Cancer Clinical Trial Eligibility Criteria: Minimum Age Considerations for Inclusion of Pediatric Patients

Description

This guidance is one in a series of guidances that provide recommendations regarding eligibility criteria for clinical trials of drugs or biological products regulated by CDER and CBER for the treatment of cancer. Specifically, this guidance includes recommendations regarding the inclusion of pediatric patients (i.e., children and adolescents) when appropriate. This guidance is intended to assist stakeholders, including sponsors and institutional review boards (IRBs), responsible for the development and oversight of clinical trials.

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

2

drugs

Includes human drug products and biological products

biological products

Regulated by CDER and CBER

Stakeholders

3

IRB

Institutional Review Board providing study approvals

Regulatory Context

Regulatory Activities

2

First in human studies

Initial clinical trials intended for adults

clinical trials

monitoring growth and pubertal development in clinical trials

Document Types

2

Pediatric formulations

Evaluation of formulations taking into account age and size

labeling

Document where unexpected behaviors might be listed as possible.

Attributes

4

Body weight

relationship between body weight and drug exposure

Body surface area

covariate associated with sex affecting device performance

minimum age

Criteria for inclusion of pediatric patients

eligibility criteria

Defining characteristics of the study population

Technical Details

Testing Methods

3

Pharmacodynamics

assessment of PD in animals and clinical testing

Pharmacokinetics

Proof-of-concept studies may evaluate tolerability, pharmacokinetics, and preliminary clinical efficacy.

Modeling and simulation

Approaches used to select pediatric doses based on exposure matching.

Clinical Concepts

3

Adolescents

Pediatric population that may be included in adult clinical trials.; inclusion in adult clinical trials recommended when disease is similar

cancer

The disease setting for which the clinical trials are conducted

pediatric patients

Target patient population

Standards & References

Specifications

1

Recommended phase 2 dose

Dose selection for pediatric cohorts

Related CFR Sections (2)

21CFR50.52§ 50.52 Clinical investigations involving greater than minimal risk but presenting the prospect of direct benefit to individual subjects.
Any clinical investigation within the scope described in §§ 50.1 and 56.101 of this chapter in which more than minimal risk to children is presented by an intervention or procedure that holds out the prospect of direct benefit for the individual subject, or by a monitoring procedure that is likely tRead full regulation →
21CFR56.111§ 56.111 Criteria for IRB approval of research.
(a) In order to approve research covered by these regulations the IRB shall determine that all of the following requirements are satisfied:Read full regulation →

Enforcement Impact

Deficiencies cited in Warning Letters referencing the same regulations

Failure to obtain informed consent

5

Failure to ensure that the investigation was conducted according to the investigational plan

3

Failure to submit an Investigational New Drug application (IND)

2

Failure to prepare, maintain, and follow written procedures governing the functions and operations of the IRB

1

Failure to ensure proper monitoring of the investigation

1

Failure to secure clinical investigator's compliance

1

Failure to submit an IDE application and obtain FDA approval

1

Failure to immediately conduct an evaluation of unanticipated adverse device effects

1

Failure to review proposed research at convened meetings with a majority of members present

1

Failure to prepare and maintain adequate documentation of IRB activities, including minutes of IRB meetings