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E8(R1) General Considerations for Clinical Studies

FinalCenter for Drug Evaluation and Research04/08/2022
Quality by DesignInformed ConsentGood Clinical PracticeData IntegrityRisk Management

Description

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) has the mission of achieving greater regulatory harmonization worldwide to ensure that safe, effective, and high-quality medicines are developed, registered, and maintained in the most resource-efficient manner. By harmonizing the regulatory expectations in regions around the world, ICH guidelines have substantially reduced duplicative clinical studies, prevented unnecessary animal studies, standardized safety reporting and marketing application submissions, and contributed to many other improvements in the quality of global drug development and manufacturing and the products available to patients.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

4
Investigational Medicinal Product

Quality of investigational medicinal product in development planning; Ensuring adequate quality and characterization

Drug

Defined 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

Biological products

development program for drug and biological products

Companion Diagnostic

FDA-authorized tests required for certain drug indications

Stakeholders

8
Clinical Study Participants

Protection of participants in clinical study design

Pregnant people

Vulnerable population in clinical studies

Geriatric patients

Subpopulation with potentially different metabolism

Pediatric populations

Special population requiring specific investigation

Data Monitoring Committee

Safety oversight body for interpreting adverse events; Safety oversight body for study design

Sponsor

Entity responsible for submitting applications under section 524B

Investigator

Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining

Regulatory Authorities

Early discussion is recommended regarding safety dataset size.; Entities with whom the adequacy of RWD and extrapolation plans should be discussed.; early discussion useful when proposed endpoints differ; Entities that should agree upon success criteria in advance.

Regulatory Context

Regulatory Activities

3
Marketing Application

Submission for product approval

Marketing Approval

Confirmatory studies intended to provide an adequate basis for approval

Marketing authorization

Submission requiring RVLP quantification data; Requires data from at least three lots of purified bulk

Document Types

6
Protocol

Defines the standard of veterinary practice and limits for anesthetic regimens

Protocol Amendment

A document describing changes made to the original clinical trial protocol.

Informed Consent

Process by which a subject voluntarily confirms his or her willingness to participate in a particular trial; Integral feature of the ethical conduct of a trial

Statistical Analysis Plan

Document for protocol execution; The sponsor should develop a statistical analysis plan that is consistent with the trial protocol; Deviations from this plan must be justified

Study protocol

Should include biological rationale for subgroups

Case Report Forms

Used to delineate dose and timing of administration

Attributes

3
Critical-to-Quality Factors

Factors essential to the study and operational practice; Attributes fundamental to the protection of participants and reliability of results; identify the factors that are critical to the study; address issues related to critical-to-quality factors observed during the course of the study

Primary Endpoint

Capable of providing clinically relevant evidence related to the primary objective

Bioavailability

Biological property that may be altered by manufacturing changes; safety narrative should address bioavailability of the ingredients

Technical Details

Testing Methods

8
Statistical Analysis

plan for evaluating equivalent or improved performance

Bioequivalence

studies available to validate the EDDO specifications

Interim Analysis

Whether an interim analysis is planned.; Rationale for any interim analysis planned with respect to its purpose and timing.; Analyses conducted while the trial is ongoing for stopping or adapting

Sensitivity Analyses

Statistical considerations in clinical trials

Blinding

Measure taken to minimize bias

Randomization

Measure taken to minimize bias

Bioequivalence Studies

additional bioequivalence studies depending on the dosage form

Modelling and Simulation

may inform drug development throughout the process; Used to support the design of later confirmatory studies

Processes

9
Nonclinical Studies

The results of all relevant nonclinical pharmacology, toxicology, pharmacokinetic, and investigational product metabolism studies should be provided.

Pharmacodynamics

Study of drug effects, which may be impacted by physiological changes in pregnancy.

Pharmacokinetics

Dosing considerations for pregnant women in trials; Study of drug movement in the body, which may change during pregnancy.

Human Pharmacology

Clinical study type in development planning; Types of clinical studies: Human Pharmacology

Clinical study design

Focus of the guidance to ensure reliable information

Randomization

Process of assigning trial participants to treatment or control groups using an element of chance; Process of assigning participants to treatment groups.; Process of assigning participants to groups using chance to reduce bias.

Blinding

Manufacturing and handling should maintain blinding

Drug Development Planning

general principles to consider in drug development planning

Feasibility Assessment

consideration of study design and implementation elements

Clinical Concepts

5
Adverse Reactions

List included in the RSI to determine expectedness.

Intercurrent Events

Events requiring handling strategies within the estimand rationale.; Events occurring after treatment initiation that affect the interpretation of the outcomes.

Adverse Events

Reporting adverse events when engaging with patients.; changes may be related to benefits, tolerability, and/or unintended effects

Pediatric Populations

clinical studies planned in pediatric populations

Special populations

Participants requiring specific protection in clinical investigations

Standards & References

External Standards

1
Declaration of Helsinki

Ethical principles that are the origin of GCP standards.; Ethical principles for medical research involving human subjects

ICH References (10)

ICH E8(R1)

General Considerations for Clinical Studies

ICH E2F

Development Safety Update Report guidance

ICH E2E

Pharmacovigilance Planning

ICH E2D

Provides guidance on identifiable patients and reporters; Provides definitions for seriousness criteria

ICH E2C(R2)

Periodic Benefit-Risk Evaluation Report (PBRER) standard for marketed products; Periodic Benefit-Risk Evaluation Report guidance; The primary guidance document for Periodic Benefit-Risk Evaluation Reports.; Periodic Benefit-Risk Evaluation Report (PBRER) guidance

ICH E2B(R3)

Implementation Guide for Electronic Transmission of Individual Case Safety Reports; Electronic Transmission of Individual Case Safety Reports Implementation Guide; Implementation Guide for electronic transmission of Individual Case Safety Reports; fully structured format using the relevant E2B(R3) data elements; Details of ICH E2B(R3) Data Elements and message specifications.; Implementation guide for electronic transmission of ICSRs; Implementation Guide for electronic transmission of ICSRs; Im

ICH E2A

Provides definitions for seriousness criteria; ICH E2A recommends blinded therapy should not be reported

ICH E3

document remedial actions in the clinical trial report; Structure and Content of Clinical Study Reports standards.

ICH E10

Choice of Control Group in Clinical Trials.

ICH E9(R1)

Statistical Principles for Clinical Trials: Addendum: Estimands and Sensitivity Analysis in Clinical Trials

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (8)