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Format and Content of the Nonclinical Pharmacology/Toxicology Section of an Application*: Guidance for Industry

FinalCenter for Drug Evaluation and Research02/01/1987
GLP statementsPharmacokinetics

Description

This guideline describes an acceptable format for organizing and presenting the pharmacology and toxicology data required under 21 CF 314.50(d)(2), and any related date, in the nonclinical section of the application. These recommendations pertain only to organization of existing data. This guideline is not intended to affect documentation required by the Good Laboratory Practices (GLP) reporting regulations under 21 CFR 58.185, nor does it describe the specific stufy requirements for particular therapeutic uses or regimens: these are addressed in other guidelines.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Stakeholders

4
Sponsor

Entity responsible for submitting applications under section 524B

U.S. GOVERNMENT PRINTING OFFICE

Entity responsible for printing the document

Laboratory

Entity establishing competence in the use of the assay

Contact Person

The name, title, address, and telephone number of the person FDA may contact

Regulatory Context

Regulatory Activities

2
IND

Investigational New Drug submissions

NDA

New Drug Application

Document Types

10
Pharmacology and Toxicology Studies

General principles apply to the submission of all pharmacology and toxicology studies

Multidose Toxicity/Carcinogenicity Studies

Summary table format for toxicity and carcinogenicity data

ADME Studies

Absorption, Distribution, Metabolism, Excretion studies organization

Mutagenicity Studies

Results of available studies should be tabulated

Segment I study

paternal effects in Segment I study

Pathology table

The pathology table should identify organs, tissues, and lesions.; Table 3 showing number of tumor-bearing animals

Table of Contents

Submission contains a Table of Contents

Pharmacology Studies

Studies related to therapeutic indication and adverse reactions

Acute Toxicity Studies

Studies describing toxic signs and lethal dose data

Subchronic/Chronic/Carcinogenicity Studies

Long-term studies grouped by species and duration

Attributes

10
body weight

Drug doses should be expressed on a body weight basis

Positive Controls

Control group expected to show response (n=50)

Untreated Controls

Control group in the current study (n=50)

Historical Controls

Reference data from previous studies (n=500)

Prevalence rate

Report the prevalence rate of nonfatal tumors.

Incidence rate

Single cohort studies can measure incidence

Malignancy

An assessment of the malignancy of the tumor.; Column in carcinogenicity study data chronology listing

Lethal dose data

Approximate or calculated median or limit doses

ED₅₀

Pharmacodynamic median effective dose

body surface basis

reason to express doses in some other way

Technical Details

Substances

5
Metabolites

Tested if they occur as residues in food and have structural alerts

Administration vehicle

Substance used to deliver the drug

Active Moiety

Basis for determining drug sameness

drug substance

Active ingredient in the CGT product

radioactive drug

Studies with a radioactive drug should indicate the molecular location

Testing Methods

10
Blood chemistry

Chemical analysis of blood samples

In vivo mammalian system

Order of presentation for mutagenicity studies

In vitro non-mammalian cell system

Order of presentation for mutagenicity studies

In vitro hemolysis

Used to evaluate compatibility with blood for intravenous products.

Topical irritation studies

Special toxicity studies include topical irritation studies.

Parenteral irritation studies

Special toxicity studies include parenteral irritation studies.

Necropsy

Procedure for D. immitis worm counts

statistical methods

Used for analyzing study data

ECG

Monitoring required when concomitant use is unavoidable.

Hematology

Clinical data required for safety evaluation

Processes

10
Necropsy

Procedure where worms are identified and counted for effectiveness calculations.

Perinatal-Postnatal

Segment III of reproduction studies.

Teratology

Segment II of reproduction studies.

Fertility and Reproductive Performance

Segment I of reproduction studies.

Special Toxicity Studies

Include studies appropriate to a particular formulation or route of administration.

Gavage

Method of oral administration in carcinogenicity studies; oral administration method in Lijinsky study; method of administration in NTP studies for p-chloroaniline; administration in the feed to rats and mice or by gavage; Route of administration in short-term carcinogenicity models; oral route of administration for carcinogenicity studies; Oral gavage study of Wester et al.; Method of administration in carcinogenicity study; Method of administration in NTP studies; Hydrazine sulfate was adminis

pathologic diagnosis procedures

study reports should include description of pathologic diagnosis procedures

Pharmacology Studies

Overview of nonclinical assessments including Pharmacology Studies

Acute Toxicity Studies

Nonclinical safety assessment

Multidose Toxicity Studies

Subchronic, Chronic, Carcinogenicity

Clinical Concepts

10
Toxic Signs

Data base should document relevant observations for each animal including toxic signs

Liver

Organ evaluated for microscopic findings

fibroadenoma

Specific tumor type observed in the liver

adenoma

Specific tumor type observed in the liver

Tumor-bearing Animals

Reporting the number of animals with tumors in study groups

Tumor Type

Data element for carcinogenicity study reporting

Skeletal abnormalities

Observations recorded for fetuses grouped by litter

Visceral abnormalities

Observations recorded for fetuses grouped by litter

Tumor

P is the proportion of animals with the specified tumor type observed at a certain dose.

Neoplastic pathology

Maintain consistency of order of presentation of non-neoplastic and neoplastic pathology.

Standards & References

External Standards

1
Toxicology and Applied Pharmacology

Standard abbreviations acceptable to a refereed U.S. pharmacology/toxicology journal

Specifications

1
p = .05 level

For each tumor found to be statistically significant at the p = .05 level.

Related CFR Sections (1)

  • 21CFR314.50§ 314.50 Content and format of an NDA.

    NDAs and supplements to approved NDAs are required to be submitted in the form and contain the information, as appropriate for the particular submission, required under this section. Three copies of the NDA are required: An archival copy, a review copy, and a field copy. An NDA for a new chemical enRead full regulation →

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)

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