Using human experience to identify drug repurposing opportunities: theory and practice

Area of Science:

• Pharmacology
• Oncology
• Drug Discovery

Background:

• Drug repurposing (DRP) identifies new uses for existing medications.
• Real-world evidence (RWE) from actual patient use offers a robust DRP approach.
• Traditional DRP methods like preclinical studies or in silico associations have limitations.

Purpose of the Study:

• To highlight the significance of real-world evidence in drug repurposing.
• To emphasize the potential of DRP in oncology and complex diseases.
• To discuss strategies for optimizing repurposed drugs.

Main Methods:

• Utilizing retrospective real-world data from medicine usage.
• Analyzing large-scale human experience incidence data.
• Combining DRP with pathway analysis and prospective preclinical studies.

Main Results:

• Real-world evidence represents the most validated form of drug repurposing.
• Cancer is a primary area for successful drug repurposing examples.
• DRP strategies can yield first-in-class treatments and optimized drug candidates.

Conclusions:

• Real-world evidence provides a powerful and validated approach to drug repurposing.
• Drug repurposing holds significant promise for developing new cancer therapies and treatments for complex diseases.
• Integration with other research methods enhances the discovery and optimization of repurposed drugs.