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Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs

DraftOncology Center of Excellence09/16/2024

Description

This guidance provides recommendations to sponsors who are planning global clinical development programs for drugs intended to treat cancer, on improving the evidence obtained from one or more multiregional clinical trials (MRCTs) intended to support a marketing application. This guidance expands on principles described in FDA’s existing guidance documents related to this topic by providing additional recommendations for the planning, design, conduct, and analysis of an oncology MRCT that may facilitate FDA’s assessment of applicability of the data to the U.S. population with the cancer being investigated and to U.S. medical practice.

Scope & Applicability

Product Classes

4
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

Oncologic drug

Drugs intended to treat cancer in multiregional trials

Oncology drug

Sponsors who plan to conduct any aspect of the CDP for an oncology drug outside the U.S.

Stakeholders

2
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 Context

Attributes

5
U.S. standard of care

the control arm of the MRCT should be sufficiently large to permit a robust evaluation... compared to the U.S. standard of care

Treatment effect consistency

Assessment of consistency between U.S. participants and overall population

Demographic representativeness

Need for guidance to address demographic representativeness in MRCTs

Intrinsic and extrinsic factors

Factors impacting study outcomes and applicability to U.S. patients

Genetic ancestry

Information about biological descent relevant to U.S. population diversity

Related CFR Sections (4)

Related Warning Letters (1)

  • Sponsor

    Applied Therapeutics, Inc.

    2024-12-03

See Also (8)

Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs | Guideline Explorer | BioRegHub