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Nonclinical Safety Evaluation of Drug or Biologic Combinations

FinalCenter for Drug Evaluation and Research03/15/2006
Nonclinical Safety EvaluationSafety PharmacologyMargins of Safety

Description

The Food and Drug Administration (FDA) is announcing the availability of a guidance for industry entitled ``Nonclinical Safety Evaluation of Drug or Biologic Combinations.'' This guidance provides recommendations on nonclinical approaches to support the clinical study and approval of fixed-dose combination products (FDCs), co-packaged products, and some adjunctive therapies.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

8
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.

Biologics

Products for which batch/lot information is particularly important

NME

New Molecular Entities requiring assessment

Fixed-dose combination products

Two or more separate drug components combined in a single dosage form

Co-packaged products

Products involving drug and device components like injector and vial

Adjunctive therapies

Second drug product used together with primary treatment

New Molecular Entity

A situation requiring additional pediatric safety data collection.

Biologic

Product of biological origin applicable to prevention or treatment of disease.

Stakeholders

2
Sponsor

Entity responsible for submitting applications under section 524B

Women of childbearing potential

Inclusion in clinical trials supported by pEFD data

Regulatory Context

Regulatory Activities

2
Investigational new drug application

IND phase of drug development

Clinical study

Proceed with doses derived from toxicology studies

Document Types

1
Product labels

Clinical safety and efficacy described in labels

Attributes

4
Pregnancy Category D

PLLR requires the removal of pregnancy categories

Labeling Category X

Significant risk for developmental toxicity

Labeling Category D

Significant risk for developmental toxicity

Pregnancy Category X

PLLR requires the removal of pregnancy categories

Technical Details

Substances

4
New molecular entities

Combinations involving one or more NMEs

NSAIDs

nonsteroidal anti-inflammatory drugs that may be more toxic in animals than humans; Example of drugs more toxic in animals than humans

ACE inhibitors

Pharmacologic class for heart failure; Class of drugs with well-established safety profiles

Antibiotics

Concomitant medication used to manage bacterial MVGT risks.

Testing Methods

8
Animal Models of Efficacy

Used to support combinations of drugs or biologics

PK clinical study

Pharmacokinetic study conducted if metabolic interaction is identified

In vitro metabolism studies

determine if a new GVHD drug is a substrate, inhibitor, or inducer

Bridging Study

general toxicity bridging study of 3 months' duration for a chronic indication

Embryo-fetal Development Studies

conducted per the timing described in ICH M3

Genotoxicity Studies

Section VII of the guidance.

Carcinogenicity Study

Carcinogenicity studies are relevant to the safety assessment of FCSs and their constituents.

In vitro drug metabolism studies

Conducted early in drug development to assess interactions

Processes

10
General Toxicology Studies

Standard nonclinical safety evaluation

Embryo-fetal developmental study

Conducted on the combination product

Toxicology study

Conducted for up to 90 days on the combination

General toxicology bridging study

Study of up to 90 days on the combination

Carcinogenicity studies

Nonclinical in vivo studies to be summarized in the DSUR.

Reproductive and Developmental Toxicology

Toxicology studies for combinations involving NMEs; Evaluation of embryo-fetal development and fertility

Safety Pharmacology

assessment of the potential effect of the pharmaceutical on vital organ functions

PK/ADME

Pharmacokinetics and metabolism studies; Studies to assess potential for PK interaction

Toxicokinetics

General considerations in the assessment process; Evaluation of Cmax and AUC in relation to dose; Substantial differences in PK were noted; PK data available in various rat strains; Assessment of drug exposure in animal models; Assessment of systemic exposure in toxicity studies; Extrapolation of Cmax and AUC values from animal data.; PK data are also available on GD8

Genetic Toxicology

Toxicology studies for NME combinations; Generally not needed for biologic or drug/biologic combinations

Clinical Concepts

4
Human immunodeficiency virus

Infectious disease risk factor discussed in relation to donor eligibility.

Preneoplastic Lesions

observed at a new organ or tissue site

QT prolongation

Potential safety concern/adverse effect of some antiemetic drugs.

Renal impairment

Patient condition increasing risk of aluminum toxicity.; Patient population sensitive to aluminum exposure

Identified Hazards

Hazards

3
Toxicologic Interaction

target organs for toxicity are similar for each drug/biologic

Metabolic interaction

A safety concern that triggers specific testing pathways

Chemical Interaction

one drug may oxidize, methylate, or ethylate the other

Standards & References

Specifications

2
No-effect Doses

toxicologic interaction may result in lowering of previously determined no-effect doses

Margin of Safety

concern if drugs have a narrow margin of safety

ICH References (3)

ICH

International Council for Harmonisation cited for scientific standards

ICH S6

Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals

ICH M3

Nonclinical Safety Studies for the Conduct of Human Clinical Trials

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)

Nonclinical Safety Evaluation of Drug or Biologic Combinations | Guideline Explorer | BioRegHub