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Determining the Extent of Safety Data Collection Needed in Late Stage Premarket and Postapproval Clinical Investigations

FinalCenter for Drug Evaluation and Research Center for Biologics Evaluation and Research02/19/2016
risk-based monitoringSelective safety data collection

Description

This guidance is intended to help sponsors determine the amount and types of safety data to collect during late-stage premarket and postapproval clinical investigations, (e.g., phase 3 clinical trials, studies of new uses, long-term outcomes). This guidance discusses a selective approach to safety data collection during late-stage premarket development or during the postapproval stage based on what is already known about a drug’s safety profile. This guidance provides recommendations on when to consider selective safety data collection and how to do so to maintain a balance between eliminating the collection of data that will not be useful and collecting sufficient data to allow adequate characterization of the safety profile of a drug. In addition, this guidance provides information to sponsors about consulting with the relevant FDA review division or divisions to determine whether a selective approach to safety data collection would be appropriate (see section V of this guidance).

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

2
Gene Therapy

A type of ATMP involving recombinant nucleic acids or viral vectors.

Biological drug products

Includes therapeutic proteins and recombinant DNA products.; Encouraged to use enhanced modelling

Stakeholders

1
Sponsor

Entity responsible for submitting applications under section 524B

Regulatory Context

Regulatory Activities

8
inspection

Ensuring records are accessible for regulatory review

phase 3 trial

focus on phase 3 trial designs to support an indication

end-of-phase 2 meeting

milestone for submitting initial pediatric study plans

Biologics License Application

BLA for biological products

New Drug Application

Rule does not apply to products marketed under an NDA

IND

Investigational New Drug submissions

Postapproval clinical investigations

studies of new uses or long-term outcomes

Phase 3 clinical trials

Adult trials that should include adolescent participants.

Document Types

2
monitoring plan

The agreement should be incorporated into the procedures for safety data collection in the protocol, the monitoring plan

Protocol

Defines the standard of veterinary practice and limits for anesthetic regimens

Attributes

2
Grade 4 adverse event

Results in the need for life-saving and urgent intervention; should always be collected

Grade 3 adverse event

Results in severe or clinically significant consequences; should always be collected

Technical Details

Testing Methods

1
Routine laboratory monitoring

possible to eliminate or decrease the frequency of monitoring certain laboratory parameters

Processes

1
selective safety data collection

A sponsor considering selective safety data collection should consult with the relevant FDA review division

Clinical Concepts

5
Hepatotoxicity

Risk increased by concomitant use of hepatotoxic drugs with DRUG-X.

Non-serious adverse events

Types of safety data where it may be appropriate to limit or stop collection; collection may be discontinued in selective approach

Serious adverse events

Anticipated risks that must be outweighed by benefits

Rare diseases

Diseases with low prevalence in certain regions

Adverse events

Safety findings including deaths and post-mortem examinations

Standards & References

External Standards

1
Common Terminology Criteria for Adverse Events

Standard used for grading the severity of adverse events.

View on FDA.govDownload PDF

Related CFR Sections (3)

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)

Determining the Extent of Safety Data Collection Needed in Late Stage Premarket and Postapproval Clinical Investigations | Guideline Explorer | BioRegHub