Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry

FEBRUARY 27th, 2019FDA Modernizes Clinical Trials with Master Protocols

The innovative regulatory approaches resulting from the 21st Century Cures Act are modernizing new drug development. The use of clinical trials with Master Protocol design is one example of a modern approach to expedite the development of oncology drugs and biologics. Because of the complexity of these trials and the potential regulatory impact, it is important that such trials are well designed and well conducted to ensure patient safety and to obtain quality data that may support drug approval.

Traditionally, oncology drug development involves a series of clinical trials studying one or two drugs in a single disease. Other clinical trials are intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure in adult and pediatric cancers. The Draft Guidance for Industry – Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Cancer Drugs and Biologics provides recommendations on the design and conduct of the latter type of clinical trials.

Master protocols may incorporate specific design features that require special considerations. Examples of types of master protocol design include trials commonly referred to as basket trials and umbrella trials:

A Basket Trial involves a single investigational drug or drug combination that is studied across multiple cancer populations defined by disease stage, histology, number of prior therapies, genetic or other biomarkers, or demographic characteristics. It is usually designed as a single-arm, activity-estimating trial with overall response rate as the primary endpoint. A strong response signal seen in a sub-study may allow for expansion to generate data that could potentially support a marketing approval.