Recent advances in clinical trial design considerations in Thera"nostics"

Area of Science:

• Pharmacology
• Radiology
• Clinical Trials

Background:

• Theranostics combines therapeutic and diagnostic drugs, with diagnostics aiding patient selection for investigational therapeutics.
• The patient selection performance of investigational diagnostic imaging drugs is often not rigorously assessed.
• Current clinical trials may prioritize therapeutic development over validating diagnostic imaging drug performance.

Purpose of the Study:

• To propose an "In-Parallel with Leveraging" development pathway for diagnostic imaging drugs in theranostics.
• To define minimum statistical metrics for rigorous diagnostic imaging drug development and patient selection.
• To explore broader clinical utilities of diagnostic imaging beyond patient selection.

Main Methods:

• Development of a novel "In-Parallel with Leveraging" pathway for diagnostic imaging drugs.
• Rationalization of essential statistical metrics for patient selection performance assessment.
• Identification of potential clinical indications and utilities for diagnostic imaging.

Main Results:

• The proposed pathway enables rigorous development of diagnostic imaging drugs alongside therapeutics.
• Minimum statistical metrics are identified to ensure robust patient selection validation.
• Broader applications including prognostic, predictive, risk stratification, and treatment monitoring are highlighted.

Conclusions:

• The proposed pathway enhances the development of diagnostic imaging drugs within theranostics.
• Leveraging group sequential or adaptive designs in therapeutic trials is encouraged for optimal benefit.
• This approach supports comprehensive clinical utility and improved patient management.