How many tumor indications should be initially screened in development of next generation immunotherapies?

Area of Science:

• Oncology
• Immunotherapy
• Clinical Trial Design

Background:

• Experimental oncology immunotherapies show potential across numerous tumor indications.
• A staggered approach for efficacy screening is proposed for indications with unmet medical needs.
• The effectiveness of novel immunotherapies is uncertain during the planning phase.

Purpose of the Study:

• To determine the optimal number of tumor indications for the first wave of investigation to maximize cost-effectiveness.
• To balance the expected benefits (correctly identified effective indications) against the expected costs (total sample size).

Main Methods:

• A benefit-cost ratio was defined as expected effective indications identified divided by expected total sample size.
• Analysis focused on a two-wave staggered screening approach for immunotherapy development.
• The model accounts for uncertainties in treatment effectiveness at the planning stage.

Main Results:

• Benefit-cost ratio analyses suggest initiating three to five tumor indications in the first wave.
• The optimal number depends on resource availability and the need to balance risk and benefit.
• Adequate investment is crucial for maintaining high-quality statistical design.

Conclusions:

• A phased approach with 3-5 initial tumor indications can optimize immunotherapy development.
• Careful planning and resource allocation are essential for cost-effective efficacy screening.
• Statistical design quality is paramount for reliable results in early-phase immunotherapy trials.