Master protocols in immuno-oncology: do novel drugs deserve novel designs?

Area of Science:

• Oncology
• Clinical Trials
• Biomarkers

Background:

• Immuno-oncology (IO) drugs have significantly impacted cancer treatment.
• The development of IO agents increasingly utilizes nonconventional clinical trial designs.
• Master protocols with adaptive features and biomarker-driven patient selection are prominent.

Purpose of the Study:

• To review characteristics of recent and ongoing trials testing IO drugs with unconventional designs.
• To highlight trends and critical aspects of these novel trial methodologies.
• To discuss the implications of these designs for evidence generation and regulatory acceptance.

Main Methods:

• Review of recent and ongoing clinical trials involving immuno-oncology drugs.
• Analysis of trial designs, focusing on master protocols and adaptive features.
• Examination of the role of biomarkers in patient selection for IO therapies.

Main Results:

• IO drugs are increasingly developed using master protocols and adaptive trial designs.
• Biomarkers play a crucial role in identifying patient populations likely to benefit from IO therapies.
• These novel designs aim to maximize clinical benefit but present challenges for widespread adoption.

Conclusions:

• Unconventional trial designs, particularly master protocols, are a growing trend in IO drug development.
• Successful integration of these designs requires significant cultural shifts among stakeholders.
• Further evaluation is needed to establish these novel designs as a robust evidence base for clinical practice.