Translational clinical trials: an entropy-based approach to sample size

Area of Science:

• Biomedical research
• Clinical pharmacology
• Experimental therapeutics

Background:

• Translational clinical trials are small, early-stage studies of novel therapies.
• These trials are critical for assessing treatment effects on disease targets and informing future research.
• The unique statistical properties of translational trials are often overlooked within traditional clinical trial frameworks.

Purpose of the Study:

• To discuss the translational clinical trial setting.
• To present an information (entropy)-based approach for understanding translational trial properties and applications.
• To propose a method for motivating sample size in translational trials.

Main Methods:

• Review of the translational clinical trial setting.
• Application of an information (entropy)-based framework.
• Development of a sample size motivation approach.

Main Results:

• Identification of translational trials as crucial for early therapeutic evaluation.
• Presentation of an entropy-based method to analyze translational trial characteristics.
• Proposal of a novel approach for determining sample size in early-phase trials.

Conclusions:

• Translational trials, combining biological insights with experimental design, effectively reduce uncertainty for emerging therapies.
• An information-based approach offers valuable insights into the statistical properties and utility of translational trials.
• A structured method for sample size determination is presented to enhance the design and interpretation of these vital studies.