment of infectious diseases

Combination Product

Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.

Preventive and Therapeutic Vaccines

Primary focus of the guidance for infectious disease indications; Subject of the developmental toxicity study guidance

Stakeholders

5
Sponsor

Entity responsible for submitting applications under section 524B

F1 generation

Offspring evaluated for normal growth and development

maternal animals

Animal models used in the toxicity study

Females of childbearing potential

Target population for vaccines who may become pregnant

Pregnant Women

Scientific and ethical considerations for inclusion in clinical trials

Regulatory Context

Regulatory Activities

2
IND

Investigational New Drug submissions

BLA

Biologics License Application

Attributes

5
Risk versus benefit

Assessment required for immunization programs

Immune Response

Distinguishing feature of vaccines compared to other pharmaceuticals

fertility index

Endpoint for natural delivery group

gestation index

Endpoint for natural delivery group

live birth index

Endpoint for natural delivery group

Technical Details

Substances

8
cord blood

Samples used to assess placental transfer of maternal antibodies

Adjuvant

CQAs should be evaluated during vaccine stability studies

Preservative

Exception excipient for parenteral, ophthalmic, or otic use.

Stabilizer

Constituent of the vaccine product

Plasmid DNA vaccine

Type of vaccine derived from polynucleotides

vaccine antigen

Component of the vaccine formulation

excipients

Assessment of excipient differences

preservatives

Example of a permissible inactive ingredient

Testing Methods

6
Caesarean Examination

Routine uterine and fetal examinations at the end of pregnancy

Pilot Studies

Used to obtain information about onset and duration of antibody response

Caesarean sectioning

Method for terminal examination of maternal animals and fetuses

necropsy

necropsy results including gross pathology

histological evaluations

Microscopic examination of tissues with macroscopic findings

auditory and visual function tests

Tests to screen for normal neuro-development in F1 generation

Processes

2
Maternal Immunization

Products specifically intended for use in pregnant women

Developmental Toxicity Studies

Non-clinical studies to evaluate effects on pregnancy and offspring

Clinical Concepts

5
Infectious Disease

Indications for which the vaccines are developed

Pregnancy

Example of a condition to be captured in the medical history section.

Developmental Toxicity

detecting malformed offspring or reduced birth weight

Embryo/Fetal Development

Evaluation of risks to the fetus during animal studies

organogenesis

Critical phase of pregnancy from implantation to closure of the hard palate

Identified Hazards

Hazards

4
Developmental adverse effects

Potential risks caused by vaccine biological activity

embryotoxic effects

Potential direct effects of vaccine components on the embryo

Maternal immune modulation

Potential cause of adverse effects on pregnancy status

Adverse Developmental Effects

Potential risks to offspring screened in animal models

Standards & References

Specifications

1
antibody response

Assessment to verify exposure of the embryo/fetus to maternal antibody

ICH References (1)

ICH S5A

Detection of Toxicity to Reproduction for Medicinal Products

Related CFR Sections (2)

Enforcement Impact

Deficiencies cited in Warning Letters referencing the same regulations

Failure of the study director to assure protocol compliance and accurate data recording
1
Failure of the study director to assure experimental data are accurately recorded and verified
1
Failure to properly identify specimens
1
Failure to assure experimental data were accurately recorded and verified
1
Failure to authorize and document deviations from standard operating procedures
1
QAU failed to assure reported results accurately reflect raw data
1
Failure to maintain a Quality Assurance Unit
1
Study director failed to assure protocol compliance and data accuracy
1
Fabrication of animal weight data
1
Failure to establish procedures for handling test and control articles
1

Related Warning Letters (9)

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)