FDA Guidance on Efficient Clinical Trial Design Strategies for Cancer Drugs and Biologics

Recommendation

7/8 May 2024

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Regulatory Requirements and Practical Implementation

The ongoing development of new biologics and drugs for the treatment of cancer is accompanied by an increasing use of clinical trials to evaluate more than one investigational drug within the same overall trial structure or to evaluate more than one cancer type in one trial, a so-called Master Protocol. It is defined as follows: "A protocol designed with multiple substudies, which may have different objectives and involves coordinated efforts to evaluate one or more investigational drugs in one or more disease subtypes within the overall trial structure."
Therefore, the FDA published the new Guidance for Industry "Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics" to support sponsors of trial designs testing multiple drugs and/or multiple cancer subpopulations in parallel under a single protocol. It is generally intended for drugs for which the recommended  phase 2 dose (RP2D) has been established. In summary, the new document will cover  "aspects of master protocol designs and trial conduct and related considerations, such as biomarker codevelopment and statistical analysis considerations, and provides advice on the information that sponsors should submit to FDA and on how sponsors can interact with FDA to facilitate efficient review."

Please be aware that this new guidance provides no support on clinical trial design, statistical analysis, or the biomarker development process which will be covered in other relevant guidance documents such as the ICH guidances for industry "E9 Statistical Principles for Clinical Trials" and E10 "Choice of Control Group and Related Issues in Clinical Trials" and the guidance for industry and FDA staff In Vitro Companion Diagnostic Devices.